PPMD has been working for many years to engage critical policy makers and stakeholders within the regulatory and access environments. While we have learned a great deal over the past two years as we have worked with our partners to navigate the complex access environment, we have gleaned insights and that we now plan to expand upon. The bottom line is that the potential promise of the innovative therapies that are moving through the Duchenne pipeline will only be realized when families are able to access them. This is PPMD’s goal.
Today, PPMD is hosting our first Duchenne Clinical Outcomes Workshop just outside of Washington, DC. This full day meeting brings together regulatory agencies, clinicians, advocacy organizations, and industry in an effort to:
- Develop a common understanding about the clinical outcome data being collected by Duchenne researchers and registries;
- Identify gaps in those data that are most relevant to decision makers about clinical use;
- Discuss potential approaches and opportunities to facilitate collection of data that would help fill the identified gaps; and
- Build consensus around prioritization of activities to undertake to help fill the identified gaps.
We are proud to convene this incredible group of thought leaders in the Duchenne community and begin, what we hope will be the first of many important discussions around clinical outcomes. PPMD will provide the community with a full report, including next steps, in the coming weeks.