March 10, 2020 / Advocacy

PPMD Leads Duchenne Community to Nation’s Capital for Annual Advocacy Conference

Parent Project Muscular Dystrophy (PPMD) heads to Washington, D.C., March 8–10, for our annual Advocacy Conference. Over 100 families are expected to join PPMD on Capitol Hill to help inform Members of Congress about the critical need to continue investing federal resources in Duchenne specific priorities and programs and to garner support on the recently introduced BENEFIT Act.

PPMD’s Founding President and CEO, Pat Furlong, credits the success of the organization’s advocacy program with the families that attend the annual meeting in Washington: “PPMD has been advocating in Washington D.C. for close to two decades. Duchenne families have been able to change the landscape of Duchenne care and research because of their consistent, unwavering advocacy efforts and an ability to build relationships with members of Congress. By sharing their stories, their personal experience with Duchenne, lawmakers better understand the importance of federal support in the fight to end Duchenne.”

As they do every year, the advocates will be stressing the importance of supporting federal funding for research and essential programs. This year’s request focuses on:

  • Funding for the Centers for Disease Control and Prevention (CDC) Muscular Dystrophy program, as well as evaluation of the impact of efforts and initiatives on outcomes, such as reducing delays to diagnosis;
  • Improving research models and data sharing at the National Institutes of Health (NIH);
  • Encouraging FDA to include in the benefit-risk framework for drug approval, a description of how patient experience data was considered; and
  • Funding for Duchenne research within the Department of Defense’s Congressional Directed Medical Research Program (CDMRP).

In addition, the advocates will ask Members of Congress for their support of S. 3385 the Better Empowerment Now to Enhance Framework and Improve Treatments (BENEFIT) Act, recently re-introduced by Senator Roger Wicker (R-MS) and Senator Amy Klobuchar (D-MN). This legislation builds on the Food and Drug Administration (FDA)’s implementation of Patient Focused-Drug Development (PFDD) provisions from the Food and Drug Administration Safety and Innovation Act (FDASIA) and the 21st Century Cures Act by requiring that FDA include PFDD data in its benefit-risk assessment for evaluating new drugs.

“PPMD has long advocated for the incorporation of patient and caregiver perspectives in regulatory decision making and therapy development. We are now in a new era of patient engagement in drug development and we believe the BENEFIT Act will send an important signal to all stakeholders that patient experience data and PFDD data will be fully incorporated into the agency’s review process,” said Ryan Fischer, PPMD’s SVP of Community Engagement.

About the Advocacy Conference

On Sunday, March 8, advocates received extensive training and support so that they feel empowered and prepared as they attend meetings with their Members of Congress. New families are paired with seasoned advocates and meetings are scheduled throughout the day on Monday and Tuesday.

Though registration is required for PPMD’s Advocacy Conference, there is no fee to attend. To learn more, click here.

If you are unable to attend the Conference in person, you can advocate from home. Click the link below to take action and ensure that your voice, your story is included in PPMD’s messaging to lawmakers.

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