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Post-Approval Evaluation of Effectiveness of Risk Minimisation: Methods, Challenges and Interpretation

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Abstract

Evaluation of the effectiveness of drug risk-minimisation measures is mandatory for both risk evaluation and mitigation strategies (REMS) in the United States and risk management plans in the European Union (EU-RMPs). Such evaluations aim to assess the impact of risk-minimisation measures on the knowledge, attitudes or behaviours of healthcare professionals or patients, the incidence of safety concerns, and their impact on the overall benefit-risk balance. Although many effectiveness evaluation models and methods are available, regulatory guidance and policy are still evolving. This paper considers evaluation strategies, challenges in evaluating risk minimisation post-authorisation, possible outcome measures and their interpretation, and potential emerging regulatory policy issues. Particular challenges include appropriate data collection, perceived and real burdens of performing evaluation on clinical practice, lack of comparators and benchmarking, and uncertainty about the best outcome measures.

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Acknowledgments

The authors wish to gratefully acknowledge the guidance and advice of Sabine M.J.M. Straus M.D., Ph.D., M.Sc. (Department of Medical Informatics, Erasmus University Medical Centre, and The Dutch Medicines Evaluation Board and the Pharmacovigilance and Risk Assessment Committee (PRAC) of the European Medicines Agency) during the preparation of this paper.

Conflict of interest

Anjan K Banerjee is employed by and is a Director of Pope Woodhead & Associates and has a COI related to holding PWA stock, employment and expert testimony, and has provided consultancy to multiple global pharmaceutical companies. Drs Ingate, Mayall and Wooder are employed by Pope Woodhead & Associates and have a COI related to employment and have provided consultancy to multiple global pharmaceutical companies. Ms Zomerdijk is also employed by the Dutch Medicines Evaluation Board and the opinions presented in this paper are her personal opinion and do not necessarily reflect those of the board. Anjan Kumar Banerjee, Inge M Zomerdijk, Stella Wooder, Simon Ingate and Stephen J. Mayall have no other conflicts of interest that are directly relevant to the content of this article.

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Authors and Affiliations

  1. Pope Woodhead & Associates, The Old Grammar School, 1 Ramsay Road, St Ives, Cambs, PE27 5BZ, UK

    Anjan Kumar Banerjee, Stella Wooder, Simon Ingate & Stephen J. Mayall

  2. Institute of Biotechnology, University of Cambridge, Tennis Court Road, Cambridge, CB2 1QT, UK

    Anjan Kumar Banerjee

  3. Department of Medical Informatics, Erasmus University Medical Centre, Rotterdam, The Netherlands

    Inge M. Zomerdijk

  4. The Dutch Medicines Evaluation Board, Graadt van Roggenweg 500, 3531 AH, Utrecht, The Netherlands

    Inge M. Zomerdijk

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  1. Anjan Kumar Banerjee
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Correspondence to Anjan Kumar Banerjee.

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Banerjee, A.K., Zomerdijk, I.M., Wooder, S. et al. Post-Approval Evaluation of Effectiveness of Risk Minimisation: Methods, Challenges and Interpretation. Drug Saf 37, 33–42 (2014). https://doi.org/10.1007/s40264-013-0126-7

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