About the Author Reprints PharmalotPharmalot, Pharmalittle: Glaxo names new CEO to succeed Andrew Witty By Ed Silverman Sept. 20, 2016 Reprints Alex Hogan/STAT Pharmalot Columnist, Senior Writer Ed covers the pharmaceutical industry. Australia’s Therapeutics Goods Administration plans to allow drug makers to submit reports from international regulators as one of several steps to speed the approval process for new drugs, according to BioCentury. The TGA estimates that the changes could shorten the approval time by as much as four months. Among the pathways will be an accelerated assessment and consideration of surrogate endpoints.Allergan agreed to buy Tobira Therapeutics in a deal worth up to nearly $1.7 billion, its second major acquisition in less than a week. Several days ago, Allergan agreed to purchase Vitae Pharmaceuticals for $639 million to bolster its dermatology product pipeline. The latest deal moves Allergan into treatments for nonalcoholic steatohepatitis, or NASH, and other liver diseases.The US Food and Drug Administration banned products from Laxachem Organics, a maker of active pharmaceutical ingredients, due to a bacterial outbreak, the Economic Times tells us. In July, the Centers for Disease Control and Prevention and the FDA began investigating a multi-state outbreak and, in one state, traced the problem to contaminated Docusate Sodium, an active ingredient supplied by Laxachem.The European Medicines Agency recommended banning all nonessential drugs made by Pharmaceutics International in the US due to manufacturing problems, according to this notice. Hello, everyone, and how are you today? We are just fine, thank you, especially now that the Pharmalot campus has settled down. The shortest of the short people has left for the local schoolhouse and the official mascots have descended into a deep snooze. We, however, must remain wide awake, which calls for a few cups of stimulation. As always, we invite you to join us. Meanwhile, here are the tidbits. Have a lovely day and do keep in touch …GlaxoSmithKline named Emma Walmsley to succeed Andrew Witty as chief executive when he retires next March. Walmsley, who will become the only woman to head a global brand-name drug maker, currently runs the Glaxo consumer health business, a unit that Witty has emphasized following a deal last year that created a joint venture with Novartis as part of a strategic realignment.Janet Woodcock, who heads the drug review division at the US Food and Drug Administration, won a huge and consequential power struggle this week when she successfully pressed the agency to approve a controversial new drug for treating Duchenne muscular dystrophy — even though there is scant evidence it works, STAT writes. “She’s tough as nails,” said Marc Boutin, who heads the National Health Council, a coalition of patient groups and drug makers.advertisement Bayer is considering dropping the Monsanto name to avoid “sullying its reputation,” Bloomberg News reports. The drug and chemical conglomerate wants to avoid association with a corporate name known for making the herbicide Agent Orange that was used in the Vietnam War and for tangling with environmental groups over genetically modified crops.Regeneron Pharmaceuticals and Teva Pharmaceuticals will jointly develop and market an experimental pain drug with a new mechanism of action that, if approved, could make it a non-addictive alternative to opioids, Reuters says. The injectable drug, called fasinumab, blocks Nerve Growth Factor, a protein involved in transmission of pain signals. Eli Lilly and Pfizer plan by 2018 to seek approval for a similar medicine.advertisement Ed Silverman [email protected] Tags FDAGlaxoSmithKlineSarepta Therapeutics @Pharmalot
Tags HIV/AIDSresearch By Linda-Gail Bekker and Anthony S. Fauci March 8, 2017 Reprints One of the most critical issues facing women at risk of contracting HIV in many parts of the world is the lack of prevention tools they can discreetly use, without needing to negotiate protection with a male sexual partner. More than 3,500 women in sub-Saharan Africa helped test a vaginal ring infused with an anti-HIV drug in two separate trials. Both studies showed that the ring provided modest protection from HIV infection. Women continue to use the rings in follow-up studies, helping scientists learn how this prevention strategy fits into women’s lives, how well it protects against HIV, and the complexities women face when relying on such a product.The fight against HIV/AIDS has always been an ambitious challenge. As HIV researchers, we see every day how our work to end this pandemic depends on strong women. We have met countless women along this journey who have given unconditionally, volunteering their time, their bodies, and their hope to drive the scientific process. We thank and salute them.Linda-Gail Bekker, MD, is president of the International AIDS Society and co-director of the Desmond Tutu HIV Center at