The funding needed to develop individualized medicines remains a significant challenge. At present, it relies largely on the dedication of parents of children with unique genetic diseases, with only a limited number of additional stakeholders supporting their efforts to identify effective treatments. Regulatory agencies are now exploring new regulatory approaches to streamline the approval process for individualized therapies. However, robust payment and reimbursement models that would ensure the sustainable development of these therapies are still lacking.
The funding and access work group is focusing on building a comprehensive map of nonprofit, for‑profit, and governmental stakeholders to help the community identify partners they can reach out to for support. We also identify key economic and clinical points to build a data set that could potentially be used in future reimbursement negotiations with health economic agencies.