Advanced Parkinson’s disease treatment eligibility in France: an epidemiological study (EPIPARK)

Study type

• Study topic:
  Disease /health condition
• Study type:
  Non-interventional study
• Scope of the study:
  Disease epidemiology
• Data collection methods:
  Primary data collection

Non-interventional study

• Non-interventional study design:
  Cross-sectional

Study drug and medical condition

• Medical condition to be studied:
  Parkinson's disease

Population studied

• Short description of the study population:
  Parkinson's disease (PD) patients at advanced stage of disease (or their legal representatives), who have received verbal and written information about the study.
• 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:
  Parkinson's disease patients
• Estimated number of subjects:
  442

Study design details

• Main study objective:
  The main objective of this study is to estimate, in Metropolitan France, the size of the population of Parkinson’s disease patients eligible to Duodopa® in accordance with the conditions defined by the French Authority for Health in its Transparency Committee’s opinion.
• Outcomes:
  -Number and percentage of patients not eligible for Deep Brain Stimulation -Number and percentage of patients presenting contra-indication, intolerance or failure to subcutaneous apomorphine continuous infusion -Number and percentage of patients without contra-indications and precautions for Duodopa® use as they are defined in the drug SmPC (ANSM, Duodopa®), -To estimate, in Metropolitan France, the size of the population of PD patients eligible for Duodopa® according to the indication defined by its marketing authorization -To describe advanced PD patients and disease characteristics (including background history) -To estimate the proportion of PD patients eligible for each invasive therapy (Deep Brain Stimulation, Apomorphine or Duodopa®)
• Data analysis plan:
  1. Descriptive analysis of patients data :-quantitative variables (sample size (data provided, missing values), mean, standard deviation, median, mode if relevant, first and third quartiles and range. If relevant, 95% confidence interval (CI) will be presented.-qualitative variables : number of non-missing values, number of missing values, frequency and percentage per modality. Note that missing values will be excluded from the calculation of percentages, unless their proportion is important (i.e. >10%). If relevant, 95% CI of the proportion will be presented2. Statistical adjustments to correct for:-potential duplicates, -incompleteness of the census, -non-inclusion of eligible patients3. Statistical extrapolations to ensure generalizability.