PPMD’s Patient Preference Study about Pulmonary Outcomes – What we learned and why it matters

Late in 2015, Parent Project Muscular Dystrophy (PPMD) and our research partners at Johns Hopkins Bloomberg School of Public Health (JHSPH) released the results of our second patient-centered benefit-risk assessment (PCBR) study. This study aimed to evaluate patient and caregiver preferences regarding pulmonary therapies for Duchenne/Becker muscular dystrophy. The study builds on an ongoing collaboration between PPMD and investigators at JHSPH.  Santhera Pharmaceuticals helped sponsor the study. The variables that we explored in this study are based on data from Santhera’s phase III clinical trial of Raxone®/Catena® (idebenone). This potential therapy is targeted at slowing the loss of lung function in patients with Duchenne.


PPMD once again utilized a model of community engagement in determining research priorities, designing a research instrument, and interpreting and disseminating the findings.


Today we are pleased to release the Community Report (download), which takes a deeper look into the results of this study. Later this year, an extended version of the report data will be published in multiple publications.


What We Aimed to Learn in the Study


The study aimed to evaluate:


  • To what degree our community viewed pulmonary outcomes as a meaningful benefit; 

  • What the maximal acceptable risks, harms, and/or burden given the pulmonary benefit; and 

  • The degree to which preferences differed for caregivers and patients. 


Survey Activities


Caregivers and Patients were surveyed through a number of different activities:

Read the full community report about the study for a closer look at the activities and results. 


What We Learned and Why it Matters 

Based on this study, PPMD and our collaborators from JHSPH are hopeful the FDA will have a better understanding of patient preferences in Duchenne patients and caregivers in regards to a specific treatment to maintain lung function. Especially when faced with low drug-associated risk and few side effects, we hope that this data will inform a permissive response by FDA in light of data showing patient and caregiver prioritization of pulmonary benefits. Importantly, Santhera will use this patient reported/patient preference data in their regulatory submission to the FDA to incorporate the patient voice into the regulatory process.


Studies like these can aid industry in designing trials and selecting drug targets and end points that address what matters most to patients and caregivers. These studies provide patients and caregivers a practical and active way to engage with industry and regulators that goes beyond focus groups and testimony submissions. The goal, as always, is to collect meaningful patient data that can inform companies and regulators to speed the development of potential therapies.


We’d like to thank everyone who participated in the study, those who served on our community stakeholder advisory board to develop the survey and all those who took the survey.


Interested in participating in research like this?

Please contact us and let us know.

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