Molly Ferguson for STAT Max, left, 13, and Austin Leclaire, 16, outside of their apartment in Pembroke, Mass.. Both boys have Duchenne muscular dystrophy, and are taking an experimental drug. Kayana Szymczak for STATBut given the amount of suffering these little boys go through, should the FDA approve them, anyway? How much evidence is enough? And as companies start to tackle drugs for rare diseases, is this is a dilemma the FDA is going to be seeing more of?Subscribe to Signal on iTunes or Stitcher.The Signal podcast is produced by Katie Hiler.Music:Time to Move and Motivate, by The InsiderSnowing, by Peter RudenkoOur Ego [feat. Different Visitor], by Broke For FreeGloci, by Graham BoleElefly, by Eino Toivanen UPDATE (Nov. 25):An FDA panel reviewed a drug for Duchenne muscular dystrophy called drisaperson on Tuesday. What it found is not encouraging. The Signal podcast takes a look at what happened and where things go from here._______________________________________In this episode we turn the spotlight on a rare but debilitating disease affecting young boys, Duchenne muscular dystrophy. Duchenne boys have a genetic mutation that affects their ability to produce a protein called dystrophin, which is essential for normal muscle function. By their pre-teen years, boys with Duchenne are almost always confined to wheelchairs. They are only expected to live into their 20s. And despite decades of research and telethon fundraising, there is still no cure.advertisement Signal PodcastEpisode 2: For boys with Duchenne, and two drug companies, a moment of shared hope Meg Tirrell About the Authors Reprints Into this hopelessness step two companies: Sarepta Therapeutics and BioMarin Pharmaceutical. Both have developed drugs to treat Duchenne and are currently racing to get them approved by the Food and Drug Administration. But the path to get there hasn’t been easy — each has had their own corporate saga. Company bigwigs were ousted, stocks soared and tanked, partnerships were created and dissolved. What once looked like promising data has dwindled to a questionable body of evidence for both these drugs. As these dramas play out, parents of boys with Duchenne are waiting. And their kids are only getting worse. But the wait, and the drama, may be over soon. On Nov. 24, an outside panel of FDA advisors is meeting to discuss whether BioMarin’s drug should be approved. It’s unclear if the drug will meet the rigorous standards set by the FDA to balance safety concerns against the potential benefit of a drug.advertisement Co-host “The Readout LOUD,” CNBC senior health and science reporter By Meg Tirrell and Luke Timmerman Nov. 19, 2015 Reprints @megtirrell Tags duchenne muscular dystrophyexperimental drugsFDA
First Opinion‘Science works best in the open’: Experts weigh in on trial data reporting Failure to report: A STAT investigation of clinical trials reporting Editor, First Opinion Patrick Skerrett is the editor of First Opinion, STAT’s platform for perspective and opinion on the life sciences writ large, and the host of the First Opinion Podcast. Hospitals, medical schools, businesses, and even the National Institutes of Health are routinely violating a federal law requiring clinical trials to be reported to the public, a STAT investigation recently found. In response, we asked experts to offer their thoughts on why low rates of clinical trial reporting is a problem, and what can be done about it.Christine Laine: ClinicalTrials.gov registration has important shortcomingsTim Errington: Science works best when it is openKarl Schwartz: Patients need accurate clinical trial informationBy Christine Laine: The timely and transparent reporting of clinical trials is of utmost importance. It has been furthered by organizations such as the International Committee of Medical Journal Editors. For example, the Committee’s trial registration policy markedly increased the number of clinical trials with prospectively documented methods in a public registry. However, the STAT investigation’s laser focus on whether the results appeared in ClinicalTrials.gov as an indicator of responsible trial reporting is troubling to me as a journal editor who oversees the peer review and publication of many clinical trials.advertisement Related: Related: While the registration of results in ClinicalTrials.gov is a baby step toward responsible trial reporting, it has important shortcomings. Anyone who examines the registry will quickly see that results reported in this manner are difficult to digest and are unlikely to be meaningfully useful to patients and physicians as they make real world clinical decisions. Further, the registry allows investigators to post results that have not been vetted by peer review. In my more than 15 years as a journal editor, I have never seen a clinical trial report published without requiring substantive changes in response to expert peer review. Thus, reporting clinical trial results in journals after peer review, and in a format that provides context, detailed methods, and explanation of trial strengths and limitations, may be the most transparent, responsible, and useful way to disseminate what we learn from trials.The STAT investigation does not appear to have gathered information about whether the results of the trials it reviewed