Background The UK MHRA has introduced a risk-proportionate approach to the approval and management of clinical trials of investigational medicinal products (CTIMP). The aim is to reduce the complexity of regulations and governance by identifying key hazards and promoting risk mitigation. Prospective risk management will reduce dependence on quality controls and monitoring during trials.

Methods A risk assessment template for neonatal CTIMPs was developed, based on the TINN Ciprofloxacin Pharmacokinetic Trial. The study was compared to standard medical care, an analysis of study design was undertaken to ensure the protection of participants’ rights and data reliability. The tool was moderated by experts in regulations and clinical trials.

Results The trial was classified as low-risk (Type A) from an IMP perspective. This allows reduced regulatory monitoring and IMP management. Ciprofloxacin is administered off label and involves vulnerable participants. The evidence required to mitigate this included the fact that the drug is administered as standard clinical care, there is published use of Ciprofloxacin for neonates and national prescribing guidelines.

4 key risk areas were identified: 1) patient safety/rights 2) reliability of results 3) research sites 4) governance. To mitigate the risks during trial conduct and the collection of data required specific neonatal trial management tools, training, standard operating procedures and systems for cross checking data.

Conclusion Neonatal CTIMPs are not always high risk. Regulations and inspections need to be proportionate to the risks arising from the trial-specific interventions and procedures. Risks can be mitigated by bespoke neonatal trial design.