Does Outcomes Research Reduce Policy Maker Uncertainty?

Rotterdam, The Netherlands – Health care payers increasingly request additional data from clinical practice to assess whether a drug is worth public spending. The data gathered should reduce the initial uncertainty regarding the clinical and economic performance of expensive drugs in a real-world setting.  Although policies such as outcomes research requirements, finance and/ or outcomes based risk-sharing agreements seem attractive, many unanswered questions remain regarding their actual value and feasibility.

Researchers at the Institute of Medical Technology Assessment, in collaboration with the VU University Medical Centre and Erasmus University Medical Centre, investigated whether outcomes research reduced policy maker uncertainty regarding the value for money of bortezomib in multiple myeloma. The study showed that it was feasible to develop evidence on bortezomib’s use, effects and costs in clinical practice, however, much uncertainty remained regarding its relative cost-effectiveness. This illustrates that outcomes research may not always be the best option to reduce uncertainty regarding the real-world clinical and economic performance of an expensive drug. The actual value of each policy option depends on the type of evidence required and the type of uncertainty addressed.

Margreet Franken, MSc, a researcher at the Erasmus University and lead author of the study, states “Policy makers should carefully consider which type of policy, for example a finance or an outcomes-based risk-sharing agreement, is most appropriate to ensure sufficient value for money of expensive drugs.”  The full study, “Policymaker, Please Consider Your Needs Carefully: Does Outcomes Research in Relapsed or Refractory Multiple Myeloma Reduce Policymaker Uncertainty Regarding Value for Money of Bortezomib?” is published in Value in Health.


Value in Health (ISSN 1098-3015) publishes papers, concepts, and ideas that advance the field of pharmacoeconomics and outcomes research as well as policy papers to help health care leaders make evidence-based decisions. The journal is published bi-monthly and has over 8,000 subscribers (clinicians, decision-makers, and researchers worldwide).

International Society for Pharmacoeconomics and Outcomes Research (ISPOR) is a nonprofit, international, educational and scientific organization that strives to increase the efficiency, effectiveness, and fairness of health care resource use to improve health.

For more information: www.ispor.org

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