Unbiased Analysis of Today's Healthcare Issues
That is the title of a Journal of Economic Literature article by Gottlieb and Nicholson (2026). Here is a Q&A summary of their article. What are the defining features of physician competition? The authors argue that competition in the physician market is a two-stage process: “First, potential physicians compete to enter medical school and then…
The answer is provided by FDA’s 2025 Drug Trials Snapshots Summary Report. The report covers 46 novel drug approvals and roughly 26,000 participants across pivotal trials, organized by therapeutic area rather than a single aggregate narrative. Half (23 out of 46) of these approvals were for the treatment of rare or orphan diseases (i.e., diseases…
FDA’s January 2026 draft guidance on Bayesian methodology in drug and biologics trials signals a clear willingness to see Bayesian methods used for regulatory decisionmaking around clinical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,…
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…
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,…
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:…
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…
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…
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…