PCN152 Patient-Relevant Endpoints (PRE) in Oncology - Current Issues in the Context of Early Benefit Assessment (EBA) in Germany: An Industry Perspective

Abstract

determination. 22 drugs met NICE's EOL-SPP for the indication for which they were being appraised. Twelve of these drugs had the EOL-SPP criterion applied to the only indication for which they were licensed. The EOL-SPP criterion was applied to the cumulative populations of ten drugs which had marketing authorization for more than one indication. The seven drugs that did not meet the EOL-SPP criterion all had individual indications which were within the number of what is considered acceptable (Յ7,000), but had total cumulative populations that were greater. Two STAs in particular stand out. The appraisal committee accepted that panitumumab met the EOL-SPP criterion for its current indication but noted that the EMA recommended a marketing extension which would raise the expected patient population to 10,000. In its final appraisal determination for abiraterone NICE overturned its original decision that the drug did not meet the EOL-SPP criterion, even though it noted that abiraterone may be recommended for a marketing extension for a greater patient population. CONCLUSIONS: There is no evidence to suggest NICE applies the EOL-SPP to the cumulative populations of currently licensed indications plus potential future indications. Estimates of the quality-adjusted life-years (QALYs) gained and incremental cost per QALY gained were then compared the assessments of the Amélioration du Service Médical Rendu (ASMR) made by the Haute Autorité de Santé (HAS) in France for the same drugs in the same clinical indications. RESULTS: In the UK, the estimates of QALYs gained ranged from 0.018 to 1.85 and estimates of incremental cost-per QALY from £1800 to £458,000. The estimate of incremental cost per QALY was a good predictor of the level of restriction imposed on the use of the drug concerned. Patient access schemes, which normally imply price reductions, were proposed in 45% of cases. In France, the distribution of ASMRs was 1, 16%; 2, 8%; 3, 21%; 4, 24%; 5, 24%; and uncategorized/ non-reimbursed, 8%. Since ASMRs of 4 and above signify minor or no improvement over existing therapy, these ratings imply that, in around half the cases, the drugs concerned would face price controls. Overall, the assessments of value added in the two jurisdictions produced very similar results. A superior ASMR rating was a good predictor of both higher QALYs gained and a lower incremental cost per QALY. CONCLUSIONS: We conclude that, despite the contrasting approaches employed in France and the UK for assessing the value added by new drugs, the overall assessments of value added produced very similar results. However, the implications of these assessments for patient access to, and prices of, anticancer drugs in the two jurisdictions require further investigation.