Duchenne & Becker MD Patient-Centered Benefits and Risks Analysis Given to FDA

Parent Project Muscular Dystrophy (PPMD) announced the submission of results from its recent patient-centered benefit-risk assessment (PCBR) study to the U.S. Food & Drug Administration (FDA).

PCBR studies incorporate patient assessment information and preferences in industry and regulatory decision-making. The PPMD study, developed in partnership with and sponsored by Santhera Pharmaceuticals, incorporated survey responses and feedback from Duchenne or Becker muscular dystrophy patients, the sponsor, and clinicians, and was based on results from a phase III clinical trial of Santhera’s Raxone/Catena (idebenone), used to attenuate respiratory function loss in Duchenne patients. The study enrolled 133 people, including patients (85% with Duchenne) and caregivers, and using Best-Worst Scaling (BWS) and a community-engaged approach, was divided into four main activities, according to PPMD’s press release:

• Activity 1: Prioritization of Treatment Targets, PPMD assessed priorities for treatments that address various Duchenne/Becker signs and symptoms that are not directly related to skeletal muscle function.
• Activity 2: Preferences for Pulmonary Therapies, PPMD assessed preferences for potential pulmonary therapies for Duchenne and Becker muscular dystrophy.
• Activity 3: Intentions to Use, PPMD validated and extended the findings of activity 2. After each treatment profile in activity 2, participants were asked whether they would choose the treatment for themselves (or for their child).
• Activity 4: Perceptions of Benefits and Harms, consisted of a set of questions where participants indicated their perception of an expanded set of benefit, risk, and burden attributes.

Researchers concluded that both patients and caregivers accept risks and burdens, highly rating the importance of pulmonary benefits. There was relatively little difference between patients and caregivers (76% of whom were  biological mothers) in their treatment priorities and risk tolerance.

Lead researcher Holly Peay, PhD, commented on the importance of these conclusions for future regulatory measures: “Based on this study, PPMD and collaborators from Johns Hopkins are hopeful the FDA will have a better understanding of preferences of Duchenne patients and caregivers in regards to a specific pulmonary therapeutic target. Especially when faced with low drug-associated risk and few side effects, we hope that this data will inform a permissive response by the agency in light of data showing patient and caregiver prioritization of pulmonary benefits.”