Year after year, federal spending on poverty programs has been going up, but we still see more and more people who have no margin to guard against unexpected expenses or job loss. At the same time, for different reasons, Americans who are not impoverished have seen their wealth decline sharply. Expert Ron Haskins, co-director of the Center on Children and Families, says the problems are growing deeper, despite increased federal spending on programs to assist the poor. Haskins says everyone must sacrifice, but also says, that people in general, who finish high school, get a job, and get married and delay having children until age 21 are better off.

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• Solutions to Poverty's Rise in America

The increasing diversification of news media—from online versions of major newspapers to political bloggers, to 24-hour cable news to social media—plus the profession’s changing economics have caused the balance of power between political reporters and presidential candidates to change. Stephen Hess, senior fellow emeritus, says our very good, well-trained reporters are “almost dangerous” to presidential candidates who are trying to stay on message. Thus, says Hess, the way the press covers campaigns has changed as well, and not for the better.

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• Stephen Hess: The Changing Balance of Power in Presidential Campaign Reporting

Senior Fellows Fiona Hill and Clifford G. Gaddy discuss their book, Mr. Putin: Operative in the Kremlin in a five part podcast series.

In the book, Hill and Gaddy write that Russian President Vladmir Putin’s style of rule is influenced by his identities as a Statist, a Man of History, a Free Marketeer, a Survivalist, an Outsider, and a Case Officer; these are distinct personalities, they note, that interact and affect policy decisions. On February 6, the Center on the United States and Europe at Brookings hosted an event for the launch of Mr. Putin with a discussion featuring the authors.

Video

• Fiona Hill: Putin’s Statist Personality: Restoring the Greatness of Russia
• Clifford Gaddy: Putin the History Man and Survivalist
• Fiona Hill and Cliff Gaddy: The Outsider Influenced Putin’s “Free Market” Personality
• Fiona Hill: Putin’s History in KGB Leads to “Case Officer” Personality
• Fiona Hill: Putin’s Personalities Leveraged to Boost Russia

Editors’ Note: The attack in Nice will inevitably, and appropriately, lead to more aggressive intelligence efforts against suspected ISIS networks and tighter security in general, writes Dan Byman. Unfortunately, they are also likely to bolster anti-Muslim voices in France and throughout Europe, and ISIS may in turn exploit Muslims’ resulting shame and anger. This post originally appeared on Slate.

At least 84 people are dead in Nice, France, after a truck deliberately plowed for over a mile through packed crowds celebrating Bastille Day; ISIS or one of its supporters is believed to be linked to the attack. As we reel in horror from yet another instance of terrorist carnage, news reports are trickling in, giving us a sense of the attack, its victims, and those behind the killings.

We’ll learn more in the days to come, but we already knew Europe—and France in particular—has a terrorism problem and that it may go from bad to worse in the near future. Ironically, one of the reasons for the increased attacks is the military successes the United States, France, and other countries and local fighters are scoring against ISIS’s core in Iraq and Syria and the setbacks suffered by ISIS’s key provinces such as that in Libya. ISIS, which had long trumpeted its success in establishing the now-shrinking caliphate, needs victories to compensate for these major losses. Earlier this month, CIA Director John Brennan warned, “As the pressure mounts on ISIL, we judge that it will intensify its global terror campaign to maintain its dominance of the global terrorism agenda.”

Running down people with a truck is a new form of violence for ISIS, though ISIS propaganda has called for supporters to kill by “ramming” people, al-Qaida’s Yemen branch recommended it in the past to “mow down the enemies of Allah,” and in 2008 a Palestinian used a bulldozer to kill three Israelis. But this is the fourth major terror attack in Europe in the past eight months: ISIS-directed terrorists killed 130 people in November in Paris; in March suicide bombers killed 32 people in Brussels, and just two weeks ago three men believed to be tied to ISIS killed more than 40 at the Istanbul airport.

The attacks will inevitably, and appropriately, lead to more aggressive intelligence efforts against suspected ISIS networks and tighter security in general. Unfortunately, they are also likely to bolster anti-Muslim voices in France and throughout Europe, fostering daily humiliations and furthering Muslim communities’ sense that they are suspect and under siege. ISIS will exploit the resulting shame and anger, increasing the risk of future attacks.

European nations face several types of terror threats from ISIS. The first is the unprecedented number of fighters—more than 5,000—that Europe has exported to Iraq and Syria to fight under the banners of ISIS and other jihadi groups. More than 900 of them (including 200 women) have come from France. More than 130 are dead, and almost 250 have returned; the rest are believed to still be in Iraq and Syria. The Paris attacks, which were coordinated and practiced, illustrate the danger such fighters can pose: Time in the battle zone allows them to gain fighting skills, become indoctrinated, and develop a network to exploit for future attacks. In addition, ISIS leaders can direct their operations to achieve maximal effect. As the so-called caliphate shrinks, more of these fighters may try to return. More than 1,000 other Frenchmen are believed to be radicalized but have not gone to Iraq and Syria, and this represents another overlapping danger. In the past many might have tried to go to Syria, but now, perhaps acting on directions from ISIS leaders with whom they are communicating or perhaps mixing with returned foreign fighters, these individuals might strike directly at France or other countries.

The last category are true “lone wolves”—those inspired by ISIS or other jihadi ideologies but with no direct operational connection to a group (the attacks in San Bernardino and Orlando appear to fall into this category). Two years ago ISIS propaganda emphasized coming to the “caliphate” to help it consolidate and expand. Earlier this year, however, its spokesman and external operations leader Muhammad al-Adnani declared, “The smallest action you do in the heart of their land is dearer to us than the largest action by us and more effective and more damaging to them.”

France, as we’ve seen, is particularly vulnerable. As my Brookings colleagues Will McCants and Chris Meserole have recently argued, French political culture is a counterterrorism problem. They found that “four of the five countries with the highest rates of radicalization in the world are Francophone, including the top two in Europe (France and Belgium).” Part of the problem are the large numbers of unemployed urban youth, a fertile field for radical recruiting. But part of it is also France’s aggressive secularization programs, which prohibit girls from wearing the veil in school and are considered by many Muslims to be a deliberate attack on their religion. Muslims’ trust in the government and security services is low. Add such a sense of humiliation to a surging far-right political movement that constantly blasts Muslim immigrants and citizens, and the conditions for radicalization are strong.

The United States is less vulnerable by comparison. Fewer than 300 Americans have gone to fight in Iraq and Syria. Part of this is because of an aggressive and effective law enforcement and intelligence effort to disrupt volunteers. But part of it is because the American Muslim community is far better integrated than its French counterpart and regularly cooperates with law enforcement. U.S. politics are becoming more poisonous toward Muslims, but—we can hope—that anti-Muslim sentiment in America may have peaked, and recent polls suggest positive attitudes toward Muslims are increasing.

Continued military and intelligence operations against the ISIS core by U.S.-led coalition forces and their local partners are necessary, but they will take time to bear fruit and in the end still don’t solve the terrorism problem. Stopping an attack like the one in Nice is exceptionally difficult. The attacker chose a “soft” (undefended) target and showed that one sick person can kill many with the right combination of determination and luck. Unfortunately, the most likely reaction after the Nice attack is also the worst one: more vitriol and hostility toward French and European Muslims, furthering a cycle that makes it harder for European security services to gain the cooperation of local communities and easier for ISIS to gain recruits and score victories.

Media coverage of the Islamic State frequently refers to the group’s violent and seemingly archaic justice system without considering the institutional structures that enable this violence, or the broader function that it serves in the group’s ambitious state-building project. Legal institutions make it easier for the group to capture and retain territory by legitimizing its claim to sovereignty, justifying the expropriation of the property and land of enemies, and building goodwill with civilians by ensuring accountability.

The Islamic State’s legal system purports to strictly apply the divinely revealed body of Islamic law known as Sharia, which it regards as the only legitimate basis for governance. Although its legal system is frequently characterized as medieval, it has instrumentally supplemented the original text of the Quran with the modern rules and regulations that are needed to govern a 21st century state and punish modern day offenses—for example, traffic violations. It has the same three features that are present in any modern legal system: police, courts, and prisons.

In a region that has long been plagued by corruption, the Islamic State has attempted to ingratiate itself with civilians by claiming that its legal system is comparatively more legitimate and effective than the available alternatives. However, two emerging vulnerabilities—the system’s susceptibility to corruption and propensity for extra-legal violence—are increasingly undermining the Islamic State’s ability to obtain the trust and cooperation of civilians. Counterinsurgency efforts should be designed to undermine the legitimacy of its institutions. Long-term solutions in the region must involve a fundamental reorganization of political and legal institutions in ways that promote legitimacy and rule of law.

