Masterstudiengang "Drug Regulatory Affairs"

Master-Thesis

Medicinal products for minors - Different regulatory approaches and conduction of clinical trials on the example of antidiabetics ***

Mona Linser (Abschlußjahr: 2022)

Summary
Language: English
The issue of the lack of paediatric research is commonly known. Europe addressed this problem by introducing the Paediatric Regulation in 2006. The aim of this master’s thesis was to analyse the legal situation and development that has taken place in other industrialised countries, such as Australia, Japan and Canada in comparison to the EU. As part of this, the number of approved drugs and the number of clinical studies using the example of antidiabetics for children were considered.
No law similar to the EU Paediatric Regulation has been passed in the three countries, but there are only different support programs that point in the right direction.
In these regions, over 20 medicinal products are authorised for the treatment of type 1 and type 2 diabetes mellitus, apart from the combination products. Of these more than 20 products, only two are approved for the paediatric population in Japan. For Canadian and Australian children, only two drugs other than insulins are available. In addition, in Canada and Japan, it is not possible to provide an in-label therapy according to the S3-Guideline for children suffering type 2 diabetes mellitus due to the lack of approval for Metformin.
Reasons for not granting a child approval are usually based on an authority’s waiver with the grounds that the disease does not occur in the paediatric population or due to the lack of paediatric study results. For most of the medicines marketed in Canada, Japan and Australia, no information for the non-existence of the children's approval are available, only the EU provides partial data.
In the EU, 11% of all clinical studies and 9% of all interventional clinical trials were conducted with paediatric participation. In Canada, the number of children participating in clinical studies for diabetes mellitus is even higher: 13%. As many Canadian children take part in interventional clinical trials as in Europe. Japan and Australia bring up the rear (Japan 10% and 2%, Australia 9% and 6%). The most common reason given for not conducting or failing a study is the small number of paediatric participants. Recruiting paediatric participants appears to be a major challenge. Furthermore, justifications are commonly missing and there is a lack of traceability and transparency with regard to the further use of the study data, including whether the studies contribute to the submission of approval for a drug.
Unfortunately, the data from the registers should be treated with caution, as registration of a clinical study is not mandatory in Canada and Australia. In Japan, registrational requirement only started in 2018. Since interventional trials conducted in the EU have been registered since 2004, the EudraCT is considered to be the most comprehensive, most complete and most transparent of all the registers presented.
The number of children suffering from diabetes mellitus has increased significantly in recent years. Although the issue has been tackled more consciously and vigorously since the beginning of the 21st century and the development of paediatric medicines has progressed at unprecedented levels, especially in Europe and the USA, this should only be a start to considerably improve the situation for children all over the world.
Pages: 87
Annexes: 17 (146 pages)