I will be on vacation for the next week or so. Posts will resume on June 20th.

Why is NIH funding small trials? (there’s a good reason!)First bird flu death in humans.Employee responses to CEO activism.How rich were the rich?‘Hot hand’ and fantasy baseball.

Why is it so hard to estimate the value of orphan drugs indicated for the treatment of rare diseases? There are a variety of reasons, but a scoping review by Grand et al. (2024) provides a nice summary of these issues. Key challenges include small sample sizes for nearly all parameters and lack of data overall. More specifically, key issues identified...

Very interesting article from Sampson, Parkin and Devlin (2024). Hard to summarize but worth a read. I have two excerpts below. The first describes the role of death in QALY calculations. The value attributed to ‘dead’ cannot affect the QALYs that are gained in any context; by definition, no QALYs can be generated by a dead person. Consequently,...

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) provides the answer. …a...

That is the title of my article with Jaehong Kim, Shanshan Wang and Slaven Sikirica in the Journal of Comparative Effectiveness Research. The abstract is below. Aim: To assesses the cost–effectiveness of sotagliflozin for the treatment of patients hospitalized with heart failure and comorbid diabetes.  Materials & methods: A de novo cost–effectiveness...