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Table 2 Cases where differences at each step of the HTA process explain differences in HTA recommendations

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

Drug and indication pair Eltrombopag Imatinib Mannitol dry Mifamurtide Trabectedin
Idiopathic thrombocytopenic purpura Gastro intestinal stromal tumours (adjuvant, after surgery) Cystic fibrosis Osteosarcoma Soft tissue sarcoma
HTA recommendation Positively appraised (list or restricted) SMC, TLV, HAS (ASMR II) SMC, HAS (ASMR III) NICE, HAS (ASMR V) NICE, SMC, TLV NICE, HAS (ASMR V)
Rejected NICE NICE SMC HAS SMC
Evidence Differences in the level of evidence reported ✖ Severe bleeding events (WHO grade 3–4) (NICE)
✖ Lack of Qol data (HAS)
Qol data included for NICE, SMC and TLV
✖ CUA-standard care (NICE)
✔ CUA-romiplostim (SMC)
✔ CMA-romiplostim (TLV) 		   ✔ Progression-free survival = primary endpoint (SMC, TLV, HAS)
✖ Overal survival = primary endpoint (NICE) 		✔ Use of registry data as historical controls (NICE)
Interpretation of the evidence Different interpretation of the same evidence appraised   Short trial duration
✖ NICE, SMC
Not raised by HAS 		No reduction in hospital days and use of antibiotics
✖ HAS
Not raised by SMC, NICE
Qol not improved
✖ HAS
✔ NICE
Not raised by SMC 		  
Different
interpretation of the same uncertainty 		Short trial duration
✖ NICE (experts), SMC, TLV
✔ HAS (same as comparator) 		Overall survival not
significantly improved
✖ NICE
✔ SMC (orphan)
✔ HAS (on-going trial) 		Risk of bronchospasms
✖ HAS
✔ NICE (expert opinion)
Not raised by SMC 		Risk of interaction between treatments
✖ HAS (other study)
✔ NICE, SMC (expert opinion), TLV (longer-term data) 		Lack of comparative evidence (phase II non-comparative pivotal trial)
✖ HAS
✔ NICE (rarity, early marketing authorisation, historical controls)
✔ SMC (rarity, investigational nature of the treatment)
  1. NICE National Institute for Health and Care Excellence, SMC Scottish Medicines Consortium, TLV Pharmaceutical Benefits Board, HAS Haute autorité de Santé
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