Relative effectiveness assessments of oncology medicines for pricing and reimbursement decisions in European countries

Kleijnen, Sarah; Lipska, Iga; Alves, Teresa Leonardo; Meijboom, Kim; Elsada, Ahmed; Vervölgyi, Volker; d’Andon, Anne; Timoney, Angela; Leufkens, H.G.M.; Boer, Anthonius de; Goettsch, Wim

doi:10.1093/annonc/mdw233

Cited by 52 publications

(78 citation statements)

References 16 publications

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“…Although drug licensing agencies increasingly rely on surrogate measures to grant marketing authorization, HTA bodies have traditionally preferred patient-relevant endpoints. 12,15 In practice, HTA bodies must make decisions on licensed products even when desirable evidence is unavailable. Our results show that statistically significant benefit from a patient-relevant endpoint does not always confirm a HTA recommendation.…”

Section: Discussionmentioning

confidence: 99%

“…14 Despite these preferences, a study exploring the use of surrogate measures in cost-effectiveness models within HTA agencies found that submitted reports often lacked this evidence, yielding uncertainty surrounding the surrogate measures. 15 The objective of this study was to assess whether the use of surrogate measures in pivotal trials of cancer drugs authorized by the EMA affected funding recommendations made by HTA bodies in England (NICE) and Canada (CADTH). These 2 countries share transparency in their appraisal processes as well as publicly funded healthcare systems.…”

Section: Introductionmentioning

confidence: 99%

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Association Between the Use of Surrogate Measures in Pivotal Trials and Health Technology Assessment Decisions: A Retrospective Analysis of NICE and CADTH Reviews of Cancer Drugs

et al. 2020

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Methods: Cancer drug approvals from 2012 to 2016 were categorized by demonstrating benefit on overall survival (OS), progression-free survival, disease response, or having no comparator. Approvals were analyzed by benefit category and health technology assessment recommendation. The association between benefit (surrogate vs OS) and recommending a drug was examined using descriptive statistics and linear probability models controlling for unmet need, orphan designation, and costeffectiveness.Results: Of 42 cancer indications that NICE recommended, 15 (36%) demonstrated OS benefit. Cancer indications with OS benefit were less likely to receive a recommendation from NICE than those without (P = .04). In linear probability models, availability of OS benefit was no longer associated with a recommendation from NICE (P = .32). Cost-effective cancer drugs had a 55.6% (95% CI: 38.9%-72.3%) higher probability of receiving a recommendation from NICE than those that were not. In Canada, 15 of 37 (41%) cancer indications that were recommended showed OS benefit. There was no detectable association between surrogate measures and CADTH recommendations based on descriptive statistics (P = .62) or in linear probability models (P = .73).Conclusion: When cost-effectiveness was considered, pivotal trial endpoints were not associated with NICE recommendations. Pivotal trial endpoints, unmet need, orphan status, and cost-effectiveness did not explain CADTH recommendations.

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Section: Discussionmentioning

confidence: 99%

Section: Introductionmentioning

confidence: 99%

Association Between the Use of Surrogate Measures in Pivotal Trials and Health Technology Assessment Decisions: A Retrospective Analysis of NICE and CADTH Reviews of Cancer Drugs

et al. 2020

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“…This can also be seen in the Table 1, in which selected countries used the same assessment results based on relative effectiveness (using EUnetHTA Core Model). The diverging results could also be due to the fact that the scope (comparators and cost considerations) and the methodology used vary across countries [47]. Allen et al [48] found similar results in a study on national reimbursement decisions in nine countries for more than 100 new active substances approved by the European Medicines Agency.…”

Section: Comparison On Hta and Decision Outcomes In Different Settingsmentioning

confidence: 98%

“…The CEPS (Comité Économique des Produits de Santé), also a separate body, is responsible for price negotiations with pharmaceutical companies. [27,47] With regard to the use of HTA in decision making, it can be observed that in addition to the level of clinical benefit and cost-effectiveness, increasingly other aspects are taken into account in the appraisal [44]. For orphan drugs different criteria might apply in either the assessment phase (e.g.…”

