FDA 21 CFR 1271 Draft Guidances and 21st Century Cures Act
FDA has published 4 separate Draft Guidances pertaining pertaining to 21 CFR 1271:
On September 12-13, 2016, FDA held public hearings regarding the above draft guidances.
The purpose of this public hearing is to obtain comments on these four draft guidances. FDA is seeking feedback, both general and specific, from a broad group of stakeholders, including HCT/P manufacturers, tissue establishments, biological and device product manufacturers, health care professionals, clinicians, biomedical researchers, and the public. For example, FDA would like comments on the scope of each guidance, including the particular topics covered, the particular questions posed, whether there are additional issues for which they seek guidance, and whether FDA's recommendations for each topic are sufficiently clear and consistent within and across documents to provide meaningful guidance to stakeholders. In addition, FDA welcomes any comments that will enhance the usefulness and clarity of these documents.
On September 8, 2016, FDA held a Public Workshop; Scientific Evidence in the Development of Human Cells, Tissues, and Cellular and Tissue-Based Products Subject to Premarket Approval.
The purpose of the public workshop is to identify and discuss scientific considerations and challenges to help inform the development of human cells, tissues, and cellular and tissue-based products (HCT/Ps) subject to premarket approval, including stem cell-based products.
On December 13th, 2016, President Obama signed the '21st Century Cures Act', which is a sweeping piece of healthcare legislation intended to in part accelerate the arrival of new drugs and medical devices. There are several provisions within the Act which pertain specifically to Regenerative Medicine:
(a) In general.—Section 506 of the Federal Food, Drug, and Cosmetic Act (21 U.S.C. 356) is amended—
(1) by transferring subsection (e) (relating to construction) so that it appears before subsection (f) (relating to awareness efforts); and
(2) by adding at the end the following:
“(1) IN GENERAL.—The Secretary, at the request of the sponsor of a drug, shall facilitate an efficient development program for, and expedite review of, such drug if the drug qualifies as a regenerative advanced therapy under the criteria described in paragraph (2).
“(A) the drug is a regenerative medicine therapy (as defined in paragraph (8));
“(B) the drug is intended to treat, modify, reverse, or cure a serious or life-threatening disease or condition; and
“(C) preliminary clinical evidence indicates that the drug has the potential to address unmet medical needs for such a disease or condition.
“(3) REQUEST FOR DESIGNATION.—The sponsor of a drug may request the Secretary to designate the drug as a regenerative advanced therapy concurrently with, or at any time after, submission of an application for the investigation of the drug under section 505(i) of this Act or section 351(a)(3) of the Public Health Service Act.
“(4) DESIGNATION.—Not later than 60 calendar days after the receipt of a request under paragraph (3), the Secretary shall determine whether the drug that is the subject of the request meets the criteria described in paragraph (2). If the Secretary determines that the drug meets the criteria, the Secretary shall designate the drug as a regenerative advanced therapy and shall take such actions as are appropriate under paragraph (1). If the Secretary determines that a drug does not meet the criteria for such designation, the Secretary shall include with the determination a written description of the rationale for such determination.
“(5) ACTIONS.—The sponsor of a regenerative advanced therapy shall be eligible for the actions to expedite development and review of such therapy under subsection (a)(3)(B), including early interactions to discuss any potential surrogate or intermediate endpoint to be used to support the accelerated approval of an application for the product under subsection (c).
“(A) eligible for priority review, as described in the Manual of Policies and Procedures of the Food and Drug Administration and goals identified in the letters described in section 101(b) of the Prescription Drug User Fee Amendments of 2012; and
“(i) surrogate or intermediate endpoints reasonably likely to predict long-term clinical benefit; or
“(ii) reliance upon data obtained from a meaningful number of sites, including through expansion to additional sites, as appropriate.
“(7) POSTAPPROVAL REQUIREMENTS.—The sponsor of a regenerative advanced therapy that is granted accelerated approval and is subject to the postapproval requirements under subsection (c) may, as appropriate, fulfill such requirements, as the Secretary may require, through—
“(A) the submission of clinical evidence, clinical studies, patient registries, or other sources of real world evidence, such as electronic health records;
“(B) the collection of larger confirmatory data sets, as agreed upon pursuant to subsection (a)(3)(B); or
“(C) postapproval monitoring of all patients treated with such therapy prior to approval of the therapy.
