US Code

Public Law 119-142

21 U.S.C. § 360bbb

Expanded access to unapproved therapies and diagnostics

Section text and notes

(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)
section 355(i) of this titlesection 360j(g) of this title 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 or investigational device exemption in effect under ; 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.
section 282(i)(3) of title 42A 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 .
(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 Pub. L. 105–115, title IV, § 402111 Stat. 2365 Pub. L. 109–482, title I, § 102(f)(2)120 Stat. 3685 (, as added , , ; amended , , .)

Editorial Notes

Amendments

Pub. L. 109–4822007—Subsec. (c). substituted “section 282(i)(3)” for “section 282(j)(3)” in concluding provisions.

Statutory Notes and Related Subsidiaries

Effective Date of 2007 Amendment

Pub. L. 109–482section 109 of Pub. L. 109–482section 281 of Title 42Amendment by applicable only with respect to amounts appropriated for fiscal year 2007 or subsequent fiscal years, see , set out as a note under , The Public Health and Welfare.

Effective Date

section 501 of Pub. L. 105–115section 321 of this titleSection effective 90 days after , except as otherwise provided, see , set out as an Effective Date of 1997 Amendment note under .

Investigational Drugs

Pub. L. 115–52, title VI, § 610(a)131 Stat. 1051

“(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 [], 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 .—

21 U.S.C. 360bbb(b)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 (). The report shall include—
“(A)
21 U.S.C. 360bbb–0 a description of actions taken by manufacturers and distributors under section 561A of the Federal Food, Drug, and Cosmetic Act ();
“(B)
21 U.S.C. 360bbb 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 () and improved clarity for patients, physicians, and drug manufacturers about such process;
“(C)
21 U.S.C. 360bbb consideration of whether the guidance or regulations issued to implement section 561 of the Federal Food, Drug, and Cosmetic Act () 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 .—

21 U.S.C. 360bbb(b)Not later than 1 year after the date of enactment of this Act [], 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 (). 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.”
, (b), , , 1053, provided that:

Executive Documents

Ex. Ord. No. 14401. Accelerating Medical Treatments for Serious Mental Illness

Ex. Ord. No. 14401, , 91 F.R. 21709, provided:

By the authority vested in me as President by the Constitution and the laws of the United States of America, it is hereby ordered:

SectionPurpose and Policy 1. . Policymakers and the medical field have long struggled to address the burden of suicide and serious mental illness rates in America. Today, over 14 million American adults have a serious mental illness, defined as having a diagnosable mental, behavioral, or emotional disorder that substantially interferes with a person’s life and ability to function, and about 8 million are on prescription medication for these conditions. Suicide rates tragically increased by 37 percent from 2000 to 2018. During my first term, we made historic progress in helping those struggling with some of the most insidious mental illnesses, and suicide rates decreased by 5 percent from 2018 to 2020. The COVID–19 pandemic and the Biden Administration’s prolonged shutdown stunted this progress and suicide rates rebounded upwards again to their peak rate in 2022. Critically, veterans often suffer in greater measure from this tragedy. For over 20 years, there have been more than 6,000 veteran suicides per year, and the current veteran suicide rate is more than twice as much as the non-veteran adult population.

Individuals suffering from major depressive disorder and substance abuse disorder, among other serious mental illnesses, can relapse or not fully respond to standard medical and psychiatric therapies. Despite massive Federal investment into researching potential advancements in mental health care and treatment, our medical research system has yet to produce approved therapies that promote enduring improvements in the mental health condition of these most complex patients. Innovative methods are needed to find long-term solutions for these Americans beyond existing prescription medications.

Psychedelic drugs, including ibogaine compounds, show potential in clinical studies to address serious mental illnesses for patients whose conditions persist after completing standard therapy. Indeed, the Food and Drug Administration (FDA) has granted Breakthrough Therapy designation to specific psychedelic drugs, and there are numerous products currently in the clinical trial pipeline for review of safety and efficacy. It is the policy of my Administration to accelerate innovative research models and appropriate drug approvals to increase access to psychedelic drugs that could save lives and reverse the crisis of serious mental illness in America.

Sec.FDA Review Prioritization and Right to Try 2. . (a) The Commissioner of Food and Drugs shall provide Commissioner’s National Priority Vouchers to appropriate psychedelic drugs that have received a Breakthrough Therapy designation and are in accordance with the criteria of the National Priority Voucher Program.

Pub. L. 115–17621 U.S.C. 360bbb–0a21 U.S.C. 823section 303 of Pub. L. 91–51321 U.S.C. 801(b) The FDA and Drug Enforcement Administration shall facilitate and establish a pathway for eligible patients to access psychedelic drugs, including ibogaine compounds, under the [Trickett Wendler, Frank Mongiello, Jordan McLinn, and Matthew Bellina] Right to Try Act [of 2017, ] (), including any necessary Schedule I handling authorizations for treating physicians and researchers, consistent with [], and any applicable waiver authority under the Controlled Substances Act [ et seq.].

Sec.Department of Health and Human Services Funding for Federal-State Collaboration 3. . The Secretary of Health and Human Services shall, through the Advanced Research Projects Agency for Health, allocate at least $50 million from existing funds to support and partner with State governments that have enacted or are developing programs to advance psychedelic drugs for serious mental illnesses, including through Federal funding, technical assistance, and data sharing as appropriate and consistent with applicable law.

Sec.Department of Health and Human Services and FDA Collaboration with the Department of Veterans Affairs and the Private Sector5 U.S.C. 552aPub. L. 104–19121 U.S.C. 355 4. . The Department of Health and Human Services (HHS) and FDA shall collaborate with the Department of Veterans Affairs (VA) and, as appropriate and consistent with applicable law, including any privacy restrictions from the Privacy Act of 1974 [] and the Health Insurance Portability and Accountability Act of 1996 [, see Tables for classification], with the private sector, to increase clinical trial participation, data sharing, and real-world evidence generation regarding psychedelic drugs, and shall prioritize drugs that have received a Breakthrough Therapy designation. The HHS, FDA, and VA are directed to sign data-sharing memoranda as appropriate to ensure that data from relevant clinical studies conducted by other executive departments and agencies is made available to FDA to facilitate the timely evaluation and approval of drugs that meet standards for approval under section 505 of the Federal Food, Drug, and Cosmetic Act [].

Sec.Timely Rescheduling21 U.S.C. 811section 201 of Pub. L. 91–513 5. . The Attorney General shall, in consultation with HHS, initiate and complete review of any product containing a Schedule I substance that has successfully completed Phase 3 clinical trials for a serious mental health disorder, so that rescheduling, if appropriate under [], may proceed as quickly as practicable for such specific products that are ultimately approved under section 505 of the Federal Food, Drug, and Cosmetic Act.

Sec.General Provisions 6. . (a) Nothing in this order shall be construed to impair or otherwise affect:

(i) the authority granted by law to an executive department or agency, or the head thereof; or

(ii) the functions of the Director of the Office of Management and Budget relating to budgetary, administrative, or legislative proposals.

(b) This order shall be implemented consistent with applicable law and subject to the availability of appropriations.

(c) This order is not intended to, and does not, create any right or benefit, substantive or procedural, enforceable at law or in equity by any party against the United States, its departments, agencies, or entities, its officers, employees, or agents, or any other person.

(d) The costs for publication of this order shall be borne by the Department of Health and Human Services.

Donald J. Trump.
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