Uncertainty tolerance among experts involved in drug reimbursement recommendations: Qualitative evidence from HTA committees in Canada and Poland

Wranik, Wiesława Dominika; Gambold, Liesl L.; Peacock, Stuart

doi:10.1016/j.healthpol.2020.12.007

Cited by 10 publications

(10 citation statements)

References 48 publications

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“…However, evidence shows unclear correlations between surrogate endpoints and OS, which means such surrogate endpoints may not accurately predict clinical benefit ( 17 – 20 ). Scholars are divided on drugs of uncertain clinical benefit, with some opposing the positive reimbursement decisions of drugs with unclear clinical benefits ( 21 ) and others advocating that a new standard for drugs with surrogate endpoints should be established ( 22 ). Previous studies have analyzed the relevance between reimbursement decisions and clinical trial endpoints ( 23 – 28 ), the situation is not consistent across countries, while few studies have focused on the situation in China.…”

Section: Introductionmentioning

confidence: 99%

Correlation between clinical trial endpoints of marketed cancer drugs and reimbursement decisions in China

Ling

Qin

Feng

et al. 2022

Front. Public Health

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ObjectiveThis study aimed to assess whether different clinical trial endpoints in pivotal trials of cancer drugs were associated with reimbursement decisions in China.Materials and methodsCancer drugs marketed before June 30th, 2021 with publicly available technical review reports for application of drug registration on Center for Drug Evaluation (CDE) website were reviewed. The trial design characteristics and relevant clinical outcomes [e.g., overall survival (OS), progression-free survival (PFS) and objective response rate (ORR)] were extracted from the technical review reports, while the reimbursement decisions were reviewed from National Healthcare Security Administration (NHSA) website. The differences in trial characteristics and clinical outcomes between drugs with positive reimbursement decisions and negative ones were compared by hypothesis test (Pearson's chi-squared test, Fisher's exact test, independent samples t-test and Mann-Whitney U test). The correlation between different clinical trial endpoints and reimbursement decisions was analyzed by multivariate logistic regression.ResultsThere were 112 cancer drug indications included in this study. Among these indications, 76 received a positive reimbursement decision, and the most common primary endpoints of them were PFS (42.1%) and ORR (30.3%). Taking PFS (OR = 7.333) and ORR (OR = 5.271) as the primary endpoints were more likely to receive a positive reimbursement decision compared with OS (P = 0.003). The proportion of drugs marketed with phase I (75.0%) and phase II (85.7%) clinical trials receiving positive reimbursement decisions are significantly higher than those marketed with phase III clinical trials (61.3%, P = 0.043). The magnitude of clinical benefit only had subtle influences (Prisk benefit − OS = 0.627, Prisk benefit − PFS = 0.087, Psurvival benefit − OS = 0.545, Psurvival benefit − PFS = 0.189) on the drug reimbursement decisions, however, the drug prices and clinical needs also made a difference on that.ConclusionThis study found that, in Chinese drug price negotiations from 2017 to 2021, policymakers have focused more on meeting clinical needs and filling therapeutical gaps in National Reimbursement Drug List (NRDL), while requirements for the selection of primary endpoints, clinical trial phases, and clinical benefits have been reduced. In the future, emphasis should be put on the use of surrogate endpoints and clinical benefits.

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Section: Introductionmentioning

confidence: 99%

Correlation between clinical trial endpoints of marketed cancer drugs and reimbursement decisions in China

Ling

Qin

Feng

et al. 2022

Front. Public Health

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“…Poland has a publicly funded health system, and an HTA agency and an expert advisory committee work together to make drug-funding recommendations to the national Ministry of Health [ 29 ].…”

Section: Resultsmentioning

confidence: 99%

How Can the EU Beating Cancer Plan Help in Tackling Lung Cancer, Colorectal Cancer, Breast Cancer and Melanoma?

et al. 2022

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Cancer is the second leading cause of mortality in EU countries, and the needs to tackle cancer are obvious. New scientific understanding, techniques and methodologies are opening up horizons for significant improvements in diagnosis and care. However, take-up is uneven, research needs and potential outstrip currently available resources, manifestly beneficial practices—such as population-level screening for lung cancer—are still not generalised, and the quality of life of patients and survivors is only beginning to be given attention it merits. This paper, mainly based on a series of multistakeholder expert workshops organised by the European Alliance for Personalised Medicine (EAPM), looks at some of those specifics in the interest of planning a way forward. Part of this exercise also involves taking account of the specific nature of Europe and its constituent countries, where the complexities of planning a way forward are redoubled by the wide variations in national and regional approaches to cancer, local epidemiology and the wide disparities in health systems. Despite all the differences between cancers and national and regional resources and approaches to cancer care, there is a common objective in pursuing broader and more equal access to the best available care for all European citizens.

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“…36 Uncertainty also poses an important challenge to the HTA process and has been shown to have significant impact on reimbursement decisions. 29,40,41 The current HTA approaches are not well suited when dealing with paediatric health and illness and there is a need to develop innovative and sustainable solutions for the methodological challenges in value assessment.…”

Section: Methodological Challenges In Performing Hta In the Paediatri...mentioning

confidence: 99%

“…23 Drug reimbursement decisions, at national level, are often contingent on health technology assessment (HTA). 29 HTA is a comprehensive evaluation framework that helps ascertain the value of new interventions, including medicines, diagnostics, medical devices and procedures. HTA evaluates safety, clinical utility, cost-effectiveness and also takes into account social, legal and ethical parameters which are especially important in the case of vulnerable groups such as the paediatric population.…”

Section: Market Access and Access To Treatmentmentioning

confidence: 99%

Health technology assessment of paediatric medicines: European landscape, challenges and opportunities inside the conect4children project

Moretti

Ruiz

Bonifazi

et al. 2022

Brit J Clinical Pharma

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The medicine development process is complex and requires time and effort to ensure safety, efficacy and quality. In paediatrics, this process is even more challenging, as it involves a subgroup of the population that already faces a considerable gap in the clinical evaluation of medicines and devices compared to the adult population. Moreover, access to therapies is heavily influenced by national health technology assessment (HTA) recommendations, which often form the basis for pricing and reimbursement decisions that affect the effective availability of treatments within the national health systems. Yet performing an HTA to assess the relative effectiveness and cost-effectiveness of a new children's treatment has several nontrivial implications, creating a critical issue for the paediatric population. In addition, the advent of innovative health technologies for children emphasises the need to empower the role of HTAs in paediatrics.This article aims at describing the most relevant elements of the drug development process in the paediatric field by focusing on the HTA assessment. Particular attention will be paid to the factors that influence market access for new paediatric medicines and patients' access to treatment. The paper will also highlight some central methodological challenges in conducting HTA in the paediatric field. Finally, the article will provide insight into how initiatives, such as conect4children, may subsequently reinforce HTA awareness in the paediatric community and strengthen collaborations through network mechanisms.

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Uncertainty tolerance among experts involved in drug reimbursement recommendations: Qualitative evidence from HTA committees in Canada and Poland

Cited by 10 publications

References 48 publications

Correlation between clinical trial endpoints of marketed cancer drugs and reimbursement decisions in China

Correlation between clinical trial endpoints of marketed cancer drugs and reimbursement decisions in China

How Can the EU Beating Cancer Plan Help in Tackling Lung Cancer, Colorectal Cancer, Breast Cancer and Melanoma?

Health technology assessment of paediatric medicines: European landscape, challenges and opportunities inside the conect4children project

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