History of Orphan Drug Regulation—United States and Beyond

Haffner, Mph Marlene E.

doi:10.1002/cpt.426

Cited by 25 publications

(23 citation statements)

References 2 publications

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“…The current upward trend in designations may be the result of an overall increase in industry interest in the development of drugs for rare diseases due to advances in science and the incentives for rare disease product development . The number of approvals overall for orphan designated drugs increased from 15 in 2010 to 49 in 2015 .…”

Section: Discussionmentioning

confidence: 99%

“…The development of treatments for patients with rare diseases, such as sickle cell anemia, amyotrophic lateral sclerosis, and neuroblastoma, has historically been challenging based on the small number of patients available to study the safety and effectiveness of potential treatments and limited commercial viability. Because development of drugs for these populations were either not pursued or were ultimately abandoned, these drugs eventually became known as “orphan drugs.” The Orphan Drug Act (ODA), which was passed in 1983 in the United States to address this important public health challenge, has been lauded as one of the most successful pieces of US public health legislation . The fiscal and regulatory incentives contained in the ODA have shepherded over 700 additional orphan drug indications to market, compared with just 10 that were developed solely by industry prior to its enactment…”

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confidence: 99%

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Precision Medicines’ Impact on Orphan Drug Designation

Mueller

Rao²

2019

Clinical Translational Sci

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The incentives provided under the Orphan Drug Act (ODA) have been credited for catalyzing the marketing approval of drugs for the treatment of rare diseases by the US Food and Drug Administration. Orphan drug designation, the granting of special status to drugs or biologics (“drugs”) for the treatment of rare diseases, one of the ODA's key incentive programs, has seen major increases in volume over recent years. The new era of precision medicine and the development of therapies directed toward smaller “orphan” subsets of common diseases have been suggested as being a major driver. We evaluated the basis for orphan drug designations and orphan subsets in relation to the impact of precision medicines. We found that the increasing numbers of orphan drug designation determinations were not driven by precision medicines separating common diseases into orphan subsets and that orphan subsets overall also represented a relatively small proportion of designations.

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Section: Discussionmentioning

confidence: 99%

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confidence: 99%

Precision Medicines’ Impact on Orphan Drug Designation

Mueller

Rao²

2019

Clinical Translational Sci

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“…In 1982, the US Orphan Drug Act was passed. This legislation provided incentives to drug developers that made it more attractive to investigate therapeutics for rare diseases.…”

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confidence: 99%

“…Without this legislation and similar legislation passed in other jurisdictions there would be less progress than we have seen to date in fighting rare diseases. As is pointed out by Haffner, since the act was passed there have been 500 drugs approved in the United States for rare diseases. Without this act how many of these therapeutics would have been developed?…”

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Challenges and Opportunities in Rare Disease Drug Development

2016

Clin Pharma and Therapeutics

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Each month, Clinical Pharmacology & Therapeutics focuses on a particular theme. Twice a year, the associate editors, editors, and staff get together to discuss journal business and spend time setting up the calendar of themes. Often, there are no experts among us to take on a particular topic that we have chosen. The consequence is that one or two of us take on the theme and then have a crash course to learn as much as they can about it in order to solicit meaningful articles. This month's theme on rare diseases is such a case.

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The future of rare disease drug development: the rare disease cures accelerator data analytics platform (RDCA-DAP)

Barrett

Betourne

Walls

et al. 2023

J Pharmacokinet Pharmacodyn

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Rare disease drug development is wrought with challenges not the least of which is access to the limited data currently available throughout the rare disease ecosystem where sharing of the available data is not guaranteed. Most pharmaceutical sponsors seeking to develop agents to treat rare diseases will initiate data landscaping efforts to identify various data sources that might be informative with respect to disease prevalence, patient selection and identification, disease progression and any data projecting likelihood of patient response to therapy including any genetic data. Such data are often difficult to come by for highly prevalent, mainstream disease populations let alone for the 8000 rare disease that make up the pooled patient population of rare disease patients. The future of rare disease drug development will hopefully rely on increased data sharing and collaboration among the entire rare disease ecosystem. One path to achieving this outcome has been the development of the rare disease cures accelerator, data analytics platform (RDCA-DAP) funded by the US FDA and operationalized by the Critical Path Institute. FDA intentions were clearly focused on improving the quality of rare disease regulatory applications by sponsors seeking to develop treatment options for various rare disease populations. As this initiative moves into its second year of operations it is envisioned that the increased connectivity to new and diverse data streams and tools will result in solutions that benefit the entire rare disease ecosystem and that the platform becomes a Collaboratory for engagement of this ecosystem that also includes patients and caregivers.

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History of Orphan Drug Regulation—United States and Beyond

Cited by 25 publications

References 2 publications

Precision Medicines’ Impact on Orphan Drug Designation

Precision Medicines’ Impact on Orphan Drug Designation

Challenges and Opportunities in Rare Disease Drug Development

The future of rare disease drug development: the rare disease cures accelerator data analytics platform (RDCA-DAP)

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