BACKGROUND: As part of the evaluation of the overall effectiveness of European additional Risk Minimization Measures (aRMM) and the United States Risk Evaluation and Mitigation Strategies (REMS) , two types of indicators may be considered: process indicators, including physician and patient knowledge and awareness of product risks and actions that should be taken to assure safe use of a medicine; and, whenever possible, outcome indicators, aiming to provide an overall estimate of the level of risk control attained with a RMM.
OBJECTIVE: To provide an update on the upcoming C IOMS Working Group IX consensus on risk minimization evaluation and to describe challenges faced by studies evaluating aRMM/REMS, with a main focus on process indicators.
DESCRIPTION: The ability to apply robust study methodology is challenged by numerous factors. For studies of knowledge and behavior, challenges include lack of baseline information on risk behavior as well as adequate ascertainment and representativeness of study populations. For studies assessing outcome indicators, challenges include the dearth of pre-existing data sources that include the necessary exposure, outcome, and confounder information and adequate choice of relevant outcomes. We will present the views of 4 key stakeholders providing specific examples on the trade-offs made between the ideal and practical study designs to minimize bias and maximize generalizability of study results while addressing regulatory requirements and expectations. This will be followed by a panel/audience discussion and sharing of experiences. - S. Perez-Gutthann: Chair, welcome, goals and introducing speakers. - Y. Moride: CIOMS Working Group IX consensus: General principles for risk minimization evaluation and field experience. - E. Andrews: Methodological considerations for the design and implementation of risk minimization evaluation studies assessing process indicators - M. Soriano-Gabarró: Experiences and challenges with risk minimization evaluation studies: Assessing risks in diverse prescriber and patient populations. - J. Raine: Regulatory perspective on risk minimization evaluation in EU.
