Note to readers: After a period of inactivity CardioBrief is coming back, but with some big differences. This website, CardioBrief.Org, will remain my personal website. A new website, CVCTCardiobrief.com, will be the new home for my “professional” blogging activities. To develop this website I have joined forces with the global CVCT Forum. I look forward to...

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It is often said that medicine is both an art and a science. In an imperfect world this is both inevitable and desirable. But it is extremely important that the two should not be confused with each other. In particular, because the “science” side of the equation has achieved overwhelming prestige and authority, it is...

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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…...

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At this moment in time the pre-pandemic cardiology research agenda needs to be completely reprioritized. There are two broad areas that now take precedence over all existing research concerns. On the one hand, researchers need to achieve a better understanding of the staggering incidence of deferred or delayed treatment of cardiovascular events and conditions as...

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<p>On 19&nbsp;September 2024, the European Medicines Agency’s (EMA) Committee for Medicinal Products for Human Use (CHMP)&nbsp;adopted a positive opinion,&nbsp;recommending the granting of marketing authorization&nbsp;for&nbsp;two aflibercept biosimilars:&nbsp;&nbsp;Sandoz’s Afqlir and Samsung Bioepis’s Opuviz.&nbsp;These products are biosimilars of the reference product Eylea, developed by Regeneron and Bayer.</p>

<p>The US Food and Drug Administration (FDA) granted approval for two&nbsp;biosimilars, Formycon’s FYB202/Otulfi (ustekinumab-aauz) and Samsung Bioepis’ Soliris biosimilar, Epysqli (eculizumab-aagh), on 27 September and 22 July 2024, respectively. FYB202/Otulfi, a biosimilar referencing&nbsp;Johnson &amp; Johnson’s Stelara, while Epysqli is a biosimilar referencing Alexion’s Soliris.</p>

<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&nbsp;|&nbsp;</b><b><a href="https://youtu.be/X6-dYZ7fjhM" target="_blank">WATCH REPLAY</a></b></p>

<p>In May 2024, Malaysia’s National Pharmaceutical Regulatory Agency (NPRA) announced that it will trial new timelines for variation applications&nbsp;of registered pharmaceutical products and natural health supplements (TMHS).</p>

<p>In October 2024, China based Bio-Thera Solutions&nbsp;(Bio-Thera)&nbsp;and Hungary’s Gedeon Richter announced they have reached an exclusive commercialization and license agreement for BAT2206, a biosimilar candidate to&nbsp;Johnson &amp; Johnson’s Stelara (ustekinumab).</p>

<p>The European Commission (EC) granted marketing authorization for<b>&nbsp;</b>three ustekinumab biosimilars<b>:&nbsp;</b>Samsung Bioepis’ Eksunbi on 12 September 2024; Formycon’s Fymskina, and Fresenius Kabi’s&nbsp;Otulfi on 25 September 2024.</p>

<p> <b>Abstract</b><br />CinnaGen, the largest biopharmaceutical company in the MENA region, is a leader in developing follow-on biologicals/biosimilars. Dr&nbsp;Haleh Hamedifar, Chairperson of CinnaGen, spoke to GaBI<i>&nbsp;</i>(Generics and Biosimilars Initiative) about the company’s strategic focus, which includes expanding its product portfolio, entering highly regulated global markets, and advancing affordable treatments for conditions such as multiple sclerosis and&nbsp;immunological diseases—transforming healthcare in underserved regions.</p><p><b>Keywords</b>: Biosimilars, clinical development, commercialization, MENA</p>

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.

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.

The Pharmaceutical Services Negotiating Committee is in talks with the government over potential plans to distribute COVID-19 treatments in primary care.

Community pharmacies should be reimbursed for their additional costs during the COVID-19 pandemic “as soon as possible”, the prime minister has told The Pharmaceutical Journal.

Starting COVID-19 patients on prophylactic anticoagulation within 24 hours of being admitted to hospital has been linked to a reduced risk of mortality.

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.

