PPMD Lends Leadership to NCATS Rare Disease Research Toolkit Development

There are some days when I feel like our community is in its infancy. When I look at where we are in terms of access, therapeutic options, combinations, care implementation…

But more often than not, I am overwhelmed with gratitude for the infrastructure and resources available to our Duchenne community. The rich collaborations and strong partnerships. Federal agencies, Members of Congress, academic networks worldwide, Industry, other advocacy organizations. Data sets that we are working to synthesize, upgrade, and analyze. Animal models. Clinical networks and clinical trial networks. Game-changing legislation. And more.

But make no mistake. This wasn’t all here 30 years ago.

PPMD and our community built this framework, created this landscape, and convened these partnerships – one day at a time. And we are so grateful to those communities who went before us, shared their expertise, and helped us navigate. HIV. CF. Pediatric cancer. We are indebted to them and we are committed to ensuring that our Duchenne community pays that generosity forward. When we break new ground, we work to publish our methods so that others may benefit. When partners in other organizations reach out and ask for our guidance, we do all we can to lean in.

So, when my friend Dr. Petra Kaufman, the Director of NCATS Office of Rare Diseases Research and Office of Clinical Innovation at NCATS asked me to join another dear friend Ron Bartek, Founding President of the Friedreichs Ataxia Research Alliance (FARA) to lend leadership to the development of a global resource for advocacy organizations – I was all in.

NCATS Rare Disease Research Toolkit Launch

Over the last two years, I have had the distinct pleasure of serving as a part of a leadership team at NCATS who has worked collaboratively to create the NCATS Rare Disease Research Toolkit. Developed in partnership with patient community innovators, we convened patient community leaders with expertise in facilitating therapeutic development across the research continuum.

We asked these leaders to share their tools and best practices, to help us collect others, and to identify areas where tools have yet to be developed. We then created a ‘navigator’ for patient advocacy groups who utilize the site to assess where to start in the pipeline based on each community’s need, who to contact, how to prioritize organizational resources. And after two years of collaboration and effort – we are gathering with a broad array of partners and stakeholders at NIH – and launching.

(PHOTO: NCATS Toolkit Leadership Team, from left to right: Dr. Chris Austin  Director NCATS; Dr. Anne Pariser, NCATS; Ron Bartek, Founder CEO FARA; Petra Kaufman, MD Director Office of Rare Disease Research & Clinical Innovation, NCATS; Annie Kennedy, SVP Legislation & Public Policy, PPMD)

I have been so proud that PPMD has been able to contribute to this resource.

The rare disease community relies on each other to learn, to move forward, to make progress. When we start an organization or foundation, it is overwhelming. Thinking about therapeutic development tools feels strange when we simply want treatments and a cure. But, over time, we realize that all of us face similar challenges -- and that models and tools are needed if we are to make progress and overcome challenges.  

The NCATS Rare Disease Research Toolkit is intended to be that common thread that ties us together, providing new organizations a solid start and veterans with new resources and hindsights from valued colleagues. And most of all - the knowledge that we are a part of a powerful network of partners and are not alone.

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