Making sure all newly developed drugs are safe for children

UCL researchers have established a freely accessible paediatric medicine ingredients database to help accelerate safe drug development and provide information for health professionals and parents.

Safety information about pharmaceutical ingredients is essential to drug development, but children’s medicines require specialised advice. The Safety and Toxicity of Excipients for Paediatrics (STEP) database, developed by Professor Catherine Tuleu and Dr Smita Salunke at UCL School of Pharmacy, is a unique, freely accessible global resource that compiles toxicity information of excipients (non-active ingredients) for paediatric medicines.

Since its launch in 2014, the STEP database has rapidly grown into an essential resource, with more than 3,000 registered users from 44 countries across six continents, with the pharmaceutical industry representing 70% of users. 

The need for easily accessible, trustworthy information 

Excipients are substances which are added to the active ingredients of a medication to help preserve them, make them easier to take (for example, by forming pills or lotions) or easier for the body to process. Excipients are inevitable to manufacture drug products but pose special challenges when making medicines for children as their bodies may respond to these substances differently to adults. 

It is important for health care professionals, but also patients and parents, to be able to easily access trustworthy information to identify and manage ‘problem’ excipients in medicines for children. Since 2007 the European Medicines Agency (EMA) has required manufacturers to justify using all excipients in children’s medicines. However, at the time no specialist database of excipient safety for children that took account of their needs and developing bodies existed.

Accelerating the development of safe paediatric medicines

Professor Catherine Tuleu founded the European Paediatric Formulation Initiative (EuPFI), a consortium to address pharmaceutical challenges with paediatric medicines. In collaboration, Professor Tuleu and Dr Salunke, the CSO of the EuPFI and the US Pediatric Formulation Initiative (NICHD), developed the database application and systematic tools to collate and quality assess the safety and toxicity information. The aim was to accelerate the development of new paediatric medicines, while ensuring prescription and administration of safer medicines in children. 

STEP’s development began with a survey of stakeholders to determine what information the database should contain and how it should be presented. Then, 150 sources of data on excipients were shortlisted using an intensive systematic selection process. Ten key excipients’ information/data were searched, reviewed and relevant data extracted from these sources. The results from this pilot study were used to test the way the database managed this information, and how it would be input into it before expanding the excipient repertoire. Finally, the new database was evaluated from the perspective of its end users for ease of use and relevance.

A freely accessible, essential resource

The resulting STEP database was launched in 2014, and it now lists 75 excipients. Because the EMA was a consortium partner it was directly involved in the database’s development and testing, and along with academic researchers, funders and manufacturers it has selected and prioritised the ingredients that are included.

The database is freely accessible and searchable via a web application. As such, it has rapidly grown into an essential resource, with more than 3,000 registered users from 44 countries across six continents, with the pharmaceutical industry representing 70% of users. 

The database has not only improved patient safety but also accelerated development of products worldwide, reduced staff time and costs. Several major pharmaceutical manufacturing companies, such as Pfizer, Roche, GSK and Novartis have cited the STEP database in their published information as well as pharmacists, clinicians, regulators

Links

• Professor Catherine Tuleu’s academic profile
• Dr Smita Salunke’s academic profile
• UCL School of Pharmacy
• UCL Faculty of Life Sciences
• UCL Faculty of Life Sciences REF 2021

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• Image credit: iStock / yaoinlove