Washington, DC (January 10, 2022) – A new report released by Alliance for Cell Therapy Now and the Alliance for Cell Therapy Foundation, independent, non-profit organizations guided by leaders representing academic and medical institutions, industry innovators, and patients (together referred to as the Alliance), assesses the level of progress made related to regenerative medicine and cell-based therapies (RMCTs) since the 21st Century Cures Act passed five years ago.
Key elements of the report include:
- An examination of the current state of the field of RMCTs;
- An overview of the provisions contained in the 21st Century Cures Act (Cures Act);
- An analysis of federal agency implementation of such provisions in the five years since the Cures Act was passed and signed into law;
- Recommendations for continuing to advance safe and effective RMCTs for patients in need.
There have been several advancements in the field since the 21st Century Cures Act was passed in December 2016:
- As of December 31, 2021, there are more than 900 active clinical trials in the U.S. exploring the use of RMCTs to treat a wide range of conditions, including cancer, cardiovascular disease, diabetes, eye-related conditions, musculoskeletal conditions, neurological conditions, and COVID-19.
- The FDA has approved 29 products—10 of those between 2017 and 2021—with several more in the pipeline.
- There have been 180 requests for the Regenerative Medicine Advanced Therapy (RMAT) designation, the new accelerated approval pathway authorized under the 21st Century Cures Act, and 67 of those requests have been granted.
Still, considerable challenges remain:
- Ninety-five percent of active clinical trials in the U.S. are Phase 1 or Phase 2 trials. Many have promising results, but large-scale, randomized, placebo-controlled trials are needed to confirm the results of these early-stage trials.
- The greatest barrier to conducting these studies is the high cost of conducting them, particularly among academic and research institutions, as well as small biotechnology companies, who are responsible for the vast majority of clinical trials being conducted. Despite this financial barrier, only 17 percent of clinical studies have received any federal funding.
- The lack of federal funding services as a key barrier to advancing RMCTs. While the Cures Act did provide limited funding—$30 million—for regenerative medicine research, such funding technical expired in 2020 and not all of the funds have been spent. Furthermore, 75% of the awards that were posted on the NIH website supported pre-clinical research, as opposed to clinical trials.
- NIH matching requirements have served as a considerable barrier to promising research applicants and will become even more of an issue with later stage, more costly trials.
- The FDA-related provisions from the 21st Century Cures Act have largely been implemented successfully, but additional resources are needed to support this growing field.
- Additional resources are needed at the FDA to support academic and research institutions, as well as small biotechnology companies, through educational sessions, workshops, and technical assistance, as they pursue regulatory approval in this emerging, promising field. In addition, resources are needed at the FDA to increase enforcement against clinics and other manufacturers who are not in compliance with relevant regenerative medicine regulations. Rogue actors continue to undermine legitimate scientific researchers and confuse patients in need of treatments for serious and life-threatening conditions.
The Alliance makes the following recommendations to address these issues and continue to advance the field of regenerative medicine and cell-based therapies as intended by Congress through passage of the 21st Century Cures Act.
- Increase Federal Funding of Clinical Trials. Significantly expand the level of investment in large-scale, randomized, placebo-controlled clinical trials exploring the use of regenerative medicine and cell and tissue-based therapies for serious and life-threatening conditions.
- Promote Collaboration on Evidence Development and Expand Cell Characterization. The federal government should take actions to promote collaboration on evidence development, including funding support and incentives for collection of data on outcomes, as well as increased cell characterization, to improve evidence development and further increase understanding of the correlation of different products with clinical outcomes.
- Support Optimization and Scaling of Manufacturing. Achieving the goal of scalable, cost-effective, high-quality therapies involving human cells will require federal support, given the complexity of biomanufacturing. This should take the form of direct funding, as well as incentives to manufacturers to invest in these activities.
- Increase Workforce Development Activities. To address the needs of this growing field, the federal government should expand workforce development activities, including those within technical and community colleges, to build capacity and support the development of a skilled, technical workforce.
- Increase Capacity at the FDA. Additional resources should be provided to the Center for Biologics Evaluation and Research to respond to and provide increased support to applicants seeking regulatory approval and to increase enforcement against clinics and others who are not in compliance with relevant regulations.
The Alliance for Cell Therapy Now and the Alliance for Cell Therapy Foundation stand ready to assist policymakers as they consider and advance these and other strategies to advance the field.
About Alliance for Cell Therapy Now and the Alliance for Cell Therapy Foundation
Alliance for Cell Therapy Now and the Alliance for Cell Therapy Foundation (together referred to as the Alliance for Cell Therapy) are independent, non-profit organizations guided by leaders representing academic and medical institutions, industry innovators, and patients, that are working to advance safe and effective regenerative medicine and cell therapies for patients in need. See www.allianceforcelltherapynow.org