Scientific advances are making new treatments possible for some of the more than 7,000 rare diseases that currently impact one in ten Americans.
Yet, some advocates say current regulatory measures are holding back further progress and lack engagement with rare disease patients and experts. Efforts to change the status quo are ongoing on Capitol Hill. Legislation introduced in Congress seeks to improve protocols for rare diseases therapies, drug review processes and patient access to treatment.Â
On World Rare Disease Day, The Hill will convene policymakers, healthcare professionals, researchers, clinicians and patient advocates for a comprehensive discussion about the nuances of regulatory reforms and policy implications for the 30 million Americans living with rare diseases.Â