had been published in a peer reviewed venue. Of course, if a serious effort to publish trial results remains unsuccessful, trialists should register their results. Anyone attempting to use results that have not been published in a peer-reviewed journal in their clinical practice or research should recognize that inability to find a home in such a journal could signal serious problems with the design, conduct, or reporting of the trial.advertisement Timely and full reporting shows respect to clinical trial participants who, in volunteering, have shouldered potential risks. Selective reporting can potentially lead to inappropriate medication use. For example, when negative trials are not published, a drug’s benefit can be exaggerated. This could lead to inappropriate off-label use, which increases the expense of treatment and exposes more patients to possible harm.The STAT investigation is helping shine a light on an issue that is very important for patients. Going forward, it would be important for the federal government or an independent organization to formally evaluate on an ongoing basis how well institutions report clinical trial results.Compliance with clinical trial reporting requirements is vitally important. It will foster patient and public trust in the research system, while improving its quality and efficiency.Karl Schwartz is the cofounder and president of Patients Against Lymphoma, a nonprofit advocacy organization that provides support, education, and evidence-based resources on lymphoma and its treatments. [email protected] Clinical trials are subjecting cancer patients to too many biopsies, study finds About the Author Reprints By Patrick Skerrett Dec. 18, 2015 Reprints Increase in industry-funded drug trials bad for public health, researcher says The world is quickly moving beyond registration of trial methods and results. There is a movement to require that clinical trialists share de-identified, patient-level data with others who wish to use these data in their own work. Such sharing holds great potential to advance science in a more efficient manner than ever before and will make registration of results passé.Christine Laine, MD, is editor in chief of Annals of Internal Medicine and senior vice president of the American College of Physicians.By Tim Errington: Science works best when it is done in the most open way possible. Unfortunately, open science is something we mostly talk about today instead of something we practice.Failure to accurately, completely, and quickly report the results of clinical trials, as well as unexpected findings and harmful side effects, poses hazards for researchers, clinicians, and patients. Not reporting this information leaves gaps that make it difficult to fully determine the effectiveness of a treatment or to understand the risks of trying a new medication. Related: Reporting clinical trial results can feel like a lot of work. It’s usually done in a separate process after the data have been collected, analyzed, and reported in a journal. That’s no excuse for delaying or failing to report results, but it is a barrier. In addition, there are no incentives for sending the required data to ClinicalTrials.gov, nor are penalties enforced for failing to deliver this information. This combination leads to the dismal results seen in the STAT investigation on clinical trial result reporting.It’s time to shift the incentive structure, which currently focuses on publishing the results of clinical trials — especially positive results — rather than on the quality of the research that has been done. It’s also time to make it easier for researchers to work in more open and more streamlined ways.The Center for Open Science and others are working to do just that. Researchers have traditionally generated ideas, collected data, run analyses, and disseminated results in virtually separate processes, and they often have to regenerate information for each step. We now have the technology to unify these processes, and should start to use it more widely.It’s also important to link related material such as registration information, data collected during the trial, analysis methods, publications based on the data set, data that didn’t make it into the publication, related analyses, and the like. That would let individuals see all of the information about a trial in one place. Open Trials is working to achieve such linkage.Although STAT’s focus on reporting to ClinicalTrials.gov was much needed, the concerns go beyond this registry and database. We must extend this kind of reporting around the world.The more open the scientific process and the more information we can share, the more the global community will benefit.Tim Errington, PhD, is project manager for Meta-science at the Center for Open Science in Charlottesville, Va.Karl Schwartz: Thank you to STAT for its investigation into the disappointing failure to report clinical trial results as directed by the Food and Drug Administration Amendments Act of 2007.The STAT report identifies failures by eminent institutions on the forefront of clinical research. It also identified some of the underlying causes, which is an important first step to help these institutions to rise to the challenge.Reporting research results is a critical aspect of collaboration that informs decision making by researchers, physicians, and patients. It also guides the direction of future research. Failure to report clinical trial data can affect future research strategies and patient safety. APStock Patrick Skerrett @PJSkerrett Tags clinical trialsFailure to ReportNIH
