at Funding Cutbacks at FDA: A Sequester Primer By www.fdamatters.com Published On :: Thu, 07 Mar 2013 17:59:38 +0000 At a time when FDA’s responsibilities continue to grow rapidly, the agency has been caught in an across-the-board reduction (sequester) in federal discretionary spending, effective March 2, 2013. Although Congress may yet reverse course and restore money to affected federal agencies, this is not considered a high probability. Altogether, FDA will lose about $209 million between now and September 30, 2013. This will reduce inspections, slow drug and device approvals, and restrict implementation of the Food Safety Modernization Act and other recent legislation. Because of the many questions about the process and outcome, this is FDA Matters’ primer on the sequester of FDA funds. Full Article FDA and Congress FDA Appropriations Planning for FDA's Future
at A Salmon on Every Plate/The Hard Road of Innovation By www.fdamatters.com Published On :: Thu, 14 Mar 2013 18:32:50 +0000 Chicken was once an expensive delicacy. In 1928, America’s quest for a better diet and a better standard of living was summarized by the campaign promise of “a chicken in every pot.” Today, chicken is a ubiquitous, low-cost source of protein, which we largely take for granted. Despite depletion of ocean-based stocks, fish hold similar potential. To begin this transformation, FDA must approve a scientifically-based innovative product—a faster growing genetically-engineered (GE) Atlantic salmon. When FDA Matters wrote about this subject 18 months ago, I believed the agency was near to approval of this first-ever food product from a GE animal. It is still not resolved and there are implications for all innovations that require FDA approval. Full Article FDA and Congress FDA and Industry FDA Leadership Food Issues
at FDA’s Indifferent Attitude Towards the First Amendment By www.fdamatters.com Published On :: Mon, 01 Apr 2013 17:09:04 +0000 The FDA doesn’t care about the First Amendment rights of the companies it regulates. It cares even less about the “free speech” rights of those companies’ sales and marketing representatives. And why should the agency care? One of FDA’s primary missions is to protect the public health and safety of the American people from illegal, adulterated and misbranded products. Doing so involves restraining food, drug, device and cosmetics companies from committing fraudulent and deceptive acts that are not protected by companies’ commercial free speech rights. Nonetheless, FDA Matters envisions opportunities for FDA and industry to broaden permissible product communications. The key is understanding history, not constitutional law. Full Article Drug Approval and Access FDA Accountability and Transparency FDA and Industry FDA Leadership Insight on FDA-regulated Industries
at Sequestration Has Less Impact on FDA? Just Not True By www.fdamatters.com Published On :: Thu, 08 Aug 2013 20:11:21 +0000 “The Hill” newspaper recently reported that: “a survey of federal budgets devoted to developing and enforcing regulations found that many agencies will spend more in 2013 and 2014 than in previous years, indicating that the writing and enforcing of new regulations is largely unimpeded by the massive cuts, known as sequestration.” That certainly sounds authoritative…until you look at the analysis. In fact, the report’s authors appear to know nothing about the federal budget and have used inherently unreliable data in calculating FY 13 and FY 14 spending levels. One can only hope that the authors—allegedly academic experts--know more about regulatory policy than they do about federal budgets. Full Article FDA and Congress FDA and Industry FDA Appropriations
at Minnesota Becomes 18th State to Adopt Consumer Data Privacy Law By cohealthcom.org Published On :: Wed, 05 Jun 2024 15:27:53 +0000 On May 24, Governor Tim Walz signed into law Minnesota’s new comprehensive data privacy law, the Minnesota Consumer Data Privacy Act (HF 4757 referenced as the MCDPA). The MCDPA goes into effect on July 31, 2025, with some exceptions for colleges and universities (who have until 2029). The MCDPA is similar to other state privacy laws, […] Full Article Data Privacy Legislative consumer privacy data privacy legislation Minnesota MN Washington Privacy Act
at Rhode Island Governor Vetoes Consumer Data Privacy Act By cohealthcom.org Published On :: Wed, 03 Jul 2024 14:32:46 +0000 On June 25th, Rhode Island Governor Daniel McKee transmitted without signature (effectively a pocket veto) the Rhode Island Data Transparency and Privacy Protection Act (SB 2500 / HB 7787). The act is based on the Washington Privacy Act model but diverges from the prevalent forms of that model in two ways. First, the act contains a unique […] Full Article Data Privacy Legislative consumer privacy data privacy legislation Rhode Island RI veto Washington Privacy Act
at FTC Revised Health Breach Notification Rule Goes into Effect By cohealthcom.org Published On :: Mon, 29 Jul 2024 14:43:12 +0000 Agencies with healthcare clients in pharmaceuticals, healthcare services, digital health apps, or health-related connected devices such as wearables should take note that the Federal Trade Commission (FTC) final rule updating its Health Breach Notification Rule (HBNR) that took effect on July 29, 2024. The FTC considers a breach to include a covered entity’s unauthorized disclosure […] Full Article Regulatory/FDA Federal Trade Commission final rule FTC Good Rx HBRN Jim Potter PHI privacy
at California Adopts a Bundle of AI & Privacy Laws, Most Controversial Bills Vetoed (Updated) By cohealthcom.org Published On :: Mon, 30 Sep 2024 19:05:07 +0000 Sorry, but you do not have permission to view this content. Full Article Artificial Intelligence (AI) Data Privacy Legislative child data privacy CO Colorado consumer privacy data privacy legislation Jim Potter
at Massachusetts High Court Rules Online Tracking Doesn’t Violate State Wiretap Law By cohealthcom.org Published On :: Wed, 30 Oct 2024 12:23:03 +0000 Sorry, but you do not have permission to view this content. Full Article Courts/First Amendment
at A COVID-19 Cardiac MRI Study: What Went Wrong? By www.cardiobrief.org Published On :: Mon, 24 Aug 2020 22:30:07 +0000 We still don’t know what COVID-19 is doing to the heart or how we should be investigating it and treating it. Last month JAMA Cardiology published a German cohort study of 100 patients recently recovered from COVID-19… A number of striking problems with the study were noted on Twitter…...Click here to continue reading... Full Article People Places & Events Policy & Ethics Prevention Epidemiology & Outcomes COVID-19 MRI scientific misconduct