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• The legal foundations of the Islamic State

In his wide-ranging interview with The New York Times, Republican presidential nominee Donald J. Trump stressed the importance of fighting ISIS while declaring “we are going to take care of this country first before we worry about everybody else in the world.” For counterterrorism (and I would argue for security in general), such thinking is dangerously wrong-headed: Fighting ISIS and stopping other foreign terrorist threats to the U.S. homeland requires close alliances and deep engagement abroad.

Allies may also have better access to a terrorist stronghold due to geography or historic ties. Saudi Arabia, Turkey, and Jordan are within easy bombing range of ISIS’ core: the U.S. homeland is not. Although military action against ISIS’ core in Iraq and Syria is vital, it is often quiet global intelligence cooperation that does much of the day-to-day counterterrorism lifting. Given the geographic span of ISIS operations, it is not realistic for the United States to have strong unilateral capabilities in every possible country where the terrorists might operate and use as a base for anti-U.S. operations. Foreign governments fill this gap, acting as a force multiplier for the United States. Shortly after 9/11, the United States was working with over 100 countries on counterterrorism; several years into the war on terror a senior CIA official testified that virtually every capture or killing of a suspected terrorist outside Iraq involved at least some help from a foreign intelligence service. 

Many allies have skilled intelligence services, and they also use their police and their domestic intelligence services to gather information. These services of course know local languages and are culturally aware. They can also take advantage of the law in their efforts to disrupt terrorism: Terrorism, after all, is a crime. Allies, particularly less savory ones, use a government’s coercive power as well. Although people automatically think torture, governments at times threaten to jail a relative of a suspected terrorist or withhold a business permit or the right to attend university. These seemingly mundane threats are something that the United States cannot do outside its own borders. 

Many of the most basic homeland security tasks do not begin or end with the homeland.

Given these advantages, the primary role of U.S. intelligence is to cajole and strengthen allies, not replace them. The United States might provide technical assistance, as many U.S. allies are far weaker in this area. In addition, U.S. intelligence often acts as a conductor of global liaison services. In 2010, al-Qaida of the Arabian Peninsula tried to bomb two cargo planes as they approached the United States. Efforts to disrupt the plot involved not only the United States and Yemen, but also the countries in transit, including Qatar, the United Arab Emirates, Germany, and the United Kingdom. And Saudi Arabia provided a key intelligence tip. 

Many of the most basic homeland security tasks do not begin or end with the homeland. Foreign governments provide information on terrorist suspects, and there is considerable cooperation on those who might travel to the United States. Much of the screening to hinder terrorist travel and operations is done “over there,” not in the United States.

Our world is too small, and terrorists too global, to think of U.S. security narrowly. If we want to fight ISIS and other foes, we need allies. That doesn’t mean we should do whatever our allies want or support them unconditionally. But we must recognize that if we expect them to help America fight its enemies, we must stand by them as well.

Media coverage of the Islamic State has focused on the group’s grotesque use of violence and archaic governance style. Less attention has been paid, however, to the institutions that make those practices possible—institutions that lend the group legitimacy, at least in the eyes of supporters, as a sovereign state. In her new Brookings Analysis Paper, “The legal foundations of the Islamic State,” Mara Revkin argues that legal institutions play a critical role in the Islamic State’s state-building project. Those structures help the group take and keep territory, as well as provide a measure of accountability to the people living under its rule.

Lesser evil?

Revkin writes that “the Islamic State has attempted to ingratiate itself with civilians by claiming that its legal system is comparatively more legitimate and effective than the available alternatives.” The Syrian and Iraqi governments, Revkin explains, are often perceived as being highly corrupt and ineffective. The Islamic State is able to gain civilians’ favor by arguing that its political and legal institutions are more legitimate than those of the Syrian and Iraqi governments or rival armed groups. She adds: “some Syrians and Iraqis seem to prefer the legal system of the Islamic State to the available alternatives not because they agree with its ideology, but simply because they regard it as the lesser evil.” 

The Syrian and Iraqi governments...are often perceived as being highly corrupt and ineffective.

Revkin writes that for the Islamic State, shariah law is “the only legitimate basis for governance.” In cases where shariah fails to address modern-day problems, she explains, religiously legitimate authorities appointed by the Islamic State—such as military commanders, police officers, and the caliph himself—can issue legal decisions as long as they do not conflict with the divine rules of shariah or harm the welfare of the greater Muslim community. Alongside this is a system of rules and regulations to “govern civilians, discipline its own officials and combatants, and control territory” in areas of rights and duties, behavior, property, trade, and warfare. 

Making the state possible

Legal institutions help the Islamic State advance three main state-building objectives, in Revkin’s view: 

1. First, they support the Islamic State’s territorial expansion by “legitimizing [its] claims to sovereignty, justifying the expropriation of the property and land of enemies, and building goodwill with civilians.” 
2. Legal institutions also allow the Islamic State to enforce compliance and accountability of its own members and maintain internal control and discipline. Revkin describes various types of punishments the Islamic State uses to discipline its own members—these punishments are important, she writes, because “no government can establish itself as legitimate and sovereign without policing the behavior of the people who are responsible for implementing its policies.”
3. Finally, Revkin explores the legal institutions surrounding the Islamic State’s tax policies, which are “critical to financing the Islamic State’s governance and military operations.” Courts and judges, she explains, are crucial to “administering and legitimizing” taxation and justifying “economic activities that might otherwise resemble theft.” 

Weaknesses in the system

Although the Islamic State claims to have legitimate governing authority, based on a defined legal system, that system faces vulnerabilities. Revkin writes, for instance, that reports of corruption and extra-legal violence are “threatening the organization’s long-term sustainability and undermining its ability to win the trust and cooperation of civilians.”

Amid recent signs that the group is losing strength, Revkin argues that it’s struggling to maintain its own moral standards. To further weaken the Islamic State, she recommends working to undermine those institutions. The trouble is, as Revkin points out: “the Islamic State came to power largely by exploiting the weakness and illegitimacy of existing institutions” in Iraq and Syria. Thus, a sustainable plan for ultimately destroying the organization must also involve strengthening political and legal institutions in those countries. 

Since mid-2014, the world’s attention has been transfixed on the aesthetically shocking actions of ISIS and the threat it poses to regional and international security. However, it is arguably Jabhat al-Nusra in Syria—and perhaps the al-Qaida movement more broadly—that looks more likely to survive over the long term and to threaten local, regional and international security interests. Since its emergence in Syria in late 2011, Jabhat al-Nusra has transformed itself from an unpopular outsider accused of Islamic State in Iraq (ISI)-like brutality towards one of the most powerful armed actors in the Syrian crisis. Moreover, its break away from the ISI in April 2013 set it further down a path of deep integration into the broader Syrian armed opposition in its fight against Bashar Assad’s regime.

Nearly five years after its formation, Jabhat al-Nusra has demonstrated the potential value of its "long game" approach. By adopting a strategy of gradualism, it has socialized populations into first accepting, and then supporting and defending, this al-Qaida-like movement. Jabhat al-Nusra aims to epitomize the realization of al-Qaida’s evolved thinking. It seeks to build localized bases of influence by embedding itself in within popular revolutionary dynamics and, eventually, establish zones of territorial control from which it can launch attacks against the Western world. By establishing a durable presence in Syria and potentially considering separating itself from al-Qaida internationally, Jabhat al-Nusra seeks to realize its long-term vision of establishing Islamic Emirates inside Syria, as components of a future Caliphate.

Jabhat al-Nusra has successfully prepared its surroundings in such a way as to give it an improved chance of surviving in the long-term, despite international counterterrorism efforts. The international community must work to de-escalate the situation in Syria and more forcefully push for a diplomatic settlement so as to prevent the establishment of a longstanding jihadi safe haven. Policy decisions, including expanding support to Syria’s mainstream opposition, encouraging opposition-Kurdish dialogue, and interrupting al-Qaida’s finances, can diminish Jabhat al-Nusra’s chances of survival.

