Table 1.
Details of the cancer drugs available through the NHS Cancer Drugs Fund prior to the first update in January 2015
| Drug | Site | Indication | Author of index trial [REF] | Primary endpoint (s) | Median age | PS 0/1 (%) | PFS (HR) | OS (HR) | Approval on ASCO criteria | ESMO score | NICE status Jan 2015 | CDF status Jan 2015 | CDF status Nov 2015 |
|---|---|---|---|---|---|---|---|---|---|---|---|---|---|
| Abiraterone | Prostate | 1st line CRPC (pre chemo) | Ryan 2014 [5] | PFS/OS | 71 | 100 | 8.3 (0.53) | 4.3 (0.80) | Yes | 3 | Not approved | Approved | Approved |
| Aflibercept | Bowel | 2nd line metastastic CRC with irinotecan/5FU | Van Cutsem 2012 [6] | OS | 61 | 98 | 2.2 (0.76) | 1.4 (0.82) | No | 1 | Not approved | Removed | Removed |
| Albumin bound paclitaxel | Pancreas | 1st line metastatic pancreatic cancer with gemcitabine | Von Hoff 2013 [7] | OS | 63 | KS > 80 = 92% | 1.8 (0.69) | 1.8 (0.72) | No | 3 | Not approved | Approved | Removed |
| Axitinib | Renal | 2nd line advanced RCC | Motzer 2013 [8] | PFS | 61 | 99 | 2.6 (0.66) | NS | No | 3 | Awaiting appraisal | Approved | Approved by NICE therefore removed |
| Bevacizumab | Cervix | 1st line Metastatic cervical ca | Tewari 2014 [9] | OS/Toxicity | 46 | 96 | 2.3 (0.67) | 3.7 (0.71) | Yes | 3 | Awaiting appraisal | Approved | Approved |
| Bevacizumab | Breast | Metastatic triple negative breast cancer | Miller 2007 [10] | PFS | 56 | 98 | 5.9 (0.6) | NS | No | 2 | Not approved | Approved | Removed |
| Bevacizumab | Bowel | 1st line advanced CRC with 5FU | Cunningham 2013 [11] | PFS | 76 | 91 | 4 (0.53) | NS | No | 2 | Not appraised | Removed | Removed |
| Bevacizumab | Bowel | 1st line metastatic CRC with oxaliplatin based regimen | Saltz 2008 [12] | PFS | 60 | 100 | 1.4 (0.83) | NS | No | 1 | Not approved | Removed | Removed |
| Bevacizumab | Bowel | 1st line metastatic CRC with irinotecan based regimen | Hurwitz 2004 [13] | OS | 59 | 99 | 4.4 (0.54) | 4.7 (0.66) | Yes | 3 | Not approved | Removed | Removed |
| Bevacizumab | Bowel | 2nd line/3rd line metastatic CRC with oxaliplatin based chemo | Giantonio 2007 [14] | OS | 61 | 96 | 2.6 (0.61) | 2.1 (0.75) | No | 2 | Not approved | Approved | Removed |
| Bevacizumab | Bowel | 3rd line in low grade gliomas of childhood with irinotecan | Gururangan 2013 [15] | PFS | 8.4 | KS > 50 = 100% | PFS 85% at 6 months, 48% at 2 yrs | NR | Uncertain | 2 | Not appraised | Approved | Approved |
| Bevacizumab | Ovarian | 1st line advanced ovarian, peritoneal or fallopian cancer | Burger 2011 [16] | PFS | 60 | 93 | 3.8 (0.72) | NS | No | 3 | Not approved | Approved | Approved |
| Bevacizumab | Ovarian | 2nd line advanced ovarian, fallopian or primary peritoneal cancers (platinum sensitive) | Aghajanian 2012 [17] | PFS | 61 | 99.5 | 4 (0.48) | NS | No | 3 | Not approved | Removed | Removed |
| Cabazitaxel | Prostate | Metastatic CRPC previously treated with docetaxel | De bono 2010 [18] | OS | 67 | 93 | 1.4 (0.7) | 2.4 (0.7) | No | 2 | Not approved | Removed | Reinstated |
| Cabozantinib | Thyroid | 1st line advanced medullary thyroid cancer | Elisei 2013 [19] | PFS | 55 | 56% PS = 0 | 7.2 (0.28) | NS | Yes | 2 | Awaiting appraisal | Approved | Approved |
