Characterization of the epidemiology, treatment patterns and burden of Pulmonary Hypertension Group 1 and 3 in France, Germany and the UK: a real-word evidence study (PHILD_RWE_FR_DE_UK)

Study type

• Study topic:
  Disease /health condition
• Study type:
  Non-interventional study
• Scope of the study:
  Disease epidemiology

  Drug utilisation

  Healthcare resource utilisation
• Data collection methods:
  Secondary use of data

Non-interventional study

• Non-interventional study design:
  Cohort

  Other
• Non-interventional study design, other:
  Retrospective longitudinal study

Study drug and medical condition

• Medical condition to be studied:
  Pulmonary hypertension

  Chronic respiratory disease

  Interstitial lung disease

  Pulmonary arterial hypertension

Population studied

• Short description of the study population:
  The study focused on two main cohorts and a subgroup: group 1 patients with pulmonary arterial hypertension (PAH) cohort, group 3 patients with pulmonary arterial hypertension (PAH) and/or hypoxia cohort, and subgroup 3.2 of patients with pulmonary hypertension associated with interstitial lung disease.
• Age groups:
  Adults (18 to < 46 years)

  Adults (46 to < 65 years)

  Adults (65 to < 75 years)

  Adults (75 to < 85 years)

  Adults (85 years and over)
• Special population of interest:
  Other
• Special population of interest, other:
  Patients with hypertension and lung disease
• Estimated number of subjects:
  15000

Study design details

• Main study objective:
  To estimate prevalence and incidence of PH with lung diseases and/or hypoxia (PH-LD) and PH with interstitial lung disease (PH-ILD) in Germany, France, and the UK. To characterize baseline characteristics, comorbidities, resource use, and real-world outcomes of PH-LD and PH-ILD patients. To describe treatment patterns of three cohorts: pulmonary arterial hypertension (PAH), PH-LD, and PH-ILD.
• Outcomes:
  1. Estimation of the prevalence and incidence of PH-LD and PH-ILD in Germany, France, and the UK. - 2. The characterization of the treatment patterns of PAH, PH-LD, and the sub-group PH-ILD patients, 1. Estimation of burden of disease of PH-LD and PH-ILD patients in terms of resource use and costs, evaluating hospital care (inpatient and outpatient), expenditure on tests, procedures, diagnostics, and medications. - 2. Characterization of the patient profile, real-world outcomes in terms of demographic and clinical characteristics, comorbidity burden, survival rate, yearly hospitalization rate
• Data analysis plan:
  The queries, management and statistical analysis of the data used will be developed by Alira Health using R and RStudio software or Python for France and UK databases, whereas for the German claims data, SAS and Excel will be used. Descriptive statistics will be presented for all evaluation criteria for each population, as follows: - Quantitative variables a. Quantitative variables will be described with standard statistics including, the mean, standard deviation (SD), median, quartiles, and minimum and maximum. The number of patients with missing data will be reported for each variable and will be described accordingly. Quantitative variables may be categorized into quantiles as required. - Categorical variables - Counts and percentages will be computed for categorical variables. - If relevant, all statistical tests will be two-sided and considered significant at the 5% level. A more detailed Statistical Analysis Plan of the study will be developed.