RESUMO
BACKGROUND: Better insights into the natural course of cystic fibrosis (CF) have led to treatment approaches that have improved pulmonary health and increased the life expectancy of affected individuals. This study evaluated how the combination of modified demographics and changes in CF management impacted resource consumption and the cost of care. METHODS: Medical records of CF patients from 2006 to 2016 in the French CF Registry were linked to their corresponding claims data (SNDS). Medications, medical visits, procedures, hospitalisations, and indirect costs were annualized by calendar year from 2006 to 2017. RESULTS: Of the 7,671 patients included in the French CF Registry, 6,187 patients (80.7%) were linked to the SNDS (51.9% male, mean age = 24.7 years). The average cost per patient was 14,174 in 2006, 21,920 in 2011 and 44,585 in 2017. Costs associated with hospital stays increased from 3,843 per patient in 2006 to 6,741 in 2017. In 2017, the mean cost per CF patient was allocated as follows: 72% for medications (of which 51% for modulator therapies), 15% for hospital stays, 7% for medical visits, 3% for indirect costs, 2% for medical devices, 1% for outpatient medical procedures. CONCLUSION: There was a strong increase in the mean annual cost per CF patient between 2006 and 2017, mostly due to the cost of therapy after the introduction of cystic fibrosis transmembrane conductance regulator (CFTR) modulators. The combination of an increase in the number of CF patients - particularly adult patients - and an increase in the annual cost per patient led to a substantial increase in the total cost of CF disease care for the health systems.
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Fibrose Cística/economia , Fibrose Cística/terapia , Custos de Cuidados de Saúde/tendências , Adolescente , Adulto , Criança , Pré-Escolar , Estudos Transversais , Feminino , Humanos , Masculino , Aceitação pelo Paciente de Cuidados de Saúde , Adulto JovemRESUMO
BACKGROUND: Lumacaftor-ivacaftor combination is a promising treatment for cystic fibrosis (CF) patients homozygous for the F508del-CFTR mutation. Optimal adherence is essential to achieve full health outcomes benefits. METHODS: This retrospective study used pharmacy refills data to calculate proportion of days covered (PDC). Adherence was defined as a PDC ≥80%. A logistic regression analysis was conducted to examine factors associated with medication adherence. RESULTS: Ninety-six patients were included in the final cohort for analysis. The mean PDC was 96% ± 14 at 6 months, and 91% ± 17 at 12 months. The proportion of adherent patients was 89% and 83% at 6 and 12 months respectively. Age and ppFEV1 were found to affect medication adherence. CONCLUSIONS: Considering the medico-economic impact of CFTR modulator therapy, high adherence rates to lumacaftor-ivacaftor found in this study are encouraging.
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Aminofenóis , Aminopiridinas , Benzodioxóis , Agonistas dos Canais de Cloreto , Regulador de Condutância Transmembrana em Fibrose Cística/genética , Fibrose Cística , Adesão à Medicação/estatística & dados numéricos , Quinolonas , Adulto , Fatores Etários , Aminofenóis/economia , Aminofenóis/uso terapêutico , Aminopiridinas/economia , Aminopiridinas/uso terapêutico , Benzodioxóis/economia , Benzodioxóis/uso terapêutico , Agonistas dos Canais de Cloreto/economia , Agonistas dos Canais de Cloreto/uso terapêutico , Análise Custo-Benefício , Fibrose Cística/tratamento farmacológico , Fibrose Cística/economia , Fibrose Cística/epidemiologia , Fibrose Cística/genética , Combinação de Medicamentos , Feminino , Volume Expiratório Forçado , França/epidemiologia , Homozigoto , Humanos , Masculino , Quinolonas/economia , Quinolonas/uso terapêutico , Testes de Função Respiratória/métodos , Testes de Função Respiratória/estatística & dados numéricos , Estudos Retrospectivos , Resultado do TratamentoRESUMO
Background: Although prognosis of Chronic Granulomatous Disease (CGD) has greatly improved, few studies have focused on its long-term outcome. We studied the clinical course and sequelae of CGD patients diagnosed before age 16, at various adult time points. Method: Cross-sectional French nationwide retrospective study of patients screened through the National Reference Center for Primary Immunodeficiencies (CEREDIH) registry. Results: Eighty CGD patients (71 males [88.7%], 59 X-linked [73.7%], median age 23.9 years [minimum, 16.6; maximum, 59.9]) were included, Median ages at diagnosis and last follow-up were 2.52 and 23.9 years, respectively. Seven patients underwent hematopoietic stem cell transplantation. A total of 553 infections requiring hospitalization occurred in 2017 patient-years. The most common site of infection was pulmonary (31%). Aspergillus spp. (17%) and Staphylococcus aureus (10.7%) were the commonest pathogens. A total of 224 inflammatory episodes occurred in 71 patients, mainly digestive (50%). Their characteristics as well as their annual frequency did not vary before and after age 16. Main sequelae were a small adult height and weight and mild chronic restrictive respiratory failure. At age 16, only 53% of patients were in high school. After age 30 years, 9/13 patients were working. Ten patients died during adulthood. Conclusions: Adult CGD patients displayed similar characteristics and rates of severe infections and inflammatory episodes that those of childhood. The high rate of handicap has become a matter of medical and social consideration. Careful follow-up in centers of expertise is strongly recommended and an extended indication of curative treatment by HSCT should be considered.
