Whatever your opinion of the FDA is you really can’t fault the agency for trying to get new therapies to patients as quickly as possible.
Here’s a brief rundown of some of the programs available:
Fast Track is a process designed to facilitate the development, and expedite the review of drugs to treat serious conditions and fill an unmet medical need.
Breakthrough Therapy designation is designed to expedite the development and review of drugs that are intended to treat a serious condition and preliminary clinical evidence indicates that the drug may demonstrate substantial improvement over available therapies.
Accelerated Approval regulations allow drugs for serious conditions that filled an unmet medical need to be approved based on a surrogate endpoint that enables faster approval.
Priority Review designation means FDA’s goal is to take action on an application within 6 months - compared to the usual 10 months.
When a drug gets one of these designations, it’s fairly common for the developing company to announce this to the press. After all, achieving one of these designations makes the company look good both to the public in general and its stockholders – a win all around.
But there is one FDA designation which has been getting some notice recently due to its lack of use. Expanded Access, sometimes called "Compassionate Use," is the use of an investigational drug outside of a clinical trial to treat a patient with a life-threatening disease who has no comparable or satisfactory alternative treatment options.
Recently, there have been at least two cases where patients have appealed to companies to allow access to drugs under the Compassionate Use designation and have been turned down.
Personally, I think this is wrong. If the denials were based on legal or safety concerns, I’m sure these issues could be worked out.
If companies are quick to announce when developing products receive one of the other designations – I’m sure the public relations benefits of a compassionate use tag would far outweigh any negatives.
What do you think?