As the NFL kicks off this week—with Buffalo defeating champion Los Angeles Rams yesterday—college athletes are helping battle rare diseases through the Young Investigator Draft, Good Day BIO writes.
Rare diseases are defined as diseases or conditions that affect less than 200,000 people in the United States, according to the U.S. Food and Drug Administration (FDA).
However, according to the National Institutes of Health (NIH), there are approximately 7,000 rare diseases affecting between 25 and 30 million Americans. This equates to 1 in 10 Americans—or one on every elevator and four on every bus.
And half of the known rare diseases affect children, while 30% of those children with rare diseases will not reach their fifth birthday, according to Uplifting Athletes, a nonprofit founded and led by university athletes to fight rare disease.
Rare disease research is needed
Uplifting Athletes holds the annual Young Investigator Draft, recognizing top young rare disease researchers. Patient advocacy organizations can nominate researchers serving the rare disease community here by Oct. 14. Winners receive $20,000 grants, co-funded by Uplifting Athletes and the nominating patient advocacy organization.
Research in this segment is of utmost importance as this is a very challenging matter. It is very difficult for drug makers to invest in years of development for a treatment that only a few thousand people will use, according to Good Day BIO.
Biotech and rare disease
Many Biotechnology Innovation Organization (BIO) members are sponsoring the draft, which gives special recognition to researchers from underrepresented backgrounds.
Meanwhile, efforts like the Institute for Life Changing Medicines, a non-profit with a for-profit model, are seeking to help.
And as just one example, the success of Global Blood Therapeutics, which was just sold to Pfizer after successfully launching groundbreaking therapeutics for sickle cell disease, shows the possibilities when we invest in rare disease research.
According to Good Day BIO, the discussion of the extension of the Prescription Drug User Fee Act (PDUFA), which expires at the end of September, includes ill-considered proposals to narrow FDA’s Accelerated Approval pathway. However, studies show the Accelerated Approval pathway actually benefits drug development for rare disease.
This is why more good policies are urgently needed, like the 40-year-old Orphan Drug Act, which was a game changer for rare diseases.
In the meantime, we can uplift the next generation of researchers to ensure we have the best minds to develop the cures of tomorrow.
