| | | | |  | | By Lauren Gardner and David Lim | | | | | 
The FDA's Center for Drug Evaluation and Research needs funds to boost its inspector workforce and help prevent drug shortages. | Andrew Harnik/AP | CHECK-IN WITH CAVAZZONI — The FDA’s top drug regulator wants Congress to know that flat taxpayer funding is forcing the FDA to make choices about how it deploys its funds to infrastructure, information technology systems and other priorities. “While the user fee programs have an adjustment for inflation, this has not been the case for the [budget authority] base,” Patrizia Cavazzoni, director of the Center for Drug Evaluation and Research, told the Alliance for a Stronger FDA on Monday. “Effectively, we get a cut in our base every year.” Foreign inspections: The agency especially wants to invest in its inspector workforce as it works on a major overhaul of its Office of Regulatory Affairs structure. Retaining experienced investigators is a specific focus because it takes new hires a long time to gain a similar level of proficiency. “The investigator’s job is the most difficult job we have here at FDA,” Cavazzoni said. “Particularly those who have to travel all over the world to do foreign inspections have a very, very challenging schedule. We need to make sure that we reward them accordingly.” Drug shortages: Cavazzoni is concerned drug shortages last longer and become more complex to fix because the supply chain isn’t resilient. “We are increasingly reliant on one or two manufacturers to make very important essential drugs,” Cavazzoni said. The FDA is piloting an approach in which manufacturers are encouraged not to stop production if they hear negative feedback during an inspection and instead call the FDA’s drug shortage surveillance team. “We want to start problem-solving with manufacturers as soon as possible during the inspection,” Cavazzoni said. “We can try to put in place some mitigation approaches that we have to make sure that we don’t find ourselves with downright stoppages of manufacturing of essential drugs like we have seen over the past year.” Next up: FDA Commissioner Robert Califf is slated to again testify before Senate appropriators in charge of FDA funding on Wednesday. The agency asked for $157 million in additional taxpayer funds for fiscal 2025 compared with its fiscal 2023 level. IT’S TUESDAY. WELCOME BACK TO PRESCRIPTION PULSE. Merck would really like the D.C. District Court to issue its opinion on the company’s challenge to the Medicare drug price negotiation program — even though fellow drugmakers haven’t notched any wins yet. Send news and tips to David Lim (dlim@politico.com or @davidalim) and Lauren Gardner (lgardner@politico.com or @Gardner_LM).
| | | | THE GOLD STANDARD OF HEALTHCARE POLICY REPORTING & INTELLIGENCE: POLITICO has more than 500 journalists delivering unrivaled reporting and illuminating the policy and regulatory landscape for those who need to know what’s next. Throughout the election and the legislative and regulatory pushes that will follow, POLITICO Pro is indispensable to those who need to make informed decisions fast. The Pro platform dives deeper into critical and quickly evolving sectors and industries, like healthcare, equipping policymakers and those who shape legislation and regulation with essential news and intelligence from the world’s best politics and policy journalists.
Our newsroom is deeper, more experienced and better sourced than any other. Our healthcare reporting team—including Alice Miranda Ollstein, Megan Messerly and Robert King—is embedded with the market-moving legislative committees and agencies in Washington and across states, delivering unparalleled coverage of health policy and the healthcare industry. We bring subscribers inside the conversations that determine policy outcomes and the future of industries, providing insight that cannot be found anywhere else. Get the premier news and policy intelligence service, SUBSCRIBE TO POLITICO PRO TODAY. | | | | | | | | | REINING IN ‘GAIN-OF-FUNCTION’ RESEARCH — The White House announced a new policy late Monday for strengthening the oversight of high-risk research — like the kind that some experts, lawmakers and intelligence officials believe caused the Covid pandemic, POLITICO’s Carmen Paun reports. The policy, from the Office of Science and Technology Policy, will apply to federal agencies that fund research into pathogens that could pose a risk to animal or human health, agriculture or national security. It takes effect in a year. Researchers who seek federal funding to conduct research on a pathogen that could increase its transmissibility, make it more virulent, make a known toxin more dangerous or create a new toxin must submit a risk-mitigation plan to the agency funding the work. The agency will weigh the benefits and risks to decide whether to fund the project. Additionally, a panel of scientific experts will review the research plan before it’s approved.
