Episode 2: For boys with Duchenne, and two drug companies, a moment of shared hope

first_img 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 drugsFDAlast_img read more

‘Science works best in the open’: Experts weigh in on trial data reporting

first_imgFirst 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 Reprintscenter_img 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 ReportNIHlast_img read more

Harvard panel explores challenges of Alzheimer’s drug trials

first_img 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.advertisementcenter_img Sharon Begley In the LabHarvard panel explores challenges of Alzheimer’s drug trials @sxbegle Tags Alzheimer’sclinical trialsHarvard T.H. Chan School of Public Healthlast_img read more

A guide to the changing science of flu shots

first_img Nasal flu vaccine loses its luster — or does it?In late June an expert committee that advises the US government on vaccination policy recommended that the nasal spray vaccine FluMist, which has been used by millions, not be used this season.The reason: Studies conducted by the CDC had shown that for the past three flu seasons the vaccine wasn’t protecting the people who got it.It’s not clear why. Adding to the confusion, a study by the vaccine’s manufacturer, MedImmune, suggested the mist was effective. (MedImmune is a division of AstraZeneca.)Both the CDC and the Food and Drug Administration are working with the company to try to figure out what’s going on.In Canada, meanwhile, kids will have the option of getting FluMist this fall because data gathered there showed the vaccine was working — though not quite as well as injectable vaccine, said Dr. Danuta Skowronski, a flu expert at British Columbia’s Center for Disease Control in Vancouver.One of the Canadian studies, led by Dr. Mark Loeb at McMaster University in Hamilton, Ont., compared kids vaccinated with the nasal spray vaccine to kids who got flu shots over three flu seasons. He found the nasal spray vaccine worked as well as the flu shot.“I think they struck the right balance in that decision,” Skowronski said of the experts who decided to retain the vaccine in Canada.How can we make sense of all of this conflicting information? There are some theories.FluMist seemed to work well in the United States until MedImmune changed its formulation a few years ago to put an extra component in the vaccine. It had protected against three flu virus families; now it protects against four. Maybe something happened in that process?Or perhaps repeatedly vaccinating children with this vaccine is diminishing its effectiveness?For now, there’s no answer. While the search for one continues, the CDC has recommended US doctors not administer FluMist this flu season. How does the flu vaccine work?Volume 90%Press shift question mark to access a list of keyboard shortcutsKeyboard ShortcutsEnabledDisabledPlay/PauseSPACEIncrease Volume↑Decrease Volume↓Seek Forward→Seek Backward←Captions On/OffcFullscreen/Exit FullscreenfMute/UnmutemSeek %0-9 facebook twitter Email Linkhttps://www.statnews.com/2016/09/28/2016-flu-season-shots-science/?jwsource=clCopied EmbedCopiedLive00:0000:4900:49  The vaccine triggers the immune system to create antibodies, preparing the body for viral battle. Alex Hogan/STAT Questions over the impact of repeat vaccinationsThere have been suspicions for decades that getting a flu shot year after year might invoke a law of diminishing returns. Those suspicions stem from a 1970s study in which a researcher observed that boarding school students who were vaccinated each year were more likely to contract influenza.In the late 1990s a British researcher named Derek Smith hypothesized that repeated vaccination could trigger beneficial results — which he called positive interference — when the viruses the vaccine targets were different from one year to the next. Why my patients refuse the flu shot. It’s not what you think Newsletters Sign up for First Opinion A weekly digest of our opinion column, with insight from industry experts. Newsletters Sign up for Morning Rounds Your daily dose of news in health and medicine. About the Author Reprints So are the shots worth the bother? STAT asked some influenza vaccine experts to break down what we’ve learned lately about flu shots and what you need to know as the winter draws near. Nasal flu vaccine is not working and should not be used this season, expert panel concludes The staff at Courage’s Anchor Brewery in London receive a flu vaccine in January 1968. Harry Todd/Fox Photos/Getty Images Related: Please enter a valid email address. Inspired by that work, the researchers at Marshfield did a study of their own. They found statin use seemed to lower antibody production to one family of influenza A viruses (the H3N2s) but not to another (H1N1s) and not to influenza B viruses. In other words, this is another question that needs answering.Loeb said the issue bears studying, but hasn’t yet been proved. “I think there’s some biological rationale [but] it’s definitely not a slam dunk by any means.”The $64,000 questionSo how good is flu vaccine at protecting against influenza?In the not-too-distant past, the CDC and other public health institutes estimated that flu shots cut one’s risk of contracting flu by between 70 percent and 90 percent.But that new way of assessing vaccine effectiveness we talked about earlier has shed more light on that question, and the effectiveness estimate was seen to be too high.The more common