In collaboration with reimbursement expert Eric Faulkner, former global VP of patient value and access at Novartis Gene Therapies, and the Muscular Dystrophy Association, we have developed an initial report to establish a novel pathway to advance treatments for ultra-rare diseases.
This is the first comprehensive approach mapped to date in the U.S. that offers a potential pathway for ultra-rare disease patients to access potentially life-saving therapies. The proposed pathway offers a precedent upon which we can rapidly build and execute this pathway in the U.S.
In addition to drafting the initial proposal for an Angel Treatment Pathway, our team held meetings with industry, payers and advocacy groups to gather feedback, insight, and pressure test the details of the proposal from various perspectives in the rare disease ecosystem.
The next step is to conduct pilot clinical studies to generate data necessary to convince public and private payers (insurance companies) to adopt a reimbursement program for non-commercial therapeutic efforts for neglected populations.
Complete the form below to download the full text of the initial report.