Spring Forward: Bringing the MD-CARE Act to Life

Despite great odds in a political climate that was stacked against us – we achieved what was considered to have been impossible. After months of outreach to congressional champions and visits to Capitol Hill, the MD-CARE Act Amendments were passed and signed into law in the end of 2014.

Now what? Well… now the real work of implementation actually begins.

But the truth is that our community has so successfully built champions and partners across the federal agencies over the last two decades, that the implementation of the MD-CARE Amendments started long before the bill ever passed. Our federal partners ‘anticipated’ its passage and began to implement aspects of what was being called for in the new law, even before Congress was telling them they had to.

One of the most important elements of the MD-CARE Act was the authorization of the establishment of the Muscular Dystrophy Coordinating Committee (MDCC) to coordinate activities across NIH and with other Federal health programs and activities relevant to the various forms of muscular dystrophy. The MD-CARE Act directed the Committee to develop a plan for conducting and supporting research and education on muscular dystrophy through the national research institutes, and to submit this plan to Congress. This ‘plan’ became known as the MD Action Plan and over the years, activities within the Plan were achieved, others became less relevant, and other efforts needs to be added to the plan due to the evolution of our community and progress in the field. So long before the MD-CARE Amendments were passed, the MDCC came together (under the leadership of Dr. Alan Gutmacher, the Chair of the MDCC and the Director of NICHD and Dr. John Porter of NINDS) and began a year-long effort to update the federal MD Action Plan.

The new MD Action Plan draft is currently under review by the Muscular Dystrophy community and we encourage you to participate – in whatever capacity you are able – and engage in the process. This is intended to be a public process and every effort is made to allow for engagement and input. Many weeks ago, John Porter did a terrific blog alerting our community to the start of the MD Action Plan comment period, and we would now like to alert you to the fact that PPMD has submitted comments on behalf of our community. Our PPMD Adult Advisory Committee (PAAC) also submitted comments on behalf of the PAAC.

Next Steps for the MD Action Plan

On Tuesday, March 17, the MDCC will convene to discuss the MD Action Plan draft and comments will be made on behalf of PPMD by both me and Brian Denger (Brian serves as PPMD’s representative on the MDCC). This is a public meeting and all are encouraged to attend. In addition, the meeting is being streamed live and we encourage you to watch the meeting if you are interested.

The MDCC meeting will be available by video cast at the NIH site on March 17th. All of the video cast meetings on any one day are listed at this site. To access the meeting, simply go to the site on the day of the meeting. The meeting starts at 8:30 EDT and the agenda can be found here

Following the meeting, we will provide a meeting summary, as well as a summary of the comments that PPMD submitted on the MD Action Plan. To get a sense of the heart and purpose of the effort, I wanted to share an excerpt from the 2015 MD Action Plan draft:

“As the prospects for efficacious therapies improve for all of the muscular dystrophies, it is clear that the opportunities and challenges that the field faces are only increasing—issues such as newborn screening, pediatric patients living into adulthood, and reimbursement for very expensive drugs were not on the horizon ten years ago. This is reflected in the 2015 MDCC Action Plan in the form of recommendations for: deeper understanding of disease mechanisms and more careful vetting of therapeutic targets; better aggregation of mutation/polymorphism, patient sample, and genotype-phenotype data to improve diagnostics, to identify people with muscular dystrophy earlier and with more reliability, and to qualify biomarkers; improvement of the efficiency of preclinical and clinical vetting of candidate therapeutics in order to avoid failures in late stages of clinical trials that can be catastrophic to the field; and increasing the efforts and urgency to address the quality of life, education, and employment of people living with muscular dystrophies.

Taken together, the muscular dystrophy landscape is considerably more complex, and adequate solutions to the myriad of problems will require a new, considerably higher level of cooperation among stakeholders in the field, and collaborations with new partners. The 2015 MDCC Action Plan for the Muscular Dystrophies provides a roadmap for those collaborations.”

– MD Action Plan 2015 draft, page 6-7

So beautifully described. The 2015 MDCC Action Plan for the Muscular Dystrophies was developed by a broad range of stakeholders within our muscular dystrophy community to provide a roadmap for collaboration. Next week, the MDCC will be taking the first formal step to bring the MD-CARE Amendments to life since they were signed into law in late 2014 – and our community will officially Spring forward!

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