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

J 2025

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

BÁRTOVÁ, Adéla; Filippo RUMI; Joao Vasco SANTOS; Adam SVOBODNÍK; Barbora ŘÍHOVÁ et. al.

Základní údaje

Originální název

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

Autoři

BÁRTOVÁ, Adéla (203 Česká republika, domácí); Filippo RUMI; Joao Vasco SANTOS; Adam SVOBODNÍK (203 Česká republika, domácí) a Barbora ŘÍHOVÁ (203 Česká republika, domácí)

Vydání

HEALTH ECONOMICS REVIEW, LONDON, BMC, 2025, 2191-1991

Další údaje

Jazyk

angličtina

Typ výsledku

Článek v odborném periodiku

Obor

30230 Other clinical medicine subjects

Stát vydavatele

Velká Británie a Severní Irsko

Utajení

není předmětem státního či obchodního tajemství

Odkazy

URL

Impakt faktor

Impact factor: 3.300 v roce 2024

Organizační jednotka

Lékařská fakulta

DOI

https://doi.org/10.1186/s13561-025-00646-3

UT WoS

001505066000001

EID Scopus

2-s2.0-105007546935

Klíčová slova anglicky

non-small cell lung cancer immunotherapy; performance-based risk-sharing agreements

Štítky

14110516, 14119710, rivok

Příznaky

Mezinárodní význam, Recenzováno

Změněno: 7. 7. 2025 12:53, Mgr. Tereza Miškechová

Anotace

V originále

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.

Návaznosti

MUNI/A/1722/2024, interní kód MU

Název: Specifický farmakologický výzkum v oblasti farmakokinetiky, behaviorální neuropsychofarmakologie a personalizované farmakoterapie v onkologii

Investor: Masarykova univerzita, Specifický farmakologický výzkum v oblasti farmakokinetiky, behaviorální neuropsychofarmakologie a personalizované farmakoterapie v onkologii

Citovat

BÁRTOVÁ, Adéla; Filippo RUMI; Joao Vasco SANTOS; Adam SVOBODNÍK a Barbora ŘÍHOVÁ. Implementing performance-based risk-sharing agreements in non-small cell lung cancer immunotherapy: a real-world data case study. HEALTH ECONOMICS REVIEW. LONDON: BMC, 2025, roč. 15, č. 1, s. 1-15. ISSN 2191-1991. Dostupné z: https://doi.org/10.1186/s13561-025-00646-3.

@article{2507565,
   author = {Bártová, Adéla and Rumi, Filippo and Santos, Joao Vasco and Svobodník, Adam and Říhová, Barbora},
   article_location = {LONDON},
   article_number = {1},
   doi = {https://doi.org/10.1186/s13561-025-00646-3},
   keywords = {non-small cell lung cancer immunotherapy; performance-based risk-sharing agreements},
   language = {eng},
   issn = {2191-1991},
   journal = {HEALTH ECONOMICS REVIEW},
   title = {Implementing performance-based risk-sharing agreements in non-small cell lung cancer immunotherapy: a real-world data case study},
   url = {https://healtheconomicsreview.biomedcentral.com/articles/10.1186/s13561-025-00646-3},
   volume = {15},
   year = {2025}
}

TY  - JOUR
ID  - 2507565
AU  - Bártová, Adéla - Rumi, Filippo - Santos, Joao Vasco - Svobodník, Adam - Říhová, Barbora
PY  - 2025
TI  - Implementing performance-based risk-sharing agreements in non-small cell lung cancer immunotherapy: a real-world data case study
JF  - HEALTH ECONOMICS REVIEW
VL  - 15
IS  - 1
SP  - 1-15
EP  - 1-15
PB  - BMC
SN  - 21911991
KW  - non-small cell lung cancer immunotherapy
KW  - performance-based risk-sharing agreements
UR  - https://healtheconomicsreview.biomedcentral.com/articles/10.1186/s13561-025-00646-3
N2  - 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.
ER  -

BÁRTOVÁ, Adéla; Filippo RUMI; Joao Vasco SANTOS; Adam SVOBODNÍK a Barbora ŘÍHOVÁ. Implementing performance-based risk-sharing agreements in non-small cell lung cancer immunotherapy: a real-world data case study. \textit{HEALTH ECONOMICS REVIEW}. LONDON: BMC, 2025, roč.~15, č.~1, s.~1-15. ISSN~2191-1991. Dostupné z: https://doi.org/10.1186/s13561-025-00646-3.

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