Experimental Drugs and the Compassionate Use Doctrine

People with chronic illnesses and incurable diseases are often willing to do almost anything to improve their health. That includes seeking alternative treatments and trying unapproved drugs. While gaining access to drugs not yet approved by the Food and Drug Administration (FDA) can be a difficult task, many people will go to nearly any lengths to get them, to include suing a drug manufacturer to force it to provide them the drug. That's what happened in the case of Gunvalson v. PTC Therapeutics Inc., Slip Copy, 2008 WL 4003377 (D.N.J.,2008), a case recently overturned on appeal. In this month's issue, we look at the reasoning of the district court. In next month's issue, we'll see why the appellate court disagreed, and discuss the implications of the compassionate use doctrine for drug research and for individual patient outcomes.