| Cetuximab | Head and Neck cancer | Advanced Head and Neck Cancer | Vermoken 2008 [20] | OS | 57 | KS > 80 = 80% | 2.3 (0.54) | 2.7 (0.8) | No | 3 | Not approved | Approved | Approved |
| Cetuximab | Bowel | 1st line metastatic CRC (K ras wild type) with oxaliplatin or irinotecan based regimens | Tejpar 2012 [21] | Pooled subgroup analysis | 60 | 95 | 1.4 (0.47) | NS | Yes | 3 | Not approved for all indications | Approved | Approved |
| Cetuximab | Bowel | 2nd or 3rd line treatment of metastatic CRC (K ras wild type) with irinotecan | Sobrero 2008 [22] | OS | 61 | 94 | 2.4 (0.69) | NS | No | 1 | Not approved | Removed | Removed |
| Cetuximab | Bowel | 3rd or 4th line metastatic CRC (K ras wild type) as single agent | Karapetis 2008 [23] | OS | 63 | 80 | 1.8 (0.40) | 4.7 (0.55) | Yes | 4 | Not approved | Approved | Removed |
| Crizotinib | Lung | 2nd line ALK +ve advanced/metastastic NSCLC | Shaw 2013 [24] | PFS | 51 | 92 | 4.7 (0.49) | NR | Yes | 4 | Not approved | Approved | Approved |
| Dabrafenib | Melanoma | Unresectable or metastatic melanoma with a BRAF V600 mutation and intolerance to Vemurafenib | Hauschild 2014 [25] | PFS | 52 | 66% PS = 0 | 2.4 (0.3) | NR | Yes | 4 | Appraisal ongoing | Approved | Approved by NICE therefore removed |
| Enzalutamide | Prostate | 1st line CRPC (pre-chemo) | Beer 2014 [26] | PFS/OS | 72 | 100 | >12 months (0.19) | 2.2 (0.71) | Uncertain | 3 | Awaiting appraisal | Approved | Approved |
| Eribulin | Breast | 3rd line metastatic breast cancer | Cortes 2011 [27] | OS | 55 | 92 | 1.5 (0.87) | 2.5 (0.81) | No | 2 | Not approved | Removed | Reinstated |
| Everolimus | Breast | Metastatic breast cancer in combination with exemestane | Baselga 2012 [28]/Piccart 2014 [29] | PFS | 62 | 96 | 4.6 (0.43) | NS | No | 2 | Not approved | Removed | Reinstated |
| Everolimus | PNET | 1st or 2nd line moderately differentiated PNET | Yao 2011/2014 [30,31] | PFS | 58 | 97 | 6.4 (0.27) | NS | No | 2 | Awaiting appraisal | Approved | Removed |
| Everolimus | PNET | Well differentiated PNET | Yao 2011/2014 [30,31] | PFS | 58 | 97 | 6.4 (0.27) | NS | No | 2 | Awaiting appraisal | Removed | Removed |
| Everolimus | Renal | Metastatic RCC | Motzer 2010 [32] | PFS | 61 | KS > 80 = 91% | 3 (0.33) | NS | No | 3 | Not approved | Removed | Reinstated |
| Imatinib | Sarcoma (GIST) | Adjuvant therapy for completely resected GIST at high relapse risk | Dematteo 2009 [33] | RFS | 59 | 99 | RFS gain 13% | NR | Yes | A | Awaiting appraisal | Approved | Approved by NICE therefore removed |
| Lapatinib | Breast | Advanced breast cancer, Her 2 +ve | Geyer 2006 [34]/cameron 2008 [35] | PFS | 54 | 100 | 4 (0.47) | NS | No | 3 | Not approved | Removed | Removed |
| Panitumimab | Bowel | 3rd or 4th line metastatic CRC (Kras wild type) as single agent | Van Cutsem 2007 [36] | PFS | 62 | 82 | 1.1 (0.54) | NS | No | 2 | Not approved | Approved | Removed |
| Panitumimab | Bowel | 1st line metastatic CRC with Folofox 4 or irinotecan (Kras wild type) | Douillard 2010 [37]/Douillard 2014 [38] | PFS | 62 | 94 | 1.4 (0.80) | 4.4 (0.88) | Yes | 3 | Awaiting appraisal | Approved | Approved |
| Pazopinib | Sarcoma | Advanced non-adipocytic soft tissue carcinoma | van der Graaf 2012 [39] | PFS | 56 | 100 | 3 (0.31) | NS | No | 2 | Not appraised | Removed | Removed |