| | |  The FDA's Peter Marks says hiring experienced specialists in cell and gene therapy has been a challenge for the agency. | Pool photo by Anna Moneymaker | FOCUS ON CELL, GENE THERAPIES — Hiring personnel with expertise in cell and gene therapies remains a challenge at the FDA, but senior officials said Monday they’re optimistic about the progress they’ve made so far. CBER’s Office of Therapeutic Products has done well in hiring the allotted personnel under the prescription drug user fee program, which was reauthorized in 2022, Dr. Peter Marks, head of the FDA’s Center for Biologics Evaluation and Research, said during a Friends of Cancer Research event in Washington. “Right now, it’s not a bad time because there are … a fair number of people that are skilled in [chemistry, manufacturing and controls] that are out there that we've been able to hire — physicians that are earlier on in their career who we’ve been able to hire — so I’m pretty encouraged with the people who we brought on board recently,” Marks said. “It’s exciting because we have a diversity of experience and expertise that will serve us well for these products.” Making the therapy: A major challenge for cell and gene therapies is the manufacturing process. The therapies are generated using the patient’s cells, known as autologous therapies, or a donor’s cells, known as allogeneic therapies. Allogeneic treatments can be made in large batches for “off-the-shelf” access, while autologous therapies are personalized for the donor patient. Marks said the FDA is neutral on centralized manufacturing versus decentralized manufacturing. “There are advantages potentially to decentralized manufacturing for certain products — particularly ones that are made fewer times — but we are just there to support this process forward just as long as the ultimate product, whether centralized or decentralized, and manufacturer is at the same high quality,” he said. Covid-19 lessons: The agency has launched a pilot program for rare disease therapy development in which makers of prospective cell and gene therapies have more frequent communication with FDA staff to help with clinical data generation. The pilot seeks to emulate Covid vaccine makers’ access to regulators when they worked to rapidly test and deploy shots to control the pandemic. “If you get rid of a lot of 30-day or 75-day waits, development can occur much more rapidly,” Marks said, referring to how long it typically takes to schedule a meeting with the agency. “We hypothesize that, for other products, that kind of communication could help, particularly ones for serious diseases, cancers.” If the pilot demonstrates that those interactions can slash development timelines, Marks said, it could help make the case for the FDA to hire more personnel. The agency currently employs more than 18,000 people. “Even a 10 or 20 percent reduction in time of development can really have an impact on patient lives when you’re dealing with a life-threatening illness,” he said.
| | | | SANDERS: HE’S RUNNING — Senate HELP Chair Bernie Sanders (I-Vt.) announced Monday he’s running for reelection this cycle. “I have been, and will be if re-elected, in a strong position to provide the kind of help that Vermonters need in these difficult times,” the Vermont independent said in a statement, referring to his chairmanship and senior positions on other Senate committees. Should Democrats hold the chamber this November, drugmakers can expect to remain under the microscope of Sanders’ Health, Education, Labor and Pensions Committee, which has pressured several companies to lower the prices of their products. Some have followed suit, lowering or capping prices on insulins and asthma inhalers, though other factors — like Medicaid rebate changes — have factored into those decisions.
| | | | LISTEN TO POLITICO'S ENERGY PODCAST: Check out our daily five-minute brief on the latest energy and environmental politics and policy news. Don't miss out on the must-know stories, candid insights, and analysis from POLITICO's energy team. Listen today. | | | | | | | | IRA FEEDBACK OPPORTUNITY — Have thoughts on the Biden administration’s Medicare drug price negotiation program? CMS wants input on its newly released draft guidance for the second cycle of negotiations slated for 2027 by July 2. “We continue to engage drug companies and the public as we build on what we are learning in the first cycle of negotiations,” CMS Administrator Chiquita Brooks-LaSure said. PhRMA, which represents major U.S. drugmakers, immediately slammed the CMS document, saying the agency “continues to undervalue and discourage the research and development of medicines.” “CMS’ draft guidance fails to meaningfully improve transparency in the process, ignores patient and provider concerns around how prices are set and turns a blind eye to insurers who are choosing to limit coverage options for seniors,” said Alex Schriver, PhRMA senior vice president of public affairs.
| | | Kelly Ngan is now director of the FDA’s Counter-Terrorism and Emergency Coordination Staff in the CDER director’s office. She has served as acting director since former leader Rosemary Roberts retired in July. Dr. Andrew Baum will join Pfizer as chief strategy and innovation officer from Citi, where he led health care research.
| | The first patient to receive a new gene therapy for sickle cell disease outside of clinical trials has begun treatment in Washington, The New York Times reports. State agricultural officials are at odds with a CDC effort to study avian flu on the ground, David and his POLITICO colleagues Meredith Lee Hill and Marcia Brown report.
| | The FDA’s cancer center has launched a “crowdsourcing initiative” to encourage clinical data collection in real-world settings. | | | | Follow us on Twitter | | | | Follow us | | | | |
