Comparative Clinical Effectiveness of Medicinal Products: Evaluation of International Criteria and Application to the NCPE HTA Process

OBJECTIVES: Assessing the comparative clinical effectiveness of pharmaceuticals includes a critical appraisal of their relative efficacy and associated degree of certainty. Certain Health Technology Assessment (HTA) agencies provide an explicit tool-informed conclusion usually in the form of a rating, on the comparative clinical effectiveness. This research aims to review the methods used to rate comparative clinical effectiveness internationally and to apply some of these methods to a selected National Centre for Pharmacoeconomics (NCPE) HTA appraisal.

METHODS: A targeted literature search was conducted to identify methods used to evaluate comparative clinical effectiveness. Strengths and limitations of applying each method were highlighted. A selection of methods (n=3) was applied to the NCPE HTA appraisal of sacituzumab govitecan for the treatment of adult patients with unresectable or metastatic triple negative breast cancer (mTNBC) (HTA ID 22007). This HTA was selected due to the format of outcome data presented and to capture a direct treatment comparison. Methods were chosen based on data availability and suitability of application.

RESULTS:: Methods used by five HTA agencies were identified. Those employed by IQWiG (Germany), HAS (France), and ICER (United States) were considered the most applicable to NCPE processes, given data availability. The application of these methods to the NCPE appraisal of sacituzumab govitecan yielded heterogeneous conclusions. Applying the IQWiG method yielded a conclusion of “indication of major added benefit”, the HAS method yielded “moderate therapeutic improvement”, and the ICER method yielded “B+”. None of the identified methods were readily applicable to this NCPE HTA appraisal due to differences in reporting domains.

CONCLUSIONS: There is an overlap of criteria considered for rating comparative clinical effectiveness internationally. The rationale for how these criteria inform comparative clinical effectiveness ratings is often unclear, leading to heterogeneous conclusions across the methods. Further research will involve the application of these appraisal methods to other NCPE HTA appraisals.