Parent Project Muscular Dystrophy (PPMD) and Duchenne UK announced the joint funding of a project to create a set of agreed and validated Patient Reported Outcomes (PROs) for use in Europe and the US. The $200,000 grant from these organizations is awarded to the lead researcher of the project, Dr. Chad Heatwole at the University of Rochester, New York.

This project aims to create an additional assessment tool for use in clinical trials to measure and report the small day-to-day benefits a drug might be having on patients with Duchenne muscular dystrophy (Duchenne) and their caregivers. Currently it is not possible to record whether, for example, patients on a trial find it easier to get in and out of their car, get themselves dressed or feed themselves.

Since disease progression in Duchenne is complex, there is a need for highly sensitive, disease-specific outcome measures to be developed to identify small, yet significant, treatment benefits. The symptoms of Duchenne are varied, and while very familiar to the patients themselves, have never have been studied to determine how truly common they are and what is most meaningful from the patient’s point of view.

This project aims to develop a set of globally agreed, highly sensitive, comprehensive and fully validated patient and caregiver reported outcome measures for Duchenne drug development. This will provide a more patient and caregiver focused evaluation of the effect of Duchenne treatments in clinical trials.

Dr. Heatwole will conduct a series of interviews with patients from Europe and the US to identify symptoms that are important, and using statistical analysis methods, develop a comprehensive set of patient reported outcomes according to regulatory guidelines from both the FDA and EMA.  Dr.  Heatwole and his team will draw from their experience developing Health Indices for close to 30 other diseases. This work will complement awards granted to Dr. Heatwole by other foundations.

The hope is that, if successful, this PRO will support drug development by providing novel and sensitive patient and caregiver information that better facilitates clinical trial assessment and subsequent drug approval.

Abby Bronson, PPMD’s Senior Vice President of Research Strategy said:

“We believe that incorporating the voice of the patient through PROs is an extremely powerful tool to support and accelerate drug development. We are grateful to be working with the Duchenne UK team to develop tools that ensure patients are heard.”

Emily Crossley, Co-founder of Duchenne UK, agreed, adding:

“Duchenne UK is committed to accelerating the drug development process in every possible way we can. We are very pleased to be collaborating with our friends at PPMD on this project which we hope will have a global impact on ensuring we can capture all the benefits a drug is having.”