Perspectives on how to build bridges between regulation, health technology assessment and clinical guideline development: a qualitative focus group study with European experts
Hogervorst, Milou A.; Møllebæk, Mathias; Vreman, Rick A.; Lu, Ting An; Wang, Junfeng; De Bruin, Marie Louise; Leufkens, Hubert G.M.; Mantel-Teeuwisse, Aukje; Goettsch, Wim
(2023) BMJ Open, volume 13, issue 8
(Article)
Abstract
OBJECTIVE: Improving synergy among regulation, health technology assessment (HTA) and clinical guideline development is relevant as these independent processes are building on shared evidence-based grounds. The two objectives were first to assess how convergence of evidentiary needs among stakeholders may be achieved, and second, to determine to what extent convergence
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can be achieved. DESIGN: Qualitative study using eight online dual-moderator focus groups. SETTING: Discussions had a European focus and were contextualised in four case studies on head and neck cancer, diabetes mellitus, multiple sclerosis and myelodysplastic syndromes. PARTICIPANTS: Forty-two experienced (over 10 years) European regulators, HTA representatives and clinicians participated in the discussion. INTERVENTIONS: Participants received information on the case study and research topic in advance. An introductory background presentation and interview guide for the moderators were used to steer the discussion. RESULTS: Convergence may be achieved through improved communication institutionalised in multistakeholder early dialogues, shared definitions and shared methods. Required data sets should be inclusive rather than aligned. Deliberation and decision-making should remain independent. Alignment could be sought for pragmatic clinical trial designs and patient registries. Smaller and lower-income countries should be included in these efforts. CONCLUSION: Actors in the field expressed that improving synergy among stakeholders always involves trade-offs. A balance needs to be found between the convergence of processes and the institutional remits or geographical independence. A similar tension exists between the involvement of more actors, for example, patients or additional countries, and the level of collaboration that may be achieved. Communication is key to establishing this balance.
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Keywords: Decision Making, Health Equity, Health policy, Organisational development, Qualitative research, Public health, General Medicine
ISSN: 2044-6055
Publisher: BMJ Publishing Group Ltd
Note: Funding Information: The HTx project has received funding from the European Commission’s Horizon 2020 research and innovation programme under grant agreement No 825162. This dissemination reflects only the authors’ views, and the Commission is not responsible for any use that may be made of the information it contains. Funding Information: At the time of the project, MLDB was employed by Copenhagen Centre for Regulatory Sciences (CORS). CORS is a cross-faculty university anchored institution involving various public (Danish Medicines Agency, Copenhagen University) and private (Novo Nordisk, Lundbeck, Ferring Pharmaceuticals, LEO Pharma) stakeholders as well as patient organisations (Rare Diseases Denmark). The Centre is purely devoted to the scientific aspects of the regulatory field and with a patient-oriented focus and the research is not company-specific product or directly company related. In the past 5 years, CORS has received funding from Novo Nordisk, Lundbeck, Ferring Pharmaceuticals and LEO Pharma for projects not related to this study. Currently, MLDB is employed by Utrecht University and conducts research under the umbrella of the Utrecht-WHO Collaborating Centre for Pharmaceutical Policy and Regulation. This Centre receives no direct funding or donations from private parties, including the pharmaceutical industry. Research funding from public–private partnerships, eg, IMI, and The Escher Project ( http://escher.lygature.org/ ) is accepted under the condition that no company-specific product or company-related study is conducted. The Centre has received unrestricted research funding from public sources, e.g. World Health Organisation (WHO), the Netherlands Organisation for Health Research and Development (ZonMW), the Dutch National Health Care Institute (ZIN), EC Horizon 2020, the Dutch Medicines Evaluation Board (MEB) and the Dutch Ministry of Health. None of the abovementioned companies had any involvement in the current study. Funding Information: A qualitative design was used, consisting of eight focus groups contextualised in the HTx case studies on diabetes mellitus (DM), head and neck cancer (HN), multiple sclerosis (MS) and myelodysplastic syndromes (MDS). HTx is a Horizon 2020 project supported by the European Union lasting for 5 years from January 2019, with the aim to create a framework for the next generation HTA to support patient-centred, societally oriented, real-time decision-making on access to and reimbursement for health technologies throughout Europe. Case studies were used to make the discussion more specific and the high diversity across the four cases ensured generalisability of the results to other disease areas. Results are reported in accordance with the Consolidated criteria for Reporting Qualitative research checklist. Funding Information: WG is employed by Utrecht University and conducts research under the umbrella of the Utrecht-WHO Collaborating Centre for Pharmaceutical Policy and Regulation. The Centre has received unrestricted research funding from public sources, eg, WHO, the Netherlands Organisation for Health Research and Development (ZonMW), the Dutch National Health Care Institute (ZIN), EC Horizon 2020, the Dutch Medicines Evaluation Board (MEB) and the Dutch Ministry of Health. None of the abovementioned public funding sources had any involvement in the current study. WG is also employed by the National Health Care Institute. Publisher Copyright: © Author(s) (or their employer(s)) 2023. Re-use permitted under CC BY-NC. No commercial re-use. See rights and permissions. Published by BMJ.
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