The type 2 diabetes mellitus drug semaglutide is effective for weight loss in non-diabetic overweight or obese adults, when taken alongside a reduced-calorie diet and exercise, researchers have found.

Community pharmacies will receive an estimated 12 referrals from the Discharge Medicines Service per year.

Around half of Wales’ community pharmacies have expressed interest to health boards in providing COVID-19 vaccinations as part of the national programme.

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.

Pharmacy negotiators have discussed proposals to take “a patient registration-based approach” to the community pharmacy contractual framework.

The Royal Pharmaceutical Society has expressed its sadness at the death of Joy Wingfield, honorary professor of Pharmacy Law and Ethics at the University of Nottingham.

By Haojing Rong and Aimee Raleigh, as part of the From The Trenches feature of LifeSciVC This blog post is the second in a series on key diligence concepts and questions. If you missed the intro blog post yesterday, click

The post Pharmacology: The Anchor for Nearly Every Diligence appeared first on LifeSciVC.

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.

By Arthur O. Tzianabos, PhD, CEO of Lifordi Immunotherapeutics, as part of the From the Trenches feature of LifeSciVC As the biotech industry continues to pick up steam, I have been getting a number of phone calls from folks in

The post Stars and Scars… Some Lessons Learned About Leadership appeared first on LifeSciVC.

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.

Having just turned 50, I’ve been reflecting on my first half-century of late… many fun and impactful moments, a few regrets, and a life I’ve tried to live to the fullest. One thread that has run throughout it has been

The post A Molecular Biologist’s Advice For Life appeared first on LifeSciVC.

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.

By Aimee Raleigh, Principal at Atlas Venture, as part of the From The Trenches feature of LifeSciVC From the outside, one might assume all biotech venture capital (VC) firms are more similar than different. However, once you look under the

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By Peter Tummino, CSO of Nimbus Therapeutics, as part of the From The Trenches feature of LifeSciVC  “Over the next five to 10 years, our goal is to become a company that’s leading the world in personalized medicines, a company

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By Ivana Magovčević-Liebisch, CEO of Vigil Neuroscience, as part of the From The Trenches feature of LifeSciVC In an industry where boom and bust cycles occur regularly and 90 percent of drug candidates fail to reach the market, an outstanding

The post Reflections On My Experience As A Board Member appeared first on LifeSciVC.

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 […]

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 […]

New research has shown that the majority of clinical trials which should be following the US law on reporting results aren’t. Less than half (41%) of clinical trial results were reported on time and 1 in 3 trials (36%) remain unreported. The research also found that clinical trials sponsored by companies are the most likely […]

PDUFA V commitments signal a strong commitment to tolerance of open debate in the face of uncertainty.

I can admit to a rather powerful lack of enthusiasm when reading about interpersonal squabbles. It’s even worse in the scientific world: when I read about debates getting mired in personal attacks I tend to simply stop reading and move on to something else.

However, the really interesting part of this week’s meeting of an FDA joint Advisory Committee to discuss the controversial diabetes drug Avandia – at least in the sense of likely long-term impact – is not the scientific question under discussion, but the surfacing and handling of the raging interpersonal battle going on right now inside the Division of Cardiovascular and Renal Products. So I'll have to swallow my distaste and follow along with the drama.

Two words that make us mistrust Duke:  Anil Potti Christian Laettner

Not that the scientific question at hand – does Avandia pose significant heart risks? – isn't interesting. It is. But if there’s one thing that everyone seems to agree on, it’s that we don’t have good data on the topic. Despite the re-adjudication of RECORD, no one trusts its design (and, ironically, the one trial with a design to rigorously answer the question was halted after intense pressure, despite an AdComm recommendation that it continue).  And no one seems particularly enthused about changing the current status of Avandia: in all likelihood it will continue to be permitted to be marketed under heavy restrictions. Rather than changing the future of diabetes, I suspect the committee will be content to let us slog along the same mucky trail.

