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

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

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

Differences in time to patient access to innovative cancer medicines in six European countries

Patients across Europe face inequity regarding access to anticancer medicines. While access is typically evaluated through reimbursement status or sales data, patients can receive first access through early access programs (EAPs) or off-label use. This study aims to assess the time to patient access at the hospital level, considering different indications and countries. (Pre-)registered access to six innovative medicines (Olaparib, Niraparib, Ipilimumab, Osimeritinib, Nivolumab and Ibritunib) was measured using a cross-sectional survey. First patient access to medicines and indications were collected using the hospital databases. Nineteen hospitals from Hungary, Italy, the Netherlands, Belgium, Switzerland and France participated. Analysis showed that some hospitals achieved patient access before national reimbursement, primarily through EAPs. The average time from EMA-approval to patient access for these medicines was 2.1 years (Range: −0.9-7.1 years). Hospitals in Italy and France had faster access compared to Hungary and Belgium. Variation was also found within countries, with specialized hospitals (x̄: −0.9 years; SD: 2.0) more likely to provide patient access prior to national reimbursement than general hospitals (x̄: 0.4 years; SD: 2.9). Contextual differences were observed, with EAPs or off-label use being more prevalent in Switzerland than Hungary. Recent EMA-approved indications and drug combinations reached patients at a later stage. Substantial variation in patient access time was observed between and within countries. Improving pricing and reimbursement timelines, fostering collaboration between national health authorities and market authorization holders, and implementing nationally harmonized, data-generating EAPs can enhance timely and equitable patient access to innovative cancer treatments in Europe.</p