Unbiased Analysis of Today's Healthcare Issues
Conventional wisdom holds that it takes 17 years from medical innovation to adoption in the real-world. However, with advances in information technology, perhaps adoption timelines have shortened. A paper by DiCicca et al. (2024) examines how new clinical trial evidence on breech births impact practice patterns. Specifically, they note that: Using the United States Birth…
Delays in drug approvals cost pharmaceutical firms money. These costs include both lost sales as well as additional cost if clinical trials need to span a longer duration. While these facts are not in doubt, a key question is how much do delays in approval cost firms? A paper by Smith, DiMasi and Getz (2024)…
Progression-free survival (PFS) is a surrogate endpoint is defined as follows (by ChatGPT): In essence, PFS refers to the length of time during a clinical study or treatment regimen where a patient’s disease doesn’t worsen or progress. For instance, in cancer research, it measures the time from the start of treatment until the cancer shows…
A paper by Meyer et al. (2020) has a nice summary of potential mechanisms through which the presence of COVID-19 could impact clinical trial results. What should a researcher do when a clinical trial is impacted by COVID-19? Here are some analysis considerations. The full article is interesting throughout.
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…