Your Action Plan for the Muscular Dystrophies

During my time at the National Institutes of Health (NIH) one of my key roles was serving as Executive Secretary for the Muscular Dystrophy Coordinating Committee (the MDCC). To help coordinate activities across the NIH and with activities of other Federal health agencies, the MDCC has undertaken two major planning efforts. Before leaving NIH at the start of this year, I organized the efforts leading to the 2015 MDCC Action Plan for the Muscular Dystrophies.

The 2015 Action Plan is now available for public comment. PPMD encourages all members of the Duchenne community to provide comments, in particular to ensure that the patient and family voice is heard in the Action Plan.


What is the MDCC? 


The MDCC was created by you and for you—it was a major provision of the MD-CARE Act in 2001 and has been reauthorized in each renewal of the MD-CARE Act. The MDCC is chaired by one of the Directors of the NIH institutes with responsibility for muscular dystrophy, currently Dr. Alan Guttmacher (Director, Eunice Kennedy Shriver National Institute of Child Health and Human Development). MDCC members include representatives from four different institutes at the NIH, the Centers for Disease Control and Prevention (CDC), Food and Drug Administration (FDA), Health Resources Services Administration, Departments of Education and Defense, and representatives from patient advocacy (currently Cure CMD, the FSH Society, MDA, PPMD, and an individual with myotonic dystrophy).

With its latest reauthorization, the MDCC will be adding representatives from the Social Security Administration, the Administration on Community Living, and an additional public member. We worked with our congressional champions to have these agencies added to the MDCC, reflecting the need for increased federal resource emphasis around supports for 'daily life' with muscular dystrophy. The MDCC meets twice annually; all meetings are open to the public, with the next meeting on March 17, 2015, in Bethesda, Maryland.


What is the history of the MDCCs planning and research coordination efforts?


In 2005, the MDCC completed an intensive planning process that led to 72 specific objectives to guide the individual missions of MDCC member agencies and organizations and these have served as a central focus for coordination of efforts. At the NIH, we used the 2005 Action Plan in a variety of ways to foster research that filled knowledge gaps, to launch targeted therapy development initiatives, and to partner with other agencies and patient advocacy groups, who also used the Action Plan to help guide their efforts. The 2005 Action Plan made a difference in how Federal agencies and patient advocacy groups made decisions.


In the decade since the 2005 MDCC Action Plan, there has been tremendous progress. Yet, as we move closer to better care and novel treatments for the muscular dystrophies, the questions that we are faced with have become harder.  In July 2014, while still at the NIH, I helped convene a working group of 34 experts to evaluate progress on the 2005 Action Plan and develop new objectives to serve as a blueprint for the muscular dystrophy community.  

The 2015 Action Plan is comprehensive—addressing issues from better understanding of disease mechanisms to quality of life, education, and employment. Its purpose is to guide all stakeholders in academics, companies, governments, patient advocacy groups, and patients and their families as to the priority needs to improve the management of and treatment for all types of muscular dystrophy.


Why is it important for YOU to comment on the Action Plan? 


The 2015 Action Plan is a plan for the entire community. MDCC members (including Brian Denger from PPMD) and an expert panel (including Annie Kennedy, now PPMD’s Senior VP for Legislation and Public Policy) have put considerable effort into identifying objectives for the future attention of the muscular dystrophy community.

What is needed now is your input. All stakeholders have shared responsibility for achieving the priorities described in the 2015 Action Plan, and, by extension, for improving the lives of people living with muscular dystrophy.

Your voice, the experts on living with muscular dystrophy, is now needed. Please let us know if you have any questions or need any assistance in accessing and commenting on the Action Plan during the public comment period.

Thanks for your help.

Review & Comment on the 2015 Action Plan

(Deadline for comments is March 4,2015) 

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Comment by John D. Porter, PhD on February 27, 2015 at 7:09am

Thanks Brid.  Again, I'd strongly encourage the community to go to the NIH site and comment on the Action Plan.  It is actually open for public comments until March 10, so you have plenty of time. 

Comment by Brid Murray on February 18, 2015 at 10:50am

Thank you all.  This is wonderful. 

Comment by Shelly on February 13, 2015 at 9:52pm
Thanks John for the information. Any possible help to end duchenne will be my pleasure....
Comment by Deb Robins on February 13, 2015 at 8:20pm

Ok, will promote on our networks.Thanks for your responsiveness John - on all levels.

Comment by John D. Porter, PhD on February 13, 2015 at 6:48am

Deb, anyone can comment on the Action Plan.  The Plan is a community plan and the community has to be international for us to improve care and develop treatments for Duchenne.  In particular, you may want to look closely at the Living with Muscular Dystrophy section.  Thanks for your help! 

Comment by Deb Robins on February 12, 2015 at 8:54pm

John, are non-US citizens' comments accepted?

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