Grand challenges in pharmacoeconomics and health outcomes.

Abstract

Pharmacoeconomics and health outcomes research are playing an increasingly important role in informing clinical development and market access decisions of new innovative medicines. Both disciplines are dealing with the evaluation of the costs and outcomes of healthcare interventions and can be considered as two branches of the same “value for money” tree. Pharmacoeconomics is the part of health economics that focuses on the economic evaluation of pharmaceuticals. Health outcomes research, and patient-reported outcomes (PRO) in particular, aim at understanding patient value in terms of impact of disease and its treatment on physical functioning and psychosocial wellbeing, known also as “health-related quality of life” (HRQL). PRO's are usually measured by self-reported questionnaires, thereby reflecting the patient's own viewpoint on the value of a new medicinal product. In many clinical development studies, HRQL is nowadays routinely measured to help establish the product's value for purposes of pricing and reimbursement. Despite this growing interest, both disciplines face numerous challenges going forward. The key question remains to what extent pharmacoeconomic evaluations and outcomes research are delivering better value-based decisions. Today, economic evaluation of new medicines is mandatory in many countries, so the question is no longer whether or not pharmacoeconomics is here to stay. The train has left the station. But challenges remain, mainly related to methodological issues. In contrast, I believe the jury is still out on HRQL and PRO research. A recent survey suggests that although clinicians recognize the importance of PRO's, limited experience and information is a barrier to the use of quality of life assessment in their own clinical practice. Interestingly, a large majority acknowledged that they would use more expensive medicines if these could improve HRQL (Bossola et al., 2010).