claim is that the vaccine lowers one’s risk by an average of about 50 percent to 60 percent — though some years the protection is far less, depending on how well matched the viral targets in the vaccine are to the viruses making people sick.Everyone wishes the vaccines were more effective. Research is underway to try to develop a flu shot that would provide broader protection that wouldn’t have to be administered every year.But the science isn’t there yet. And bringing a brand new type of flu vaccine through licensure and to market would cost boatloads of money. So for now, manufacturers in the crowded flu vaccine market are, as Skowronski put it, “tinkering” — adding the fourth component to the vaccine, creating a high-dose product for seniors, who don’t respond as well to flu vaccine, or adding an adjuvant (a compound meant to amp up antibody production) to another vaccine for seniors.“You get maybe little marginal benefits here, there. But in terms of a real game-changer … I don’t see it,” Treanor said. Privacy Policy @HelenBranswell By Helen Branswell Sept. 28, 2016 Reprints Please enter a valid email address. Tags fluinfluenzavaccine Privacy Policy While the flu is a common illness, that hardly means the science around it is static. Some recent studies have suggested that getting a yearly shot may actually diminish the benefit of successive vaccinations. Others have raised the possibility that statins —  the commonly used cholesterol-lowering drugs — may actually interfere  with your immune system’s response to influenza vaccine.Meanwhile, the Centers for Disease Control and Prevention recently recommended against the use of the nasal mist vaccine that many kids prefer over injected vaccine.advertisement Helen Branswell Leave this field empty if you’re human: Yes, there are questions about flu vaccine that need to be explored through scientific study. But in the meantime, Skowronski said, people should stay the course.“You could become overwhelmed by the smorgasbord of issues that have arisen when we started to really systematically evaluate influenza vaccine performance on an annual basis,” she admitted.Treanor agreed.“The recommendation and the message that people should be vaccinated still holds true. Because it’s clear from all these studies that although many factors can influence how well the vaccine works, getting the vaccine is always better than not getting the vaccine,” he said. HealthA guide to the changing science of flu shots Related: Senior Writer, Infectious Disease Helen covers issues broadly related to infectious diseases, including outbreaks, preparedness, research, and vaccine development. You’re seeing the signs in pharmacies and perhaps around your workplace. Your doctor’s office may be calling to schedule an appointment. It’s just that time.Flu vaccination efforts are in full swing.But you may have been hearing puzzling things about flu shots over the past couple of years.advertisement Leave this field empty if you’re human: But he also suggested when the vaccine targets the same specific virus in successive years, the antibodies created in the first year might dampen antibody production the second year. He called that negative interference.In recent years, a new method of measuring the effectiveness of the flu vaccine has put this theory back on the table. In 2014 scientists from Marshfield Clinic Research Foundation in Marshfield, Wisc., found that people who received the flu vaccine generated higher levels of antibodies — compared with people who received an annual shot — if they hadn’t been vaccinated in the previous five years.So, is it a thing?Dr. John Treanor, an influenza vaccine expert at the University of Rochester in New York, said it appears there’s something there.“It’s really unclear exactly what the mechanism is and I think that’s going to be an area of very intensive investigation over the next few years,” said Treanor.The problem is, even if the theory proves true, it would be hard to act on the information, he noted. Flu vaccine contains protection against three or four types of viruses, depending on the brand. It is very rare that all four viruses would change from year to year.So one year you might not really need a repeat of one component, but you would need the other two or three. Because of the way vaccines are made, it’s impossible to unbundle the components.Still, understanding what is going on is important, said Skowronski.“For me anyway, these repeat vaccine effects are among the most important developments in influenza vaccinology of the past decade,” she said, noting the issue could have implications for universal vaccination programs, such as the one in the United States, where it’s recommended that everyone get vaccinated against influenza.“The long-term implications of that frankly are not known and these repeat vaccine effects may have a huge bearing on that.”In the meantime, experts stress that while repeat vaccinations could lead the body to generate fewer antibodies, it’s still recommended to get a yearly shot. Some protection is better than none.The statin factorStatin use has become ubiquitous among people in late-middle age and older seeking to lower their cholesterol.But two studies published last fall (here and here) suggest that people on statins don’t mount as vigorous an immune response to flu vaccine as people not taking the drugs. The effect was particularly pronounced for people taking synthetic statins.last_img read more

