Developing medications for children poses unique regulatory complexities. In the UK, the Medicines and Healthcare Products Regulatory Agency oversees paediatric drug development and requires a Paediatric Investigation Plan, referred to as a PIP. A PIP provides a framework for systematically evaluating investigational drugs in paediatric clinical trials. However, navigating these standards requires careful planning. 

Developing safe and effective medications for children presents unique challenges. Paediatric populations are diverse, with significant differences in how drugs are absorbed, distributed, and eliminated based on factors like age and development. At the same time, children have specific healthcare needs that deserve tailored medical solutions.  

In the UK, the Medicines and Healthcare Products Regulatory Agency (MHRA) oversees paediatric drug development and regulation. The MHRA recognises both the vulnerability of paediatric patients and the importance of advancing paediatric therapies. Its guidelines aim to ensure medications are properly tested and authorised for paediatric use when clinically appropriate. 

The Paediatric Investigation Plan

One key MHRA requirement is the Paediatric Investigation Plan, referred to as a PIP. A PIP provides a framework for systematically evaluating investigational drugs in paediatric clinical trials. It outlines proposed study designs, safety assessments, dosing strategies and other critical trial components. 

Submitting a well-designed PIP is a prerequisite for marketing approval of new drugs intended for paediatric populations in the UK. However, it requires navigating complex regulatory standards and procedures.  

The overarching aim of a PIP is to ensure the necessary data are obtained through clinical trials in this vulnerable population to support authorisation of a medicine specifically for paediatric use. This includes assessing safety, dose selection, age appropriateness of the dose form and efficacy. The MHRA defines the paediatric population as individuals from birth to less than 18 years of age. 

Applying for Waivers and Deferrals

In some cases, the MHRA may grant a waiver or deferral of the PIP requirement, provided certain conditions are met. It is important to understand the criteria for a waiver or deferral and include a well-justified request in the application. 

The agency conducts compliance checks to ensure the PIP is being conducted in accordance with the PIP decision, including compliance with the agreed timelines for completion of measures. This review examines the necessary documentation such as a clinical study report to ensure the measures agreed in the PIP have been completed as agreed.  Applicants for marketing authorisation or variation will need to consider whether a PIP compliance check is required prior to submission of their applications.  

Key Steps to a Successful PIP

Once the PIP has been submitted, the MHRA will review it and provide feedback. . If the PIP is incomplete or poorly justified, the MHRA will request additional information or clarification. 

The following are some key steps in the road to an agreed PIP: 

  • Provide a detailed description of the condition and existing treatment methods in the UK and existing quality, nonclinical and clinical data on the medicine. 
  • Include a well-justified rationale for the proposed study(s) and the methods that will be used to assess the quality, safety and efficacy of the medicine in children. 
  • Address any feedback or concerns raised by the MHRA. 

The MHRA adopts a pragmatic approach to PIPs in the UK, acknowledging the unique needs of children and the necessity for tailored development of medicines for them.  

Conclusion

The MHRA mandates the implementation of PIPs to ensure a thorough evaluation of medications intended for paediatric use. The approval of these plans depends on how complete they are, and the justifications provided. The MHRA provides guidance to assist companies in adhering to these requirements. By following these guidelines, pharmaceutical companies can contribute to the development of safe and effective paediatric medications, thus ultimately enhancing children's health outcomes. 

Are you planning to apply for approval in the UK? At NDA, we have extensive experience of MHRA procedures. Contact us by using the form below, to discuss how our regulatory expertise can help you adhere to MHRA guidelines for your product's development. 

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