21 U.S.C. § 360bbb (FOOD AND DRUGS)
§ 360bbb. Expanded access to unapproved therapies and diagnostics
(a) Emergency situations
The Secretary may, under appropriate conditions determined by the Secretary, authorize the shipment of investigational drugs or investigational devices for the diagnosis, monitoring, or treatment of a serious disease or condition in emergency situations.
(b) Individual patient access to investigational products intended for serious diseases
Any person, acting through a physician licensed in accordance with State law, may request from a manufacturer or distributor, and any manufacturer or distributor may, after complying with the provisions of this subsection, provide to such physician an investigational drug or investigational device for the diagnosis, monitoring, or treatment of a serious disease or condition if—
(1) the licensed physician determines that the person has no comparable or satisfactory alternative therapy available to diagnose, monitor, or treat the disease or condition involved, and that the probable risk to the person from the investigational drug or investigational device is not greater than the probable risk from the disease or condition;
(2) the Secretary determines that there is sufficient evidence of safety and effectiveness to support the use of the investigational drug or investigational device in the case described in paragraph (1);
(3) the Secretary determines that provision of the investigational drug or investigational device will not interfere with the initiation, conduct, or completion of clinical investigations to support marketing approval; and
(4) the sponsor, or clinical investigator, of the investigational drug or investigational device submits to the Secretary a clinical protocol consistent with the provisions of section 355(i) or 360j(g) of this title, including any regulations promulgated under section 355(i) or 360j(g) of this title, describing the use of the investigational drug or investigational device in a single patient or a small group of patients.
(c) Treatment investigational new drug applications and treatment investigational device exemptions
Upon submission by a sponsor or a physician of a protocol intended to provide widespread access to an investigational drug or investigational device for eligible patients (referred to in this subsection as an “expanded access protocol”), the Secretary shall permit such investigational drug or investigational device to be made available for expanded access under a treatment investigational new drug application or treatment investigational device exemption if the Secretary determines that—
(1) under the treatment investigational new drug application or treatment investigational device exemption, the investigational drug or investigational device is intended for use in the diagnosis, monitoring, or treatment of a serious or immediately life-threatening disease or condition;
(2) there is no comparable or satisfactory alternative therapy available to diagnose, monitor, or treat that stage of disease or condition in the population of patients to which the investigational drug or investigational device is intended to be administered;
(3)
(A) the investigational drug or investigational device is under investigation in a controlled clinical trial for the use described in paragraph (1) under an investigational drug application in effect under section 355(i) of this title or investigational device exemption in effect under section 360j(g) of this title; or
(B) all clinical trials necessary for approval of that use of the investigational drug or investigational device have been completed;
(4) the sponsor of the controlled clinical trials is actively pursuing marketing approval of the investigational drug or investigational device for the use described in paragraph (1) with due diligence;
(5) in the case of an investigational drug or investigational device described in paragraph (3)(A), the provision of the investigational drug or investigational device will not interfere with the enrollment of patients in ongoing clinical investigations under section 355(i) or 360j(g) of this title;
(6) in the case of serious diseases, there is sufficient evidence of safety and effectiveness to support the use described in paragraph (1); and
(7) in the case of immediately life-threatening diseases, the available scientific evidence, taken as a whole, provides a reasonable basis to conclude that the investigational drug or investigational device may be effective for its intended use and would not expose patients to an unreasonable and significant risk of illness or injury.
A protocol submitted under this subsection shall be subject to the provisions of section 355(i) or 360j(g) of this title, including regulations promulgated under section 355(i) or 360j(g) of this title. The Secretary may inform national, State, and local medical associations and societies, voluntary health associations, and other appropriate persons about the availability of an investigational drug or investigational device under expanded access protocols submitted under this subsection. The information provided by the Secretary, in accordance with the preceding sentence, shall be the same type of information that is required by section 282(i)(3) of title 42.
(d) Termination
The Secretary may, at any time, with respect to a sponsor, physician, manufacturer, or distributor described in this section, terminate expanded access provided under this section for an investigational drug or investigational device if the requirements under this section are no longer met.
(e) Definitions
In this section, the terms “investigational drug”, “investigational device”, “treatment investigational new drug application”, and “treatment investigational device exemption” shall have the meanings given the terms in regulations prescribed by the Secretary.
(June 25, 1938, ch. 675, § 561, as added Pub. L. 105–115, title IV, § 402, Nov. 21, 1997, 111 Stat. 2365; amended Pub. L. 109–482, title I, § 102(f)(2), Jan. 15, 2007, 120 Stat. 3685.)
heading: Editorial Notes
heading: Amendments
text: 2007—Subsec. (c). Pub. L. 109–482 substituted “section 282(i)(3)” for “section 282(j)(3)” in concluding provisions.
heading: Statutory Notes and Related Subsidiaries
heading: Effective Date of 2007 Amendment
text: Amendment by Pub. L. 109–482 applicable only with respect to amounts appropriated for fiscal year 2007 or subsequent fiscal years, see section 109 of Pub. L. 109–482, set out as a note under section 281 of Title 42, The Public Health and Welfare.
heading: Effective Date
text: Section effective 90 days after Nov. 21, 1997, except as otherwise provided, see section 501 of Pub. L. 105–115, set out as an Effective Date of 1997 Amendment note under section 321 of this title.
heading: Investigational Drugs
text: Pub. L. 115–52, title VI, § 610(a), (b), Aug. 18, 2017, 131 Stat. 1051, 1053, provided that:
“(a) Patient Access to Investigational Drugs.—
“(1) Public meeting.—
“(A) In general.—
The Secretary of Health and Human Services (referred to in this section as the ‘Secretary’), acting through the Commissioner of Food and Drugs, in coordination with the Director of the National Institutes of Health, and in consultation with patients, health care providers, drug sponsors, bioethicists, and other stakeholders, shall, not later than 270 days after the date of enactment of this Act [Aug. 18, 2007], convene a public meeting to discuss clinical trial inclusion and exclusion criteria to inform the guidance under paragraph (3). The Secretary shall inform the Comptroller General of the United States of the date when the public meeting will take place.
