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Abstract Purpose Development of innovative drugs for melanoma is occurring rapidly. Incremental gains in overall survival amongst innovative products may be difficult to measure in clinical trials, and their use may be associated with increased toxicity profiles. Therefore, HTA agencies increasingly require information on HRQoL for the assessment of such drugs. This study explored the feasibility of social media to assess patient perspectives on HRQoL in melanoma, and whether current cancer- and melanoma-specific HRQoL questionnaires represent these perspectives. Methods A survey was distributed on the social media channels of Melanoma Patient Network Europe to assess melanoma patients’ perspectives regarding HRQoL. Two researchers independently conducted content analysis to identify key themes, which were subsequently compared to questions from one current cancer-specific and two melanoma-specific HRQoL questionnaires (i.e. EORTC QLQ-C30, EORTC QLQ-MEL38, FACT-M). Results In total, 72 patients and 17 carers completed the survey. Patients indicated that family, having a normal life, and enjoying life were the three most important aspects of HRQoL for them. Carers indicated that being capable, having manageable adverse events, and being pain-free were the three most important aspects of HRQoL for patients. Respondents seem to find some questions from HRQoL questionnaires relevant (e.g. ‘Have you felt able to carry on with things as normal?’) and others less relevant (e.g. ‘Have you had swelling near your melanoma site?’). Additionally, wording may differ between patients and HRQoL questionnaires, whereby patients generally use a more positive tone. Conclusions Social media may provide a valuable tool in assessing patient perspectives regarding HRQoL. However, differences seem to emerge between patient and carer perspectives. Additionally, patient perspectives did not seem to fully correlate to questions posed in cancer- (i.e. EORTC QLQ-C30) and melanoma-specific (i.e. EORTC QLQ-MEL38, FACT-M) HRQoL questionnaires examined.

Abstract Standard outcome sets developed by the International Consortium for Health Outcomes Measurement (ICHOM) facilitate value‐based health care in healthcare practice and have gained traction from regulators and Health Technology Assessment (HTA) agencies that regularly assess the value of new medicines. We aimed to assess the extent to which the outcomes used by regulators and HTA agencies are patient‐relevant, by comparing these to ICHOM standard sets. We conducted a cross‐sectional comparative analysis of ICHOM standard sets, and publicly available regulatory and HTA assessment guidelines. We focused on oncology due to many new medicines being developed, which are accompanied by substantial uncertainty regarding the relevance of these treatments for patients. A comparison of regulatory and HTA assessment guidelines, and ICHOM standard sets showed that both ICHOM and regulators stress the importance of disease‐specific outcomes. On the other hand, HTA agencies have a stronger focus on generic outcomes in order to allow comparisons across disease areas. Overall, similar outcomes are relevant for market access, reimbursement, and in ICHOM standard sets. However, some differences are apparent, such as the acceptability of intermediate outcomes. These are recommended in ICHOM standard sets, but regulators are more likely to accept intermediate outcomes than HTA agencies. A greater level of alignment in outcomes accepted may enhance the efficiency of regulatory and HTA processes, and increase timely access to new medicines. ICHOM standard sets may help align these outcomes. However, some differences in outcomes used may remain due to the different purposes of regulatory and HTA decision‐making.

BackgroundThere is a general agreement on the importance of health-related quality of life (HRQoL). This type of information is becoming increasingly important for the value assessment of health technology assessment agencies in evaluating the benefits of new health technologies, including medicines. However, HRQoL data are often limited, and additional sources that provide this type of information may be helpful. ObjectiveWe aim to identify the HRQoL topics important to patients with melanoma based on web-based discussions on public social media forums. MethodsWe identified 3 public web-based forums from the United States and the United Kingdom, namely the Melanoma Patient Information Page, the Melanoma International Forum, and MacMillan. Their posts were randomly selected and coded using qualitative methods until saturation was reached. ResultsOf the posts assessed, 36.7% (150/409) of posts on Melanoma International Forum, 45.1% (198/439) on MacMillan, and 35.4% (128/362) on Melanoma Patient Information Page focused on HRQoL. The 2 themes most frequently mentioned were mental health and (un)certainty. The themes were constructed based on underlying and more detailed codes. Codes related to fear, worry and anxiety, uncertainty, and unfavorable effects were the most-often discussed ones. ConclusionsWeb-based forums are a valuable source for identifying relevant HRQoL aspects in patients with a given disease. These aspects could be cross-referenced with existing tools and they might improve the content validity of patient-reported outcome measures, including HRQoL questionnaires. In addition, web-based forums may provide health technology assessment agencies with a more holistic understanding of the external aspects affecting patient HRQoL. These aspects might support the value assessment of new health technologies and could therefore help inform topic prioritization as well as the scoping phase before any value assessment.