RARE Daily

Cure Rare Disease Gets MDA Grant to Develop Reimbursement Strategies for Ultra-Rare Disease Therapies

February 16, 2023

Rare Daily Staff

The Muscular Dystrophy Association has awarded the nonprofit biotech company Cure Rare Disease an advocacy collaboration $30,000 grant to support research of novel reimbursement strategies for drugs developed for ultra-rare diseases, a key barrier to scaling the development of n-of-1 therapies and therapies for small patient populations.

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.

“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,” said Rich Horgan, founder and CEO of Cure Rare Disease. “Our goal is to find a solution to this critical economic challenge.”

The Muscular Dystrophy Association 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. The grants are for MDA collaborations 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.

Ultra-rare diseases are 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 said it considers the challenge of reimbursement for ultra-rare diseases closely tied to its mission of developing 18 potential therapeutics in its pipeline of candidates to treat 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.”

Photo: Rich Horgan, founder and CEO of Cure Rare Disease

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