21st Century Cures Act

On May 9, 2017, Scott Gottlieb, M.D. was confirmed by the Senate as the new Commissioner of the Food and Drug Administration (“FDA”).  As Commissioner, he will be immediately responsible for shaping FDA policy on a number of current issues, including addressing and implementing several mandates stemming from the 21st Century Cures Act, (“Cures Act”), which was signed into law on December 13, 2016 with tremendous bipartisan support. The Cures Act contains over 200 sections that create new obligations for FDA; however, most pressing for Commissioner Gottlieb are three requirements that must be fulfilled within 180 days of the Cures Act’s passage (June 11th, 2017).

These requirements are:

  • Submission of a work plan to the Committee on Health, Education, Labor, and Pensions and the Committee on Appropriations of the Senate and the Committee on Energy and Commerce and the Committee on Appropriations of the House of Representatives for any projects, which will use funding from the FDA Innovation Account created under Section 1002 of the Cures Act;
  • Development of “a plan to issue draft and final versions of one or more guidance documents, over a period of 5 years, regarding the collection of patient experience data, and the use of such data and related information in drug development” pursuant to Section 3002 of the Cures Act, which is codified at 21 U.S.C. 360bbb-8c; and
  •  Publication of “a list of reusable device types” pursuant to Section 3059 of the Cures Act, which is codified at 21 U.S.C. 360.

Commissioner Gottlieb has a long professional history in the pharmaceutical industry working in both the public and private sectors. His firsthand experience as a former Deputy Commissioner at the FDA provides him with unique insights into the internal workings of the administration. As a former consultant advising on FDA policies to the pharmaceutical industry, Commissioner Gottlieb is also familiar with recent issues and trends affecting the industry, many of which are addressed within the Cures Act.  Despite having only one month to organize and address the mandates of the three above-referenced sections of the Cures Act, we believe Commissioner Gottlieb will likely meet these deadlines based on his prior knowledge and experience.

We will continue to monitor and provide insight on Commissioner Gottlieb’s activity as FDA Commissioner, and the implementation of key Cures Act provisions as they develop. For insight into how Commissioner Gottlieb has historically viewed key issues impacting the FDA, and mandates under the Cures Act, please view our previously published client alert.

On January 19, 2017, the United States Food and Drug Administration (“FDA”) unveiled a new drug designation process for regenerative advanced therapies, an important first step toward implementation of the regenerative medicine provisions of the 21st Century Cures Act.  Products for which a designation as a regenerative advanced therapy (“RAT”) is obtained are eligible for accelerated approval under the 21st Century Cures Act, which was signed into law by former President Obama on December 13, 2016 with sweeping bipartisan support.

The accelerated approval provisions for RATs under the 21st Century Cures Act are intended to facilitate expedited review and approval of stem cell therapies and other cellular and tissue products for use in serious or life threatening diseases, which are currently subject to regulation as unapproved drugs. Under the 21st Century Cures Act, regenerative medicine therapies eligible for a RAT designation may include any “cell therapy, therapeutic tissue engineering product, human cell and tissue product, or any combination product using such therapies or products, except for those products regulated solely under Section 361 of the Public Health Service Act (“PHS”), and part 1271 of Title 21, Code of Federal regulations.”[1]

Under the 21st Century Cures Act, the sponsor of a product must show the following to be eligible for a RAT designation:

  • The drug is a regenerative medicine therapy;
  • The drug is intended to treat, modify, reverse, or cure a serious or life-threatening disease or condition;[2] and
  • Preliminary clinical evidence indicates that the drug has the potential to address unmet medical needs for such disease or condition.

Pursuant to the FDA website on the Regenerative Advanced Therapy Designation, a sponsor requesting a RAT designation for its product must make such a request either concurrently with submission of an Investigational New Drug application (“IND”), or as an amendment to an existing IND. Consistent with requests for fast track and breakthrough therapy designations, the FDA only requires that a sponsor describe the preliminary clinical evidence that supports a RAT designation, and does not require the sponsor to submit primary data.  Information that will be considered includes: a description of any available therapies for the disease or condition already in existence, the study design, the population studied, the endpoints used, and a description of the study results and statistical analyses.

The RAT designation process will be overseen by the newly created Office of Tissues and Advanced Therapies (OTAT). The OTAT will manage the application process for RAT designation, and will notify the sponsor within 60 days of receiving an application as to whether the RAT designation is granted. If a sponsor does not receive a RAT designation for its product the OTAT will provide an explanation in writing of its rationale for the denial.

A sponsor that obtains a RAT designation for its product is entitled to meet with the FDA early in its development program to discuss the potential use of surrogate or intermediate endpoints that may be used to support accelerated approval of the product. RATs may be eligible for accelerated approval based upon surrogate or intermediate endpoints reasonably likely to predict a long-term clinical benefit, and based on data obtained from a “meaningful number of sites” with subsequent expansion to additional sites, along with the collection of additional data in the post-market phase.

The implementation of the RAT designation process will enable manufacturers to begin to take advantage of the less burdensome review process enabled by the 21st Century Cures Act.  While some patient advocates have expressed concern that the availability of an accelerated approval pathway for regenerative medicine products may impede the development of robust evidence establishing their safety and effectiveness, and may ultimately result in patient harm, 21st Century Cures’ accelerated approval provisions are likely to be a harbinger of a new wave of regenerative medicine therapies that provide additional options for patients facing serious or life threatening conditions.

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[1] 21st Century Cures Act Sec., Sec. 3033(8).  Human Cells, tissues, and cellular and tissue-based products (HCT/Ps) are regulated solely under section 361 of the PHS Act and the regulations of 21 C.F.R. Part 1271 if all of the following criteria are met: the HCT/P is minimally manipulated, intended for homologous use (as reflected in labeling and advertising), is not manufactured by combining cells or tissues with another article, except for water, crystalloids, or a sterilizing, preserving or storage agent, and does not have a systemic effect nor is dependent upon the metabolic activity of living cells for its primary function. Therefore, if a product meets all of the aforementioned criteria, the HCT/P will still be regulated under 21 C.F.R. Part 1271 and will not be subject to regulation as a drug product.

[2] The FDA will use its standard definitions found in its Expedited Program Guidance as a guide to determining whether a product meets the required criteria, such as whether a condition is “serious or life-threatening” or whether a drug is “intended to treat a serious disease or condition.”