Amid a period of upheaval at the FDA, several pivotal drug approval news items are making waves. For example, the agency recently released two draft guidance documents that will impose new restrictions on the details of the Accelerated Approval program. The Accelerated Approval program allows drugs that treat serious and unmet medical needs to be approved based on surrogate endpoints while requiring ongoing confirmatory trials. It has led to significant breakthroughs in oncology and rare diseases, but it has also generated concerns about high drug prices, delayed follow-up studies and uncertainty over clinical benefits in some cases.

The new draft guidances will require that sponsors align promotional claims with verified benefits and will review all materials before they are distributed. The FDA will also strengthen its oversight of accelerated approvals and their confirmatory trials. These changes will have major implications for market strategies and pricing strategies. A new study analyzing 129 cancer drugs granted accelerated approval between 2013 and 2023 found that 63% of those drugs were converted to regular approval, with most being withdrawn due to lack of clinical benefit. Drugs like Clolar (clofarabine) and Xydelig (idelalisib) illustrate the danger of using surrogate or intermediate endpoints to approve therapies.

This year, the FDA has already approved 50 novel drugs, a slight decrease from last year’s total of 55. The majority of those approved were first-in-class medicines, including the first new schizophrenia drug in three decades and the first treatment for metabolic-associated steatohepatitis (MASH). In addition, a number of drugs received Breakthrough Therapy designation, signaling accelerated development. At Serve You Rx, we track these trends to help our clients optimize their pharmacy benefits and cost management strategies.