at Regulatory update for post-registration of biological products in Brazil By www.gabionline.net Published On :: Tue, 29 Oct 2024 09:23:29 +0000 <p>On 3<sup> </sup>June 2024, Resolution RDC No. 876 was published in Brazil in the Official Journal of the Union (DOU)[1], modifying the current regulations regarding the post-registration of biological products (RDC 413/2020).</p> Full Article
at ASBM/GaBI 2024 webinar on BIOSIMILAR RED TAPE ELIMINATION ACT (S2305) By www.gabionline.net Published On :: Tue, 05 Nov 2024 09:01:43 +0000 <p> <b>BIOSIMILAR RED TAPE ELIMINATION ACT (S2305):</b><br /> <b><i>Weakening FDA Regulatory Standards for Biosimilars, Undermining Physician Confidence and Jeopardizing Patient Health</i></b><br /><b>31 October 2024 | </b><b><a href="https://youtu.be/X6-dYZ7fjhM" target="_blank">WATCH REPLAY</a></b></p> Full Article
at Medicines for Europe 23rd Regulatory Affairs Conference 2025 By www.gabionline.net Published On :: Tue, 05 Nov 2024 09:04:58 +0000 <p> <b>23rd Regulatory Affairs Conference 202</b><b>5</b><br /> <b>27</b><b>‒</b><b>28 February 2025</b><br /> Hilton Amsterdam Airport Schiphol<br />Amsterdam, The Netherlands</p> Full Article
at NPRA Malaysia trials new timelines for variation applications By www.gabionline.net Published On :: Tue, 05 Nov 2024 09:13:55 +0000 <p>In May 2024, Malaysia’s National Pharmaceutical Regulatory Agency (NPRA) announced that it will trial new timelines for variation applications of registered pharmaceutical products and natural health supplements (TMHS).</p> Full Article
at Bio-Thera and Gedeon Richter partner to commercialize Stelara biosimilar BAT2206 By www.gabionline.net Published On :: Tue, 05 Nov 2024 09:14:16 +0000 <p>In October 2024, China based Bio-Thera Solutions (Bio-Thera) and Hungary’s Gedeon Richter announced they have reached an exclusive commercialization and license agreement for BAT2206, a biosimilar candidate to Johnson & Johnson’s Stelara (ustekinumab).</p> Full Article
at RPS and pharmacy students' association call for rethink over overseas exam decision By www.pharmaceutical-journal.com Published On :: Tue, 9 Feb 2021 16:42 GMT The Royal Pharmaceutical Society and the British Pharmaceutical Students’ Association have called for all overseas candidates to sit the March 2021 registration assessment in their home countries. Full Article
at Government 'miscommunicated' PPE stock levels to pharmacies during first COVID-19 wave, MPs told By www.pharmaceutical-journal.com Published On :: Wed, 10 Feb 2021 12:17 GMT The government implied wholesalers had more personal protective equipment in stock than was the case during the first wave of the COVID-19 pandemic, the Healthcare Distribution Association has said. Full Article
at Pharmacy negotiators in talks over plans to distribute COVID-19 treatments in primary care By www.pharmaceutical-journal.com Published On :: Wed, 10 Feb 2021 15:25 GMT The Pharmaceutical Services Negotiating Committee is in talks with the government over potential plans to distribute COVID-19 treatments in primary care. Full Article
at Regulator looking at 'flexibility' that would allow overseas candidates to sit registration assessment By www.pharmaceutical-journal.com Published On :: Thu, 11 Feb 2021 14:53 GMT The General Pharmaceutical Council has said it is “double, treble, quadruple-checking” for any “flexibility” that would allow all overseas candidates to sit the March 2021 registration assessment exam in their countries of residence. Full Article
at Risk of mortality drops in COVID-19 patients given anticoagulation within a day of hospital admission, research finds By www.pharmaceutical-journal.com Published On :: Fri, 12 Feb 2021 13:58 GMT Starting COVID-19 patients on prophylactic anticoagulation within 24 hours of being admitted to hospital has been linked to a reduced risk of mortality. Full Article
at Nearly 200 women were prescribed valproate during pregnancy between April 2018 and September 2020 By www.pharmaceutical-journal.com Published On :: Fri, 12 Feb 2021 15:47 GMT Some 180 women were prescribed valproate, a medicine used to treat epilepsy and bipolar disorder, during their pregnancy within a 2.5 year interval, NHS data has revealed. Full Article
at Pharmacies estimated to receive one referral per month through hospital-to-pharmacy referral service By www.pharmaceutical-journal.com Published On :: Mon, 15 Feb 2021 15:27 GMT Community pharmacies will receive an estimated 12 referrals from the Discharge Medicines Service per year. Full Article
at Overseas candidates will be allowed to sit registration assessment remotely, regulator says By www.pharmaceutical-journal.com Published On :: Tue, 16 Feb 2021 12:05 GMT The General Pharmaceutical Council has said most candidates living in countries with a two-hour or more time difference from the UK will be able to apply to sit the registration assessment at home. Full Article
at NHS England lowers threshold for COVID-19 vaccination site applications By www.pharmaceutical-journal.com Published On :: Wed, 17 Feb 2021 12:13 GMT Community pharmacies able to administer up to 400 COVID-19 vaccines per week can now apply to become designated vaccination sites, NHS England has said. Full Article
at New drug cuts the risk of death in bladder cancer by 30% compared with chemotherapy, study suggests By www.pharmaceutical-journal.com Published On :: Thu, 18 Feb 2021 15:30 GMT A new type of drug that targets chemotherapy directly to cancer cells reduces the risk of death from the most common type of bladder cancer by 30%, a phase III trial in the New England Journal of Medicine has suggested. Full Article
at Pharmacy negotiators discuss patient registration with community pharmacies By www.pharmaceutical-journal.com Published On :: Thu, 18 Feb 2021 17:07 GMT Pharmacy negotiators have discussed proposals to take “a patient registration-based approach” to the community pharmacy contractual framework. Full Article
at Half of asthma patients in the UK overusing SABAs, study finds By www.pharmaceutical-journal.com Published On :: Thu, 18 Feb 2021 17:10 GMT More than half of patients with asthma in the UK are “potentially overusing” short-acting β2-agonists, according to research. Full Article
