User Fees
Pink Sheet editors and special guest Michael McCaughan of Prevision Policy discuss Robert F. Kennedy Jr.’s first act as HHS Secretary, former FDA Commissioner Scott Gottlieb’s suggestion that industry extend the existing user fee agreement rather than negotiate a new one, and President Trump’s policies impacting FDA advisory committee schedules and staffing.
Given the Trump Administration’s intense focus on cutting costs and increasing efficiency, industry and the FDA should focus on what is and is not working in PDUFA VII when negotiating the next agreement, lobbyists said at the BIO CEO and Investor Conference.
Experts worry new restrictions on remote work, plans to eliminate certain employees’ job protections and workforce cuts will make the FDA a less attractive employer and hurt pharma’s efforts to get innovative medicines to patients.
Top areas for potential 2025 approvals were shaped by R&D focused on increasingly tightly targeted therapies, including the eight new candidates to join the still-burgeoning kinase inhibitor class and RNA interference, as well as many varieties of antibodies.
CBER has at least 14 and CDER another 10 novel biologics among the more than 60 candidates with a user fee goal in 2025.
Trump’s former FDA commissioner sees ‘nothing good’ coming from negotiating a new user fee agreement in the ‘current political environment.’
Among the more than 100 the drug center actions while the FDA's communications have been constrained are approvals of two novel agents, two new indications, and a new combination therapy.
Only one-third of novel agents with 2025 goal dates come from the traditional oncology, hematology and neuroscience strongholds. Immunodermatology also is cooling, while cardiovascular drugs return to the front burner.
Prospects are promising for many of the 16 novel candidates that received complete response letters in 2024 after half of the 2023 CRL class was approved the following year.
Novel agents from the biologics center had a median review time of 10.6 months, beating the drugs center's median of 11.8 months thanks to fewer multicycle and more priority reviews.
The rush to submit applications before user fees increased may have motivated the sudden drop, a generic industry trend that continues to grow.
The December calendar features nearly 20 user fee goal dates, from Novo Nordisk’s Alhemo to Lexicon’s Zynquista.
While annual fees for marketing authorization applications and annual payments to the European Medicines Agency are set to increase from 2025, micro-sized companies and SMEs that make orphan drugs will still benefit from full reductions.
Pink Sheet reporter and editors discuss concerns that the incoming administration could try to influence the upcoming FDA user fee reauthorization, as well as the changes Dr. Mehmet Oz, President-elect Donald Trump’s controversial selection to run the US Centers for Medicare and Medicaid Services, could impose if confirmed.
Expected HHS Secretary nominee Robert F. Kennedy Jr.'s dislike of user fees and interest in major FDA reforms could come to a head in the 2027 PDUFA reauthorization.
The FDA’s Peter Marks warned novel approvals will suffer if the agency is forced to spend its time relitigating vaccines. At the top HHS post Kennedy could impact pharma from basic research funding to drug pricing.
Another record-breaking year for novel approvals looks out of reach, but the six novel agents with November goal dates show the continued strength of rare disease drug development.
The number of standard and priority reviews also decreased significantly in FY 2023 compared to the previous year, which caused the fee for redeeming a voucher to rise.
Advisory committee concerns cast clouds over Iterum’s oral antibiotic, Intercept’s Ocaliva, and perioperative immuno-oncology regimens, while CSL and Pfizer aim to take their hematology franchises in new directions.
Inflation accounted for a larger portion of user fee revenue target increases for fiscal year 2025, compared to previous years, according to a Pink Sheet analysis.