Most of you know I am an advocate of NORD (National Organization for Rare Disorders). They have done a lot of good representing those of us living with rare disorders including Kennedy’s Disease. This year is the 30th anniversary of the Orphan Drug Act. Below is a press release the KDA received today.
NORD Press Release
Launching a Year-Long Celebration: 30th Anniversary of the Orphan Drug Act and NORD
Washington DC, Jan. 4, 2013----Thirty years ago today, President Ronald Reagan signed the
Orphan Drug Act into law to encourage the development of treatments for the millions of Americans with rare diseases.
The new law represented a triumph of patient advocates working with government partners, the media, and other supporters to address a critically important unmet need.
To get the law passed, an ad hoc coalition of leaders of rare disease patient organizations gave themselves a name -- the National Organization for Rare Disorders (NORD) -- and taught themselves how to be advocates for the desperate patients and families they represented.
Actor Jack Klugman, whose brother was dying of a rare disease, lent his support with an episode of his popular TV show, Quincy, M.E., dedicated to showing the need for treatments for rare disease patients. Klugman later testified before a Congressional subcommittee and, when the bill became stalled in Congress, aired another Quincy episode that helped get the bill passed.
Exactly four months after President Reagan signed the bill into law, on May 4, 1983, the coalition of patient advocates formally established NORD as a nonprofit organization to provide advocacy, education, research and patient/family services for all Americans affected by rare diseases.
"It was an amazing accomplishment to get this desperately needed law enacted at that time," says NORD President and CEO Peter L. Saltonstall. "Today and throughout 2013, we will be saluting all the pioneers who helped make it happen, celebrating progress to date, and working with our partners to find ways to accelerate the pace of progress."
Saltonstall added that the recent death of Jack Klugman (on Dec. 24th) added poignancy to the occasion.
NORD's president for its first 25 years, Abbey S. Meyers, is widely considered the primary patient advocate for the
Orphan Drug Act. She retired in 2008 and Saltonstall joined the organization at that time.
Government partners at the National Institutes of Health (NIH) and Food and Drug Administration (FDA) have provided major support and encouragement to the rare disease community through their implementation of the law.
On Monday, the FDA Office of Orphan Products Development will host an event for FDA staff and guests to observe the anniversary of the law. FDA Commissioner Margaret Hamburg, MD, will speak, as will Saltonstall, with a video message from Meyers. Special guests will include individuals and representatives of organizations being honored as "rare disease heroes" by FDA during the anniversary year.
NORD's year-long observance of the 30th anniversary will include a
special section on its website where rare disease patients, researchers, and others will be invited to share stories about rare disease milestones and achievements.
On Rare Disease Day 2013 (Feb. 28th), which NORD sponsors nationally, there will be special activities to honor the history of the rare disease community. In addition, NORD's annual gala in May and annual conference in October will have an anniversary theme. There will be other special activities throughout the year.
The
Orphan Drug Act provides financial incentives such as seven years of marketing exclusivity for products developed to treat diseases affecting fewer than 200,000 Americans. There are 7,000 diseases considered rare in the U.S. affecting nearly 30 million Americans, or about 1 in 10 people.
In the decade before the law was passed, only 10 new drugs were developed by industry for patients with rare diseases. Since 1983, more than 2,700 potential treatments have entered the research pipeline as "orphan products" and more than 400 have been approved by FDA.
Highlights over the years since the law was enacted have included the
Rare Diseases Act of 2002, promoted by NORD, which made possible a Rare Diseases Clinical Research Network at NIH, and most recently the
FDA Safety and Innovation Act of 2012, which includes many major provisions related to rare diseases.
"This year, we will be saluting the progress made to date but we'll also be focusing on the fact that much remains to be done," Saltonstall said. "Most people with rare diseases still have no FDA-approved treatment. And, even when treatments exist, many patients have trouble accessing them.
"During this anniversary year, NORD will move forward with renewed commitment toward the development of safe, effective treatments for all, and assurance that all patients with serious medical problems will have access to the treatments and services they need."
The last few years IGF-1 for muscles and ASC-J9 are two drugs that have been discussed by researchers as having a potential benefit for Kennedy’s Disease (SBMA or Spinal Bulbar Muscular Atrophy). They are both currently being tested with small mammals. One of the big issues with both drugs is how to decide upon a dosage that will work, but will do no harm. And, if it does work, another issue is how to make the drug available (injection, pill, IV, etc.) and convenient. 
Recently, several articles have been published about athletes taking a banned substance called IGF-1 or a supplement made from deer antler velvet. The athletes are using the drug to improve their strength (in other words, cheating). Several people with Kennedy’s Disease have asked about using IGF-1 that you can by over the internet or in a foreign country.
