Journal of General Internal Medicine

, Volume 34, Issue 3, pp 338–340 | Cite as

Out-of-pocket Spending on Orphan Drug Prescriptions Among Commercially Insured Adults in 2014

  • Kao-Ping ChuaEmail author
  • Rena M. Conti
Concise Research Report


Orphan drugs are products with at least one Food and Drug Administration (FDA)-approved indication to treat rare diseases affecting fewer than 200,000 Americans. Many orphan drugs also have FDA-approved non-orphan indications.1

Manufacturers of orphan drugs enjoy extended periods free from generic competition,1 allowing them considerable latitude on pricing. In 2016, annual list price spending per patient exceeded $50,000 for 40.9% of orphan drugs,1 prompting concerns over the burden of high prices on payers. Commercially insured patients, however, may also be burdened by these prices, particularly if they pay a percentage of drug prices through deductibles and co-insurance.2 Though prior studies have examined orphan drug benefit design,3 none have examined out-of-pocket spending for these drugs. We estimated out-of-pocket spending on orphan drug prescription fills at retail and mail-order pharmacies among a national sample of commercially insured adults.




Compliance with Ethical Standards


Dr. Conti served on the National Academy of Sciences, Engineering, and Medicine Committee “Ensuring Patient Access to Affordable Drug Therapies.” Dr. Conti reports funding from The Commonwealth Fund and the American Cancer Society for her work on this study.

Conflict of Interest

The authors declare that they do not have a conflict of interest.


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Copyright information

© Society of General Internal Medicine 2018

Authors and Affiliations

  1. 1.Department of Pediatrics and Communicable Diseases, Child Health Evaluation and Research CenterUniversity of Michigan Medical SchoolAnn ArborUSA
  2. 2.Department of Markets, Public Policy, and LawBoston University Questrom School of BusinessBostonUSA
  3. 3.Institute for Health System Innovation and PolicyBoston University Questrom School of BusinessBostonUSA

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