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Closing the cycle of innovation in healthcare in Europe

Published online by Cambridge University Press:  17 January 2020

Frank Hulstaert*
Affiliation:
Belgian healthcare knowledge centre (KCE), Brussels, Belgium
Alric Ruether
Affiliation:
International Affairs, Institut für Qualität und Wirtschaftlichkeit im Gesundheitswesen (IQWiG), Cologne, Germany
Jacques Demotes
Affiliation:
European Clinical Research Infrastructure Network (ECRIN), Paris, France
Øyvind Melien
Affiliation:
Reviews and HTA, Norwegian Institute of Public Health, Chair of the Clinical Research Initiative for Global Health (CRIGH), Oslo, Norway
*
Author for correspondence: Frank Hulstaert, E-mail: frank.hulstaert@kce.fgov.be

Abstract

Pragmatic or practice-oriented comparative effectiveness trials may be conducted to fill the evidence gaps that are revealed after the private sector has performed the trials needed for bringing their product to the market. A tool of increasing importance to identify such evidence gaps is resulting from health technology assessments (HTA) whereby the data derived from clinical research are examined in a systematic manner with reference to effect, safety, as well as additional parameters. Practice-oriented trials are informative for healthcare decision makers, practice-changing and may even be cost-saving for the healthcare payers. There are however only a limited number of funding sources for such trials. Public and private healthcare payers should stimulate the conduct of practice-oriented trials in their effort to maximize patient benefit within the limitation of the available resources. Pragmatic randomized trials can be performed at low cost when based on existing coded electronic health records and as well health registries. Public health decision makers are increasingly taking advantage of results from health technology assessments to support priority setting. In accordance with this it would appear reasonable that decision makers should get more involved in priority setting and funding also in the field of clinical research in order to provide further evidence needed for assessments, reassessments, and subsequent qualified decisions and resource allocations in health care. A closer dialogue and collaboration between the clinical research and HTA communities would facilitate a more efficient utilization of such opportunities.

