WASHINGTON, DC—Robert Califf, MD, commissioner of the US Food and Drug Administration (FDA), today announced 19 new grants worth more than $38 million over the next 4 years to support clinical trials, natural history studies, and regulatory science tools related to rare diseases.
The grants, funded by the FDA’s Orphan Products Grants Program, are aimed at accelerating access to critical therapies for amyotrophic lateral sclerosis (ALS) and other neurodegenerative diseases. In addition, the agency awarded more than $25 million spread over the next 4 years to 11 clinical trials that support product development for rare disease treatments; 7 of the awards fund studies on rare cancers.
Dr. Califf’s keynote speech at this year’s Rare Diseases & Orphan Products Breakthrough Summit—sponsored by the National Organization for Rare Disorders (NORD)—kicked off NORD’s first in-person event since the 2019 summit, which preceded the COVID-19 pandemic.
“Patient involvement and engagement is a critical component of our efforts at the FDA,” he said. “By listening to patient voices, and by incorporating and learning from that engagement and guidance, we gain a better understanding of diseases and their treatments. The experience and input of patients can play a key role in scientific discovery, clinical evaluation of interventions, and other aspects of medicine.”
Significantly, Dr. Califf’s speech followed presentations by 3 patient advocates: Nakisha Isom, who has Hold-Oram syndrome; Annie Papik, whose son has an ultrarare mutation of the MTHFS gene; and Brittany Clayborne, a heart transplant recipient with peripartum cardiomyopathy and post-transplant lymphoproliferative disorder.
Dr. Califf, who led the FDA from February 2016 to January 2017, was sworn in as the agency’s 26th commissioner on February 17, 2022. A longtime professor of medicine at Duke University in Durham, North Carolina, he’s a noted cardiologist and has led many landmark clinical trials. One of the most frequently cited authors in biomedical science, Dr. Califf has published more than 1300 articles in peer-reviewed literature.
Dr. Califf said that at present, scientists know of more than 7000 rare diseases that cumulatively affect 30 million Americans, half of them children. Since the passage of the Orphan Drug Act in 1983, the FDA has approved some 1000 therapies, including 50 in 2021 alone—more than half of which are aimed specifically at treating rare or orphan diseases.
“Against this backdrop of success, much more needs to be done,” he said. “We still face enormous challenges which come with significant costs. The nature of rare diseases means there’s a limited number of people to participate in clinical trials. While we will continue to see important and often remarkable progress, the vast majority of drugs will probably not make it to market because of unexpected toxicity or failure to have beneficial effects.”