Implementing outcomes-based risk-sharing agreements: an integrative review of applications in blood cancer in the UK and beyond

ABSTRACT

Introduction

Outcomes-based risk-sharing agreements (OBRSA) have been increasingly used worldwide to manage uncertainty in value assessments. This review aimed to summarize motivations, barriers, and facilitators to implementing OBRSAs with a specific focus on therapies for hematological cancer.

Areas covered

An integrative review was conducted based on a scoping of existing reviews on the topic and reports published by UK NICE. Findings from 16 articles and 10 reports were summarized and categorized into three themes: applications in blood cancer drugs, motivations for adoption, and barriers and facilitators to implementation.

Expert opinion

There was a dissociation between the theoretical basis for opting for OBRSAs, and reasons stated or inferred from practice. The administrative burden was considered a notable barrier to implementation, which affects not only payers and manufacturers but also healthcare providers. Effective stakeholder engagement and building mutual trust among key groups were identified as factors enabling successful implementation. The review raises essential considerations in implementing OBRSAs and implications for their future role, particularly for blood cancer drugs where uncertainty is rife. Carefully designed and managed schemes may remain an option for health systems to manage risks involved when funding high-cost treatments.

KEYWORDS:

• Blood cancer
• health technology assessment
• managed-entry
• outcomes-based risk-sharing agreements
• uncertainty

Article highlights

• This review summarizes motivations, barriers, and facilitators to implementing outcomes-based risk-sharing agreements with a specific focus on blood cancer therapies.

• Schemes for blood cancer drugs were put in place mainly to address uncertainty arising from incomplete trial data, use of subsequent therapies, treatment duration, and epidemiological measures that impact estimation of costs.

• Administrative burden was considered a notable barrier, while effective stakeholder engagement and mutual trust between payers and companies were identified as enabling factors to implementation.

Declaration of interest

The authors have no other relevant affiliations or financial involvement with any organization or entity with a financial interest in or financial conflict with the subject matter or materials discussed in the manuscript apart from those disclosed.

Reviewer disclosures

Peer reviewers on this manuscript have no relevant financial or other relationships to disclose.

Acknowledgments

We express our gratitude to the National University of Singapore for awarding a Research Scholarship to the first author, for which this study was conducted as a part of a PhD program.

Supplementary material

Supplemental data for this article can be accessed online at https://doi.org/10.1080/14737167.2023.2240515

Additional information

Funding

The study is conducted as part of a doctoral thesis of the first author who is a recipientof a research scholarship from the National University of Singapore.