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• Profiling Jabhat al-Nusra

Executive Summary

Large-scale antipoverty programs have achieved significant and positive results in many developing countries around the world in the past decade. This paper explores the challenges of “scaling up” small-scale antipoverty programs—taken here to mean the processes by which successful efforts to raise the incomes of the poorest citizens in developing counties are expanded in coverage over time and across geography. In particular, I advocate supplementing approaches that highlight resource and program constraints with an expanded focus on the political dynamics involved in expanding pro-poor policies. Thus, greater emphasis should be placed on understanding the political factors that limit the expansion and survivability of antipoverty programs. A broader view along these lines highlights the bargaining strength of beneficiaries, the need to secure public support, the potential for political misuse of antipoverty programs, and how institutional fragilities affect their sustainability. Antipoverty programs can be effectively scaled up if attention is paid to addressing these political and institutional challenges. An agenda for future research is also identified.

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A key objective of the United Nations Development Programme (UNDP) is to assist its member countries in meeting the Millennium Development Goals (MDGs). UNDP pursues this objective in various ways, including through analysis and advice to governments on the progress towards the MDGs (such as support for the preparation and monitoring Poverty Reduction Strategies, or PRSs, in poor countries), assistance for capacity building, and financial and technical support for the preparation and implementation of development programs.

The challenge of achieving the MDGs remains daunting in many countries, including Tajikistan. To do so will require that all development partners, i.e., the government, civil society, private business and donors, make every effort to scale up successful development interventions. Scaling up refers to “expanding, adapting and sustaining successful policies, programs and projects on different places and over time to reach a greater number of people.” Interventions that are successful as pilots but are not scaled up will create localized benefits for a small number of beneficiaries, but they will fail to contribute significantly to close the MDG gap.

This paper aims to assess whether and how well UNDP is supporting scaling up in its development programs in Tajikistan. While the principal purpose of this assessment was to assist the UNDP country program director and his team in Tajikistan in their scaling up efforts, it also contributes to the overall growing body of evidence on the scaling up of development interventions worldwide.

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Today, Egyptian President Abdel-Fattah el-Sissi is witnessing the most vocal and angry objection to his rule since he took power via a military coup in 2013. Across Cairo and beyond, Egyptians are gathering and chanting some of the same slogans from the January 2011 revolution—such as “the people want the fall of the regime” and “down with military rule.” These protests are not a spontaneous uprising. They were planned and announced on April 15, when thousands of Egyptians took to the streets, protesting the latest in a series of bold and controversial decisions that are slowly and steadily chipping away at Sissi’s once solid support structure abroad and at home.

During Saudi King Salman’s recent visit to Cairo, the Egyptian government announced that it had agreed to transfer sovereignty of two Red Sea islands—Tiran and Sanafir—to Saudi Arabia. This decision, which coincided with a $22 billion oil and aid deal, has a clear short term pay-off: a substantial Band-Aid on Egypt’s gaping economic wounds. But Sissi and his government are once again dramatically underestimating just how self-destructive their behavior can be. As my colleague Tamara Wittes eloquently noted, Egypt “continues to throw obstacles in the road of U.S.-Egyptian cooperation.” But even worse than the self-sabotage in Egypt’s foreign relations is the damage Sissi is doing to his reputation at home. 

The decision to transfer the islands to Saudi Arabia may turn out to be the final nail in Sissi’s coffin.

To the streets, again

Following the announcement of this decision, Egyptians took to Twitter, with the hashtag “leave” and “I didn’t elect Sissi” trending in Egypt. Lawyers filed lawsuits in Egyptian courts opposing the agreement. And plans were made for a much larger protest today, Sinai Liberation Day. 

But today’s protests are different than in the past. First, while the anti-Sissi protesters had time to plan and coordinate their actions, so did the regime. Today, pro-Sissi supporters organized their own protests, proudly waving the Saudi flag in Cairo’s symbolic Tahrir Square. The Egyptian Air Force painted the Egyptian flag in the sky. And the security forces came out in droves early today across greater Cairo, closing off access to most of the usual protests sites (such as the Journalists’ Syndicate and the Doctors’ Syndicate) and making a massive show of force to deter people from coming out. 

The government clearly learned a few lessons since Mubarak’s fall. A law passed in 2013 requires pre-approval from the Interior Ministry for any protest activity. That gave Sissi’s henchmen a green light to round up actual and suspected protesters as they have been doing since Thursday, arresting hundreds of suspected agitators and human rights activists on charges related to organizing today’s protests. (Notably, the pro-Sissi demonstrators have not been touched.) As each new anti-regime protest pops up today, security forces are there, arresting protesters and journalists and dispersing them with tear gas and rubber bullets. Regardless of the final outcome of today’s events, Sissi should pay attention to the growing dissatisfaction among the Egyptian people. 

The symbolism of holding today’s protests on Sinai Liberation Day is potent. Threats to Egypt’s nationalism and national sovereignty have long been key drivers of Egyptian rage, allowing the protest organizers to tap in to the anger and frustration shared by Egyptians across the political spectrum. The outrage citizens have expressed in the streets, online, and in the media should be a red flag to Sissi, who is hemorrhaging support. 

Notably, he’s now struck a nerve not just with Islamists or others in the anti-Sissi crowd, but with one of the few remaining bastions of Sissi supporters—the everyday Egyptians who are not normally politically engaged. This is a group of people who, following five years of political turmoil, see Sissi as Egypt’s best chance at stability in an increasingly unstable neighborhood. And they’re generally willing to forgive Sissi for his transgressions. They don’t believe the theory that the Egyptian security services are responsible for Italian PhD student Giulio Regeni’s death. They agree that foreign funding of NGOs is a form of Western meddling in Egyptian affairs. They justify the brutal crackdown on free expression in the name of security. But secretly concocting a deal to give away Egyptian land—that is one pill even they can’t swallow. 

Final straws?

Making matters worse are reports that Egypt consulted with Israel and the United States prior to the transfer. While the Israeli-Egyptian peace treaty remains active, Egypt and Israel’s peace is cold, at best. The notion that Sissi would consult with Israel over something that he kept secret from his own people is the ultimate insult and betrayal to many Egyptians. The facts behind the transfer matter very little. What matters is the perception of the Egyptian public that President Sissi has duped them. 

The decision to transfer the islands to Saudi Arabia may turn out to be the final nail in Sissi’s coffin. Over the past several months, he has lost other pillars of support—including secular revolutionaries, who saw former President Morsi and the Muslim Brotherhood as subverting the revolution and supported the military’s return to power. The far-reaching and brutal crackdown on Egyptian journalists and NGOs turned many of them off from Sissi. And wealthy Egyptians, who believed Sissi’s promises to grow the economy and protect their assets, have increasingly questioned their leader as Egypt’s economy continues to plummet. 

Sissi is not only running out of supporters, he is also running out of excuses.

Sissi is not only running out of supporters, he is also running out of excuses. Rather than admit his mistakes, Sissi has defended his actions, shifting the blame and feeding conspiracy theories. While protests were growing across Egypt on April 15, Sissi spoke to a group of Egyptian youth, referencing a “hellish scheme” to destabilize Egypt from within. 

Unfortunately for Sissi, there is no such “scheme.” In 2011 it was not a Western plot, as some Egyptian conspiracy theories have suggested, that ousted Mubarak—it was the Egyptian people, fed up with actions Mubarak carried out as president. In 2013, the coup that ousted Morsi succeeded because the people were fed up with decisions he made in office to consolidate power and reject democratic reforms. Had either Mubarak or Morsi spent as much time responding to the wants and needs of their citizenry as they had quashing dissent, one of them might still be in office. Much like his predecessors, what Sissi fails to understand is that the thing most likely to destabilize his government is neither an external conspiracy not an internal scheme—it’s him. 

A central function of the United States Congress is oversight of the executive branch. Congressional oversight, as exercised from the beginning of the nation, is an essential tool in making the separation of powers real by empowering Congress to check the executive. In recent years, however, as polarization has reached paralyzing levels, Congress has largely gotten out of the business of routine and prospective “police-patrol” oversight.  In the absence of the will and the capacity to do prospective oversight, Congress is at risk of losing its power to the executive branch and thus failing one of its most important constitutional roles.

This paper assesses whether or not anything can be done to get Congress back into the oversight business. Specifically, author Elaine Kamarck examines the following question: Assuming that future Congresses develop the political will to conduct oversight, do they have the capacity to do oversight of a large, modern, and complex executive branch?

As Kamarck illustrates, mismatched resources may make it difficult for Congress to resume its oversight function. The modern federal government is a complex and enormous enterprise. But as the executive branch has grown considerably over the past decades, Congress has adopted budget cuts that make the legislative branch less and less capable of undertaking the kinds of systemic oversight that can solve or prevent problems. Congress employs a mere 17,272 professional staff to oversee an executive branch consisting of 4.2 million civil servants and uniformed military. 

