Cystic fibrosis prognosis in Europa : chronicle of an announced dilemma

In cystic fibrosis, lung disease is early and insidious. It almost always conditions the prognosis. A pragmatic way of looking at prognostic factors is to distinguish those on which care management has little (environmental factors) or no grip (genetic factors) and those related to the quality of ca...

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Bibliographic Details
Published inRevue médicale de Liège Vol. 76; no. 3; p. 202
Main Authors Lebecque, P, Thimmesch, M
Format Journal Article
LanguageFrench
Published Belgium 01.03.2021
Subjects
Aminophenols
Aminopyridines
Benzodioxoles
Cystic Fibrosis - diagnosis
Cystic Fibrosis - therapy
Cystic Fibrosis Transmembrane Conductance Regulator - genetics
Europe
Humans
Mutation
Prognosis
Quinolones
Europe
effectiveness
CTFR
Prognosis
Cystic fibrosis
Cost
Modulators
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Summary:In cystic fibrosis, lung disease is early and insidious. It almost always conditions the prognosis. A pragmatic way of looking at prognostic factors is to distinguish those on which care management has little (environmental factors) or no grip (genetic factors) and those related to the quality of care, the latter being crucial. Recently, a triple-combination CFTR («Cystic Fibrosis Transmembrane conductance Regulator») modulator regimen has been shown a highly effective therapy. Ultimately, at least 90 % of Belgian patients with cystic fibrosis should benefit from this drug. However, its official price is extremely high (712 €/day), lacks transparency and illustrates problematic aspects of current orphan legislations. For the majority of citizens in Western Europe, a social ideal still prevails that healthcare should be accessible to all in an equitable fashion. Somewhere between this price and the necessity for national health systems based on solidarity to keep the costs of orphan drugs at a sustainable level, patients are looking like hostages.
ISSN:0370-629X