Should health technology assessment be more patient centric? If so, how?

Drummond, Michael; Torbica, Aleksandra; Tarricone, Rosanna

doi:10.1007/s10198-020-01182-z

Cited by 12 publications

(9 citation statements)

References 21 publications

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“…This hesitation is also reflected in the overall performance of the jurisdictions regarding patient involvement as a “good practice” to orphan drug reimbursement ( Table 5 ). As Drummond et al described, the current, overall approach towards patient involvement is considered to be “reactive rather than proactive” ( Douglas et al, 2015 ; Drummond et al, 2020 ). Furthermore, the choice for a specific set of patients (those with or without the diseases) may be culturally defined ( Torbica et al, 2017 ; Drummond et al, 2020 ).…”

Section: Discussionmentioning

confidence: 99%

“…As Drummond et al described, the current, overall approach towards patient involvement is considered to be “reactive rather than proactive” ( Douglas et al, 2015 ; Drummond et al, 2020 ). Furthermore, the choice for a specific set of patients (those with or without the diseases) may be culturally defined ( Torbica et al, 2017 ; Drummond et al, 2020 ). Although there has been increasing guidance on patient involvement, as well as interest in tools to make overall patient involvement more robust, for example via patient-reported “outcome” or “experience” measures (PROMs and PREMs), their use in value assessment/HTA is still limited ( Hunter et al, 2018 ; Janssens et al, 2019 ; Drummond et al, 2020 ; Jommi et al, 2021 ; Wale et al, 2021 ).…”

Section: Discussionmentioning

confidence: 99%

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How Can We Optimize the Value Assessment and Appraisal of Orphan Drugs for Reimbursement Purposes? A Qualitative Interview Study Across European Countries

et al. 2022

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Introduction: The expansion of orphan drug treatment at increasing prices, together with uncertainties regarding their (cost-)effectiveness raises difficulties for decision-makers to assess these drugs for reimbursement. The present qualitative study aims to gain better insight into current value assessment and appraisal frameworks for orphan drugs, and provides guidance for improvement.Methods: 22 European experts from 19 different countries were included in a qualitative survey, followed by in-depth semi-structured interviews. These experts were academics, members of reimbursement agencies or health authorities, or members of regulatory or health/social insurance institutions. Adopting a Grounded Theory approach, transcripts were analysed according to the QUAGOL method, supported by the qualitative data analysis software Nvivo.Results: Although participants indicated several good practices (e.g., the involvement of patients and the presence of structure and consistency), several barriers (e.g., the lack of transparency) lead to questions regarding the efficiency of the overall reimbursement process. In addition, the study identified a number of “contextual” determinants (e.g., bias, perverse effects of the orphan drug legislation, and an inadequate consideration of the opportunity cost), which may undermine the legitimacy of orphan drug reimbursement decisions.Conclusion: The present study provides guidance for decision-makers to improve the efficiency of orphan drug reimbursement. In particular, decision-makers can generate quick wins by limiting the impact of contextual determinants rather than improving the methods included in the HTA. When implemented into a framework that promotes “Accountability for Reasonableness” (A4R), this allows decision-makers to improve the legitimacy of reimbursement decisions concerning future orphan drugs.

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Section: Discussionmentioning

confidence: 99%

Section: Discussionmentioning

confidence: 99%

How Can We Optimize the Value Assessment and Appraisal of Orphan Drugs for Reimbursement Purposes? A Qualitative Interview Study Across European Countries

et al. 2022

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“…When considering policy decisions, it is therefore important to carefully consider whose views should be prioritised—a long-standing debate within the field of health technology assessment. 41 Arguably, it is important to consider both perspectives; priorities of women with test experience help to identify unmet or inadequate aspects of current service provision, whereas preconceived views of testing-naïve women may reveal underlying barriers and facilitators of testing since the initial decision to undergo testing is based on these pre-existing judgements. In both instances, mismatches in priorities and practice may lead to delays in seeking help or testing for future symptoms.…”

