The Duchenne Community’s Voice Was Essential Every Step of the Way & Contains PPMD’s Provision to Strengthen Patient Focused Drug Development Efforts

To everyone in our PPMD community who called, reached out, emailed, and met with your elected officials – WE DID IT!

Today, we are thrilled that Congress has passed the 21st Century Cures (21CC) bill – which includes almost every provision that our Duchenne community championed and led throughout this process.

The importance of this bill: 

The 21CC bill contains incentives and innovations that will accelerate therapy development and are designed to translate into therapeutic opportunities for THIS generation living with Duchenne. It will help bring drugs and devices to market more quickly and at less cost by making needed reforms to the FDA, including:

• Expedited review for breakthrough devices
• Increased patient involvement in the drug approval process
• A streamlined review process for combination products that are both a drug and device
• Freedom from red tape for innovative software

Our community’s impact:

Beginning with Pat Furlong’s testimony at one of Representative Upton’s first Congressional Hearings on the momentum behind the 21CC effort in July of 2014, PPMD’s powerful and visionary community played a critical role in shaping and passing the 21CC package over the last three years.

From the Duchenne community leaders who participated in Congressional District roundtables in Florida (Colleen Labbadia) and Illinois (Ellen Wagner), to PPMD’s hosting of three Congressional Briefings focused on 21CC– and PPMD leadership testifying in five others led by our community partners, and hundreds of meetings with Hill staff and member of Congress.

Our community’s voice was heard.

But more… When our efforts to incorporate specific language into the Patient Focused Drug Development section of House version of the 21CC bill only served to ‘strengthen and influence’ that section, rather than yield our exact language and provision – we opted for another approach in the Senate.

In June of 2016, our longtime Duchenne community champions Senator Roger Wicker (R-MS) and Senator Amy Klobuchar (D-MN) worked with us to introduce the Patient Focused Impact Assessment Act (S.1597). Introduced during PPMD's Connect Conference, we took 300 members of our community to the Hill and engaged with our Senate champions to ensure they understood the concerns of our Duchenne community.

We then followed with another year and a half of outreach from our national community… and we have achieved great success!

President Obama has said he’ll sign the bill as soon as it reaches his desk – and then 21^st Century Cures will be the law of the land and PPMD will begin engaging on implementation efforts.

Highlights of the bill include:

• Provide $4.8 billion to National Institutes of Health
• Provide $500 million to the Food and Drug Administration
• Patient Focused Impact Assessment Act (PFIA) provision to increase transparency in application of PFDD tools within product reviews
• Expanded Access policy disclosures
• Advancing Targeted Therapies Act
• Data Sharing Incentives
• PRV extension (incentive for industry to work within our Duchenne space)
• A strong section on Patient Focused Drug Development
• Incentives for pediatric rare disease research – both U.S. and global

Thank you!

Today’s passage of the 21CC bill is a bold reminder of just how far we’ve come together – and that there is so much momentum in this community!

We must continue to keep Duchenne on the front lines of agency priorities. Please consider joining us February 12-14 in Washington DC for PPMD's 2017 Advocacy Conference. Your presence in DC is a critical part of our Duchenne community’s strength and impact going forward! 

Related links

• PPMD 21CC Endorsement Letter to Energy & Commerce Committee
• PPMD 21CC Endorsement Letter to HELP Committee