“The existing infrastructure that is supposed to help Congress be on top of the executive branch has fallen prey to a mindless dumbing down of Congress,” Kamarck states. She details the five entities that are meant to support Congress in its oversight role: committee staff, the Congressional Research Service, the Government Accountability Office, the Congressional Budget Office, and the Inspectors General, all of which are understaffed and under-budgeted. Kamarck recommends the first thing Congress should do to fix its oversight problem is to properly staff the agencies it already has and to stop nickel and diming and degrading its own capacity.

Furthermore, Kamarck calls for a “Congressional Oversight Office,” a body charged with evaluating governmental performance before a crisis arises. This office should be staffed by implementation professionals who can gather the signals from all the other oversight organizations annually and in sync with the budget cycle.

“Congress needs to get back into the business of productive executive branch oversight,” concludes Kamarck. A Congressional Oversight Office is certainly a step in that direction.

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Editor’s Note: In the aftermath of the United Kingdom's vote to leave the European Union, we are left with more questions than answers. Dhruva Jaishankar writes that with all the questions about what happens next, there's a bigger question worth asking: What are the implications of Brexit for democracy? Arguably, Brexit represents the first major casualty of the ascent of digital democracy over representative democracy. This piece was originally posted by The Huffington Post.

In the aftermath of the United Kingdom's vote to leave the European Union, we are left with more questions than answers. What kind of relationship will the UK now forge with the EU, and how will that affect economic relations and migration? Will Scotland and Northern Ireland opt to leave? What is the future of British politics, given turbulence within both the Conservative and Labour Parties? Will a successful Brexit set a precedent for other EU members -- perhaps even some eurozone members-- to leave the union? What are the long-term economic consequences of the resulting uncertainty? Will Brexit even happen at all, given the absence of a clear post-referendum plan, the apparent unwillingness of 'Leave' campaign leaders to invoke Article 50 of the Lisbon Treaty, and the fact that the referendum was advisory and non-binding? Answers to these questions will make themselves evident in the coming weeks, months, and years.

[D]igital democracy... has contributed to polarization, gridlock, dissatisfaction and misinformation.

But there's a bigger question worth asking: What are the implications of Brexit for democracy? Arguably, Brexit represents the first major casualty of the ascent of digital democracy over representative democracy. This claim deserves an explanation.

When historians look back at the world of the past 25 years, they will likely associate it not with terrorism or growing inequality but with the twin phenomena of the "rise of the rest" (particularly China and India) and of globalization. Globalization involves the easier, faster and cheaper flow of goods, people, capital and information. One big enabler of globalization is the internet, the global network of networks that allows billions of people to cheaply and easily access enormous amounts of digital information. The rise of service and high-technology industries, trade liberalization, container shipping, and the development of financial markets have also been important enablers, as is the increased ease and lower cost of travel, particularly by air.

Many technology optimists have assumed that globalization would lead to the democratization of information and decision-making, and also greater cosmopolitanism. Citizens would be better informed, less likely to be silenced, and able to communicate their views more effectively to their leaders. They would also have greater empathy and understanding of other peoples the more they lived next to them, visited their countries, read their news, communicated, and did business with them. Or so the thinking went.

[L]eaders only exploit the vulnerabilities of a post-fact world. The conditions have been laid by the digital sphere.

But there has been little to justify such panglossianism. There is some evidence for a correlation between greater information, political democratization and economic progress, in that all three have advanced steadily, if at different paces, over the past two decades. But that correlation is weak. Instead, digital democracy -- the ability to receive information in almost real time through mass media and to make one's voice heard through social media -- has contributed to polarization, gridlock, dissatisfaction and misinformation. This is as equally applicable to the countries in which modern democracy took root -- in the United States and Europe -- as it is to India, the biggest and most complex democracy in the developing world.

The ascent of digital democracy around the world has some shared features. One characteristic is that access to greater information has, rather counterintuitively, contributed to a "post-fact" information environment. Nick Cohen -- speaking of British pro-"Leave" journalists-turned-politicians Boris Johnson and Michael Gove --called out their use of bold claims, their contempt for practical questions, their sneering disregard for expertise, and their transgressions of the bounds of political spin. These tactics are not all that dissimilar to Donald Trump's assertions about Barack Obama's birth certificate or immigration policies, or Subramanian Swamy's insinuations about the nationality of senior Indian policymakers.

But leaders only exploit the vulnerabilities of a post-fact world. The conditions have been laid by the digital sphere. A recent example springs to mind. There is a widespread belief on Indian social media that US presidential candidate Hillary Clinton is somehow anti-India, pro-Pakistan, and/or anti-Modi. I am no supporter of Ms. Clinton, but as someone who worked on foreign affairs in Washington and knows many of her advisors, I found these claims baffling. In fact, Clinton's political opponents (whether Barack Obama in 2008 or Donald Trump in 2016) have accused her of being too close to India, while Pakistanis often view her as critical of their country and Prime Minister Modi appears to enjoy cordial relations with her. After some inquiries, and a few tips, I managed to trace these sentiments to a single publication, a poorly sourced and misleading column that gained widespread circulation upon its release. The article's contents were deemed sufficiently credible to have now become instilled as absolute fact in the minds of many Indians active online. In a digital democracy, a lie or (better yet) a half-lie if told enough times becomes truth.

In a digital democracy, a lie or (better yet) a half-lie if told enough times becomes truth.

Another outcome of digital democracy may be a variation of what the psychologist Barry Schwartz has called the paradox of choice. Quite possibly, the greater abundance of political choice leads to less satisfaction, and the result is citizens increasingly voicing their displeasure with their available political and policy choices. The political platforms of mainstream parties rarely adhere entirely to individual voters' views. That may explain why many voters are gravitating towards parties, factions or leaders who offer the simplest messages, and project themselves as alternatives to the mainstream.

A third result of digital democracy, and one that has been better documented, is the political echo chamber. Social media, rather than creating connections with people who possess differing views and ideologies, tends to reinforce prejudices. As the psychologist Nicholas DiFonzo has noted, "Americans across the political spectrum tend to trust the news media (and 'facts' provided by the media) less than their own social group." This makes it easier for views and rumours to circulate and intensify within like-minded groups. Similar digital gerrymandering was evident in the EU Referendum in Britain and the polarization is palpable in the Indian online political space.

Finally, instant information has increased the theatricality of politics. With public statements and positions by governments, political parties and individual leaders now broadcast to constituents in real time, compromise, a necessary basis of good governance, has become more difficult. When portrayed as a betrayal of core beliefs, compromise often amounts to political suicide. Political grandstanding also contributes to legislative gridlock, with elected representatives often resorting to walkouts, sit-ins, or insults -- all manufactured for maximum viral effect -- instead of trying to reach solutions behind closed doors. Even as ease of travel allows legislators to spend more time in their constituencies, making them more sensitized to their constituents' concerns, less gets done at the national or supranational level. It is a trend that, once again, applies equally to the United States, Europe, and India.

Social media, rather than creating connections with people who possess differing views and ideologies, tends to reinforce prejudices.

The unintended consequences of digital democracy -- misinformation and discontent, polarization and gridlock -- mean that the boundary between politician and troll is blurring. The tone of democratic politics increasingly reflects that of anonymous online discourse: nasty, brutish, and short. And successful politicians are increasingly those who are able to take advantage of the resulting sentiments. Exploiting divisions, appealing to base instincts, making outlandish claims, resorting to falsehoods, and pooh-poohing details and expertise. All that could just as easily describe the playbooks of populists around the world, on the right and left: Marine Le Pen, Frauke Petry, Donald Trump or Subramanian Swamy as much as Jeremy Corbyn, Beppe Grillo, Bernie Sanders or Arvind Kejriwal.

The unintended consequences of digital democracy -- misinformation and discontent, polarization and gridlock -- mean that the boundary between politician and troll is blurring.

In all these cases, populists are willing to cross the lines that mainstream parties have flirted with, becoming forces that the centre cannot hold. US Republicans fanned the anti-immigration sentiments that first the Tea Party and then Trump are only taking to their natural conclusions, just as mainstream Democrats' economic protectionism has been seized upon by Sanders. Cameron's euroscepticism, explained away initially as constructive criticism, spiralled out of control with Brexit, just as those who pronounced the death of New Labour helped paved the way for Corbyn. Will the same one day apply in India, to the economic populism of the Congress, of which Kejriwal has become a new torchbearer, or to the chauvinism of the right, which Swamy now threatens to run away with?

