Unbiased Analysis of Today's Healthcare Issues
These are becoming increasingly common. Agrawal et al. (2022) use data between 2002-2021, and found that FDA approved: …176 new malignant hematology and oncology indications based on single-arm trials, including 116 accelerated approvals (AAs) and 60 traditional approvals. Overall, 87 approvals (49%) were for new molecular entities or original biologics and 89 (51%) were supplemental…
While randomized controlled trials (RCTs) are the gold standard of experimental research, there are some limitations. For instance, in some cases use of an RCT is not feasible (e.g., the sample size may be too small as in the case of rare disease), not ethical (e.g., if the previous standard of care is not effective)…
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…
What outcomes should be used to measure if a medicine is a “good” medicine? Improved survival? Improved ability to function? Few side effects? Ability to get back to work? Living to attend your daughter’s wedding? All of the above? Oftentimes, health economists measure value of a treatment for the average person. Preferences for efficacy, safety,…
FDA draft guidance published this month says you should. In most cases, adjusting for covariates is not necessary. Randomization generally insurers that covariates are balanced across clinical trial arms. Randomization, however, may not always result in perfectly balanced trial arms. In these cases, the FDA notes that covariate adjustment is perfectly acceptable. There are some…
The cost to bring a drug to market is a hotly debated topic. The costs include not only the costs for basic science and clinical trials. However, one must also take into account the cost of capital (since most cost are incurred up front, but revenue begins only after drug approval) and the likelihood of…
Clinical trials are typically of (relatively) short duration, but innovative treatments may impact patient survival over multiple years. Health economists and outcomes researchers often are faced with the challenge of extrapolating clinical trial survival curves to estimate long-term survival gains for the typical patient. These estimates may be done parameterically (e.g., exponential, Weibull, Gompertz, log-logistic,…
Should we infect people with COVID-19? Although most people would say ‘no’, this prospect could help speed the development of a vaccine. In these trials, half the people would be treated with a test vaccine for COVID-19 and half the people would receive a placebo. Then, people in both arms would be infected with COVID-19…
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…
How do you design a good clinical trial? Clearly, the trial design depends on what question you are answering. For instance consider the dichotomy between an explanatory and pragmatic trial: A pragmatic randomised trial is undertaken in the “real world” and with usual care and is intended to help support a decision on whether to…