A national evaluation analysis and expert interview study of real-world data sources for research and healthcare decision-making.

Data quality Data quality criteria Data quality recommendations FAIR data principles GPMed Gesellschaft für Pharmazeutische Medizin Health data strategy Health data use Healthcare decision-making Pharmaceutical research Quality criteria for RWD in health care Real-world data Real-world evidence Secondary use of health data

Journal

Scientific reports
ISSN: 2045-2322
Titre abrégé: Sci Rep
Pays: England
ID NLM: 101563288

Informations de publication

Date de publication:
28 Apr 2024
Historique:
received: 09 10 2023
accepted: 11 04 2024
medline: 29 4 2024
pubmed: 29 4 2024
entrez: 28 4 2024
Statut: epublish

Résumé

Real-world data (RWD) can provide intel (real-world evidence, RWE) for research and development, as well as policy and regulatory decision-making along the full spectrum of health care. Despite calls from global regulators for international collaborations to integrate RWE into regulatory decision-making and to bridge knowledge gaps, some challenges remain. In this work, we performed an evaluation of Austrian RWD sources using a multilateral query approach, crosschecked against previously published RWD criteria and conducted direct interviews with representative RWD source samples. This article provides an overview of 73 out of 104 RWD sources in a national legislative setting where major attempts are made to enable secondary use of RWD (e.g. law on the organisation of research, "Forschungsorganisationsgesetz"). We were able to detect omnipresent challenges associated with data silos, variable standardisation efforts and governance issues. Our findings suggest a strong need for a national health data strategy and data governance framework, which should inform researchers, as well as policy- and decision-makers, to improve RWD-based research in the healthcare sector to ultimately support actual regulatory decision-making and provide strategic information for governmental health data policies.

Identifiants

pubmed: 38679653
doi: 10.1038/s41598-024-59475-9
pii: 10.1038/s41598-024-59475-9
doi:

Types de publication

Journal Article Research Support, Non-U.S. Gov't

Langues

eng

Sous-ensembles de citation

IM

Pagination

9751

Informations de copyright

© 2024. The Author(s).

