Unbiased Analysis of Today's Healthcare Issues
That is according to the FDA. Nature Reviews Drug Discovery summarizes the progress to date as follows. The FDA’s Center for Drug Evaluation and Research (CDER) and Center for Biologics Evaluation and Research (CBER) approved 57 new therapeutic drugs (NTD) in 2024. This is slightly above the average of 53 NTDs per year for 2014–2024,…
A study by Fermaglich and Miller (2023) evaluated trends in orphan drug designations and approvals after the Orphan Drug Act of 1983 was passed in the US. The authors use FDA data to evaluate between 1983 and 2022 and find that: Over the 40 years of the ODA, 6,340 orphan drug designations were granted, representing…
In July 2018, FDA released the Biosimilars Action Plan (BAP), which outlined FDA’s approach for expanding access to biosimilars for the American public. The plan focused on 4 key areas: A recent FDA report reviews some of their accomplishments since then. Most of these efforts revolve around guidance documents, additional staff, education products and websites,…
It appears changes are coming to the FDA Advisory Committee process for evaluating new drugs. The FDA is planning significant reforms to its advisory committee process, as announced by Commissioner Rob Califf. While specifics are scarce, these changes are spearheaded by Namandjé Bumpus, the FDA’s chief scientist, and newly appointed deputy commissioner.Califf noted improvements in…
Last week, FDA authorized the State of Florida to import pharmaceuticals from Canada. The price of branded pharmaceuticals in the US is nearly three times the cost of identical pharmaceuticals in Canada, so this approach could save the US money. While there are some administrative issues to work out on the US side, the biggest…
2023 was a strong year for drug discovery. Mullard et al. (2024) report: The FDA’s Center for Drug Evaluation and Research (CDER) approved 55 new drugs in 2023, as the small molecule and biologic pharmacopoeia continues to grow. This cohort is nearly 50% bigger than the new approval class of 2022, which fell below the…
Last month, FDA released guidance on the use of real-world evidence to support regulatory decision-making for new drugs. Below are some of the key points from this guidance document: Legislative mandate. The 21st Century Cures Act (Cures Act), signed December 2016, mandated that the FDA consider how to incorporate real world data (RWD) into regulator…
That is the title of a very interesting paper by Casey Mulligan (2023) [NBER WP version]. The full title includes the phrase “Peltzman revisited”. This refers to a paper by Peltzman (1973).When speaking of the 1962 “Drug Efficacy Amendment” (EA) to the Federal Food, Drug, and Cosmetic Act a key piece of research was Peltzman…
The year 2022 was a good, but perhaps not great year for innovation. According to the FDA’s Center for Drug Evaluation and Research’s (CDER) New Drug Therapy Approvals 2022 report, there were 37 novel drugs approved in 2022. This number is down from the historical rate of approvals between 2013-2021 (43.4 approvals per year) and…
As part of The 21st Century Cures Act, FDA is required to consider additional mechanisms to expedite access to new drugs, including those currently under investigation. In fact, the 21st Century Cure Act mandates that pharmaceutical manufacturers have to post their policies on expanded access (a.k.a. “compassionate use”) for investigational drugs by: (1) the first…