Skip to main content

Table 5 ESMO-MCBS scores and comparison of OS before marketing authorization and after marketing authorization

From: Oncologic orphan drugs approved in the EU – do clinical trial data correspond with real-world effectiveness?

Drug Indication Score ESMO-MCBS Median OS of standard of care (before authorization) Median OS of OMP (after authorization) OS gain (before authorization vs after authorization)
Imatinib GIST A 9 – 22 months 41.1 months ≥3 months
Imatinib DFSP n.a. 3-year OS rate 66% 3-year OS rate 60 – 77% No gain
Mitotane Adrenocortical carcinoma n.a. 3 – 9 months No post-marketing studies n.a.
Sunitinib GIST 3 9 – 22 months 14.1 – 17.6 months Gain of unknown magnitude
Sunitinib RCC 4 10 – 13 months 18.2 – 27.2 months ≥3 months
Sorafenib RCC 3 10 – 13 months 17.8 – 29.3 months ≥3 months
Sorafenib HCC 3 < 6 months 5 – 10.2 months ≥3 months
Sorafenib Thyroid carcinoma 2 2.5 – 3.5 years 2.4 – 4.7 years Gain of unknown magnitude
Temsirolimus RCC 4 6 – 12 months 11.6 – 18 months ≥3 months
Trabectedin STS 1 6 months 11.9 – 19.3 months ≥3 months
Trabectedin Ovarian cancer 2 >  30 months 16.3 – 22.2 months No gain
Mifamurtide Osteosarcoma C 13 – 28 months No post-marketing studies n.a.
Everolimus RCC 2 5-year survival rate 9.5% 14.8 – 32 months n.a.
Cabozantinib Medullary thyroid cancer 3 10-year survival rate < 40% No post-marketing studies n.a.
Olaparib Ovarian cancer 2 5-year OS 44% for BRCA1 carriers, 52% for BRCA2 carriers No post-marketing studies n.a.
Ramucirumab Gastric cancer 2 12 months No post-marketing studies n.a.
Dinutuximab Neuroblastoma A0 EFS rates are 30 - 40% for children with high-risk neuroblastoma No post-marketing studies n.a.
Lenvatinib Thyroid cancer 2 2.5 – 3.5 years No post-marketing studies n.a.
Olaratumab STS 4 11 – 15 months No post-marketing studies n.a.
Nanoliposomal irinotecan Pancreatic cancer 2 < 12 months No post-marketing studies n.a.
  1. BRCA breast cancer, DFSP dermatofibrosarcoma protuberans, EFS event-free survival, ESMO-MCBS European Society for Medical Oncology - Magnitude of Clinical Benefit Scale, GIST gastrointestinal stromal tumor, HCC hepatocellular carcinoma, OMP orphan medicinal product, OS overall survival, pNET pancreatic neuroendocrine tumor, RCC renal cell carcinoma, STS soft tissue sarcoma