Novel statistics to handle rare diseases and small sample sizes using Bayesian techniques: Application to Multiple Sclerosis (MS) and Systemic Lupus Erythematosus (SLE) in pregnancy

Study identification

• PURI:
  https://redirect.ema.europa.eu/resource/43421
• EU PAS number:
  EUPAS43420
• Study ID:
  43421
• Official title and acronym:
  Novel statistics to handle rare diseases and small sample sizes using Bayesian techniques: Application to Multiple Sclerosis (MS) and Systemic Lupus Erythematosus (SLE) in pregnancy
• DARWIN EU® study:
  No
• Study countries:
  Finland

  France

  Germany

  Italy

  Norway

  Spain
• Study description:
  The aim of the study is to evaluate the safety to the mother and child of medications that are used by women suffering from SLE or MS during pregnancy using novel statistical methods. The ConcePTION platform will be used to analyse observational data from multinational cohort studies based on population based secondary data sources in Europe. The following studies will be undertaken: a. Identification of the most appropriate algorithm to identify MS and SLE in women of child bearing age and childhood infections in children in selected data sources on the ConcePTION platform. b. Evaluation of the utilisation trends and patterns of medications in women with SLE and MS (a) of childbearing age and (b) in pregnant women before, during and after pregnancy c. Evaluation of the safety to the mother and child of medications that are used by women suffering from SLE or MS during pregnancy. Analytic results from each data sources will be combined using novel meta-analytic techniques to overcome the issue of small numbers that will occur due to rare exposures and outcomes being studied. d. Evaluation of the increased risk of the occurrence of a major congenital malformation associated with first trimester exposure to medications that are used by women suffering from SLE or MS during pregnancy. EUROmediCAT data will be used to perform case-malformed analyses and to compile case series of congenital anomaly cases to identify possible safety indications.
• Study status:
  Ongoing

Research institutions and networks

Institutions

CHU de Toulouse - Hôpital des Enfants

Pharmacoepidemiology and Drug Safety Research Group (PharmaSafe), University of Oslo

Norway

Drugs and Pregnancy, Finnish Institute for Health and Welfare (THL)

Finland

Ulster University

United Kingdom (Northern Ireland)

University of Bordeaux

France

The Foundation for the Promotion of Health and Biomedical Research of Valencia Region (FISABIO)

Spain

University of Swansea Wales, UK, University of Ferrara Emilia Romagna, Italy, Ulster University North Ireland, UK, Institute for prevention Research and epidemiology Leipniz, Germany, University of Bordeaux France, The Foundation for the Promotion of Health and Biomedical Research of Valencian Region Spain

Networks

ConcepTION

Study timelines

Date when funding contract was signed

Planned: 01/04/2021

Actual: 01/04/2021

Study start date

Actual: 08/05/2020

Date of final study report

Planned: 31/03/2024

Sources of funding

EU institutional research programme

More details on funding

IMI ConcePTION

Regulatory

• Was the study required by a regulatory body?:
  No
• Is the study required by a Risk Management Plan (RMP)?:
  Not applicable