The really interesting question, that will potentially impact CDER for years to come, is how it can function with frothing, open dissent among its staffers. As has been widely reported, FDA reviewer Tom Marciniak has written a rather wild and vitriolic assessment of the RECORD trial, excoriating most everyone involved. In a particularly stunning passage, Marciniak appears to claim that the entire output of anyone working at Duke University cannot be trusted because of the fraud committed by Duke cancer researcher Anil Potti:

I would have thought that the two words “Anil Potti” are sufficient for convincing anyone that Duke University is a poor choice for a contractor whose task it is to confirm the integrity of scientific research. 

(One wonders how far Marciniak is willing to take his guilt-by-association theme. Are the words “Cheng Yi Liang” sufficient to convince us that all FDA employees, including Marciniak, are poor choices for deciding matter relating to publicly-traded companies? Should I not comment on government activities because I’m a resident of Illinois (my two words: “Rod Blagojevich”)?)

Rather than censoring or reprimanding Marciniak, his supervisors have taken the extraordinary step of letting him publicly air his criticisms, and then they have in turn publicly criticized his methods and approach.

I have been unable to think of a similar situation at any regulatory agency. The tolerance for dissent being displayed by FDA is, I believe, completely unprecedented.

And that’s the cliffhanger for me: can the FDA’s commitment to transparency extend so far as to accommodate public disagreements about its own approval decisions? Can it do so even when the disagreements take an extremely nasty and inappropriate tone?

• Rather than considering that open debate is a good thing, will journalists jump on the drama and portray agency leadership as weak and indecisive?
• Will the usual suspects in Congress be able to exploit this disagreement for their own political gain? How many House subcommittees will be summoning Janet Woodcock in the coming weeks?

I think what Bob Temple and Norman Stockbridge are doing is a tremendous experiment in open government. If they can pull it off, it could force other agencies to radically rethink how they go about crafting and implementing regulations. However, I also worry that it is politically simply not a viable approach, and that the agency will ultimately be seriously hurt by attacks from the media and legislators.

Where is this coming from?

As part of its recent PDUFA V commitment, the FDA put out a fascinating draft document, Structured Approach to Benefit-Risk Assessment in Drug Regulatory Decision-Making. It didn't get a lot of attention when first published back in February (few FDA documents do). However, it lays out a rather bold vision for how the FDA can acknowledge the existence of uncertainty in its evaluation of new drugs. Its proposed structure even envisions an open and honest accounting of divergent interpretations of data:

When they're frothing at the mouth, even Atticus doesn't let them publish a review

A framework for benefit-risk decision-making that summarizes the relevant facts, uncertainties, and key areas of judgment, and clearly explains how these factors influence a regulatory decision, can greatly inform and clarify the regulatory discussion. Such a framework can provide transparency regarding the basis of conflicting recommendations made by different parties using the same information.

(Emphasis mine.)

Of course, the structured framework here is designed to reflect rational disagreement. Marciniak’s scattershot insults are in many ways a terrible first case for trying out a new level of transparency.

The draft framework notes that safety issues, like Avandia, are some of the major areas of uncertainty in the regulatory process. Contrast this vision of coolly and systematically addressing uncertainties with the sad reality of Marciniak’s attack:

In contrast to the prospective and highly planned studies of effectiveness, safety findings emerge from a wide range of sources, including spontaneous adverse event reports, epidemiology studies, meta-analyses of controlled trials, or in some cases from randomized, controlled trials. However, even controlled trials, where the evidence of an effect is generally most persuasive, can sometimes provide contradictory and inconsistent findings on safety as the analyses are in many cases not planned and often reflect multiple testing. A systematic approach that specifies the sources of evidence, the strength of each piece of evidence, and draws conclusions that explain how the uncertainty weighed on the decision, can lead to more explicit communication of regulatory decisions. We anticipate that this work will continue beyond FY 2013.

I hope that work will continue beyond 2013. Thoughtful, open discussions of real uncertainties are one of the most worthwhile goals FDA can aspire to, even if it means having to learn how to do so without letting the Marciniaks of the world scuttle the whole endeavor.

[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.