Brexit is not anti-globalization so much as a product of globalization. It is also a product of democracy rather than an affront to it. But it is a democracy of a different sort, one that many of its ideological forebears anticipated. When James Madison warned of "the superior force of an interested and overbearing majority," or John Stuart Mill cautioned against "a social tyranny more formidable than many kinds of political oppression," or BR Ambedkar argued (in a slightly different context) that "political tyranny is nothing compared to social tyranny," they could just as easily have been speaking in 2016 as in 1787, 1859, or 1936. Democrats around the world may not yet be married to the mob, but plenty have been betrothed.

None of this should be interpreted as some kind of nostalgia for an older, simpler world. That world was not necessarily simpler, but it was more violent and chaotic, prejudiced and unfair, and poor and backward. It may be hard to discern amid the smoke and noise, but there are some benefits to digital democracy. Information is no longer in the hands of the few. It is easier than ever to bring injustices to light. And the same process can throw up mainstream leaders from backgrounds that are far from privileged, such as a Barack Obama, Angela Merkel, or Narendra Modi. Two of the three, Obama and Modi, rose to power on the backs of unprecedented social media movements.

But representative democracy as we have come to know it is under threat, and Brexit represents the first major casualty. Rather than fight the tide, a collective rethink is needed about how to make democracies resilient and productive in the digital age. It won't be easy.

Turkish President Recep Tayyip Erdoğan has responded to last week’s coup attempt with a hammer. Over the span of just a few days, more than 50,000 people have been fired from their jobs or detained on suspicions that they’re connected to the coup or to the Gülenist movement (which President Erdoğan blames for the coup attempt). Now emergency rule has been imposed, suggesting that more detentions may follow.

Turkish leaders are assuring everyone that the state of emergency is meant to control the situation and to preserve Turkish democracy. But many observers, including in the West, aren’t buying it: There are legitimate fears that these measures will actually further consolidate Erdoğan’s authoritarian rule. And the consequences of Turkey’s continued drift away from democracy isn’t only a human rights or governance problem—it could become a real geopolitical challenge for the West. 

The swinging pendulum

Turkey—literally the bridge between Europe and Asia—sometimes seems of two minds on governance issues. On the one hand, its leaders express a commitment to a Western form of governance based on the rule of law, liberal democracy, transparency, and accountability. On the other—and more in the vein of governance styles in Russia, Iran, and China—they sometimes reject what they see as outside interference, restrict civil liberties and government transparency, and promote a heavy state role in the economy. 

Although Turkey was welcomed into NATO and other transatlantic institutions after World War II—at a time when Soviet expansionism was a real fear—its commitment to democratic values has always been shaky. The military’s shadow loomed large over Turkish politics (last week’s coup attempt was far from the first) and the country’s human rights record was poor, particularly on minority rights. 

Many thought that all this would change when Erdoğan and his Justice and Development Party (AKP) came to power in 2002. They introduced political reforms that propelled Turkey toward EU membership. The Turkish economy excelled: Many people in Turkey once depended on remittances sent by Gastarbeiters (guest workers) in Germany and other West European countries, for instance, but the country quickly became host itself to immigrants from neighboring countries. Tourists, business people, students, athletes, and artists poured into the country in the millions. And Turkey enjoyed considerable soft power in the region and the world, often touted as a model in the wake of the Arab Spring of a country that properly paired mainstream Islamism and democratic governance. None of this would have been possible were it not for Turkey’s growing adherence to Western governance norms and its membership in the transatlantic community. 

But the picture has since become rather grim. The events of the past week have renewed concerns about the state of Turkish democracy, yes—but those concerns have in fact been growing for years. Turkey’s commitment to supporting freedom of expression, freedom of the media, anti-corruption efforts, and liberal markets has been in serious doubt for a while. Meanwhile, the economy has stalled, related in part to political developments and to a recent spate of terror attacks that have seriously damaged the overall security situation. It is no wonder that Turkish per capita income—which peaked at $10,800 in 2013—has now fallen to 2009 levels, at $9,950. (That’s an almost 10 percent drop in the span of just two years.) Turkey’s further slide away from Western governance norms would likely only make matters worse, making Erdoğan’s promise of putting Turkey among the largest 10 economies in the world a fantasy.

If you ask Erdoğan and his AKP colleagues why reforms sputtered out, they’re likely to answer with conspiracy theories: They’ll blame the West, the EU, the interest rate lobby, and others. But the AKP has failed to be self-critical, which could have helped it succeed. 

Turkey’s choice of orbit

So if Turkey seems to be moving away from Western norms, is it also moving away from the West? Possibly. In November 2013—after years of stop-and-go accession talks with the EU—Erdoğan sought Russian President Vladimir Putin’s support for accepting Turkey into Eurasian organizations like the Shanghai Five. That could be a big geostrategic gain for Russia, something not lost on the Russian press.

Western Europe and the United States would be the biggest losers if Turkey moved closer to Russia’s camp. Losing their partnership with Turkey would deliver a serious blow to the fight against ISIS in Syria and Iraq, for one thing. But it would also further dim prospects that Turkey might really embrace Western-style democracy any time soon. As Ted Piccone has written, Turkey has the potential to be a linchpin of the liberal international order—and a long-term downturn in the country could have wide detrimental effects in regional and global governance. 

The path ahead

Finally, is there a role for the United States in all this? In the short term, as Ömer Taşpınar has argued, the United States should offer real help to Ankara in investigating the role of Pennsylvania resident Fethullah Gülen and his movement in last week’s coup attempt. Extradition is a highly sensitive issue, and the United States must defend its legal standards. At the same time, that kind of cooperation could build trust in U.S.-Turkey relations, calm Ankara’s paranoia about a potential U.S. role in the coup attempt (and therefore possibly help minimize the damage to Turkish democracy that Ankara itself might cause in its heavy-handed response), and help the United States build credibility on the rule of law. A thorough investigation—including into the Gülenists—is important for determining who was behind the coup attempt. And it’s in U.S. interests to know: As Turkey is a NATO member, a threat against it should be considered a threat against all members. It is in no NATO member’s interest to allow a political earthquake like this to push Turkey from its fold or towards a rival mode of governance. 

This isn’t to downplay the burden now on Ankara; the Turkish government shouldn’t forget that its respect for civil liberties and the rule of law once helped earn it a lot of international respect and a place in the Western community. It’s disappointing that the AKP and Erdoğan supporters have failed to capitalize on their country’s potential. Among its peers in the Muslim world, Turkey had once made the most progress in terms of democratic values and economic growth. Many would still like to believe that that Turkey still exists, in spite of recent setbacks. But for Turkey to win back those gains, its leadership will have to proceed very cautiously and with reason. 

Please note that this Engelberg Center for Health Care Reform Health Policy Issue Brief first appeared in the Health Affairs Blog on July 31, 2014. Click here for the Health Affairs Blog version.

Abstract

Individual patient expanded access is a process by which patients can obtain investigational drugs that have not been approved by the Food and Drug Administration (FDA) outside of a clinical trial setting from biopharmaceutical companies when no other alternative therapy is available. Currently, no industry-wide structural principles exist to help companies navigate this process while balancing the needs of getting a drug to the market as quickly as possible with providing potentially life-saving treatment to individual patients. The Engelberg Center convened a stakeholder group to identify common themes and identify common principles related to expanded access, as none currently exist. The result was 4 A’s - Anticipation, Accessibility, Accountability, and Analysis – to help assist patients, providers, and companies with expanded access. Process and capacity building recommendations for the FDA also were proposed to assist companies with sustaining expanded access programs.

Call to Action: The Importance of Expanded Access Programs

Individual patient expanded access, sometimes termed “compassionate use,” refers to situations where access to a drug still in the development process is granted to patients on a case-by-case basis outside of a clinical trial, prior to completion of mandated clinical trials and approval by the Food and Drug Administration (FDA). This typically involves filing a single patient or emergency investigational new drug (IND) request with the Food and Drug Administration and voluntary release of the drug by the manufacturer. Generally, the following criteria must be met: there is reasonable expectation of meaningful benefit despite the absence of definitive clinical trial data, the patient has a serious or life-threatening condition, there are no comparable or satisfactory treatment alternatives, and there are no suitable clinical trials for the drug available to the patient. This form of expanded access, which is the focus of this paper, is different from the situation in which a drug is discharged to a large group of needy patients in the interval between successful phase 3 trials and presumed FDA approval, a strategy often termed a “treatment” IND or protocol, which was initially used in the 1980s for releasing zidovudine to patients with acquired immune deficiency syndrome.

