Registration is now open for a workshop on the importance of dose finding and dose selection for the successful development, licencing and lifecycle management of medicines. The workshop is jointly organised by the European Medicines Agency (EMA) and the European Federation of Pharmaceutical Industries and Associations External link icon (EFPIA) and will take place at the EMA on 4 and 5 December 2014.

The workshop is open to all stakeholders, including industry, academia, regulators and patients’ and healthcare professionals’ representatives, and will be broadcasted live.

Investigating and understanding dose-exposure-response (D-E-R) relationships and selecting a dose-range for confirmatory phase-III trials and licensing is among the most difficult problems that drug developers and regulators are called to solve. Regulatory experience indicates that dose finding is often confined to determining a single dose to carry forward in phase-III based on the results of pair-wise comparisons between a few dose levels in small trials on single efficacy parameters. This type of evidence tends to form a weak basis for decision-making, increasing the risks associated with confirmatory trials. D-E-R information is often not available or not reported and questions of importance to product labelling are not answered precisely, which complicates the licensing process. In addition, despite recommendations in the ICH E4: Dose Response Information to Support Drug Registration guideline, there is a common misperception that dose ranging/finding and D-E-R characterisation are limited to exploratory development.

The workshop will discuss the utility of methods for D-E-R relationship estimation and dose finding, and the implications for the sponsor, the regulator, the prescriber and the patient, when adequate information on important questions related to dose are not generated or are sub-optimal. The impact that a lack of these data might have on benefit-risk assessment and post-approval studies will be examined. The goal of the meeting is to provide recommendations on which regulators and drug developers agree will result in improved dose-finding strategies and a more systematic D-E-R investigation in drug development. This should not only optimise the chances for successful development and approval, but should also result in improved dose recommendations in the Summary of Product Characteristics (SmPC) and lifecycle management for the medicinal products.