Cure Rare Disease, a clinical-stage nonprofit biotechnology company, has been awarded an advocacy collaboration grant from the Muscular Dystrophy Association (MDA) to support research on novel reimbursement strategies for drugs developed for ultra-rare diseases, a key challenge for rare disease patients, including muscular dystrophy patients. As reimbursement decisions are dependent upon commercial approval through large clinical trials, potential therapies developed for the rare and ultra-rare diseases are unlikely to reach the threshold of criteria to support reimbursement.
“We are honored to have MDA’s support as we look to tackle one of the greatest barriers for ultra-rare disease patients to access potentially life-saving medicines,” said Rich Horgan, founder and chief executive officer of Cure Rare Disease. “Lack of reimbursement makes this an inequitable playing field for ultra-rare disease families, many of whom have shouldered the cost of drug development for their loved ones, while simultaneously acting as caregiver and parent. Our goal is to find a solution to this critical economic challenge.”
A leading health organization in the United States for people living with muscular dystrophy, ALS and related neuromuscular diseases, the Muscular Dystrophy Association has awarded the grant to Cure Rare Disease as part of the organization’s Advocacy Collaboration Grants Program, which seeks to fund public policy and advocacy projects that will benefit the neuromuscular disease community.
According to the Food and Drug Administration, rare diseases are defined as any condition impacting fewer than 200,000 people in the United States. Ultra-rare diseases impact even fewer, making them a challenging fit for traditional drug development, which often takes place in a commercial setting and relies upon investment that may favor science that could impact diseases affecting large populations of people. In addition to being commercially unattractive due to individually small prevalence numbers, these conditions cannot readily be studied through well-powered clinical trials that recruit large numbers of participants. A reimbursement process has yet to be established for these circumstances.
SOURCE: Businesswire