By Sharon Begley April 15, 2016 Reprints Dina Rudick/The Boston Globe Senior Writer, Science and Discovery (1956-2021) Sharon covered science and discovery. [email protected] About the Author Reprints The challenges of clinical trials, particularly those testing Alzheimer’s disease drugs, was the focus of an hour-long panel discussion on Friday. The event was hosted by the Forum at Harvard T.H. Chan School of Public Health, in collaboration with STAT.The program was presented in partnership with HHMI Tangled Bank Studios and in association with NOVA, whose film “Can Alzheimer’s Be Stopped?” examines the hurdles to developing and testing experimental treatments for the disease.Missed the event live? The video is available in its entirety below.advertisement Sharon Begley In the LabHarvard panel explores challenges of Alzheimer’s drug trials @sxbegle Tags Alzheimer’sclinical trialsHarvard T.H. Chan School of Public Health
Log In | Learn More What is it? @HelenBranswell What’s included? The director of the World Health Organization issued a thinly veiled rebuke of critics suggesting that the Food and Drug Administration should be overhauled, including several candidates reported to be in consideration to head the agency.Speaking at a conference in Seattle on Wednesday, Dr. Margaret Chan warned against loosening the rules governing the safety and effectiveness data that drug companies must supply to win marketing approval from the FDA. GET STARTED Daily reporting and analysis The most comprehensive industry coverage from a powerhouse team of reporters Subscriber-only newsletters Daily newsletters to brief you on the most important industry news of the day STAT+ Conversations Weekly opportunities to engage with our reporters and leading industry experts in live video conversations Exclusive industry events Premium access to subscriber-only networking events around the country The best reporters in the industry The most trusted and well-connected newsroom in the health care industry And much more Exclusive interviews with industry leaders, profiles, and premium tools, like our CRISPR Trackr. Senior Writer, Infectious Disease Helen covers issues broadly related to infectious diseases, including outbreaks, preparedness, research, and vaccine development. By Helen Branswell Feb. 9, 2017 Reprints Unlock this article by subscribing to STAT+ and enjoy your first 30 days free! GET STARTED Francois Mori/AP Helen Branswell Health WHO director issues thinly veiled rebuke of FDA critics About the Author Reprints STAT+ is STAT’s premium subscription service for in-depth biotech, pharma, policy, and life science coverage and analysis. Our award-winning team covers news on Wall Street, policy developments in Washington, early science breakthroughs and clinical trial results, and health care disruption in Silicon Valley and beyond. Tags drug developmentpolicypublic healthVaccines
By Ed Silverman March 30, 2017 Reprints Log In | Learn More About the Author Reprints What’s included? Pharmalot GET STARTED What is it? [email protected] A Nevada lawmaker has introduced a bill to fight the rising cost of medicines, one of dozens state legislators to attempt such a move. But this one has a twist — the legislation targets only companies that make diabetes drugs and insulin.Specifically, the bill requires drug makers to report pricing histories; disclose a slew of costs, such as research, manufacturing, and patient assistance; and notify state officials and insurers at least 90 days before prices are increased above the inflation rate for the preceding year. Unlock this article — plus daily coverage and analysis of the pharma industry — by subscribing to STAT+. First 30 days free. GET STARTED Tags drug pricingpharmaceuticalsSTAT+ @Pharmalot STAT+ is STAT’s premium subscription service for in-depth biotech, pharma, policy, and life science coverage and analysis. Our award-winning team covers news on Wall Street, policy developments in Washington, early science breakthroughs and clinical trial results, and health care disruption in Silicon Valley and beyond. Ed Silverman Pharmalot Columnist, Senior Writer Ed covers the pharmaceutical industry. A Nevada bill targets drug makers over price hikes, but only for diabetes meds Insulin bottles at a Novo Nordisk factory in France. Francois Monier/AFP/Getty Images Daily reporting and analysis The most comprehensive industry coverage from a powerhouse team of reporters Subscriber-only newsletters Daily newsletters to brief you on the most important industry news of the day STAT+ Conversations Weekly opportunities to engage with our reporters and leading industry experts in live video conversations Exclusive industry events Premium access to subscriber-only networking events around the country The best reporters in the industry The most trusted and well-connected newsroom in the health care industry And much more Exclusive interviews with industry leaders, profiles, and premium tools, like our CRISPR Trackr.