Are we looking at our enrollment data in the right way?

Session Details:

June 25, 1:45PM - 3:15PM

• Session Number: 241
• Room Number: 205B

1. Enrollment Analytics: Moving Beyond the Funnel
Paul Ivsin
VP, Consulting Director
CAHG Clinical Trials

2. Use of Analytics for Operational Planning
Diana Chung, MSc
Associate Director, Clinical Operations
Gilead
3. Using Enrollment Data to Communicate Effectively with Sites
Gretchen Goller, MA
Senior Director, Patient Access and Retention Services
PRA

Results reporting requirements are pretty clear. Maybe critics should re-check their methods?

Ben Goldacre has rather famously described the clinical trial reporting requirements in the Food and Drug Administration Amendments Act of 2007 as a “fake fix” that was being thoroughly “ignored” by the pharmaceutical industry.

Pharma: breaking the law in broad daylight?

He makes this sweeping, unconditional proclamation about the industry and its regulators on the basis of  a single study in the BMJ, blithely ignoring the fact that a) the authors of the study admitted that they could not adequately determine the number of studies that were meeting FDAAA requirements and b) a subsequent FDA review that identified only 15 trials potentially out of compliance, out of a pool of thousands.


Despite the fact that the FDA, which has access to more data, says that only a tiny fraction of studies are potentially noncompliant, Goldacre's frequently repeated claims that the law is being ignored seems to have caught on in the general run of journalistic and academic discussions about FDAAA.

And now there appears to be additional support for the idea that a large percentage of studies are noncompliant with FDAAA results reporting requirements, in the form of a new study in the Journal of Clinical Oncology: "Public Availability of Results of Trials Assessing Cancer Drugs in the United States" by Thi-Anh-Hoa Nguyen, et al.. In it, the authors report even lower levels of FDAAA compliance – a mere 20% of randomized clinical trials met requirements of posting results on clinicaltrials.gov within one year.

Unsurprisingly, the JCO results were immediately picked up and circulated uncritically by the usual suspects.

I have to admit not knowing much about pure academic and cooperative group trial operations, but I do know a lot about industry-run trials – simply put, I find the data as presented in the JCO study impossible to believe. Everyone I work with in pharma trials is painfully aware of the regulatory environment they work in. FDAAA compliance is a given, a no-brainer: large internal legal and compliance teams are everywhere, ensuring that the letter of the law is followed in clinical trial conduct. If anything, pharma sponsors are twitchily over-compliant with these kinds of regulations (for example, most still adhere to 100% verification of source documentation – sending monitors to physically examine every single record of every single enrolled patient - even after the FDA explicitly told them they didn't have to).

I realize that’s anecdotal evidence, but when such behavior is so pervasive, it’s difficult to buy into data that says it’s not happening at all. The idea that all pharmaceutical companies are ignoring a highly visible law that’s been on the books for 6 years is extraordinary. Are they really so brazenly breaking the rules? And is FDA abetting them by disseminating incorrect information?

Those are extraordinary claims, and would seem to require extraordinary evidence. The BMJ study had clear limitations that make its implications entirely unclear. Is the JCO article any better?

Some Issues

In fact, there appear to be at least two major issues that may have seriously compromised the JCO findings:

1. Studies that were certified as being eligible for delayed reporting requirements, but do not have their certification date listed.

The study authors make what I believe to be a completely unwarranted assumption:

In trials for approval of new drugs or approval for a new indication, a certification [permitting delayed results reporting] should be posted within 1 year and should be publicly available.

It’s unclear to me why the authors think the certifications “should be” publicly available. In re-reading FDAAA section 801, I don’t see any reference to that being a requirement. I suppose I could have missed it, but the authors provide a citation to a page that clearly does not list any such requirement.

But their methodology assumes that all trials that have a certification will have it posted:

If no results were posted at ClinicalTrials.gov, we determined whether the responsible party submitted a certification. In this case, we recorded the date of submission of the certification to ClinicalTrials.gov.

