PCN232 DIVERGENT HTA DECISIONS IN EUROPE FOR ONCOLOGY DRUGS GRANTED BREAKTHROUGH DESIGNATION

Abstract

The Food and Drug Administration Safety and Innovation Act (FDASIA) introduced in 2012 the Breakthrough Therapy Designation (BTD) to expedite approval of new health care technologies for use by patients with serious or life-threatening diseases/conditions. From 77 BTDs, 35 were granted for oncology indications so far. The aim of this study is compare the HTA decisions in the three biggest EU agencies for BTD oncology medicines. The FDA website was examined to identify oncology drugs granted BTD by 1 December 2018. The NICE (England), IQWiG/GBA (Germany) and HAS/TC (France) websites were reviewed to compare the decisions. Out of 35 oncology breakthrough medicines 33 drugs has been submitted to NICE, with 21 recommended for all indications, 7 recommended for some indications, 5 decision pending, one was rejected and for 2 information was not found. 27 were submitted to IQWiG with 3 considerable additional benefit, 7 additional benefit not proved, 4 additional benefit low, 5 additional benefit not quantifiable, 6 considerable additional benefit for some indications and not proved or not quantifiable for others and 2 procedures pending. For 8 BTDs drugs from the list no results were found. In France information was only available for 17 BTDs recommended by the TC for inclusion in the list of reimbursable products: 2 with ASMR rating III, 2 with ASMR rating IV, 2 with ASMR rating V, 2 with ASMR rating III and V, 7 with ASMR rating III and IV and 1 with ASMR rating IV and V. One has been claimed to be with insufficient clinical benefit to justify reimbursement. Overall, only 5 matching positive decision were identified between the three authorities. The lack of consistent approach entails heterogeneous HTA decisions for the breakthrough oncology drugs amongst the three main European competent bodies.