PATIENT REPORTED OUTCOMES AND THEIR RELEVANCE IN REIMBURSEMENT DECISIONS
Author(s)
Stoddart S1, Lis Y2, Malmenas M3
1PAREXEL International, London, UK, 2PAREXEL International, Uxbridge, UK, 3PAREXEL International, Stockholm, Sweden
OBJECTIVES Payers all need evidence of clinical effectiveness, reasonable cost, and safety. Although, patient-reported outcomes (PROs) are an important component of this evidence it is not always clear which PROs to use and reliance on an incorrect PRO can have negative consequences. The objective was to describe the factors determining which PROs are most likely to meet payer evidence needs. METHODS A structured discussion of factors influencing PRO choice was held between experts in late phase studies, evidence review, and economic modelling. These were then validated against a sample of published reimbursement decisions across multiple disease areas. RESULTS Focus of most questions is on whether to use a generic, disease-specific or novel measure. Disease specific PROs are very frequently of value in demonstrating clinical effectiveness and may be the standard clinical outcome. For example, migraine studies include pain assessment and headache frequency. To facilitate Payers’ decision making generic PROs such as EQ-5D and SF-36 are commonly used for assessing quality of life (QoL). However these instruments may not be reliable in disease areas characterised by focal impacts on patient wellbeing. For example, pain is only a small component of the SF-36 consequently changes in pain levels cannot be measured with great precision and the extreme effects of pain may be missed when generating estimates of utility. CONCLUSIONS When choosing a PRO, it is important to consider the disease, treatment and payer reimbursement decision context. Disease specific QoL measures may be more sensitive than a generic tool but may also need mapping to a general QoL measure. Acceptance will be contingent on the existence of robust evidence either from published literature or additionally collected data. Reviews of previous HTA submissions and payer decisions help identify requirements / standard practices in key markets and identify common criticisms or problems.
Conference/Value in Health Info
2014-11, ISPOR Europe 2014, Amsterdam, The Netherlands
Value in Health, Vol. 17, No. 7 (November 2014)
Code
PHP101
Topic
Economic Evaluation
Topic Subcategory
Cost/Cost of Illness/Resource Use Studies
Disease
Multiple Diseases