Global position statement on medicines for patients with rare diseases (Orphan drugs)
Developing orphan drugs to treat rare disease patients is a complex undertaking. The number of patients is small, and they are widely dispersed. There are also major logistical and regulatory issues due to minimal clinical research centers and sufficient expertise.
BMS recognizes the crucial need for sustained research and development of orphan drugs, and shares the goal of timely, equitable and sustainable access to orphan drug treatments.
BMS encourages the use of real-world evidence (RWE) and real-world data (RWD) when appropriate to keep pace with the rapidly advancing science and changing treatment paradigms to inform global regulatory bodies on continued innovation in orphan drug development. BMS also supports health technology assessment (HTA) practices that appropriately assess orphan drugs into the system and do not to delay patient access. Meanwhile, BMS opposes price controls that threaten patient access to needed orphan drugs. Special consideration and flexibility should be given to ensure that vulnerable patients with rare diseases have a sustained pathway to access.