Since 2001, the MD CARE Act has had a tremendously positive impact on MD research — but its reauthorization this year will require active outreach
Note: The MD CARE Act is a critical part of the historic effort to find cures for muscular dystrophies. Watch for articles in the coming weeks that delve further into the remarkable progress attributable to this important legislation.
Centers of research excellence. Clinical trials. New insights into disease causation and progression. Optimal patient care guidelines. A robust pipeline of promising research.
These are the tangible results of the MD CARE Act (H.R. 594/S. 315) — and these are what advocates are seeking to preserve and expand in MDA's all-out push that began Feb. 8, 2013, to convince Congress to reauthorize this critical legislation for muscular dystrophy.
(To read the Senate and House bill text, visit thomas.loc.gov and type in the bill number in the search box.)
Since the original passage of the MD CARE Act in 2001, “there have been 67 clinical trials of drugs or therapies for the muscular dystrophies, with 37 clinical trials currently under way,” says Annie Kennedy, MDA senior vice president for advocacy.
“We’ve ‘moved the needle’ in muscular dystrophy care and research. We must keep the momentum going strong.”
Although a number of congressional champions strongly support reauthorization of the Act, passage is far from guaranteed in the current political climate. Nonetheless, MDA — in concert with Parent Project Muscular Dystrophy and the Foundation to Eradicate Duchenne — has helped to shape the 2013 reauthorization bill so that it expands on a decade of progress and continues the push toward the ultimate goal: improved health outcomes and effective, available treatments for people with muscular dystrophy.
At the turn of the new millennium, total governmental funding for muscular dystrophy research was $17 million, there were no new drugs in development, there were no care guidelines and no patient registries, and the average life span for someone with Duchenne muscular dystrophy (DMD) was the late teens.
In 2001, after extensive lobbying by the muscular dystrophy community (including congressional testimony by former MDA National Chairman Jerry Lewis and former National Goodwill Ambassador Ben Cumbo), Congress passed the Muscular Dystrophy Community Assistance, Research and Education (MD CARE) Act.
The Act provided essential government funding for the infrastructure needed to accelerate research and bring drugs to market for all nine forms of muscular dystrophy.
The ensuing decade saw an unprecedented national focus on coordinating and intensifying MD research:
Today, total governmental funding for muscular dystrophy research has swelled to $75 million annually. Nine new drugs are in trial for DMD and almost a dozen others in preclinical development. Care guidelines for DMD have been disseminated worldwide, and guidelines are in development for facioscapulohumeral muscular dystrophy (FSHD), myotonic muscular dystrophy (MMD), limb-girdle muscular dystrophy (LGMD) and congenital muscular dystrophy (CMD).
And in just over a decade, life span in DMD had increased by 10 years.
MD CARE reauthorization bill expands on progress
The 2013 reauthorization bill was introduced in the U.S. Senate (S. 315) on Feb. 13, 2013 by Senators Amy Klobuchar (D-Minn.), Roger Wicker (R-Miss.), Bernie Sanders (I-Vt.), Susan Collins (R-Maine), Robert Menendez (D-N.J.), Johnny Isakson (R-Ga.), Barbara Mikulski (D-Md.), Patrick Leahy (D-Vt.), Frank Lautenberg (D-N.J.) and Bill Nelson (D-Fla.).
The bill was introduced in the U.S. House of Representatives (H.R. 594) on Feb. 8, 2013, by co-sponsors Eliot Engel (D-N.Y.) and Michael Burgess (R-Texas), who also spearheaded the the reauthorization in 2008.
It seeks to expand the scope of the bill in several ways, including:
If passed, the bill also would expand the mission of the MDCC (the public-private coordinating committee) to include:
MDA is at the forefront of the effort to convince Congress to reauthorize and allocate funding for the MD CARE Act. Advocacy by the muscular dystrophy community is key to this effort.
“The MD CARE Act has been a ‘game changer’ for our MD community,” Kennedy says. “We must ensure that our federal research funding remains robust, that we optimize our clinical trial infrastructure, and that we are able to deliver effective therapies to our loved ones who await them. We need the active outreach of every member of the MDA community — every clinician, researcher, family, sponsor, volunteer, advocate — to ensure legislative success.
“Our community has overcome amazing challenges before, and we will do it again in 2013. Please ‘Take 5’ and contact your elected officials to urge their support of the MD CARE Act of 2013 and keep our lifesaving research and care momentum strong.”
To learn more about MDA's efforts to reauthorize the MD CARE Act of 2013, see: