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Table 3 Input data for the economic impact evaluation of CUP

From: The economic impact of compassionate use of medicines

# of CUP

Molecule

Indication (short)

Standard of Care

Combination medicines

Diagnostic test

Hospitilisations for side effects*

MO29499

Alectinib

Non-Small Cell Lung Cancer 1

Ceritinib (1)

Crizotinib (2)

-

Partially used with the SoC

0/21

MO40066

Alectinib

Non-Small Cell Lung Cancer 2

Docetaxel (1)

Pemetrexed low dosage (2)

Pemetrexed high dosage (3)

Ceritinib (4)

-

Used with the SoC

0/226

ML39740

Atezolizumab

Urothelial Carcinoma

Docetaxel (1)

Nivolumab (2)

Pembrolizumab (3)

-

Used with the SoC

7/222 (3.2 %)

AL41528

Atezolizumab

Non-Small Cell Lung Cancer

Pemetrexed low dosage (1)

Pemetrexed high dosage (2)

Bevacizumab+carboplatino+paclitaxel (3)

-

Used with the SoC

1/125 (0.8 %)

AL41712

Atezolizumab

Triple-Negative Breast Cancer

Nab-paclitaxel

Nab-paclitaxel

Covered by the sponsor

Covered by the sponsor

2/41 (4.9 %)

M029476

Cobimetinib

Melanoma

Nivolumab (1)

Vemurafenib (2)

Dabrafenib + trabetinib (3)

Vemurafenib

Not covered by the sponsor

Used with the SoC

14/228 (6.1 %)

MA30130

Ocrelizumab

Primary Progressive Multiple Sclerosis

Rituximab

-

Not covered by the sponsor

13/1,045 (1.2 %)

AG40852

Entrectinib

Solid tumours (NTRK)

No

-

Not covered by the sponsor

Not included in the analysis

Non-Small Cell Lung Cancer

Crizotinib

-

Used with the SoC

0/5

AG40661

Polatuzumab Vedotin

Diffuse Large B-Cell Lymphoma

Bendamustine + rituximab (1)

Lenalidomide (648 List) (2)

Bendamustine + rituximab

Covered by the sponsor

-

20/151 (13.2 %)

AG41381

Risdiplam

Spinal Muscular Atrophy Type 1

Nusinersen

-

-

0/28

AG42025

Risdiplam

Spinal Muscular Atrophy Type 2

No

-

-

Not included in the analysis

AL41711

Trastuzumab emtansine

Breast Cancer

Trastuzumab + Capecitabina (1)

Lapatinib +

Capecitabina (2)

-

Not covered by the sponsor

0/621

Total

 

2,713 patients with SoC

-

-

57/2,713 (2.1 %)

  1. * # of patients / % of patients recruited