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

Kód výsledku v 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>

Výsledek na webu

<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>

Jazyk výsledku

angličtina

Název v původním jazyce

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

Popis výsledku v původním jazyce

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.

Název v anglickém jazyce

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

Popis výsledku anglicky

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.

Druh

J_imp - Článek v periodiku v databázi Web of Science

CEP obor

—

OECD FORD obor

30230 - Other clinical medicine subjects

Projekt

—

Návaznosti

S - Specificky vyzkum na vysokych skolach

Rok uplatnění

2025

Kód důvěrnosti údajů

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

Název periodika

HEALTH ECONOMICS REVIEW

ISSN

2191-1991

e-ISSN

2191-1991

Svazek periodika

15

Číslo periodika v rámci svazku

1

Stát vydavatele periodika

GB - Spojené království Velké Británie a Severního Irska

Počet stran výsledku

15

Strana od-do

1-15

Kód UT WoS článku

001505066000001

EID výsledku v databázi Scopus

2-s2.0-105007546935