at IFM’s Hat Trick and Reflections On Option-To-Buy M&A By lifescivc.com Published On :: Wed, 13 Mar 2024 11:00:37 +0000 Today IFM Therapeutics announced the acquisition of IFM Due, one of its subsidiaries, by Novartis. Back in Sept 2019, IFM granted Novartis the right to acquire IFM Due as part of an “option to buy” collaboration around cGAS-STING antagonists for The post IFM’s Hat Trick and Reflections On Option-To-Buy M&A appeared first on LifeSciVC. Full Article Capital efficiency Exits IPOs M&As External R&D IFM option deals Option To Buy
at Lessons From A Private Funding Round: Science, Relationships, And Experience By lifescivc.com Published On :: Tue, 02 Apr 2024 11:00:46 +0000 By Mike Cloonan, CEO of Sionna Therapeutics, as part of the From The Trenches feature of LifeSciVC An insightful piece on this blog following the JPM healthcare conference noted the “refreshing burst of enthusiasm” in the biotech sector. It’s true The post Lessons From A Private Funding Round: Science, Relationships, And Experience appeared first on LifeSciVC. Full Article Biotech financing From The Trenches Fundraising CFTR Sionna Therapeutics
at The Biotech Startup Contraction Continues… And That’s A Good Thing By lifescivc.com Published On :: Fri, 26 Apr 2024 11:00:38 +0000 Venture creation in biotech is witnessing a sustained contraction. After the pandemic bubble’s over-indulgence, the venture ecosystem appears to have reset its pace of launching new startups. According to the latest Pitchbook data, venture creation in biotech hit its slowest The post The Biotech Startup Contraction Continues… And That’s A Good Thing appeared first on LifeSciVC. Full Article Biotech financing Biotech investment themes Capital markets Fundraising Biotech startups venture creation
at Mariana Oncology’s Radiopharm Platform Acquired By Novartis By lifescivc.com Published On :: Mon, 06 May 2024 10:42:46 +0000 Novartis recently announced the acquisition of Mariana Oncology, an emerging biotech focused on advancing a radioligand therapeutics platform, for up to $1.75 billion in upfronts and future milestones. The capstone of its three short years of operations, this acquisition represents The post Mariana Oncology’s Radiopharm Platform Acquired By Novartis appeared first on LifeSciVC. Full Article Exits IPOs M&As Leadership Portfolio news Talent #RLT Mariana Oncology Novartis radioligand therapy Radiopharm
at Neuro-Immunology: The Promise Of A Differentiated Approach To Neurodegenerative Disease By lifescivc.com Published On :: Wed, 05 Jun 2024 11:00:30 +0000 By Ivana Magovčević-Liebisch, CEO of Vigil Neuroscience, as part of the From The Trenches feature of LifeSciVC In the last decade, our industry has made great strides in combating cancer by harnessing the body’s own immune system. As it was The post Neuro-Immunology: The Promise Of A Differentiated Approach To Neurodegenerative Disease appeared first on LifeSciVC. Full Article Drug discovery From The Trenches Science & Medicine neurodegeneration neuroimmunology neuroinflammation TREM2
at Looking for Opportunities to Accelerate Clinical Research in Rare Diseases By lifescivc.com Published On :: Wed, 17 Jul 2024 11:00:40 +0000 By Mike Cloonan, Chief Executive Officer of Sionna Therapeutics, as part of the From The Trenches feature of LifeSciVC The drug development process in rare diseases is rife with challenges especially when companies target significant differentiation or first-in-class targets. Identifying The post Looking for Opportunities to Accelerate Clinical Research in Rare Diseases appeared first on LifeSciVC. Full Article Business Development From The Trenches Portfolio news Rare Diseases Science & Medicine CFTR Cystic Fibrosis NBD1 Sionna Therapeutics
at Keeping It Simple: What Really Matters For Emerging Enterprises By lifescivc.com Published On :: Wed, 04 Sep 2024 11:00:46 +0000 By Ankit Mahadevia, chairman of Spero Therapeutics, as part of the From The Trenches feature of LifeSciVC A common theme in startup literature is that by cutting a range of unnecessary tasks, a step-change in results will follow. I’ve found The post Keeping It Simple: What Really Matters For Emerging Enterprises appeared first on LifeSciVC. Full Article Bioentrepreneurship Biotech startup advice Corporate Culture From The Trenches
at Biotech Risk Cycles: Assets And Platforms By lifescivc.com Published On :: Mon, 28 Oct 2024 10:00:52 +0000 Today’s market likes products. Platforms aren’t in vogue anymore. Investors, especially in the public markets, only want late stage de-risked assets. Pharma only seems to be buying these kinds of asset. VCs need to focus on clinical stage companies. Or The post Biotech Risk Cycles: Assets And Platforms appeared first on LifeSciVC. Full Article Biotech financing Biotech investment themes Capital efficiency Capital markets Exits IPOs M&As
at Tell the UK’s research regulator to do more on clinical trial transparency By www.alltrials.net Published On :: Mon, 29 Jul 2019 13:41:09 +0000 The UK body that oversees health research is writing a new strategy on clinical trial transparency and it wants to hear opinions on it. The Health Research Authority (HRA) says its strategy aims to “make transparency easy, make compliance clear and make information public.” It has opened a public consultation on the strategy and some […] Full Article News
at UK universities and NHS trusts that flout the rules on clinical trials identified in report to Parliament By www.alltrials.net Published On :: Thu, 24 Oct 2019 00:19:34 +0000 An AllTrials report for the House of Commons Science and Technology Select Committee this week has found that 33 NHS trust sponsors and six UK universities are reporting none of their clinical trial results, while others have gone from 0% to 100% following an announcement from the Select Committee in January that universities and NHS […] Full Article News Uncategorized
