At 31, she runs one of the hottest biotech companies in the country

first_img NewslettersSign up for The Readout Your daily guide to what’s happening in biotech. “It makes me feel incredibly lucky,” Haurwitz said. “I am here because I was in the right place at the right time, with the right people and the right science around me.”But even if she was the beneficiary of some good fortune, Haurwitz, associates say, deserves ample credit for helping to transform the company from a one-person operation into a growing enterprise with tens of millions of dollars in investment — all as a 31-year-old woman working in an industry still dominated by men.advertisement Privacy Policy BERKELEY, Calif. — The question on the test was about CRISPR, but Rachel Haurwitz, then a graduate student at the University of California, Berkeley, botched it. She had never heard the term.Less than a decade later, Haurwitz is the CEO of Caribou Biosciences, one of the leading companies pursuing commercial applications of CRISPR, a remarkable gene-editing tool that could help scientists develop new medical treatments and advance other industries.Haurwitz readily admits she chanced into running a company at the forefront of one of the hottest areas in science. As a graduate student, she happened to land in a lab that was starting to explore CRISPR systems, just a few years before the field exploded.advertisement @DrewQJoseph Leave this field empty if you’re human: None of the other three cofounders wanted to leave academia, and Haurwitz had known she wanted to pursue a career outside an academic lab. She became Caribou’s CEO, and started working on the company full time when she finished her PhD in 2012.(“Caribou” is a combination of Cas, a term meaning CRISPR-associated, and ribo, as in ribonucleic acid, or RNA. Haurwitz’s boyfriend at the time came up with it. He’s now her husband.)Haurwitz’s first office was in a startup incubator in the basement of the same building as Doudna’s lab; it lacked some of the lab equipment she needed and didn’t have cell service. Now with about 30 employees, Caribou has office and lab space here in West Berkeley, just a few blocks from the bay.The company has raised more than $40 million.“She really grew into [being CEO],” said Jinek, now on the faculty at the University of Zurich. “At the beginning, she worked at the bench when it was really small. But then she transitioned and did this, I would say, flawlessly.”Caribou is often described as the nontherapeutic CRISPR company. It helped launch a separate business, Intellia Therapeutics, in 2014 to build treatments with CRISPR, a goal also pursued by companies including Editas Medicine and CRISPR Therapeutics. Related: By Andrew Joseph June 22, 2016 Reprints Related: [email protected] Disciplined, competitive, and decisive, Haurwitz has been eager and quick to learn new skills as an executive, growing as a leader as the company has advanced, people who know her say.“She was always cool under pressure — she never got rattled,” said Jennifer Doudna, a Caribou cofounder and the UC Berkeley CRISPR pioneer in whose lab Haurwitz did her PhD research. “I was always struck by that. I think that plays to her strength in the business world.”Haurwitz acknowledges that much of the excitement around the company is because it is working with CRISPR, considered a biotech darling. That’s helped Caribou avoid the painful slogs that trip up many young biotech companies, though she added, “No doubt that’s coming.” Meet ‘CRISPR’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 Link EmbedCopiedLive00:0000:5700:57  CRISPR is a tool that acts as a microscopic pair of scissors with the ability to slice DNA. Dom Smith/STAT Haurwitz, who grew up in Austin, Texas, first caught the science bug as a kid, and it was at Harvard where she picked up an interest in RNA, specifically. She then headed to graduate school in 2007, where she spent time in a few labs studying RNA before winding up in Doudna’s in 2008.It was there she met CRISPR again.