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Effectiveness of National Pricing Policies for Patent-Protected Pharmaceuticals in the OECD: A Systematic Literature Review

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Abstract

Objectives

The aim of this review is to assess the current state of empirical research regarding the effectiveness of national pricing regulations of the patent-protected market for prescription pharmaceuticals. Effectiveness is understood to be the capacity of policies to have a desired impact on outcomes, such as health status, patient access, healthcare expenditure, and research investments, among others.

Methods

A systematic review of the published literature on pricing regulations in OECD countries was performed. The PubMed, MEDLINE, Scopus, Web of Science, Cochrane Library and the OECD iLibrary databases were searched in September 2016 and December 2017, with an update in August 2018. Interrupted time series studies and additional empirical studies were included, as well as systematic reviews if appropriate methods were applied. The risk of bias was assessed based on the recommendations of the BMJ guidelines, Cochrane EPOC criteria, QHES instrument, HTA good practice guidelines, CRD’s guidance and the CHEC criteria. The quality of evidence was evaluated using the suggestions from EPOC and GRADE.

Results

Thirty-one publications met the inclusion criteria. Most of the assessed empirical research included therapeutic (TRP) and/or external reference pricing (ERP), with a clear majority focusing on TRP. The main outcomes that were analysed were drug prices, expenditures and drug use. For value-based pricing (VBP), only limited empirical data were found.

Conclusions

We found evidence that TRP may reduce pharmaceutical prices and expenditures in the short term. Furthermore, TRP may lead to substitution effects towards lower-priced pharmaceuticals. The effects of TRP on patient access, healthcare utilisation and R&D investments were found to be uncertain. No conclusions were drawn for ERP and VBP. No evidence was found for the effects on health outcomes for any of the analysed policies. There is a strong need for evidence generation regarding effective pricing policies, particularly for VBP, managed entry agreements and non-financial outcomes.

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Notes

  1. The three papers from the OECD and EXPH refer to some of the recent literature on MEAs, which we excluded since none of them provides a policy effect analysis (see above).

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Contributions

All of the authors (DJW and SB) fulfilled the authorship criteria and contributed to the design of the study, review of the search protocol, analysis and interpretation of the results, and writing and review of the manuscript. DJW prepared the search protocol, performed the database searches, screenings, categorisations, and assessments and prepared the draft of the manuscript. All of the authors are aware of the submission and are in agreement with the manuscript.

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Correspondence to Dominik J. Wettstein.

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No funding was received for the research performed or the writing of the manuscript.

Conflict of interest

DJW is an external PhD student at the University of Lucerne and was employed at the pharmaceutical company Takeda Pharma while performing this research. Neither the former employer of the first author nor any other private or public entity supported or influenced this research in any relevant way. SB has no conflicts of interest to declare. All of the authors submitted a signed Conflict of Interest disclosure form.

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Wettstein, D.J., Boes, S. Effectiveness of National Pricing Policies for Patent-Protected Pharmaceuticals in the OECD: A Systematic Literature Review. Appl Health Econ Health Policy 17, 143–162 (2019). https://doi.org/10.1007/s40258-018-0437-z

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