Rare diseases are characterized by a broad diversity of disorders and symptoms that vary not only from disease to disease but also from patient to patient suffering from the same disease. Rare diseases can be chronic, progressive, degenerative, and often life-threatening. Given the small patient populations, traditional development pathways may not be relevant or necessary, requiring novel and innovative approaches. From a market access perspective, lack of comparative effectiveness and long-term outcomes data can lead to delays in reimbursement and significant post-launch evidence generation commitments. Treatment costs and patient benefits may not be matched over time giving rise to difficulties with assessing the value and establishing the necessary funding pathways. VCLS is at the forefront of designing new strategies to bring effective and timely treatments to patients in these high unmet need rare disease areas.
This presentation provides an outline for smarter orphan drug development through:
- Integrating development approach
- Reducing cost and time
- Maximizing patient access