PDB63 Reimbursement Strategies of First-in-Class Drugs for Type 2 Diabetes Mellitus in Health Technology Assessment in Australia: A Qualitative Review

Abstract

To gain insights into reimbursement strategies of first-in-class anti-diabetic drugs reflected in public summary documents (PSDs) by the Pharmaceutical Benefits Advisory Committee (PBAC) in Australia. A qualitative review was conducted on PSDs for exenatide, sitagliptin and dapagliflozin. Both initial and re-submissions were reviewed. No further reviews were performed once the indications proposed in the initial submission were recommended. Key information was extracted using a standardized data extraction form and summarized by narrative synthesis. All retrieved submissions were focused on combination therapies. All three drugs in combination with metformin (MET) and/or sulfonylurea (SU) were recommended eventually following an average of 2.3 attempts. Indirect comparisons were generally adopted to assess the comparative effectiveness and safety. In initial submissions, thiazolidinediones was nominated as the main comparator for sitagliptin and exenatide, whereas sitagliptin being the comparator for dapagliflozin. HbA1c reduction was the primary endpoint across all comparisons with a non-inferiority margin of 0.3% - 0.4% agreed by the PBAC. Cost-minimization analyses (CMA) were mainly employed except that cost-utility analyses were applied in the first two submissions for exenatide. All initial submissions were deemed to have high uncertainties with respect to comparative effectiveness, and exenatide was further characterized with high uncertainties in cost-effectiveness and safety concerns. These uncertainties were addressed in resubmissions by providing additional clinical evidence and adopting the comparator and the CMA approach suggested by the PBAC. All submissions estimated a net cost to the government of less than $10 million in Year 5 post listing. Furthermore, PBAC suggested a risk-sharing agreement to manage the potential usage beyond the listing of dapagliflozin to assure its nil net cost to the government. The selection of comparator(s) and the robustness of clinical and economic evaluation should be emphasized by the sponsors. Cost neutral and appropriate risk-sharing agreements may facilitate positive recommendations.