A group of legislators requested information on Wednesday from FDA Commissioner Margaret Hamburg about patient access to therapies that are not approved by the FDA to treat life threatening or terminal conditions.
Sens. Lamar Alexander (R-Tenn.), Tom Coburn (R-Okla.) and Richard Burr (R-N.C.) requested data from Hamburg on the risks and benefits related to the FDA’s expanded access program.
“We are interested in understanding how the expanded access, or ‘compassionate use,’ program at the U.S. Food and Drug Administration is serving patients who seek access to new, unapproved therapies outside of clinical trials to treat serious or life threatening conditions,” the senators said. “The work of our nation’s biomedical research infrastructure and potential for medical innovation on behalf of patients is astounding. Yet, each day patients nationwide are burdened by diseases and conditions that no FDA-approved products can cure or treat.”
Many patients who face terminal or life threatening medical conditions turn to therapies that have not completed clinical trials or other requirements of the FDA approval process, the senators said.
The senators also requested information related to investigational new drugs (INDs), specifically on the number of requests for their use, the number of request approvals, average length of the approval process, conditions the drugs were approved for and the effectiveness of the INDs.
“We look forward to working with (the FDA) on this issue to ensure the FDA’s compassionate use program is operating well for patients, providers and the agency,” the senators said.