Compassionate Use Access to Promising Drugs -Mid-October2009

[Fictional]

The FDA recently updated its policy regarding compassionate use access for individuals with serious or life-threatening illnesses. Some FDA officials have said that it may mean promising drugs even in Phase I may become accessible to patients - this is an important issue for ASPS because the trials are often few and difficult traveling to get access to promising drugs. The thing to keep in mind, though is that companies usually don't like giving their drug away without patients being 'on study' - they can't use the data if it works and they worry that patients will all prefer compassionate use over enrolling in a trial e.g. where randomized to placebo. There is an added legislation though that says the company can charge somewhat for the drug (which reduces the disincentive of companies having to give away 'free' drug), but I don't know what that would translate into. I don't think they can charge as much for a drug that is unproven. The FDA has decreed they cannot make a profit from the drug given under expanded access.

Anyway - something to think about as there are now several promising drugs - and still often very sites to enroll etc. The guidelines go into effect in mid-October.

http://www.fda.gov/Drugs/DevelopmentApp ... 172492.htm

http://online.wsj.com/article/SB1000142 ... smartbrief