Funds will support the design of novel reimbursement strategies for drugs developed for ultra-rare diseases

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. Applications for the program were announced in 2022 for MDA to collaborate with organizations focused on non-partisan, non-political advocacy initiatives to achieve legislative and public policy victories for the neuromuscular community at the federal, state, local level.

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.

Cure Rare Disease considers the challenge of reimbursement for ultra-rare diseases to be closely tied to its mission of developing 18 potential therapeutics in its pipeline of rare, neuromuscular conditions.

“While drug development is our primary focus, we’re dedicated to making this emerging ecosystem sustainable for drug developers and accessible and equitable for patient families,” Horgan said. “A new reimbursement model is necessary to finish the creation of this ecosystem, and to ensure that rare and ultra-rare disease patients are no longer left behind.”

ABOUT CURE RARE DISEASE

Cure Rare Disease, a 501(c)(3) nonprofit biotechnology company based in Boston, Mass., is transforming possibilities for people with rare diseases by developing advanced therapeutics in time to save lives. Through a collaborative community of forward-thinking families, patients, scientists and supporters, Cure Rare Disease has enabled an ecosystem of innovation and discovery to overcome the obstacles inherent in existing models of medicine and to expedite life-saving genetic technologies from research to the clinic. Learn more at www.cureraredisease.org.

ABOUT MUSCULAR DYSTROPHY ASSOCIATION

Muscular Dystrophy Association (MDA) is the #1 voluntary health organization in the United States for people living with muscular dystrophy, ALS, and related neuromuscular diseases. For over 70 years, MDA has led the way in accelerating research, advancing care, and advocating for the support of our families. MDA’s mission is to empower the people we serve to live longer, more independent lives. To learn more visit mda.org and follow MDA on Instagram, Facebook, Twitter, TikTok, and LinkedIn.

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