Unbiased Analysis of Today's Healthcare Issues
Diversity in clinical trials Recently, the FDA’s Center for Drug Evaluation and Research’s (CDER’s) released its 2020 Drug Trials Snapshots Summary Report. Part of the report examines the demographics of participants in clinical trials for the 53 drugs that FDA approved in 2020 either as new molecular entities (NMEs) under new drug applications (NDAs) or…
How do you evaluate treatment efficacy and safety outside of the clinical trial setting? This is not just a question of academic interest. In last week’s JAMA, Rubin 2021 writes about some of the challenges of evaluating COVID-19 vaccines outside of a clinical trial setting. In an interesting, two-day workshop last week, the Duke Margolis…
The FDA approval process is slow. Even for COVID-19 vaccines, the process can be time consuming. However, regulators around the world do have some expedited programs. However, a number of countries have programs that expedite review, particularly for the diseases with unmet need, orphan drugs, or when new treatments represent a significant advance over the…
In many cases, the answer is ‘no’. At least that is the findings from a paper by Ryan and Sood (2019). As reported in FiercePharma: Sood and Ryan reviewed 166 drug pricing laws passed between 2015 and 2018 and found that 35 bills in 22 states included transparency mandates. Only seven of the laws were…
Not surprisingly, the answer is yes, but not by as much as one might guess. In part due to a measles outbreak at Disneyland, California outlawed non-medical exemptions for vaccinations. An NBER working paper by Richwine et al. (2019) provides background on the situation in California prior to enacting the ban. Prior to the policy…
That is the title of my recent piece in STAT News. Earlier this month, the Food and Drug Administration announced the creation of Project Facilitate. This pilot program facilitates access to innovative treatments for cancer that have not yet been approved by individuals who aren’t able to enroll in clinical trials. This move suggests that the…
This week, the UK’s National Health Service (NHS) and National Institute for Health and Care Excellence (NICE) released a document titled Evidence standards framework for digital health technologies. An accompanying piece in The Lancet by Greaves et al. (2018) aims to describe the goals of the document at a high level. The authors state that key…
Darius Lakdawalla has an very interesting review article in the Journal of Economic Literature on the Economics of the pharmacuetical industry. Do read the whole thing, but below I have listed some highlights. A model of firm R&D decisions Lakdawalla uses a simple model based on Nordhaus (1969) to derive some important implications about pharmaceutical…
The government strictly regulates what information that pharmaceutical firms can share with payers. There is good reason for this. We don’t want drug companies making false claims that a drug can treat/cure a disease when there is no evidence to support that claim. Randomized controlled trials are the gold standard of clinical evidence and studies…
How frequently are pharmaceuticals used off label? Perhaps more than you think. Although these figures are a bit dated, Tabarrok (2000) details the extent of off-label prescribing in the U.S. as follows: According to a study by the U.S. General Accounting Office, 56 percent of cancer patients have been given non-FDA-approved prescriptions, and 33 percent…