at Pediatric Trial Enrollment (Shameless DIA Self-Promotion, Part 1) By www.placebocontrol.com Published On :: Wed, 19 Jun 2013 22:19:00 +0000 [Fair Warning: I have generally tried to keep this blog separate from my corporate existence, but am making an exception for two quick posts about the upcoming DIA 2013 Annual Meeting.] Improving Enrollment in Pediatric Clinical Trials Logistically, ethically, and emotionally, involving children in medical research is greatly different from the same research in adults. Some of the toughest clinical trials I've worked on, across a number of therapeutic areas, have been pediatric ones. They challenge you to come up with different approaches to introducing and explaining clinical research – approaches that have to work for doctors, kids, and parents simultaneously. On Thursday June 27, Don Sickler, one of my team members, will be chairing a session titled “Parents as Partners: Engaging Caregivers for Pediatric Trials”. It should be a good session. Joining Don are 2 people I've had the pleasure of working with in the past. Both of them combine strong knowledge of clinical research with a massive amount of positive energy and enthusiasm (no doubt a big part of what makes them successful). However, they also differ in one key aspect: what they work on. One of them – Tristen Moors from Hyperion Therapeutics - works on an ultra-rare condition, Urea Cycle Disorder, a disease affecting only a few hundred children every year. On the other hand, Dr. Ann Edmunds is an ENT working in a thriving private practice. I met her because she was consistently the top enroller in a number of trials relating to tympanostomy tube insertion. Surgery to place “t-tubes” is one of the most common and routine outpatients surgeries there is, with an estimated half million kids getting tubes each year. Each presents a special challenge: for rare conditions, how do you even find enough patients? For routine procedures, how do you convince parents to complicate their (and their children’s) lives by signing up for a multi-visit, multi-procedure trial? Ann and Tristen have spent a lot of time tackling these issues, and should have some great advice to give. For more information on the session, here’s Don’s posting on our news blog. Full Article DIA pediatric trials
at Questionable Enrollment Math at the UK's NIHR By www.placebocontrol.com Published On :: Mon, 16 Sep 2013 18:04:00 +0000 There has been considerable noise coming out of the UK lately about successes in clinical trial enrollment. First, a couple months ago came the rather dramatic announcement that clinical trial participation in the UK had "tripled over the last 6 years". That announcement, by the chief executive of the Sweet creature of bombast: is Sir John writing press releases for the NIHR? National Institute of Health Research's Clinical Research Network, was quickly and uncritically picked up by the media. That immediately caught my attention. In large, global trials, most pharmaceutical companies I've worked with can do a reasonable job of predicting accrual levels in a given country. I like to think that if participation rates in any given country had jumped that heavily, I’d have heard something. (To give an example: looking at a quite-typical study I worked on a few years ago: UK sites were overall slightly below the global average. The highest-enrolling countries were about 2.5 times as fast. So, a 3-fold increase in accruals would have catapulted the UK from below average to the fastest-enrolling country in the world.) Further inquiry, however, failed to turn up any evidence that the reported tripling actually corresponded to more human beings enrolled in clinical trials. Instead, there is some reason to believe that all we witnessed was increased reporting of trial participation numbers. Now we have a new source of wonder, and a new giant multiplier coming out of the UK. As the Director of the NIHR's Mental Health Research Network, Til Wykes, put it in her blog coverage of her own paper: Our research on the largest database of UK mental health studies shows that involving just one or two patients in the study team means studies are 4 times more likely to recruit successfully. Again, amazing! And not just a tripling – a quadrupling! Understand: I spend a lot of my time trying to convince study teams to take a more patient-focused approach to clinical trial design and execution. I desperately want to believe this study, and I would love having hard evidence to bring to my clients. At first glance, the data set seems robust. From the King's College press release: Published in the British Journal of Psychiatry, the researchers analysed 374 studies registered with the Mental Health Research Network (MHRN). Studies which included collaboration with service users in designing or running the trial were 1.63 times more likely to recruit to target than studies which only consulted service users. Studies which involved more partnerships - a higher level of Patient and Public Involvement (PPI) - were 4.12 times more likely to recruit to target. But here the first crack appears. It's clear from the paper that the analysis of recruitment success was not based on 374 studies, but rather a much smaller subset of 124 studies. That's not mentioned in either of the above-linked articles. And at this point, we have to stop, set aside our enthusiasm, and read the full paper. And at this point, critical doubts begin to spring up, pretty much everywhere. First and foremost: I don’t know any nice way to say this, but the "4 times more likely" line is, quite clearly, a fiction. What is reported in the paper is a 4.12 odds ratio between "low involvement" studies and "high involvement" studies (more on those terms in just a bit). Odds ratios are often used in reporting differences between groups, but they are unequivocally not the same as "times more likely than". This is not a technical statistical quibble. The authors unfortunately don’t provide the actual success rates for different kinds of studies, but here is a quick example that, given other data they present, is probably reasonably close: A Studies: 16 successful out of 20 Probability of success: 80% Odds of success: 4 to 1 B Studies: 40 successful out of 80 Probability of success: 50% Odds of success: 1 to 1 From the above, it’s reasonable to conclude that A studies are 60% more likely to be successful than B studies (the A studies are 1.6 times as likely to succeed). However, the odds ratio is 4.0, similar to the difference in the paper. It makes no sense to say that A studies are 4 times more likely to succeed than B studies. This is elementary stuff. I’m confident that everyone involved in the conduct and analysis of the MHRN paper knows this already. So why would Dr Wykes write this? I don’t know; it's baffling. Maybe someone with more knowledge of the politics of British medicine can enlighten me. If a pharmaceutical company had promoted a drug with this math, the warning letters and fines would be flying in the door fast. And rightly so. But if a government leader says it, it just gets recycled verbatim. The other part of Dr Wykes's statement is almost equally confusing. She claims that the enrollment benefit occurs when "involving just one or two patients in the study team". However, involving one or two patients would seem to correspond to either the lowest ("patient consultation") or the middle level of reported patient involvement (“researcher initiated collaboration”). In fact, the "high involvement" categories that