At the heart of PPMD’s mission to end Duchenne lies our unwavering commitment to accelerate research. As part of our comprehensive research strategy, this includes a focus on enhancing the drug development process to ensure we have an environment that increases the likelihood of approvals and the speed of development.

One of the programs we utilize to facilitate those advancements is PPMD’s Duchenne Drug Development Roundtable (DDDR). The DDDR is comprised of a number of pharmaceutical stakeholders who are brought together in a pre-competitive space to address challenges in drug development for Duchenne.

November 2021 Meeting Recap

Last month, PPMD was happy to host our first in-person meeting with our 2021 DDDR members at the TWA Hotel in New York City. This one-day meeting focused on increasing industry’s understanding on the current state of using external controls in clinical trial design.

External controls groups could be used in clinical trials to help reduce the number of patients on placebo, while increasing the power of studies to get us to answers about investigational products faster. The workshop focused on where the field is today and examples of how to deploy external controls in clinical trials. We hope to continue building on this effort to get answers on potential therapies faster.

Additionally, we held a sub-group meeting specific to gene therapy to discuss important ethical issues around clinical trials in the gene therapy landscape. Enhancing the informed consent process to better educate, empower, and prepare families was a central theme to the meeting. Those discussions around the informed consent process can be expanded beyond just gene therapy trials to ensure all families participating in any clinical trial feel informed and capable of making the right choices for their families.

We thank all our DDDR members for collaborating pre-competitively to solve issues in drug development for the benefit of our entire community.

2021 Duchenne Drug Development Roundtable Members