“(8) DEFINITION.—For purposes of this section, the term ‘regenerative medicine therapy’ includes cell therapy, therapeutic tissue engineering products, human cell and tissue products, and combination products using any such therapies or products, except for those regulated solely under section 361 of the Public Health Service Act and part 1271 of title 21, Code of Federal Regulations.”.
(1) to approve drugs pursuant to the Federal Food, Drug, and Cosmetic Act (21 U.S.C. 301 et seq.) and section 351 of the Public Health Service Act (42 U.S.C. 262) as authorized prior to the date of enactment of the 21st Century Cures Act, including the standards of evidence, and applicable conditions, for approval under such Acts; or
(2) to alter the authority of the Secretary to require postapproval studies pursuant to such Acts, as authorized prior to the date of enactment of the 21st Century Cures Act.
(c) Conforming amendment.—Section 506(e)(1) of the Federal Food, Drug, and Cosmetic Act (21 U.S.C. 356(e)(1)) is amended by inserting “and the 21st Century Cures Act” after “Food and Drug Administration Safety and Innovation Act”.
(a) Draft guidance.—Not later than 1 year after the date of enactment of the 21st Century Cures Act, the Secretary of Health and Human Services, acting through the Commissioner of Food and Drugs, shall issue draft guidance clarifying how, in the context of regenerative advanced therapies, the Secretary will evaluate devices used in the recovery, isolation, or delivery of regenerative advanced therapies. In doing so, the Secretary shall specifically address—
(1) how the Food and Drug Administration intends to simplify and streamline regulatory requirements for combination device and cell or tissue products;
(2) what, if any, intended uses or specific attributes would result in a device used with a regenerative therapy product to be classified as a class III device;
(3) when the Food and Drug Administration considers it is necessary, if ever, for the intended use of a device to be limited to a specific intended use with only one particular type of cell; and
(4) application of the least burdensome approach to demonstrate how a device may be used with more than one cell type.
(b) Final guidance.—Not later than 12 months after the close of the period for public comment on the draft guidance under subsection (a), the Secretary of Health and Human Services shall finalize such guidance.
(a) Report to Congress.—Before March 1 of each calendar year, the Secretary of Health and Human Services shall, with respect to the previous calendar year, submit a 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—
(1) the number and type of applications for approval of regenerative advanced therapies filed, approved or licensed as applicable, withdrawn, or denied; and
(2) how many of such applications or therapies, as applicable, were granted accelerated approval or priority review.
(b) Regenerative Advanced Therapy.—In this section, the term “regenerative advanced therapy” has the meaning given such term in section 506(g) of the Federal Food, Drug, and Cosmetic Act, as added by section 3033 of this Act.
Subchapter A of chapter V of the Federal Food, Drug, and Cosmetic Act (21 U.S.C. 351 et seq.) is amended by inserting after section 506F the following:
“(a) In general.—Not later than 2 years after the date of enactment of the 21st Century Cures Act, the Secretary, in consultation with the National Institute of Standards and Technology and stakeholders (including regenerative medicine and advanced therapies manufacturers and clinical trial sponsors, contract manufacturers, academic institutions, practicing clinicians, regenerative medicine and advanced therapies industry organizations, and standard setting organizations), shall facilitate an effort to coordinate and prioritize the development of standards and consensus definition of terms, through a public process, to support, through regulatory predictability, the development, evaluation, and review of regenerative medicine therapies and regenerative advanced therapies, including with respect to the manufacturing processes and controls of such products.
“(A) identity opportunities to help advance the development of regenerative medicine therapies and regenerative advanced therapies;
“(B) identify opportunities for the development of laboratory regulatory science research and documentary standards that the Secretary determines would help support the development, evaluation, and review of regenerative medicine therapies and regenerative advanced therapies through regulatory predictability; and
“(C) work with stakeholders, such as those described in subsection (a), as appropriate, in the development of such standards.
“(2) REGULATIONS AND GUIDANCE.—Not later than 1 year after the development of standards as described in subsection (a), the Secretary shall review relevant regulations and guidance and, through a public process, update such regulations and guidance as the Secretary determines appropriate.
“(c) Definitions.—For purposes of this section, the terms ‘regenerative medicine therapy’ and ‘regenerative advanced therapy’ have the meanings given such terms in section 506(g).”.