Why orphan drug coverage reimbursement decision-making needs patient and public involvement

Conor M.W. Douglas, Elizabeth Wilcox, Michael Burgess and Larry D. Lynd

Health Policy, 2015, vol. 119, issue 5, 588-596

Abstract: Recently there has been an increase in the active involvement of publics and patients in healthcare and research, which is extending their roles beyond the passive recipients of medicines. However, there has been noticeably less work engaging them into decision-making for healthcare rationing exercises, priority setting, health technology assessment, and coverage decision-making. This is particularly evident in reimbursement decision-making for ‘orphan drugs’ or drugs for rare diseases. Medicinal products for rare disease offer particular challenges in coverage decision-making because they often lack the ‘evidence of efficacy’ profiles of common drugs that have been trialed on larger populations. Furthermore, many of these drugs are priced in the high range, and with limited health care budgets the prospective opportunity costs of funding them means that those resources cannot be allocated elsewhere. Here we outline why decision-making for drugs for rare diseases could benefit from increased levels of publics and patients involvement, suggest some possible forms that involvement could take, and advocate for empirical experimentation in this area to evaluate the effects of such involvement. Focus is given to the Canadian context in which we are based; however, potentialities and challenges relating to involvement in this area are likely to be similar elsewhere.

Keywords: Orphan drugs; Rare diseases; Public and patient involvement; Coverage; Decision-making; Policy (search for similar items in EconPapers)
Date: 2015
References: View references in EconPapers View complete reference list from CitEc
Citations: View citations in EconPapers (6)

Downloads: (external link)
http://www.sciencedirect.com/science/article/pii/S0168851015000123
Full text for ScienceDirect subscribers only

Related works:
This item may be available elsewhere in EconPapers: Search for items with the same title.

Export reference: BibTeX RIS (EndNote, ProCite, RefMan) HTML/Text

Persistent link: https://EconPapers.repec.org/RePEc:eee:hepoli:v:119:y:2015:i:5:p:588-596

DOI: 10.1016/j.healthpol.2015.01.009

Access Statistics for this article

Health Policy is currently edited by Katrien Kesteloot, Mia Defever and Irina Cleemput

More articles in Health Policy from Elsevier
Bibliographic data for series maintained by Catherine Liu () and ().