PIH39 - Patient-Reported Outcomes within Health Technology Assessment Decision Making: Current Status and Implications for Future Policy

Abstract

In contrast to regulatory settings, research on using patient-reported outcome (PRO) data within health technology assessment (HTA) is limited. The objectives of this research were to: 1) document PRO guidance within HTA; 2) explore manufacturers’ compliance with this; 3) understand the HTA body’s acceptance of deviations from the guidance; and 4) identify areas of improvement within this process to close the gap between HTA and PRO research. After identifying PRO-related guidance from the National Institute for Health and Care Excellence (NICE), documentation on new single technology appraisals (STAs) published throughout 2014 was reviewed to identify PRO data usage and its compliance with NICE’s guidance. Reviews of existing STAs, and medical device STAs were excluded. PRO data supporting cost-effectiveness and clinical-effectiveness was explored. NICE published new guidance on 19 pharmaceutical products throughout 2014; 16 documented a recommendation. Regarding cost-effectiveness, PRO approaches mostly adhered to NICE guidance, with 87% of recommended STAs measuring health-related quality of life (HRQoL) using the EQ-5D. However, transparency regarding the valuation of HRQoL appears to be lacking; 52% of submissions did not provide the valuation method. Over half (62%) of recommended guidance documents cited PRO data to support clinical-effectiveness. Despite NICE guidance on outcome measures used to support clinical-effectiveness (requesting evidence of reliability or validity) this was only mentioned by the manufacturer for 25% of the measures used. Interestingly, neither the evidence review group nor committee made any comment regarding this disconnect. Although PRO data’s role in supporting cost-effectiveness is clearly guided and adhered to, transparency issues remain. In contrast, clinical-effectiveness guidance is vague and compliance is very low which appears to be currently unrecognised as an issue. Therefore, a more stringent approach is needed when assessing PRO data within HTA, to ensure accurate measurement of treatment effectiveness to inform better decision making.