Reflection on the Pharmaceutical Formulation Challenges Associated with a Paediatric Investigation Plan for an...

Area of Science:

• Pharmacology
• Pharmaceutical Sciences
• Pediatric Medicine

Background:

• Regulatory requirements in Europe mandate pediatric investigation plans (PIPs) for new patented drugs.
• Developing pediatric formulations for off-patent medicines remains a significant unmet need, despite incentives.
• The EU-funded LENA project addresses this gap for the off-patent drug enalapril.

Purpose of the Study:

• To develop a novel pediatric dosage form for enalapril.
• To support the treatment of heart failure in pediatric patients aged 0-18 years.
• To navigate the Paediatric Investigation Plan (PIP) process for off-patent medications.

Main Methods:

• Formulation development of a new pediatric enalapril dosage form.
• Selection of appropriate excipients and dosage form.
• Methodology for product administration and patient acceptability evaluation.
• Addressing challenges within the Paediatric Investigation Plan (PIP) framework.

Main Results:

• Successfully developed a new pediatric dosage form for enalapril.
• Identified key formulation and administration challenges.
• Gathered insights into patient acceptability for the new dosage form.
• Successfully navigated the PIP process for an off-patent drug.

Conclusions:

• The LENA project successfully developed a pediatric enalapril formulation, addressing a critical unmet need.
• Key formulation, administration, and acceptability challenges were overcome during product development.
• This work provides a model for developing pediatric formulations of off-patent drugs, facilitating PIP submissions.