Patients living with rare and chronic illnesses are all too familiar with unknowns. Only about 5% of the more than 7,000 known rare diseases have an FDA-approved treatment, meaning that the vast majority of patients, including myself, are living with no treatment for their condition. Many patients in the rare disease community are holding out hope that we’ll be able to access new treatments and cures as they’re researched and developed. Yet, several state and federal policies intended to lower drug prices can have disastrous consequences for rare disease patients.
Maryland policymakers must protect rare disease patients
January 10, 2024