“She pitched to me the project that she wanted me to join,” Haurwitz said. “There was one scientist in the lab working on it, and it was something she called CRISPR.”At the time, Doudna told her that it appeared to be a bacterial immune system but that people were not sure how it worked. It had some interesting biochemistry, structural biology, and RNA components, and it became Haurwitz’s job to help understand parts of it.“I felt, well, that sounds wacky. I like wacky, let’s do it,” Haurwitz said.Blake Wiedenheft, the postdoctoral researcher with whom Haurwitz worked, said Haurwitz jumped into the research, demonstrating her determination and commitment to the project during an experiment that required them to remain in the lab for 24 hours.“Rachel would seem to get stronger, while I was certainly fading,” said Wiedenheft, now on the faculty at Montana State University.Haurwitz in the Caribou offices in Berkeley. Elizabeth D. Herman for STATOf course, scientists in Doudna’s lab and others soon realized much more was going on. Researchers discovered they could harness CRISPR systems to cut and edit DNA in plant, animal, and human cells. As scientists uncovered more about CRISPR, Doudna and Haurwitz started discussing the commercial possibilities.Eventually in 2011, Doudna and Haurwitz teamed up with Martin Jinek, then a postdoctoral researcher, and James Berger, then a professor at UC Berkeley and now at Johns Hopkins University, to found Caribou. A debate: Should we edit the human germline? General Assignment Reporter Andrew covers a range of topics, from addiction to public health to genetics. Federal panel approves first use of CRISPR in humans Caribou, meanwhile, has been focused on enhancing what CRISPR can do and thinking about how the tool could be adapted for use in agriculture, industrial biosciences, and other fields. It has research partnerships with Novartis Institutes for Biomedical Research and DuPont, and recently finalized a deal with the animal genetics company Genus that will allow the company to use Caribou’s technology in some livestock.Haurwitz said the goal for Caribou is to strike more partnerships and licensing agreements, and possibly create more spinouts like Intellia. But it also intends to pick a few fields to design and build its own products, which Haurwitz said could include human therapeutics.Another issue that the company will have to deal with is the dispute over key CRISPR patents. Caribou licensed some of its technology from the University of California and its research partners. UC, however, is in a fight with the Cambridge, Mass.-based Broad Institute and the Massachusetts Institute of Technology before the US Patent and Trademark Office over which side deserves the intellectual property for that technology.The uncertainty used to cast a cloud over companies working with CRISPR. But if UC loses, Caribou would likely license technology from another institution. Executives at other companies have echoed that point, and they say investors have come to realize that, as well.“The last time we were out fundraising, the first five questions, the middle five questions, and the last five questions were always IP, IP, IP,” Haurwitz said. Now, she added, investors see some risk, but “it’s just a question of who’s going to be paying a royalty to whom.”Despite Caribou’s successes so far, as a young woman leading a company, Haurwitz stands out in the industry. But she said there have only been a few times when she felt disrespected, which she attributed more to her age than her gender.Still, she seems to take some pride in making others realize they have a gender imbalance.“I have, multiple times, been in a boardroom with a bunch of VCs who are proudly going through their deck of who their team is … and at some point they’ll be about halfway through the slides and freeze,” Haurwitz said. “Because they realize they’re showing me a slide of [only] men.” About the Author Reprints BusinessAt 31, she runs one of the hottest biotech companies in the country Rachel Haurwitz is the CEO of Caribou Biosciences, a biotech pursuing commercial applications of CRISPR. Elizabeth D. Herman for STAT Andrew Joseph Please enter a valid email address. Tags biotechCRISPRprofileslast_img read more

