Measuring Outcomes

Regenerative Medicine and Cell Therapies

Regenerative medicine and cell therapies focus on repairing, replacing, and restoring function to tissues or organs that have been damaged due to disease, trauma, or congenital issues. Many different types of cells are being explored for use in these therapies, including mesenchymal stromal cells (MSCs, which can be derived from bone marrow, cord blood or cord tissue, adipose tissue, placenta or other sources), hematopoietic stem cells, skeletal muscle stem cells, lymphocytes, induced pluripotent stem cells, and many others.

There are thousands of clinical trials underway to explore the use of RMCTs for many conditions, including cancer, cardiovascular disease, diabetes, degenerative joint conditions, neurological conditions, wound healing, and most recently, COVID-19.

The 21st Century Cures Act laid the groundwork for making safe and effective regenerative medicine and cell 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 by providing initial funds for research on regenerative medicine through the National Institutes of Health.

A method for systematically tracking health outcomes, safety, and efficacy of treatments is needed, which can be accomplished through an outcomes database or registry.

Benefits of an Outcomes Database or Registry

An outcomes database for regenerative cell therapy offers many benefits:

  • Creating a community of high-quality researchers, providers, and manufacturers who agree to share data and increase accountability to patients and the public;
  • Monitoring safety of novel products undergoing testing in the clinic;
  • Measuring and improving short- and long-term outcomes for patients;
  • 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; and
  • Providing another means of real-world evidence development for regulatory decision-making.

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.

Stakeholder Support for an Outcomes Database 

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.

Congressional Support for an Outcomes Database

Congress also supports an outcomes database and support for a registry and demonstrated such support through the inclusion of language encouraging the NIH, in collaboration with FDA and HRSA, to explore the feasibility and utility of an outcomes database for adult cellular therapies that are either FDA-approved or are being administered under FDA Investigational New Drug or Investigational Device Exemption protocol, engaging experts and stakeholders to define data types and standards necessary for such a database.