Chapter 6 112 DISCUSSION In this review, we combined automated documentation searches with double, independent manual review to screen NICE documentation on the usage of expanded access data for HTA. We have found that data from expanded access programs are frequently included: 21.1% of the TAs used expanded access data to evaluate safety, efficacy/effectiveness, or resource use of the appraised technology. The use of data from expanded access programs appears to remain stable over the years. Additionally, patients and physicians share their treatment experience from an expanded access program in 14.2% of the appraisals. The disease areas of the appraisals that included expanded access data differed significantly from the overall distribution of disease areas from all appraisals investigated between 2010 and 2021. Oncology and hematology account for the lion’s share (66%) of expanded access data usage, yet account for half (50%) of all TAs conducted. Although ‘the life-threatening or seriously debilitating’ prerequisite for expanded access is often present in hemato-oncologic malignancies, cardiac or ophthalmologic illnesses can also be severely limiting.55,56 Cardiology and ophthalmology account for 8.4% of all TAs, but none (0%) of these programs used expanded access data (or even mentioned it). There is a range of possible explanations for this discrepancy. Perhaps, drug developers in these areas may be less familiar with collecting and using expanded access data, or cardiologists and ophthalmologists may be less acquainted with expanded access than haemato-oncologists – simply because expanded access may be less warranted in these disease areas.57,58 Compared with regulatory submissions to the EMA and the FDA, submissions to NICE more frequently include expanded access data. The EMA and FDA used expanded access data to support efficacy in 49 regulatory approvals over 25 years (± 2 annually).50 In this work, we find that NICE used expanded access to inform cost-effectiveness in 76 over 11 years (± 7 annually). One reason for this may be that payers have a higher uptake of RWD in their decision making. Furthermore, they also assess comparative effectiveness rather than efficacy. Modelling cost and comparative effectiveness by definition necessitates a variety of input parameters, every one of them potentially coming from different sources, such as expanded access. Whether using expanded access data (or other non-randomized data) for payer decision making is wise, depends in part on the robust design and execution of the expanded access program, and the relevance to the decision problem.59 The instances in which the FDA and the EMA assessed efficacy mainly based on expanded access data, are scarce, and characterized by (i) a high unmet medical need (ii) a rare disease population and (iii) large treatment effects.50 Additionally, we witnessed twice (TA391, TA491) that health-related quality-of-life (HRQoL) data were not
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