Estimating the value of orphan drugs for rare diseases is challenging due to various reasons outlined in a scoping review by Grand et al. (2024). Key issues include small sample sizes, lack of data, and uncertainties in disease characteristics, clinical effectiveness, costs, quality of life, cost-effectiveness, budget impact, and value/reimbursement. The authors suggest solutions such as collaborating with patient advocacy groups, establishing disease registries, and exploring outcomes-based payment models. While these approaches have benefits, they also present challenges, particularly in terms of cost. Learn more about these challenges and opportunities in rare disease economic evaluations.
