Grand Challenges in Pharmacoeconomics and Health Outcomes

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). Shared challengeS and concernS As is the case for pharmacoeconomics, there is a huge need for training and education on what HRQL is all about, how to measure it, and – above all – how to analyze and interpret results. The biggest challenge is with the interpretation and effective communication of the clinical meaningfulness of HRQL data. Indeed, the question that many clinicians and payers are asking about PRO’s is “What do these numbers and scores really mean?” Another major challenging area of interest for both pharmacoeconomics and outcomes research is the role of Quality Adjusted Life Years (QALYs) in economic evaluation. The QALY concept used in costutility analyses is increasingly subject of methodological debate. The main concern is that different methods for valuing health state preferences may yield different results (Drummond et al., 2009). A related issue is the respective role of preference-based utilities versus other PRO’s to facilitate market access. The use of cost-utility analysis is the preferred form of economic evaluation in several countries to inform market access decisions. Other countries are in favor of disease-specific outcomes. The challenge is to convince the decision makers that there is more to HRQL than health utilities alone. Therefore, HRQL PRO’s should be given a more prominent role in support of value-based pricing and reimbursement. The predominant role of generic measures to estimate utilities is also reason for concern. Advances in methods for mapping disease-specific quality of life measures onto generic preference-based measures open new prospects for better capturing clinically meaningful changes in response to treatment.