Activists Call on FDA to Balance Safety, Access to Investigational Drugs

Two proposed rules from the FDA strike a "generally reasonable balance" between allowing patient access to investigational drugs and ensuring patient safety, although some changes still need to be made, Public Citizen said.

The group is concerned about the agency's definition of which patients can have access to investigational drugs and when, Public Citizen said in a letter to the FDA. The agency also needs to make information on investigational drugs publicly available, including results from use, the group added.

The FDA is proposing to expand access to investigational drugs for patients with "serious or life-threatening illnesses." While the agency provides a sufficient definition for "life-threatening illnesses," it does not specify which illnesses are "serious," Public Citizen said.

Public Citizen said it approves of the agency's decision that the FDA should require less evidence for approving investigational drugs for the most serious illnesses and more evidence for drugs for less serious diseases. However, the threshold of required evidence is too low for patients with immediately life-threatening diseases, the group said. Under the current recommendations, a patient could receive the first human exposure to a drug, which is too dangerous, Public Citizen said.

In addition, the agency needs to disseminate the information included in its proposed rule in different ways, not just as a Federal Register notice, Public Citizen said. The group also recommended that the FDA make the safety and efficacy data on the investigational drugs publicly available.