A REVIEW OF THE USE OF PROS IN SUBMISSIONS TO NICE
Author(s)
Lenderking WRUnited BioSource Corporation, Lexington, MA, USA
Presentation Documents
OBJECTIVES: To review the use of Patient Reported Outcomes (PROs) in HTA submissions to date, with respect to the types of findings based on PROs, and the quality of the evaluations of the PRO-based evidence. METHODS: A review of the NICE website with respect to HTA appraisals that incorporated a PRO as part of the evidence base. Focus was on appraisals that departed from the 2007 reference case, and the use of the EQ-5D. RESULTS: At the time of review, 142 appraisals had been published, with 59 in progress. In particular, two case studies were identified, somatropin in growth hormone deficiency and Alzheimer’s disease. In GH deficiency, QOL was the primary outcome of interest and 23 scales were evaluated across 17 RCTs. In spite of pooling and use of generic instruments, there was insufficient evidence to conclude that somatropin had an effect on quality of life (QOL). In observational study, EQ-5D results were 40% higher than disease-specific QOL-AGHDA; committee recommended use of QOL-AGHDA, and treatment guidelines that recommended treatment for fewer patients than currently being treated. In Alzheimer’s, multiple scales used, but treatment benefits on QOL less clear (with donepezil for example, one study showed benefit, one was neutral, one showed worsening). CONCLUSIONS: PRO information has been incorporated in HTA appraisals, but there are significant limitations on the quality of evidence using PROs, due to study design (small sample size primarily), and lack of evidence for mapping value based on PROs.
Conference/Value in Health Info
2009-10, ISPOR Europe 2009, Paris, France
Value in Health, Vol. 12, No. 7 (October 2009)
Code
PHP79
Topic
Health Technology Assessment
Topic Subcategory
Decision & Deliberative Processes
Disease
Multiple Diseases