Scientists discover new threat to antibiotic of last resort

first_img Cash prizes for pharma are needed to fight superbugs By Andrew Joseph July 7, 2016 Reprints Privacy Policy General Assignment Reporter Andrew covers a range of topics, from addiction to public health to genetics. About the Author Reprints Related: European scientists have discovered another gene that makes bacteria resistant to the antibiotic of last resort, one that could spread more easily among other kinds of bacteria.The gene, called mcr-2, was found in E. coli bacteria from pigs in Belgium, the scientists reported Thursday in the journal Eurosurveillance. It is similar to the gene mcr-1, which was first identified in China last fall and has now been seen in 30 countries across five continents, including the United States.Both genes confer resistance to a drug called colistin, an antibiotic used to cure infections that have already developed resistance to other antibiotics.advertisement While mcr-1 and mcr-2 have the same clinical effect, mcr-2 might be able to be passed among different bacteria more easily than mcr-1, the new study found.advertisement Superbugs for dummies: Explaining the battle between bacteria and antibiotics Related: Please enter a valid email address. Newsletters Sign up for Daily Recap A roundup of STAT’s top stories of the day.center_img Colistin resistance has been seen before, but both mcr-1 and mcr-2 pose a particularly worrisome threat. The genes are carried on plasmids, mobile pieces of DNA that can be swapped from one bacterium to another, even from different families. That means they can wind up in bacteria that infect people. HealthScientists discover new threat to antibiotic of last resort @DrewQJoseph [email protected] Mcr-1 has already been found in bacteria retrieved from people, including a woman in the United States. The fear is that such a gene could find its way into bacteria that are already resistant to other antibiotics, creating a pan-resistant bug that cannot be treated.The emergence of superbugs has been blamed on the overuse of antibiotics in both people and in livestock. Colistin is not used in animal husbandry in the United States, but is in some parts of the world, including China.US officials have said they are expanding surveillance efforts to hunt for mcr-1, and the authors of the new report called for the “immediate inclusion” of mcr-2 in such efforts around the world.Price noted that many reports of colistin-resistant bacteria are coming out of Europe because researchers there are doing genetic analysis of bacteria more frequently than elsewhere. He said US scientists should be doing the same.“We need more active surveillance looking for these bugs so we can get ahead of these things,” he said. Andrew Joseph A gene that makes bacteria resistant to the antibiotic of last resort was found in E. coli from pigs. Carsten Koall/Getty Images “They showed that this has a higher transfer frequency, so this thing has the potential to disseminate more quickly than mcr-1,” said Lance Price, who leads the Antibiotic Resistance Action Center at George Washington University.For the report, scientists studying E. coli samples from piglets and calves in Belgium found bacteria from the pigs that were resistant to colistin but that did not contain mcr-1. Genetic sequencing led them to the new gene.The mcr-2 gene was more prevalent than mcr-1 in the samples of E. coli, but the scientists noted they were working with a small number of samples.That another gene or another version of a gene also makes superbugs resistant to colistin is not unexpected, said Barry Kreiswirth of the Public Health Research Institute at Rutgers. Some genes that confer resistance to other antibiotics have multiple variants as well.“There are more of these guys out there, including different flavors,” Kreiswirth said. “This is what you expect when people start looking.” Leave this field empty if you’re human: Tags antibiotic resistanceantibioticssuperbuglast_img read more

German agency criticizes European program for speeding some drug approvals

