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Federal Register/Vol. 74, No. 155/Thursday, August 13, 2009 40900 Federal Register / Vol. 74, No. 155 / Thursday, August 13, 2009 / Rules and Regulations addition to the direct costs described in Application which clarifies the efforts to expand access to paragraph (d)(1)(i) of this section, a circumstances in which charging for an investigational drugs for treatment use. sponsor may recover the costs of investigational drug in a clinical trial is Before 1987, there was no formal monitoring the expanded access IND or appropriate, sets forth criteria for recognition of treatment use in FDA’s protocol, complying with IND reporting charging for an investigational drug for regulations concerning INDs, but requirements, and other administrative the different types of expanded access investigational drugs were made costs directly associated with the for treatment use described in this final available for treatment use informally. expanded access IND. rule, and clarifies what costs can be In 1987, FDA revised the IND (3) To support its calculation for cost recovered for an investigational drug. regulations in part 312 (21 CFR part recovery, a sponsor must provide DATES: This rule is effective October 13, 312) to explicitly provide for one supporting documentation to show that 2009. specific kind of treatment use of the calculation is consistent with the FOR FURTHER INFORMATION CONTACT: investigational drugs (52 FR 19466, May requirements of paragraphs (d)(1) and, if Colleen L. Locicero, Center for Drug 22, 1987). Section 312.34 authorized applicable, (d)(2) of this section. The Evaluation and Research, Food and access to investigational drugs for a documentation must be accompanied by Drug Administration, 10903 New broad population under a treatment a statement that an independent Hampshire Ave., Bldg. 22, rm. 4200, protocol or treatment IND when certain certified public accountant has Silver Spring, MD 20993–0002, criteria were met. Section 312.35 reviewed and approved the calculations. 301–796–2270; or described the submission requirements Dated: July 20, 2009. Stephen M. Ripley, Center for for such treatment use. The 1987 IND Jeffrey Shuren, Biologics Evaluation and Research regulations also implicitly acknowledged the existence of other Associate Commissioner for Policy and (HFM–17), Food and Drug Planning. Administration, 1401 Rockville kinds of treatment use, notably use in individual patients, by adding a [FR Doc. E9–19004 Filed 8–12–09; 8:45 am] Pike, Rockville, MD 20852, 301– 827–6210. provision for obtaining an BILLING CODE 4160–01–S SUPPLEMENTARY INFORMATION: investigational drug for treatment use in an emergency situation (§ 312.36). Table of Contents DEPARTMENT OF HEALTH AND However, § 312.36 did not describe HUMAN SERVICES I. Background criteria or requirements that must be II. Overview of the Final Rule Including met to authorize individual patient Food and Drug Administration Changes to the Proposed Rule treatment use. A. Overview In response to criticisms that this lack 21 CFR Parts 312 and 316 B. Changes to the Proposed Rule of criteria and submission requirements III. Comments on the Proposed Rule resulted in inconsistent policies, [Docket No. FDA–2006–N–0238] (formerly A. General Comments on the Proposed Docket No. 2006N–0062) Rule inequitable access, and preferential access for certain categories of patients, RIN 0910–AF14 B. Comments Related to Proposed Rule as Congress included in the Food and Drug a Whole Administration Modernization Act of Expanded Access to Investigational C. Comments on Specific Provisions of the 1997 (FDAMA) (Public Law 105–115), Drugs for Treatment Use Proposed Rule which amended the Federal Food, Drug, IV. Legal Authority AGENCY: and Cosmetic Act (the act), specific Food and Drug Administration, V. Environmental Impact HHS. provisions concerning expanded access VI. Analysis of Economic Impacts to investigational drugs for treatment ACTION: Final rule. A. Objectives of the Final Action B. Nature of the Problem Being Addressed use (Expanded Access to Unapproved SUMMARY: The Food and Drug C. Baseline for the Analysis Therapies and Diagnostics, section 561 Administration (FDA) is amending its D. Nature of the Impact of the act (21 U.S.C. 360bbb)). regulations on access to investigational E. Benefits of the Final Rule FDA proposed this rule in December new drugs for the treatment of patients. F. Costs of the Final Rule 2006 to further address the concerns The final rule clarifies existing G. Minimizing the Impact on Small Entities that motivated the FDAMA changes, regulations and adds new types of H. Alternatives including problems of inconsistent expanded access for treatment use. VII. Paperwork Reduction Act of 1995 application of access policies and A. The Final Rule Under the final rule, expanded access to B. Estimates of Reporting Burden programs and inequities in access based investigational drugs for treatment use VIII. Federalism on the relative sophistication of the is available to individual patients, setting in which a patient is treated or including in emergencies; intermediate- I. Background on