The Engelberg Center for Health Care Reform at the Brookings Institution recently invited senior leaders from several pharmaceutical companies, two bioethicists, a senior FDA representative, and a patient advocate to share experiences and discuss organizational strategies related to expanded access (see acknowledgements). A driving factor for this meeting was a recent flurry of highly public cases of desperate patients seeking access to experimental drugs, which lead to social media campaigns and media coverage. Such cases included 7-year-old Josh Hardy (brincidofovir from Chimerix for disseminated adenovirus infection), 45-year-old Andrea Sloan (BMN673 from BioMarin for ovarian cancer), 41-year-old Nick Auden (pembrolizumab from Merck for melanoma), and 6-year-old Jack Fowler (intrathecal idursulfase from Shire for Hunter Syndrome). Expanded access requests to the FDA for new patients are increasing, from 1,000 patients nationwide in 2010 to more than 1,200 in 2012.[i] (This is likely an underestimate, since it does not include appeals made directly to companies.)

In the wake of these events, it became clear that many biopharmaceutical companies had varying experiences and policies related to such access. From the domestic regulatory standpoint, the FDA revised its expanded access regulations in 2009, which define criteria that must be met to authorize expanded access, list requirements for expanded access submissions, describe safeguards that will protect patients, and preserve the ability to develop meaningful data about the use of the drug. Biopharmaceutical companies typically face a complex global environment in which legal and regulatory frameworks can differ substantially. At the meeting, a senior FDA representative indicated the agency has approved over 99 percent of expanded access requests submitted via single patient or emergency INDs since 2009, suggesting the regulatory agency is not a major barrier to expanded access. As such, provided the access request is reasonably related to the potential benefits of the drug, the biopharmaceutical company is almost solely responsible for the decision and liability regarding whether to grant expanded access to an individual. Still, the public belief persists that the FDA is the main bottleneck that restricts access. In April 2014, Representative Morgan Griffith (R-VA) proposed H.R. 4475, The Compassionate Freedom of Choice Act of 2014, designed to restrict the FDA’s ability to prevent the use of investigational drugs in terminally ill patients. Similarly, some states have passed “Right to Try” legislation to reduce FDA oversight, but contains no requirement that companies must make drugs available.[ii]

The goal of our meeting was to identify common themes and possibly broad outlines to suggest industry-wide policies related to expanded access, as none currently exist. The group first discussed background issues related to expanded access and agreed on definitions. The meeting then focused on three topics. First, the group participants who play key roles in evaluating expanded access requests were invited to share narrative experiences in specific clinical cases, in an effort to lay the groundwork for trust and open discussion. Second, the group was asked to identify internal industry-specific structural barriers, such as the existence of clear procedures or tracking mechanisms within companies to handle requests. Finally, the participants reflected on situations in which expanded access may not be appropriate, or where regulatory barriers or liability concerns may hinder expanded access. This paper reflects the authors’ observations and assessment of the internal and external landscape, based upon information provided by the meeting participants.

Laying the Groundwork with Shared Experiences

The FDA allows companies to provide drugs and charge individual patients that do not meet the enrollment criteria for clinical trials geared towards regulatory approval through expanded access programs.[iii] These programs are meant to provide the drug directly to treat the patient’s condition, rather than having the primary goal of collecting efficacy or detailed safety data in support of approval. Before 1987, the FDA lacked formal recognition of expanded access, although investigational drugs were provided informally.[iv] Since then, the FDA has instituted novel classes of individual INDs so that a company sponsor or licensed physician can legally obtain treatment access from the FDA to provide a drug while it is still in the approval process.[v] Essentially, this provides companies a legal exception from the law to ship unapproved drugs across state lines, and if they desire, to charge for them. These INDs are designed solely for the potential benefit of desperate patients and not intended to formally collect safety or efficacy data that could potentially inform a regulatory decision, but can have regulatory impact, nonetheless.

At the outset, several participants objected to the term “compassionate use,” since it introduces inherent value decisions, can emotionally charge discussions, and does not recognize that there may be valid and ethically appropriate reasons for denial. The generally agreed upon term “expanded access,” is used throughout this paper. (One participant suggested the term “early access.”) Ideally, the term would make it obvious that this is access to an unapproved drug, in order to temper expectations of favorable results. Somewhat confusingly, the FDA uses the terms “expanded access,” “access,” and “treatment use” interchangeably to refer to the use of a drug, and of which none clearly identify the stage of development.[vi]

Participants shared numerous examples of requests for expanded access and explained that their companies handle anywhere from a handful to several hundred requests per year. The following selected stories illustrate the wide range of experiences and situations that companies encounter when navigating the complex decisions involved in administering an expanded access program. Several other examples were discussed and the specific participants expressed that they would be willing to share these particular examples publicly.

Chimerix, a 54-employee company based in Durham, North Carolina, is developing the drug brincidofovir and previously had created an intermediate expanded access protocol for the drug (CMX001-350) as encouraged by the FDA following over 200 emergency INDs granted for access to brincidofovir.[vii] One such case was for an armed services member with previously undiagnosed acute myelogenous leukemia who developed life-threatening vaccinia infection following smallpox vaccination in 2009.[viii] The patient received the drug from Chimerix through an emergency IND. After two years, the company had not secured FDA approval for the drug and eliminated expanded access in February 2012 in order to focus on studies which would inform a regulatory decision. In March 2014, Chimerix originally rejected an emergency IND request for 7-year old, Josh Hardy, who was critically ill from disseminated adenovirus infection after bone marrow transplantation. A highly public social media campaign targeted the company in the wake of this decision, and the experience was traumatizing for many of the employees. Following discussion with the FDA, Chimerix initiated a new clinical trial for the treatment of adenovirus infection in order to collect safety and efficacy data to support an NDA submission. Hardy was the first patient enrolled in the clinical trial, and his family reported through several media outlets that he recovered from the adenovirus infection and was discharged home.

One biopharmaceutical company representative described receiving a middle-of-the-night telephone call directly at home, with an emergent, time-sensitive request for an experimental therapy for a critically ill child with a rare acute disease in a foreign pediatric intensive care unit, where regulatory standards were different from those in the U.S. The ideal pediatric dosage was unknown, and only limited safety data and clinical details were available. Urgent efforts were made to gather more information and the request was approved, but despite these efforts the patient did not survive.

Bristol-Myers Squibb began a clinical trial for a cancer drug several years ago.[ix] A woman with pancreatic cancer enrolled in the trial and saw that her tumor was no longer growing. After the 3.5 year trial, the study closed because the drug was deemed ineffective for all other patients and was not approved for further development. However, the company continued to provide the drug for the one woman for whom the drug was effective through a single patient IND for an additional 9 years.

To demonstrate the volume of expanded access requests, one participant showed several messages on his mobile device during the half-day discussion, directly from patients who had located his email addresses through on-line searches, to plead for expanded access to an anticancer therapy.

Development of Structural Principles: The Four A's 

Broadly, no specific industry-wide consensus on expanded access procedures exists. As a result, there is significant variation in company policies and procedures. During this phase of discussion, participants shared their own company strategies and suggested possible areas of consensus that might form the basis for shared principles and industry-wide practices. These suggestions fell into four categories, which we termed the 4 “A’s”: Anticipation, Accessibility, Accountability, and Analysis (see Figure 1).

First, the group agreed that large and small companies should anticipate the need for and creation of expanded access programs when developing drugs expected to generate expanded access requests and as part of the drug development plan. This is particularly important for drugs that might be considered for priority or breakthrough designation during FDA approval. In these cases, companies should strongly consider developing a written expanded use policy with clear guidelines for inclusion and exclusion, which would also feature a defined review process, clear decision making criteria, and a defined time frame for response to requests. This also allows companies to plan for the demands that may be placed on their supply chain and staff resources to ensure sufficient supply for investigational and expanded use purposes. Identifying a decision maker within each company and for each disease area/product will also help patients or physicians reach the appropriate contact when requesting a drug, as well as assist the company in gaining expertise in responding to these requests. For example, one large company identifies one point of contact for all expanded access requests regarding each product and posts that individual’s contact information on the website.

