As the approval of therapies in Duchenne becomes more of a reality for our community, access becomes critical. Potential challenges with insurance coverage and approvals, as well as specialty pharmacy access begin to become a very real concern. And typically new and innovative therapies marketed to small patient communities are very expensive. Lingering questions remain about coverage for drugs that go through the accelerated approval process.
For many months, PPMD has been actively engaged with payer groups and coalition partners convened by FasterCures including the CF Foundation, JDRF, Michael J. Fox Foundation, and others around efforts to ensure that the families who are eligible for these life-altering treatments will have ACCESS to them. As the Duchenne Therapy Pipeline heats up and we work to ensure that reviews are conducted swiftly, be assured that PPMD is focused on making sure that these promising drugs are more than just approved; they must be delivered into the hands of the families who need them.
All of PPMD’s policy efforts – including DuchenneConnect, the Benefit-Risk study, the Duchenne Guidance, the MD-CARE Act, the Putting Patients First and Patients Are Waiting white papers, the burden of disease publication from DuchenneConnect, 21st Century Cures and PDUFA efforts, and much more — are all a part of the evidence base that will be necessary to justify therapy access and payment once the life-altering drugs we’ve long worked for are approved. For a better understanding of this landscape, check out the video below created by our partners at FasterCures.
PhRMA has also unveiled “Four Facts About Spending on Prescription Medicines,” to help put medicine costs in context of overall health care spending.