Section: Comparison On Hta and Decision Outcomes In Different Settingsmentioning

confidence: 99%

“…Other suggestions to help improve future pricing and reimbursement considerations in challenging areas include multi-stakeholder debates to better align the needs for robust evidence requirements, given concerns with surrogate markers, and a collectively shared definition and acceptance on what are clinically relevant benefits for patients and society across disease areas [149]. As a result, help better shape the concepts of value to improve pricing and reimbursement deliberations in the future and reduce current controversies.…”

Section: New Cancer Medicinesmentioning

confidence: 99%

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Role of Health Technology Assessment in Pharmaceutical Market Access in Developed Countries

Kahveci¹,

Oortwijn²,

Godman³

et al. 2018

Pharmaceutical Market Access in Developed Markets2018

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Association of Disease Progression With Health-Related Quality of Life Among Adults With Breast, Lung, Pancreatic, and Colorectal Cancer

et al. 2020

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IMPORTANCE Mortality, morbidity, and health-related quality of life (HRQoL) are patient-relevant end points generally considered in the early benefit assessments of new cancer treatments. Progression-related end points, such as time to progression or progression-free survival, are not included, although patients and physicians testify to the detrimental association of disease progression with HRQoL. OBJECTIVE To examine the association of disease progression and HRQoL in 4 prevalent solidcancer entities in routine clinical practice. DESIGN, SETTING, AND PARTICIPANTS This cohort study evaluated data from 4 prospective, nonintervention, multicenter registries collected between 2011 and 2018 in 203 centers in Germany. Patients' HRQoL was assessed regularly for up to 5 years. The change in HRQoL scores after disease progression was examined with linear mixed models, adjusting for demographic and clinical covariates. Patients with metastatic breast, pancreatic, lung, and colorectal cancer were recruited at the start of systemic first-line treatment. Data analysis was performed from February 2019 to April 2019. EXPOSURES All patients received systemic, palliative first-line treatment according to their physician's choice. MAIN OUTCOMES AND MEASURES The primary outcome was deterioration of HRQoL associated with disease progression, as measured by 4 validated questionnaires: Functional Assessment of Cancer Therapy-General version 4, European Organization for Research and Treatment of Cancer QLQ-C30 version 3.0, European Organization for Research and Treatment of Cancer QLQ-C15-PAL version 1, and Hospital Anxiety and Depression Scale. RESULTS More than 8000 questionnaires from 2314 patients with 2562 documented disease progressions were analyzed. In total, 464 patients had breast cancer (464 [100.0%] female; median [range] age, 61.6 [26.4-90.1] years), 807 patients had pancreatic cancer (352 [43.6%] female; median [range] age, 70.0 [39.0-93.0] years), 341 patients had lung cancer (118 [34.6%] female; median [range] age, 65.9 [28.4-88.2] years), and 702 patients had colorectal cancer (248 [35.3%] female; median [range] age, 66.9 [26.9-92.1] years). The first disease progression was associated with a statistically significant worsening of 37 of 45 HRQoL scales; for 17 of these scales, the worsening was clinically meaningful.

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Relative effectiveness assessments of oncology medicines for pricing and reimbursement decisions in European countries

Cited by 52 publications

References 16 publications

Association Between the Use of Surrogate Measures in Pivotal Trials and Health Technology Assessment Decisions: A Retrospective Analysis of NICE and CADTH Reviews of Cancer Drugs

Association Between the Use of Surrogate Measures in Pivotal Trials and Health Technology Assessment Decisions: A Retrospective Analysis of NICE and CADTH Reviews of Cancer Drugs

Role of Health Technology Assessment in Pharmaceutical Market Access in Developed Countries

Association of Disease Progression With Health-Related Quality of Life Among Adults With Breast, Lung, Pancreatic, and Colorectal Cancer

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