Skip to main content

Table 2 Compassionate Use Programs available in the CUP Database

From: The economic impact of compassionate use of medicines

# of CUP

Molecule

Indication

Indication (short)

# of patients

Starting month of CUP

MO29499

Alectinib

Adult patients with Anaplastic Lymphoma Kinase (ALK)-positive advanced Non-Small Cell Lung Cancer (NSCLC)

Non-Small Cell Lung Cancer 1

21°

March 2015

ML40066

Alectinib

Adult patients with ALK-positive advanced NSCLC, previously treated with crizotinib

Non-Small Cell Lung Cancer 2

226°

May 2017

ML39740

Atezolizumab

Adult patients with locally advanced or metastatic Urothelial Carcinoma (UC) after prior platinum-containing chemotherapy, or considered cisplatin ineligible, and whose tumours have a PD-L1 expression ≥ 5 %

Urothelial Carcinoma

222°

February 2017

AL41528

Atezolizumab

Adult patients with locally advanced or metastatic NSCLC after prior chemotherapy (patients with EGFR mutant or ALK-positive NSCLC should also have received targeted therapies before)

Non-Small Cell Lung Cancer

125°

June 2019

AL41712

Atezolizumab

Adult patients with unresectable locally advanced or metastatic Triple-Negative Breast Cancer (TNBC), whose tumours have PD-L1 expression ≥ 1 % and who have not received prior chemotherapy for metastatic disease

Triple-Negative Breast Cancer

41°

November 2019

M029476

Cobimetinib

Adult patients with unresectable or metastatic Melanoma with a BRAF V600 mutation

Melanoma

228°

May 2015

MA30130

Ocrelizumab

Adult patients with Primary Progressive Multiple Sclerosis (PPMS) (in terms of disease duration and level of disability) and with imaging features characteristic of inflammatory activity

Primary Progressive Multiple Sclerosis

1,045°

June 2017

AG40852

Entrectinib

Adult and paediatric patients 12 years of age and older with solid tumours expressing a Neurotrophic Tyrosine Receptor Kinase (NTRK) gene fusion, who have a disease that is locally advanced, metastatic or where surgical resection is likely to result in severe morbidity, and who have not received a prior NTRK inhibitor or who have no satisfactory treatment options

Solid tumours (NTRK)

1

August 2019

Adult patients with ROS1-positive advanced NSCLC, not previously treated with ROS1 inhibitors

Non-Small Cell Lung Cancer

5°

AG40661

Polatuzumab Vedotin

Adult patients with relapsed/refractory Diffuse Large B-Cell Lymphoma (DLBCL) who are not candidates for haematopoietic stem cell transplant.It is indicated in combination with the treatment of adult patients with bendamustine and rituximab

Diffuse Large B-Cell Lymphoma

151°

May 2019

AG41381

Risdiplam

Patients from 2 months old with 5q spinal muscular atrophy (SMA) Type 1, Type 2 or Type 3, or those who have up to 4 copies of SMN2 gene (Type 1)

Spinal Muscular Atrophy Type 1

28°

January 2020

AG42025

Risdiplam

Patients from 2 months old with 5q Spinal Muscular Atrophy (SMA) Type 1, Type 2 or Type 3, or those who have up to 4 copies of SMN2 gene (Type 2)

Spinal Muscular Atrophy Type 2

31

January 2020

AL41711

Trastuzumab emtansine

Adult patients with HER2-positive, unresectable locally advanced or metastatic Breast Cancer who previously received trastuzumab and a taxane, separately or in combination. Patients should have either: (i) received prior therapy for locally advanced or metastatic disease, or (ii) developed disease recurrence during or within six months of completing adjuvant therapy.

Breast Cancer

621°

September 2019

Total # of patients

2,745

° 2,713 included in the analysis