Just in case anyone is interested in the act that allows for...

Just in case anyone is interested in the act that allows for accelerated approval:

In 2012, Congress passed the Food and Drug Administration Safety Innovations Act (FDASIA). Section 901 of FDASIA amends the Federal Food, Drug, and Cosmetic Act (FD&C Act) to allow the FDA to base accelerated approval for drugs for serious conditions that fill an unmet medical need on whether the drug has an effect on a surrogate or an intermediate clinical endpoint.


TITLE IX—DRUG APPROVAL AND PATIENT ACCESS SEC. 901. ENHANCEMENT OF ACCELERATED PATIENT ACCESS TO NEW MEDICAL TREATMENTS. (a) FINDINGS; SENSE OFCONGRESS.— (1) FINDINGS.—Congress finds as follows: (A) The Food and Drug Administration (referred to in this section as the ‘‘FDA’’) serves a critical role in helping to assure that new medicines are safe and effective. Regu-latory innovation is 1 element of the Nation’s strategy to address serious and life-threatening diseases or condi-tions by promoting investment in and development of innovative treatments for unmet medical needs. (B) During the 2 decades following the establishment of the accelerated approval mechanism, advances in med-ical sciences, including genomics, molecular biology, and bioinformatics, have provided an unprecedented under-standing of the underlying biological mechanism and patho-genesis of disease. A new generation of modern, targeted medicines is under development to treat serious and life- threatening diseases, some applying drug development strategies based on biomarkers or pharmacogenomics, pre-dictive toxicology, clinical trial enrichment techniques, and novel clinical trial designs, such as adaptive clinical trials. (C) As a result of these remarkable scientific and med-ical advances, the FDA should be encouraged to implement more broadly effective processes for the expedited develop-ment and review of innovative new medicines intended to address unmet medical needs for serious or life-threat-ening diseases or conditions, including those for rare dis-eases or conditions, using a broad range of surrogate or clinical endpoints and modern scientific tools earlier in the drug development cycle when appropriate. This may result in fewer, smaller, or shorter clinical trials for the intended patient population or targeted subpopulation without compromising or altering the high standards of the FDA for the approval of drugs. (D) Patients benefit from expedited access to safe and effective innovative therapies to treat unmet medical needs for serious or life-threatening diseases or conditions. (E) For these reasons, the statutory authority in effect on the day before the date of enactment of this Act gov-erning expedited approval of drugs for serious or life-threat-ening diseases or conditions should be amended in order to enhance the authority of the FDA to consider appropriate scientific data, methods, and tools, and to expedite develop-ment and access to novel treatments for patients with a broad range of serious or life-threatening diseases or conditions. (2) SENSE OF CONGRESS.—It is the sense of Congress that the Food and Drug Administration should apply the accelerated approval and fast track provisions set forth in section 506 of the Federal Food, Drug, and Cosmetic Act (21 U.S.C. 356), as amended by this section, to help expedite the development and availability to patients of treatments for serious or life- threatening diseases or conditions while maintaining safety and effectiveness standards for such treatments. (b) EXPEDITEDAPPROVAL OFDRUGS FORSERIOUS ORLIFE- THREATENINGDISEASES ORCONDITIONS.—Section 506 (21 U.S.C. 356) is amended to read as follows:

