Compassionate Access: No Good Answer

Here's a very good look (via the New York Times magazine) at the area of "compassionate use" medications - the practice of allowing desperate patients access to an investigational drug long before it's approved by the FDA. At first look, you'd think that this would be a simple question to answer: if someone's going to die shortly, they should be able to take a crack at whatever investigational drug they want, because what's to lose? But it's not that simple, unfortunately: But not all companies willingly allow compassionate access to drugs in their pipelines, and ImClone’s and AstraZeneca’s reluctance makes sense on some level. Only 6 percent of early-stage cancer drugs ever come to market, because many are found to have severe side effects or simply don’t work. Given those odds, companies hesitate to do anything to jeopardize a product too soon. If they give drugs away, a disastrous side effect or other poor outcome could spur bad publicity and extra scrutiny from regulators. Even more important, if doctors simply let people take untested medicines without going through all the clinical trials, drug companies would most likely never get anyone to enroll in them, never get the data on safety and efficacy for F.D.A. approval and never pass the gateway to big sales. “Even if patients with cancer are willing buyers,” writes George Annas, a Boston University expert on medical law, “drug manufacturers are not willing sellers.” As a public-health advocate, I know th...
Source: In the Pipeline - Category: Chemists Tags: Clinical Trials Source Type: blogs