The study must yield meaningful data and combination studies are limited
What the document says“A molecularly targeted pediatric cancer investigation referred to in subparagraph (A) shall be designed to yield clinically meaningful pediatric study data that is gathered using appropriate formulations for each age group for which the study is required, regarding dosing, safety, and preliminary efficacy to inform potential pediatric labeling.”
The rewritten paragraph requires the investigation to be designed to produce clinically meaningful pediatric data on dosing, safety and preliminary effectiveness, using appropriate formulations for each age group, to inform potential pediatric labeling. A combination study may be required only where the application drug has a single new active ingredient, or more than one where the combination has not been approved before but each ingredient is in a product already approved for an adult cancer.
What the document actually says“A molecularly targeted pediatric cancer investigation referred to in subparagraph (A) shall be designed to yield clinically meaningful pediatric study data that is gathered using appropriate formulations for each age group for which the study is required, regarding dosing, safety, and preliminary efficacy to inform potential pediatric labeling.”
The study must give real answers about children. It must use a form of the drug fit for each age group. It must cover dose, safety and early results.
A drug made for adults may not come in a form a child can take. Data from a study that ignores that is of little use. The rule says the study must fit the child.
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