Calls grow for a rethinking of FDA voucher program

An FDA approval pathway that promises one- to two-month reviews for drugs that have been deemed to be aligned with “national priorities” is under scrutiny. Patient advocates, former agency officials, and many industry players are urging the agency to pause the program and reintroduce it through formal rulemaking, STAT’s Lizzy Lawrence writes.

At a public hearing yesterday, speakers warned that the Commissioner’s National Priority Voucher program — launched last year by then-commissioner Marty Makary — lacks transparency and is vulnerable to political interference.

“The ambiguity of the criteria in this context and lack of transparency has given life to rumors about political influences determining winners and losers,” said Janet Krommes, chair of the FDA Task Force of Doctors For America. “This is extremely corrosive to patient care.”

The agency will accept public comments through June 29 as it decides whether to continue the program.

cancer

RevMed's cancer drug feels out of reach for some

A remarkable experimental pancreatic cancer drug from Revolution Medicines is generating enormous hope, but also frustration, as patients scramble to access it through a newly launched expanded access program. Daraxonrasib nearly doubled median survival in a pivotal trial, STAT’s Ed Silverman writes, fueling demand from patients who have exhausted other options and view the drug as a rare lifeline in a disease with few effective treatments.

But the rollout has been slowed by paperwork, institutional review board approvals, and communication challenges, and there’s worry that some patients may run out of time to be treated.

Revolution says demand is high but insists supply is not expected to be a limiting factor, and analysts report the company is confident in manufacturing capacity.

“This all takes time, but the fact they can ship the drug within a month is remarkable,” said Anna Berkenblit, chief scientific and medical officer at the Pancreatic Cancer Action Network, an advocacy group. “This hasn’t happened to this extent before in pancreatic cancer. It’s a new thing. It’s an unusual situation and everyone is learning.”

podcast

What RevMed's drug meant for one patient

Why did oncologists give a standing ovation to a data presentation on Revolution Medicines' pancreatic cancer drug, daraxonrasib? Why did biotech stocks perform so badly this week? And are concrete beaches better than normal beaches?

We discuss all that and more on this week's episode of “The Readout LOUD,” STAT's biotech podcast.

We bring on Leanna Stokes, who was diagnosed with metastatic pancreatic cancer in 2023. She discusses how participating in RevMed's trial for daraxonrasib has affected her disease and life, and what the drug means for patients and the field.

We also chat about other data presented during last week's meeting of the American Society of Clinical Oncology, the market reaction to Abivax's ulcerative colitis data, and the upcoming meeting of the American Diabetes Association.

Listen here.

legal

Supreme Court preserves key generic drug pathway

The Supreme Court yesterday handed a major win to generic drugmakers, STAT’s Ed Silverman writes. In a unanimous decision, it ruled that Hikma Pharmaceuticals did not infringe patents held by Amarin — reinforcing the legality of so-called “skinny labels.”

The case centered on Amarin’s fish-oil-derived drug Vascepa. Amarin argued that Hikma’s labeling and public statements could still encourage doctors to prescribe the generic for a cardiovascular indication that was still under patent protection. The court disagreed, finding that Hikma’s actions did not actively induce patent infringement.

“The decision is pretty much a complete victory for Hikma and the generic drug industry,” one attorney and Hatch-Waxman expert told STAT. “Although this decision may still allow for inducement claims to be brought when active steps are taken by a generic company to encourage infringement, it will certainly be a sigh of relief for the generic drug industry and will support the continued use of skinny labeling to avoid infringement of method of use patents.”

Thanks for reading! Until next week,

@megkesh

WASHINGTON