[Pharmaceutical Directive/Annex XII: Voxelotor (haemolytic anaemia in sickle cell disease, monotherapy or combination with hydroxycarbamide, ≥ 12 years)]

The Federal Joint Committee [Gemeinsamer Bundesausschuss] (G-BA)
Record ID 32018004241
English, German
Original Title: Arzneimittel-​​​Richtlinie/Anlage XII: Voxelotor (Hämolytische Anämie bei Sichelzellkrankheit, Monotherapie oder Kombination mit Hydroxycarbamid, ≥ 12 Jahre)
Authors' objectives: The Federal Joint Committee [Gemeinsamer Bundesausschuss (G-BA)] has had the legal task of carrying out an (additional) benefit assessment for all newly approved drugs with new active ingredients immediately after market entry (§ 35a SGB V). The result of this assessment is the basis for deciding how much the statutory health insurance pays for a new drug with a new active ingredient. The G-BA was commissioned to carry out the benefit assessment through the Pharmaceuticals Market Reorganisation Act [Gesetz zur Neuordnung des Arzneimittelmarktes (AMNOG)]. In the context of the early benefit assessment of medicinal products containing new active substances, the following rules apply to orphan drugs: According to the legal requirements (§ 35a SGB V), the additional medical benefit of these drugs is already considered to be proven by the approval. The G-BA determines the extent of the additional benefit for orphan drugs that do not exceed a turnover of 50 million Euros in the last twelve calendar months, on the basis of the approval and the studies justifying the approval.
Authors' results and conclusions: Voxelotor is indicated for the treatment of haemolytic anaemia due to sickle cell disease (SCD) in adults and paediatric patients 12 years of age and older as monotherapy or in combination with hydroxycarbamide. The pivotal study for the benefit assessment was a randomized, double-blind, multicenter, placebo-controlled Phase III study with an adaptive design, the HOPE trial. Patients with SCD of different genotypes aged 12 years and older with at least one vaso-occlusive crisis during the last 12 months were eligible. A total of 274 patients were enrolled and randomly assigned to treatment with voxelotor 1,500 mg/day (N = 90), voxelotor 900 mg/day (N = 92), or placebo (N = 92) in a 1:1:1 ratio. However, only the 1,500 mg group was considered as the recommended dose. Patients were treated for 72 weeks. Primary endpoint was haemoglobin response after 24 weeks (not considered as relevant for patients: RR = 7.69, 95% CI 3.57;16.67; p<0.001). Relevant endpoints for benefit assessment were mortality, vaso-occlusive crises, acute thoracic syndrome, health status (EQ-5D) and safety. The risk of bias at the study level was considered low. There was one fatality in each treatment arm. There was no difference between the groups for acute thoracic syndrome (RR 1.34, 95% CI 0.64;2.84),and vaso-occlusive crises (RR 0.86, 95% CI 0.61;1.22). Response rates for health status were insufficient and therefore not considered. For severe adverse events, and adverse events that led to discontinuation of study medication, there were no statistically significant differences between voxelotor and placebo, both with and without underlying disease events.
Project Status: Completed
Year Published: 2022
Requestor: The Federal Joint Committee [Gemeinsamer Bundesausschuss] (G-BA)
English language abstract: An English language summary is available
Publication Type: Full HTA
Country: Germany
MeSH Terms
  • Anemia, Hemolytic
  • Anemia, Sickle Cell
  • Hematologic Agents
  • Hydroxyurea
  • Adolescent
  • Adult
  • Voxelotor
  • haemolytic anaemia
Organisation Name: The Federal Joint Committee
Contact Address: Gutenbergstr. 13, 10587 Berlin, Germany
Contact Name: Fachberatung Medizin [Department of Medical Consultancy]
Contact Email: Fachberatung-Medizin@g-ba.de
Copyright: https://www.g-ba.de/sys/impressum/
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