The impact of quality-of-life data in relative effectiveness assessments of new anti-cancer drugs in European countries

Kleijnen, Sarah; Alves, Teresa Meneses Leonardo; Meijboom, Kim; Lipska, Iga; Boer, Anthonius de; Leufkens, H.G.M.; Goettsch, Wim

doi:10.1007/s11136-017-1574-9

Cited by 24 publications

(19 citation statements)

References 21 publications

(35 reference statements)

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“…To compare and evaluate HTA practices, many studies exist that investigate HTA recommendations for groups of medicines. [2][3][4][5][6][7][8][9][10][11] They differ in the extent to which they include practice-related factors as explanatory for differences between HTA jurisdictions. Based on our results, it is recommended to take a systematic approach to investigating practice differences between HTA jurisdictions.…”

Section: Discussionmentioning

confidence: 99%

“…Consequently, differences in HTA recommendations have been the subject of extensive research. [2][3][4][5][6][7][8][9][10][11][12][13][14][15][16][17][18][19] Studies qualitatively assessing recommendations for groups of drugs in several HTA jurisdictions report that a series of sources contribute to observed differences. These sources can be roughly divided into two categories.…”

Section: Introductionmentioning

confidence: 99%

“…Second, even when jurisdictions apply the same practice (eg, a cost-effectiveness assessment [CEA]), there may still be differences in HTA outcomes, stemming from differences in the inclusion and evaluation of evidence, the impact of evidence on the final recommendation, and the integral leveraging of different types of evidence. [2][3][4][5][6]8 These can be classified as value assessment differences. Within HTA practices, a nonsubmission means that an HTA body provides a recommendation on reimbursement, even though no submission has been provided by the relevant company.…”

Section: Introductionmentioning

confidence: 99%

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Differences in Health Technology Assessment Recommendations Among European Jurisdictions: The Role of Practice Variations

et al. 2020

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Background: Health technology assessment (HTA) plays an important role in reimbursement decision-making in many countries, but recommendations vary widely throughout jurisdictions, even for the same drug. This variation may be due to differences in the weighing of evidence or differences in the processes or procedures, which are known as HTA practices. Objective: To provide insight into the effects of differences in practices on interpretation of intercountry differences in HTA recommendations for conditionally approved drugs. Methods: HTA recommendations for conditionally approved drugs (N = 27) up until June 2017 from England/Wales, France, Germany, the Netherlands, and Scotland were included. Recommendations and practice characteristics were extracted from these five jurisdictions and this data was validated. The effect of nonsubmissions, resubmissions, and reassessments; costeffectiveness assessments; and price negotiations on changes in the percentage of negative recommendations and the interpretation of intercountry differences in HTA outcomes were analyzed using Fisher exact tests. Results: The inclusion of cost-effectiveness assessments led to significant increases in the proportion of negative recommendations in England/Wales (from 4% to 50%, P,.01) and Scotland (from 21% to 71%, P,.01). The subsequent inclusion of price negotiations led to significant reductions in the proportion of negative recommendations in England/ Wales (from 50% to 14%, P,.01), France (from 31% to 3%, P=.012), and Germany (from 34% to 0%, P,.01). Results indicated that the inclusion of nonsubmissions and resubmissions might affect Scottish negative HTA recommendations (from 7% to 21%), but this effect was not significant. No significant effects were observed in the Netherlands, possibly owing to sample size. Conclusion: Variations in HTA practices between international jurisdictions can have a substantial and significant impact on conclusions about recommendations by HTA bodies, as exemplified in this cohort of conditionally approved products. Studies comparing international HTA recommendations should carefully consider possible practice variations between jurisdictions.

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Section: Discussionmentioning

confidence: 99%

Section: Introductionmentioning

confidence: 99%

Section: Introductionmentioning

confidence: 99%

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Differences in Health Technology Assessment Recommendations Among European Jurisdictions: The Role of Practice Variations

et al. 2020

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“…While national health technology assessment guidelines within the European Union recognize the relevance of quality of life (QoL) to determine the relative effectiveness of new therapies, it is still a factor poorly reflected during the actual assessment [ 8 ]. This is mainly due to methodological concerns relating to the collection and quality of these QoL data.…”

Section: Introductionmentioning

confidence: 99%

Psychosocial aspects and life project disruption in young women diagnosed with metastatic hormone-sensitive HER2-negative breast cancer

et al. 2020

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Metastatic breast cancer (MBC) diagnosis in young women negatively impacts on quality of life (QoL) and daily activities, disrupting their life project and forcing them to face new psychosocial challenges. The recently published results on the improvement of the overall survival of pre- or perimenopausal women with hormone-receptor-positive, HER2-negative MBC treated with CDK4/6 inhibitors plus endocrine therapy, while preserving, and in some items improving their QoL, will change the landscape of the management of this patient population. Their extended survival and potential improvement in QoL will, therefore, modify their specific needs in terms of psychosocial support. The complexity of the care of young women with MBC is described herein, based on an extensive literature review. Further research about the specific psychosocial requirements of these women and a new multidisciplinary holistic approach is paramount to properly address their concerns and preferences. The communication with and support of their partners, parents and children is an important factor affecting the QoL of these patients. Altogether, a multidisciplinary care, open communication and personalized support is required to address the psychosocial implications of the new prognostic expectations on these patients with the incorporation of new targeted therapies.

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“…In this scenario, RWD can act as a historical control for singlearm studies and provide a natural course of disease data (3). Third, the main focus of many oncology RCTs is survival benefit with the endpoints of overall survival (OS) or progression-free survival (PFS), which do not provide accurate long-term safety or quality of life data; RWD can be tailored to satisfy this informational shortfall (6).…”

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confidence: 99%

The essence of real-world study in cancer research

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Shi²,

Fan³

et al. 2020

Transl Breast Cancer Res

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BackgroundDespite the rapid development of gene engineering and other novel therapies, advanced cancer is still a largely intractable disease, and its high mortality necessitates the development of better treatment (1). In general, the regulatory approval of new cancer treatment is built on the objective criteria of efficacy and safety derived from randomized controlled trials (RCTs). However, with advances in genetic and molecular treatment, the number of patients available for a specific clinical trial may be too small for proper assessment of a benefit-risk paradigm (2). As a result, drug developers and regulators have become increasingly interested in data external from clinical trials, and there is growing need to rethink the traditional approaches to treatment development and approval (3). One potential solution to this may be to further extend and validate the results from RCTs with the wider experience of real-world data (RWD) or real-world evidence (RWE).Compared to the rigorous admission criteria and strict research conditions of RCTs, RWD is collected from the real world to provide evidence for clinical and policyrelevant questions that cannot be addressed with data from RCTs, such as patient health status, cancer incidence and mortality, quality of and access to care delivered in routine practice, rare cancers, and rare adverse events and toxicities

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The impact of quality-of-life data in relative effectiveness assessments of new anti-cancer drugs in European countries

Cited by 24 publications

References 21 publications

Differences in Health Technology Assessment Recommendations Among European Jurisdictions: The Role of Practice Variations

Differences in Health Technology Assessment Recommendations Among European Jurisdictions: The Role of Practice Variations

Psychosocial aspects and life project disruption in young women diagnosed with metastatic hormone-sensitive HER2-negative breast cancer

The essence of real-world study in cancer research

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