| Pegylated liposomal doxirubicin | Sarcoma | 1st or 2nd line for angiosarcoma | Judson 2001 [40] | Toxicity/RR | 52 | 86 | NA | NA | Uncertain | Uncertain | Not appraised | Removed | Removed |
| Pegylated liposomal doxirubicin | Sarcoma | For patients with sarcoma with cardiac impairment or contraindication to doxorubicin | Judson 2001 [40] | Toxicity/RR | 52 | 86 | NA | NA | Uncertain | 4 | Not appraised | Approved | Approved |
| Pemtrexed | Lung | Maintenance post 4 cycles cisplatin/pemetrexed for NSCLC | Ciuleanu 2009 [41] | PFS | 60 | 100 | 1.7 (0.5) | 2.8 (0.79) | Yes | 2 | Not approved | Removed | Reinstated |
| Pemetrexed | Lung | 2nd line NSCLC | Hanna 2004 [42] | OS | 59 | 88 | NS | NS | No | 1 | Not approved | Removed | Removed |
| Lutetium Octreotate/Yttrium Octreotide | NETs | For inoperable well-differentiated neuroendocrine tumours (NETs) with progressive disease | Kam 2012 [43] | Symptoms/tumour regression | NR | NR | NA | NA | No | 1 | Not appraised | Approved | Removed |
| Pertuzumab | Breast | 1st line locally advanced Her2 +ve breast cancer | Swain 2013 [44] | PFS | 54 | 99 | 6.3 (0.69) | 15.7 (0.68) | Yes | 4 | Not approved | Approved | Approved |
| Radium 223 | Prostate | CRPC and bone mets, no visceral mets | Parker 2013 [45] | OS | 71 | 87 | NR | 3.6 (0.7) | Yes | 5 | Awaiting appraisal | Approved | Approved by NICE therefore removed |
| Regorafinib | Sarcoma (GIST) | Imatinib and sunitinib resistant GIST | Demetri 2013 [46] | PFS | 60 | 100 | 3.9 (0.27) | NR | No | 3 | Not appraised | Removed | Reinstated |
| Sorafenib | Liver | 1st line HCC | Llovet 2008 [47] | OS/Time to symptom progression | 65 | 92 | 2.7 (0.58) | 2.8 (0.69) | Yes | 3 | Not approved | Approved | Approved |
| Sorafenib | Thyroid | Metastatic/inoperable thyroid cancer refractory to radioiodine | Brose 2014 [48] | PFS | 63 | 97 | 5 (0.8) | NR | Yes | 2 | Not appraised | Approved | Approved |
| Sunitinib | PNET | well differentiated PNET | Raymond 2011 [49] | PFS | 56 | 100 | 5.9 (0.42) | Trial stopped early | Uncertain | 2 | Not appraised | Approved | Approved |
| Temsirolimus | Renal | Advanced RCC | Hudes 2007 [50] | OS | 59 | KS > 70 = 17% | 2.4 | 3.6 (0.73) | Yes | 4 | Not approved | Approved | Approved |
| Trastuzumab emtansine (Kadcyla) | Breast | Relapsed Her 2 +ve breast cancer | Verma 2012 [51] | PFS/OS and safety | 53 | 99 | 3.2 (0.65) | 5.8 (0.68) | Yes | 5 | Not approved | Approved | Approved |
| Vandetinib | Thyroid | Medullary thyroid ca | Wells 2012 [52] | PFS | 51 | 96 | 11.2 (0.46) | NR | Yes | 2 | Not appraised | Approved | Approved |
| Vismodegib | Skin | Metastatic basal cell cancer | Sekulic 2012 [53] | Objective response rate | 62 | NR | NA | NA | Uncertain | 2 | Not appraised | Approved | Approved |
OS, overall survival (months); PFS, progression free survival (months); HR, median hazard ratio; TTP, Time to progression; ASCO, American Society of Clinical Oncology; ESMO, European Society of Medical Oncology; PS, Eastern cooperative oncology group performance score; KS, Karnofsky score; NR; not reported; NS, not statistically significant; NA, endpoint not assessed; CRC, colorectal cancer; RCC, renal cell cancer; CRPC, castrate resistant prostate cancer; PNET, pancreatic neuroendocrine tumour; HCC, hepatocellular carcinoma; GIS, gastrointestinal stromal tumour; NSCLC, non small cell lung cancer.