Back in the 1980s when the HIV epidemic was sweeping through large cities and there were few treatment options available, there was a great deal of agitation at the grassroots level for the FDA to reform the process and do two things – (1) speed up approval of new drugs and (2) expand access to drugs before their approval. There were two results PDUFA and FDAMA – FDA reforms that brought us priority review and accelerated approval.
Priority review is the consideration of a new drug application within a 6-month time period, as opposed to the traditional 10-month period. Accelerated approval is the provisional approval of a drug prior to the time when all of its clinical trials are yet finished and reviewed to allow access without marketing to populations without treatment options.
This week, the FDA has proposed new rules to overhaul expanded access to experimental drugs as well as addressing the charges for the drugs used under such programs.
The proposed rule is open for public comment for a period of 90 days.
The aim of the changes, according to the news release, are:
1 – modernize applicable regulations to include all circumstances under which access to experimental drugs is permitted
2- make experimental drugs more widely available in appropriate situations by establishing criteria that link the level of evidence needed to support the use of an experimental drug, and
3- revise the current regulation regarding manufacturers’ recovery of the costs associated with the use of an experimental drug.
This is a great thing for patients suffering from life-threatening illnesses with few treatment options.
The next step is for the FDA to be packaging these kinds of positive steps and announcements into a coherent plan that works to restore public confidence in the agency. As a stand alone milestone, it is great, but don’t expect people to connect the dots and believe that the agency has turned a corner. That takes a plan.