‘‘SEC. 506. EXPEDITED APPROVAL OF DRUGS FOR SERIOUS OR LIFE- THREATENING DISEASES OR CONDITIONS. ‘‘(a) DESIGNATION OFDRUG ASFASTTRACKPRODUCT.— ‘‘(1) IN GENERAL.—The Secretary shall, at the request of the sponsor of a new drug, facilitate the development and expedite the review of such drug if it is intended, whether alone or in combination with one or more other drugs, for the treatment of a serious or life-threatening disease or condi-tion, and it demonstrates the potential to address unmet med-ical needs for such a disease or condition. (In this section, such a drug is referred to as a ‘fast track product’.) ‘‘(2) REQUEST FOR DESIGNATION.—The sponsor of a new drug may request the Secretary to designate the drug as a fast track product. A request for the designation may be made concurrently with, or at any time after, submission of an application for the investigation of the drug under section 505(i) or section 351(a)(3) of the Public Health Service Act. ‘‘(3) DESIGNATION.—Within 60 calendar days after the receipt of a request under paragraph (2), the Secretary shall determine whether the drug that is the subject of the request meets the criteria described in paragraph (1). If the Secretary finds that the drug meets the criteria, the Secretary shall designate the drug as a fast track product and shall take such actions as are appropriate to expedite the development and review of the application for approval of such product. ‘‘(b) ACCELERATEDAPPROVAL OF ADRUG FOR ASERIOUS ORLIFE-THREATENINGDISEASE ORCONDITION, INCLUDING AFASTTRACKPRODUCT.— ‘‘(1) IN GENERAL.— ‘‘(A) ACCELERATED APPROVAL.—The Secretary may approve an application for approval of a product for a serious or life-threatening disease or condition, including a fast track product, under section 505(c) or section 351(a) of the Public Health Service Act upon a determination that the product has an effect on a surrogate endpoint that is reasonably likely to predict clinical benefit, or on a clinical endpoint that can be measured earlier than irreversible morbidity or mortality, that is reasonably likely to predict an effect on irreversible morbidity or mortality or other clinical benefit, taking into account the severity, rarity, or prevalence of the condition and the availability or lack of alternative treatments. The approval described in the preceding sentence is referred to in this section as ‘accelerated approval’. ‘‘(B) EVIDENCE.—The evidence to support that an end-point is reasonably likely to predict clinical benefit under subparagraph (A) may include epidemiological, pathophysiological, therapeutic, pharmacologic, or other evidence developed using biomarkers, for example, or other scientific methods or tools. ‘‘(2) LIMITATION.—Approval of a product under this sub-section may be subject to 1 or both of the following require-ments: ‘‘(A) That the sponsor conduct appropriate postapproval studies to verify and describe the predicted effect on irreversible morbidity or mortality or other clinical benefit. ‘‘(B) That the sponsor submit copies of all promotional materials related to the product during the preapproval review period and, following approval and for such period thereafter as the Secretary determines to be appropriate, at least 30 days prior to dissemination of the materials. ‘‘(3) EXPEDITED WITHDRAWAL OF APPROVAL.—The Secretary may withdraw approval of a product approved under acceler-ated approval using expedited procedures (as prescribed by the Secretary in regulations which shall include an opportunity for an informal hearing) if— ‘‘(A) the sponsor fails to conduct any required post-approval study of the drug with due diligence; ‘‘(B) a study required to verify and describe the pre-dicted effect on irreversible morbidity or mortality or other clinical benefit of the product fails to verify and describe such effect or benefit; ‘‘(C) other evidence demonstrates that the product is not safe or effective under the conditions of use; or ‘‘(D) the sponsor disseminates false or misleading pro-motional materials with respect to the product. ‘‘(c) REVIEW OFINCOMPLETEAPPLICATIONS FORAPPROVAL OFAFASTTRACKPRODUCT.— ‘‘(1) INGENERAL.—If the Secretary determines, after preliminary evaluation of clinical data submitted by the sponsor, that a fast track product may be effective, the Sec-retary shall evaluate for filing, and may commence review of portions of, an application for the approval of the product before the sponsor submits a complete application. The Sec-retary shall commence such review only if the applicant— ‘‘(A) provides a schedule for submission of information necessary to make the application complete; and‘‘(B) pays any fee that may be required under section 736. ‘‘(2) EXCEPTION.—Any time period for review of human drug applications that has been agreed to by the Secretary and that has been set forth in goals identified in letters of the Secretary (relating to the use of fees collected under section 736 to expedite the drug development process and the review of human drug applications) shall not apply to an application submitted under paragraph (1) until the date on which the application is complete. ‘‘(d) AWARENESSEFFORTS.—The Secretary shall— ‘‘(1) develop and disseminate to physicians, patient organizations, pharmaceutical and biotechnology companies, and other appropriate persons a description of the provisions of this section applicable to accelerated approval and fast track products; and ‘‘(2) establish a program to encourage the development of surrogate and clinical endpoints, including biomarkers, and other scientific methods and tools that can assist the Secretary in determining whether the evidence submitted in an applica-tion is reasonably likely to predict clinical benefit for serious or life-threatening conditions for which significant unmet med-ical needs exist. ‘‘(e) CONSTRUCTION.— ‘‘(1) PURPOSE.—The amendments made by the Food and Drug Administration Safety and Innovation Act to this section are intended to encourage the Secretary to utilize innovative and flexible approaches to the assessment of products under accelerated approval for treatments for patients with serious or life-threatening diseases or conditions and unmet medical needs. ‘‘(2) CONSTRUCTION.—Nothing in this section shall be con-strued to alter the standards of evidence under subsection (c) or (d) of section 505 (including the substantial evidence standard in section 505(d)) of this Act or under section 351(a) of the Public Health Service Act. Such sections and standards of evidence apply to the review and approval of products under this section, including whether a product is safe and effective. Nothing in this section alters the ability of the Secretary to rely on evidence that does not come from adequate and well- controlled investigations for the purpose of determining whether an endpoint is reasonably likely to predict clinical benefit as described in subsection (b)(1)(B).’’.