Federal Funding Needed to Support Large-Scale, Randomized, Placebo-Controlled Clinical Trials

Thousands of clinical trials are underway in the United States to explore the use of cell-based therapies for many conditions, including neurological conditions, musculoskeletal conditions, cancer, cardiovascular disease, diabetes, wound healing, and immunological disease (including COVID-19). Cell-based therapies hold great hope for a range of conditions for which—to date—there has been no cure.

More than 90 percent of clinical trials involving cell-based therapies in the U.S. are either Phase 1 or Phase 2 trials. While results of early clinical studies are promising, the primary barrier to advancing such therapies is the significant cost of conducting large-scale, randomized clinical trials, which are a precursor to bringing safe and effective therapies to patients. The financial barriers are particularly problematic for academic and research institutions, as well as small biotechnology companies, who are responsible for the vast majority of clinical trials investigating the use of cell-based therapies in the United States.

Current federal support of cell-based therapies is lacking:

  • Less than one-half of one percent of total National Institutes of Health (NIH) funding over the last several years has been used to support clinical trials for cell-based therapies.
  • While the bipartisan 21st Century Cures Act did provide limited funding—$30 million—for regenerative medicine research, such funding expired on September 30, 2020.
  • A small number of states have recognized the need for funding and in fact, California voters approved a ballot measure in November 2020, providing $5.5 billion to support cell-based therapies through the California Institute of Regenerative Medicine (CIRM). Similar funding is needed to support these therapies for all Americans.

Federal support is urgently needed for cell-based therapies, including large-scale, randomized, placebo-controlled clinical trials to confirm early results, cell characterization, and optimization and scaling of manufacturing. Without federal support, the promise of cell-based therapies for patients in need will not be realized.

We urge policymakers to support the development and manufacturing of cell-based therapies for patients in need, including:

  • Large-scale, randomized, placebo-controlled clinical trials exploring the use of cell-based therapies for serious and life-threatening conditions;
  • Collaborative evidence development, including an outcomes database;
  • Clinical trials network to support sharing of best practices, lessons learned;
  • Support for characterization, optimization and scaling of manufacturing for such therapies;
  • Workforce development; and
  • Expert committee to oversee a program on cell-based therapies that cuts across multiple centers at the National Institutes of Health.