Coverage with Evidence Development of Cancer Treatments in the Netherlands | “Bringing Innovations to the patient"
Joost Verbeek is a PhD student in the Department of Health Technology & Services Research. (Co)Promotors are prof. dr. W.H. van Harten from the Faculty of Behavioural, Management and Social Science University of Twente and prof. dr. V.P. Retèl from NKI Netherlands Cancer Institute.
Healthcare spending in the EU has steadily increased due to factors like aging populations, medical advancements, and rising chronic diseases. Despite more investment, cancer incidence and mortality remain high, highlighting the need for innovative cancer care solutions. Health Technology Assessment (HTA) and Coverage with Evidence Development (CED) are crucial in balancing early patient access to promising health technologies with evidence-based decision making, utilizing methods like cost-effectiveness analysis and preference elicitation to inform reimbursement and resource allocation.
This dissertation examines patient access to innovative health technologies under CED programs and uses early HTA methods to support timely decision-making and adoption in two real-world examples: cytoreductive surgery combined with hyperthermic intraperitoneal chemotherapy (CRS/HIPEC) for gastric cancer with carcinomatosis (“PERISCOPE II study”) and high-dose chemotherapy (HDCT) with stem cell rescue for high-risk BRCA1-like breast cancer (“SUBITO study”). Moreover, a cost-effectiveness model was constructed for internet-based cognitive behavioural therapy (iCBT) for menopausal symptoms in breast cancer survivors (“EVA-online study”).
Chapter 2 presents a comprehensive overview of CED schemes in eight countries, and timelines and methods of finalized CED programs (n = 100) within those countries. Moreover, we identified eighteen factors influencing patient access to promising health technologies before, during, or after a CED program and formulated five key recommendations to improve timely and equitable patient access. Chapter 3 shows findings from qualitative interviews with 28 stakeholders, identifying 88 factors to support high-quality, equitable clinical implementation of HDCT for stage III, HER2-negative, BRCA1-like breast cancer patients in the Netherlands. In Chapter 4, we present the results of a discrete choice experiment. Our goal was to explore healthcare professionals’ treatment preferences, specifically focusing on how they weigh trade-offs between different treatment characteristics and especially outcomes. Our analysis revealed that the most important treatment attributes for healthcare professionals in determining the optimal treatment plan were the 10-year overall survival rate and the degree of long-term cognitive impairment. Therefore, results in the SUBITO study on those outcomes for all treatment arms are imperative for decision making. By better understanding healthcare providers’ priorities, more personalized and collaborative decision-making processes can be facilitated that align with both patient values and professional judgment. Chapter 5 presents an early cost-utility analysis of adding CRS/HIPEC to chemotherapy for gastric cancer with limited peritoneal carcinomatosis. CRS/HIPEC showed promise by generating more QALYs but at higher costs, resulting in an incremental cost-utility ratio (ICUR) of €50,990/QALY, which falls below the Dutch threshold. However, significant uncertainty remained, and the EVPI (€4 million) indicated substantial value in reducing this uncertainty, particularly regarding quality of life, overall survival, and identifying heterogeneity in the comparator arms. Further research on those aspects, such as within the ongoing PERISCOPE II trial, is therefore recommended. Chapter 6 evaluates the cost-effectiveness of guided and self-managed iCBT for menopausal symptoms in breast cancer survivors. Both formats were cost-effective, with ICURs below €30,000/QALY. Self-managed iCBT had lower costs, remained cost-effective in most scenarios, and showed potential for broader implementation. A stepped care approach is recommended, prioritizing self-managed iCBT and including therapist support when needed.
The last chapter summarizes the key findings and places them in a broader scientific and societal context to highlight potential future (methodological) directions. In summary, this dissertation aided in the evaluation of three different health technologies and highlights how CED programs and early HTA methods can support more equitable and timely access to promising health technologies while maintaining rigorous evaluations.
