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Patient Registries: An Underused Resource for Medicines Evaluation

Author

Listed:
  • Patricia McGettigan

    (Queen Mary University of London)

  • Carla Alonso Olmo

    (European Medicines Agency)

  • Kelly Plueschke

    (European Medicines Agency)

  • Mireia Castillon

    (European Medicines Agency)

  • Daniel Nogueras Zondag

    (European Medicines Agency)

  • Priya Bahri

    (European Medicines Agency)

  • Xavier Kurz

    (European Medicines Agency)

  • Peter G. M. Mol

    (University of Groningen
    Dutch Medicines Evaluation Board)

Abstract

Introduction Patient registries, ‘organised systems that use observational methods to collect uniform data on a population defined by a particular disease, condition, or exposure, and that is followed over time’, are potentially valuable sources of data for supporting regulatory decision-making, especially for products to treat rare diseases. Nevertheless, patient registries are greatly underused in regulatory assessments. Reasons include heterogeneity in registry design and in the data collected, even across registries for the same disease, as well as unreliable data quality and data sharing impediments. The Patient Registries Initiative was established by the European Medicines Agency in 2015 to support registries in collecting data suitable to contribute to regulatory assessments, especially post-authorisation safety and effectiveness studies. Methods We conducted a qualitative synthesis of the published observations and recommendations from an initiative-led multi-stakeholder consultation and four disease-specific patient registry workshops. We identified the primary factors facilitating the use of registry data in regulatory assessments. We generated proposals on operational measures needed from stakeholders including registry holders, patients, healthcare professionals, regulators, marketing authorisation applicants and holders, and health technology assessment bodies for implementing these. Results Ten factors were identified as facilitating registry use for supporting regulatory assessments of medicinal products. Proposals on operational measures needed for implementation were categorised according to three themes: (1) nature of the data collected and registry quality assurance processes; (2) registry governance, informed consent, data protection and sharing; and (3) stakeholder communication and planning of benefit-risk assessments. Conclusions These are the first explicit proposals, from a regulatory perspective, on operational methods for increasing the use of patient registries in medicines regulation. They apply to registry holders, patients, regulators, marketing authorisation holders/applicants and healthcare stakeholders broadly, and their implementation would greatly facilitate the use of these valuable data sources in regulatory decision-making.

Suggested Citation

  • Patricia McGettigan & Carla Alonso Olmo & Kelly Plueschke & Mireia Castillon & Daniel Nogueras Zondag & Priya Bahri & Xavier Kurz & Peter G. M. Mol, 2019. "Patient Registries: An Underused Resource for Medicines Evaluation," Drug Safety, Springer, vol. 42(11), pages 1343-1351, November.
  • Handle: RePEc:spr:drugsa:v:42:y:2019:i:11:d:10.1007_s40264-019-00848-9
    DOI: 10.1007/s40264-019-00848-9
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    Citations

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    Cited by:

    1. Rita Francisco & Sandra Brasil & Carlota Pascoal & Andrew C. Edmondson & Jaak Jaeken & Paula A. Videira & Cláudia de Freitas & Vanessa dos Reis Ferreira & Dorinda Marques-da-Silva, 2022. "A Community-Led Approach as a Guide to Overcome Challenges for Therapy Research in Congenital Disorders of Glycosylation," IJERPH, MDPI, vol. 19(11), pages 1-19, June.
    2. Emanuel Raschi & Matteo Bianchin & Milo Gatti & Alessandro Squizzato & Fabrizio De Ponti, 2019. "Comparative Effectiveness and Safety of Direct Oral Anticoagulants: Overview of Systematic Reviews," Drug Safety, Springer, vol. 42(12), pages 1409-1422, December.
    3. Kamila Sienkiewicz & Monika Burzyńska & Izabela Rydlewska-Liszkowska & Jacek Sienkiewicz & Ewelina Gaszyńska, 2021. "The Importance of Direct Patient Reporting of Adverse Drug Reactions in the Safety Monitoring Process," IJERPH, MDPI, vol. 19(1), pages 1-16, December.
    4. Patricia McGettigan & Carla Alonso Olmo & Kelly Plueschke & Mireia Castillon & Daniel Zondag & Priya Bahri & Xavier Kurz & Peter G. M. Mol, 2019. "Authors’ Reply to Ravi Jandhyala’s Comment on “Patient Registries: An Underused Resource for Medicines Evaluation: Operational Proposals for Increasing the Use of Patient Registries in Regulatory Asse," Drug Safety, Springer, vol. 42(12), pages 1517-1518, December.
    5. Ravi Jandhyala, 2019. "Comment on: “Patient Registries: An Underused Resource for Medicines Evaluation: Operational Proposals for Increasing the Use of Patient Registries in Regulatory Assessments”," Drug Safety, Springer, vol. 42(12), pages 1515-1516, December.

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