Chapter 6 110 ‘The company did not collect data on health-related quality of life in TROPIC (the RCT, red.), so it took utility values from the UK Early Access Programme (EAP) for cabazitaxel. The programme measured the health-related quality of life (using the EQ-5D) of men who had been treated with cabazitaxel after docetaxel.(…)’ ‘(…) One hundred and twelve patients participated in the UK EAP at 12 UK Cancer Centres. All had mCRPC with disease progression during or after docetaxel and were similar in baseline patient characteristics to the population in TROPIC. (…) Safety assessments were performed prior to each cycle and HRQL recorded at alternate cycles using the EQ-5D-3L questionnaire and visual analogue scale (VAS).’ Committee papers, Health-related quality of life, TA391 Resource use Expanded access data can also be used to inform other parameters in cost-effectiveness modelling. Such models are often based on Markov chains, which describe the state of the disease that patients are in at a given time point. These models require cost per state and transition probabilities or rates between states. Registries, or other real-world data sources, are frequently used to estimate such data. In the appraisal of sofosbuvir-velpatasvir-voxilaprevir for treating chronic hepatitis C, transition probabilities from decompensated liver cirrhosis to death are modelled via a Beta-distribution and the input parameters are provided from the expanded access program: ‘Variable: From decompensated cirrhosis to death Distribution and parameters: Beta; α=46.5; ꞵ=147.2 Source: EAP data (expanded access program, red.)’ Manufacturer submission, Sensitivity analyses, TA507 A different, direct resource use example is given in the evaluation of ipilimumab for previously treated irresectable malignant melanoma. The dosing of ipilimumab is weight dependent. Hence, to estimate the number of vials needed for treatment of UK patients, an estimate of the (UK) patient population weight is required. This weight is calculated via: ‘Patient level analysis of the weight of UK clinical trial patients in MDX010-20 (n=55), and the weight of UK patients in the ipilimumab compassionate use program (n=258), from these weights, the mean number of vials required (assuming no vial sharing) is calculated.’ ‘Results from these analyses showed that the dose of ipilimumab given per patient per induction has a large impact on the ICER with the minimum dose given in the trial and compassionate use programme (3 x 50 mg) resulting in an ICER of £38,387 per QALY gained
RkJQdWJsaXNoZXIy MTk4NDMw