are supposed to be associated with enrollment success are studies that were either fully designed by patients, or were initiated by patients and researchers equally. So, if there is truly a causal relationship at work here, improving enrollment would not be merely a function of adding a patient or two to the conversation. There are a number of other frustrating aspects of this study as well. It doesn't actually measure patient involvement in any specific research program, but uses just 3 broad categories (that the researchers specified at the beginning of each study). It uses an arbitrary and undocumented 17-point scale to measure "study complexity", which collapses and quite likely underweights many critical factors into a single number. The enrollment analysis excluded 11 studies because they weren't adequate for a factor that was later deemed non-significant. And probably the most frustrating facet of the paper is that the authors share absolutely no descriptive data about the studies involved in the enrollment analysis. It would be completely impossible to attempt to replicate its methods or verify its analysis. Do the authors believe that "Public Involvement" is only good when it’s not focused on their own work? However, my feelings about the study and paper are an insignificant fraction of the frustration I feel about the public portrayal of the data by people who should clearly know better. After all, limited evidence is still evidence, and every study can add something to our knowledge. But the public misrepresentation of the evidence by leaders in the area can only do us harm: it has the potential to actively distort research priorities and funding. Why This Matters We all seem to agree that research is too slow. Low clinical trial enrollment wastes time, money, and the health of patients who need better treatment options. However, what's also clear is that we lack reliable evidence on what activities enable us to accelerate the pace of enrollment without sacrificing quality. If we are serious about improving clinical trial accrual, we owe it to our patients to demand robust evidence for what works and what doesn’t. Relying on weak evidence that we've already solved the problem ("we've tripled enrollment!") or have a method to magically solve it ("PPI quadrupled enrollment!") will cause us to divert significant time, energy, and human health into areas that are politically favored but less than certain to produce benefit. And the overhyping those results by research leadership compounds that problem substantially. NIHR leadership should reconsider its approach to public discussion of its research, and practice what it preaches: critical assessment of the data. [Update Sept. 20: The authors of the study have posted a lengthy comment below. My follow-up is here.] [Image via flikr user Elliot Brown.] Ennis L, & Wykes T (2013). Impact of patient involvement in mental health research: longitudinal study. The British journal of psychiatry : the journal of mental science PMID: 24029538 Full Article NIHR patient recruitment trial delays UK trials
at Questionable Enrollment Math(s) - the Authors Respond By www.placebocontrol.com Published On :: Fri, 20 Sep 2013 04:09:00 +0000 The authors of the study I blogged about on Monday were kind enough to post a lengthy comment, responding in part to some of the issues I raised. I thought their response was interesting, and so reprint it in its entirety below, interjecting my own reactions as well. There were a number of points you made in your blog and the title of questionable maths was what caught our eye and so we reply on facts and provide context. Firstly, this is a UK study where the vast majority of UK clinical trials take place in the NHS. It is about patient involvement in mental health studies - an area where recruitment is difficult because of stigma and discrimination. I agree, in hindsight, that I should have titled the piece “questionable maths” rather than my Americanized “questionable math”. Otherwise, I think this is fine, although I’m not sure that anything here differs from my post. 1. Tripling of studies - You dispute NIHR figures recorded on a national database and support your claim with a lone anecdote - hardly data that provides confidence. The reason we can improve recruitment is that NIHR has a Clinical Research Network which provides extra staff, within the NHS, to support high quality clinical studies and has improved recruitment success. To be clear, I did not “dispute” the figures so much as I expressed sincere doubt that those figures correspond with an actual increase in actual patients consenting to participate in actual UK studies. The anecdote explains why I am skeptical – it's a bit like I've been told there was a magnitude 8 earthquake in Chicago, but neither I nor any of my neighbors felt anything. There are many reasons why reported numbers can increase in the absence of an actual increase. It’s worth noting that my lack of confidence in the NIHR's claims appears to be shared by the 2 UK-based experts quoted by Applied Clinical Trials in the article I linked to. 2. Large database: We have the largest database of detailed study information and patient involvement data - I have trawled the world for a bigger one and NIMH say there certainly isn't one in the USA. This means few places where patient impact can actually be measured 3. Number of studies: The database has 374 studies which showed among other results that service user involvement increased over time probably following changes by funders e.g. NIHR requests information in the grant proposal on how service users have been and will be involved - one of the few national funders to take this issue seriously. As far as I can tell, neither of these points is in dispute. 