WHO director issues thinly veiled rebuke of FDA critics

first_img 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 healthVaccineslast_img read more

A Nevada bill targets drug makers over price hikes, but only for diabetes meds

first_img 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 STARTEDcenter_img 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.last_img read more

Soon-Shiong’s top executive steps down at NantHealth

first_img 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+last_img read more

Is the FDA approving drugs faster than ever? No, as the data show

first_img @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.center_img 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 STARTEDlast_img read more

A South Texas county drags PBMs into nationwide lawsuit over opioids

first_img 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 STARTEDcenter_img 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/STATlast_img read more

As Atul Gawande steps into a risky health CEO role, here are five challenges he faces

first_imgChallenge: Controlling costs while improving qualityHow: Leverage technology to inform patients, demand lower pricesRisk: Coming off as a corporate stooge, rather than a steward of better healthGawande’s biggest test will be to make changes that will simultaneously lower costs while improving the quality of care for 1.2 million employees who range from investment bankers, to short-order cooks, to Amazon pickers in fulfillment centers across the U.S.The combined workforce is a microcosm of America, which is why it can serve as a powerful example for the rest of the country and why it will be so difficult to develop effective solutions.To succeed, Gawande will need to make clear who his real boss is — the employees or the business leaders who have an inherent interest in reducing their runaway health care expenses. Gawande has emphasized that the new venture will be a nonprofit, and therefore somewhat insulated from corporate financial pressures. But health care business specialists said he will need to set the tone early among patients who will be skeptical of the new venture’s motives.“It’s a sensitive political area: Are we here to just cut costs? Will we lose the trust of those under our care if that’s a stated mandate?” asked Zen Chu, faculty director of Health Care Ventures at the Massachusetts Institute of Technology’s Sloan School of Management. “Setting it up as a nonprofit helps, but there will still be trust issues.”Gawande will need to muster his considerable talents as a communicator to explain the rationale behind his changes — whether it’s sending employees to a particular provider to get care, or more carefully scrutinizing when they get certain tests and procedures. 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. About the Author Reprints Related: By Casey Ross July 9, 2018 Reprints Related: After interviews with leaders across the world of health care, STAT has identified five challenges Gawande will take on in his new role. Each one carries substantial risks and complications that range from the political, to the personal, to the professional and ethical:advertisement Leave this field empty if you’re human: The new venture could help improve their care and cut costs by standardizing treatment regimens and directing employees to providers with proven track records. Given the expertise of Amazon, JPMorgan and Berkshire Hathaway in analyzing data and assessing risks, Gawande will be able to use machine learning software and other technologies to better predict the onset of disease and take preventive measures.That’s where Blumenthal sees the greatest potential for Gawande and the new health venture — using the power of technology to help inform patients and deliver care at the time when it can make the most difference.“The one major advantage that this combination has is Amazon and its IT capabilities and the network of consumers that Alexa touches,” he said, adding that the PillPack acqusition further enhances its ability to leverage technology.