| dc.description.abstract | Abstract
Background: Larotrectinib and entrectinib are tumour-agnostic therapies that received marketing authorization from the European Medicines Agency (EMA) in 2019 and 2020. In the Netherlands, both therapies are conditionally covered in the healthcare package while awaiting a definitive reimbursement decision. The limited evidence base caused uncertainties which complicated the evaluation for (cost-)effectiveness and reimbursement.
Objective: This study aimed to develop a strategic framework for pricing and reimbursement decisions for the two tumour-agnostic therapies by linking the uncertainties and corresponding risks to various managed entry agreements (MEAs).
Methods: The most pressing uncertainties of larotrectinib and entrectinib were prioritized based on previous research. Existing MEAs were identified from the literature and linked to the uncertainties if they were capable of mitigating the uncertainty or the risk, which led to the development of the reimbursement framework. The MEAs were linked based on the root cause of the uncertainty, the type of uncertainty, and the risks associated. Stakeholder interviews with Dutch healthcare stakeholders were conducted to better understand the preferences and implementation considerations for each of the MEAs.
Results: The developed framework consists of two parts. First, the four most pressing uncertainties and their associated risks are mentioned. The second part lays down the reimbursement models, including the level and timing of payment, that are linked or not linked to the uncertainties. Stakeholder interviews displayed that the healthcare payer and the healthcare insurer majorly preferred a simple implementation strategy. The pharmaceutical company for most uncertainties preferred neither of the proposed strategies and suggested other models. Whereas, the other stakeholders preferred the complex strategy for the majority of the implementation strategies.
Conclusions: As tumour-agnostic therapies are surrounded by many uncertainties, focusing on mitigating the most pressing ones can be a good starting point. The developed reimbursement framework for these uncertainties has the potential to ease the decision-making of reimbursement of these therapies.
Keywords: Tumour-agnostic therapies, larotrectinib, entrectinib, managed entry agreements, reimbursement framework | |
