Real-world data from expanded access programmes in health technology assessments: a review of NICE technology appraisals 109 6♦ patients with advanced or metastatic NSCLC. The majority of ADRs associated with gefitinib are mild in nature and those most commonly reported are grade 1/2 diarrhoea and skin reactions.’ Manufacturer submission, Safety and tolerability, TA192 Alternatively, safety signals from expanded access programs can be quantitatively incorporated in cost-effectiveness analyses. When evaluating ocrelizumab for treating relapsing–remitting multiple sclerosis, the committee noted that an important safety signal from the compassionate use program is lacking from the current analysis: ‘The committee heard that there has been the 1 case of PML (progressive multifocal leukoencephalopathy, red.) following treatment with ocrelizumab in the compassionate-use programme in Germany, (…). It concluded that the economic model should have included a risk of PML for ocrelizumab.’ Appraisal consultation, Adverse events in the economic model, TA533 Efficacy Efficacy data from expanded access programs can also be used, together with data from clinical trials, to estimate overall efficacy of the technology appraised. In the evaluation of lutetium (177Lu) oxodotreotide for treating irresectable or metastatic neuroendocrine tumors, response rates were obtained from the ‘Erasmus study’. The Erasmus study was a compassionate use program conducted at the Erasmus MC. The data from this program are summarized as: ‘In a single centre non-controlled phase I/II open-label study (The Erasmus study, red.), conducted in 810 Dutch patients with different somatostatin receptor positive tumour types, the objective response rate (ORR) for the full analysis set (FAS) population with GEP-NETs and bronchial NETs (360 patients) was 44% (95% confidence interval [CI] 38% - 49%).’ Manufacturer submission, Executive summary, TA539 NICE requires that benefits of technologies are evaluated using quality-adjusted life years (QALYs), as NICE’s decision to recommend or not recommend a product for reimbursement depends (among other things) on the willingness-to-pay for an incremental year in perfect health – the socalled cost-per-QALY approach. In the evaluation of cabazitaxel for hormone-relapsed metastatic prostate cancer treated with docetaxel, the expanded access program was used to gather quality of life data not collected during the routine clinical development:
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