If a sponsor gets approval from FDA to delay reporting (as is routine for all drugs that are either not approved for any indication, or being studied for a new indication – i.e., the overwhelming majority of pharma drug trials), but doesn't post that approval on the registry, the JCO authors deem that trial “noncompliant”. This is not warranted: the company may have simply chosen not to post the certification despite being entirely FDAAA compliant.

2. Studies that were previously certified for delayed reporting and subsequently reported results

It is hard to tell how the authors treated this rather-substantial category of trials. If a trial was certified for delayed results reporting, but then subsequently published results, the certification date becomes difficult to find. Indeed, it appears in the case where there were results, the authors simply looked at the time from study completion to results posting. In effect, this would re-classify almost every single one of these trials from compliant to non-compliant. Consider this example trial:

• Phase 3 trial completes January 2010
• Certification of delayed results obtained December 2010 (compliant)
• FDA approval June 2013
• Results posted July 2013 (compliant)

In looking at the JCO paper's methods section, it really appears that this trial would be classified as reporting results 3.5 years after completion, and therefore be considered noncompliant with FDAAA. In fact, this trial is entirely kosher, and would be extremely typical for many phase 2 and 3 trials in industry.

Time for Some Data Transparency

The above two concerns may, in fact, be non-issues. They certainly appear to be implied in the JCO paper, but the wording isn't terribly detailed and could easily be giving me the wrong impression.

However, if either or both of these issues are real, they may affect the vast majority of "noncompliant" trials in this study. Given the fact that most clinical trials are either looking at new drugs, or looking at new indications for new drugs, these two issues may entirely explain the gap between the JCO study and the unequivocal FDA statements that contradict it.

I hope that, given the importance of transparency in research, the authors will be willing to post their data set publicly so that others can review their assumptions and independently verify their conclusions. It would be more than a bit ironic otherwise.
Thi-Anh-Hoa Nguyen, Agnes Dechartres, Soraya Belgherbi, and Philippe Ravaud (2013). Public Availability of Results of Trials Assessing Cancer Drugs in the United States JOURNAL OF CLINICAL ONCOLOGY DOI: 10.1200/JCO.2012.46.9577

• 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

• 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]

But while we can all agree that "patient engagement is good" in a highly general sense, we don't have much consensus on what the implications of that idea might be. There is precious little hard evidence about how to either attract engaged patients, or how we might effectively turn "regular patients" into "engaged patients".

On this date 349 years ago, Samuel Pepys relates in his famous diary a remarkable story about an upcoming medical experiment. As far as I can tell, this is the first written description of a paid research subject.

According to his account, the man (who he describes as “a little frantic”) was to be paid to undergo a blood transfusion from a sheep. It was hypothesized that the blood of this calm and docile animal would help to calm the man.

Some interesting things to note about this experiment:

• Equipoise. There is explicit disagreement about what effect the experimental treatment will have: according to Pepys, "some think it may have a good effect upon him as a frantic man by cooling his blood, others that it will not have any effect at all".
• Results published. An account of the experiment was published just two weeks later in the journal Philosophical Transactions. 
• Medical Privacy. In this subsequent write-up, the research subject is identified as Arthur Coga, a former Cambridge divinity student. According to at least one account, being publicly identified had a bad effect on Coga, as people who had heard of him allegedly succeeded in getting him to spend his stipend on drink (though no sources are provided to confirm this story).
• Patient Reported Outcome. Coga was apparently chosen because, although mentally ill, he was still considered educated enough to give an accurate description of the treatment effect. 

Depending on your perspective, this may also be a very early account of the placebo effect, or a classic case of ignoring the patient’s experience. Because even though his report was positive, the clinicians remained skeptical. From the journal article:

The Man after this operation, as well as in it, found himself very well, and hath given in his own Narrative under his own hand, enlarging more upon the benefit, he thinks, he hath received by it, than we think fit to own as yet.

…and in fact, a subsequent diary entry from Pepys mentions meeting Coga, with similarly mixed impressions: “he finds himself much better since, and as a new man, but he is cracked a little in his head”.

