[Pharmaceutical Directive/Annex XII: Voretigene Neparvovec (reassessment after the deadline: inherited retinal dystrophy)]

The Federal Joint Committee [Gemeinsamer Bundesausschuss] (G-BA)
Record ID 32018004248
English, German
Original Title: Arzneimittel-Richtlinie/Anlage XII: Voretigen Neparvovec (Erbliche Netzhautdystrophie) – Neubewertung nach Fristablauf
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: Voretigen Neparvovec is approved for the treatment of adult and pediatric patients with vision loss due to hereditary retinal dystrophy based on proven biallelic RPE65 mutations and who have sufficient viable retinal cells. The benefit assessment was based on reassessment of the pivotal study 301, a randomized, multicenter, open-label study with study duration of 1 year and a multi-center, non-randomized follow-up study (LTFU) with data available 5 years after baseline. In study 301 the investigational product (N=21) was compared to observational wait-and-see (N=10). Randomization was performed in a 2:1 ratio. Subjects in the control group could receive the study medication after one year. Risk of bias was rated high due to the open label study design of the RCT. In Study 301 one year after baseline, the Multi-Luminence-Mobility-Test showed a statistically significant result in favor of the intervention group (mean change intervention-control: 1.6 [95% K´CI: 0.7; 2.4]; p < 0.001). No one in the intervention group scored worse. Results on FST measured with white, blue and red light and Goldmann perimetry with stimulus III4e showed comparable statistically significant results in favor of the intervention. In the macular region but not in the fovea region, a statistically significant result in favor of the intervention was shown for Humphrey perimetry. Visual acuity was comparable between treatment arms. In both treatment groups at least one adverse event (AE) was reported. Severe AEs and serious AEs occurred only in the intervention group in 2-3 subjects, respectively, but not in the control group. Results of the LTFU study suggest that the beneficial effects achieved in study 301 are maintained in their magnitude up to 5 years after administration.
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
  • Retinal Dystrophies
  • Genetic Therapy
  • cis-trans-Isomerases
  • retinal dystrophy
  • adult and pediatric patients
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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