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March 26, 2020

Evidence Underlying Policy Proposals for Sustainable Anticancer Drug Prices: A Systematic Review

Author Affiliations
  • 1Department of Health Technology and Services Research, University of Twente, Enschede, the Netherlands
  • 2The Netherlands Cancer Institute, Amsterdam, the Netherlands
  • 3Rijnstate Hospital, Arnhem, the Netherlands
JAMA Oncol. 2020;6(6):909-916. doi:10.1001/jamaoncol.2019.6846
Key Points

Questions  What policy proposals to reduce drug prices at market launch exist and what is the quantitative evidence on which these proposals are based?

Findings  This systematic review identified 4775 articles on oncologic policy proposals to reduce drug prices; 80 were included, and an inventory of 23 policy proposals was created. Because the existing evidence was insufficient to deduct price effects, 6 promising proposals that need further analysis were identified: transparency, delinkage, 2-part pricing, public research, orphan drug reform, and public clinical trials.

Meaning  The findings suggest that the identified proposed policies should be tested with small-scale experiments, simulations, and pilots to establish price effects.


Importance  The financial consequences of high-expenditure innovative drugs and the association of these consequences with access to cancer treatment are substantial. With oncology being one of the major spending blocks of care and research, innovative policies are needed to secure the sustainability and accessibility of health care systems. Despite this strong interest, structured approaches are missing to date, and proposals are often based on opinion rather than fact.

Objectives  To evaluate an inventory of policies to reduce drug prices at market launch and analyze the quantitative evidence on which these policies are based.

Evidence Review  For this systematic review, a literature search of the Ovid MEDLINE, Embase, Business Source Premier, ABI/Inform, World Health Organization, and Organisation for Economic Co-operation and Development databases was conducted for articles published between January 1, 2001, and December 31, 2017. Publications that described proposed policies with a direct or obvious indirect association with pharmaceutical prices at market launch and with relevance to oncology and high-income countries were included. Evidence basis was assessed per article, and quantitative articles were categorized according to time and data use. Main price mechanisms and scored system disruptiveness per proposal were identified. Data were analyzed from January 1, 2018, to January 1, 2019.

Findings  Of the 4775 articles screened, 80 were selected, and an inventory of 23 policies to reduce medicine prices was created. Proposals were diverse but mainly applied the strengthening of competition as an underlying mechanism to reduce drug prices. Of the 80 studies, 23 used quantitative models, but existing evidence was insufficient to deduce price effects, especially considering system disruptiveness. The available evidence was used to identify promising proposals for which testing may be beneficial: transparency, delinkage, 2-part pricing, public research, orphan drug reform, and public clinical trials.

Conclusions and Relevance  The findings suggest that despite the urgency of the search for proposals that lead to sustainable drug prices, careful and structured testing of proposals is needed because the implications for access to drug treatment can be substantial.

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