Table 1 Summary of proposed recommendations
From: Rare disease challenges and potential actions in the Middle East
Survey domain | Recommendations |
|---|---|
Disease awareness | Collaboration between the Ministry of Health (MoH) and pharmaceutical companies to improve rare disease policies, registries, awareness, and diagnosis |
Increasing centers for diagnosis of rare diseases | |
Training healthcare professionals on using diagnostic tools | |
Establishing patient registries to reduce uncertainty in clinical evidence and improve patient access to drugs (UAE) | |
Marketing authorization | Developing a separate registration process for orphan drugs with clear requirements and timelines |
Reimbursement, pricing, & HTA | Establishing a pricing process for rare diseases which does not depend on external price referencing |
Introducing a cost-effectiveness threshold for non-orphan drugs and a differential higher cost-effectiveness threshold for orphan drugs | |
Creating affordability programs to improve patients’ access to orphan products | |
Supporting patient advocacy groups to allow for patients’ engagement in decision-making in the long run | |
Orphan designation | Developing a guideline that considers how to identify rare diseases and implementing such guidelines in registration, pricing, and reimbursement |
Policy incentives & special access programs | Increasing the use of managed entry agreements for orphan drugs |
Increase the use of outcomes-based managed entry agreements (Egypt) |