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HHS/FDA RIN: 0910-AF14 Publication ID: Fall 2005 
Title: Expanded Access to Investigational Drugs for Treatment Use 
Abstract: To amend the regulations governing investigational new drugs (INDs) to describe the ways patients may obtain investigational drugs for treatment use. Treatment use of investigational drugs would be available to: (1) individual patients, including in emergencies; (2) intermediate size patient populations; and (3) larger populations under a treatment protocol or IND. 
Agency: Department of Health and Human Services(HHS)  Priority: Other Significant 
RIN Status: Previously published in the Unified Agenda Agenda Stage of Rulemaking: Proposed Rule Stage 
Major: No  Unfunded Mandates: No 
CFR Citation: 21 CFR 312.42    21 CFR 312.300    21 CFR 312.305    21 CFR 312.310    21 CFR 312.315    21 CFR 312.320   
Legal Authority: 21 USC 321    21 USC 331    21 USC 351 to 353    21 USC 353    21 USC 355    21 USC 371    42 USC 262   
Legal Deadline:  None

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 proposed rule is needed to incorporate into FDAs regulations this and other provisions of the Modernization Act concerning access to investigational drugs. In addition, by this proposed rule, the agency seeks to increase awareness and knowledge of expanded programs and the procedures for obtaining investigational drugs for treatment use. The proposed rule would 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 proposed rule explains procedures and criteria for obtaining FDA authorization for treatment uses of investigational drugs. The Modernization Act provides significant additional authority for this proposed rule. 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 existing FDAs existing regulation at 21 CFR 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 proposed rule is expected to aid in the efficient enforcement of the act.

Alternatives: One alternative to the proposed rule that FDA considered was not to propose regulations implementing the expanded access provisions of the Modernization Act. However, the agency believes that implementing 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 to propose a regulation describing only those types of treatment use that are specifically described in the Modernization Act. However, the agency concluded that it would be preferable to establish, as authorized by the Modernization Act, a third category of treatment use that would be used for more than an individual patient, but fewer than the large numbers of patients in treatment INDs or treatment protocols.

Anticipated Costs and Benefits: FDA expects that the total one-time costs of the proposed rule will be negligible. The agency expects that the annual and annualized costs of the proposed rule will range from a low of about $130,000 to $260,000 in the first year following publication of any final rule based on this proposal, to a high of about $350,000 to $690,000 in the fourth and fifth years. These estimates suggest that total annual and annualized costs for the proposed rule would be between $1.4 million and $2.7 million for the 5-year period following implementation of any final rule based on this proposal. The agency also expects that the estimated incremental cost burdens associated with this proposed rule are likely to be widely dispersed among affected entities. The benefits of the proposed 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 proposed rule.

Action Date FR Cite
NPRM  04/00/2006    
Regulatory Flexibility Analysis Required: Yes  Government Levels Affected: None 
Small Entities Affected: Organizations  Federalism: No 
Included in the Regulatory Plan: Yes 
Agency Contact:
Christine F. Rogers
Regulatory Counsel
Department of Health and Human Services
Food and Drug Administration
Suite 3059 (HFD-7), Center for Drug Evaluation and Research, 10903 New Hampshire Avenue, Bldg. 51, Room 6316,
Silver Spring, MD 20993-0002
Phone:301 796-3601
Fax:301 847-8440

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