Do orphan drugs deliver more survival gains per patient than non-orphan drugs?

Conventional wisdom holds that orphan drugs treat rare (by definition) and more severe diseases. Because they treat diseases with significant unmet needs, their health benefits per person are large. But are they really? Does conventional wisdom align with the evidence? To answer the question myself and colleagues at FTI Consulting, including co-author Marie Steele-Adjognon, conducted…

Links

Sanofi joins 340B lawsuit. Kaiser behavioral health workers on strike. Apologia of PBMs. Status of brain-computer interfaces.  How old is the moon?

What is HEMA?

The Pink Sheet reports on a new international health technology assessment (HTA) body: Health Economics Methods Advisory group (HEMA). The Health Economics Methods Advisory group (HEMA) is a new, independent group of health technology assessment agencies from the US, England and Canada that will work together to develop recommendations on adopting novel health economic methods…

Speculation on RFK Jr. as HHS Secretary

From Tyler Cowen at Marginal Revolution: One of the problems with an RFK Jr. ascendancy is that his core views, which run strongly against vaccines and pharmaceuticals, make it unlikely that any of these reimbursement revisions will be done in a rational or scientific way. The best evidence indicates that pharmaceuticals are a relatively cost-effective ways of saving…