Study identification

PURI

https://redirect.ema.europa.eu/resource/37984

EU PAS number

EUPAS37983

Study ID

37984

Official title and acronym

Comparative Effectiveness and Safety of Drugs used in Rare Neuromuscular and Neurodegenerative Diseases (CAESAR)

DARWIN EU® study

No

Study countries

Italy

Study description

CAESAR 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.

Study status

Planned
Research institution and networks

Institutions

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

Contact details

Ursula Kirchmayer

Primary lead investigator
Study timelines

Date when funding contract was signed

Planned:
Actual:

Study start date

Planned:

Data analysis start date

Planned:

Date of interim report, if expected

Planned:

Date of final study report

Planned:
Sources of funding
Other

More details on funding

Italian Medicines Agency, Regional Drug Departme
Regulatory

Was the study required by a regulatory body?

No

Is the study required by a Risk Management Plan (RMP)?

Not applicable