Table 1.
Characteristics of the 30 anticancer drugs included in the analysis
| Medicine name | Active substance | Clinical setting | Treatment group | Control group | PFS TRT (median in weeks) | PFS CRT (median in weeks) | OS TRT (median in weeks) | OS CRT (median in weeks) | ORR TRT (%) | ORR CRT (%) | Year first authorization | Official negotiated price (€) | Discounted price (€) |
| Teysuno | Tegafur/gimeracil/oteracil | Advanced gastric cancer in combination with cisplatin | Tegafur/gimeracil/oteracil 25 mg/m2+cisplatin 75 mg/m2 |
5-fluorouracil 1000 mg/m2/24+ cisplatin 100 mg/m2 | 34.4 | 31.6 | 2011 | 4942 | 3479 | ||||
| Jevtana | Cabazitaxel | Hormone-refractory metastatic prostate cancer | Cabazitaxel+prednisone | Mitoxantrone +prednisone |
11.2 | 5.6 | 60.4 | 50.8 | 14.4 | 4.4 | 2011 | 52 983 | 38 254 |
| Yervoy | Ipilimumab | Advanced (unresectable or metastatic) melanoma | Ipilimumab+Peptide vaccin | Peptide vaccine glycoprotein 100 (gp100) | 11.04 | 11.04 | 39.8 | 25.8 | 5.7 | 1.5 | 2011 | 71 400 | 45 107 |
| Votubia | Everolimus | Renal angiomyolipoma associated with tuberous sclerosis complex (TSC) | Everolimus | Placebo | 45.48 | 41.8 | 0 | 2011 | 66 521 | 41 424 | |||
| Votubia | Everolimus | Subependymal giant cell astrocytoma associated with TSC | Everolimus | Placebo | 34.6 | 0 | 53 216 | 33 139 | |||||
| Halaven | Eribulin | Locally advanced or metastatic breast cancer | Eribulin 1.23 mg/m2 (equivalent to 1.4 mg/m2 eribulin mesylate) | Treatment of physician's choice | 16.14 | 9.71 | 57.57 | 45.86 | 12.2 | 4.7 | 2011 | 32 300 | 28 130 |
| Zytiga | Abiraterone acetate | Metastatic castration-resistant prostate cancer | Abiraterone acetate | Placebo | 22.4 | 14.4 | 68.9 | 48.7 | 29.1 | 5.5 | 2011 | 46 842 | 33 397 |
| Dacogen | Decitabine | Newly diagnosed de novo or secondary acute myeloid leukaemia | Decitabine | Patient's choice | 14.8 | 8.4 | 30.8 | 20 | 2012 | 54 366 | 34 346 | ||
| Caprelsa | Vandetanib | Aggressive and symptomatic medullary thyroid cancer unresectable locally advanced or metastatic disease | Vandetanib | Placebo | 122 | 77.2 | 45 | 13 | 2012 | 67 405 | 53 533 | ||
| Zelboraf | Vemurafenib | BRAF-V600-mutation-positive unresectable or metastatic melanoma | Vemurafenib | Dacarbazine | 21.28 | 6.44 | 52.8 | 39.6 | 48.4 | 5.5 | 2012 | 119 929 | 108 236 |
| Xalkori | Crizotinib | Anaplastic lymphoma kinase (ALK) positive advanced non-small-cell lung cancer (NSCLC) | Crizotinib | Pemetrexed or docetaxel | 30.8 | 12 | 65.3 | 19.5 | 2012 | 79 538 | 57 427 | ||
| Xalkori | Crizotinib | First-line treatment of adults with ALK positive advanced NSCLC | Crizotinib | Chemotherapy | 43.6 | 28 | 74.4 | 45 | 79 538 | 57 427 | |||
| Inlyta | Axitinib | Advanced renal-cell carcinoma (RCC) | Axitinib | Sorafenib | 26.8 | 18.8 | 19.4 | 9.4 | 2012 | 57 632 | 39 295 | ||
| Perjeta | Pertuzumab | HER2-positive metastatic or locally recurrent unresectable breast cancer | Pertuzumab +trastuzumab+docetaxel | Placebo +trastuzumab + docetaxel | 74 | 49.6 | 80.2 | 69.3 | 2013 | 51 643 | 46 608 | ||
| Kadcyla | Trastuzumab emtansine | HER2-positive, unresectable locally advanced or metastatic breast cancer | Trastuzumab emtansine (tdm1) | Lapatinib +capecitabine | 38.4 | 25.6 | 123.9 | 100.4 | 2013 | 87 215 | 75 877 | ||
| Giotrif | Afatinib | Locally advanced or metastatic NSCLC with activating EGFR mutation(s) | Afatinib | Pemetrexed (lyophilised powder)+cisplatin (solution for infusion) | 44.56 | 27.6 | 56.1 | 22.6 | 2013 | 29 528 | 21 853 | ||
| Stivarga | Regorafenib | Metastatic colorectal cancer (MCRC) | Regorafenib+best supportive care | Placebo+best supportive care | 8.4 | 7.4 | 28 | 21.6 | 1 | 0.4 | 2013 | 85 800 | 77 434 |
