Accelerating Rare Disease Drug Development: Lessons Learned from Muscular Dystrophy Patient Advocacy Groups
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ANALYTICAL REPORT
Accelerating Rare Disease Drug Development: Lessons Learned from Muscular Dystrophy Patient Advocacy Groups Raymond A. Huml, MS, DVM, RAC1 · Jill Dawson, PhD2 · Michelle Bailey, MD1 · Nermina Nakas, MD, MPH1 · Jane Williams, MD, MPH1 · Maryna Kolochavina, PharmD, PMP1 · Jonathan R. Huml, BA, BSc3 Received: 1 July 2020 / Accepted: 9 September 2020 © The Drug Information Association, Inc 2020
Abstract With scientific and molecular advancements related to disease pathogenesis, advances in gene and stem cell therapies, and the promise of lucrative markets for biopharmaceutical companies, there has been a rapid expansion in the number of potential new muscular dystrophy (MD) treatments. The first champion for a newly diagnosed MD patient and their caregivers is typically an MD-specific patient advocacy group (PAG). Muscular dystrophy PAGs have been among the most active in the rare disease drug development space. Notable achievements in the last decade include promulgating the first U.S. clinical research guidance, setting up registries and natural history studies, and investing in companies—some of which have brought potentially disease-modifying products to the market. This paper will discuss five key strategies that have been successfully employed by MD PAGs to advance treatments: (1) creating a national registry, (2) understanding the barriers to identifying patients with certain subtypes of muscular dystrophy to participate in clinical trials, (3) partnering with the biopharmaceutical industry, (4) collaborating with the regulators, and (5) incorporating market access and use insights early in clinical development. While clearly helpful within the MD community, these tactics could also be employed by PAGs representing other types of rare diseases. Keywords Rare disease drug development · Muscular dystrophy · Patient advocacy
Background: Muscular Dystrophies Rare diseases were once a neglected aspect of pharmaceutical research, relegated to non-profits and philanthropic projects. Today, with new blockbuster drugs being few and far between, and regulators incentivizing drug developers to pursue unmet needs, drug targets are increasingly being elucidated and companies are investing billions in rare disease research—with impressive results. Gene and stem cell therapies are the breakthrough treatments for a majority of rare diseases. Examples include a gene therapy treatment for spinal muscular atrophy (SMA), an ultra-rare neuromuscular disease, in children under two years of age (AveXis’ * Raymond A. Huml [email protected] 1
Syneos Health®, Morrisville, NC, USA
2
Danville, CA, USA
3
University of North Carolina at Chapel Hill, Chapel Hill, NC, USA
Zolgensma®), and the first gene therapy for an inherited retinal disease (Spark Therapeutics’ Luxturna®). Muscular dystrophy (MD)—a group of inherited, rare, degenerative muscle diseases that cause progressive muscle weakness due to defects in genes affecting the expression of muscle proteins—has a range of complex features
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