Unbiased Analysis of Today's Healthcare Issues
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…
Today, the Food and Drug Administration (FDA) approved the drug Relyvrio (sodium phenylbutyrate/taurursodiol; also known as AMX0035), which is indicated for the treatment of patients with amyotrophic lateral sclerosis (ALS), also known as Lou Gehrig’s disease. Most people with ALS die within 2-5 years of diagnosis. In justifying the approval, FDA’s press release stated: “This…
FDA has two competing priorities. On the one hand, they want to collect as much data as possible to insure drugs are safe and effective; on the other hand, patients want access to innovative medicines as quickly as possible. To try to balance these priorities, FDA often will approve treatments based on evidence from surrogate…