A 30-year retrospective: National Organization for Rare Disorders, the Orphan Drug Act, and the role of rare disease patient advocacy groups

Abstract

This 30-year retrospective looks at the history of the US Orphan Drug Act and how it originally came to be enacted, with particular emphasis on the role of patient advocacy organizations. The article explores how rare-disease patient advocacy groups learned to work with government partners in the National Institutes of Health and US Food and Drug Administration, members of Congress, and the media to promote their cause. It also discusses their awakening to the value of collaboration, which led to the formation of an umbrella organization to represent their shared interests. Milestones over the years are briefly discussed, and current areas of focus for rare disease patient organizations -most notably, globalization -are also examined.

The involvement of patient organisations in rare disease research: a mixed methods study in Australia

“…Australian RDPOs currently rely mainly on their own expertise and resources when trying to advance research, as there is no policy framework to help them develop mutually beneficial relationships with researchers. In contrast, European patient organisations are actively engaged in RD-Connect, a EU-funded initiative linking rare disease registries, biobanks and other databases [ 28 ] and in the development of outcome measures for clinical trials, therapy approval, and post-marketing drug surveillance [ 23 , 49 ]. In the US, each of the research consortia in the National Institutes of Health’s Rare Diseases Clinical Research Network program directly involves patient organisations as well as researchers – enabling scientists, clinicians and people affected by rare diseases to work together on disease-specific projects [ 50 ].…”

Using four decades of FDA orphan drug designations to describe trends in rare disease drug development: substantial growth seen in development of drugs for rare oncologic, neurologic, and pediatric-onset diseases

“…In 1983, the United States Congress passed the Orphan Drug Act (ODA) to incentivize the development of drugs for rare diseases, defined in the ODA as affecting fewer than 200,000 people in the US. These incentives were primarily financial, to stimulate the biopharmaceutical industry’s interest in developing drugs for the relatively small populations of patients affected by these diseases, many of which are debilitating or life-threatening [ 1 ]. These incentives currently include a 25% tax credit on applicable research and development expenditures, waived user fees when submitting applications to the Food and Drug Administration (FDA), and the potential for a seven-year period of orphan drug exclusivity for the approved rare indication [ 2 ].…”

“…There have been significant changes in the way drugs are priced and reimbursed since the passage of the ODA [15]. We have also seen the creation of active patient groups for rare diseases, many of whom play a central role in creating patient registries and centers of excellence, and who fund the development of diagnostics, devices and drugs for their conditions [1,[16][17][18].…”

Rare diseases in Tanzania: a National Call for Action to address policy and urgent needs of individuals with rare diseases

“…In the United States (US) for example, the National Organization for Rare Disorders (NORD) which was established in 1983, advocates for policy changes to improve the lives of Americans impacted by RDs at the federal and state levels [ 16 ]. Within a period of 3 decades, NORD championed many initiatives leading to remarkable achievements [ 17 ]. These include establishment of the ClinicalTrials.gov website, where all studies receiving funding from the US government must be listed.…”