Non-interventional prospective study in patients with pulmonary fibrosis treated with nintedanib participating in a patient support program in Spain, to describe patient satisfaction with the program and to monitor quality of life. BALANCE Study

Study type

• Study topic:
  Disease /health condition
• Study topic, other:
  Pulmonary Fibrosis
• Study type:
  Non-interventional study
• Scope of the study:
  Evaluation of patient-reported outcomes
• Data collection methods:
  Primary data collection

Non-interventional study

• Non-interventional study design:
  Cohort

Study drug and medical condition

• Name of medicine:
  OFEV
• Study drug International non-proprietary name (INN) or common name:
  NINTEDANIB
• Anatomical Therapeutic Chemical (ATC) code:
  (L01EX09) nintedanib

  nintedanib

Population studied

• Short description of the study population:
  This study will include patients with IPF and PPF (other than IPF) participating in the PSP Balance Program. The study duration will be from the start of the recruitment period (which will last approximately 6 months) to the last follow-up visit of the last patient (approximately 18 months of total study period). Patients will be followed-up at 6 months and at 12 months. Eligible patients will be recruited according to the following inclusion and exclusion criteria:; ; Inclusion criteria:; Adults (≥18 years old at baseline).; Patients included in the PSP Balance Program.; Ability to read and speak Spanish correctly according to the investigator criteria.; Agree to participate and sign informed consent at baseline.; ; Exclusion criteria:; Suspicion or diagnosis of any relevant cognitive impairment at the discretion of the investigator.
• Age groups:
  Adult and elderly population (≥18 years)

  Adults (18 to < 65 years)

  Adults (18 to < 46 years)

  Adults (46 to < 65 years)

  Elderly (≥ 65 years)

  Adults (65 to < 75 years)

  Adults (75 to < 85 years)

  Adults (85 years and over)

Study design details

• Study design:
  This is an observational and non-interventional study based on newly collected data with the cohort of IPF and PPF (other than IPF) patients participating in the PSP Balance Program. The study will not interfere with the pre-defined procedures of the PSP and will merely observe and and collect PROs.
• Main study objective:
  Primary objective:; To describe patient satisfaction with PSP Balance Program at study inclusion.; ; Secondary objectives:; ; Secondary objectives related to Program satisfaction:; - To describe the change in patient satisfaction with PSP Balance Program at 6 and 12 months after the inclusion in the study.; ; Secondary objectives related to treatment management and adverse events:; - To evaluate patient dosing pattern of nintedanib at 6 and 12 months after the inclusion in the study.; - To describe nintedanib management during the study period.; ; Secondary objectives related to patient reported outcomes:; - To describe the quality of life at baseline and 12 months after the inclusion in the study.; - To describe depression and anxiety symptoms at baseline and 12 months after the inclusion in the study.; - To describe the disease symptoms at baseline and 12 months after the inclusion in the study.; - To determine the number of hospitalization and visits to emergency service related to Interstitial Lung Disease from baseline to 12 months.; ;
• Setting:
  In this study there is one unique study site, which is the following:; Evidenze Health España S.L.U.; Passatge de Ferrer i Vidal, 8, Sant Martí. 08005 Barcelona; This study site has already been selected as it will be the site providing the PSP Balance Program services. ; ; 3 study visits are planned in this study:; - Baseline; - 6 months (+/- 1 month); - 12 months (+/- 1 month); All study visits will coincide with routine PSP Balance Program visits, that means, no new visits will be schedule for this study. Following the PSP Balance Program criteria, if 6 or 12 months study visits could not be performed face to face, it will be done by phone. Baseline study visit will always coincide with one face-to-face PSP Balance Program visit.; ; All visits will be performed by study investigators. After the study visit, all study documents obtained during the visit (e.g., ICF, questionnaires, (S)AE form), will be sent to the study site for processing (data entry into the eCRF) and archival.; ; The Principal Investigator (PI) will delegate the collection of the study informed consent form and the study information to nurses providing the PSP Balance Program services. All nurses participating will be delegated as co-investigators.; The study site will complete a site delegation list with all study tasks delegated to others than the PI.; The Sponsor will sign a contract with the study site and the Principal Investigator. All co-investigators will have a contract with the study site.
• Outcomes:
  Primary Outcome: Patient satisfaction with PSP Balance Program at baseline using the Baker Questionnaire.; ; Secondary Outcomes:; - Change in the satisfaction with PSP Balance Program at 6 and 12-month follow-up after inclusion in the study, using the Baker Questionnaire. ; - Patient satisfaction with PSP Balance Program at baseline, 6 and 12 months after the inclusion in the study using the Likert scale.; - Dosing pattern of nintedanib calculated as absolute number of doses taken correctly divided by all the doses prescribed in that period (6 and 12 months); - Time from first dose to permanent discontinuation of nintedanib within 12-month follow-up.; - Absolute number of nintedanib dose interruptions. ; - HRQoL at baseline and 12-month follow-up using the L-PF Impacts Questionnaire. ; - Anxiety and depression scores in HADS Questionnaire at baseline and 12-month follow-up. ; - Disease symptoms score in L-PF Symptoms Questionnaire at baseline and 12 months follow-up.; - Absolute number of visits to emergency service and hospitalization related to ILD between baseline and 12-month follow-up.; ;
• Data analysis plan:
  A description of all patients included in the study will be performed. All patients participating in the study who meet the eligibility criteria will be included in the study population.; Descriptive statistics will be presented as absolute and relative frequencies for categorical variables. Mean, standard deviation, median, 25th – 75th quartiles, minimum and maximum will be reported for quantitative variables. Number of valid values (N valid) and number of missing values (N missing) will be reported for each variable.; A linear regression will be performed to assess the association between the outcomes and the independent variables (PSP duration). To evaluate the change in PSP satisfaction mixed models will be used.; Questionnaire scores provided by patients will be described using the descriptive measures described above, and according to the manual user.; Data will be analysed for the total evaluable population and for different subgroups (PSP duration) and different timepoints (baseline and 6 and 12- months follow-up), if applicable. If sample population allows, it will also be analysed by received treatment dose.; Same methodology will be used for the interim analysis (after basal data for all patients is collected) and the final analysis.