MS08.01 How Can Real World Data Improve Clinical Evidence Generation and Impact Regulatory Bodies - European Perspective

Abstract

Health technology assessment (HTA) evaluates the efficacy and effectiveness of new interventions, integrates these data with the costs to define efficiency and addresses future availability and distribution. As such, HTA focuses on accessibility, affordability and equity. Economic evaluations are central in this concept: weighing costs and effects, they support evidence-based decisions on reimbursement, thus endorsing the introduction of innovative healthcare interventions in daily practice. After more than 20 years of voluntary cooperation on HTA in Europe, more than 50 HTA bodies are currently operating in the European Union (EU), be it still fragmented with different systems, different procedures and different requirements regarding the type of clinical evidence. Efficacy, the outcome a new intervention provides in the well-defined circumstances of a randomised controlled trial (RCT) and typically the input used to derive cost-effectiveness evidence, may not provide the best insight into the impact of an intervention in daily clinical care. Moreover, in contrast to what is the case for pharmaceuticals, it is much more difficult to perform RCTs on radiotherapy or surgery, especially when it comes to evaluating the incremental evolution typical for new techniques and technologies, or the long-term benefits anticipated to follow more accurate treatment delivery. This has resulted in different regulatory systems for systemic and non-systemic treatment strategies, with attempts to come to a more homogenised HTA approach in the EU having so far failed. Real-world data are gradually expanding their role in the evidence generation of lung cancer radiotherapy. A first step moving away from RCTs is to adopt a more pragmatic approach to evidence generation, as is the case in the OligoCare project. This joint ESTRO-EORTC initiative is evaluating the outcome of oligometastatic patients, amongst others from primary lung cancer, treated with radical radiotherapy in a large prospective cohort study. Various patterns of care studies have generated clinical evidence on the uptake of new treatment techniques such as SBRT (Stereotactic Body Radiotherapy), on the value of multimodality treatments for locally-advanced non-small cell lung cancer (NSCLC) beyond the context of RCTs, or have allowed to develop prediction models to support shared decision-making. Furthermore, the wealth of data available in cancer registries and other nation-wide databases can be leveraged to learn more about the quality of care, actual access to different treatment strategies, geographical and institutional variations and their potential impact on lung cancer survival. Real-life data can also be used to generate the cost information necessary to perform cost-effectiveness evaluations. Besides the more frequent approach to derive this evidence from reimbursement data, actual resource costs can also be computed in daily practice. One such initiative was undertaken in Belgium, using Time-Driven Activity-Based Costing to compute real-life costs of radiotherapy, and more specifically of innovative radiotherapy techniques such as SBRT. Whereas all these examples provide interesting insight into the clinical and financial consequences of access to standard-of-care and innovative lung cancer radiotherapy, there is a dearth of information on how this evidence defines policy. One interesting approach to change practice is using coverage with evidence generation to provide early access to radiotherapy innovations, while stimulating the further generation of data. Such a programme of provisional financing has been set-up in Belgium to generate evidence from daily practice on SBRT for primary tumours, most typically early-stage NSCLC, and for oligometastatic disease. While this programme allowed radiation oncology centres to develop and provide SBRT without being financially penalised, it also generated the reassuring clinical evidence that will soon lead to the inclusion of SBRT in the formal national radiotherapy reimbursement system. Keywords: Effectiveness, costs, real-world data