OBJECTIVES : We analyzed the attributes of biologics appraised for severe asthma to understand the challenges for acceptance.

METHODS : Health technology assessment (HTA) reports (including re-submissions) from NICE, CADTH & ICER were systematically searched by two independent reviewers. Multiple technology appraisals and requests for advice were excluded. The HTA agencies recommendations along with their rationale and limitations (if any) were extracted.

RESULTS : Reports were available for omalizumab (OMA), mepolizumab (MEPO) and reslizumab (RES). NICE and CADTH recognized the potential benefits of biologics and considered the endpoints assessed in the clinical trials to be relevant. The cost-effectiveness models submitted to NICE had uncertainties around age at onset and mortality benefits, and underestimated the sparing effect of oral corticosteroids. CADTH highlighted a limited target population, high cost, and potential over prescribing. The indirect treatment comparisons of MEPO & RES with OMA in overlapping patient populations were not methodologically robust and had high degree of uncertainty, thus warranting the need for head-to-head trial data. The appraisal of MEPO by ICER considered the limited long-term follow-up data and an uncertainty around the projected budget impact as the major hurdles for reimbursement. Price negotiations and patient access schemes have helped pharmaceutical industries to gain access in the UK and Canada. NICE recommended all three biologics and reported an ICER of £29,870, £29,163 and £23,200 per QALY for RES, MEPO and OMA, respectively, in allergic and eosinophilic asthma. CADTH suggested an ICER of $50,000 per QALY for RES and MEPO after substantial discounts (~90%) and $63,000 per QALY for OMA.

CONCLUSIONS : While biologics offer a much-needed treatment option for patients with severe asthma, however, access controls, limited real-world data and high prices can restrict access. The strict approach to assessments despite robust clinical data present challenges for upcoming biologics in severe asthma.