Rare Disease Advocates Urge for Early Patient Input in Research

Early patient engagement should be a federal requirement for companies looking to launch clinical trials on rare disease treatments, medical researchers and patient advocates said Monday.

Both the Food and Drug Administration and Congress could act to ensure patient voices are a key component of the research development process, advocates argued in a webinar held in honor of World Rare Disease Day. Early input before a trial, rather than just during or afterward, can help ensure researchers use effective biomarkers to measure a potential treatment’s impact on patients.

“Unless we are rewarding and acknowledging and really pursuing that companies make ...