By Rebecca Robbins May 5, 2017 Reprints Dr. Patrick Soon-Shiong’s NantHealth is losing another top executive.Bob Watson, the company’s president and chief growth officer, is leaving. The company announced this week that he will “relocate to the East Coast for family reasons.” What is it? Dr. Patrick Soon-Shiong is the founder, CEO, and majority owner of NantHealth. Danny Moloshok/AP GET STARTED STAT+ is STAT’s premium subscription service for in-depth biotech, pharma, policy, and life science coverage and analysis. Our award-winning team covers news on Wall Street, policy developments in Washington, early science breakthroughs and clinical trial results, and health care disruption in Silicon Valley and beyond. Log In | Learn More Soon-Shiong’s top executive steps down at NantHealth Unlock this article — plus daily market-moving biopharma analysis — by subscribing to STAT+. First 30 days free. GET STARTED Business Daily reporting and analysis The most comprehensive industry coverage from a powerhouse team of reporters Subscriber-only newsletters Daily newsletters to brief you on the most important industry news of the day STAT+ Conversations Weekly opportunities to engage with our reporters and leading industry experts in live video conversations Exclusive industry events Premium access to subscriber-only networking events around the country The best reporters in the industry The most trusted and well-connected newsroom in the health care industry And much more Exclusive interviews with industry leaders, profiles, and premium tools, like our CRISPR Trackr. What’s included? Tags biotechnologycancerdiagnosticsfinanceSTAT+
@damiangarde Politics By Damian Garde Nov. 16, 2017 Reprints [email protected] STAT Unlock this article by subscribing to STAT+ and enjoy your first 30 days free! GET STARTED Damian Garde What is it? Tags cancerdrug developmentpolicySTAT+ Daily reporting and analysis The most comprehensive industry coverage from a powerhouse team of reporters Subscriber-only newsletters Daily newsletters to brief you on the most important industry news of the day STAT+ Conversations Weekly opportunities to engage with our reporters and leading industry experts in live video conversations Exclusive industry events Premium access to subscriber-only networking events around the country The best reporters in the industry The most trusted and well-connected newsroom in the health care industry And much more Exclusive interviews with industry leaders, profiles, and premium tools, like our CRISPR Trackr. What’s included? About the Author Reprints STAT+ is STAT’s premium subscription service for in-depth biotech, pharma, policy, and life science coverage and analysis. Our award-winning team covers news on Wall Street, policy developments in Washington, early science breakthroughs and clinical trial results, and health care disruption in Silicon Valley and beyond. Is the FDA approving drugs faster than ever? No, as the data show Log In | Learn More With each breathless announcement and triumphant press release, it seems clear that the Food and Drug Administration is approving new drugs at a record pace under Dr. Scott Gottlieb.But we wanted to put that appearance to the test. So we gathered more than five years of approval data from Evaluate Pharma, covering more than 200 first-time drug approvals. And it turns out, the FDA has actually been reliably speedy under multiple administrations, consistently OK’ing drugs ahead of schedule. National Biotech Reporter Damian covers biotech, is a co-writer of The Readout newsletter, and a co-host of “The Readout LOUD” podcast. GET STARTED
By Casey Ross Feb. 26, 2018 Reprints [email protected] @caseymross National Technology Correspondent Casey covers the use of artificial intelligence in medicine and its underlying questions of safety, fairness, and privacy. He is the co-author of the newsletter STAT Health Tech. Tags legalopioidspharmaceuticals Pharma Daily reporting and analysis The most comprehensive industry coverage from a powerhouse team of reporters Subscriber-only newsletters Daily newsletters to brief you on the most important industry news of the day STAT+ Conversations Weekly opportunities to engage with our reporters and leading industry experts in live video conversations Exclusive industry events Premium access to subscriber-only networking events around the country The best reporters in the industry The most trusted and well-connected newsroom in the health care industry And much more Exclusive interviews with industry leaders, profiles, and premium tools, like our CRISPR Trackr. What’s included? STAT+ is STAT’s premium subscription service for in-depth biotech, pharma, policy, and life science coverage and analysis. Our award-winning team covers news on Wall Street, policy developments in Washington, early science breakthroughs and clinical trial results, and health care disruption in Silicon Valley and beyond. Unlock this article — plus daily coverage and analysis of the pharma industry — by subscribing to STAT+. First 30 days free. GET STARTED Log In | Learn More GET STARTED What is it? About the Author Reprints A South Texas county drags PBMs into nationwide lawsuit over opioids Casey Ross A massive lawsuit over the nation’s opioid crisis has largely ignored an influential group of companies in the prescription drug business — the financial middlemen who brokered access to the powerful pills that got so many people hooked.Perhaps that’s because their role is simply harder to see. Known as pharmacy benefit managers (PBMs), these companies secretly make the rules that determine the availability of drugs, and how much patients must pay out of pocket to get them. Alex Hogan/STAT