Dataset Information

Estimating the clinical cost of drug development for orphan versus non-orphan drugs.

ABSTRACT:

Background

High orphan drug prices have gained the attention of payers and policy makers. These prices may reflect the need to recoup the cost of drug development from a small patient pool. However, estimates of the cost of orphan drug development are sparse.

Methods

Using publicly available data, we estimated the differences in trial characteristics and clinical development costs with 100 orphan and 100 non-orphan drugs.

Results

We found that the out-of-pocket clinical costs per approved orphan drug to be $166 million and $291 million (2013 USD) per non-orphan drug. The capitalized clinical costs per approved orphan drug and non-orphan drug were estimated to be $291 million and $412 million respectively. When focusing on new molecular entities only, we found that the capitalized clinical cost per approved orphan drug was half that of a non-orphan drug.

Conclusions

More discussion is needed to better align on which cost components should be included in research and development costs for pharmaceuticals.

SUBMITTER: Jayasundara K

PROVIDER: S-EPMC6327525 | biostudies-literature | 2019 Jan

REPOSITORIES: biostudies-literature

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Publications

Estimating the clinical cost of drug development for orphan versus non-orphan drugs.

Jayasundara Kavisha K Hollis Aidan A Krahn Murray M Mamdani Muhammad M Hoch Jeffrey S JS Grootendorst Paul P

Orphanet journal of rare diseases 20190110 1

<h4>Background</h4>High orphan drug prices have gained the attention of payers and policy makers. These prices may reflect the need to recoup the cost of drug development from a small patient pool. However, estimates of the cost of orphan drug development are sparse.<h4>Methods</h4>Using publicly available data, we estimated the differences in trial characteristics and clinical development costs with 100 orphan and 100 non-orphan drugs.<h4>Results</h4>We found that the out-of-pocket clinical cos ...[more]

PMID: 30630499

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Project description:BackgroundOrphan drug designations are a useful proxy to investigate trends in rare disease drug development. Drug developers must receive a designation before they are eligible for the economic incentives of the Orphan Drug Act in the United States. We created a database of all orphan drugs designated between 1983 and 2019 that included numerous drug characteristics, including therapeutic area. In addition, we constructed a "broad disease" categorization of designations as an alternative to therapeutic area, based on disease etiology and age of onset rather than organ system. By looking at the pattern of orphan drug designations over the past four decades, this analysis studied the impact of the evolving rare disease drug development landscape and considers the future of rare disease therapies over the coming decades.ResultsBetween 1983 and 2019, a total of 5099 drugs and biologics received orphan drug designation. Designations more than doubled between the 1980s and 1990s, almost doubled between the 1990s and 2000s, and almost tripled in number between the 2000s and 2010s. The top three therapeutic areas represented in the orphan drug designations were: oncology (1910, 37%), neurology (674, 13%), and infectious diseases (436, 9%). The broad disease categorization found that the proportion of designations for pediatric-onset diseases has increased in the most recent decade to 27%.ConclusionsAnalysis of the last four decades of orphan drug designation indicates seismic shifts have occurred in the rare disease drug development space. The number of designations granted more than quadrupled between the 1990s and 2010s. While these substantial increases led to growth in the absolute number of designations within all therapeutic areas (bar one) and broad disease categories, the relative proportions have seen considerable change over time. In the most recent decade, there have been notable increases in the proportion of drugs in oncology, pediatric-onset diseases, and neurologic disorders. The dramatic rise in overall orphan designations over the past four decades suggests we may continue to see an upward trajectory in designations leading to an increased number of approvals for drugs and biologics designed specifically for diagnosing, preventing, and treating rare diseases in the coming decades.

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Dataset Information

Estimating the clinical cost of drug development for orphan versus non-orphan drugs.

Background

Methods

Results

Conclusions

Publications

Estimating the clinical cost of drug development for orphan versus non-orphan drugs.

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