Pediatric diseases are those that affect adolescents and children under 18 years old. While these diseases represent a substantial public health concern, most drugs have traditionally been developed and approved only in adults.
In an effort to promote the development of drugs for pediatric use, the US Congress passed the Pediatric Research Equity Act (PREA) and the Best Pharmaceuticals for Children Act (BPCA). Together, these laws require drug companies to study their products in children, with some exceptions, but also offer valuable incentives to perform these studies.
Understanding Sponsor-side requirements under these statutes is critical. Failure to comply with these laws may result in a drug being considered misbranded and subject to enforcement action. On the other hand, performing pediatric studies in compliance with these statutes comes with the possibility of extended patent exclusivity. Transferable priority review vouchers are also available to reward companies developing drugs for rare pediatric diseases.
While these incentives have encouraged more pediatric studies, they have not eliminated the challenges associated with them. Pediatric drug development can be fraught with difficulties, including a lack of correlation between safety, pharmacokinetics (PK), and pharmacodynamics (PD) in adults and children, study design restrictions, and difficulty in study enrollment.
When it comes to developing pediatric drugs, it is vitally important to understand the nuances inherent in the field. Ensuring the appropriate study designs, understanding the many ways in which PK and PD can differ in young people, and establishing early engagement with regulatory authorities like the FDA are critical for success.
Nuventra’s Pediatric Disease Experience
Nuventra can perform any of our wide range of services for drugs to treat Pediatric Disease indications. Some of our recent project experience in this area includes:
- Data Analysis and Interpretation
- Data Management
- Initial Pediatric Study Plans (iPSPs)
- Meetings with Regulatory Agencies and Study Centers
- Modeling and Simulation
- Multiple Exposure Bridging Modeling & Simulation Project
- Overall Program Strategy
- Pediatric Investigation Plans (PIPs)
- Pediatric ODAC Meeting
- Phases 1-4 Clinical Studies
- Protocol and Study Design
- Regulatory Support
- Sampling Schedule Development
- Strategy and Decisions
Visit our services page to see our full range of services and to learn more about what our drug development consultants can offer your program.
Pediatric Disease Indications
Nuventra has experience with a number of Pediatric Disease indications, including:
- Allergic Rhinitis
- Atopic Dermatitis
- Eosinophilic Esophagitis
- Fragile X Syndrome
- Nausea and Vomiting
- Sickle Cell Disease