Registration for the PPMD Advocacy Conference is live! Join us March 8-10, 2020 in Washington, DC to advocate for Duchenne directly with your Members of Congress.
Because of our community’s presence in Washington for the last two decades, lawmakers better understand our clinical, research, and regulatory priorities. Next month, we hope you will join us for several powerful and critical days, including a special event on Monday, March 9 discussing federal agency involvement in Duchenne. It’s a week in which we have the opportunity to make an indelible impact and continue to change the landscape in the fight to end Duchenne. Please join us!Register >
- Sunday, March 8, 3pm-7:30pm
Advocate Training (The Dupont Circle Hotel)
- Monday, March 9, 9am-12pm
Breakfast Forum for Advocates – The Duchenne Community & Federal Agencies: Yesterday, Today, & Tomorrow
- Monday, March 9, 12pm-5pm
Advocates on Senate Side for Meetings (Capitol Hill)*
- Tuesday, March 10, 9am-5pm
Advocates on House Side for Meetings (Capitol Hill)*
* PPMD will handle scheduling all of your meetings on the Hill with your specific Members of Congress
What will we be Advocating about?
FEDERAL FUNDING FOR DUCHENNE RESEARCH & PROGRAMS
Every year, PPMD advocates head to Washington to ensure the agencies that matter most to Duchenne – the Centers for Disease Control and Prevention (CDC), the National Institutes of Health (NIH), the Food and Drug Administration (FDA), and even the Departments of Defense (DOD) – are funded at appropriate levels and include specific report language guiding these agencies when they make decisions about Duchenne programs and priorities. To date, that funding has yielded over $600 million specifically for Duchenne research and care programs. Advocates will be encouraging members of Congress to support Duchenne-specific funding and programs that impact Duchenne.
PASSING THE ‘BETTER EMPOWERMENT NOW TO ENHANCE FRAMEWORK AND IMPROVE TREATMENTS (BENEFIT) ACT’
While much progress has been made to ensure the patient voice is incorporated into drug development through our previous legislative efforts, some significant gaps remain. One such gap is the lack of any requirement in laws today that the FDA include, as part of its decision-making tool called the Benefit-Risk Framework, any patient experience or patient-focused drug development (PFDD) data. This includes Patient Reported Outcome Data, Patient Preference Data, and data from interviews, testimonials, and focus groups with patients, collectively termed Patient Experience Data.
This means that the agency’s signature tool for evaluating benefit-risk is not currently required to include data from the patient perspective that could be critical to informing the agency’s evaluation and, ultimately, their decision on whether or not to approve a product. The BENEFIT Act would close this existing gap. Advocates will be requesting members of Congress support passage of BENEFIT.
Currently, we have families from the following states attending the conference. BUT, we need more advocates from each state AND to fill in the states where we don’t have representation!