JOHN D. JOHNSTON
c/o 35 Indescon Square, London E14 9DR UK

Abstract

Multi-criteria decision analysis has been supported by the European Medicines Agency to clarify how a decision on the benefit – risk balance for a medicinal product has been reached. Such analysis quantifies benefit and risk and reaches a total score by subtracting risk from benefit. New European pharmaceutical legislation has been proposed to permit timely access to custom-made medicinal products that have licences that are conjoined to those of corresponding master medicinal products. Multi-criteria decision analysis may be used to inform the regulatory position on a master medicinal product but would not be appropriate to decide (prior to first administration) if a named patient would benefit from a custom-made medicine. Multi-criteria decision analysis may be appropriate after a suitable length of exposure to the custom-made medicine.

INTRODUCTION

Evaluation of the balance between benefits and risks of medicinal products is important to everyone involved in the development, registration and use of these products. Healthcare providers and those people who need to take medicines for the treatment or prevention of disease need to be able to weigh up the likely benefits and risks of the various therapeutic options before deciding what to take. Regulators make decisions about benefits and risks of medicinal products on the basis of trials conducted prior to licensing and continue to monitor products after approval so that updated information may be made available to the public on the basis of emerging information. Pharmaceutical companies may also carry out benefit-risk assessment throughout the life-cycle of a medicinal product in order to make decisions on product development, to make changes to the product information and to fine-tune risk mitigation activities. It is only recently, however, that structured approaches to decision-making have been advocated to be part of the regulatory process (1-5).
The benefit-risk assessment of medicines is characterised by taking into account ...