Drugmakers seek tweaks to FDA guidance on measuring development in pediatric trials

Bristol Myers Squibb, Novartis, AstraZeneca and industry group BIO are all seeking alterations to the FDA’s recent draft guidance to help sponsors measure the growth and evaluate the pubertal development in pediatric clinical trials.

The 10-page draft guidance, released last October, explains how if a drug could have an impact on the growth or pubertal development of participants in a trial, such trials should include accurate, serial measurements and recordings of growth parameters. The FDA calls on and explains how sponsors should look into participants’ changes in age, length/height, weight, head circumference in some circumstances, and more.

“Sponsors enrolling pediatric participants in clinical trials should use a sexual maturity rating (e.g., Tanner Staging) to evaluate and document pubertal development at baseline (i.e., trial entry) and at regular intervals based on the potential safety concerns associated with the drug and the pubertal development stage of the pediatric participant,” the draft says.

Commenting on the draft, BIO noted that the FDA is being prescriptive on which tools should be used to measure growth and development, such as via an orchidometer or digital scale, etc.

“These tools may not be readily available in sites with fewer resources, especially in underserved communities,” BIO wrote. “We suggest that the guidance clarify flexibility around what tools may be used to avoid unintended consequences, i.e., creating challenges to enrollment of a greater population of patients from such communities.”

Bristol Myers even went so far as to question why the guidance is necessary for some pediatric trials, noting:

It would be helpful to provide scientific justification as to why the assessment of growth and pubertal development are important in the setting of chronic illnesses and literature supporting the need for this specific regulatory guidance.

Novartis tells FDA to clarify that length is only recommended to be measured up to 2 years of age, so the draft should say, “length or height.” The large pharma also sought to ensure that the FDA not place restrictions on how long companies should collect this type of info, recommending that the agency call for “at least” 12 months rather than just 12 months of data.

And AstraZeneca’s entire comment on the draft boils down to a suggestion that the FDA delete one sentence:

We suggest FDA delete the sentence, ‘For single-arm trials, the sponsor should discuss with the appropriate review division how the trial could be designed to avoid biased measurement of growth parameters.’, as growth measurements are not subjective and highly unlikely be biased. Thus, this sentence does not seem necessary and should be deleted or, alternatively, modified for better clarity of what FDA is concerned about.

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