By Maggie Fick and Patrick Wingrove
SAN FRANCISCO, Jan 16 (Reuters) - More than half a dozen major drugmakers are participating in the Trump administration's speedier review program for new medicines, yet other leading pharmaceutical companies are hesitating over what they see as potential legal risks, six industry sources told Reuters.
Under the FDA Commissioner's National Priority Voucher Program unveiled in June, U.S. Food and Drug Administration chief Marty Makary can issue approval decisions in one to
two months on a limited number of drugs deemed critical to public health or national security, or if they were manufactured in the U.S. or offered at low prices.
The FDA has long offered a fast-track review process of six to eight months for medicines that address critical U.S. health needs, while standard reviews can take up to a year.
In interviews this week at the J.P. Morgan Healthcare Conference in San Francisco, drugmaker executives and advisers said one of their chief concerns was that a two-month review could fall short of the scientific rigor needed to evaluate a medicine's safety and efficacy.
That could undermine the credibility of the FDA decision and create more legal risk for a drugmaker if patients experience serious side effects post-approval, they said.
One top pharmaceutical executive said their company would not seek a priority voucher because they were not convinced the program would endure beyond this administration and worried that FDA approvals could later be challenged in court. They spoke on condition of anonymity in order to describe concerns about a Trump administration initiative.
A Washington-based lawyer specializing in FDA regulatory affairs said a key concern is whether the voucher program will impact the agency's decisions on what adverse events or side effects need to be included on a medicine's prescribing information.
If plaintiffs can convince courts that the FDA label requirements are not reliable because they were issued too hastily, that will put more responsibility on manufacturers to warn about potential risks of using their products.
"This opens up greater risks for drug manufacturers seeking FDA approval for their products," the lawyer said.
DEEPER ISSUES WITHIN THE AGENCY
Richard Pazdur, a longtime FDA veteran, in an interview said questions about the voucher program were emblematic of deeper issues with the agency under Makary.
They include growing politicization of the agency's work, a lack of transparency over how some decisions are made, and a disregard for drug reviewers’ concerns, which he said ultimately drove him to leave the FDA in December.
"There are companies that came up to me at conferences and said, 'you know, they are not going ahead with it'," Pazdur said, of participation in the voucher program. Some companies asked whether it would be viewed negatively if they did not apply. He said he told them the FDA would expedite any deserving drug.
A Department of Health and Human Services spokesperson said "all decisions are grounded in evidence-based science and the agency continues to operate as a stable, science-based regulator".
WEIGHT-LOSS PILL ON PRIORITY LIST
The FDA has issued vouchers for 18 drugs so far, including Eli Lilly's widely anticipated weight-loss pill and products from Merck, GSK, Johnson & Johnson and Regeneron. Some candidates were nominated by the agency rather than their manufacturers seeking to participate.
Regeneron declined to comment on whether it sees legal risks associated with the program. The others did not immediately respond to requests for comment.
Only one drug - a generic antibiotic previously sold for years in the U.S. but later discontinued - has been approved under the program to date.
Reviewers are taking around four months to review some drugs in the program, including Lilly's pill, Reuters reporting shows. Lilly said its priority review began in early November. Reuters reporting showed a decision is now expected in April.
Reviews for two other drugs have been delayed after agency scientists flagged safety and efficacy concerns, including the death of a patient while taking one of the medicines, internal documents seen by Reuters show.
Supporters of the program include Eli Lilly research chief Dan Skovronsky.
“We’ve just started, so let’s see how it goes. But I’m excited about it," Skovronsky said in an interview. The program moves top-priority products to the front of the queue rather than cut corners, he said. "We welcome anything that gets medicines to patients faster.”
Still, he said, companies want to ensure the FDA remains "science-driven."
Pfizer is discussing potential participation in the program with the Trump administration, Bloomberg reported on Monday. Pfizer did not say which product it hoped to put through the pathway.
Industry experts say the hesitation of some drugmakers may be short-lived as more products move through the program.
"If this is a highly impactful drug that's safe and effective... and there aren't compromises in safety, review, or quality, just relatively faster, it'll be hard to unmake history," said Greg Graves, a senior partner in consultancy McKinsey's U.S. life sciences business.
A senior executive at another drugmaker, also speaking anonymously to avoid criticising the Trump administration or the FDA publicly, said the voucher program worries some because it ties the agency's role of regulating drug safety with pricing or other economic considerations. That has "never been in the FDA's purview," the executive said.
"A product shouldn't be approved because it has this price or that price," the executive said. "A product should be approved because there has been rigorous testing (and) it's safe and effective for patients."
(Reporting by Maggie Fick in San Francisco and Patrick Wingrove in New York, Additional reporting by Michael Erman and Sabrina Valle; Editing by Michele Gershberg and Bill Berkrot)