the University of Cape Town, South Africa. Anthony S. Fauci, MD, is director of the National Institute of Allergy and Infectious Diseases, which is part of the National Institutes of Health. Women are setting examples, breaking down barriers, and demonstrating the value that inclusivity brings in scientific research. Because of their efforts, more trials will ensure that the unique biology of women is taken into account as new HIV treatment and prevention tools are developed, tested, and ultimately used by both sexes.advertisement @LindaGailBekker Anthony S. Fauci There is a saying in South Africa: Wathint’ abafazi, wathint’ imbokodo. You strike the women, you strike the rock.On International Women’s Day, we want to celebrate the strong women who have always been at the heart of fighting the HIV/AIDS epidemic. For more than 35 years, women have modeled strength and resilience as researchers, nurses and physicians, caregivers, volunteers, advocates, social workers, and community leaders.As the HIV/AIDS pandemic unfolded, women also became one of the most affected populations. Women now represent half of the people living with HIV around the world.advertisement Women are at the center of the fight against HIV/AIDS as researchers, clinicians, clinical trial participants, and more. MUJAHID SAFODIEN/AFP/Getty Images To end this pandemic, women are advancing research on the front lines as scientists in laboratories and clinics and as leaders of large, international clinical trial efforts. Women are also making a difference in clinics around the world as participants in clinical trials, volunteering to help us better understand and fight the disease, one person at a time. Revised US tally: HIV infections fell 18 percent in 6 years Ending HIV requires new prevention methods for women Linda-Gail Bekker [email protected] @NIAIDnews First OpinionWomen are leading the way in HIV research The participation of young women will be essential for testing a new HIV vaccine in the recently launched HVTN 702 study, the only currently active HIV vaccine efficacy trial in the world. It aims to enroll 5,400 women and men between the age of 18 and 35 in South Africa. Another 1,500 young women will participate in the antibody-mediated prevention studies, in which HIV-negative participants receive intravenous infusions of anti-HIV antibodies to see if this new approach can reduce their risk of becoming infected with the virus.Women living with HIV are playing an important role in helping all people with the disease as participants in the REPRIEVE trial. This ambitious study, funded by the National Institutes of Health, was launched in 2015. It aims to determine if taking a cholesterol-lowering statin drug can reduce the risk of cardiovascular disease among people with HIV. Women living with HIV are three times as likely as their HIV-negative counterparts to develop heart disease; men with HIV are up to twice as likely. Trial investigators, attuned to the disparities affecting women in clinical research, are enrolling a racially and ethnically diverse group of women alongside men in five countries. Related: [email protected] About the Authors Reprints Related:
Pioneering cancer therapy makes two CAR-T drugs the approvals of the year About the Author Reprints BRENDAN SMIALOWSKI/AFP/Getty Images Related: Related: John Maraganore Support a productive, consistent, and transparent FDA. The Food and Drug Administration ended 2017 with more than 50 approvals, including the first gene and chimeric antigen receptor T-cell (CAR-T) therapies that could transform the treatment of cancer and other life-threatening — and often rare — diseases. Thanks to remarkable scientific advances and a positive regulatory environment, many more innovations stand at the cusp of FDA approval in 2018, including the first RNA-interference-based therapeutics that my company has pioneered. The president should talk about the importance of a strong and efficient regulatory process for our industry. We have observed important progress over the past year — legislation to streamline the clinical trial process, acceleration of the approval of generic medicines, and providing patients a stronger voice in regulatory decision-making. We should continue to build upon it.Continue to select strong leaders for HHS. The president should feel free to take a victory lap on the leadership he’s already assembled at the Department of Health and Human Services FDA Commissioner Scott Gottlieb and Centers for Medicare and Medicaid Services Administrator Seema Verma both win high marks for results to date, and are tremendous leaders who have a strong, broad understanding of FDA and CMS and its many programs that support health care for Americans. Newly appointed HHS Secretary Alex Azar is an excellent recent appointment who will extend the strong leadership already in place.advertisement We live in a time where the pace of biomedical discovery has never been faster and the opportunity for patients’ access to transformative medicines has never been greater. We are in the era of precision medicine, attacking disease with genetic therapies. This is an exhilarating time for patients, many of whom previously faced a future with little hope of getting better.It’s also an exciting time for U.S.