[Cerliponase alfa in neuronal ceroid lipofuscinosis type 2]

Donato M, Pichon-Riviere A, Augustovski F, García Martí S, Alcaraz A, Bardach A, Ciapponi A.
Record ID 32018001532
Original Title: Cerliponasa alfa en lipofuscinosis neuronal ceroide tipo 2
Authors' recommendations: CONCLUSIONS No evidence assessing survival in patients with neuronal ceroid lipofuscinosis type 2 under treatment with cerliponase alfa has been identified. Low-quality evidence suggests that in patients three to nine years old with neuronal ceroid lipofuscinosis type 2, treatment with intraventricular cerliponase alfa might slow progression towards disability of the motor function and speech areas at short term when compared with historical controls. No evidence related to the potential impact on other disease manifestations such as seizures or visual acuity loss has been found. Serious adverse events took place very frequently and included hypersensitivity, upper respiratory tract infection, epilepsy, pharyngitis, gastroenteritis, pyrexia and device-related infection. No evidence assessing the effectiveness of this treatment at long term has been identified. In Argentina, this technology has been not approved for the assessed indication. The clinical practice guidelines identified from high-income countries do not mention this technology for the assessed indication. Among the healthcare payers consulted only Germany and one United States private institution cover this technology for the assessed indication. One preliminary analysis carried out by the United Kingdom’s NICE (National Institute for Health and Clinical Excellence) highlights the uncertainty about the potential long-term benefit of this technology and its high cost; therefore it would not be cost-effective in the United Kingdom considering its current values. In Argentina, no economic studies for this technology have been found. An informal budget impact analysis from Chile showed a high budgetary impact.
Project Status: Completed
Year Published: 2019
URL for published report: https://www.iecs.org.ar/home-ets/
English language abstract: An English language summary is available
Publication Type: Not Assigned
Country: Argentina
MeSH Terms
  • Neuronal Ceroid-Lipofuscinoses
  • Dipeptidyl-Peptidases and Tripeptidyl-Peptidases
  • Enzyme Replacement Therapy
Organisation Name: Institute for Clinical Effectiveness and Health Policy
Contact Address: Dr. Emilio Ravignani 2024, Buenos Aires - Argentina, C1414 CABA
Contact Name: info@iecs.org.ar
Contact Email: info@iecs.org.ar
Copyright: Institute for Clinical Effectiveness and Health Policy (IECS)
This is a bibliographic record of a published health technology assessment from a member of INAHTA or other HTA producer. No evaluation of the quality of this assessment has been made for the HTA database.