4. Analysis of patient involvement involves the 124 studies that have completed. You cannot analyse recruitment success unless then. I agree you cannot analyze recruitment success in studies that have not yet completed. My objection is that in both the KCL press release and the NIHR-authored Guardian article, the only number mentioned in 374, and references to the recruitment success findings came immediately after references to that number. For example: Published in the British Journal of Psychiatry, the researchers analysed 374 studies registered with the Mental Health Research Network (MHRN). Studies which included collaboration with service users in designing or running the trial were 1.63 times more likely to recruit to target than studies which only consulted service users. Studies which involved more partnerships - a higher level of Patient and Public Involvement (PPI) - were 4.12 times more likely to recruit to target. The above quote clearly implies that the recruitment conclusions were based on an analysis of 374 studies – a sample 3 times larger than the sample actually used. I find this disheartening. The complexity measure was developed following a Delphi exercise with clinicians, clinical academics and study delivery staff to include variables likely to be barriers to recruitment. It predicts delivery difficulty (meeting recruitment & delivery staff time). But of course you know all that as it was in the paper. Yes, I did know this, and yes, I know it because it was in the paper. In fact, that’s all I know about this measure, which is what led me to characterize it as “arbitrary and undocumented”. To believe that all aspects of protocol complexity that might negatively affect enrollment have been adequately captured and weighted in a single 17-point scale requires a leap of faith that I am not, at the moment, able to make. The extraordinary claim that all complexity issues have been accounted for in this model requires extraordinary evidence, and “we conducted a Delphi exercise” does not suffice. 6. All studies funded by NIHR partners were included – we only excluded studies funded without peer review, not won competitively. For the involvement analysis we excluded industry studies because of not being able to contact end users and where inclusion compromised our analysis reliability due to small group sizes. It’s only that last bit I was concerned about. Specifically, the 11 studies that were excluded due to being in “clinical groups” that were too small, despite the fact that “clinical groups” appear to have been excluded as non-significant from the final model of recruitment success. (Also: am I being whooshed here? In a discussion of "questionable math" the authors' enumeration goes from 4 to 6. I’m going to take the miscounting here as a sly attempt to see if I’m paying attention...) I am sure you are aware of the high standing of the journal and its robust peer review. We understand that our results must withstand the scrutiny of other scientists but many of your comments were unwarranted. This is the first in the world to investigate patient involvement impact. No other databases apart from the one held by the NIHR Mental Health Research Network is available to test – we only wish they were. I hope we can agree that peer review – no matter how "high standing" the journal – is not a shield against concern and criticism. Despite the length of your response, I’m still at a loss as to which of my comments specifically were unwarranted. In fact, I feel that I noted very clearly that my concerns about the study’s limitations were minuscule compared to my concerns about the extremely inaccurate way that the study has been publicized by the authors, KCL, and the NIHR. Even if I conceded every possible criticism of the study itself, there remains the fact that in public statements, you Misstated an odds ratio of 4 as “4 times more likely to” Overstated the recruitment success findings as being based on a sample 3 times larger than it actually was Re-interpreted, without reservation, a statistical association as a causal relationship Misstated the difference between the patient involvement categories as being a matter of merely “involving just one or two patients in the study team” And you did these consistently and repeatedly – in Dr Wykes's blog post, in the KCL press release, and in the NIHR-written Guardian article. To use the analogy from my previous post: if a pharmaceutical company had committed these acts in public statements about a new drug, public criticism would have been loud and swift. Your comment on the media coverage of odds ratios is an issue that scientists need to overcome (there is even a section in Wikipedia). It's highly unfair to blame "media coverage" for the use of an odds ratio as if it were a relative risk ratio. In fact, the first instance of "4 times more likely" appears in Dr Wykes's own blog post. It's repeated in the KCL press release, so you yourselves appear to have been the source of the error. You point out the base rate issue but of course in a logistic regression you also take into account all the other variables that may impinge on the outcome prior to assessing the effects of our key variable patient involvement - as we did – and showed that the odds ratio is 4.12 - So no dispute about that. We have followed up our analysis to produce a statement that the public will understand. Using the following equations: Model predicted recruitment lowest level of involvement exp(2.489-.193*8.8-1.477)/(1+exp(2.489-.193*8.8-1.477))=0.33 Model predicted recruitment highest level of involvement exp(2.489-.193*8.8-1.477+1.415)/(1+exp(2.489-.193*8.8-1.477+1.415)=0.67 For a study of typical complexity without a follow up increasing involvement from the lowest to the highest levels increased recruitment from 33% to 66% i.e. a doubling. So then, you agree that your prior use of “4 times more likely” was not true? Would you be willing to concede that in more or less direct English? This is important and is the first time that impact has been shown for patient involvement on the study success. Luckily in the UK we have a network that now supports clinicians to be involved and a system for ensuring study feasibility. The addition of patient involvement is the additional bonus that allows recruitment to increase over time and so cutting down the time for treatments to get to patients. No, and no again. This study shows an association in a model. The gap between that and a causal relationship is far too vast to gloss over in this manner. In summary, I thank the authors for taking the time to response, but I feel they've overreacted to my concerns about the study, and seriously underreacted to my more important concerns about their public overhyping of the study. I believe this study provides useful, though limited, data about the potential relationship between patient engagement and enrollment success. On the other hand, I believe the public positioning of the study by its authors and their institutions has been exaggerated and distorted in clearly unacceptable ways. I would ask the authors to seriously consider issuing public corrections on the 4 points listed above. Full Article NIHR patient recruitment trial delays UK trials
