The use of Real-World Data (RWD) by Health Technology Assessment (HTA) agencies

Abstract

Abstract Issue There are divergent views between HTA bodies and pharmaceutical industry on the potential for RWD to inform determination of added benefit or value for money of new medicines. However, exploration of use of RWD is now essential as more high-cost innovative therapies come to market with short-term uncontrolled trials. Description of problem Payers are increasingly seeing use of RWD to fill evidence gaps in clinical development programmes, including external control arms or collection of RWD after initial assessment to determine real-life effectiveness or duration of benefit. An international multi-stakeholder initiative, RWE4Decisions, led by the Belgian Payer - RIZIV/INAMI - has explored these issues over the past few years. They have proposed that all stakeholders including industry, clinicians, regulators, patients and methodologists need to be involved to determine what questions can be answered by RWD and ensuring data is of sufficient quality to generate robust real-world evidence (RWE). Alongside this, the IMPACT HTA Project has reviewed use of RWD in specific rare disease case studies. Results Several HTA/Payers have implemented RWD collection schemes, but few have been successful in delivering sufficient evidence to enable re-assessment of treatment value. Italy is an exception, with a national web-based registry system. Other countries prefer to rely on secondary use of health system data and links to clinical registries, but curating and analysing these data can be complex. The case studies show how more recently some countries are overcoming these challenges by entering into more formal agreements with stakeholders to ensure purposive data collection. Lessons There is a need to publish protocols and reports related to RWD collection in an international portal. If possible, dialogues among bodies should happen in advance to align RWD collection requirements and facilitate data analysis.