-based biopharma innovators, as we lead the world into the new frontier of medicine in an industry that employs 1.7 million Americans. Continuing to foster this culture of innovation will be key to ensuring that important new therapies reach patients in need.As President Trump prepares to address the nation in his first State of the Union address, I’d encourage him to foster this momentum in several ways:advertisement By John Maraganore Jan. 30, 2018 Reprints First OpinionA biopharma innovator’s 5 wishes for Trump’s State of the Union address Acknowledge that innovation is part of solving the opioid crisis. We need to consider new plans to stimulate research into and development of innovative treatments to treat pain and opioid addiction. This means increasing resources at the FDA, streamlining the drug development and review process for non-opioid alternatives, and establishing expedited approval pathways to speed the commercialization of non-addictive painkillers. The pharmaceutical industry responded to the HIV crisis with new medicines that saved lives. We’re prepared to do the same with the opioid crisis.When it comes to biopharmaceutical innovation, America leads the world. The president has a unique opportunity tonight to advance policies that can cement U.S. global leadership for generations to come.John Maraganore is CEO of Boston-based Alnylam Pharmaceuticals and chairman of the Biotechnology Innovation Organization (BIO), the world’s largest biotechnology trade association. Keep discussions on drug pricing grounded in facts. The trend on drug costs is moving in the right direction. CMS reports that spending on retail drugs increased by just 1.3 percent in 2016. Outside groups have affirmed this finding; the Altarum Institute recently reported a meager 1 percent increase in drug prices over the last year. Meanwhile, administration officials have taken important steps to help make prescription drugs even more affordable. Under Gottlieb’s leadership, the FDA approved 1,027 generic drugs and 56 novel drugs and biologics in 2017 — both record highs. Moreover, the FDA has taken numerous regulatory actions to facilitate the entry of generic versions of complex drugs, such as biosimilars. A robust generic drug market that can’t be gamed is a clear and proven approach to lower drug prices that also preserves innovation.Support targeted reforms that benefit patients. Policies written in the last century should be modernized to change how our health care system pays for prescription medicines. Under Verma’s leadership, CMS has worked to lead on new value-based approaches to drug pricing. This approach will bend the cost curve in health care and help patients access therapies they need with out-of-pocket costs they can afford. But these efforts can only take us so far. It’s time to modernize federal rules so more patients enjoy the benefits of value-based pricing. CMS has also recently made significant changes to the 340B drug pricing program rules, which lower patients’ out-of-pocket costs. Here, too, more fulsome programmatic reforms would help lower out-of-pocket costs for patients. A drug with the startling power to mute defective genes raises hopes for a new class of therapies — but hurdles abound Tags Donald Trumpdrug developmentpharmaceuticalsWhite House @jmaraganore
Tags drug developmentgovernemnt agenciespharmaceuticalsSTAT+ The FDA disagrees with its own expert panels how often? Unlock this article by subscribing to STAT+ and enjoy your first 30 days free! GET STARTED @Pharmalot By Ed Silverman July 15, 2019 Reprints What’s included? Log In | Learn More What is it? 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. FDA [email protected] 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. Pharmalot Columnist, Senior Writer Ed covers the pharmaceutical industry. About the Author Reprints Pharmalot Ed Silverman As the Food and Drug Administration grapples with rising pressure to approve new medicines more quickly, a new analysis finds the agency disagreed with its expert advisory panels about one-fifth of the time. And the disagreements were more likely to occur over safety issues than over approving new products or additional uses for existing products.Of those disagreements, 75% resulted in the FDA making more restrictive decisions after an advisory panel made a favorable recommendation, while 25% resulted in the agency making less restrictive decisions after unfavorable panel recommendations, according to the analysis, which was published in The Milbank Quarterly and reviewed 376 panel meetings that took place between 2008 and 2015.
