President Joe Biden garnered national attention this year when he formally launched his cancer moonshot initiative, which, most notably, aims to cut the cancer death rate by 50% in the next 25 years.
Now, European stakeholders are rallying for a moonshot program of their own, this time focused on rare diseases. The new coalition, announced Tuesday evening as part of the European Health Summit, unites seven European organizations involved in drug development to pool resources and direct attention toward advancing rare disease therapies.
The announcement, admittedly, has few details. But the project is set to focus on bolstering the research ecosystem so more early-stage assets can be born, optimizing clinical trials so they can function with few rare disease participants and improving the diagnosis to treatment infrastructure.
The coalition's members span all corners of drug development. In all, the group includes the Critical Path Institute, the European Infrastructure for Translational Medicine, the European Clinical Research Infrastructure Network, the European Federation of Pharmaceutical Industries and Associations, the European Confederation of Pharmaceutical Entrepreneurs, EuropaBio and EURORDIS-Rare Diseases Europe. The announcement says the coalition formed in response to the European Commission’s call for addressing (PDF) unmet medical needs.
But the calls for prioritizing rare diseases are not unique to Europe. Following the U.S.' effort to rapidly develop COVID-19 vaccines and therapies—dubbed “Operation Warp Speed”—government officials and advocates have pushed to replicate the effort in other disease areas, including rare conditions. In May, the FDA’s Center for Drug Evaluation and Research (CDER) launched the new Accelerating Rare disease Cures program to try and do just that.
Peter Marks, M.D., Ph.D., head of CDER’s sister organization the Center for Biologics Evaluation and Research, echoed this objective on a call with an FDA lobbying group in May, focusing on efforts to improve regulatory feedback for the cell and gene therapy field. To do that, the agency needs to focus on hiring and retaining more permanent staff, Marks said. It’s a tall task that’s long plagued the FDA, given how lucrative private sector jobs can be.
“Parents of patients, of kids who have severe diseases, they don't want to hear about Type A, Type B and Type C meeting timelines,” he said at the time. “They want to know what we're doing to truly make a difference in trying to bring something better to their children's lives.”