When the pandemic began in March 2020, the scientific community reacted with a dramatic increase in COVID-19 clinical trials, but progress in other therapeutic areas stalled due to safety concerns. The new safety and logistical challenges added by the pandemic resulted in increased costs, delays, and uncertainty in trial initiation and completion for rare disease drug manufacturers. Rare disease patients have conditions that make them more susceptible to and higher risk for COVID-19, making it especially difficult for this population to participate in clinical trials.
By October 2020, an estimated 80 percent of non-COVID trials were stopped or delayed during the pandemic. Over 700 trials experienced disruptions since the beginning of the pandemic, with over 250 suspending enrollment due to the pandemic. In May 2020, NIH Director, Dr. Francis Collins estimated that $10 billion of research outside of COVID-19 had been lost. Given the tumultuous two years that have followed, the amount of lost research has continued to multiply.
Many of the companies investing in rare disease research are small biotechnology companies that face particularly stark financial challenges, leaving them with exceedingly difficult choices on whether to resume their trials or stay in business altogether. Much of the critically necessary rare disease clinical research remains stalled, while millions of patients are waiting for breakthrough treatments and cures for the diseases that threaten their lives.
The RDCC is grateful for the leadership of Representative Josh Gottheimer (D-NJ) and Representative Don Bacon (R-Neb) to co-sponsor Leo’s Law, formerly called The Orphan Drug COVID-19 Mitigation Act, and push for its inclusion in the next available legislative vehicle. Learn more on this important issue which will help to bring hope and needed cures to the at least 95 percent of rare diseases currently lacking an FDA-approved treatment in this letter.