| Stivarga | Regorafenib | Unresectable or metastatic gastrointestinal stromal tumours | Regorafenib+best supportive care | Placebo+best supportive care | 21 | 4 | 1.5 | 4.5 | 85 800 | 77 434 | |||
| Tafinlar | Dabrafenib | Unresectable or metastatic melanoma with a BRAF V600 mutation | Dabrafenib | Dacarbazine | 27.6 | 10.8 | 72.8 | 62.4 | 59 | 24 | 2013 | 107 935 | 87 670 |
| Zaltrap | Aflibercept | MCRC | Aflibercept+folfiri | Placebo+folfiri | 27.6 | 18.7 | 54 | 48.4 | 19.8 | 11.1 | 2013 | 30 576 | 27 591 |
| Xtandi | Enzalutamide | Metastatic castration resistant prostate cancer | Enzalutamide | Placebo | 33.2 | 11.6.6 | 73.6 | 54.4 | 2013 | 49 184 | 31 960 | ||
| Imnovid | Pomalidomide | Relapsed and refractory multiple myeloma | Pomalidomide+ Ld-dexamethasone |
Hd dexamethasone | 15.7 | 8 | 34 | 2013 | 127 985 | 101 646 | |||
| Lynparza* | Olaparib | Platinum-sensitive relapsed BRCA-mutated (germline and/or somatic) high-grade serous epithelial ovarian, fallopian tube or primary peritoneal cancer | Olaparib | Placebo | 33.6 | 19.2 | 119.2 | 111.2 | 2014 | 70 517 | 52 142 | ||
| Cyramza | Ramucirumab | Advanced gastric cancer or gastro-oesophageal junction adenocarcinoma with disease progression | Ramucirumab +paclitaxel | Placebo+paclitaxel | 17.6 | 11.6 | 38.4 | 29.6 | 27.9 | 16.1 | 2014 | 87 360 | 78 842 |
| Mekinist | Trametinib | Unresectable or metastatic melanoma with a BRAF V600 mutation | Trametinb | Chemotherapy (dacarbazine or paclitaxel) | 19.6 | 6 | 62.4 | 45.2 | 19 | 5 | 2014 | 62 398 | 28 157 |
| Imbruvica | Ibrutinib | Chronic lymphocytic leukaemia (CLL) | Ibrutinib | Chlorambucil | 75.6 | 82.4 | 35.3 | 73 805 | 51 663 | ||||
| Zydelig | Idelalisib | CLL | Idelalisib+rituximab | Placebo+rituximab | 22 | 74.5 | 14.5 | 2014 | 48 667 | 34 067 | |||
| Sylvant | Siltuximab | Multicentric Castleman disease | Siltuximab+best supportive care | Placebo+best supportive care | 37.7 | 3.8 | 2014 | 66 104 | 29 829 | ||||
| Keytruda | Pembrolizumab | Advanced (unresectable or metastatic) melanoma | Pembrolizumab | Ipilimumab | 22 | 11.2 | 33.7 | 11.9 | 2015 | 90 400 | 81 586 | ||
| Opdivo | Nivolumab | Advanced (unresectable or metastatic) melanoma | Nivolumab 3 mg/kg | Investigator choice I | 18.8 | 16.8 | 31.7 | 10.6 | 2015 | 81 310 | 71 181 | ||
| Opdivo | Nivolumab | Locally advanced or metastatic NSCLC | Nivolumab 3 mg/kg | Docetaxel 75 mg/m2 | 9.32 | 16.8 | 48.8 | 37.4 | 19.2 | 12.4 | 81 310 | 71 181 | |
| Opdivo | Nivolumab | Advanced RCC | Nivolumab 3 mg/kg | Everolimus 100 mg | 18.4 | 17.8 | 100 | 78.2 | 25.1 | 5.4 | 81 310 | in negotiation | |
| Lenvima | Lenvatinib mesylate | Progressive, locally advanced or metastatic, differentiated (papillary/follicular/Hurthle cell) thyroid carcinoma, refractory to radioactive iodine | Lenvatinib | Placebo | 73.2 | 14.4 | 64.8 | 1.5 | 2015 | 68 433 | 58 673 | ||
| Cotellic | Cobimetinib hemifumarate | Unresectable or metastatic melanoma with a BRAF V600 mutation | Cobimetinib +vemurafenib | Placebo +vemurafenib | 45.2 | 24 | 67.6 | 44.8 | 2015 | 75 374 | 54 420 | ||
| Kyprolis | Carfilzomib | Multiple myeloma who have received at least one prior therapy | Carfilzomib +lenalidomide +dexamethasone |
Lenalidomide +dexamethasone | 105.2 | 70.4 | 87.1 | 67.7 | 2015 | 75 900 | 44 525 |
*During the evaluation at the EMA, lymparza was found to be more effective in a subgroup of patients with a specific biomarker with the following outcome results: PFS lymparza: 44.8 w; PFS control: 17.2 w; OS lymparza: 139.6 w; OS control: 127.6.
CRT, Control Patients; EGFR, Epidermal Growth Factor Receptor; EMA, European Medicines Agency; ORR, objective response rate; OS, overall survival; PFS, progression free survival; TRT, Patients with treatment.