Prevent Pandemics Act Discussion Draft
February 4, 2022
The Honorable Patty Murray
United States Senate
154 Russell Senate Office Building
Washington, DC 20510
The Honorable Richard Burr
United States Senate
217 Russell Senate Office Building
Washington, DC 20510
Dear Senators Murray and Burr:
The American Society of Gene and Cell Therapy (ASGCT) welcomes the opportunity to provide comments on the PREVENT Pandemics Act discussion draft. ASGCT is a nonprofit professional membership organization comprised of more than 4,800 scientists, physicians, and other professionals working in gene and cell therapy in settings such as universities, hospitals, and biotechnology companies. Many of our members have spent their careers in this field performing the underlying research that has led to today’s robust pipeline of transformative therapies.
A core portion of the Society’s mission is to advance the discovery and clinical application of genetic and cellular therapies to alleviate human disease. Therefore, the development and accessibility to patients of such therapies is of paramount importance to ASGCT’s membership. Our comments on the PREVENT Pandemics Act discussion draft primarily focus on the application of Sections 506 and 518 to gene and cell therapies during both peacetime and emergencies.
Sections 506 and 518 establish new Food and Drug Administration (FDA) designations for drug and manufacturing platforms (in proposed new Federal Food Drug and Cosmetic Act Sections 506K and 506L, respectively) that would enable greater use of data across products that share common chemistries (such as gene delivery vectors) or critical processes. We believe that these sections, if included in final legislation and drafted to reflect their intent, would facilitate the development of gene and cell therapies across more disease areas by harnessing the power of platforms and result in greater availability to patients. Comments on specific provisions are included below.
Proposed FFDCA Section 506K(g) – Changes to an advanced platform technology (APT).
Unlike traditional drug products, gene and cell therapy product manufacturing often develops in parallel with clinical development, with sponsors making 2 changes to improve yield and efficacy based on early clinical findings. In addition, manufacturing process improvements may occur at any time during product development, including post market, and in many gene and cell therapy development programs process changes are made to scale up manufacturing during late stages after demonstration of early clinical benefit. In this respect, chemistry, manufacturing, and controls (CMC) data for gene and cell therapy products often come throughout the product lifecycle. However, the current requirements for making changes after products are on the market were developed with small molecule chemistry in mind.
The proposed Section 506K(g) enables a single application for a major CMC change to an APT to facilitate and permit that change to be effectuated across all products utilizing that APT. As multiple gene therapies come to market on APTs, with potentially many more in the pipeline, this section will enable the latest CMC learnings to be applied across products to ensure products on the market are not lagging.
Proposed FFDCA Section 506L – Advanced Manufacturing Technologies Designation Pilot Program.
Gene and cell therapy manufacturing technologies need improvements to increase efficiencies and capacity as more products receive FDA licensure and approval to meet real-world patient demand. However, new innovation in manufacturing has lagged behind other areas. One reason for this is the lack of market incentive for an approved product using a novel technology coupled with the regulatory risk added to the product. The National Academies of Medicine published a report in 2021 which suggested that FDA implement a pathway to review novel advanced manufacturing technologies separately from individual products to de-risk their use product applications
.
We are pleased that the Committee has included language that establishes a pathway to review manufacturing technologies, including those for gene and cell therapies. However, we recommend the following changes in order for the pathway to meet its intent:
-
Allow the submission of information on a manufacturing technology for review and designation by the FDA to occur at any time, rather than tying submission to an individual product IND.
-
Ensure that the sunset of the pilot program does not remove designations granted before the sunset, in order to maintain the benefits of the pathway.
Advanced Research Projects Agency for Health (ARPA-H).
While not included in the draft text, ASGCT appreciates the mention of continued work to develop authorization language for a new Advanced Research Projects Agency for Health (ARPA-H). We believe that the ARPA-H concept could be especially helpful for advancing gene and cell therapies through investments in platform technologies and manufacturing innovation. We suggest that you consider creating a strong mandate that ARPA-H have tangible collaboration with the FDA throughout product development. We also believe that your proposed changes to the Food Drug and Cosmetic Act in Sections 506 and 518 are critical to the success of ARPA-H or any similar research effort. For product platforms and new manufacturing modes to advance from development to treatments, FDA will need the authority and Congressional charge to facilitate product agnostic reviews of platforms and allow sponsors to rely on previous data supporting a product’s safety and efficacy.
Thank you for your consideration of these comments. Please contact Christina Mayer, Senior Manager of Government Affairs, at cmayer@asgct.org with any questions. We look forward to further engaging with you in your legislative development process.
Sincerely,
David Barrett, JD
Chief Executive Officer