Expert Insights

Expert Insights

Key Considerations in Pediatric Protocol Development

Key Considerations in Pediatric Protocol Development
19 April 2019

Developing a protocol for medicine aimed at children is significantly more complex than simply modifying a corresponding adult protocol for the age of the children. Numerous types of pediatric trials and trial designs can be leveraged depending on the objective of the investigation. Although these trial designs are not unique to pediatric studies, there are numerous specific elements that need to be carefully considered in pediatric clinical studies.

Common trial types and designs include:

  • Pharmacokinetic and combination pharmacokinetic and pharmacodynamic (PK and PK/PD) studies
  • Efficacy, pharmacodynamic (PD), safety studies
    • Monotherapy, or add‐on (combination), or multiple doses
    • Parallel group, or Cross‐over
    • Multicenter, single center
    • Multinational
    • Blinded or un-blinded trials
    • Controlled or uncontrolled trials
    • Consecutive phases or periods

The following approaches may also be of special interest in pediatric trials:

  • Adaptive Trial Designs - allows modifications to be made to the trial as it progresses based on predefined rules. This approach may help decrease the number of children that would be required to participate in a trial.
  • Age‐Staggered Inclusion - to protect the most vulnerable participants, these trials start in older children before including younger groups.

Pediatric trial protocols should address the same design elements typically adopted in adult trial protocols (see ICH E3) with equivalent quality standards. There are also a number of specific needs that need to be incorporated into the design of pediatric protocols, including:

  • Patient assent (if feasible)
  • Informed consent of one/both parents
  • Age-appropriate and therapeutically-specific endpoints
  • Additional safeguards for children that address risk versus benefit (according to 21 CFR 50 subpart D)
  • Detailed monitoring and safety safeguards that include rescue plans if appropriate
  • Justification for the inclusion of a placebo or sham procedures

Protocols should also strive to minimize the number of invasive procedures, such as blood draws for routine labs and PK/PD measurements. The incorporation of sparse sampling designs and PopPK analyses to minimize the frequency and volume of blood draws is highly desirable.

Careful attention should be paid to development and age-appropriate formulations that are palatable and acceptable for use in different age groups. Factors such as breastfeeding versus formula feeding in newborns and infants, which may impact gastric emptying time, may also be relevant for certain drugs administered by orally.

The availability of patients (particularly in rare diseases) should also be considered from both enrollment and statistical analysis perspectives in the early planning phases of protocol development. Also, complex protocol designs may pose a significant barrier to enrollment, especially if the protocol places unreasonable procedural demands on both patients and staff.

Synteract’s pediatricians, pharmacologists, and clinical operations teams work together to design well-thought out protocols that strive to minimize the burden on patients and sites while maximizing the quality of data to support pediatric labelling.

At Synteract, we draw on extensive experience in pediatric clinical trials. to discuss your pediatric protocol development needs. Learn more about our experience or contact us  to discuss your pediatric protocol development needs.

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