Making Expanded Access Programs Work For –Not Against– Your Trial Drug

2010 Jun 7, E Hayes

Providing an investigational drug outside of a controlled trial carries inherent development risks, but it can also be a win-win proposition for patients and sponsors when managed well, veterans of "expanded access" programs say.

According to the FDA's broad definition, "expanded access" could mean a one-time supply of a drug to a particular patient, or a program that serves tens of thousands of patients. Globally, the terms "early access" and "compassionate use" are also used for similar programs.

Demand for access to investigational drugs is on the rise for a number of reasons. For one, it's become much easier for patients to access information about trial drugs and also to publicize their own experiences through social media. The globalization of the biopharma markets and a growing focus on development of treatments, including biologics, for less common disorders are also driving interest, said Carol Collins, corporate vice president of the Peri-Approval Clinical Excellence program at Parexel.

Another key factor is the evolving regulatory environment. FDA has widely publicized guidance on three paths toward gaining access outside of trials for patients who do not qualify for ongoing studies.

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