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. 2023 Aug 28;13(8):e072309.
doi: 10.1136/bmjopen-2023-072309.

Perspectives on how to build bridges between regulation, health technology assessment and clinical guideline development: a qualitative focus group study with European experts

Milou A Hogervorst  1   2 , Mathias Møllebæk  3 , Rick A Vreman  1 , Ting-An Lu  1 , Junfeng Wang  1 , Marie Louise De Bruin  1   3 , Hubert G M Leufkens  1 , Aukje Mantel-Teeuwisse  1 , Wim Goettsch  4   2
Affiliations

Perspectives on how to build bridges between regulation, health technology assessment and clinical guideline development: a qualitative focus group study with European experts

Milou A Hogervorst et al. BMJ Open. .

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 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.

Keywords: Decision Making; Health Equity; Health policy; Organisational development; PUBLIC HEALTH; QUALITATIVE RESEARCH.

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Conflict of interest statement

Competing interests: 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.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.The other authors declare no competing interests.

Figures

Figure 1
Figure 1
Participant selection and distribution over case studies and focus group days. HTA, health technology assessment.
Figure 2
Figure 2
Final node tree with (sub)themes for each of the two research questions.
See this image and copyright information in PMC

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References

    1. Schünemann HJ, Reinap M, Piggott T, et al. . The Ecosystem of health decision making: from fragmentation to synergy. Lancet Public Health 2022;7:e378–90. 10.1016/S2468-2667(22)00057-3 - DOI - PubMed
    1. Hoekman J, Boon WPC, Bouvy JC, et al. . Use of the conditional marketing authorization pathway for oncology medicines in Europe. Clin Pharmacol Ther 2015;98:534–41. 10.1002/cpt.174 - DOI - PubMed
    1. Bloem LT, Mantel-Teeuwisse AK, Leufkens HGM, et al. . Postauthorization changes to specific obligations of conditionally authorized medicines in the European Union: A retrospective cohort study. Clin Pharmacol Ther 2019;105:426–35. 10.1002/cpt.1169 - DOI - PubMed
    1. Goring S, Taylor A, Müller K, et al. . Characteristics of non-randomised studies using comparisons with external controls submitted for regulatory approval in the USA and Europe: a systematic review. BMJ Open 2019;9:e024895. 10.1136/bmjopen-2018-024895 - DOI - PMC - PubMed
    1. Vreman RA, Naci H, Goettsch WG, et al. . Decision making under uncertainty: comparing regulatory and health technology assessment reviews of medicines in the United States and Europe. Clin Pharmacol Ther 2020;108:350–7. 10.1002/cpt.1835 - DOI - PMC - PubMed

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