Section: Discussionmentioning

confidence: 99%

Women’s priorities towards ovarian cancer testing: a best–worst scaling study

et al. 2022

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ObjectiveTo investigate the importance of key characteristics relating to diagnostic testing for ovarian cancer and to understand how previous test experience influences priorities.DesignCase 1 best–worst scaling embedded in an online survey.SettingPrimary care diagnostic testing in England and Wales.Participants150 women with ovaries over 40 years old living in England and Wales.MethodsWe used best–worst scaling, a preference-based survey method, to elicit the relative importance of 25 characteristics relating to ovarian cancer testing following a systematic review. Responses were modelled using conditional logit regression. Subgroup analysis investigated variations based on testing history.Main outcome measuresRelative importance scores.Results‘Chance of dying from ovarian cancer’ (0.380, 95% CI 0.26 to 0.49) was the most important factor to respondents, closely followed by ‘test sensitivity’ (0.308, 95% CI 0.21 to 0.40). In contrast, ‘time away from usual activities’ (−0.244, 95% CI −0.33 to −0.15) and ‘gender of healthcare provider’ (−0.243, 95% CI −0.35 to −0.14) were least important to respondents overall. Women who had previously undergone testing placed higher importance on certain characteristics including ‘openness of healthcare providers’ and ‘chance of diagnosing another condition’ at the expense of reduced emphasis on characteristics such as ‘pain and discomfort’ and ‘time away from usual activities’.ConclusionsThe results clearly demonstrated items at the extreme, which were most and least important to women considering ovarian cancer testing. Differences in priorities by testing history demonstrate an experience effect, whereby preferences adapt over time based on evidence and experience. Acknowledging these differences helps to identify underlying barriers and facilitators for women with no test experience as well as shortcomings of current service based on women with experience.

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“…Although the evidence available corroborates the importance of integrating the views of the patients in HTA, at present, there is no consensus on what “patient-centric HTA” actually implies. In the context of a private insurance-based health system, in which an important proportion of the therapeutic payments may be performed directly by the patients, the consideration of the patient perspective in HTA might be more relevant, than in a publicly funded national health system [ 21 ].…”

Section: Policy Frameworkmentioning

confidence: 99%

“…Similarly, patient-reported experience measures (PREMs) that aim to capture elements that include burden of disease, route of administration and impact on caregivers, might be considered. In the US, FDA has endorsed the use of PROMs to support label claims for regulatory decisions, yet there is still limited evidence of their use by HTA bodies [ 21 ].…”

Section: Policy Frameworkmentioning

confidence: 99%

Value-based pricing for advanced therapy medicinal products: emerging affordability solutions

Gonçalves

2021

Eur J Health Econ

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The emergence of advanced therapy medicinal products (ATMPs), a disruptive class of health technologies, is generating important challenges in terms of value assessment and their high prices introduce critical access and affordability concerns. The aim of this article is to analyze the challenges of traditional value assessment and price and reimbursement methods in the evaluation of ATMPs and to characterize the current and prospective financing solutions that may ensure patient access and affordability for these health technologies. Standard Health Technology Assessment (HTA) is not designed for ATMPs, and may delay access to these health technologies, thus a broader concept of value is required. As a consequence, value-based pricing methodologies have been gaining terrain to cope with the specific challenges of ATMPs. The pricing and reimbursement framework should ensure the balance between encouragements to innovation and maximization of value for money for payers, through the attribution of a fair price to new health technologies. Early scientific advice by regulatory and HTA bodies to developers is key, as it will contribute to diminish the perspective gap between developers, regulators and payers. The high efficacy/high price dynamic of many advanced therapies will demand novel financing models, both in the EU and US. Managed entry agreements (MEA), with financing being conditional to the submission of additional evidence, associated with methods of leased payments, may offer effective strategies to address the uncertainties caused by the evidence gap associated with ATMPs, ensuring affordable and sustained access.

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Should health technology assessment be more patient centric? If so, how?

Cited by 12 publications

References 21 publications

How Can We Optimize the Value Assessment and Appraisal of Orphan Drugs for Reimbursement Purposes? A Qualitative Interview Study Across European Countries

How Can We Optimize the Value Assessment and Appraisal of Orphan Drugs for Reimbursement Purposes? A Qualitative Interview Study Across European Countries

Women’s priorities towards ovarian cancer testing: a best–worst scaling study

Value-based pricing for advanced therapy medicinal products: emerging affordability solutions

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