Regulatory and Evidence Strategy for GTx Pediatric Expansion
- Built an evidence base to expand from adolescent into pediatric.
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Regulators required a compelling justification for pediatric inclusion and for the use of a non-traditional study
design in a rare disease.
- The program needed additional evidence beyond trial data to support regulatory decision-making.
- Engaged FDA in parallel during trial execution to support transition from adolescents to pediatric patients.
- Committed to a natural history study to support pediatric expansion discussions with FDA.
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Used burden-of-disease evidence to persuade health authorities that a delay-to-treat arm was sufficient in place
of a traditional control arm.
- Strengthened the regulatory case for pediatric expansion using a broader evidence package.
- Secured health authority acceptance of a more feasible study design in a rare disease setting.
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Contributed to a successful approval by combining trial evidence with RWE and natural history support.
