Today is Rare Disease Day, and amid cuts to the NIH and FDA, ?it's worth recognizing their critical role in shaping biotech and bringing relief to millions.
Nearly 10% of the U.S. population has a rare disease, and many are lifelong, life-threatening, and with no approved treatments. Before 1983, the rare disease landscape was bleak: little research, no incentives for drug development, and fewer than 10 drugs ever developed for rare diseases.
Then came the Orphan Drug Act of 1983 that changed everything. It introduced market exclusivity, tax credits, and grant funding. The FDA launched new programs like Orphan Drug Designation, Rare Pediatric Disease Designation, Fast Track, and Breakthrough Therapy Designation, fundamentally shifting the incentives for biotech. Since then, over 1,000 drugs have been approved for rare diseases.
Rare disease drug development is often more successful than other areas because the genetic mechanism of action is well understood—and that's almost always thanks to basic research done and funded by the NIH. These researchers uncover the drivers of these diseases. Even more directly, the NIH invests ~5% of its budget into early-stage biotech startups—the lifeblood of the pharmaceutical industry, to directly translate their discoveries into approved therapies.
These breakthroughs have changed countless lives. In cystic fibrosis, NHLBI and NCATS funded key research leading to the discovery of the CFTR gene. (Approved drugs Kalydeco, Orkambi, Trikafta). NIAMS and NINDS funded early exon-skipping research into Duchenne Muscular Dystrophy. (Exondys 51, Vyondys 53, Elevidys). NHLBI invested heavily in sickle cell disease research, leading to gene therapy breakthroughs. (Casgevy & Lyfgenia)
Our startup, ResVita Bio, has been awarded over $2.6M in NIH funding, supporting more than 10 scientists and advancing our therapy for Netherton Syndrome—a severe, life-threatening genetic skin disorder—from a concept to the brink of the clinic. The NIH SEED program provided critical early regulatory support, allowing us to engage with over a dozen FDA experts through the INTERACT program, an initiative designed to accelerate the development of novel therapies like gene and cell therapies. We can't bring therapies to Netherton patients without the NIH and FDA—and neither could any U.S. therapy.
Sickness isn’t political. Every person should be on the same side in this fight.