the patient’s disease or condition. By size patient populations; and larger In the Federal Register of December describing in detail in the final rule the populations under a treatment protocol 14, 2006 (71 FR 75147), FDA proposed criteria, submission requirements, and or treatment investigational new drug to amend its regulations permitting safeguards for the different types of application (IND). The final rule is access to investigational drugs to treat expanded access for treatment use of intended to improve access to patients with serious or immediately investigational drugs, FDA hopes to investigational drugs for patients with life-threatening diseases or conditions increase awareness and knowledge of serious or immediately life-threatening when there is no comparable or expanded access programs and the diseases or conditions who lack other satisfactory alternative therapy to procedures for obtaining investigational therapeutic options and who may diagnose, monitor, or treat the patient’s drugs for treatment use. The agency benefit from such therapies. Elsewhere disease or condition. believes that the final rule appropriately in this issue of the Federal Register, As discussed in greater detail in the authorizes access to promising drugs for FDA is publishing the final rule on preamble to the proposed rule (71 FR treatment use, while protecting patient Charging for Investigational Drugs 75147 at 75148 to 75149), there have safety and avoiding interference with Under an Investigational New Drug been several statutory and regulatory the development of investigational VerDate Nov<24>2008 18:59 Aug 12, 2009 Jkt 217001 PO 00000 Frm 00030 Fmt 4701 Sfmt 4700 E:\FR\FM\13AUR2.SGM 13AUR2 srobinson on DSKHWCL6B1PROD with RULES2 Federal Register / Vol. 74, No. 155 / Thursday, August 13, 2009 / Rules and Regulations 40901 drugs for marketing under approved B. Changes to the Proposed Rule insurance companies, hospitals, a trade applications. The final rule has been revised in organization representing hospitals, and In 2007, after the proposed rule on response to comments received on the a professional society representing expanded access was published, proposed rule. The responses are oncologists. Congress passed the Food and Drug discussed in section III of this A. General Comments on the Proposed Administration Amendments Act of document. The final rule: Rule 2007 (FDAAA) (Public Law 110–85). • Revises proposed § 312.300(a) to Most of the comments strongly One provision, codified in 505–1(f)(6) of clarify that subpart I is intended to the act (21 U.S.C. 355–1(f)(6)), requires supported the goal of expanding access apply not only to the use of to investigational drugs for treatment the Secretary of Health of Human investigational new drugs but also to Services (the Secretary) to promulgate use. The vast majority of these approved drugs whose availability is comments expressed strong support for regulations concerning how a physician limited because the drugs are subject to may provide a drug under the the proposed rule as a way to expand a risk evaluation and mitigation strategy access. As a category, the largest volume mechanisms of section 561 when the (REMS) in accordance with section 505– drug is subject to elements to assure safe of comments came from individuals, 1(f)(6) of the act. and the vast majority of those supported use under a risk evaluation and • Also revises proposed § 312.300(a) the proposed rule. Healthcare and mitigation strategy (REMS). The to clarify that subpart I is intended to expanded access mechanisms described consumer advocacy organizations apply to all those with a serious disease provided the next largest volume of in this final rule can be used by a or condition, regardless of whether the patient seeking access to a drug with a comments. Comments from these patient would currently be considered organizations spanned the spectrum REMS in the event that the drug is not seriously ill with that disease or available to the patient under the from strongly supportive to strongly condition. negative. Many healthcare and criteria of the REMS, provided the drug • Revises proposed § 312.300(b) to consumer advocacy organizations and the patient meet the criteria for an include a definition of ‘‘serious disease expanded access program. Therefore, commented that they believe the rule or condition.’’ strikes the appropriate balance between this rule fulfills the FDAAA • Revises proposed § 312.305(c)(5) to requirement. increased access and patient safety clarify that a sponsor should make an without impeding enrollment in clinical This final rule applies both to drug investigator’s brochure available to products that are subject to section 505 trials or otherwise jeopardizing the licensed physicians in an expanded development of new drugs for marketing of the act (21 U.S.C. 355) and biological access program whenever such a products subject to the licensing approval. brochure exists. Healthcare and consumer advocacy provisions of the Public Health Service • Revises proposed § 312.310(a)(2) to organizations who opposed the Act (42 U.S.C.
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