Challenges and facilitators in the development of orphan and paediatric medicines
Illustrative case studies on five different therapies developed in collaboration with the Office of Health Economics.
Challenges and facilitators in the development of orphan and paediatric medicines
We suggest that many of the barriers identified in this report – especially a lack of flexibility and pragmatism in regulatory and reimbursement policies – could be reduced through closer and earlier collaboration between industry and regulators/payers. Such collaboration could reduce uncertainty, time, and costs in the development process and encourage greater developments in the most urgent areas of unmet need.