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Statement of Need: The Food and Drug Administration Modernization Act of 1997 (Modernization Act) amended the Federal Food, Drug, and Cosmetic Act (the Act) to include specific provisions concerning expanded access to investigational drugs for treatment use. In particular, section 561(b) of the Act permits any person, acting through a licensed physician, to request access to an investigational drug to diagnose, monitor, or treat a serious disease or condition provided that a number of conditions are met. The rule is needed to incorporate into FDA’s regulations this and other provisions of the Modernization Act concerning access to investigational drugs. In addition, the agency seeks to increase awareness and knowledge of expanded access programs and the procedures for obtaining investigational drugs for treatment use. The rule will assist in achieving this goal by describing in detail the criteria, submission requirements, and safeguards applicable to different types of treatment uses.

Summary of the Legal Basis: FDA has the authority to impose requirements concerning the treatment use of investigational drugs under various sections of the Act, including sections 505(i), 561, and 701(a) (21 U.S.C. 355(i), 360bbb, and 371(a)). Section 505(i) of the Act directs the Secretary to promulgate regulations exempting from the operation of the new drug approval requirements drugs intended solely for investigational use by experts qualified by scientific training and expertise to investigate the safety and effectiveness of drugs. The rule explains procedures and criteria for obtaining FDA authorization for treatment uses of investigational drugs. The Modernization Act provides significant additional authority for this rulemaking. Section 561(a) states that the Secretary may, under appropriate conditions determined by the Secretary, authorize the shipment of investigational drugs for the diagnosis, monitoring, or treatment of a serious disease or condition in emergency situations. Section 561(b) allows any person, acting through a physician licensed in accordance with State law, to request from a manufacturer or distributor an investigational drug for the diagnosis, monitoring, or treatment of a serious disease or condition if certain conditions are met. Section 561(c) closely tracks FDA’s existing regulation at 21 CFR part 312.34 providing for treatment use by large patient populations under a treatment protocol or treatment IND if a number of conditions are met. Section 701(a) provides the Secretary with the general authority to promulgate regulations for the efficient enforcement of the Act. By clarifying the criteria and procedures relating to treatment use of investigational products, this rule is expected to aid in the efficient enforcement of the Act.

Alternatives: One alternative to this rulemaking that FDA considered was not to promulgate regulations implementing the expanded access provisions of the Modernization Act. However, the agency believes that promulgating regulations would further improve the availability of investigational drugs for treatment use by providing clear direction to sponsors, patients, and licensed physicians about the criteria for authorizing treatment use and what information must be submitted to FDA. Another alternative FDA considered was a regulation describing only individual patient and large scale expanded access criteria. However, the agency concluded that it would be preferable to have a third category of expanded access for intermediate-size patient populations.

Anticipated Costs and Benefits: FDA expects that the costs of the rule will range from a low of about $109,350 to $218,700 in the first year following implementation of the final rule, to a high of about $325,500 to $567,825 in the fourth and fifth years. These estimates suggest that total annual costs for the rule would be between $1.2 million and $2.2 million for the 5-year period following implementation of the final rule. The agency also expects that the estimated incremental cost burdens associated with this rule are likely to be widely dispersed among affected entities. The benefits of the rule are expected to result from improved patient access to investigational drugs generally and from treatment use being made available for a broader variety of disease conditions and treatment settings. In particular, the clarification of eligibility criteria and submission requirements would enhance patient access by easing the administrative burdens on individual physicians seeking investigational drugs for their patients and on sponsors who make investigational drugs available for treatment use.

Risks: The agency foresees no risks associated with the rule.