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Table 1 List of drug-indication pairs included in the study

From: Why do health technology assessment coverage recommendations for the same drugs differ across settings? Applying a mixed methods framework to systematically compare orphan drug decisions in four European countries

Generic/brand name Indication NICE
England
SMC
Scotland
TLV
Sweden
HAS—Francea
SMR (coverage)
ASMR (pricing)
Eltrombopag
REVOLADE
Chronic idiopathic thrombocytopenic purpura DNL LWC LWC Important (65 %)
II (EU)
Romiplostim
NPLATE
Chronic idiopathic thrombocytopenic purpura LWC LWC LWC Important (65 %)
II (EU)
Everolimus
AFINITOR
Renal cell carcinoma (2nd line, advanced) DNL DNL L Important (100 %)
IV (comp)
Lenalidomide
REVLIMID
Multiple myeloma (3rd line) LWC LWC L Important (65 %)
III (EU)
Mifamurtide
MEPACT
Osteosarcoma LWC L LWC Insufficient (0 %)
DNL
Azacitidine
VIDAZA
Myelodysplastic syndrome LWC LWC NA Important (65 %)
II (EU)
Imatinib
GLIVEC
Gastro intestinal stromal tumour (adjuvant, after surgery) DNL LWC NA Important (100 %)
III (EU)
Mannitol dry
BRONCHITOL
Cystic fibrosis LWC DNL NA Weak (15 %)
V (comp)
Ofatumumab
ARZERRA
Chronic lymphocytic leukemia DNL DNL NA Moderate (35 %)
V (comp)
Trabectedin
YONDELIS
Soft tissue sarcoma LWC DNL NA Important (65 %)
V (comp)
  1. NICE National Institute for Health and Care Excellence (NICE), SMC Scottish Medicines Consortium, TLV Dental and Pharmaceutical Benefits Board, HAS Haute Autorité de Santé, L list, LWC list with restrictions, DNL do not list, NA not applicable, EU price negotiation at European price levels, comp price set below comparator price
  2. aThe ASMR (Amélioration du Service Médical Rendu) ranks drugs according to their relative improvement in clinical benefit in five levels, from a major innovation (level I) to no improvement (level V). The pricing scheme is determined by the ASMR ranking [e.g. ASMR I–III = price negotiations within European price levels (EU), ASMR IV–V = price set below comparators (comp)]. The SMR (Service Médical Rendu) ranks the drug according to the drug’s clinical benefit in four levels (insufficient, weak, moderate, important) and drives the coverage rate (0, 15, 35, 65 %)