Measuring Outcomes

Regenerative Cell Therapies

Regenerative cellular therapy—which involves the replacement or regeneration of human cells or tissues to restore or establish normal function in the human body—represents the next generation of groundbreaking treatments that are showing great promise in cardiology, neurology, oncology, orthopedics, ophthalmology, and other areas. Research is also now underway to explore the use of regenerative cell therapies to help patients with Alzheimer’s disease, cancer, cerebral palsy, diabetes, and Parkinson’s disease.

The 21st Century Cures Act contained several provisions to make safe and effective regenerative cellular therapies available to patients, including extending Food and Drug Administration (FDA) expedited programs to include regenerative therapies through the Regenerative Medicine Advanced Therapy program and providing funds for research on regenerative medicine through the National Institutes of Health.

The next step is to establish a method for systematically tracking health outcomes, safety, and efficacy of treatments through a national outcomes database or registry.

Benefits of an Outcomes Database or Registry

Key benefits and goals for a national outcomes database or registry for regenerative cell therapies include the following:

  • Measuring and improving short- and long-term outcomes for patients
  • Monitoring safety of novel products undergoing testing in the clinic
  • Advancing the science of cell therapy by assessing trends, identifying promising approaches, and facilitating collaboration
  • Improving development, manufacturing, and delivery of treatments by tracking clinical and patient-reported outcomes, including long-term results
  • Informing decision-making regarding coverage and payment
  • Providing another means of evidence development for regulatory decision-making (e.g., real-world evidence)
  • Creating a community of high-quality providers who agree to share data and be accountable to patients and the public

The development and launch of a national registry can help separate quality providers from “bad actors” that administer unsafe, unapproved treatments that put patients’ health at risk. Any federally-funded outcomes database or registry should only include treatments approved by the FDA or conducted under an FDA-administered IND or IDE.

Support for a Registry

Organizations representing scientists and researchers, patients, practitioners, and other health care organizations support the launch of a national registry for regenerative cell therapy. The latest list of supporters can be found here.