title: Post-approval evaluation of effectiveness of risk minimisation: methods, challenges and interpretation.
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.
authors.affiliation: Pope Woodhead & Associates, The Old Grammar School, 1 Ramsay Road, St Ives, Cambs, PE27 5BZ, UK, swapu.banerjee@popewoodhead.com.
authors.forename: Anjan Kumar
authors.initials: AK
authors.lastname: Banerjee
authors.forename: Inge M
authors.initials: IM
authors.lastname: Zomerdijk
authors.forename: Stella
authors.initials: S
authors.lastname: Wooder
authors.forename: Simon
authors.initials: S
authors.lastname: Ingate
authors.forename: Stephen J
authors.initials: SJ
authors.lastname: Mayall
mesh_terms: D019985:Benchmarking; D003625:Data Collection / Q000379:methods; D017277:Drug Approval; D005062:European Union; D007722:Health Knowledge, Attitudes, Practice; D006801:Humans; D017063:Outcome Assessment, Health Care / Q000379:methods*; D060735:Pharmacovigilance; D018570:Risk Assessment / Q000379:methods*; D012308:Risk Management / Q000379:methods*; D014481:United States