In the early stages of drug development, supplies of investigational drugs are extremely limited. This is often because the technically-challenging process of optimizing drug product manufacture takes a considerable amount of time. Low yielding manufacture batches are not uncommon at the early phases of research. Some companies do not approve expanded access requests because they do not have enough of the drug in stock to supply these external requests and meet the needs of investigational study patients and individuals participating in clinical trials, an issue which may be particularly acute for biologics. Smaller companies may have more resource constraints, such as inadequate staff to manage requests or supply chain and logistics issues. One representative suggested that if a company had early transparency from regulators about the final numbers of subjects they would be willing to accept to achieve drug development milestones, it would make it much easier for the company to feel less reservation about its drug supply. (It may be beneficial for companies to analyze their financial ability to provide drugs potentially at no cost or when there is not a large enough supply, ideally in a transparent manner.)

Once an expanded access policy is anticipated and developed, the second key principle the group identified was making the policy accessible to all individuals who may qualify. First, for patients, with guidance from their treating physician, the company making the drug should always steer the patient to enter a clinical trial (if they meet eligibility criteria). If the contacted company cannot accommodate the patient, they should steer them to other open trials if possible, even if sponsored by another company. Many of our participants noted that this already occurs.

The group was particularly cognizant of the disparity in access to drug companies and their expanded access programs: patients with savvy social media strategies are more likely to succeed in navigating across organizational constraints than without similar sophistication. The group believes that increased accessibility would assist in making opportunities for expanded access more equitable. In addition, these policies could help educate patients and physicians about submitting legitimate expanded access requests and help decrease the costs of reviewing inappropriate requests on the company (for example, if there are other proven therapies or the situation is not life threatening).

If the patient is ineligible for a trial, the patient should be able to easily access the written expanded access policy online. For example, both large and small companies like Pfizer, Bristol-Myers Squibb, Shire, and Merck post their expanded access policies on their websites, though the terminology may in some cases be complex. In addition, Janssen has developed a video explaining their policies in non-technical terms. Ideally, such policies should be available in some web based or public facing platform to both patients and physicians and written in a clear manner that is jargon free and accessible to individuals at various education levels. Most participants felt strongly that requests for expanded access should originate from a medical provider, not from a patient, since expertise is needed to first screen appropriate candidates. This is consistent with current FDA regulations for an IND, in which a physician or qualified medical expert must sponsor an IND or serve as an investigator under an existing IND for expanded access.

Third, companies should have accountability to the requesting party for expanded use requests that they receive and review them within a specified, transparent amount of time. If the request could not be approved, the company should consider clear communication and provide an explanation of why the request was turned down. In these cases, some participants suggested that the company might also consider instituting an appeals process by which a patient can receive an additional review if not approved, potentially from a non-binding third party such as an independent, multidisciplinary body or a regulatory agency like the FDA. (Two participants, however, were uncomfortable with any third party review.)

The final principle would encourage companies to release timely analysis of data from expanded access patients. In addition to tracking communication, companies should keep a database of the number of requests and outcomes, in a manner that doesn’t slow getting drugs to needy patients rapidly. One company refined its internal tracking tools to determine who was requesting drugs, for what conditions, and where they lived. Where possible, companies might be encouraged to share anecdotal or preliminary safety or efficacy data from expanded access in peer-reviewed or other refereed venues in a prudent time frame following collections, if this is available or known. This is not always possible, because emergency INDs do not require provision of safety or outcome data to the company.

There are several challenges associated with operationalizing this in the current model, namely the appropriateness of anecdotal data, the level of detailed safety and efficacy data currently available through expanded access, suitability for publication, and funding for these activities in the current budget climate. One potential approach to address this is funding from federal or state regulatory agencies or payers for the reasonable costs of follow-up and reporting outcomes.

Regulatory Considerations

The participants then discussed the types of risks, including regulatory and financial, that may affect companies’ expanded access policies. When a company is considering expanded access requests, they consider the risks-benefits of providing the drug outside of a clinical trial as well as the potential for any regulatory issues in an era of litigation and an increased threshold for demonstration of safety. While a company’s provision of a drug for expanded access is voluntary, the FDA does require the company to collect and report safety data. Notably, none of the representatives felt that the FDA is a major regulatory barrier to processing and approving expanded access requests once the sponsor has reviewed the request, assessed the benefit-risk, and determined the request meets FDA requirements and evidentiary standards. In addition, the attendees felt that adverse effects and related liability risk were not of particular concern given that the drugs are assessed on a risk-benefit analysis.

However, companies that make drugs in particularly limited markets with small numbers of patients (for example, for unusual diseases with less than 200,000 patients nationwide which may justify a special designation called “orphan status”) may be more concerned about restrictive labeling if an unusual adverse event occurred even in one or two patients during expanded access of an orphan or small market therapy. However, there is no data of which participants were aware and no public reports that an adverse event during expanded access has harmed regulatory approval.[x] The group opinion was that that safety data would be available eventually in any event and an FDA “safe harbor” provision would not necessarily affect companies’ willingness to accept more requests for expanded access. A final concern was that there is no regulatory mechanism to consider data from expanded access in the evidence generation process for approval.

An Expanded Role for the FDA

While the FDA may not serve as a strong barrier to expanded access, the group considered strategies to promote equitable and fair access. For example, some argued that the breakthrough or priority review categories for FDA review might identify products that could have high potential for expanded access requests. This designation expedites “the development and review of drugs for serious or life-threatening conditions.”[xi] As of mid-April 2014, the FDA had received nearly 180 requests for breakthrough designation, with 44 requests granted.[xii] By hastening the drug development process, the FDA has already begun to bring drugs that have a reasonable expectation of benefit to the market faster. In order to receive breakthrough therapy designation, current legislation might be amended so companies could be asked to provide evidence that the 4 A’s are being followed in some capacity.

The FDA might also assist companies in establishing expanded access programs during open clinical trials in two main areas: process and capacity building. First, in terms of process, the FDA could be asked to create a defined path for regulatory approval with provisions that would encourage companies, both large and small, to include plans for expanded access programs when developing a drug. While FDA’s draft guidance related to INDs notes that larger expanded access programs could threaten enrollment in clinical trials,[xiii] and some participants agreed that this was a significant issue, not all companies have had difficulties enrolling patients in both clinical trials and expanded access programs. For example, one large pharmaceutical company left a Phase 1 clinical trial open for a promising therapy while concurrently enrolling individuals who didn’t qualify for open clinical trials into an expanded access program, without appreciable leakage of enrollees in their advanced phase trials that might affect the key development pathway.

Second, the FDA could support convening around capacity building and sharing best practices with companies. With the understanding that there are many small biotechnology or pharmaceutical companies with limited budgets and staff, the FDA could foster a partnership of large and small companies. This partnership could be achieved by convening meetings where companies share their experiences in creating and sustaining expanded access programs. This could be supported by creating a database for these shared ideas, as well as any expanded access data that can be made legally available, such as how many requests are granted or patient outcomes.

To ensure equitable, consistent, and transparent review of requests, some companies suggested the use of an impartial external advisory board. Similar to an unbiased review from an institutional review board (IRB), this committee could have an advisory or decision making function. Companies with supply constraints may feel that if they cannot give the drug to everyone who requests it, then they should give it to no one. This committee could help the company triage the patients who would benefit the most, and would be protected from liability.

Next Steps

The most efficient and equitable way to make new effective treatments to the largest number of needy patients is regulatory approval, accelerated or otherwise, following successful demonstration of efficacy and safety for a given indication in a specific population. Until that process is complete, access to an experimental therapy is by definition an additional risk, as the agreed necessary safety and efficacy have not yet been demonstrated. True informed consent in this setting is difficult to obtain (i.e. studies have shown that severely ill patients, such as those with life-threating circumstances requesting expanded access, had less retention of information discussed in the informed-consent process and less-clear understanding of the risks of therapy compared to healthier patients[xiv]).

One position companies and regulators can consider is that the default answer to expanded access requests should be affirmative, unless there are compelling reasons for not approving requests to patients with life-threatening illnesses. (Such reasons, for example, might include limited treatment supply or lack of reasonable expectation of benefits versus risks.) Such a position would require, however, that there be broader industry, clinician, regulatory, and patient advocacy agreement of shared principles. This paper outlines the experiences, structural principles, and regulatory considerations of a small group, but further meetings may convene a broader group of stakeholders to build upon these concepts. Such consensus-based approaches might lead to durable systems that meet the needs of desperate patients who have run out of options—while allowing innovation to continue to benefit those who may come afterwards.