“(B) Topics.—
The Secretary shall make available on the internet website of the Food and Drug Administration a report on the topics discussed at the meeting described in subparagraph (A) within 90 days of such meeting. Such topics shall include discussion of—
“(i) the rationale for, and potential barriers for patients created by, research clinical trial inclusion and exclusion criteria;
“(ii) how appropriate patient populations can benefit from the results of trials that employ alternative designs;
“(iii) barriers to participation in clinical trials, including—
“(I) information regarding any potential risks and benefits of participation;
“(II) regulatory, geographical, and socioeconomic barriers; and
“(III) the impact of exclusion criteria on the enrollment in clinical trials of particular populations, including infants and children, pregnant and lactating women, seniors, individuals with advanced disease, and individuals with co-morbid conditions;
“(iv) clinical trial designs and methods, including expanded access trials, that increase enrollment of more diverse patient populations, when appropriate, while facilitating the collection of data to establish safe use and support substantial evidence of effectiveness, including data obtained from expanded access trials; and
“(v) how changes to clinical trial inclusion and exclusion criteria may impact the complexity and length of clinical trials, the data necessary to demonstrate safety and effectiveness, and potential approaches to mitigating those impacts.
“(2) Report.—
Not later than 1 year after the Secretary issues the report under paragraph (1)(B), the Comptroller General of the United States shall report to the Committee on Health, Education, Labor, and Pensions of the Senate and the Committee on Energy and Commerce of the House of Representatives on individual access to investigational drugs through the expanded access program under section 561(b) of the Federal Food, Drug, and Cosmetic Act (21 U.S.C. 360bbb(b)). The report shall include—
“(A) a description of actions taken by manufacturers and distributors under section 561A of the Federal Food, Drug, and Cosmetic Act (21 U.S.C. 360bbb–0);
“(B) consideration of whether Form FDA 3926 and the guidance documents titled ‘Expanded Access to Investigational Drugs for Treatment Use—Questions and Answers’ and ‘Individual Patient Expanded Access Applications: Form FDA 3926’, issued by the Food and Drug Administration in June 2016, have reduced application burden with respect to individuals and physicians seeking access to investigational new drugs pursuant to section 561(b) of the Federal Food, Drug, and Cosmetic Act (21 U.S.C. 360bbb) and improved clarity for patients, physicians, and drug manufacturers about such process;
“(C) consideration of whether the guidance or regulations issued to implement section 561 of the Federal Food, Drug, and Cosmetic Act (21 U.S.C. 360bbb) have improved access for individual patients to investigational drugs who do not qualify for clinical trials of such investigational drugs, and what barriers to such access remain;
“(D) an assessment of methods patients and health care providers use to engage with the Food and Drug Administration or drug sponsors on expanded access; and
“(E) an analysis of the Secretary’s report under paragraph (1)(B).
“(3) Guidance.—
“(A) In general.—
Not later than 1 year after the publication of the report under paragraph (1)(B), the Secretary, acting through the Commissioner of Food and Drugs, shall issue one or more draft guidances regarding eligibility criteria for clinical trials. Not later than 1 year after the public comment period on each such draft guidance ends, the Secretary shall issue a revised draft guidance or final guidance.
“(B) Contents.—
The guidance documents described in subparagraph (A) shall address methodological approaches that a manufacturer or sponsor of an investigation of a new drug may take to—
“(i) broaden eligibility criteria for clinical trials and expanded access trials, especially with respect to drugs for the treatment of serious and life-threatening conditions or diseases for which there is an unmet medical need;
“(ii) develop eligibility criteria for, and increase trial recruitment to, clinical trials so that enrollment in such trials more accurately reflects the patients most likely to receive the drug, as applicable and as appropriate, while establishing safe use and supporting findings of substantial evidence of effectiveness; and
“(iii) use the criteria described in clauses (i) and (ii) in a manner that is appropriate for drugs intended for the treatment of rare diseases or conditions.
“(b) Improving Institutional Review Board Review of Single Patient Expanded Access Protocol.—
Not later than 1 year after the date of enactment of this Act [Aug. 18, 2017], the Secretary, acting through the Commissioner of Food and Drugs, shall issue guidance or regulations, or revise existing guidance or regulations, to streamline the institutional review board review of individual patient expanded access protocols submitted under [section] 561(b) of the Federal Food, Drug, and Cosmetic Act (21 U.S.C. 360bbb(b)). To facilitate the use of expanded access protocols, any guidance or regulations so issued or revised may include a description of the process for any person acting through a physician licensed in accordance with State law to request that an institutional review board chair (or designated member of the institutional review board) review a single patient expanded access protocol submitted under such section 561(b) for a drug. The Secretary shall update any relevant forms associated with individual patient expanded access requests under such section 561(b) as necessary.”