The 25th anniversary of the US Orphan Drug Act: an occasion to review achievements and consider future directions
A conference and a gala celebration were held in Washington, DC, on 19-20 May in order to commemorate the twenty-fifth anniversary of the Orphan Drug Act. The National Organization for Rare Disorders (NORD) was simultaneously celebrating 25 years of non-profit service for rare disease patients and their families. NORD, established in 1983 by rare disease patient group representatives and individuals, provided the advocacy behind the US Orphan Dug Act. The first such legislation in the world, versions of this vital regulation have since been adopted in other countries and continents around the world, including Australia, the European Union, Japan, South Korea and Taiwan.
The anniversary of the Orphan Drug Act was marked by a conference co-sponsored by the Food and Drug Administration (FDA) and the Drug Information Association (DIA), which took stock of the past 25 years of orphan drug legislation and considered future perspectives. Intended for researchers, biopharmaceutical representatives, patient groups, policy makers and clinical trial decision makers, the day-long event sought to identify the challenges and obstacles encountered in rare disease trials, and consider how to best optimise the orphan drug act provisions to bring rare disease drugs to market. An array of sessions considered the role of the orphan drug act, industry and patient perspectives, cooperation between the FDA and EMEA, the orphan product grants program, clinical trial regulatory monitoring, trial design for small patient populations, orphan drug application contents, and interactions with FDA review divisions.
A particularly interesting presentation was made by NORD founder and former president Abbey Meyers, who delineated the history of the Orphan Drug Act. In the 1970s, prior to passage of the legislation, the FDA created two task forces to study the issue of creating medicinal products with inherently low commercial value. At the time, Abbey Meyers was a housewife from Connecticut whose son was taking an experimental treatment that became unavailable. Trying to find a solution, she began contacting other patient organisations to see if they had experienced similar problems. This became the genesis for a coalition of patient organisations that eventually grew into NORD. With an actor from a popular television show bringing additional attention to the issue, the first legislation was created. This first bill proposed establishing a pool of government money from which companies could borrow for orphan drug development. If the product was approved, the company would pay the money back to the pool, along with any profits earned from sales. Both industry and congress soundly rejected this scheme. Meanwhile, the results of the task forces showed that pharmaceutical companies would be interested in rare disease products if there was a potential for profit, and at the very least the means to avoid losing money. The task forces recommended regulation that contained incentives such as exclusivity, tax credits, and protocol assistance. These recommendations, coupled with the lobbying of the burgeoning patient group coalition, led to the development of the Orphan Drug Act, which was signed into law in 1983.
Most of the original elements of the regulation are still intact today, despite several amendments over the years. In 1985, the definition of "orphan drugs" was broadened to include biologics. In addition, after heavy debate, the term rare disease was officially defined as a condition affecting less than 200,000 persons in the USA. In 1987, congress tried unsuccessfully to amend the bill to allow for "shared exclusivity" for two companies developing the same product for the same indication. In 1992, an amendment was proposed that would extend market exclusivity to nine years. This amendment would also impose a 120 million dollar sales ceiling for any given orphan drug product. Once reached, a competing product would be eligible for approval. Although passed by congress, this amendment was ultimately vetoed by then-president George Bush. Summing up the lessons gleaned from the past 25 years of the Orphan Drug Act, Abbey Meyers pointed out that the major controversies have occurred around the big money-making products, and were often sparked by those competitors that lost the race to marketing approval. Proposed changes have often favoured one corporation or one product. Abbey Meyers sums up the greatest achievement of NORD as their role in protecting the Orphan Drug Act from changes that would ultimately diminish its force.
Other notable presentations included an assessment of the accomplishments of the Orphan Drug Act from former Office of Orphan Products Development director Marlene Haffner, and a consideration of future challenges from Office of Rare Diseases director Stephen Groft. An exciting feature of the day was the unveiling of the Undiagnosed Diseases Program, a National Institutes of Health clinical research initiative specifically designed for medical cases that elude diagnosis. Read more about the Undiagnosed Diseases Program (below)
The next day it was NORD’s turn to blow out the candles. A gala dinner in the evening featured a speech by incoming NORD president and CEO Peter Saltonstall, as well as remarks by NORD board members Nancy Harris and Carolyn Asbury, and comments by the US Social Security Administration commissioner Michael Astrue. Attendees watched a video commemorating 25 years of orphan drug success, and were entertained by the Children’s Hospital Jazz Band.
Going forward, it is important to emphasise ways in which the international community can continue to share expertise and perspectives in order to fine-tune orphan drug legislation to maximise efficiency. The US and the EU enjoy a streamlined procedure for obtaining orphan drug designation that serves to simplify sponsor application in both territories and encourage orphan product development even further. Although in existence for a shorter period of time, the EMEA can inform the US of ways to accelerate productivity, while continuing to learn from the original orphan drug act pioneers, who initially found the way to bring relief for rare disease patients and continue to offer hope to many more.