Table 2 EBAs included in the analysis set and respective primary endpoints

 Abiraterone acetate

Prostate carcinoma

OS

BDU, DGHO, DVPZ

 Abiraterone acetate^a

Prostate carcinoma

OS, rPFS

DGHO

 Afatinib^c

Non-small cell lung cancer

PFS

DGHO, DKG (Working Group on Thoracic Oncology of the AIO)

 Aflibercept

Metastatic colorectal cancer

OS

DGVS

 Axitinib

Renal cell carcinoma

PFS

DGHO

 Bosutinib

Chronic myeloid leukaemia

MCR

DGHO

 Brentuximab vedotin

Hodgkin-Lymphoma, anaplastic large cell lymphoma

ORR

DGHO

 Cabazitaxel

Prostate carcinoma

OS

DGHO, DKG

 Cabozantinib

Thyroid gland neoplasia

PFS

DGE (thyroid gland section), DGHO, DGN

 Crizotinib

Non-small cell lung cancer

PFS, ORR

DGHO, POA, representatives from leading oncology centres

 Decitabine

Myeloid leukaemia

OS

DGHO

 Enzalutamide

Prostate carcinoma

OS

DGHO, DGU

 Enzalutamide^a

Prostate carcinoma

OS, rPFS

DGHO

 Eribulin

Breast cancer

OS

DGHO

 Eribulin^ac

Breast cancer

OS, rPFS

DGHO

 Ibrutinib

Chronic lymphocytic leukaemia, relapsed or refractory mantle cell lymphoma (MCL)

ORR

DGHO

 Idelalisib

Chronic lymphocytic leukaemia, follicular lymphoma

PFS

DGHO, GLSG, representative from the University Hospital Gießen

 Ipilimumab

Melanoma

OS

DGHO

 Lenvatinib

Thyroid gland neoplasia

PFS

DGHO

 Nintedanib

Non-small cell lung cancer

PFS

DGHO, DKG (Working Group on Thoracic Oncology of the AIO), representative from the LungenClinic Grosshansdorf

 Nivolumab

Melanoma

OS, PFS

ADO, DGHO

 Obinutuzumab

Chronic lymphocytic leukaemia

PFS

DGHO

 Olaparib

Ovarian cancer

PFS

DGHO

 Pertuzumab

Breast cancer

PFS, ORR

AGO, DGHO

 Pomalidomide

Multiple myeloma

PFS

DGHO, representatives from the University Hospitals of Heidelberg, Tübingen and Würzburg

 Radium-223-dichloride

Prostate carcinoma

OS

DGHO

 Ramucirumab

Stomach cancer

OS

DGHO, DGVS

 Regorafenib

Colorectal cancer

OS

DGHO

 Ruxolitinib^d

Chronic myeloproliferative diseases

≥35 % reduction in spleen volume

DGHO

 Ruxolitinib^a

Polycythaemia vera

Haematocrit control without phlebotomy and ≥35 % reduction in spleen volume

DGHO

 Siltuximab

Multicentric Castleman’s disease

Durable tumour & symptomatic response (complete and partial response)

DGHO

 Sipuleucel-T

Prostate carcinoma

OS

DGHO, representative from the University Hospital Tübingen, Department of Urology

 Trastuzumab emtansine

Breast cancer

OS, PFS

AGO, DGHO

 Vandetanib^b

Thyroid gland neoplasia

PFS

DGHO

 Vemurafenib^c

Melanoma

OS, PFS

ADO, DGHO

 Vismodegib

Basal cell cancer

ORR

DGHO, DGMKG, German Society of Dermatology

 Albiglutide

Diabetes mellitus type 2

HbA1c

DDG, Diabetes Research Group HZM, representative from the University Hospital Carl Gustav Carus (Dresden)

 Dulaglutide

Diabetes mellitus type 2

HbA1c

BVND, DDG, Diabetes Research Group HZM

 Eliglustat

Gaucher disease type 1

Stable health status (decrease in spleen and liver volume, Hb, thrombocytes) and %-change in spleen volume

ASIM, DGVS, representative from the Charité University Medicine Berlin

 Elosulfase alfa

Mucopolysaccharidose type IVA (Morquio A syndrome)

6MWT

ZSE Wiesbaden

 Insulin degludec^a

Diabetes mellitus type 1

HbA1c

DDG

 Ivacaftor

Cystic fibrosis

FEV1%

-

 Ivacaftor^a

Cystic fibrosis

FEV1%

-

 Linagliptin^b

Diabetes mellitus type 2

HbA1c

BVND, DDG, Diabetes Research Group HZM

 Pasireotide

Pituitary gland dysfunction

mUFC ≤ ULN

-

 Pasireotide^a

Acromegaly

Biochemical control

-

 Saxagliptin

Diabetes mellitus type 2

HbA1c

DDG, Working Group on Pharmacoepidemiology, Diabetes Research Group HZM, Diabetes Centre Bad Lauterberg

 Saxagliptin/metformin

Diabetes mellitus type 2

HbA1c

BVND

 Sitagliptin

Diabetes mellitus type 2

HbA1c

DDG, Working Group on Pharmacoepidemiology, Diabetes Research Group HZM, Diabetes Centre Bad Lauterberg

 Sitagliptin/metformin

Diabetes mellitus type 2

HbA1c

DDG, Diabetes Centre Bad Lauterberg, Diabetes Research Group HZM, Working Group on Pharmacoepidemiology

 Vildagliptin^b

Diabetes mellitus type 2

HbA1c

BVND, DDG

 Boceprevir

Chronic hepatitis C

SVR

bng, DGVS, German Liver Foundation

 Daclatasvir

Chronic hepatitis C

SVR

bng, dagnä, DGIM, DGVS

 Dasabuvir

Chronic hepatitis C

SVR

bng, dagnä, DGIM, DGVS

 Dolutegravir

HIV infection

VR

dagnä, DAIG

 Dolutegravir/abacavir/lamivudine

HIV infection

VR

-

 Elvitegravir/cobicistat/emtricitabin/tenofovir-disoproxil

HIV infection

VR

dagnä, DAIG

 Emtricitabine/rilpivirine/tenofovirdisoproxil

HIV infection

VR

dagnä, DAIG

 Fidaxomicin

Clostridium infection

Overall cure

DGHO, DGVS

 Ledipasvir/sofosbuvir

Chronic hepatitis C

SVR

bng, dagnä, DGIM, DGVS

 Ombitasvir/paritaprevir/ritonavir

Chronic hepatitis C

SVR

bng, dagnä, DGIM, DGVS

 Rilpivirine

HIV infection

VR

DAIG, dagnä

 Simeprevir

Chronic hepatitis C

SVR

bng, dagnä, DGI, DGIM, DGVS

 Sofosbuvir

Chronic hepatitis C

SVR

bng, dagnä, DGIM

 Telaprevir

Hepatitis C

SVR

bng, DGVS, German Liver Foundation