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Doença Granulomatosa Crônica/epidemiologia , Adolescente , Fatores Etários , Antibioticoprofilaxia , Autoimunidade , Infecções Bacterianas/tratamento farmacológico , Infecções Bacterianas/epidemiologia , Infecções Bacterianas/etiologia , Infecções Bacterianas/prevenção & controle , Criança , Pré-Escolar , Efeitos Psicossociais da Doença , Estudos Transversais , Feminino , França/epidemiologia , Doença Granulomatosa Crônica/complicações , Doença Granulomatosa Crônica/diagnóstico , Doença Granulomatosa Crônica/mortalidade , Humanos , Lactente , Recém-Nascido , Masculino , Micoses/tratamento farmacológico , Micoses/epidemiologia , Micoses/etiologia , Micoses/prevenção & controle , Fenótipo , Vigilância da População , Sistema de Registros , Estudos Retrospectivos , Análise de Sobrevida , Avaliação de SintomasRESUMO
INTRODUCTION: Following the generalization of neonatal screening, the French CF Care Network has become structured around 45 qualified centres, the French CF Society, 2 national expertise centres, the Patient Registry and the National Protocol of CF Care in collaboration with the Vaincre Ia Mucoviscidose patient association. This organization and progress in treatment have resulted in the outpatient follow-up of a growing number of patients. Since 2010, the CF Network representatives have been conducting an assessment of outpatient follow-up to identify difficulties in complying with national and international clinical practice guidelines. METHODS: Two complementary quantitative and qualitative approaches were used to characterize and quantify the activities carried out by professionals in 8 centres both for outpatient visits and patient care coordination. RESULTS: Two thirds of the 1,4 75 patients followed in the centres were managed over the period, less than half (40%) of them attended outpatient visits, but all of them were concerned by care coordination activities, whether or not they were related to the visit. The core team (doctor, nurse, physio-therapist) is not mobilized at each scheduled outpatient visit as recommended. Professionals devote 40% less time for follow-up in adult centres than in paediatric centres, all activities included. The multidisciplinary outpatient visit process is complicated by the lack of available resources and the unsuitability of certain premises. DISCUSSION: With a constantly growing number of patients, CF centres are struggling to comply with good clinical practice and meet the specific needs of adult patients and transplant recipients. An upgrade of professional resources and an update of the National Protocol appear to be necessary.
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Assistência Ambulatorial/métodos , Fibrose Cística/terapia , Guias de Prática Clínica como Assunto , Qualidade da Assistência à Saúde , Adulto , Comportamento Cooperativo , Seguimentos , Necessidades e Demandas de Serviços de Saúde , Humanos , Comunicação Interdisciplinar , Equipe de Assistência ao Paciente/organização & administração , TransplantadosRESUMO
OBJECTIVES: The aim of this study was to evaluate how advances in CF management in France between 2000 and 2003 impacted CF-related costs. METHODS: The analysis of direct medical costs was done in 2000 and 2003 from the perspective of the French national healthcare insurance system. The patients, 65 in 2000 and 64 in 2003, were followed-up in one pediatric and one adult CF reference center (CFRC). We quantified and valued CF-related home and hospital care costs. RESULTS: We found an average cost of euro16474/patient/year in 2000, and euro22725 in 2003 (based on the 2003 euro value). Hospital care increased from 15% of the total cost in 2000 to 22% in 2003. Medications accounted for 45% of the total cost for the two periods, with an average cost of euro7229/patient/year in 2000 and euro10336 in 2003. Home intravenous antibiotic therapy accounted for 20% of the total cost for the two periods. CONCLUSIONS: We highlighted an increase in CF care costs between 2000 and 2003, which might be related to the changes in practice patterns that followed guidelines implementation, such as the use of new medications (dornase alpha and tobramycin) and more frequent follow-up in the CFRC.
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Fibrose Cística/economia , Fibrose Cística/terapia , Fidelidade a Diretrizes/economia , Guias de Prática Clínica como Assunto , Adulto , Assistência Ambulatorial/economia , Distribuição de Qui-Quadrado , Criança , Pré-Escolar , Custos e Análise de Custo , Feminino , França , Serviços de Assistência Domiciliar/economia , Custos Hospitalares/estatística & dados numéricos , Humanos , Lactente , Masculino , Estatísticas não ParamétricasRESUMO
OBJECTIVES: In France, new guidelines for clinical practices concerning cystic fibrosis came out in 2002, underscoring the need for early and intensive management of this disease. Because no recent health economic studies on cystic fibrosis in France were available, we conducted a cost-analysis study before the new guidelines were put into practice, with a view to a later study on the medical and economic impact of these guidelines. METHODS: A cost-analysis study was performed of the inpatient and outpatient costs of patients with cystic fibrosis for the 2000-2001 period. The various direct costs were estimated on a sample of sixty-five adult and pediatric patients managed for cystic fibrosis in two reference medical centers. Data were obtained from medical records, and questionnaires were filled out by the patients. Analysis was made from the perspective of the French healthcare system. RESULTS: We studied sixty-five patients, 54 percent male patients and 72 percent children under 18 years of age. The total cost of cystic fibrosis care totaled 16,189 euros per year and per patient. Outpatient costs accounted for 88 percent of the total cost versus 12 percent for inpatient costs; medication costs were the highest with 21 percent of the total cost for home intravenous antibiotic treatments and 49 percent of the total cost for chronic medications. CONCLUSIONS: The results show that outpatient costs were higher than inpatient costs, which could be related to the importance granted to home health care in France, notably for intravenous antibiotic treatments given for pulmonary complications.