PPMD’s PRO Project: Adding more tools to help with upcoming clinical trials in Duchenne

What’s a PRO?

Several patient-report outcomes (PROs) have been validated for use in people with Duchenne and their caregivers. Patient-report outcomes are a way for patients and families to provide structured input about their own experiences. PROs include a set of survey questions focused on one area such as physical ability, pain, or fatigue. For researchers, often the best PROs are ones that are “validated”. For a PRO to be validated means it has been studied in a standardized way in specific groups of people. During this validation process each question is checked, as well as the ability of the group of questions to measure something distinct and meaningful.

As you may have noticed, clinical trials are almost always focused on looking for a change in function—for example, a change to how far someone can walk or how well they climb stairs. While these outcomes are important to determining if a drug has a positive effect, there are other important quality of life outcomes such as energy level, social inclusion, and moods that may play an important role in how a person with Duchenne and his family manage (and possibly benefit from) being in a clinical trial.

PPMD PRO Project

PPMD’s DuchenneConnect, with support from Bristol-Myers Squibb, is working to identify areas that are important to our community but have been previously underrepresented in clinical trials. An example of a PRO that has been validated for neuromuscular conditions is the the Pediatric Outcomes Questionnaire (PODCI). This PRO was developed by the American Academy of Orthopedic Surgeons, the Orthopedic Society of North America, the American Academy of Pediatrics, and Shriner’s Hospitals for parental reporting of function in children 2-10 years old who have bone or muscle disorders. Areas of focus or “domains” include activities of daily living, mobility, and participation in sports and gym activities. The measure includes few items on social inclusion, sleep, and energy levels, and all of the questions are answered by the parents. We want to build off of measures like to PODCI to assess things that are important to you.

PROs are an important tool that can be used in clinical trials and other research to help measure meaningful differences over time. Our community needs more meaningful PROs so that we have as many tools in our toolbox to help with upcoming clinical trials. That’s why PPMD wants to take a deeper dive into what is most important to you – what would you value and expect as a result of a new therapeutic?

We (will) need you!

Soon, we will be reaching out to you via DuchenneConnect to share your experiences through a preliminary survey, so make sure to register on DuchenneConnect today. If you are already registered, please remember to update your Profile Survey at least once a year. 

Never has the patient voice been more critical in the fight to end Duchenne. And PPMD and DuchenneConnect will continue to collect your stories and your data so that this information can help reshape the Duchenne landscape.



Recently Cindy Nowinski, MD, PhD hosted a webinar on “Making PROs Fit Your Needs: Practical basics of patient reported outcome measures” as part of the PCORnet Best Practice Sharing Session. Click here to watch the webinar and learn more about PROs.

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