INFLUENCE OF PATIENT-REPORTED OUTCOMES ON HTA REIMBURSEMENT DECISIONS

Author(s)

Kulich K1;Versoza L*2;Jaksa A2;Demuro-Mercon C3, Gnanasakthy A4 1Novartis Pharma AG, Basel, Switzerland, 2Context Matters, Inc., New York, NY, USA, 3RTI Health Solutions, Research Triangle Park, NC, USA, 4Novartis Pharmaceuticals, East Hanover, NJ, USA

OBJECTIVES: To understand how patient-reported outcomes (PROs) influence decisions made by Health Technology Assessment (HTA) agencies. METHODS: Reports from five HTA agencies that make reimbursement decisions (NICE, HAS, SMC, PBAC and CADTH’s CDR) were selected. The reports, taken from January 2005-April 2013, cover disease conditions in neurological and respiratory therapeutic areas. PROs within the HTA reports were identified and four analysts independently examined the stated rationales for the agencies’ decisions to determine whether PROs had a positive, negative, or neutral influence on the decision. Discrepancies between the analysts were discussed in-depth until consensus was reached. RESULTS: A total of 262 HTA reports were analyzed from the five agencies selected.  PROs were mentioned in 34% of the HTA reports, and were the primary endpoint in 6%. Twenty-five (10%) reports mentioned PRO in the clinical rationale for their decisions. Twelve of the 25 HTA reports (48%) contained PRO as a primary outcome, indicating that a PRO is more likely to influence the decision-making process if it is a primary outcome (p <0.001).  In 12 out of the 25 HTA reports, the clinical rationale for the decision stated that, for the PRO, the drug performed better than placebo or comparator. Ten (83%) of these reports resulted in a positive decision. In eight reports, the drug compared equivalently or unfavorably to placebo or comparator for the PRO and resulted in four (50%) positive decisions. There was no significant difference in agency decisions between the reports that positively reported PROs and the reports with negative or similar PRO results (p=0.16). CONCLUSIONS: In respiratory and neurological diseases, the use of PROs is more likely to influence decision-making by HTA agency when PROs are specified as primary outcomes. Future research directions include comparing these findings to the effect other clinical outcomes have on reimbursement decisions.

Conference/Value in Health Info

2013-11, ISPOR Europe 2013, The Convention Centre Dublin

Value in Health, Vol. 16, No. 7 (November 2013)

Code

PHP163

Topic

Health Technology Assessment

Topic Subcategory

Decision & Deliberative Processes

Disease

Multiple Diseases

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