By Charles K. Fisher Nov. 7, 2019 Reprints Newsletters Sign up for STAT Health Tech Your weekly guide to how tech is transforming health care and life sciences. AI startups are racing into drug development. Here’s 5 burning questions about which will survive Please enter a valid email address. Artificial intelligence has become a crucial part of our technological infrastructure and the brain underlying many consumer devices. In less than a decade, machine learning algorithms based on deep neural networks evolved from recognizing cats in videos to enabling your smartphone to perform real-time translation between 27 different languages. This progress has sparked the use of AI in drug discovery and development.Artificial intelligence can improve efficiency and outcomes in drug development across therapeutic areas. For example, companies are developing AI technologies that hold the promise of preventing serious adverse events in clinical trials by identifying high-risk individuals before they enroll. Clinical trials could be made more efficient by using artificial intelligence to incorporate other data sources, such as historical control arms or real-world data. AI technologies could also be used to magnify therapeutic responses by identifying biomarkers that enable precise targeting of patient subpopulations in complex indications.Innovation in each of these areas would provide substantial benefits to those who volunteer to take part in trials, not to mention downstream benefits to the ultimate users of new medicines.advertisement First OpinionThe FDA needs to set standards for using artificial intelligence in drug development Related: @charleskfisher Dom Smith/STAT [email protected] The FDA has an opportunity to ease regulatory uncertainty by proposing a framework that guides how sponsors can use AI tools within drug development programs. By engaging with industry to develop a workable regulatory framework, the FDA can balance the opportunity for artificial intelligence to provide significant public health benefits with its mission to protect public health by ensuring that these new technologies are reliable. At the same time, the FDA could create a pathway for formal qualification of AI-based drug-development tools to ensure that these tools are sufficiently vetted.In addition, it could encourage the exploratory use of AI-based technologies in drug development that would allow sponsors and regulators to better understand their advantages and disadvantages through use of new regulatory pathways, such as the Complex Innovative Trial Designs Pilot Program.These concrete actions would open the door to innovative approaches to clinical trials that will make drug development more efficient and so help deliver new treatments to patients who need them as quickly as possible.Charles K. Fisher, Ph.D., is the founder and CEO of San Francisco-based Unlearn.AI, Inc. Misapplication of these technologies, however, can have unintended harmful consequences. To see how a good idea can turn bad, just look at what’s happened with social media since the rise of algorithms. Misinformation spreads faster than the truth, and our leaders are scrambling to protect our political systems.Could artificial intelligence and machine learning similarly disrupt our ability to identify safe and effective therapies?advertisement About the Author Reprints Leave this field empty if you’re human: Even well-intentioned researchers can develop machine learning algorithms that exacerbate bias. For example, many datasets used in medicine are derived from mostly white, North American and European populations. If a researcher applies machine learning to one of these datasets and discovers a biomarker to predict response to a therapy, there is no guarantee the biomarker will work well, if at all, in a more diverse population. If such a biomarker was used to define the approved indication for a drug, that drug could end up having very different effects in different racial groups simply because it is filtered through the biased lens of a poorly constructed algorithm.Concerns about bias and generalizability apply to most data-driven decisions, including those obtained using more traditional statistical methods. But the machine learning algorithms that enable innovations in drug development are more complex than traditional statistical models. They need larger datasets, more sophisticated software, and more powerful computers. All of that makes it more difficult, and more important, to thoroughly evaluate the performance of machine learning algorithms.Companies operating at the intersection of drug development and technology need standards to ensure that artificial intelligence tools function as intended.The FDA has already issued several proposals around the regulation of AI products, and it now has an opportunity to build on these efforts. The Center for Devices and Radiological Health has reviewed and cleared a number of devices that use AI. The center has also released a proposed framework, “Artificial Intelligence and Machine Learning in Software as a Medical Device.” These proposals, though, don’t necessarily apply to AI-based tools used as part of the drug development process. As a result, biopharmaceutical and technology companies aren’t sure how these tools fit into current regulatory frameworks.I’m the founder and CEO of a company that uses artificial intelligence to streamline clinical trials and make them more efficient. You might expect me to counsel the FDA to back off on creating hurdles for companies that want to apply artificial intelligence to drug development. Not so. In a presentation to the FDA on Thursday, I’ll argue that the agency should play an important role in ensuring that AI-based drug development tools meet appropriate standards. Charles K. Fisher Tags Artificial Intelligencedrug developmentgovernment agencies
Unlock this article — plus daily coverage and analysis of the pharma industry — by subscribing to STAT+. First 30 days free. GET STARTED By Ed Silverman July 6, 2020 Reprints Pharmalot Columnist, Senior Writer Ed covers the pharmaceutical industry. Pharmalot About the Author Reprints Good morning, everyone, and welcome to another working week. We hope the weekend respite, which was extended on this side of the pond due to a holiday, was somehow relaxing and refreshing. Well, regardless, the usual routine of Zoom meetings, Skype calls and deadlines has predictably returned. What can you do? The world keeps spinning, no matter what. So hold on tight, grab a cup of stimulation, and feel free to peruse the tidbits we have assembled for you. Hope your day goes well, and do keep in touch. One more thing, stay safe and wear a mask. …Researchers have designed a staggering 1,200 clinical trials aimed at testing treatment and prevention strategies against Covid-19 since the start of January, but a new STAT analysis shows the effort has been marked by disorder and disorganization, with huge financial resources wasted. For instance, one in every six trials was designed to study the decades-old malaria drugs hydroxychloroquine or chloroquine, which have been shown to have no benefit in hospitalized patients. Ed Silverman Pharmalittle: Covid-19 trials are marked by disorganization; FDA approves a new type of HIV treatment Alex Hogan/STAT 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. @Pharmalot Log In | Learn More GET STARTED [email protected] What is it? Tags HIV/AIDSSTAT+ What’s included? 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.