Status: Planned First registered on: 10/11/2020
Last updated on: 10/11/2020
1. Study identification
EU PAS Register NumberEUPAS37983
Official titleComparative Effectiveness and Safety of Drugs used in Rare Neuromuscular and Neurodegenerative Diseases
Study title acronymCAESAR
Study typeObservational study
Brief description of the studyCAESAR is an Italian multicenter retrospective cohort study on Rare Neuromuscular and Neurodegenerative diseases (RND), based on information available in regional administrative healthcare databases. The aims are 1) to describe the prescriptive patterns of drugs used for the treatment of patients affected by RND and identify patient characteristics associated to these patterns in the three Italian regions (Tuscany, Umbria, Lazio), accounting for over 10 million residents; and 2) to perform a comparative evaluation of the effectiveness and safety of drugs used for the treatment of RND, with a focus on specific active agents. The study population will be enrolled in the period 2009-2019, with a two-year look-back and at least 1 year of follow-up, including patients, resident in one of the three regions and affected by Amyotrophic lateral sclerosis (ALS), Progressive muscular atrophy (PMA), Pseudobulbar palsy, Progressive bulbar palsy (PBP), Primary lateral sclerosis (PLS), Other motor neuron disease, Myasthenia gravis (MG). Patients will be defined from Hospital discharge records (ICD-9-CM codes), disease specific copayment exemptions, and, as far as possible, disease specific drug treatments. Drug utilisation patterns will be based on drugs (ATC codes) prescribed to outpatients using the DDDs. Safety and effectiveness will be investigated using a new-user approach and applying both, intention-to-treat and as-treated analysis. Clinical data available in two of the participating regions for ALS patients will be used to 1) validate the algorithm used for patient identification, 2) validate the exposure definition, and 3) perform external adjustment. Data and analysis ill be managed through a common data model, with shared data scripts, performing the analysis at regional level (in-house) and pooling aggregated anonymous data to obtain overall results.
Was this study requested by a regulator?No
Is the study required by a Risk Management Plan (RMP)? Not applicable
Regulatory procedure number (RMP Category 1 and 2 studies only)
Other study registration identification numbers and URLs as applicable
2. Research centres and Investigator details
Coordinating study entity
Centre to which the investigator belongsDepartment of Epidemiology Lazio Region, Italy
Department/Research groupDepartment of Epidemiology
Organisation/affiliationLazio Regional Health Service
Website/Homepagewww.deplazio.net
Details of (Primary) lead investigator
Title Ms
Last name Kirchmayer
First name Ursula
Is this study being carried out with the collaboration of a research network?
No
Other centres where this study is being conducted
Multiple centres

In total how many centres are involved in this Study?8

Department of Epidemiology Lazio Region, Italy
ARS
National Center for Disease Prevention and Health Promotion, National Institute of Health, Rome, Italy
University of Florence, Neurofarba Department, Florence, Italy
Unit of Adverse Drug Reactions Monitoring, University Hospital of Pisa, Pisa, Italy
Scarab Lab, Florence, Italy
Umbria Region, Regional Pharmacovigilance Center, Perugia, Italy
Umbria Region Perugia Hospital - Neuropathophysiology, Perugia, Italy
Countries in which this study is being conducted
National study

Italy
3. Study timelines: initial administrative steps, progress reports and final report
PlannedActual
Date when funding contract was signed23/09/202023/09/2020
Start date of data collection01/03/2021
Start date of data analysis01/06/2021
Date of interim report, if expected25/10/2021
Date of final study report24/10/2022
4. Sources of funding
Please provide estimates of the percentage of funding by source for this study
Names(s)Approximate % funding
Pharmaceutical companies
Charities
Government bodyItalian Medicines Agency12
Research councils
EU funding scheme
OtherRegional Drug Departme88
5. Contact details for enquiries
Scientific Enquiries
Title Ms
Last name Kirchmayer
First name Ursula
Address line 1Via Cristoforo Colombo, 112
Address line 2 
Address line 3 
CityRoma 
Postcode00147 
CountryItaly
Phone number (incl. country code)390699722160 
Alternative phone number3999722129 
Fax number (incl. country code)390699722111 
Email address u.kirchmayer@deplazio.it
Public Enquiries
Title Ms 
Last name Kirchmayer 
First name Ursula 
Address line 1Via Cristoforo Colombo, 112 
Address line 2 
Address line 3 
CityRoma 
Postcode00147 
CountryItaly 
Phone number (incl. country code)390699722160 
Alternative phone number3999722129 
Fax number (incl. country code)390699722111 
Email address u.kirchmayer@deplazio.it 
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