at Patient Recruitment: Taking the Low Road By www.placebocontrol.com Published On :: Thu, 19 Dec 2013 22:36:00 +0000 The Wall Street Journal has an interesting article on the use of “Big Data” to identify and solicit potential clinical trial participants. The premise is that large consumer data aggregators like Experian can target patients with certain diseases through correlations with non-health behavior. Examples given include “a preference for jazz” being associated with arthritis and “shopping online for clothes” being an indicator of obesity. We've seen this story before. In this way, allegedly, clinical trial patient recruitment companies can more narrowly target their solicitations* for patients to enroll in clinical trials. In the spirit of full disclosure, I should mention that I was interviewed by the reporter of this article, although I am not quoted. My comments generally ran along three lines, none of which really fit in with the main storyline of the article: I am highly skeptical that these analyses are actually effective at locating patients These methods aren't really new – they’re the same tactics that direct marketers have been using for years Most importantly, the clinical trials community can – and should – be moving towards open and collaborative patient engagement. Relying on tactics like consumer data snooping and telemarketing is an enormous step backwards. The first point is this: certainly some diseases have correlates in the real world, but these correlates tend to be pretty weak, and are therefore unreliable predictors of disease. Maybe it’s true that those struggling with obesity tend to buy more clothes online (I don’t know if it’s true or not – honestly it sounds a bit more like an association built on easy stereotypes than on hard data). But many obese people will not shop online (they will want to be sure the clothes actually fit), and vast numbers of people with low or average BMIs will shop for clothes online. So the consumer data will tend to have very low predictive value. The claims that liking jazz and owning cats are predictive of having arthritis are even more tenuous. These correlates are going to be several times weaker than basic demographic information like age and gender. And for more complex conditions, these associations fall apart. Marketers claim to solve this by factoring a complex web of associations through a magical black box – th WSJ article mentions that they “applied a computed algorithm” to flag patients. Having seen behind the curtain on a few of these magic algorithms, I can confidently say that they are underwhelming in their sophistication. Hand-wavy references to Big Data and Algorithms are just the tools used to impress pharma clients. (The down side to that, of course, is that you can’t help but come across as big brotherish – see this coverage from Forbes for a taste of what happens when people accept these claims uncritically.) But the effectiveness of these data slice-n-dicing activities is perhaps beside the point. They are really just a thin cover for old-fashioned boiler room tactics: direct mail and telemarketing. When I got my first introduction to direct marketing in the 90’s, it was the exact same program – get lead lists from big companies like Experian, then aggressively mail and call until you get a response. The limited effectiveness and old-school aggressiveness of these programs comes is nicely illustrated in the article by one person’s experience: Larna Godsey, of Wichita, Kan., says she received a dozen phone calls about a diabetes drug study over the past year from a company that didn't identify itself. Ms. Godsey, 63, doesn't suffer from the disease, but she has researched it on the Internet and donated to diabetes-related causes. "I don't know if it's just a coincidence or if they're somehow getting my information," says Ms. Godsey, who filed a complaint with the FTC this year. The article notes that one recruitment company, Acurian, has been the subject of over 500 FTC complaints regarding its tactics. It’s clear that Big Data is just the latest buzzword lipstick on the telemarketing pig. And that’s the real shame of it. We have arrived at an unprecedented opportunity for patients, researchers, and private industry to come together and discuss, as equals, research priorities and goals. Online patient communities like Inspire and PatientsLikeMe have created new mechanisms to share clinical trial opportunities and even create new studies. Dedicated disease advocates have jumped right into the world of clinical research, with groups like the Cystic Fibrosis Foundation and Michael J. Fox Foundation no longer content with raising research funds, but actively leading the design and operations of new studies. Some – not yet enough – pharmaceutical companies have embraced the opportunity to work more openly and honestly with patient groups. The scandal of stories like this is not the Wizard of Oz histrionics of secret computer algorithms, but that we as an industry continue to take the low road and resort to questionable boiler room tactics. It’s past time for the entire patient recruitment industry to drop the sleaze and move into the 21st century. I would hope that patient groups and researchers will come together as well to vigorously oppose these kinds of tactics when they encounter them. (*According to the article, Acurian "has said that calls related to medical studies aren't advertisements as defined by law," so we can agree to call them "solicitations".) Full Article Big Data CFF direct mail direct to patient ethics Inspire MJFF patient engagement patient recruitment PatientsLikeMe telemarketing
at These Words Have (Temporarily) Relocated By www.placebocontrol.com Published On :: Tue, 18 Mar 2014 14:17:00 +0000 Near the end of last year, I had the bright idea of starting a second blog, Placebo Lead-In, to capture a lot of smaller items that I found interesting but wasn't going to work up into a full-blown, 1000 word post. According to Murphy’s Law, or the Law of Unintended Consequences, or the Law of Biting Off More Than You Can Chew, or some such similar iron rule of the universe, what happened next should have been predictable. First, my team at CAHG Trials launched a new blog, First Patient In. FPI is dedicated to an open discussion of patient recruitment ideas, and I’m extremely proud of what we've published so far. Next, I was invited to be a guest blogger for the upcoming Partnerships in Clinical Trials Conference. Suddenly, I've gone from 1 blog to 4. And while my writing output appears to have increased, it definitely hasn't quadrupled. So this blog has been quiet for a bit too long as a result. The good news is that the