The amount Coga was paid for his participation? Twenty shillings – at the time, that was exactly one Guinea.

[Image credit: Wellcome Images]

Maybe those pesky sites are good for something after all. 

It's been six years since Pfizer boldly announced the launch of its "clinical trial in a box". The REMOTE trial was designed to be entirely online, and involved no research sites: study information and consent was delivered via the web, and medications and diaries were shipped directly to patients' homes.

Despite the initial fanfare, within a month REMOTE's registration on ClinicalTrials.gov was quietly reduced from 600 to 283. The smaller trial ended not with a bang but a whimper, having randomized only 18 patients in over a year of recruiting.

Still, the allure of direct to patient clinical trials remains strong, due to a confluence of two factors. First, a frenzy of interest in running "patient centric clinical trials". Sponsors are scrambling to show they are doing something – anything – to show they have shifted to a patient-centered mindset. We cannot seem to agree what this means (as a great illustration of this, a recent article in Forbes on "How Patients Are Changing Clinical Trials" contained no specific examples of actual trials that had been changed by patients), but running a trial that directly engages patients wherever they are seems like it could work.

The less-openly-discussed other factor leading to interest in these DIY trials is sponsors' continuing willingness to heap almost all of the blame for slow-moving studies onto their research sites. If it’s all the sites’ fault – the reasoning goes – then cutting them out of the process should result in trials that are both faster and cheaper. (There are reasons to be skeptical about this, as I have discussed in the past, but the desire to drop all those pesky sites is palpable.)

However, while a few proof-of-concept studies have been done, there really doesn't seem to have been another trial to attempt a full-blown direct-to-patient clinical trial. Other pilots have been more successful, but had fairly lightweight protocols. For all its problems, REMOTE was a seriously ambitious project that attempted to package a full-blown interventional clinical trial, not an observational study.

In this context, it's great to see published results of the TAPIR Trial in vasculitis, which as far as I can tell is the first real attempt to run a DIY trial of a similar magnitude to REMOTE.

TAPIR was actually two parallel trials, identical in every respect except for their sites: one trial used a traditional group of 8 sites, while the other was virtual and recruited patients from anywhere in the country. So this was a real-time, head-to-head assessment of site performance.

And the results after a full two years of active enrollment?

• Traditional sites: 49 enrolled
• Patient centric: 10 enrolled

Even though we’re six years later, and online/mobile communications are even more ubiquitous, we still see the exact same struggle to enroll patients.

Maybe it’s time to stop blaming the sites? To be fair, they didn’t exactly set the world on fire – and I’m guessing the total cost of activating the 8 sites significantly exceeded the costs of setting up the virtual recruitment and patient logistics. But still, the site-less, “patient centric” approach once again came up astonishingly short.
Krischer J, Cronholm PF, Burroughs C, McAlear CA, Borchin R, Easley E, Davis T, Kullman J, Carette S, Khalidi N, Koening C, Langford CA, Monach P, Moreland L, Pagnoux C, Specks U, Sreih AG, Ytterberg S, Merkel PA, & Vasculitis Clinical Research Consortium. (2017). Experience With Direct-to-Patient Recruitment for Enrollment Into a Clinical Trial in a Rare Disease: A Web-Based Study. Journal of medical Internet research, 19 (2) PMID: 28246067

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?

More people are getting cancer in their 20s, 30s, and 40s, and surviving, thanks to rapid advancement in care. Many will have decades of life ahead of them, which means they face greater and more complex challenges in survivorship. Lourdes Monje is navigating these waters at age 29.

NPR's Leila Fadel talks with actor Patrick Dempsey about his efforts to raise money for cancer treatment and prevention.

Sudan's civil war has displaced 10 million citizens. Here are profiles of two young people from the most vulnerable groups: an unaccompanied minor caring for twin brothers, a woman who was raped.

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."

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The post Kumpulan Game Slot Gacor Dengan Persentase RTP Tertinggi Hari Ini appeared first on Biosimilarnews.