Rare Disease Day Reminds Biopharma of Opportunities, Challenges

Yesterday was Rare Disease Day 2024. It was a reminder of the importance of raising awareness for rare diseases—defined as those that affect fewer than 200,000 people in the U.S., but collectively affecting some 30 million Americans. While much has been achieved in improving access to lifesaving and life-changing treatments, much more remains to be done for patients with these disorders.