“I would extend that well beyond the pharmaceutical market to begin to try to influence the decisions that consumers make with their clinicians about health care in a very personal way,” Blumenthal said. “That doesn’t mean changing the way providers behave so much as it means changing the way consumers behave, making them smarter purchasers.”Challenge: Writing for the New Yorker while leading a national effort to reform health careHow: Carefully selecting subjects and avoiding conflictsRisk: Compromising the progress of the new venture, or undermining his credibility as a journalistGawande holds many jobs — surgeon, writer, teacher, entrepreneur — and intends to retain them while leading the new venture. But it may be difficult to keep the roles separate, particularly as it relates to his most public job as a staff writer for the New Yorker.The power of his writing is what put him on the radar of the executives who formed the new health venture. In 2010, Warren Buffett publicly praised “The Cost Conundrum,” Gawande’s New Yorker piece, which examined why health care was so much more expensive in some parts of the U.S. than others, despite little difference in the quality of care or the sickness of people getting it. HealthAs Atul Gawande steps into a risky health CEO role, here are five challenges he faces @caseymross Andrew Dreyfus, chief executive of Blue Cross Blue Shield of Massachusetts Challenge: Lowering hospital pricesHow: Shift care to lower-cost settings, get more employers to join the effortRisk: Alienating academic centers that dominate some marketsIt is impossible to save money without getting lower prices for medical services. Americans pay the highest health care prices in the world, which is a primary driver of the nation’s sky-high health care tab, according to a recent study by researchers at Harvard University and the London School of Economics.But several industry specialists said that Amazon and its partners, though large and influential, lack the concentration of employees in specific geographic regions to force hospitals to cut their prices.“The quickest way to get a bang for your buck in cost control in health care is for purchasers of care to negotiate better prices, and that’s what limits them in local markets — they don’t have enough purchasing power,” said Dr. David Blumenthal, president of the Commonwealth Fund, a private foundation that conducts health care research. “If you can’t negotiate better prices, the alternative is to send your consumers to places that charge lower prices.”In some cases, that might mean directing employees to steer clear of large academic medical centers that dominate local health care markets and charge the most for their care. Gawande himself is a surgeon at Brigham and Women’s Hospital, a prominent Harvard-affiliated hospital in Boston, so his effort in this new venture may run counter to the business interests of his own hospital. “All the eyes of American health care will be on this new initiative,” said Andrew Dreyfus, chief executive of Blue Cross Blue Shield of Massachusetts and an acquaintance of Gawande’s. “Atul understands this is high stakes, and I think that’s why he took the job.” Chu, who was the first investor in the company, said it accomplished that by using technology to create a better service. PillPack uses software and robots to deliver medicines in presorted packets to patients across the country that take multiple medicines. It provides them with instructions on taking the medications and avoiding adverse interactions.When Express Scripts, one of the nation’s largest pharmacy benefit managers, sought to block PillPack from its network, the company got its customers to protest the move, putting pressure on Express Scripts to reverse course. It did so in 2016.“From the beginning they knew they were competing on customer satisfaction,” Chu said of PillPack’s founders, who added that Amazon’s acquisition of the company will spur a transformation of the industry. “My prediction is this is the beginning of a sea change, as Amazon will be able to serve on the front lines of consumer health care.”Amazon will also likely become the pharmacy supplier to the 1.2 million employees covered by Gawande’s venture, which could help improve care and lead other employers to sign up.Challenge: Attacking chronic disease; and variations in careHow: Leverage technology to improve patient knowledge, contract with select providersRisk: Changing patient behavior is the hardest thing to do in health care, and may prove costlyA crucial part of Gawande’s efforts will be addressing the crushing burden of chronic disease. These preventable illnesses, such as diabetes and heart disease, dramatically magnify costs: Studies have established that 5 percent of U.S. patients account for nearly 50 percent of spending in the U.S.“Most of those 5 percent have multiple chronic illnesses that tend to not be well-managed within our fragmented health care system,” said Dreyfus, the Blue Cross chief executive. He added that one of Gawande’s first efforts will likely be trying to understand the extent of the disease burden among the 1.2 million employees. Dr. Atul Gawande is leading the new health venture funded by Amazon, JP Morgan Chase, and Berkshire Hathaway Dan Bayer/The Aspen Institute/Spotlight Health Another way to counter hospitals’ pricing power is to convince other large employers to join the new venture, which may be a relatively easy to do given the prominence and promise of the effort.