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 patient organization leaders 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 it passed.
Exactly four months after President Reagan signed the bill into law, on May 4, 1983, the coalition of patient advocates formally became NORD – 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 legislation enacted at that time," says NORD President and CEO Peter L. Saltonstall. "Today and throughout 2013, we will be saluting the pioneers from that period, 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.
Over the years, government partners at the National Institutes of Health (NIH) and Food and Drug Administration (FDA) have provided support and encouragement to the rare disease patient 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 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 submit stories of 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 conference in October will have an anniversary theme.
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 people 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 have included the Rare Diseases Act of 2002, promoted by NORD, which made possible the establishment of 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 celebrating 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."