first_img In pointed remarks, Germany’s cost-effectiveness watchdog has criticized an effort by European regulators to accelerate approval for new medicines based on limited evidence. And the concerns raised by the agency come as regulators on both side of the Atlantic increasingly look to such approaches to get new drugs to patients with unmet medical needs.At issue is a proposal called adaptive pathways, a term used to describe a method for jumpstarting drug approvals for select patient populations. Two years ago, the European Medicines Agency launched a specific pilot program with plans to compare initial data used for approval with so-called “real world” data, which is subsequently gathered after the medicines are in use.But after the EMA released a final progress report late last month, the German Institute for Quality and Efficiency in Health Care last week turned around and raised serious concerns about the effort. In short, the German watchdog agency maintained the EMA failed to make its case that this approach for approving drugs can make a demonstrable difference.advertisement About the Author Reprints APStock The Germany agency, however, was having none of it. “In view of the importance of the pilot project for drug development and the potential consequences of the considerable changes in approval procedures for patients,” IQWIG wrote, “concealment of the content and results of the discussions seems unacceptable.”Finally, IQWIG concluded by criticizing the EMA for failing to offer proposals for using real-world trial data after approvals, notably the potential for improved value. “If this is still lacking, then it would be high time to pause for a moment and rethink the whole concept, instead of considering more drugs in the consultations on adaptive pathways, as planned by EMA,” the Germany agency suggested.We asked the EMA for a response to the criticism and will update you accordingly.It is worth noting the EMA did acknowledge in its report that adaptive pathways is not a suitable approach for developing all medicines. The regulator conceded most companies were “not ready to describe real-world data plans” or strategies because they did not yet know whether their product will prove effective. And the EMA also lamented the fact that it received few proposals about value that were thought out.[UPDATE: An EMA spokeswoman later wrote us that “we reject IQWIG’s conclusion about the limitations of real-world data.” The report states that “‘the majority of the plans were vague in terms of the purpose of collection of real-world data..’ This is not a judgement of the usefulness, in general, of collecting real-world data to assess a drug’s performance, only of the post-licensing evidence generation plan submitted by some companies. The purpose of repeat discussions among stakeholders is to refine and clarify real-world data collection plans. In several instances, EMA advised companies on what kind of real-world data and methodologies of analysis would be expected.”She added that “methodological challenges to the use of real-world data are recognized in the report and the pilot was intended to be a learning exercise of what was feasible within the current regulatory framework, not the definition of a new regulatory standard.” A workshop is to be held in December “to further discuss best practice examples and methodological challenges.”] Leave this field empty if you’re human: In particular, the German agency chastised the EMA for pilot programs that were “vague” about collecting real-world data to supplement existing clinical trial results. And the IQWIG, as the agency is called, also complained that there was “insufficient detail” in the proposals submitted by companies to bolster and refine safety and effectiveness information.advertisement Pharmalot Columnist, Senior Writer Ed covers the pharmaceutical industry. IQWIG also complained that the EMA “justifies this information gap” by pointing to a need to keep certain company data confidential. This has been a contentious point between the EMA and drug makers. After protracted debate, the European regulator last March released new rules that attempt to create limits for companies that seek to redact trial data. European ombudsman urges regulator to get tough on redacting study data @Pharmalot Please enter a valid email address. [email protected] Ed Silverman Related: “Neither industry nor EMA has a concept as to how real-world data can be used after drug approval to allow drawing reliable conclusions on benefit and harm,” the agency wrote in a statement. Moreover, “a critical discussion on the quality, potential for bias, and reliability of the data acquired” following regulatory approval “was lacking.” Newsletters Sign up for Pharmalot Your daily update on the drug industry. Privacy Policy By Ed Silverman Aug. 15, 2016 Reprints PharmalotGerman agency criticizes European program for speeding some drug approvals Tags drug approvalEuropean Medicines Agencylast_img read more

