Evaluation of the new medication Kalydeco to treat cystic fibrosis

Description

Kalydeco is a medicine for the treatment of cystic fibrosis (CF) patients. This medicine is only for CF patients with a specific genetic mutation on the CFTR gene: G551D, G1244E, G1349R G178R, G551S, S1251N, S1255P, S549N or S549R. On 26/11/2015, the manufacturer of Kalydeco, Vertex Pharmaceuticals, and the NIHDI signed an agreement for the temporary reimbursement of Kalydeco. As part of this temporary reimbursement, additional data should be collected for all patients treated with Kalydeco. With this data, Vertex Pharmaceuticals must prepare reports that are intended for the NIHDI and that must allow to evaluate the effectiveness of the medicine based on the following questions: a) What is the (long-term) effect on disease progression, on the use of standard therapies, on the need for lung transplantation and on mortality?; b) What is the actual reduction in antibiotic use and hospitalizations?; c) What is the effect on improving survival?. Based on these reports, the NIHDI will decide whether the reimbursement can be continued.

Resources

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8 https://fair.healthdata.be/dataset/b562f5a1-baa4-4581-92aa-5b5cdb3ab743
23 https://fair.healthdata.be/dataset/b562f5a1-baa4-4581-92aa-5b5cdb3ab743
23 https://fair.healthdata.be/dataset/b562f5a1-baa4-4581-92aa-5b5cdb3ab743
54 https://fair.healthdata.be/dataset/b562f5a1-baa4-4581-92aa-5b5cdb3ab743
8 https://fair.healthdata.be/dataset/b562f5a1-baa4-4581-92aa-5b5cdb3ab743
23 https://fair.healthdata.be/dataset/b562f5a1-baa4-4581-92aa-5b5cdb3ab743
8 https://fair.healthdata.be/dataset/b562f5a1-baa4-4581-92aa-5b5cdb3ab743
8 https://fair.healthdata.be/dataset/b562f5a1-baa4-4581-92aa-5b5cdb3ab743
8 https://fair.healthdata.be/dataset/b562f5a1-baa4-4581-92aa-5b5cdb3ab743
23 https://fair.healthdata.be/dataset/b562f5a1-baa4-4581-92aa-5b5cdb3ab743
23 https://fair.healthdata.be/dataset/b562f5a1-baa4-4581-92aa-5b5cdb3ab743
8 https://fair.healthdata.be/dataset/b562f5a1-baa4-4581-92aa-5b5cdb3ab743
8 https://fair.healthdata.be/dataset/b562f5a1-baa4-4581-92aa-5b5cdb3ab743
23 https://fair.healthdata.be/dataset/b562f5a1-baa4-4581-92aa-5b5cdb3ab743
23 https://fair.healthdata.be/dataset/b562f5a1-baa4-4581-92aa-5b5cdb3ab743
54 https://fair.healthdata.be/dataset/b562f5a1-baa4-4581-92aa-5b5cdb3ab743
23 https://fair.healthdata.be/dataset/b562f5a1-baa4-4581-92aa-5b5cdb3ab743
8 https://fair.healthdata.be/dataset/b562f5a1-baa4-4581-92aa-5b5cdb3ab743
23 https://fair.healthdata.be/dataset/b562f5a1-baa4-4581-92aa-5b5cdb3ab743
8 https://fair.healthdata.be/dataset/b562f5a1-baa4-4581-92aa-5b5cdb3ab743
8 https://fair.healthdata.be/dataset/b562f5a1-baa4-4581-92aa-5b5cdb3ab743
8 https://fair.healthdata.be/dataset/b562f5a1-baa4-4581-92aa-5b5cdb3ab743
54 https://fair.healthdata.be/dataset/b562f5a1-baa4-4581-92aa-5b5cdb3ab743
23 https://fair.healthdata.be/dataset/b562f5a1-baa4-4581-92aa-5b5cdb3ab743
8 https://fair.healthdata.be/dataset/b562f5a1-baa4-4581-92aa-5b5cdb3ab743
23 https://fair.healthdata.be/dataset/b562f5a1-baa4-4581-92aa-5b5cdb3ab743
8 https://fair.healthdata.be/dataset/b562f5a1-baa4-4581-92aa-5b5cdb3ab743
23 https://fair.healthdata.be/dataset/b562f5a1-baa4-4581-92aa-5b5cdb3ab743
23 https://fair.healthdata.be/dataset/b562f5a1-baa4-4581-92aa-5b5cdb3ab743
8 https://fair.healthdata.be/dataset/b562f5a1-baa4-4581-92aa-5b5cdb3ab743
8 https://fair.healthdata.be/dataset/b562f5a1-baa4-4581-92aa-5b5cdb3ab743
23 https://fair.healthdata.be/dataset/b562f5a1-baa4-4581-92aa-5b5cdb3ab743
54 https://fair.healthdata.be/dataset/b562f5a1-baa4-4581-92aa-5b5cdb3ab743
23 https://fair.healthdata.be/dataset/b562f5a1-baa4-4581-92aa-5b5cdb3ab743
8 https://fair.healthdata.be/dataset/b562f5a1-baa4-4581-92aa-5b5cdb3ab743
23 https://fair.healthdata.be/dataset/b562f5a1-baa4-4581-92aa-5b5cdb3ab743
23 https://fair.healthdata.be/dataset/b562f5a1-baa4-4581-92aa-5b5cdb3ab743
8 https://fair.healthdata.be/dataset/b562f5a1-baa4-4581-92aa-5b5cdb3ab743
8 https://fair.healthdata.be/dataset/b562f5a1-baa4-4581-92aa-5b5cdb3ab743
8 https://fair.healthdata.be/dataset/b562f5a1-baa4-4581-92aa-5b5cdb3ab743
23 https://fair.healthdata.be/dataset/b562f5a1-baa4-4581-92aa-5b5cdb3ab743
23 https://fair.healthdata.be/dataset/b562f5a1-baa4-4581-92aa-5b5cdb3ab743
8 https://fair.healthdata.be/dataset/b562f5a1-baa4-4581-92aa-5b5cdb3ab743
23 https://fair.healthdata.be/dataset/b562f5a1-baa4-4581-92aa-5b5cdb3ab743
23 https://fair.healthdata.be/dataset/b562f5a1-baa4-4581-92aa-5b5cdb3ab743
8 https://fair.healthdata.be/dataset/b562f5a1-baa4-4581-92aa-5b5cdb3ab743

