PurposeThe aim of this study is to investigate the role of health-related quality-of-life (QoL) data in relative effectiveness assessments (REAs) of new anti-cancer drugs across European jurisdictions, during health technology assessment procedures.MethodsComparative analysis of guidelines and publicly available REAs in six European jurisdictions of anti-cancer drugs approved by EMA between 2011 and 2013.ResultsFourteen anti-cancer drugs were included, adding up to 79 REAs. Whilst all guidelines state that QoL is a relevant endpoint to determine the relative effectiveness of new cancer drugs, QoL data were included in only 54% of the 79 reports and their impact on the recommendations was limited.ConclusionsWhilst national guidelines recognize the relevance of QoL to determine the relative effectiveness of new anti-cancer drugs, this is not well-reflected in current assessments. Developing and implementing into REAs specific evidence requirements for QoL data would improve the use of this patient-centred outcome in future reimbursement and pricing decisions.Electronic supplementary materialThe online version of this article (doi:10.1007/s11136-017-1574-9) contains supplementary material, which is available to authorized users.
BackgroundAn 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.ObjectiveWe assessed the extent to which social media-generated health data has provided insights for relative effectiveness assessment.MethodsAn 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.ResultsOf 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.ConclusionsSocial 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.
PROs were also presented in the HTA submissions of 8 drugs with no EMA PRO data. In HTA submissions, PROs assessing QoL (51/57, 89.4%) and pain (18/57, 31.6%) were the most common. Statistical significance was observed in 59.6% of the 52 submissions with available results. In the final HTA decision, PROs were mentioned for 43.9% of submissions, not mentioned in 35.1% and were not reviewed by the HTA body in 21.0% of submissions due mainly to methodological issues. ConClusions: Demonstrating a statistically significant improvement in PROs does not increase the chance of a positive HTA recommendation (65.5% vs 74.1% for submissions without PROs). The value that PRO claims in oncology have to payers needs to be further clarified.
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.
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