October 21, 2019 / Advocacy,Community

Transitioning to a New Role – While Keeping My Arms Wrapped Around Duchenne

Over the last few weeks, I have spent many hours reflecting on just how privileged I have been to share this journey with so many of you, and have spent hours recounting highlights among these last five years at PPMD – and 30 years since I first began my journey in our community.

When I joined the team at PPMD 5 years ago, I knew I had been given an opportunity to join a strong team of policy experts; people who I had long collaborated with, long admired, and who shared my deep personal passion for our mission. Working alongside this team has been a tremendous honor – and I have been so proud to work for PPMD. I vividly recall when I first met Pat in 1999. I am even more in awe of Pat, this community, and our organization today — as I was then.

So, here’s what my future now holds…

In the coming weeks, I will be transitioning to a leadership role within the EveryLife Foundation for Rare Diseases. EveryLife is a DC-based organization dedicated to advancing the development of treatment and diagnostic opportunities for rare disease patients through science-driven public policy. EveryLife is a longtime advocacy partner and PPMD’s office-mate at the Rare Hub in D.C.!

As you know, we are in an era of unprecedented opportunity in rare disease. There exist great challenges for clinical trial innovation, infrastructure and recruitment, trial design, approval pathways, and reimbursement justification. Therapeutic successes yield urgent policy issues such as nationwide newborn screening, favorable access environment frameworks for novel therapies, federal policies that promote autonomy and independence for new generations living decades into adulthood — and more. The opportunities are endless. The priorities are extensive. The moment is now.

Staying in the Community

While our Duchenne/Becker community is deeply personal to me, I am eager to apply a systems approach to helping to convening partners around rare disease priorities who can serve as force multipliers to drive paradigm shifts.

Most of all, be assured that this move signifies a reaffirmation of all that I have believed in, fought for, and built over the last 30 years. The work that is done in one collaborative disease community instantly benefits all others – and you must know that it is wired into my DNA to always fight for each of you.

Being passionate about both PPMD and the EveryLife Foundation are not mutually exclusive; both organizations are fighting day and night for you — and your talents and voice are needed anywhere and everywhere you have time to offer. The EveryLife Foundation and PPMD are close partners and it is the mission of the EveryLife Foundation to support rare disease communities and seek ways to promote their priorities.  In my role I will continue to formally work closely with Ryan, Pat, Abby and the team in many ways. But there are aspects of my work at PPMD that are a part of my heart and my life’s work, and I am honored and grateful to have the opportunity to remain formally engaged in them. Thus, I will continue to provide leadership support to PPMD’s Adult Advisory Committee (PAAC) including Teen/Adult elements of the Annual Conference in June, and will provide support and guidance to the Duchenne Newborn Screening Pilot efforts.

An Amazing Journey

In just five years at PPMD, highlights of efforts I have been privileged to help lead have included:

  • MD-CARE Act Amendments 2014
  • Patient Focused Impact Assessment Act (PFIA)
  • Federal MD Action Plan
  • CDC Duchenne Becker Care Considerations update (published in Lancet 2018)
  • Transition Checklist (Pediatric supplement January 2019)
  • The National Duchenne Newborn Screening Infrastructure effort
  • The PPMD Adult Advisory Committee (PAAC)
  • PPMD Transitions Conferences
  • PDUFA VI engagements
  • 21st Century Cures Act engagement
  • National Transitions Policy efforts
  • Duchenne Drug Development Roundtable (DDDR)
  • PPMD Compass Workshop (Externally-led PFDD meeting)
  • Annual PPMD Advocacy Conferences
  • PPMD Access Forum
  • Launch of Duchenne Newborn Screening Pilot in New York State
  • Refinement of ICD-10 code for DBMD
  • CDMRP coalition efforts
  • Valuation initiatives
  • HEOR engagements including first ICER reviews for Duchenne products
  • Community’s first four Advisory Committee meetings for Duchenne (3 products, 1 protocol amendment)
  • Congressional Briefings
  • Congressional Hearings
  • Access Resource Center
  • Payer engagement
  • Robust coalition partnerships
  • Industry partnerships
  • Federal partnerships
  • And the list goes on!

Finding the Right Balance

I am occasionally asked to speak on Career development panels. On one such panel the theme was ‘Careers in Public Service’ and I was asked how I achieved ‘work/life balance’? My answer was simple, “I don’t”. It’s not about balancing demands of work and personal life for me, those lines crossed, blurred, and merged so long ago… it is really about achieving harmony. Harmony between my son and immediate family’s needs – and my vocational priorities (what one of my dear friends recently referred to as my ‘ministry’).

This ‘harmony’ has been possible because somewhere along the line, you each became a part of my personal life and my family. My son’s world is enhanced, enriched, and blessed daily by you. But over the past year, this sense of  ‘harmony’ has waned.

This is what brings me to my next chapter.

I have felt a crushing sense of responsibility to you. To do more. To ensure that we are making the maximum impact possible. And within my role at PPMD, this frequently requires time away from my son. Time – that I have learned from each of you – is precious and fragile.

As many of you have heard me say before, I believe that a career of service is about taking life personally. Letting the lives that touch yours, touch you. Thank you for touching my life.

As I transition into this next season, my arms will continue to remain wrapped around Duchenne.

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