COMPARING HOW SINGLE ARM PHASE II TRIAL DATA CAN SUPPORT APPROVAL OF ONCOLOGICS BY EUROPEAN HEALTH TECHNOLOGY ASSESSMENT BODIES

Author(s)

Macaulay R
HERON Commercialization, London, UK

OBJECTIVES: The European Medicines Agency (EMA) approved 15 oncologics across 24 indications based on pivotal single-arm Phase II data (Macaulay, ISPOR Dublin 2013). Approval was typically granted for indications in which there was no therapeutic alternative where a response rate of ≥35% was demonstrated. This research aims to compare how such data can further support approval between different European Health Technology Agencies (HTAs).  METHODS: Relevant National Institute of Health and Care Excellence (NICE), Scottish Medicines Commission (SMC), Commission de la Transparence (CT), Institute for Quality and Efficiency in Health Care (IQWiG), Federal Joint Committee (G-BA), and Swedish Dental and Pharmaceutical Benefits Agency (TLV) reports were sourced for any oncologic approved by the EMA on the basis of pivotal Phase II data (up to March 2014) and the decision and key rationale were analysed.  RESULTS: CT fully reimbursed 14/14 (100%) oncologics appraised on the basis of pivotal Phase II data, with 10/14 obtaining ASMRs I-III. In Germany (IQWiG), 6/6 (100%) oncologics appraised on this basis were deemed to offer some added benefit, avoiding reference pricing (5/6 were orphan drugs which are not subject to a benefit assessment). NICE approved 5/7 (71%), SMC 6/11 (55%), and TLV 7/7 (100%) of oncologics appraised on Phase II data. For NICE/SMC/TLV rejected drugs, the clinical case was not strongly criticised, rather cost-utility values were deemed too high and uncertain. Even for approved drugs, the lack of comparative data was critiqued as introducing considerable uncertainty to submissions. CONCLUSIONS: For any oncologic approved by the EMA on the basis of Phase II data, favourable ASMR and benefit ratings can be awarded on this basis by the CT and IQWiG/G-BA, respectively. NICE, SMC, and TLV recommendations are conditional on cost-effectiveness being adequately demonstrated with additional price discounts required to offset inherent uncertainties in cost-utility modelling from such limited clinical data.

Conference/Value in Health Info

2014-11, ISPOR Europe 2014, Amsterdam, The Netherlands

Value in Health, Vol. 17, No. 7 (November 2014)

Code

PCN255

Topic

Health Policy & Regulatory, Health Technology Assessment

Topic Subcategory

Decision & Deliberative Processes, Reimbursement & Access Policy

Disease

Oncology

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