Unbiased Analysis of Today's Healthcare Issues
In short, ‘yes’. Barry Linden (2024) provides a nice overview of FDA’s approach to patient preference information (PPI): FDA is very open to doing this and they’re encouraging sponsors to use this kind of data to help them. The FDA’s desire to incorporate patient perspectives into their risk-benefit decision-making process started during the HIV/AIDS crisis,…
FDA uses expedited regulatory pathways (ERPs) in order to accelerate the availability of drugs and diagnostic tests for severe conditions that have unmet medical needs. How often is this process used and has this changed over time? A recent paper by Horn et al. (2025) provides the answer as applied to colorectal cancer (CRC) drugs.…
That is the question that a paper by Wong et al. (2024) aims to answer. FDA use the accelerated approval (AA) pathway to expedite the availability of drugs for serious or life-threatening conditions when a drug demonstrates an effect on a surrogate endpoint that is reasonably likely to predict clinical benefit, or on a clinical…
Last week, the Congressional Budget Office (CBO) released a report titled “How Changes to Funding for the NIH and Changes in the FDA’s Review Times Would Affect the Development of New Drugs.” The report evaluates two scenarios: A permanent 10 percent reduction in the amount of funding that the government provides to the NIH, and…
It seems like the answer is now ‘yes’. EndPoints reports: Stealth BioTherapeutics said the FDA would not meet its Tuesday deadline for approving or rejecting the company’s drug, elamipretide. It had been seeking regulatory clearance of elamipretide for a rare disease called Barth syndrome, which affects only about 150 people in the US and causes…
That is the subtitle of a paper by Vreman et al. (2020). The authors look at all treatments approved by FDA and EMA that were subsequently reviewed by HTA bodies in both jurisdictions between 1995 and 2018. The authors used ICER as the US HTA body; European HTA bodies included IQWiG (Germany), NICE (UK), ZIN…
The answer to this question depends, not only on how well AI tools work, but also on how AI technologies are regulated by the FDA. Scott Gottlieb has some thoughts on preferred regulatory approaches in his recent article in JAMA Health Forum. Artificial intelligence tools with advanced analytical capabilities used in clinical practice, especially tools…
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,…