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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