Date of Award
Spring 4-7-2023
Embargo Period
4-19-2023
Document Type
Dissertation - MUSC Only
Degree Name
Doctor of Health Administration
Department
Health Administration
College
College of Health Professions
First Advisor
Kit N. Simpson
Second Advisor
Jiebing Wen
Third Advisor
Zahi Jurdi
Abstract
Background: Orphan drugs are developed to treat rare diseases that affect a small number of individuals. Due to the limited patient demand, orphan drugs are expensive to develop and bring to market. After the passage of the Orphan Drug Act in 1983, over 600 drugs have been approved. However, the coverage and reimbursement for these drugs by commercial health insurance and Medicare are not well understood. Methods: The orphan drugs approved by the FDA between 2015 - 2016 were identified via FDA Search Orphan Drug. A collection of paid claims data was examined on a retrospective basis using the 2016-2020 MarketScan® (Truven Analytics) Medicare and Private Insurance databases to determine the existence and differences in the reimbursement among commercial health insurers and Medicare for first-in-class orphan drugs launched between 2015 - 2016. Two cohorts of patients were identified and matched for their clinical conditions: commercial health insurance and Medicare. Descriptive statistics were reported using mean and standard deviation for continuous measures and frequencies and percentages for categorical variables to investigate relationships between the outcome variables, controlling for age, sex, insurance plan, and the approved rare disease indication. Results: All seven orphan drugs were covered by commercial health insurance and Medicare, when applicable. However, the orphan drug coverage, out-of-pocket costs, and cost of treatment varied across commercial health insurance and Medicare programs. Further, for all orphan drugs analyzed, commercial health insurance pays higher payments to providers and commercially-insured patients pay significantly higher maximum out-of-pocket costs than Medicare beneficiaries. Conclusion: This study highlights the complex landscape of coverage and reimbursement trends for orphan drugs by commercial health insurance and Medicare. These insights provide valuable information for stakeholders in the healthcare industry to guide future research and development efforts. In addition, the study emphasizes the crucial need for continued surveillance and evaluation of the orphan drug sector to ensure patients' access to affordable and effective treatments.
Recommended Citation
Teng, Ing, "Orphan Drug Reimbursement Trends" (2023). MUSC Theses and Dissertations. 786.
https://medica-musc.researchcommons.org/theses/786
Rights
Copyright is held by the author. All rights reserved.