21st Century Cures Passes House of Representatives!

21 Century Cures. First a bold concept led by two visionary congressionary leaders on April 30th, 2014.

Then innovative ideas poured in to Washington from the world’s best and brightest developers and innovators.

Then lots of Hearings, and roundtables, and Hill meetings, and constituent visits.

Then ‘provisions’ were structured which pulled together all of these bold ideas. Some of these provisions made it into the final bill; some didn’t. Most were probably worthy.

And – voila - H.R. 6.!

Then a Committee vote.

Then more constituent visits. Then more members of the House became interested and amendments started getting tacked on to the bill; some ok — some not ok at all.

Again — our army got active.

And — today — a full House vote and 21 Century Cures has passed the House of Representatives!

So what’s does this all actually mean? After all of this — what changes?

The 21st Century Cures Act brought together stakeholders from all facets of the scientific, clinical, biomedical, and regulatory space, as well as the patient community to work towards a more streamlined drug development process.

Today’s yield includes provisions that will:

  • Increase funding for the National Institutes of Health (NIH) & the Food & Drug Administration (FDA)
  • Incorporate the patient perspective into the drug development and regulatory review process (patient focused drug development)
  • Clarify the use of platform technologies - Utilizing early data from trials to shorten follow on trials (such as in exon skipping)
  • Provide new incentives for the development of drugs for rare diseases (OPEN Act)
  • Invest in 21st century science and next generation investigators

The contribution of THIS community

But let’s talk about how this REALLY happened.

It happened because armies of powerful patient communities joined our voices together and ensured that the members of Congress who were listening — really listening – heard poignant and succinct messages.

We attended the forums they convened in D.C., we testified at their district roundtables, we showed up in force their offices in Washington, we visited their offices back home, we called, we emailed, we convened two briefings on Capitol Hill, we got to know them — and we rallied. Big time.

And what did they hear from us? Here are some of the highlights:

"14 years ago, I testified before Senator Spector and the Senate HELP Subcommittee for the passage of the MD-CARE Act. That law changed the trajectory of Duchenne. On that day, I dreamed of serving our country as Navy Pilot. While the progression of Duchenne prevented me from enlisting, I still pursued my dream of serving my country through my academic and career pursuits. Today, I testify with new dreams – and an equal hope of achieving those dreams, thanks to my faith, my support system, the therapeutic advances I see on the horizon, and the efforts being led by groups like PPMD to ensure that all stakeholders – from Industry to the FDA – have the tools they need to get therapies to me as fast as possible. We need the FDA remain engaged, allow for maximum transparency in their processes, and leave no stone unturned.

After all, your next experimental therapy – could lead to my tomorrow.”

- Benjamin Cumbo IV – Testifying at PPMD's Congressional Briefing, February 24, 2015, Capitol Hil

“I believe that patient engagement is the 'blockbuster drug of the century' – helping patients live longer, richer, more meaningful lives.”

- Pat Furlong, PPMD Founder & President – Testifying at PPMD’s Congressional Briefing December 9, 2014, Capitol Hill


"The Duchenne community has traveled a great distance over the past 15 years, thanks in significant part to the leadership of this Committee. But for too many families, my own included, this journey has not been fast enough, and we think every day about those whom we have lost.

We need treatments and therapies, right now, so we can end Duchenne and address the thousands of other rare diseases in need of treatments and cures."

- Pat Furlong, PPMD Founder & President – Testifying to Congress at the 21st Century Cures hearing on Patient Perspectives.

"Each loss of function for our boys, including my son, is a "little death"... it is loss of function that he will not get back. The ripple affect from this is devastating. The negative impact on the family and the burden on the caregiver becomes increasingly significant and grows over time….

Working through the current regulatory issues and challenges that plaque our community, would be advantageous for all boys with Duchenne, including my brave little guy. Now is the time for change. Now is the time to help these boys and give them the bright future that they deserve!"

- Colleen Labbadia, parent of Brendan and PPMD FACES Coordinator – Speaking at the Congressman Bilrakis Florida regional roundtable

"My family and I have watched Tim lose ambulation and now the ability to feed himself as he loses the use of his arms. Although a wheelchair can help with ambulation, there is no replacement for arm movement. Today boys like Tim with Duchenne are living longer than ever before, on average into their late 20s. But that is still far too short and simply unacceptable.

Our community is encouraged by the number of potential therapies that are in varying stages of development and evaluation, including three in critical late-stage clinical trials. We are hopeful that by the end of this year, the first-ever Duchenne drug applications will be submitted to the Food and Drug Administration, and we want the FDA to be ready to move as expeditiously as possible when this milestone occurs."

- Ellen Wagner, parent of Tim and PPMD Board Member - Speaking at Congressmen Davis and Shimkus 21st Century Cures regional roundtable in Illinois 

What happens next?

The Senate is currently working on creating a companion version of this legislation and – in anticipation of this effort our Duchenne community already began meeting with Senators in the D.C. Offices during the June Connect Conference and Hill Day. We have been reaching out throughout the summer.

While today signifies a tremendous step forward for our nation and our community, we must work together to ensure that these provisions are implemented as envisioned — and 'move the needle' for those living with Duchenne today.

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Comment by Shelly on July 10, 2015 at 9:10pm
Great news.!!!!!....highly appreciated step....thanks to Benjamin, .......n thanks to all who are with PPMD.
Comment by Robin Taylor on July 10, 2015 at 12:54pm

Great news on HR.6!! Did they keep the Rep. Brat (VA) amendment in?  I hope not.

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