Publications by authors named "R A Vreman"
The European Commission (EC) has proposed to redefine 'unmet medical need' to steer pharmaceutical innovation and link the definition to incentives and regulatory tools and procedures. A multistakeholder meeting of the Regulatory Science Network Netherlands (RSNN) discussed the impact of this proposal. Four principles were highlighted (flexibility, feasibility, fairness, and sensitivity to risk) that were not considered sufficiently addressed.
View Article and Find Full Text PDFObjective: To construct a framework and calculation tool to compare the consequences of implementing different payment models for high-cost, one-off potentially curative therapies and enable decision making to ultimately enhance timely patient access to innovative health interventions.
Methods: A framework outlining steps to determine potentially suitable payment models was developed. Based on the framework, a supporting calculation tool operationalised as an Excel-based model was constructed to quantify the associated costs for an average patient during the timeframe of the intended payment agreement, the total budget impact and associated benefits expressed in quality-adjusted life-years for the total expected lifetime of the patient population.
Objectives: Uncertainty regarding the long-term relative effectiveness is an important factor in health technology assessment (HTA) of medicines. This study investigated how different HTA bodies address this uncertainty in their assessments.
Methods: A total of 49 HTA reports from 6 national HTA bodies, assessing 9 medicines for spinal muscular atrophy, cystic fibrosis, and hypercholesterolemia, were included.
Article Synopsis
- - The study assesses various managed entry agreements for financing the gene therapy atidarsagene autotemcel (Libmeldy) in the Dutch healthcare system, aiming to inform future payment structures for high-cost treatments.
- - Three payment models were compared: a 60% price discount, outcome-based payments with discounts, and a willingness-to-pay linked model, focusing on costs and benefits for different patient response scenarios.
- - Results indicated that outcome-based models generally led to lower budget impacts and better financial management for payers, especially for patients responding as predicted by health assessments, compared to a simple discount approach.
Operationalization guidance is needed to support health technology assessment (HTA) bodies considering implementing lifecycle HTA (LC-HTA) approaches. The 2022 Health Technology Assessment International (HTAi) Global Policy Forum (GPF) established a Task Force to develop a position paper on LC-HTA. In its first paper, the Task Force established a definition and framework for LC-HTA in order to tailor it to specific decision problems.
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