The Impact of Achondroplasia on Quality of Life, Healthcare Resource Use, Clinical, Socio-economic and Psychosocial state of the Individual (LIAISE)

Study type

• Study topic:
  Disease /health condition
• Study type:
  Non-interventional study
• Scope of the study:
  Disease epidemiology
• Data collection methods:
  Combined primary data collection and secondary use of data

Non-interventional study

• Non-interventional study design:
  Cross-sectional

  Other
• Non-interventional study design, other:
  Retrospective

Study drug and medical condition

• Medical condition to be studied:
  Dwarfism

Population studied

• Short description of the study population:
  Achondroplasia patients aged 5-70 years.
• Age groups:
  Children (2 to < 12 years)

  Adolescents (12 to < 18 years)

  Adults (18 to < 46 years)

  Adults (46 to < 65 years)

  Adults (65 to < 75 years)
• Special population of interest:
  Other
• Special population of interest, other:
  Achondroplasia patients
• Estimated number of subjects:
  300

Study design details

• Main study objective:
  The objectives of the study are to describe the impact on the following in individuals with achondroplasia versus a normative population in Denmark, Germany, Spain and Sweden: • Quality of life• Clinical burden• Healthcare resource use• Socio-economic burden • Psychosocial burden
• Outcomes:
  1. Patient Reported Outcomes (PRO) and Activities of Daily Living (ADL)2. Clinical burden for at least the last five years3. Healthcare resource use for at least the last five years 4. Socio-economic burden5. Psychosocial burden
• Data analysis plan:
  Statistical analysis will be carried out using SAS software. An interim analysis will be performed after nine months of enrolment.Continuous variables will be described (distribution) by their mean, standard deviation, median, quartiles one and three, extreme values (minimum and maximum) and the number of missing data and will be compared to normative data.Categorical variables will be described (counts) by the absolute and relative (%) frequency of each class, and the number of missing data and will be compared to normative data.