Acknowledgements: We are grateful for the participation of the following representatives in the roundtable: Jeff Allen (Friends of Cancer Research), Michelle Berrey (Chimerix), Renzo Canetta (Bristol-Myers Squibb), Anne Cropp (Pfizer), Joseph Eid (Merck), Aaron Kesselheim (Harvard Medical School), Howard Mayer (Shire), Jeffrey Murray (FDA), Lilli Petruzzelli (Novartis), Amrit Ray (Janssen), and Robert Truog (Harvard Medical School). We thank Mark McClellan (Brookings Institution) for helpful discussions of this topic and comments on the manuscript, and to the Richard Merkin Foundation for support. The views and opinions expressed in this article were interpreted and organized by the staff of the Brookings Institution. They do not necessarily reflect the official policy or position of any individual roundtable representative, their companies, or their employers.

References

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• The Four A's of Expanded Patient Access

The Centers for Medicare and Medicaid Services (CMS) recently released more detailed ACO-level data for participants in first two years of the Pioneer ACO Model. The program, which is designed for health systems with more experience assuming financial risk for patient populations, has generated savings and improvements in quality measures, but has also struggled to retain participants. The program began with 32 provider organizations; following a series of recent announcements there are now 19 total participants.

Last month, CMS announced that the Pioneer Program was able to yield total program savings of $96 million in its second year and resulted in ACOs sharing in savings of $68 million. CMS also reported that the Pioneers were able to improve mean quality scores by 19 percent and increased performance on 28 of 33 measures between performance year one and performance year two.

Financial Results

The latest financial results provide more participant-level data and allow for a new level of analysis of performance across all these ACOs. In year one of the program, financial performance for individual Pioneers ranged from a gross loss of $9.31 million to a gross savings of $23.34 million. Thirteen Pioneers reduced costs enough to qualify for shared savings, with an average of $5.85 million returned to the ACOs, ranging from $1.00 million to $14.00 million. One ACO owed shared losses of $2.55 million. The remaining eighteen ACOs were within the minimum savings or loss rate and did not earn shared savings or owe money to Medicare due to losses.

Following year one, nine Pioneer ACOs either left the Medicare ACO program entirely, or moved to the lower risk Medicare Shared Savings Program (MSSP). Eight of the nine Pioneers that left the program failed to reduce spending in their first year. Out of the remaining 23 participants in the second performance year, three of these ACOs opted to defer reconciliation until the end of Performance Year 3. The 20 Pioneers with final Performance Year 2 data had financial performance ranging from a gross savings of $24.59 million to gross losses of $6.26 million. Fourteen ACOs reduced spending in Performance Year 2, eleven of which reduced enough to qualify for shared savings. The average shared savings for these ACOs was $6.55 million, ranging from $1.22 million to $13.41 million. Three Pioneers shared losses, averaging $2.33 million back to the Medicare program.

The table below shows the breakdown of ACOs according to whether they reduced spending, increased spending, shared in savings, or owed money back to Medicare due to losses. More than half of the Pioneers were able to reduce spending in year one (18/32) and year two (14/23), with more than one-third of total ACOs earning shared savings in each year as well.

The data also suggest that those ACOs that were most successful in reducing spending in the first year were also more likely to reduce spending in their second year. As the chart below shows, three ACOs that earned shared savings in year one owed money back to Medicare due to losses in year two, while no ACO that had shared losses in year one was able to attain shared savings in year two.  

Quality Results

CMS also released ACO-level performance on all 33 measures for Pioneer participants in year one and year two. The 23 ACOs that remain in the Pioneer Program showed overall improvement in average quality scores from the first to second performance year. The ACOs also improved overall on 28 of 33 measures, as the chart below shows.

The quality domain with the greatest improvement in year two was Domain 4 (At Risk-Populations) which saw an overall improvement from 67.5% to 83%. The marked improvement in this domain suggests that ACOs are making progress at better coordinating and delivery care for high-risk patients, many of whom have multiple chronic conditions. Chronic care management for conditions such as diabetes, coronary artery disease, and hypertension is critical for the continued success of accountable care efforts. All other domains saw average quality improvement as well, summarized below.

Likewise, almost all of the individual Pioneer ACOs improved their performance on quality measures from year one to year two. Of the ACOs that remained in the program for year two, all but one ACO was able to improve its overall quality score in its second year.

Additionally, the percentage of Pioneer ACOs performing in the 80^th or 90^th percentile in quality scores also increased from year one to year two, as shown in the chart below.

Putting Together Financial and Quality Results

In year one of the Pioneer Program there appeared to be no direct correlation between average quality scores and gross savings or losses for individual ACOs. This may not be unexpected, especially since Pioneer ACOs in their first year are eligible for shared savings simply by reporting their quality. In subsequent years, however, the ACO’s quality score impacts the level of shared savings that the Pioneers are eligible to receive, so we might expect a bit more alignment between quality and financial performance. Average quality scores and level of savings or losses for each of the 32 first year Pioneer ACOs is below.

After year two, there still does not appear to be a direct relationship between higher quality scores and level of savings or losses in the Pioneer Program. Further examination of results begs additional questions about why certain ACOs clustered in different parts of the grid relative to others.

Of those ACOs in the red circle above— higher total savings and relatively average quality scores—two of the ACOs are from the Boston area and the remaining ones from other large metropolitan areas (New York City; Orange County, CA; Phoenix, AZ; and Detroit, MI). The average per capita Medicare spending for the counties corresponding to these ACOs is $11,544, compared to an average of $10,384 for counties corresponding to all 23 of the Pioneer participants.

Meanwhile those ACOs within the yellow circle had the highest quality scores, but also experience financial losses or slight savings. Many of these ACOs are from less densely populated areas, such as Maine, Wisconsin, and Illinois. There are a number of factors that could be contributing to their quality success, but little financial savings—healthier patient populations, a smaller or more engaged patient population, financial baselines impacted by lower per capita spending in these areas, or other factors driven by their region. Further analysis of these ACOs and the other public and private ACO programs, including both their characteristics and regional market characteristics, will provide needed further insights on the factors most likely to drive success.

Next Steps

These ACO-level data reflect the range of experiences across Pioneer participants. Some ACOs have sustained positive performance to date, while others have seen diminishing rates of return. Those organizations more committed to clinical transformation, patient outreach, and organizational change may be more likely to do better, but further analysis of differences in performance could enable the Pioneer Program and ACOs to achieve bigger impacts over time.

It is hard to know what the third performance year of the Pioneer program will show, but as noted earlier, the Pioneer Program has already lost over a third of its original 32 participants. Despite the decline in participation and mixed results so far, CMS remains optimistic and committed to the program, and the overall number of Medicare, Medicaid, and privately-insured individuals in ACO arrangements continues to rise. We can anticipate a proposed rule impacting the MSSP, likely later this Fall, which will impact elements of the Pioneer ACO program. Regulatory changes that may help increase the ability of the Medicare ACO programs to support better care while ensuring sustainability include: adjustments to attribution methods, benchmark calculations, collection and sharing of data with ACOs, updating performance measures, linking to other ongoing payment and delivery reforms, and creating more financial sustainability for program participants. The current Pioneer program can be a key step toward effective payment reform, but further steps are needed to assure long-term success.

Event Information

Over the past few years, more than 600 Accountable Care Organizations (ACOs) have formed across the country, charged with the dual goals of improving health while also reducing health care costs. Increasingly, evidence on how public and private ACOs are progressing toward these goals is beginning to emerge. Based on these results, major regulatory changes are anticipated in the months ahead that will impact accountable care programs in Medicare, as well as future uptake within the private sector.

On October 20, the Engelberg Center for Health Care Reform hosted a half day forum to assess the latest evidence on accountable care, discuss strategies to overcome unique ACO challenges, and provide an overview of accountable care reforms. Sean Cavanaugh of the Centers for Medicare and Medicaid Services (CMS) provided keynote remarks on the latest Medicare ACO results and potential changes to the Medicare Shared Savings Program (MSSP). Panel sessions featured leading experts in ACO research, implementation and health care policy.

 Join the conversation on Twitter using #ACOFuture or follow @BrookingsMed

Video

• The Medicare Shared Savings Program (MSSP): Looking Ahead
• Big Issues for ACOs Going Forward
• ACO Policy Issues and Solutions on the Horizon

Audio

• The State of Accountable Care: Evidence to Date and Next Steps

Transcript

• Uncorrected Transcript (.pdf)

Event Materials

• ACO Public Event full deck FINAL
• 102014_Accountable Care_Transcript