“It wouldn’t surprise me if lots of other self-insured employers piled into whatever Atul is running,” said Chu. “It’s structurally a nonprofit and it improves with scale, so very quickly you could have 50 million covered lives, way beyond the 1 million employees the founding companies charged him with.”Challenge: Cutting out the middlemenHow: Create innovative business solutions that streamline health care’s supply chainRisk: Alienating key stakeholders needed to access or serve customersAmazon has built its business on simplifying supply chains and cutting out industry gatekeepers who either add unnecessary costs or undermine the customer experience. Think books. Think home goods. Think groceries.Gawande must accomplish the same feat in health care, where it is much harder and more complicated to do so. The market is filled with entrenched incumbents who control access to prescription drugs, medical services, and even the customers themselves.But Amazon’s recent acquisition of the mail-order pharmacy PillPack points to a way forward — and not just in pharmacy sector. PillPack successfully battled with pharmacy benefit managers, which operate their own mail-order pharmacies, to get access to networks that include hundreds of millions of U.S. customers. How Atul Gawande landed perhaps the most extraordinary (or impossible) job in health care Related: But writing on matters related to his work for the health care nonprofit could pose conflicts, potentially by disclosing information or strategies the new venture would want to keep quiet — something Amazon in particular insists upon in its business dealings. He might also have to tiptoe around certain topics to avoid infusing bias.“I see a lot that intersects pretty directly with the work he’ll be doing as part of his new venture,” said Dan Kennedy, an associate professor of journalism at Northeastern University. In addition to health care costs, Kennedy noted that Gawande has written about President Trump’s attempts to do away with the ACA, unnecessary medical procedures, and other controversial subjects.“Given his journalistic focus on policy matters, it strikes me that it’s going to be difficult for him to avoid writing about issues that he’s not involved in,” he said.More broadly, Gawande will have to learn to manage the new venture, and his other interests, in a fish bowl, as his every move and misstep will be the subject of intense media coverage and scrutiny. But keeping one foot in his existing endeavors also provides a cushion if the new venture doesn’t produce the results many are hoping for.“I do think this is brave,” Blumenthal said of Gawande’s new job. “On the other hand, he starts from a foundation of enormous prestige and influence and he’s not giving up the career that has brought him to prominence. So if it doesn’t work, he can return to being the extraordinary thinker and writer and innovator he’s always been.” Dr. Atul Gawande will step out of health care’s limelight on Monday to put himself under its microscope.Taking the helm of the new health venture funded by Amazon, JPMorgan Chase, and Berkshire Hathaway is the riskiest move of his career — one that will subject his acclaimed New Yorker narratives to a real-world stress test whose outcome is far from certain.In the balance will hang not just his reputation as a physician and writer, but perhaps the highest-profile effort to date to leverage the private sector to fix America’s fragmented and dysfunctional health care system. Gawande has made a name for himself by proposing novel solutions to the system’s many shortcomings — from surgical checklists to rooting out unnecessary care — and testing them in specific hospitals or markets around the world.advertisement [email protected] Tags hospitalsinsurancepatients Privacy Policy STAT+: “All the eyes of American health care will be on this new initiative. Atul understands this is high stakes, and I think that’s why he took the job.” Casey Ross Newsletters Sign up for Daily Recap A roundup of STAT’s top stories of the day. Please enter a valid email address. Amazon’s acquisition of PillPack is a first step, but not a fatal blow for the pharmacy industry Inside Ariadne Labs, Atul Gawande’s testing ground for new ideas in health care Exclusive analysis of biopharma, health policy, and the life sciences. last_img read more