FDA Issues “Draft Guidance” to Accelerate Drug Development for Duchenne Muscular Dystrophy: Pioneering Collaboration Between the FDA and Patient Advocates
Connexion Healthcare’s Rare Disease Center of Excellence noted with interest on June 9, 2015, that the FDA issued breakthrough “draft guidance” to help drug companies develop treatments for the rare disease Duchenne muscular dystrophy (DMD) and related conditions.
For the first time, a patient advocacy group—Parent Project Muscular Dystrophy (PPMD)—was directly involved in drafting guidance for a rare disease. This pioneering collaboration between a regulatory agency and representatives of the DMD community is a shining example of how input from patients and families can help to further the development of drugs.
DMD, one of nine types of muscular dystrophy, is a serious, life-limiting disease. DMD is an X-linked recessive disorder, and is the most common muscular dystrophy seen in children (mostly in boys). DMD has an annual incidence of one in every 3600 to 6000 newborn males. Signs and symptoms typically begin at 3 to 5 years of age. DMD results from a shortage of dystrophin in muscles, leading to progressive muscle weakness and degeneration. Children have difficulty walking, running, and climbing stairs; frequent falls; muscle stiffness and pain; and learning difficulties. DMD causes degeneration of skeletal and cardiac muscle, leading to respiratory and cardiac failure and premature death.
No cure for DMD is currently available.
Today’s DMD treatments aim for symptomatic management, helping to maintain function and comfort, improve quality of life, and sometimes increase lifespan. One major goal of the FDA draft guidance is to allow companies to initiate speedier and more efficient drug development programs for DMD.
The FDA draft guidance is an official “map” that describes the ways in which innovative therapies for DMD will move through the regulatory approval process. The guidance provides insight into the FDA’s current view of various aspects of DMD drug development, such as early clinical development, treatments at different stages of the disease, safety and efficacy, design of clinical trials, accelerated approval, and risk–benefit considerations.
Because of confidentially between the FDA and drug companies, the public is not able to know the FDA’s perspectives on DMD or the extent to which FDA decision-makers are willing to listen to the experiences of patients and families and realize their sense of urgency. Enabling easy access to the FDA’s perspectives and accelerated FDA review of potential therapies are 2 of the primary goals of this draft guidance. This draft guidance encourages the FDA and trial sponsors to engage patients and families at all stages of clinical trial development and to take into account what they consider to be “acceptable risk” in clinical trials.
A recent study in the journal Clinical Therapeutics surveyed 120 parents of children with DMD regarding “acceptable risk.” The results showed that parents will accept substantial risk related to a potential therapy when that risk is balanced against slowing or stopping of the progression of muscle weakness, even with no increase in life expectancy.
Research into the development of disease-modifying therapies for DMD is underway. The unique step by the FDA of providing this draft guidance will change the landscape of DMD and other dystrophinopathies, offering legitimate promise of effective treatment for DMD to patients and their families, possibly in the not too distant future.
For more information, please visit the Muscular Dystrophy Association Web site at: http://www.mda.org/
About the Connexion Healthcare Rare Disease Center of Excellence
The Connexion Healthcare Rare Disease Center of Excellence specializes in communicating the science behind rare diseases and orphan drugs. We strategically partner with industry leaders in the rare disease community to proactively connect all stakeholders in launching products to underserved patient populations. Our team comprises skilled communications experts who plan, develop, and disseminate scientific communications based on their advanced knowledge of the science, the patient journey, healthcare providers, and the orphan drug approval process.
At Connexion, our cross-functional teams navigate complicated treatment and market landscapes by elucidating the appropriate pathways to brand success. We understand how brand strategy drives market share. Since launching its dedicated Rare Disease Center of Excellence, Connexion has developed prelaunch, launch, and postlaunch communications in numerous rare diseases and disorders: congenital and genetic, movement, metabolic, and lysosomal storage disorders; lung, blood, skin, and endocrine diseases; and rare cancers.
For further information regarding the Rare Disease Center of Excellence at Connexion Healthcare and how we can develop rare disease communications to differentiate therapies by their unique attributes, contact:
Susan Stein, MPH
Rare Disease Center of Excellence