Pharmalot, Pharmalittle: Glaxo names new CEO to succeed Andrew Witty

first_img 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.advertisementcenter_img 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 last_img read more

Women are leading the way in HIV research

first_img 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 center_img 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:last_img read more

A former drug czar weighs in: What Trump should have done about the opioid crisis

first_img Related: Access gaps arise again in the use of medications to treat addiction. According to the president’s commission, medication-assisted therapy can “reduce overdose deaths, retain persons in treatment, decrease use of heroin, reduce relapse, and prevent spread of infectious disease.” Only about 10 percent of conventional drug treatment facilities in the United States provide medication-assisted therapy for opioid use disorder, and significant barriers exist to getting it among certain populations, such as those in rural parts of the United States and those in the criminal justice system. The commission called for the immediate establishment of, and funding for, a federal incentive to enhance access to these medications yet no such funding is included in the emergency declaration.We must also better support those who are in recovery, so they will stay in recovery. According to the National Institute on Drug Abuse, relapse rates for people with addiction and other substance use disorders are similar to rates for other chronic medical illnesses such as diabetes or hypertension. Yet resources for the supports people need to stay in recovery are typically not covered by insurance. These include peer recovery coaches, transportation and child care, as well as community resources such as housing and employment. States need additional resources to build up not only treatment programs but support services that are essential for people to maintain their recovery.Funding is also needed for first responders to purchase naloxone, the most effective antidote to overdose deaths. Yet this medication is becoming an increasingly exorbitant line item for public safety and community health budgets. With the price of naloxone increasing dramatically, and multiple doses needed to reverse fentanyl-related overdoses, many state health departments, as well as state and local law enforcement departments, simply cannot meet current demand without additional resources. About the Author Reprints Privacy Policy The best strategy we can embrace on the treatment access front is to fight to preserve the Affordable Care Act, particularly Medicaid and Medicaid expansion. According to the most recent National Survey on Drug Use and Health, only 10 percent of the nearly 21 million citizens with a substance use disorder receive any type of addiction treatment. One of the primary reasons people who seek treatment are not able to get it is lack of insurance coverage or insurance that does not provide an adequate substance use disorder treatment benefit. By Michael Botticelli Oct. 30, 2017 Reprints 4 questions about Trump’s emergency declaration on opioids Related: Why fentanyl is deadlier than heroin, in a single photo First OpinionA former drug czar weighs in: What Trump should have done about the opioid crisis President Trump may have underscored the gravity of the opioid epidemic by declaring a public health emergency, but he failed to put forth any new resources or actions that would make a significant and immediate impact on the trajectory of the worst health crisis of our time.Instead of putting the full weight of the federal government behind fighting the crisis, Trump ignored many of the most compelling recommendations from his own Commission on Combating Drug Addiction and the Opioid Crisis. Those of us on the front lines of this scourge — which is already claiming more American lives each year than motor vehicle crashes and gunshot homicides — hoped to see a more precise, thoughtful, and aggressive strategy, one that would include the rapid allocation of federal funding that such a crisis deserves.As Congress and the commission consider what impact the declaration will have, and what is still needed, there are certain approaches that should not be overlooked, starting with expanding the capacity of the health care system to treat people with opioid addiction and making sure they have access to that care.advertisementcenter_img Leave this field empty if you’re human: The urgency of this epidemic cannot be overstated and needs to be matched by actions and resources that will make a difference. Many of policies and programs put in place by previous administrations are already showing promising results. We need make sure they are fully resourced and implemented.As with most epidemics, this one continues to evolve. We need to continue to put forward new strategies, such as those in the interim report from the Commission on Combating Drug Addiction and the Opioid Crisis, and act on them with the sense of urgency that this epidemic demands.Michael Botticelli is the executive director of the Grayken Center for Addiction at Boston Medical Center. He served as the director of the White House Office of National Drug Control Policy from March 2014 until January 2017. Newsletters Sign up for First Opinion A weekly digest of our opinion column, with insight from industry experts. @MBotticelliBMC Also missing from Trump’s declaration is the mandated prescriber education that was recommended by his opioid and addictions commission. “While we acknowledge that some of this inappropriate overprescribing is done illegally and for profit, we believe the overwhelming percentage is due to a lack of education on these issues in our nation’s medical and dental schools and a dearth of continuing medical education for practicing clinicians,” it concluded. Again, the president’s declaration fell short of mandating prescriber education initiatives.One of the most effective strategies to promote safer prescribing is the use of state-based prescription drug monitoring programs to better track patient-specific prescription data. Numerous studies have shown these programs to be highly effective at reducing problematic prescribing and reducing overdose deaths. Essential to this effort is sharing data across state lines. But to do so, we need federal funding and technical support to enhance interstate data sharing and to ensure that federal health care systems, including the Department of Veterans Affairs hospitals, participate in state-based data sharing.Trump was at least correct to identify the opioid crisis as a “public health emergency.” Any future efforts should be focused on a more robust health response, not a law enforcement response. While law enforcement efforts are a vital part of the overall strategy, this country and many local law enforcement agencies have begun to finally move away from arrest and incarceration as the foundation of our response. In acknowledging this as a public health crisis, the president should also nominate someone well-respected within the public health community to lead his Office of National Drug Control Policy, a post that remains vacant nine months after his inauguration. President Donald Trump shakes hands with New Jersey Gov. Chris Christie, who chairs the president’s Commission on Combating Drug Addiction and the Opioid Crisis, after the president signed a memorandum last week to declare the opioid crisis a national public health emergency. Pablo Martinez Monsivais/AP Medicaid expansion under the Affordable Care Act — adopted by 31 states — has played a crucial role in increasing treatment access in states that have been hit hard by this epidemic. If the president really does believe that this is a public health emergency, he should drop his effort to repeal the ACA and eviscerate the Medicaid program.advertisement Michael Botticelli Please enter a valid email address. Tags addictionopioidsWhite Houselast_img read more

A biopharma innovator’s 5 wishes for Trump’s State of the Union address

first_img 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 last_img read more

The FDA disagrees with its own expert panels how often?

first_img 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. FDAcenter_img [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.last_img read more

The FDA needs to set standards for using artificial intelligence in drug development

first_img 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/STATcenter_img [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 agencieslast_img read more

Pharmalittle: Covid-19 trials are marked by disorganization; FDA approves a new type of HIV treatment

first_img 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.center_img @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.last_img read more