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Delivered every Tuesday and Friday by 12 p.m., Prescription Pulse examines the latest pharmaceutical news and policy.

Feb 21, 2024 View in browser   By Lauren Gardner With Ben Leonard Driving The Day A new treatment for melanoma could be out of reach for some patients because of its cost and availability. | Fred Tanneau/AFP via Getty Images A CELL THERAPY BREAKTHROUGH — The FDA’s Friday approval of the first cell therapy for solid tumors marks a major development in cancer treatment and individualized therapies.  Yet, the treatment’s accessibility, high costs and demanding and unpleasant regimen could limit its reach as with other approved cell and gene therapies. Iovance Biotherapeutics’ Amtagvi, indicated to treat advanced melanoma — the deadliest form of skin cancer — is a type of therapy that employs tumor-infiltrating lymphocytes, or TILs. The novel treatment is the first non-CAR T-cell therapy to hit the market. FDA-approved CAR T-cell treatments are targeted for blood cancers, which represent about 10 percent of the cancers diagnosed in the U.S. annually. How the therapy works: TILs are made by biopsying a piece of the patient’s tumor and expanding and engineering the immune cells to boost their ability to fight the cancer, Dr. Jason Bock, CEO of the Cell Therapy Manufacturing Center, told Prescription Pulse. CTMC is affiliated with MD Anderson Cancer Center in Houston. The manufacturing process takes about a month before the patient can receive the single-dose infusion, Bock said. “These are literally the most complicated medicines we’ve ever tried to develop,” he said. Like gene therapies, Amtagvi won’t be cheap for payers, with an initial list price of $515,000. Patients will face access challenges similar to those associated with gene therapies. Iovance said more than 30 authorized treatment centers that can administer the therapy are active, with about 20 more expected to come online within the next three months. Bock said next-generation products that require infusing fewer cells and rely less on co-administered medicines to prepare patients for treatment would help bring down costs. People receiving Amtagvi must take high doses of interleukin-2, used to boost the immune system’s cancer-fighting power with a regimen that carries plenty of its own serious adverse effects and high costs. Still, the accelerated approval is a breakthrough for metastatic or unresectable melanoma patients with limited treatment options, FDA officials said. IT’S WEDNESDAY. WELCOME BACK TO PRESCRIPTION PULSE. Your host’s medicine to combat this Tuesday-that-was-like-a-Monday was this New York Times Magazine piece on a favorite reality TV scandal. Send news and tips to Lauren Gardner (lgardner@politico.com or @Gardner_LM) or David Lim (dlim@politico.com or @davidalim).   CONGRESS OVERDRIVE: Since day one, POLITICO has been laser-focused on Capitol Hill, serving up the juiciest Congress coverage. Now, we’re upping our game to ensure you’re up to speed and in the know on every tasty morsel and newsy nugget from inside the Capitol Dome, around the clock. Wake up, read Playbook AM, get up to speed at midday with our Playbook PM halftime report, and fuel your nightly conversations with Inside Congress in the evening. Plus, never miss a beat with buzzy, real-time updates throughout the day via our Inside Congress Live feature. Learn more and subscribe here.     Eye on the FDA FDA Director Robert Califf is meeting with his U.K. counterparts this week. | Kevin Dietsch/Getty Images WHERE IN THE WORLD IS ROBERT CALIFF? The FDA commissioner is across the pond this week, meeting with fellow regulators in the U.K. “to discuss opportunities for closer alignment,” he said on X, formerly Twitter. The FDA collaborates with drug authorities in allied nations on regulatory harmonization, such as the agency’s work with Health Canada a decade ago to align rules on certain over-the-counter cold medicines. Califf spoke Tuesday at the Richard Doll Seminar at the University of Oxford about one of his pet issues — improving clinical research. In an FDA blog, Dr. June Raine, Califf’s counterpart at the U.K.’s Medicines and Healthcare products Regulatory Agency, reflected on her meeting this week with the commissioner and their shared goals — combating misinformation, promoting diverse clinical trial populations and making pharma supply chains more resilient. FDA: WATCH FOR FAKE DATA — The FDA warns device manufacturers and study sponsors to scrutinize third parties used to conduct performance testing and verify testing results before sending them to the agency. The regulator says it has seen a rise in recent years in entities working with device firms that have generated fabricated, duplicated or “otherwise unreliable” data. The FDA said some of the increase has come from facilities in China and India, and the “alarming trend” could result in supply-chain issues and decreased patient access to new devices. YEARS IN THE MAKING — The FDA has partially granted a Public Citizen petition seeking additional safety warnings on the labels of glucocorticosteroids, such as prednisone, the nonprofit consumer watchdog group said Tuesday. But what was perhaps more notable was that the agency’s decision came 12-and-a-half years after Public Citizen submitted its request. “Going forward, the FDA should protect patients by prioritizing the prompt response to requests for enhanced safety labeling of drugs and other medical products,” Dr. Robert Steinbrook, director of Public Citizen’s Health Research Group, said in a statement. An FDA spokesperson didn’t comment on the decision. Industry Intel PHARMA FRETS OVER EU HEALTH DATA PLAN — Drug and medical device companies warn that a new EU plan for sharing health data could deter firms from conducting research in Europe for fear of competitors learning trade secrets, POLITICO’s Mari Eccles and Rory O’Neill report. Industry says the forthcoming plan doesn’t include sufficient intellectual property protections for the companies, universities and research groups that would have to make their datasets available upon request to support innovation. While the proposed regulation text says that “all measures necessary” should be taken to ensure trade secrets are protected, it would be up to newly created health data access bodies in each EU country to decide whether a data request is legitimate. Biotech companies are concerned those agencies could too freely give out their information. DMD DRUG DECISION DATE SET— Sarepta Therapeutics said Friday that the FDA has accepted supplemental efficacy data the company filed to the application for Elevidys, its Duchenne muscular dystrophy gene therapy. The regulator set June 21 as its target date for completing its review. The data is intended to support the conversion of the drug’s accelerated approval to traditional approval, plus expand its label indication to include DMD patients with a confirmed genetic mutation. The infusion is limited to patients ages 4 through 5 with the rare and fatal neuromuscular disease. The company also said the FDA doesn’t plan to hold an advisory committee meeting to discuss the new information, which analysts suggest is a positive sign for Elevidys’ broader approval. Pharma Moves Robert A. Michael will succeed Richard A. Gonzalez as AbbVie’s chief executive officer. Michael is president and chief operating officer of the company. WHAT WE'RE READING The FDA faces daunting funding and staffing shortfalls in regulating artificial intelligence, but the technology’s rollout continues apace in health care, POLITICO’s Ruth Reader writes. A multicountry study by the Global Covid Vaccine Safety Project confirmed already-established safety signals for myocarditis, pericarditis, Guillain-Barré syndrome and cerebral venous sinus thrombosis. The analysis identified other potential safety signals for further investigation.   DON’T MISS POLITICO’S GOVERNORS SUMMIT: Join POLITICO on Feb. 22 to dive into how Governors are wielding immense power. While Washington remains gridlocked, governors are at the center of landmark decisions in AI and tech, economic development, infrastructure, housing, reproductive health and energy. How are they setting the stage for the future of American politics, policies and priorities? How are they confronting major challenges? Explore these questions and more at the 2024 Governors Summit. 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