Tags

  • cystic-fibrosis
  • alliance-nationale-des-mutualités-chrétiennes-(anmc)
  • mucoviscidose
  • national-institute-for-health-and-disability-insurance-(nihdi)
  • codage,-anonymisation-et-ttp-(ehealth)
  • rijksregisternummer-(rrn)
  • pubchem-16220172
  • numéro-d'identification-de-la-sécurité-sociale-(niss)
  • atc-l1-r
  • atc-r07ax02
  • comité-de-sécurité-de-l'information:-chambre-sécurité-sociale-et-santé
  • sexe
  • unii-1y740ill1z
  • fibrose-kystique
  • artikel-81
  • ivacaftor
  • kalydeco
  • chebi-66901
  • union-nationale-des-mutualités-libres-(unml)
  • belgië-(country)
  • hdbp0068
  • mucoviscidosis
  • kas-der-geneeskundige-verzorging-van-de-nmbs-holding-(hr-railcare)
  • sex
  • codering,-anonimisering-en-ttp-(ehealth)
  • echa-100.226.211
  • identificatienummer-van-de-sociale-zekerheid-(insz)
  • source-data-at-healthdata.be-(sciensano)
  • informatieveiligheidscomité:-kamer-sociale-zekerheid-en-gezondheid
  • landesinstitut-für-kranken-und-invalidenversicherung-(likiv)
  • hta
  • mesh-c545203
  • union-nationale-des-mutualités-neutres-(unmn)
  • numéro-inami
  • nationaal-intermutualistisch-college-(nic)
  • belgique-(country)
  • riziv-nummer
  • landsbond-der-christelijke-mutualiteiten-(lcm)
  • cas-873054-44-5
  • landsbond-van-liberale-mutualiteiten-(llm)
  • nidhi-number
  • kegg-d09916
  • beveiligde-elektronische-brievenbus-(ehealthbox)
  • caisse-des-soins-de-santé-de-la-sncb-holding-(hr-railcare)
  • health-technology-assessment-procedure
  • mesh-d003550
  • drugbank-db08820
  • healthdata.be-(sciensano)
  • landsbond-van-de-neutrale-ziekenfondsen-(lnz)
  • icd10-e00–e90
  • snomed-ct-190905008
  • union-nationale-des-mutualités-socialistes-(solidaris-unms)
  • civars
  • médicaments-chapitre-iv
  • chemspider-17347474
  • medlineplus-a612012
  • belgium-(country)
  • numéro-de-registre-national-(nrn)
  • age
  • landsbond-van-de-onafhankelijke-ziekenfondsen-(mloz)
  • collège-intermutualiste-national-(cin)
  • hulpkas-voor-ziekte-en-invaliditeitsverzekering-(hziv)
  • âge
  • cystische-fibrose
  • project-completed
  • chembl-2010601
  • rijksinstituut-voor-ziekte-en-invaliditeitsverzekering-(riziv)
  • managed-entry-agreement
  • geneesmiddelen-hoofdstuk-iv
  • hospital
  • vx-770
  • leeftijd
  • gender
  • union-nationale-des-mutualités-libérales-(unml)
  • cobrha-common-base-registry-for-healthcare-actor-(ehealth)
  • institut-national-d'assurance-maladie-invalidité-(inami)
  • geslacht
  • boîte-aux-lettres-électronique-sécurisée-(ehealthbox)
  • snomed-ct-110462006
  • ziekenhuis
  • nationaal-verbond-van-socialistische-mutualiteiten-(nvsm)
  • caisse-auxiliaire-d'assurance-maladie-invalidité-(caami)
  • iuphar-4342
  • hôpital
  • vertex-pharmaceuticals-(company)

Topics

  • HEAL

Categories