Skip to main page content
U.S. flag

An official website of the United States government

Dot gov

The .gov means it’s official.
Federal government websites often end in .gov or .mil. Before sharing sensitive information, make sure you’re on a federal government site.

Https

The site is secure.
The https:// ensures that you are connecting to the official website and that any information you provide is encrypted and transmitted securely.

Access keys NCBI Homepage MyNCBI Homepage Main Content Main Navigation
. 2022 Apr;15(4):809-812.
doi: 10.1111/cts.13270. Epub 2022 Mar 25.

The economics of moonshots: Value in rare disease drug development

Nathan Yates  1 , Jennifer Hinkel  2
Affiliations

The economics of moonshots: Value in rare disease drug development

Nathan Yates et al. Clin Transl Sci. 2022 Apr.
No abstract available

PubMed Disclaimer

Conflict of interest statement

The authors declared no competing interests for this work.

References

    1. Richter T, Nestler‐Parr S, Babela R, et al. Rare disease terminology and definitions—a systematic global review: report of the ISPOR rare disease special interest group. Value Health. 2015;18:906‐914. - PubMed
    1. Bagley N, Berger B, Chandra A, Garthwaite C, Stern AD. The Orphan Drug Act at 35: observations and an outlook for the twenty‐first century. Innov Policy Econ. 2019;19:97‐137.
    1. Sarpatwari A, Beall RF, Abdurrob A, He M, Kesselheim AS. Evaluating the impact of the Orphan Drug Act’s seven‐year market exclusivity period. Health Aff. 2018;37:732‐737. - PubMed
    1. Nabhan C, Phillips EG Jr, Feinberg BA. Orphan cancer drugs in the era of precision medicine. JAMA Oncol. 2018;4:1481‐1482. - PubMed
    1. Haendel M, Vasilevsky N, Unni D, et al. How many rare diseases are there? Nat Rev Drug Discov. 2020;19:77‐78. - PMC - PubMed