International Rare Disease Day 2011
Monday, February 28, 2011, marks the fourth annual International Rare Disease Day, in which hundreds of patient organizations from more than 40 countries conduct awareness-raising activities around the slogan “Rare but Equal.” The worldwide event is an effort to draw attention to rare diseases and the millions of people who are affected by them — including all those covered by the MDA umbrella.
In Washington D.C., Francis Collins, director of the National Institutes of Health, will be speaking about NIH’s commitment to rare disease research. As a follow-up, MDA and other patient advocacy groups are planning to hold an information session for members of Congress and their staffs about the benefits of establishing a rare disease caucus in Congress.
Families affected by rare diseases are invited to learn more about these conditions, advocate for greater awareness and funding for treatments and cures, and to share their stories. Please visit the Rare Disease Day 2011 website for more information.
GINA now fully in effect
As of January 2011, the Genetic Information Nondiscrimination Act is now fully in effect. This law, signed by President George Bush in 2008 and phased in over several years, prohibits discrimination in health coverage and employment based on genetic information. Health insurers cannot require genetic testing and may not consider the results of genetic testing when determining eligibility. Businesses with more than 15 employees also are prevented from making employment decisions based on genetic testing of individuals or their family members. For more, go to http://www.genome.gov/Pages/PolicyEthics/GeneticDiscrimination/GINAInfoDoc.pdf
Creating Hope Act of 2010
Congress is considering a new law, the Creating Hope Act of 2010, which would make it more profitable for companies to develop drugs targeting rare diseases in children. A January 19 editorial in Science Translational Medicine praised the proposed law, noting that drug development for pediatric rare diseases has been hampered by lack of financial incentives, and that this law will increase potential profits as well as hasten the regulatory process for companies who develop drugs to treat these conditions.
Formation of NCATS at NIH
In response to the slow pace of new drug development by the pharmaceutical industry, the National Institutes of Health (NIH) plans to establish a National Center for Advancing Translational Sciences (NCATS). This new agency will be assembled primarily from NIH agencies and programs already in existence. It is intended to facilitate research already being supported by the NIH that moves drugs from the laboratory to the clinic (“translational research”), and to demonstrate the value and potential of developing these drugs and therapies.
Drug development partner for hereditary IBM
RRD International in Rockville, Md., has been selected as a drug development partner for the NIH Therapeutics for Rare and Neglected Diseases program. One of the diseases RRD will be focusing on is hereditary inclusion-body myopathy (HIBM). MDA covers both the hereditary and “sporadic” (not inherited) forms of inclusion-body myositis.
Medicare competitive bidding
Comments and complaints about the Medicare competitive bidding program, which went into effect in January in nine regions nationwide, are currently being collected. The controversial program requires providers of durable medical equipment and other supplies to submit to competitive bidding in order to receive reimbursement from Medicare.
Patients, clinicians and providers have reported a wide range of problems with the program, including delays in obtaining medically required equipment and services; difficulty locating approved providers, especially in rural areas; longer hospital stays; reduced quality of equipment; fewer choices in equipment or providers; and confusing or incorrect information being provided by Medicare.
The National Institute of Arthritis and Musculoskeletal and Skin (NIAMS), part of the National Institutes of Health, has announced new resources for families affected by muscle disease. Through its websites, printed materials, multimedia presentations, and other resources, NIAMS provides a great deal of information to both the research community and the general public, including results of recent research studies, a list of current clinical trials and other health-related news. To find out more, please visit the NIAMS website, call 877-226-4267, or send an e-mail to NIAMSinfo@mail.nih.gov.
Visit MDA's Advocacy pages for more information about current advocacy efforts and how you can become involved.