Real-world data from expanded access programmes in health technology assessments: a review of NICE technology appraisals 103 6♦ INTRODUCTION Novel drug therapies are important drivers of increased health care spending. In the United Kingdom (UK), the National Institute for Health and Care Excellence (NICE) conducts technology appraisals (TAs) to evaluate cost-effectiveness of technologies (e.g., drugs, medical devices) and to determine their impact on health care budgets.44 These evaluations are conducted using a variety of data sources, such as randomized controlled trials (RCTs) or observational studies.45,46 In this research, we explore the use of data in NICE TAs from another source: expanded access programs. A positive appraisal determination from NICE forms the main pathway for novel pharmaceutical technologies to access the National Health Service (NHS) and become available for patients across the UK. The health technology assessment (HTA) usually starts with the submission of evidence on clinical effectiveness and costs by the pharmaceutical company. The submission is scrutinized by an independent Evidence Review Group (ERG), which critically reviews the manufacturer’s submission and performs additional exploratory analyses of cost-effectiveness; in some cases, the ERG even re-analyses clinical data.44,47,48 Patients, patient advocacy groups, and physicians working within the NHS also contribute to NICE’s appraisals. The resulting qualitative input is considered in the formal analyses conducted by the manufacturer and the ERG. The entire evidence is assessed by NICE’s Appraisal Committee and forms the basis of their appraisal determination.49 More detailed information on NICE’s processes can be found on their guidance website (https://www.nice.org.uk/about/what-we-do/ our-programmes/nice-guidance). HTA bodies are particularly keen to know how technologies will use resources, yield benefit, and attribute risks in the real-world patient population for which treatment will potentially be reimbursed.10 Real-World Data (RWD) are ‘information on health care that is derived from multiple sources outside typical clinical research settings’, such as electronic health records, claims and billing databases, or patient registries.34 RWD is typically generated after a drug comes to market (post-approval). At the time of the reimbursement decision however, most of the available data stems from clinical trials (pre-approval). Noteworthy, payers may use (real-world) data from patients that have been treated outside of clinical trial settings, but prior to marketing authorization.44–46 These patients can receive treatment via expanded access programs. Expanded access (EA) is a pathway to access investigational medicine for patients who suffer from life-threatening conditions, who cannot enter clinical trials, and have exhausted all approved treatment options. It is also known as ‘compassionate use’, ‘early access’ or ‘non-trial pre-approval
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