situation is temporary - Partnerships will actually happen at the end of this month. (If you’re going: drop me a line and let’s meet. If you’re not: you really should come and join us!) My contributions to FPI will settle into a monthly post, as I have a fascinating and clever team to handle most of the content. In case you've missed it, then, here is a brief summary of my posts elsewhere over the past 2 months. First Patient In How to Catalyze a Clinical Trial - My inaugural post introducing the blog and its purpose Video: Predicting Referral Conversion in Clinical Trial Advertising - A somewhat technical but very important topic, how to visualize and model the “real time” results of recruitment advertising at the sites. The Crystal Ball is on the Fritz - What to do with a broken enrollment feasibility process, and how asking will never be as good as measuring Partnerships in Clinical Trials The New Breed of Clinical Trial Matchmakers - A (hopefully pretty complete, thanks to knowledgeable commenters) listing of services looking to match interested patients to clinical trials Rethinking Patient Enrollment, in One Graphic - The perils of predictability in site-based enrollment Seize the Data! Will Big Data Save Us from Ourselves? - My take on what I consider to be the large and serious obstacles in the way of “Big Data” solutions for patient recruitment Please take a look, and I will see you back here soon. [Photo credit: detour sign via Flikr user crossley] Full Article Big Data metrics patient engagement patient recruitment site relationship management
at Patient Centered Trials - Your Thoughts Needed By www.placebocontrol.com Published On :: Thu, 31 Jul 2014 17:43:00 +0000 The good folks down at eyeforpharma have asked me to write a few blog posts in the run-up to their Patient Centered Clinical Trials conference in Boston this September. In my second article -Buzzword Innovation: The Patient Centricity “Fad” and the Token Patient - I went over some concerns I have regarding the sudden burst of enthusiasm for patient centricity in the clinical trial world. Apparently, that hit a nerve – in an email, Ulrich Neumann tells me that “your last post elicited quite a few responses in my inbox (varied, some denouncing it as a fad, others strongly protesting the notion, hailing it as the future).” In preparing my follow up post, I’ve spoken to a couple people on the leading edge of patient engagement: Abbe Steel, CEO of HealthiVibe, which is focused on bringing greater patient input into the earliest stages of trial design through focus groups and patient surveys Casey Quinlan, co-founder of Patients for Clinical Research, which aims to be a force in patient education and engagement for clinical trials In addition to their thoughts, eyeforpharma is keenly interested in hearing from more people. They've even posted a survey – from Ulrich: To get a better idea of what other folks think of the idea, I am sending out a little ad hoc survey. Only 4 questions (so people hopefully do it). Added benefit: There is a massive 50% one-time discount for completed surveys until Friday connected to it as an incentive). So, here are two things for you to do: Complete the survey and share your thoughts Come to the conference and tell us all exactly what you think Look forward to seeing you there. [Conflict of Interest Disclosure: I am attending the Patient Centered Clinical Trials conference. Having everyone saying the same thing at such conferences conflicts with my ability to find them interesting.] Full Article eyeforpharma HealthiVibe patient engagement PFCR
at Jerry Matczak By www.placebocontrol.com Published On :: Tue, 07 Feb 2017 17:16:00 +0000 Jerry Matczak passed away suddenly last Thursday at the much-too-young age of 54. I can say, without exaggeration, that Jerry embodied pretty much everything I aspire to be in my professional life. The MedCityNews headline called him a “social media guru”, but in reality he was temperamentally the exact opposite of a "guru": He was constantly curious; it seemed that every conversation I had with him was composed mainly of questions. Many of us try to be “listen first, talk second” types, but Jerry was a “listen first, ask questions, listen some more, then talk” type. He also never stopped trying to figure out how to improve whatever he was working on. He participated in a lot of pilot projects, which means he was a part of a lot of projects that didn’t meet their objectives – but I never witnessed Jerry being the least bit negative or frustrated. Every project was just another opportunity to learn more. Mostly, though, Jerry was remarkable in his ability to connect with patients, even patients who were deeply distrustful of his employer and industry. If nothing else, I hope you read the words of two such patients, coming from very different places, with remarkably similar reactions to Jerry: What Would Jerry Do? (by ALS Advocacy) Patients, Pharma, Partners (by AfternoonNapper) Jerry, thank you for your service and your example. I carry it with me. Angela @radclipatra & I rocking our #WalkingGallery of Healthcare jackets. Learn more https://t.co/uFZHW81Cts #SCOPE2017 @ReginaHolliday pic.twitter.com/6kamYOW2VZ — Jerry Matczak (@gmatczak) January 25, 2017 Full Article
at For good sleep and good health, regulate your exposure to light By www.npr.org Published On :: Sat, 09 Nov 2024 07:58:39 -0500 Your daily light exposure impacts your health. A new study finds that too much light at night and not enough natural light during the day can be harmful. This story first aired on Morning Edition on Nov. 4, 2024. Full Article
at What does a 2nd Trump term mean for the Affordable Care Act? By www.npr.org Published On :: Mon, 11 Nov 2024 04:47:01 -0500 President-elect Donald Trump tried unsuccessfully to get rid of the Affordable Care Act during his first term. What action will he take this time around? Full Article
at Patrick Dempsey aims to raise awareness of cancer disparities and encourage screening By www.npr.org Published On :: Mon, 11 Nov 2024 05:18:50 -0500 NPR's Leila Fadel talks with actor Patrick Dempsey about his efforts to raise money for cancer treatment and prevention. Full Article
at Chronic itch is miserable. Scientists are just scratching the surface By www.npr.org Published On :: Tue, 12 Nov 2024 13:33:46 -0500 Journalist Annie Lowrey has a rare disease that causes a near-constant itch that doesn't respond to most treatments. She likens the itchiness to a car alarm: "You can't stop thinking about it." Full Article
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