Advancing equity in rare disease trials

Equity in rare and genetic disease clinical trials is both a scientific necessity and an ethical responsibility. Unlike patients with more common conditions, those living with rare diseases often face longer diagnostic journeys, fewer treatment options and limited access to specialty care and clinical research trials. With these challenges, it is critical that rare disease patients and families are able to benefit from medical innovations leading to potential treatments. Advancing equity requires tailored, patient-centered approaches that reflect health care system infrastructure, as well as cultural, geographic and socioeconomic realities. Community-based site networks, thoughtful application of digital technology, flexible study designs and inclusive protocols are key to enabling broader participation in rare disease clinical trials. Sustained collaboration across industry, academia, regulators and advocacy groups can ensure that no patient is left behind in the pursuit of treatment and discovery.

Authors:

• Rose Blackburne, M.D., MBA – PPD™ clinical research business of Thermo Fisher Scientific
• Galina Nesterova, M.D. – PPD™ clinical research business of Thermo Fisher Scientific
• Radojica Savic, M.D. – PPD™ clinical research business of Thermo Fisher Scientific