To Speed Access To Compassionate Use, Look Beyond The FDA

In mid-July, the nonpartisan research arm of the federal government, the Government Accountability Office (GAO), released its long-awaited verdict on how the Food and Drug Administration (FDA) is handling compassionate use. As anyone who saw Dallas Buyers Club knows, compassionate use (also known as pre-approval access and expanded access) is when a drug company allows a patient who has no other treatment options to try a drug that is still in development and not FDA approved for use or sale. These patients are generally too sick to participate in a clinical trial—which has inclusion and exclusion criteria to try to keep the trial data from getting too “messy”—but if their physician, the drug company, the FDA, and their physician’s institution (in that order) all permit the effort, they can try these drugs in development through compassionate use. On and off over the past decades, the FDA has come under fire for being too slow or too bureaucratic in handling compassionate use requests. One of the major recent critics is the libertarian Goldwater Institute, which published a policy report stating that: “For patients suffering from terminal illnesses, the federal Food and Drug Administration (FDA) is the arbiter of life and death. … The FDA, however, often stands between the patients and the treatments that may alleviate their symptoms or provide a cure. To access these treatments, patients must either go through a lengthy FDA exemption process or wa...
Source: Health Affairs Blog - Category: Health Management Authors: Tags: Drugs and Medical Innovation Population Health Quality Compassionate Use expanded access Food and Drug Administration right-to-try laws Source Type: blogs