Advocacy is needed to pass amendments to this landmark bill that will expand muscular dystrophy research and health care efforts
Times have changed dramatically since 2001, thanks to the passage that year of the Muscular Dystrophy Community Assistance, Research and Education (MD CARE) Act — a bill designed to advance research and health care for all forms of muscular dystrophy. Among the changes:
While these achievements are cause for celebration, much remains to be done, and this is reflected in the expanded scope of the MD CARE Act, which is up for reauthorization this year.
“We’ve been thrilled with the progress that has been made through the MD CARE Act,” says Annie Kennedy, MDA senior vice president for advocacy. “The bill has been instrumental in helping us to move the needle in muscular dystrophy care and research, and we must see this momentum continue. The proposed additions in the MD CARE Act of 2013 reflect the positive changes resulting from the original legislation.”
All year, MDA advocates have been urging their legislators to reauthorize the MD CARE Act. Join the fight! Here’s a look at some of the valuable additions being proposed in the 2013 bill.
One proposed expansion to the MD CARE Act emphasizes the need to provide transition services to a growing community of young adults with various forms of muscular dystrophy.
“Transition” refers to the passage from childhood to adulthood — a difficult journey for any young person but especially complicated for those with muscle diseases. Transition services address barriers this group faces in education, housing, health care, personal care assistance and more.
“Thanks to research breakthroughs and improved clinical care interventions, young people with pediatric-onset muscular dystrophies are living into adulthood,” notes Kennedy. “The MD CARE Act reauthorization bill calls for the Muscular Dystrophy Coordinating Committee [MDCC] to create an action plan that reflects this urgent need for transition services and independent living resources that provide support for the young adult and adult populations in our community.
“To facilitate this effort, MDCC membership is strongly encouraged to include representatives of the Social Security Administration and the U.S. Department of Health and Human Services’ newly formed Administration for Community Living.”
The MDCC is a public-private committee created by the MD CARE Act to expand, strengthen and coordinate muscular dystrophy research and care efforts. Under the direction of the U.S. Secretary of Health and Human Services, representatives from patient advocacy organizations (including MDA), as well as from various federal agencies identify gaps in existing research and care programs, and look for opportunities for collaboration among the committee members.
Research into cardiac and respiratory issues
If passed, the new MD CARE Act would call on the National Institutes of Health (NIH) to expand its muscular dystrophy research focus to include cardiac and pulmonary function, which is particularly important for adults with MD. This research would be conducted at the Wellstone Muscular Dystrophy Cooperative Research Centers — the “Centers of Excellence” that are funded by the bill.
“Even though we’ve been able to increase life expectancies, it is still critically important to place emphasis on therapy development that addresses the fatal effects of these diseases related to cardiac and pulmonary function, which span many forms of muscular dystrophy,” says Kennedy.
The amendment also calls for the MDCC to develop a plan to accelerate government approval of emerging therapies and personalized medicines with potential to treat patients with muscular dystrophy.
Clinical care guidelines
Among the proposed amendments is a requirement that the MDCC place new emphasis on studying and developing acute care guidelines for all muscular dystrophies.
“This is a priority. Right now for DMD, there is simply a paragraph for emergency room care,” notes Kennedy. “For each of the muscular dystrophies, we need a consensus document written in collaboration with professional organizations that will give it credibility in acute care settings as credentialed literature.”
The bill specifically calls for updating and disseminating the existing DMD/BMD care guidelines for pediatric patients, as well as developing and widely disseminating care considerations for adults with the disease. The latter provision is critical to enhancing the quality of care for adults with DMD/BMD.
For each form of muscular dystrophy, “our goal is to provide family-friendly care guidelines on our MDA website and as a smartphone app that could link to the person’s medical records,” says Kennedy. (Care guidelines for DMD and CMD already are available on the MDA site; visit mda.org/publications/books-booklets/daily-living.)
The Centers for Disease Control and Prevention already is conducting a surveillance project (collecting a database of patient information) for people with muscular dystrophy.
The amendment requires that the national surveillance program capture data from different racial and ethnic populations and make this data available to public and private researchers. The amendment also strengthens engagement and collaboration between the surveillance program and the Wellstone Muscular Dystrophy Cooperative Research Centers.
To strengthen relationships across agencies and to help create a more robust clinical environment and expedite the MDCC’s work, the committee will be required to meet at least twice each year, where now they meet annually.
Agencies represented in the MDCC include: CDC; U.S. Food and Drug Administration; U.S. Department of Defense; U.S Department of Education; and several institutes under the purview of the National Institutes of Health — National Institute on Disability and Rehabilitation Research (NIDRR); National Institute for Neurological Disorders and Stroke (NINDS); National Institute of Arthritis and Musculoskeletal and Skin Diseases (NIAMS); the Eunice Kennedy Shriver National Institute of Child Health and Human Development (NICHD); National Heart, Lung and Blood Institute (NHLBI); and the Office of Rare Diseases Research (ORDR).
In addition, a number of patient advocacy organizations are represented, including Cure CMD, the FSH Society, the Myotonic Dystrophy Foundation and Parent Project Muscular Dystrophy. Valerie Cwik, MDA executive vice president and chief medical and scientific officer, represents MDA on the committee.
For these valuable amendments to become reality, Congress must first pass the reauthorization bill.
Do your congressional representatives understand the importance to you and your family of the MD CARE Reauthorization Act of 2013? MDA provides two ways to urge your congressional representatives to support the MD CARE Act of 2013 (H.R. 594/S. 315):
“Putting a face on our community and the real-life impact of these diseases through personal contact with elected officials will go a long way toward ensuring their support for this critical legislation,” Kennedy says.
Nancy West is a freelance writer based in Fort Washington, Pa.
The Action Plan for the Muscular Dystrophies was developed for the Muscular Dystrophy Coordinating Committee (MDCC) in 2005 with the goal of identifying and addressing the needs of the entire muscular dystrophy community. Over the next year, the plan — which currently contains 72 specific research objectives — will be evaluated and updated, and the National Institutes of Health wants your input.
Public comments on current and future action plan objectives will be accepted until Dec. 20, 2013. To comment, go to Request for Information (RFI) on the Action Plan for the Muscular Dystrophies; the page includes a link to the current 100-page action plan. Not online? Write John D. Porter, Ph.D.; NINDS; 6001 Executive Blvd; NINDS/NSC 2114; Bethesda, MD, 20892; or call (301) 496-5739.
“This is a very important activity for the MDCC,” said Porter, who is executive secretary of the committee. “It’s vital that we have input from all stakeholders to help us shape the new plan.”