3759 - Magnitude of clinical benefit of cancer drugs and time to health technology assessment (HTA) decisions in Europe

Background

A key goal of the European Society for Medical Oncology Magnitude of Clinical Benefit Scale (ESMO-MCBS) is to highlight cancer drugs with the greatest clinical benefit that should be rapidly made available by health authorities. We used ESMO-MCBS scores to determine the correlation with national benefit assessments, and evaluate the drugs’ reimbursement statuses and times to health technology assessment (HTA) decisions.

Methods

We identified all new cancer drugs for solid tumors, and subsequent indications for these drugs, approved by the EMA from January 2007 to December 2016. HTA scores (for France and Germany), appraisal evidence, and reimbursement decisions for England, France, Germany, and Scotland were extracted as of January 2018. ESMO-MCBS v1.1 scores were calculated based on the trials submitted to HTA bodies for appraisal. “Highest benefit” was defined as scores of A-B (neo/adjuvant setting) and 4-5 (palliative setting) on the ESMO-MCBS scale, and scores of “moderate”, “considerable”, or “major” benefit by HTA bodies. Fisher’s exact test, Mann-Whitney test, Cohen’s kappa, and Cox proportional hazards models were used to compare drugs categorized as “highest benefit” versus those that were not.

Results

From 2007-2016, the EMA approved 47 drugs for 77 solid tumor indications. Median times from EMA approval to HTA decision were 188 (France), 209 (Germany), 384 (Scotland), and 405 (England) days. Drugs categorized as “highest benefit” by ESMO-MCBS were associated with shorter times to HTA decision in France (median 154 vs. 198 days; hazard ratio [HR] 1.82; 95% confidence interval [CI] 1.06-3.13; p=0.03), Germany (203 vs. 213 days; HR 2.24; CI 1.27-3.93; p=0.005), and England (302 vs. 413 days; HR 1.95; CI 1.10-3.46; p=0.02) but not Scotland (349 vs. 402 days; HR 1.15; CI 0.65-2.03). 90%, 100%, 92%, and 95% of “highest benefit” drugs were reimbursed in France, Germany, England, and Scotland. There was high concordance between ESMO-MCBS and HTA scores for categorization of “highest benefit” (κ 0.8 [Germany], κ 0.6 [France]).

Conclusions

Anticancer drugs with greatest clinical benefit are associated with faster times to HTA decision, and nearly all are approved for reimbursement in Europe.

Clinical trial identification

Editorial Acknowledgement