Patient Preferences in Drug Development and Evaluation

The patient perspective has received growing attention on all levels of healthcare research and decision-making. An increasing amount of literature reports on the role of the patient perspective in drug development, regulatory assessment, health technology assessment (HTA) and clinical practice guideline development. The increased focus on patients’ perspectives has already led to the participation of patients and patient representatives in micro decision-making surrounding the individual treatment, but also in their participation in macro decision-making, including industry decisions surrounding drug development, regulatory marketing authorization and reimbursement decisions. This patient participation has been shown to be informative for decision-making, but has also been criticized for not representing the entire patient population. Consequently, methods for eliciting patient preferences are emerging as a key topic in the drug development arena. While no consensus exists about the definition of patient preferences across research fields and disciplines, patient preferences can be defined as the relative desirability or acceptability to patients of specified alternatives or choices among outcomes or other attributes that differ among alternative health interventions. Patient preferences have a potential role in various decision-making contexts along the drug life cycle, including but not limited to: drug design and clinical trial design, regulatory benefit-risk assessment (BRA) and marketing authorization (MA), HTA and reimbursement decision-making and in the context of individual treatment decision-making. Due to this broad potential of patient preferences, various health care stakeholders are interested in measuring and using them at multiple points in the course of the drug life cycle.

This Research Topic covers the broad applicability of patient preferences throughout the development and evaluation of drugs. It aims to address all aspects of measuring and using patient preferences, with a special focus on their use for industry decision-making, regulatory marketing authorization decision-making and reimbursement decision-making. Articles within the scope of this topic can be review articles as well as original qualitative or quantitative research articles. Potential articles focus on different aspects concerning patient preferences in drug development such as potential barriers and facilitators for the use of patient preferences in decision-making and the current status of patient preferences for decision-making.