Building Synergy between Regulatory and HTA Agencies beyond Processes and Procedures—Can We Effectively Align the Evidentiary Requirements? A Survey of Stakeholder Perceptions

Wang, Ting; McAuslane, Neil; Liberti, Lawrence; Leufkens, Hubert G. M.; Hövels, Anke M.

doi:10.1016/j.jval.2017.11.003

Cited by 34 publications

(67 citation statements)

References 23 publications

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“…A recent survey on the alignment between regulatory and HTA found that 75% of company respondents, 86% of regulatory respondents, and 63% of HTA respondents thought that the definition of UMN was a top area for potential alignment between stakeholders. 13 Thus, there is an aspiration to find alignment between stakeholder groups on UMN definitions and their interpretation.…”

Section: Introductionmentioning

confidence: 99%

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Unmet Medical Need: An Introduction to Definitions and Stakeholder Perceptions

Vreman

Heikkinen²,

Schuurman

et al. 2019

Value in Health

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Background: Despite increasing informal and formal use of unmet medical need (UMN) in drug development, regulation, and assessment, there is no insight into its definitions in use. This study aims to provide insight into the current definitions in use and to provide a starting point for a multi-stakeholder discussion on alignment. Methods: A scoping and a gray literature review were performed to locate definitions of UMN in literature and on stakeholder websites. These definitions were categorized and then discussed among the multi-stakeholder author group via semistructured group discussions and open session workshops with a broader stakeholder audience. Issues with the formation of a common definition and mechanisms for use were discussed. Results: The reviews yielded 16 definitions. Differences were evident, but all included 1 or more of the following elements: (adequacy of) available treatments (16 of 16: 100%), disease severity or burden (6 of 16: 38%), and patient population size (1 of 16: 6%). The stakeholder discussions led to a suggestion for a definition including the first 2 items and, depending on context, population size. The discussions also showed that quantification of UMN is highly dependent on the scope and the value framework in which it is used based on different stakeholder preferences and responsibilities. Conclusion: We encourage stakeholders that want to promote alignment on the concept of UMN to prospectively discuss the scope in which they want to apply the concept, what elements they find important for consideration in each case, and how they would measure UMN within the broader regulatory or value framework applicable.

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Section: Introductionmentioning

confidence: 99%

“…The EMA and the National Institute for Health and Care Excellence both independently concluded on the existence of UMN in the indicated patient population. 13,14 This study has 3 goals. First, it provides a (gray) literature review to outline the definitions of UMN in use by different stakeholders.…”

Section: Introductionmentioning

confidence: 99%

Unmet Medical Need: An Introduction to Definitions and Stakeholder Perceptions

Vreman

Heikkinen²,

Schuurman

et al. 2019

Value in Health

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“…Although the benefi t risk balance of a medicine will be the same in different regions of Europe, HTA bodies include social and economic aspects, as well as availability of other treatment options into their evaluation, and that frequently results in different results for different countries. Efforts are being made to align the requirements for data and information, so that evidence generated can serve both aims: marketing authorization (regulators) and patient access (HTAs, payers) 22 . Parallel scientifi c advice with regulators and HTA bodies is available at EMA to support developers in generating appropriate evidence for both purposes 23,24 .…”

Section: Adaptation Number 4: Holding a Marketing Authorization Is Nomentioning

confidence: 99%

From Laboratory to Patient: The Evolving Path to Become a Medicine

Hidalgo‐Simon¹

2019

Mol. Front. J.

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Gene editing and other laboratory developments promise to revolutionize treatment of many diseases that currently have no cure. The process to become a medicine is long and complex and requires many years. Regulation of medicines (permission to market a product and monitoring of its performance) is evolving to adapt to emerging new approaches in development. Current issues and new ideas needed to navigate the difficult waters from laboratory to patient are discussed here: novel approaches for narrow evidence base, earlier discussions of evidence with regulators, earlier planning for post-authorization requirements and better patient access.

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“…This is particularly important with the growing trend and efforts to align regulatory and HTA standards and processes both within companies as well as across agencies, in order to further increase effectiveness and efficiency during the development, review and reimbursement of medicines. 5,6 This gap in research into decision-making during medicines development was initially addressed through the research of Donelan and colleagues, which resulted in defining the best practices in decision-making during the lifecycle of medicines, namely the 10 Quality Decision-Making Practices (QDMPs) ( Figure 1). Donelan et al also developed an instrument, the Quality of Decision-Making Orientation Scheme (QoDoS), which can be used to measure the incorporation of these 10 QDMPs into the operational processes of companies and agencies.…”

Section: Introductionmentioning

confidence: 99%

A Process for Evaluating Quality Decision-Making Practices During the Development, Review and Reimbursement of Medicines

Bujar

McAuslane

Walker

et al. 2020

Int J Health Policy Manag

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Background: The development of a medicine is not only underpinned by good science but also by Quality DecisionMaking Practices (QDMPs). Indeed, it is important to ensure that all organisations involved in the lifecycle of medicines are aligning their practices in decision-making to the QDMPs to ensure quality, transparent and consistent decisionmaking processes. Methods: The aim of this study was to evaluate the practicality of QoDoS (Quality of Decision-Making Orientation Scheme) in assessing the incorporation of ten QDMPs during the development, review and reimbursement of medicines, illustrated by case studies with a pharmaceutical company, a regulatory authority and a health technology assessment (HTA) agency. Individuals from each organisation completed the 47-item QoDoS questionnaire. Results: The results demonstrate the applicability of QoDoS in identifying favourable and unfavourable practices and in assessing the consistency and transparency of the QDMPs within each organisation, as well as across the different stakeholders. Furthermore, the study established the value of the methodology in raising awareness of the biases and best practices in decision-making, as well as having a basis for discussion for differences within and across stakeholders to promote consistency and alignment in decision-making. Finally, the QoDoS demonstrated the need for improvement across a number of decision-making practices for the 3 organisations such as the evaluation of alternatives and of the decision impact. Conclusion: The QoDoS can be used to benchmark organisations’ decision-making practices to provide a basis for discussion to ultimately encourage a level of trust across and within organisations and helping to identify areas for improvement.

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Building Synergy between Regulatory and HTA Agencies beyond Processes and Procedures—Can We Effectively Align the Evidentiary Requirements? A Survey of Stakeholder Perceptions

Cited by 34 publications

References 23 publications

Unmet Medical Need: An Introduction to Definitions and Stakeholder Perceptions

Unmet Medical Need: An Introduction to Definitions and Stakeholder Perceptions

From Laboratory to Patient: The Evolving Path to Become a Medicine

A Process for Evaluating Quality Decision-Making Practices During the Development, Review and Reimbursement of Medicines

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