Cancer treatments touch a wide range of values that count for patients and other stakeholders: What are the implications for decision-making?

BACKGROUND: Cancer rates and expenditures are increasing, resulting in debates on the exact value of this care. Perspectives on what exactly constitutes worthwhile values differ. This study aims to explore all values-elements regarding new oncological treatments for patients with cancer and all stakeholders involved and to assess their implications in different decision-making procedures. METHOD: Thirty-one individual in-depth interviews were conducted with different stakeholders to identify values within oncology. A focus group with seven experts was performed to explore its possible implications in decision-making procedures. RESULTS: The overarching themes of values identified were impact on daily life and future, costs for patients and loved ones, quality of life, impact on loved ones, societal impact and quality of treatments. The expert panel revealed that the extended exploration of values that matter to patients is deemed useful in patient-level decision-making, information provision, patient empowerment and support during and after treatment. For national reimbursement decisions, implications for the broad range of values seems less clear. CONCLUSION: Clinical values are not the only ones that matter to oncological patients and the stakeholders in the field. We found a much broader range of values. Proper recognition of values that count might add to patient-level decision-making, but implications for reimbursement decisions are less clear. The results could be useful to guide clinicians and policymakers when it comes to decision-making in oncology. Making more explicit which values counts for whom guarantees a more systematic approach to decision-making on all levels.

Are CAR-T therapies living up to their hype? A study using real-world data in two cohorts to determine how well they are actually working in practice compared with bone marrow transplants.

With the increasing use of new regulatory tools, like the Food and Drug Administration's breakthrough designation, there are increasing challenges for European health technology assessors (HTAs) to make an accurate assessment of the long-term value and performance of chimeric antigen receptor T-cell (CAR-T) therapies, particularly for orphan conditions, such as acute lymphoblastic leukaemia. The aim of this study was to demonstrate a novel methodology harnessing longitudinal real-world data, extracted from the electronic health records of a medical centre functioning as a clinical trial site, to develop an accurate analysis of the performance of CAR-T compared with the next-best treatment option, namely allogeneic haematopoietic cell transplant (HCT). The study population comprised 43 subjects in two cohorts: 29 who had undergone HCT treatment and 14 who had undergone CAR-T therapy. The 3-year relapse-free survival probability was 46% (95% CI: 08% to 79%) in the CAR-T cohort and 68% (95% CI: 46% to 83%) in the HCT cohort. To explain the lower RFS probability in the CAR-T cohort compared with the HCT cohort, the authors hypothesised that the CAR-T cohort had a far higher level of disease burden. This was validated by log-rank test analysis (p=0.0001) and confirmed in conversations with practitioners at the study site. The authors are aware that the small populations in this study will be seen as limiting the generalisability of the findings to some readers. However, in consultation with many European HTAs and regulators, there is broad agreement that this methodology warrants further investigation with a larger study.

Addressing the dichotomy between individual and societal approaches to personalised medicine in oncology.

Academic, industry, regulatory leaders and patient advocates in cancer clinical research met in November 2018 at the Innovation and Biomarkers in Cancer Drug Development meeting in Brussels to address the existing dichotomy between increasing calls for personalised oncology approaches based on individual molecular profiles and the need to make resource and regulatory decisions at the societal level in differing health-care delivery systems around the globe. Novel clinical trial designs, the utility and limitations of real-world evidence (RWE) and emerging technologies for profiling patient tumours and tumour-derived DNA in plasma were discussed. While randomised clinical trials remain the gold standard approach to defining clinical utility of local and systemic therapeutic interventions, the broader adoption of comprehensive tumour profiling and novel trial designs coupled with RWE may allow patient and physician autonomy to be appropriately balanced with broader assessments of safety and overall societal benefit.

Social media as a tool for assessing patient perspectives on quality of life in metastatic melanoma: a feasibility study.

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.

Use of Social Media in the Assessment of Relative Effectiveness: Explorative Review With Examples From Oncology.

BACKGROUND: An element of health technology assessment constitutes assessing the clinical effectiveness of drugs, generally called relative effectiveness assessment. Little real-world evidence is available directly after market access, therefore randomized controlled trials are used to obtain information for relative effectiveness assessment. However, there is growing interest in using real-world data for relative effectiveness assessment. Social media may provide a source of real-world data. OBJECTIVE: We assessed the extent to which social media-generated health data has provided insights for relative effectiveness assessment. METHODS: An explorative literature review was conducted following the Preferred Reporting Items for Systematic Reviews and Meta-Analyses guidelines to identify examples in oncology where health data were collected using social media. Scientific and grey literature published between January 2010 and June 2016 was identified by four reviewers, who independently screened studies for eligibility and extracted data. A descriptive qualitative analysis was performed. RESULTS: Of 1032 articles identified, eight were included: four articles identified adverse events in response to cancer treatment, three articles disseminated quality of life surveys, and one study assessed the occurrence of disease-specific symptoms. Several strengths of social media-generated health data were highlighted in the articles, such as efficient collection of patient experiences and recruiting patients with rare diseases. Conversely, limitations included validation of authenticity and presence of information and selection bias. CONCLUSIONS: Social media may provide a potential source of real-world data for relative effectiveness assessment, particularly on aspects such as adverse events, symptom occurrence, quality of life, and adherence behavior. This potential has not yet been fully realized and the degree of usefulness for relative effectiveness assessment should be further explored.

Using Real-World Data in Health Technology Assessment (HTA) Practice: A Comparative Study of Five HTA Agencies.

BACKGROUND: Reimbursement decisions are conventionally based on evidence from randomised controlled trials (RCTs), which often have high internal validity but low external validity. Real-world data (RWD) may provide complimentary evidence for relative effectiveness assessments (REAs) and cost-effectiveness assessments (CEAs). This study examines whether RWD is incorporated in health technology assessment (HTA) of melanoma drugs by European HTA agencies, as well as differences in RWD use between agencies and across time. METHODS: HTA reports published between 1 January 2011 and 31 December 2016 were retrieved from websites of agencies representing five jurisdictions: England [National Institute for Health and Care Excellence (NICE)], Scotland [Scottish Medicines Consortium (SMC)], France [Haute Autorité de santé (HAS)], Germany [Institute for Quality and Efficacy in Healthcare (IQWiG)] and The Netherlands [Zorginstituut Nederland (ZIN)]. A standardized data extraction form was used to extract information on RWD inclusion for both REAs and CEAs. RESULTS: Overall, 52 reports were retrieved, all of which contained REAs; CEAs were present in 25 of the reports. RWD was included in 28 of the 52 REAs (54%), mainly to estimate melanoma prevalence, and in 22 of the 25 (88%) CEAs, mainly to extrapolate long-term effectiveness and/or identify drug-related costs. Differences emerged between agencies regarding RWD use in REAs; the ZIN and IQWiG cited RWD for evidence on prevalence, whereas the NICE, SMC and HAS additionally cited RWD use for drug effectiveness. No visible trend for RWD use in REAs and CEAs over time was observed. CONCLUSION: In general, RWD inclusion was higher in CEAs than REAs, and was mostly used to estimate melanoma prevalence in REAs or to predict long-term effectiveness in CEAs. Differences emerged between agencies' use of RWD; however, no visible trends for RWD use over time were observed.