Regulation – Healthcare Economist

How does FDA recommend using Bayesian Statistics to inform Regulatory Decisionmaking around clinical trials?

2026-02-04Jason Shafrin

FDA’s January 2026 draft guidance on Bayesian methodology in drug and biologics trials signals a clear willingness to see Bayesian methods used for regulatory decisionmaking around clinical trials. The guidance lays out how sponsors (i.e., pharmaceutical manufacturers) should pre-specify priors, success criteria, and simulations so that Bayesian designs remain interpretable, control error rates when needed,…

What are FDA’s Guiding Principles of Good AI Practice in Drug Development?

2026-01-29Jason Shafrin

Here they are: Human-centric by design. The development and use of AI technologies align with ethical and human-centric values. Risk-based approach. The development and use of AI technologies follow a risk-based approach with proportionate validation, risk mitigation, and oversight based on the context of use and determined model risk. Adherence to standards. AI technologies adhere…

Can patient preference information influence regulatory decision-making?

2025-11-24Jason Shafrin

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,…

What real-world data will regulators consider in Europe?

2025-11-202025-11-20Jason Shafrin

A 2024 Data Quality Framework (DQF) from the European Medicines Agency (EMA) aims to provide some guidance for drug manufacturers. Here is a summary of that document. Factors to be considered when evaluating RWD Key factors to be considered in the EMA’s DQF include: Reliability: Are the data correct? Extensiveness: Are the data enough Coherence:…

Change to UK Rare Disease Approval Process

2025-11-03Jason Shafrin

The Times reports that the UK’s Medicines and Healthcare products Regulatory Agency (MHRA) is revising it’s approach to how it approves new medicines that treat rare diseases. How? Principally by allowing for single arm trials with real-world ‘digital twin’ controls as well as expediting the approval process. The new plan, which is being launched on…

How often does FDA use expedited regulatory pathway for oncology drugs?

2025-09-012025-09-01Jason Shafrin

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.…

What are the health benefits of the accelerated approval pathway for new cancer treatments?

2025-07-302025-07-30Jason Shafrin

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…

Do Gun-Purchase Waiting Periods Save Lives?

2025-04-30Jason Shafrin

That is the title of a paper by Arnold and Priestley (2025). They examine whether gun purchase wait periods impact suicide rates using both variation in gun laws by state over time as well as variation in a county’s distance to the nearest state without a waiting period. First, some background on US gun purchase…

Are Trump/DOGE cuts to FDA staff impacting drug approval timelines?

2025-04-29Jason Shafrin

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…

Comparing Regulatory and Health Technology Assessment Reviews of Medicines in the United States and Europe

2025-02-122025-02-12Jason Shafrin

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…