Establishing cost-effectiveness of genetic targeting of cancer therapies\ud

The clinical benefit of a new genomic instrument, the 70-gene signature\ud for breast cancer patients, is being evaluated in a randomised clinical\ud trial. The early, controlled implementation process is supported by a\ud Constructive Technology Assessment to help decision-making in an\ud uncertain time of developmen

Establishing cost-effectiveness of genetic targeting of cancer therapies

The clinical benefit of a new genomic instrument, the 70-gene signature for breast cancer patients, is being evaluated in a randomised clinical trial. The early, controlled implementation process is supported by a Constructive Technology Assessment to help decision-making in an uncertain time of development

Constructive technology assessment of gene expression profiling for breast cancer

Constructive Technology Assessment (CTA) can be used as a complementary\ud approach to Health Technology Assessment (HTA), especially for the early and\ud dynamic introduction of new technologies in a controlled way. CTA is based on the\ud idea that during the course of technology development, choices are constantly\ud being made about the form, the function, and the use of that technology. In this\ud dissertation the mixed method approach of CTA covers an integral assessment of\ud clinical, economic, patient-related, ethical/juridical, and organizational domains.\ud Diffusion scenarios, which are commonly applied in industry to anticipate on their\ud strategies concerning future development, have been adapted to monitor the\ud dynamics in this study.\ud The aim of this dissertation was to contribute to the knowledge on early stage HTA\ud by performing a CTA for the introduction and diffusion of gene expression profiling\ud for breast cancer patients. As a clinical case, the introduction and diffusion of the\ud 70-gene prognosis signature (MammaPrintTM) using microarray analysis was\ud evaluated. The research objectives were twofold: first to develop the CTA method\ud in early stages of technology development and second, to apply the CTA method\ud to the case of the 70-gene signature for breast cancer, in order to support and\ud anticipate on the introduction of this new diagnostic test, specified in different CTA\ud aspects.\ud This study showed that the CTA methodology can be a useful tool to guide\ud controlled early implementation of a promising technology and its possible use for\ud coverage decisions, in this case the 70-gene signature for breast cancer patients.\ud The patient information regarding the 70-gene signature appeared to be clear and\ud satisfactory and resulted in a good understanding of (the consequences of) the\ud genomic profile. In general, the 70-gene signature seems most cost-effective in\ud terms of quality adjusted life years; the slightly more sensitive tests deliver more life\ud years, but leads to a substantial larger amount of adjuvant chemotherapy and\ud hence higher costs, thus demanding a higher willingness to pay. Developing the\ud 70-gene signature based on paraffin instead of fresh frozen tissue could establish\ud a higher cost-effectiveness and could thus be a worthwhile investment. Finally,\ud when incorporating scenarios in the decision model, it became apparent that early\ud anticipation on certain aspects is necessary to reach the potential costeffectiveness

Close cooperation with Health Technology Assessment expertise is crucial for implementation and ultimately reimbursement of innovations in oncology

The Organisation of European Cancer Institutes OECI working group on Health Economics and Cost Benefit in Oncology suggests four actions that are needed to improve alignment and integration between clinicians, researchers, and Health Technology Assessment (HTA) experts and agencies: 1) HTA expertise is necessary close to or within the comprehensive cancer centres (CCC); 2) HTA expertise should be physically present throughout the translational research process; 3) Appropriate knowledge is necessary within the research staff; 4) Close cooperation between translational researchers, clinicians, and health economists guarantees clinical ownership. Fulfilling these conditions may help the translational research field in oncology to interact with agencies and efficiently move innovative technologies through the translational research stages into that of implementation and diffusion. This brings innovative treatments faster to the patient with a greater chance of reimbursement

Relevant factors for policy concerning comprehensive genomic profiling in oncology:stakeholder perspectives

Background: Comprehensive genomic profiling (CGP) can identify targets beyond standard of care, potentially revolutionizing personalized cancer management. However, conducting well designed studies in this rapidly evolving field is complex and demands time and investments. Consequently, the total added value of CGP remains uncertain. Clinical benefit and costs often are driving factors in coverage decisions. Recently, five additional factors were identified in the literature that can influence the choice for targeted profiling vs. CGP, specifically: “feasibility”, “test journey patient/physician”, “wider implications of diagnostic results”, “organization of laboratories”, and “scientific spillover”. The objective of the current study is to examine the role and importance assigned to these five additional factors for a comprehensive technology assessment by different stakeholders.Methods: Purposive sampling was used to identify respondents from 4 stakeholder groups (i.e., medical specialists, molecular specialists, patient representatives, and policymakers) from different regions and hospital types (academic vs. non-academic) in the Netherlands. In semi-structured interviews, respondents scored the importance to decision-making of the five factors on a 0 (not important) to 5 (essential) scale. Reasoning behind the scores were elicited using open-ended follow-up questions. Transcripts were independently double-coded by two researchers using thematic analysis.Results: Nineteen stakeholders (100% response rate; medical specialists (n = 7), molecular specialists (n = 7), patient representatives (n = 2), and policymakers (n = 3)) were interviewed. We observed differences between stakeholders in the relative importance assigned to the factors (range of median importance scores: 2–5). Overall, “wider implications of diagnostic results”, primarily CGP’s potential to identify additional treatment options, was deemed the most important factor alongside clinical benefit and costs in decision-making about CGP (median range: 3–5). While the “organization of laboratories” was considered less important (median range: 3–4), opposing arguments and preferences regarding the organization of laboratories were identified, with participants from academic centers preferring a centralized approach whilst non-academics preferred a decentralized approach.Conclusions: Stakeholders deemed “wider implications of diagnostic results”, “feasibility”, and “test journey” the most important considerations for decision-making about targeted profiling vs. CGP alongside clinical benefit and costs. For policy decision-making, it is important to understand the arguments behind the heterogeneous opinions, often related to the setting they originate from.</p