Type
Article Commentary
Copyright
Copyright © Cambridge University Press 2020

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References

Leighann Kimble, L, Massoud, MR (2017) What do we mean by innovation in healthcare? EMJ Innov 1(1), 89-91.Google Scholar
Hulstaert, F, Neyt, M, Vinck, I et al. (2013) Pre-market clinical evaluations of innovative high-risk medical devices in Europe. Int J Technol Assess Health Care 28(3), 278-284.CrossRefGoogle Scholar
EMA, European Medicines Agency (2018) Parallel consultation with regulators and health technology assessment bodies. [cited 08/02/2019]. Available at: https://www.ema.europa.eu/en/human-regulatory/research-development/scientific-advice-protocol-assistance/parallel-consultation-regulators-health-technology-assessment-bodiesGoogle Scholar
FDA, US Food and Drug Administration (2018) New Program with Payors Aims to Accelerate Patient Access to Medical Devices. [cited 08/02/2019]. Available at: https://www.fda.gov/NewsEvents/Newsroom/FDAVoices/ucm621681.htmGoogle Scholar
Health Canada (2019) Health Canada and CADTH launch new initiative to provide early parallel scientific advice. [cited 9/12/2019]. Available at: https://www.canada.ca/en/health-canada/corporate/transparency/regulatory-transparency-and-openness/improving-review-drugs-devices/notice-early-panel-scientific-advice.htmlGoogle Scholar
Tafuri, G, Pagnini, M, Moseley, J et al. (2016) How aligned are the perspectives of EU regulators and HTA bodies? A comparative analysis of regulatory-HTA parallel scientific advice. Br J Clin Pharmacol 82(4), 965-973.CrossRefGoogle ScholarPubMed
Garattini, S, Chalmers, I (2009) Patients and the public deserve big changes in evaluation of drugs. Br Med J. 338, b1025.CrossRefGoogle ScholarPubMed
Wieseler, B, McGauran, N, Kaiser, T (2019) New drugs: Where did we go wrong and what can we do better? Br Med J. 366, l4340.CrossRefGoogle ScholarPubMed
Olberg, B, Perleth, M, Busse, R (2014) The new regulation to investigate potentially beneficial diagnostic and therapeutic methods in Germany: Up to international standard? Health Policy 117(2), 135-145.CrossRefGoogle ScholarPubMed
Califf, RM, Sugarman, J (2015) Exploring the ethical and regulatory issues in pragmatic clinical trials. Clin Trials 12(5), 436-441.CrossRefGoogle ScholarPubMed
Pham, CT, Karnon, JD, Middleton, PF et al. (2017) Randomised clinical trials in perinatal health care: A cost-effective investment. Med J Aust 207(7), 289-293.CrossRefGoogle ScholarPubMed
Mailankody, S, Prasad, V (2014) Comparative effectiveness questions in oncology. N Engl J Med 370(16), 1478-1481.CrossRefGoogle Scholar
Perner, A, Haase, N, Guttormsen, AB et al. (2012) Hydroxyethyl starch 130/0.42 versus Ringer's acetate in severe sepsis. N Engl J Med 367(2), 124-134.CrossRefGoogle ScholarPubMed
Rottingen, JA, Regmi, S, Eide, M et al. (2013) Mapping of available health research and development data: What's there, what's missing, and what role is there for a global observatory? Lancet 382(9900), 1286-1307.CrossRefGoogle ScholarPubMed
Chakma, J, Sun, GH, Steinberg, JD et al. (2014) Asia's ascent–global trends in biomedical R&D expenditures. N Engl J Med 370(1), 3-6.CrossRefGoogle Scholar
Bouillon, R, Slordahl, S, Nogueira, MM et al. (2015) Public investment in biomedical research in Europe. Lancet 386(10001), 1335.CrossRefGoogle ScholarPubMed
Neyt, M, Christiaens, T, Demotes, J et al. (2015) Publicly funded Practice-oriented Clinical Trials. Health Services Research (HSR). Brussels: Belgian Health Care Knowledge Centre (KCE) Report246Cs. Available at: http://www.kce.fgov.be. Accessed 2019.Google Scholar
Viergever, RF, Hendriks, TC (2016) The 10 largest public and philanthropic funders of health research in the world: What they fund and how they distribute their funds. Health Res Policy Syst 14, 12.CrossRefGoogle ScholarPubMed
Califf, RM, Zarin, DA, Kramer, JM et al. (2012) Characteristics of clinical trials registered in ClinicalTrials.gov, 2007–2010. JAMA 307(17), 1838-1847.CrossRefGoogle ScholarPubMed
Naci, H, Ioannidis, JP (2013) Comparative effectiveness of exercise and drug interventions on mortality outcomes: Metaepidemiological study. Br Med J. 347, f5577.CrossRefGoogle ScholarPubMed
European Commission STAMP Commission Expert Group (2018) Repurposing of established medicines/active substances. Available at: https://ec.europa.eu/health/sites/health/files/files/committee/stamp/stamp_9_40_en.pdf. Accessed 2019.Google Scholar
Core Outcome Measures in Effectiveness Trials (COMET) initiative (2019) Available at: www.comet-initiative.org. Accessed 2019.Google Scholar
Tunis, SR, Stryer, DB, Clancy, CM (2003) Practical clinical trials: Increasing the value of clinical research for decision making in clinical and health policy. JAMA 290(12), 1624-1632.CrossRefGoogle ScholarPubMed
Duke Margolis Center for Health Policy (2019) Leveraging randomized clinical trials to generate real-world evidence for regulatory purposes. Available at: https://healthpolicy.duke.edu/events/leveraging-randomized-clinical-trials-generate-real-world-evidence-regulatory-purposes. Accessed 2019.Google Scholar
Walters, SJ, Bonacho dos Anjos Henriques-Cadby, I, Bortolami, O (2017) Recruitment and retention of participants in randomised controlled trials: A review of trials funded and published by the United Kingdom health technology assessment programme. BMJ Open 7(3), e015276.CrossRefGoogle ScholarPubMed
The Belgian Healthcare Knowledge Centre (KCE) (2018) The KCE Trials programme. Available at: http:www.kce.fgov.be. Accessed 2019.Google Scholar
Demotes-Mainard, J (2017) Global health: Boost multinational clinical research. Nature 545(7654), 289.CrossRefGoogle ScholarPubMed
Vandenbroeck, P, Raeymaekers, P, Wickert, R et al. . (2016) Future scenarios about drug development and drug pricing. Brussels: Belgian Health Care Knowledge Centre, Diemen: Zorginstituut Nederland. Available at: https://kce.fgov.be/en/future-scenarios-about-drug-development-and-drug-pricing. Accessed 2019.Google Scholar
Gotzsche, PC (2018) Patients not patents: drug research and development as a public enterprise. Eur J Clin Invest 48(2), e12875.CrossRefGoogle Scholar
Speich, B, von Niederhausern, B, Schur, N et al. (2018) Systematic review on costs and resource use of randomized clinical trials shows a lack of transparent and comprehensive data. J Clin Epidemiol 96, 1-11.CrossRefGoogle Scholar
Martin, L, Hutchens, M, Hawkins, C et al. (2017) How much do clinical trials cost? Nat Rev Drug Discov 16(6), 381-382.CrossRefGoogle ScholarPubMed
Sertkaya, A, Wong, HH, Jessup, A et al. (2016) Key cost drivers of pharmaceutical clinical trials in the United States. Clin Trials 13(2), 117-126.CrossRefGoogle ScholarPubMed
Johnston, SC, Rootenberg, JD, Katrak, S et al. (2016) Effect of a US National Institutes of Health programme of clinical trials on public health and costs. Lancet 367(9519), 1319-1327.CrossRefGoogle Scholar
Sheffet, AJ, Flaxman, L, Tom, M et al. (2014) Financial management of a large multisite randomized clinical trial. Int J Stroke 9(6), 811-813.CrossRefGoogle ScholarPubMed
Nevens, H, Harrison, J, Vrijens, F et al. (2019) Budgeting of non-commercial clinical trials: Development of a budget tool by a public funding agency. Trials 20(1), 714.CrossRefGoogle ScholarPubMed
Ohmann, C, Banzi, R, Canham, S et al. (2017) Sharing and reuse of individual participant data from clinical trials: Principles and recommendations. BMJ Open 7(12), e018647.CrossRefGoogle ScholarPubMed
Oldgren, J (2017) Prospective registry-based randomized clinical trials – the Swedish concept for pragmatic clinical trials. Slides presented at KCE Trials symposium, Brussels, November 28. Available at: https://kce.fgov.be/en/event/real-world-evidence-randomised-trials-in-daily-practice-2nd-kce-trials-symposium. Accessed 2019.Google Scholar
Organisation for Economic Co-operation and Development (OECD) Global Science Forum (2011) Facilitating International Co-operation in Non-Commercial Clinical Trials. Available at: www.oecd.org/sti/inno/49344626.pdf. Accessed 2019.Google Scholar