OBJECTIVES: AML is a rare, aggressive cancer with few available personalized treatments. These high-cost drugs are in the focus of HTA bodies and reimbursement decisions globally. The present research examines HTA assessments for AML drugs.

 

METHODS: 7 HTA bodies were screened: US (ICER), England (NICE), Scotland (SMC), France (HAS), Canada (pCODR), Australia (PBAC) and Germany (G-BA) for the assessment of 4 drugs (Midostaurin, Gemtuzumab-ozogamicin, Daunorubicin/Cytarabine and Enasidenib) for AML treatment, approved in the last 5 years (since 2015).

 

RESULTS: ICER assessed no drugs for AML.

 

Midostaurin: All HTA bodies made a positive assessment for newly diagnosed FLT3+ AML. NICE and SMC with a restriction for the use [Patient Access Scheme (PAS) / Commercial agreement]. G-BA and HAS assessed the additional benefit with different gradings (Considerable and ASMR IV respectively). In Germany a price reduction of 26% was agreed after G-BA assessment.

 

Daunorubicin/Cytarabine: NICE, SMC, G-BA and HAS made a positive assessment for newly diagnosed, therapy-related AML or AML with myelodysplasia-related changes. In Germany, a price reduction of 18% was agreed after G-BA assessment.

 

Gemtuzumab-ozogamicin: NICE, SMC, G-BA and HAS made a positive assessment for untreated de novo CD33+ (AML) except acute promyelocytic leukemia. NICE and SMC with a restriction for the use (PAS / Commercial agreement). G-BA assessed the added benefit as Not Quantifiable, with a price reduction of 36% after G-BA assessment. HAS rated ASMR V.

 

Enasidenib: pCODR did not recommend reimbursement for relapsed/refractory AML (IDH2 mutation).

 

CONCLUSIONS: HTA assessment depicted varying outcomes and decisions due to differences in the applicable underlying laws, and patient population across drugs assessed. Major factors driving decisions were, superior efficacy and acceptable safety for HAS and G-BA; clinical benefit and cost-effectiveness for pCODR, PBAC, NICE and SMC; and end of life criteria across HTAs. Cost-effectiveness for NICE, SMC was by PAS under a commercial agreement with manufacturers.