Policymaker, Please Consider Your Needs Carefully: Does Outcomes Research in Relapsed or Refractory Multiple Myeloma Reduce Policymaker Uncertainty Regarding Value for Money of Bortezomib?

Franken, M; Gaultney, J; Blommestein, Hedwig M.; Huijgens, Peter C.; Sonneveld, Pieter; Redekop, William K.; Groot, Carin A. Uyl–de

doi:10.1016/j.jval.2013.12.009

Cited by 9 publications

(11 citation statements)

References 47 publications

Supporting

Mentioning

Contrasting

Order By: Relevance

“…This would require the use of surrogate endpoints which may give false reassurance of performance (Hettle et al, 2017;Pauwels et al, 2017;Seeley and Kesselheim, 2017;Toumi et al, 2017). Therefore, validated surrogate endpoints that are proven predictors of hard clinical endpoints and uninfluenced by other treatments should be selected (Garber and McClellan, 2007;McCabe et al, 2010;Brennan and Wilson, 2014;Franken et al, 2014;Launois et al, 2014;Carr and Bradshaw, 2016;Gerkens et al, 2017;Toumi et al, 2017;. However, proven validated surrogate endpoints may not exist for all disease areas which complicates agreement between payer and developer on the chosen outcome.…”

Section: The Organization Of Data Collectionmentioning

confidence: 99%

Barriers and Opportunities for Implementation of Outcome-Based Spread Payments for High-Cost, One-Shot Curative Therapies

et al. 2020

View full text Add to dashboard Cite

Background: The challenging market access of high-cost one-time curative therapies has inspired the development of alternative reimbursement structures, such as outcome-based spread payments, to mitigate their unaffordability and answer remaining uncertainties. This study aimed to provide a broad overview of barriers and possible opportunities for the practical implementation of outcome-based spread payments for the reimbursement of one-shot therapies in European healthcare systems.Methods: A systematic literature review was performed investigating published literature and publicly available documents to identify barriers and implementation opportunities for both spreading payments and for implementing outcome-based agreements. Data was analyzed via qualitative content analysis by extracting data with a reporting template.Results: A total of 1,503 publications were screened and 174 were included. Main identified barriers for the implementation of spread payments are reaching an agreement on financial terms while considering 12-months budget cycles and the possible violation of corresponding international accounting rules. Furthermore, outcome correction of payments is currently hindered by the need for additional data collection, the lack of clear governance structures and the resulting administrative burden and cost. The use of spread payments adjusted by population- or individual-level data collected within automated registries and overseen by a governance committee and external advisory board may alleviate several barriers and may support the reimbursement of highly innovative therapies.Conclusion: High-cost advanced therapy medicinal products pose a substantial affordability challenge on healthcare systems worldwide. Outcome-based spread payments may mitigate the initial budget impact and alleviate existing uncertainties; however, their effective implementation still faces several barriers and will be facilitated by realizing the required organizational changes.

show abstract

Section: The Organization Of Data Collectionmentioning

confidence: 99%

Barriers and Opportunities for Implementation of Outcome-Based Spread Payments for High-Cost, One-Shot Curative Therapies

et al. 2020

View full text Add to dashboard Cite

show abstract

“…In observational registries, however, data on response and progression may be biased because this may not be accurately captured in patient files [ 19 ]. Moreover, physicians in daily practice often do not report using standardized response criteria [ 20 ], whereas RCTs dictate response criteria. This may especially be the case when data are retrospectively collected by other individuals than the treating physician.…”

Section: Conducting Sound Economic Evaluations With Registry Datamentioning

confidence: 99%

A Practical Guide for Using Registry Data to Inform Decisions About the Cost Effectiveness of New Cancer Drugs: Lessons Learned from the PHAROS Registry

2015

Self Cite

View full text Add to dashboard Cite

Decision makers increasingly request evidence on the real-world cost effectiveness of a new treatment. There is, however, a lack of practical guidance on how to conduct an economic evaluation based on registry data and how this evidence can be used in actual decision making. This paper explains the required steps on how to perform a sound economic evaluation using examples from an economic evaluation conducted with real-world data from the Dutch Population based HAematological Registry for Observational Studies. There are three main issues related to using registry data: confounding by indication, missing data, and insufficient numbers of (comparable) patients. If encountered, it is crucial to accurately deal with these issues to maximize the internal validity and generalizability of the outcomes and their value to decision makers. Multivariate regression modeling, propensity score matching, and data synthesis are well-established methods to deal with confounding. Multiple imputation methods should be used in cases where data are missing at random. Furthermore, it is important to base the incremental cost-effectiveness ratio of a new treatment compared with its alternative on comparable groups of (matched) patients, even if matching results in a small analytical population. Unmatched real-world data provide insights into the costs and effects of a treatment in a real-world setting. Decision makers should realize that real-world evidence provides extremely valuable and relevant policy information, but needs to be assessed differently compared with evidence derived from a randomized clinical trial.

show abstract

“…Other policy instruments may be considered as alternatives for conditional reimbursement, for example, financeor outcome risk-sharing agreements (e.g., pay for performance and finance agreements). These alternatives, however, suffer from issues such as high implementation and transaction costs, administrative burden, and lack of transparency [32].…”

Section: Discussionmentioning

confidence: 99%

The Challenge of Conditional Reimbursement: Stopping Reimbursement Can Be More Difficult Than Not Starting in the First Place!

2017

View full text Add to dashboard Cite

show abstract

Policymaker, Please Consider Your Needs Carefully: Does Outcomes Research in Relapsed or Refractory Multiple Myeloma Reduce Policymaker Uncertainty Regarding Value for Money of Bortezomib?

Cited by 9 publications

References 47 publications

Barriers and Opportunities for Implementation of Outcome-Based Spread Payments for High-Cost, One-Shot Curative Therapies

Barriers and Opportunities for Implementation of Outcome-Based Spread Payments for High-Cost, One-Shot Curative Therapies

A Practical Guide for Using Registry Data to Inform Decisions About the Cost Effectiveness of New Cancer Drugs: Lessons Learned from the PHAROS Registry

The Challenge of Conditional Reimbursement: Stopping Reimbursement Can Be More Difficult Than Not Starting in the First Place!

Contact Info

Product

Resources

About