Implementing performance-based risk-sharing agreements in non-small cell lung cancer immunotherapy: a real-world data case study

Result code in IS VaVaI

<a href="https://www.isvavai.cz/riv?ss=detail&h=RIV%2F00216224%3A14110%2F25%3A00141523" target="_blank" >RIV/00216224:14110/25:00141523 - isvavai.cz</a>

Result on the web

<a href="https://healtheconomicsreview.biomedcentral.com/articles/10.1186/s13561-025-00646-3" target="_blank" >https://healtheconomicsreview.biomedcentral.com/articles/10.1186/s13561-025-00646-3</a>

DOI - Digital Object Identifier

<a href="http://dx.doi.org/10.1186/s13561-025-00646-3" target="_blank" >10.1186/s13561-025-00646-3</a>

Result language

angličtina

Original language name

Implementing performance-based risk-sharing agreements in non-small cell lung cancer immunotherapy: a real-world data case study

Original language description

Background Performance-based risk-sharing agreements (PBRSA) represent an innovative tool for managing uncertainty and balanced distribution of the financial risk of high-cost drugs. By linking reimbursement to real-world treatment performance, these agreements help mitigate budgetary impacts.This study poses an illustrative patient-level PBRSA reimbursement model for non-small cell lung cancer (NSCLC) immunotherapy based on collected real-world data (RWD). Methods A retrospective analysis of 266 patients with NSCLC treated with immunotherapy was performed. Progression-free survival (PFS) served as the primary outcome measure of therapeutic effectiveness. An illustrative patient-level PBRSA model was developed to quantify the manufacturer's financial participation based on deviations from established PFS thresholds reported in randomised controlled trials (RCT).The manufacturer's financial responsibility increased proportionally to greater deviations in patient outcomes from the RCT benchmark. Cost calculations were limited exclusively to the acquisition price of immunotherapies, excluding administration, toxicity management, and other indirect costs. The potential PBRSA scenario was compared with the current reimbursement situation. Results Using this reimbursement method, cost savings per checkpoint inhibitor for healthcare payers could represent between 27.3% and 66.2% of the total cost, depending on the individual PFS reached. For the RWD cohort of NSCLC patients unsuccessfully treated with pembrolizumab monotherapy was 57.5% (a reduction in cost to payers from $27 996 to $11 893 per patient); pembrolizumab in combination 51.7% ($33 595 to $16 237); nivolumab 37.1% ($5 608 to $3 531); atezolizumab 27.3% ($11 799 to $8 583); and durvalumab 66.2% ($44 005 to $14 882). Conclusions This study proposes an illustrative patient-level PBRSA reimbursement model leveraging real-world clinical data to enhance risk-sharing for high-cost therapies. Unlike conventional cost-effectiveness analyses, this method directly links clinical performance and manufacturer financial responsibility. Future research should integrate comprehensive cost considerations and validate model performance in broader clinical settings.

Czech name

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Czech description

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Type

J_imp - Article in a specialist periodical, which is included in the Web of Science database

CEP classification

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OECD FORD branch

30230 - Other clinical medicine subjects

Project

—

Continuities

S - Specificky vyzkum na vysokych skolach

Publication year

2025

Confidentiality

S - Úplné a pravdivé údaje o projektu nepodléhají ochraně podle zvláštních právních předpisů

Name of the periodical

HEALTH ECONOMICS REVIEW

ISSN

2191-1991

e-ISSN

2191-1991

Volume of the periodical

15

Issue of the periodical within the volume

1

Country of publishing house

GB - UNITED KINGDOM

Number of pages

15

Pages from-to

1-15

UT code for WoS article

001505066000001

EID of the result in the Scopus database

2-s2.0-105007546935