Références

Burns, L. et al. Real-world evidence for regulatory decision-making: Guidance from around the world. Clin. Ther. 44(3), 420–437. https://doi.org/10.1016/j.clinthera.2022.01.012 (2022).
doi: 10.1016/j.clinthera.2022.01.012 pubmed: 35181179
Valla, V. et al. Use of real-world evidence for international regulatory decision making in medical devices. Int. J. Digital Health. 3, 1. https://doi.org/10.29337/ijdh.50 (2023).
doi: 10.29337/ijdh.50
Baumfeld, A. E. et al. Trial designs using real-world data: The changing landscape of the regulatory approval process. Pharmacoepidemiol. Drug Saf. 29, 1201–1212. https://doi.org/10.1002/pds.4932 (2020).
doi: 10.1002/pds.4932
European Medicines Agency. High-Quality Data to Empower Data-Driven Medicines Regulation in the European Union. News 10/10. https://www.ema.europa.eu/en/news/high-quality-data-empower-data-driven-medicines-regulation-european-union (2022).
Rudrapatna, V. A. & Butte, A. J. Opportunities and challenges in using real-world data for health care. J. Clin. Invest. 130(2), 565–574. https://doi.org/10.1172/JCI129197 (2020).
doi: 10.1172/JCI129197 pubmed: 32011317 pmcid: 6994109
Degelsegger-Márquez, A. The future of European health (data) systems. PHIRI Spring School on Health Information. https://jasmin.goeg.at/2768/1/PHIRI%20Spring%20School%202023%20-ADM.pdf (2023).
Regulation (EU) 2022/868 of the European Parliament and of the Council of 30 May 2022 on European data governance and amending Regulation (EU) 2018/1724 (Data Governance Act). EUR-Lex. https://eur-lex.europa.eu/legal-content/EN/TXT/?uri=CELEX%3A32022R0868 (2022).
European Commission. Public Health. eHealth: Digital Health and Care. European Health Data Space. https://health.ec.europa.eu/ehealth-digital-health-and-care/european-health-data-space_en .
European Commission. Public Health. Reform of the EU Pharmaceutical Legislation. https://health.ec.europa.eu/medicinal-products/pharmaceutical-strategy-europe/reform-eu-pharmaceutical-legislation_en .
Thiel, R. et al. #SmartHealthSystems. Digitalisierungsstrategien im internationalen Vergleich. Bertelsmann Stiftung (HRSG). https://www.bertelsmann-stiftung.de/de/publikationen/publikation/did/smarthealthsystems (2018).
European Medicines Agency. Data Analysis and Real World Interrogation Network (DARWIN EU). https://www.ema.europa.eu/en/about-us/how-we-work/big-data/data-analysis-real-world-interrogation-network-darwin-eu#share .
European Medicines Agency. Global Regulators Call for International Collaboration to Integrate Real-World Evidence into Regulatory Decision-Making. News 22/07. https://www.ema.europa.eu/en/news/global-regulators-call-international-collaboration-integrate-real-world-evidence-regulatory-decision (2022).
Klimek, P. et al. Quality criteria for real-world data in pharmaceutical research and health care decision-making: Austrian expert consensus. JMIR Med. Inform. 10(6), e34204. https://doi.org/10.2196/34204 (2022).
doi: 10.2196/34204 pubmed: 35713954 pmcid: 9250059
Degelsegger‐Márquez, A. Gesundheitsdaten in Österreich—ein Überblick. Gesundheit Österreich. https://jasmin.goeg.at/id/eprint/2023 (2021).
Proposal for a Regulation of the European Parliament and of the Council on the European Health Data Space. COM/2022/197 final. https://eur-lex.europa.eu/legal-content/EN/TXT/?uri=celex%3A52022PC0197 (2022).
Feldman, K., Johnson, R. A. & Chawla, N. V. The state of data in healthcare: path towards standardization. J. Healthc. Inform. Res. 2, 248–271. https://doi.org/10.1007/s41666-018-0019-8 (2018).
doi: 10.1007/s41666-018-0019-8 pubmed: 35415409 pmcid: 8982788
Bundesgesetzblatt. Datenschutz-Anpassungsgesetz 2018—Wissenschaft und Forschung – WFDSAG 2018. BGBl. I Nr. 31/2018. https://www.ris.bka.gv.at/eli/bgbl/I/2018/31 (2018).
Bundesgesetzblatt. Änderung des Bundesstatistikgesetzes 2000 und des Forschungsorganisationsgesetzes. BGBl. I Nr. 205/2021. https://www.ris.bka.gv.at/eli/bgbl/I/2021/205 (2021).
EMA/HMA Big Data Big Data Steering Group. Workplan 2023–2025. Version 1.1—September 2023. https://www.ema.europa.eu/en/documents/work-programme/workplan-2023-2025-hma/ema-joint-big-data-steering-group_en.pdf (2023).
Staatssekretariat für Digitalisierung, Bundesministerium für Finanzen. Digital Austria Act. Das Digitale Arbeitsprogramm der Bundesregierung. Digitales Gesundheitswesen. https://www.digitalaustria.gv.at/Strategien/Digital-Austria-Act---das-digitale-Arbeitsprogramm-der-Bundesregierung.html (2023).
Aigner, G. et al. Grundlagenpapier zur Schaffung des österreichischen Gesundheitsdatenraumes. Arbeitsgruppe Digitalisierung und Register des Obersten Sanitätsrates. IERM-Working-Paper Nr. 12. https://ierm.univie.ac.at/fileadmin/user_upload/i_ierm/Varanstaltungen/WP_12_-_Aigner_et_al._-_Grundlagenpapier_zur_Schaffung_des_oesterreichischen_Gesundheitsdatenraumes.pdf (2023).
Bundesministerium für Finanzen. Pressemeldungen November 2023. Rauch/Tursky: Digitale Gesundheitsreform bringt entscheidenden Beitrag zur Entlastung des Gesundheitssystems. https://www.bmf.gv.at/presse/pressemeldungen/2023/november/digitalisierung-gesundheitsbereich.html (2023).
Parlament Österreich. Parlamentskorrespondenz Nr. 1232 vom 22.11.2023. Rauch: Insgesamt 5 Mrd. € mehr für das Gesundheitswesen bis zum Jahr 2028. https://www.parlament.gv.at/aktuelles/pk/jahr_2023/pk1232 (2023).

