Concerns about cancer drug affordability explain the need to base reimbursement and pricing decisions on clinical added value (CAV). A special attention is given to the CAV assessment by Health Technology Assessment (HTA) bodies. In 2015, The European Society for Medical Oncology (ESMO) published the ESMO Magnitude of Clinical Benefit Scale (ESMO-MCBS), to assess CAV of drugs in solid tumors. For more than 20 years, the French National Authority for Health (HAS), which is responsible for HTA, assesses CAV on a 5-point scale from I (major CAV) to V (no CAV). While the HAS CAV scale is used for pricing, it is not used for reimbursement decision. The opportunity for HAS to use the ESMO scale should be further explored.
To compare both the ESMO-MCBS CAV assessment and that done by HAS, we reviewed the 77 trials that were tested by the ESMO Task Force in its 2015 publication.
Of the 77 trials tested using ESMO-MCBS, 59 were also appraised by HAS, 16 were not assessed and 2 are still under assessment by the HAS. In about 39% (23) of the 59 indications considered, the HAS CAV appraisal was consistent with the ESMO ranking. When an inconsistency was observed (36, 61%), the HAS CAV assessment was systematically more strict. No indication had a higher score than the ESMO evaluation under the HAS evaluation. In 37 indications (62%), the ESMO-MCBS indicated a substantial improvement in CAV. Of these, 48% (13/37) obtained a similar ranking by the HAS. Thirteen indications were downgraded for toxicity by the HAS while only two were downgraded for that criteria under the ESMO-MCBS. The HAS gave an unfavorable opinion for reimbursement for 4 indications that obtained the lowest scores on ESMO-MCBS.
In most cases, disparities between the ESMO-MCBS and the HAS assessment of CAV were observed. The HAS appears to be more demanding in its appraisals, which are mainly based on the level of evidence, comparator relevance, transposability of the results and safety. In the case of a health technology evaluation, the possibility of an unfavorable opinion for drug access should be added in the ESMO-MCBS. As CAV impacts drug prices, the use of the ESMO-MCBS might have led to an increase in French cancer expenses. Nevertheless, the ESMO-MCBS provides an objective tool for HTA bodies to rate CAV.
Clinical trial identification
Legal entity responsible for the study
All authors have declared no conflicts of interest.