FDA Expands Access to Investigational Drugs for Treatment Use

Background

In response to the need for broader access to investigational drugs for patients with serious or immediately life-threatening diseases or conditions, the FDA has amended its regulations. This move is aimed at addressing the lack of formal recognition of treatment use in FDA's regulations concerning Investigational New Drugs (INDs) before 1987. The 1987 IND regulations revision explicitly provided for treatment use of investigational drugs under specific criteria. The Food and Drug Administration Modernization Act of 1997 (FDAMA) further emphasized the importance of expanded access to investigational drugs for treatment use.

Overview of the Final Rule

The final rule amends FDA regulations by detailing the ways expanded access to treatment use of investigational drugs is available, including for individual patients, intermediate-size patient populations, and treatment INDs or treatment protocols. It outlines criteria for authorization, submission requirements, and safeguards to protect patients and preserve the ability to develop meaningful data about treatment use.

Changes to the Proposed Rule

In response to comments, the final rule clarifies that it applies not only to investigational new drugs but also to approved drugs whose availability is limited by a risk evaluation and mitigation strategy (REMS). It also clarifies that the rule applies to all patients with a serious disease or condition, regardless of their current health status.

Comments on the Proposed Rule

The FDA received 119 comments on the proposed rule, with most supporting the goal of expanding access to investigational drugs for treatment use. Concerns were raised about the administrative burdens, equitable access, and the supply of investigational drugs. The FDA addressed these concerns by emphasizing the importance of patient safety, the need for equitable access, and the potential for using mechanisms to fairly allocate limited drug supply.

Legal Authority and Environmental Impact

The rule is issued under the authority of the Federal Food, Drug, and Cosmetic Act and the Public Health Service Act. The FDA has determined that this final rule will not have a significant impact on the human environment.

Analysis of Economic Impacts

The FDA analyzed the economic impacts of the final rule, concluding that the benefits of providing access to investigational drugs for treatment use outweigh the costs. The rule is expected to have a positive impact on public health by allowing patients with serious or life-threatening conditions to access potentially life-saving treatments.

Conclusion

The FDA's final rule on expanded access to investigational drugs for treatment use represents a significant step forward in ensuring that patients with serious or immediately life-threatening diseases or conditions have access to potentially beneficial treatments. By detailing the criteria, submission requirements, and safeguards for expanded access, the FDA aims to increase awareness and knowledge of these programs, ultimately facilitating wider availability of investigational drugs in appropriate circumstances.