PPMD Gathers DMD Experts to Consider Pulmonary Outcomes Measures Used in Clinical Studies

Margarida Azevedo, MSc avatar

by Margarida Azevedo, MSc |

PPMD workshop

Parent Project Muscular Dystrophy (PPMD), the largest U.S. nonprofit group focused on Duchenne muscular dystrophy (DMD), is holding a Pulmonary Outcomes Workshop this week, April 14–15. Physicians and industry experts will discuss pulmonary outcome measures used in current DMD clinical trials, how those measures might be improved, and their use in furthering drug development.

The workshop, being held in Bethesda, Maryland, has the support of Santhera Pharmaceuticals, Catabasis Pharmaceuticals, and Pfizer Inc.

As DMD progresses, respiratory muscles progressively weaken leading to changes in pulmonary function, making the process of breathing and coughing increasingly difficult for patients. In light of these organ-specific effects, clinical trials that aim to improve pulmonary function in DMD apply several measures to demonstrate changes in DMD pulmonary function. Such measures include changes in inspiratory strength, as measured by maximal inspiratory pressure (MIP), changes in expiratory strength, as measured by maximal expiratory pressure (MEP) and peak expiratory pressure (PEP) and changes in the volume of air that the lungs can hold, as measured by forced vital capacity (FVC), forced vital capacity when compared with normal (FVC%) and peak expiratory flow (PEF).

PPMD believes it is time to reassess these measures, and the goals of the Pulmonary Outcomes Workshop include answering questions regarding the current measures’ clinical meaningfulness and possible limitations. Experts will discuss if there is a need to implement new measures in light of new knowledge, and how such knowledge may improve and facilitate drug development. The forum also serves as an educational platform and an opportunity to discuss protocols and data analyses used in clinical trials.

“It is our hope that this workshop will help to clarify much of the confusion around pulmonary outcome measures.  We must begin by agreeing on which pulmonary outcome measures to collect, how to analyze these measures, and the meaningfulness of these measures,” Kathi Kinnett, MSN, CNP,  senior vice president of Clinical Care at PPMD, said  in a news release. “If we can agree on these issues, we can begin to demonstrate the efficacy of drugs on pulmonary function and, hopefully, speed up the drug approval process. It’s a lofty goal but, with this panel of experts, we believe we are up to the task.”

The workshop’s results and conclusions will be published on the organization’s website.