Auteurs

Veronika Mikl (V)

Gesellschaft für Pharmazeutische Medizin E.V. (GPMed), 1210, Vienna, Austria.
Roche Austria GmbH, Vienna, Austria.

Dejan Baltic (D)

Gesellschaft für Pharmazeutische Medizin E.V. (GPMed), 1210, Vienna, Austria.
Amgen GmbH, Vienna, Austria.

Thomas Czypionka (T)

Institut für Höhere Studien - Institute for Advanced Studies (IHS), 1080, Vienna, Austria.

Alexander Degelsegger-Márquez (A)

Gesundheit Österreich GmbH (GÖG), 1010, Vienna, Austria.

Nikolaus Forgó (N)

Faculty of Law, Department of Innovation and Digitalisation in Law, University of Vienna, 1010, Vienna, Austria.

Ghazaleh Gouya-Lechner (G)

Gesellschaft für Pharmazeutische Medizin E.V. (GPMed), 1210, Vienna, Austria.
Gouya Insights GmbH & CoKG, 1190, Vienna, Austria.

Arnold Herzog (A)

Austrian Medicines and Medical Devices Agency (AGES Medizinmarktaufsicht), 1220, Vienna, Austria.

Peter Klimek (P)

Supply Chain Intelligence Institute Austria (ASCII), 1080, Vienna, Austria.
Section for Science of Complex Systems, Center for Medical Statistics, Informatics, and Intelligent Systems, Medical University Vienna, 1090, Vienna, Austria.
Complexity Science Hub Vienna, 1080, Vienna, Austria.

David Benjamin Lumenta (DB)

Research Unit for Digital Surgery, Division of Plastic, Aesthetic and Reconstructive Surgery, Department of Surgery, Medical University of Graz, 8036, Graz, Austria.

Bernhard Mraz (B)

Gesellschaft für Pharmazeutische Medizin E.V. (GPMed), 1210, Vienna, Austria.
Novartis Pharma GmbH, Vienna, Austria.

Herwig Ostermann (H)

Gesundheit Österreich GmbH (GÖG), 1010, Vienna, Austria.

Robert Scharinger (R)

Federal Ministry of Social Affairs, Health, Care and Consumer Protection, 1010, Vienna, Austria.

Tanja Stamm (T)

Gesellschaft für Pharmazeutische Medizin E.V. (GPMed), 1210, Vienna, Austria.
Section for Outcomes Research, Center for Medical Statistics, Informatics, and Intelligent Systems, Medical University of Vienna, Medical University Vienna, 1090, Vienna, Austria.

Michael Strassnig (M)

Wiener Wissenschafts-, Forschungs- und Technologiefonds (Vienna Science and Technology Fund), 1090, Vienna, Austria.
Expertenplattform Plattform Registerforschung, c/o WWTF, 1090, Vienna, Austria.

Markus Zeitlinger (M)

Gesellschaft für Pharmazeutische Medizin E.V. (GPMed), 1210, Vienna, Austria.
Department of Clinical Pharmacology, Medical University Vienna, 1090, Vienna, Austria.

Johannes Pleiner-Duxneuner (J)

Gesellschaft für Pharmazeutische Medizin E.V. (GPMed), 1210, Vienna, Austria. johannes.pleiner@gpmed.at.
Roche Austria GmbH, Vienna, Austria. johannes.pleiner@gpmed.at.

Articles similaires

[Redispensing of expensive oral anticancer medicines: a practical application].

Lisanne N van Merendonk, Kübra Akgöl, Bastiaan Nuijen
1.00
Humans Antineoplastic Agents Administration, Oral Drug Costs Counterfeit Drugs

Smoking Cessation and Incident Cardiovascular Disease.

Jun Hwan Cho, Seung Yong Shin, Hoseob Kim et al.
1.00
Humans Male Smoking Cessation Cardiovascular Diseases Female
Humans United States Aged Cross-Sectional Studies Medicare Part C
1.00
Humans Yoga Low Back Pain Female Male

Classifications MeSH