US: Industry Calls for The Flexibility in FDA’s Paediatric Measurement Guidance

A trade association for the pharmaceutical industry and a number of pharmaceutical companies urged the US Food and Drug Administration (FDA) to be more lenient in its recommendations for paediatric measurements in clinical trials and questioned whether 12 months was long enough to provide useful data on growth and development.

The agency requested feedback on the draft guidance, which details how sponsors can systematically monitor paediatric growth in clinical trials for medicines used to treat both common and rare disorders. These comments were made in response to the request.

Industry opposes mandates that are too strict

Seven comments were sent to the agency regarding the guidelines, the majority of which expressed worry that the industry would be overburdened. According to Biotechnology Innovation Organization (BIO), FDA is advised to provide prescriptive guidelines in describing instruments used for monitoring growth and development.

BIO concern is the possibility for a disproportionate community to fulfil the requirements. Hence, asking for leniency of tools usage for the measurement so that no disturbance occurs while the community population grows bigger in future.

Meanwhile, Bristol Myers Squibb requests for leniency on guidance requires sponsors to calibrate and measure instrument for accurate data and suggest to do site visits and growth measurement were scheduled on the same day.

Otsuka also stands on the same point which site visit schedule should be on the same day as it would not disturb patient’s personal schedule and activity. Otsuka also states that rigid timing would not affect data efficiency.

Further details comment from the industry were concluded below:

• AstraZeneca: Calls for concern clarification on single-arm trials, as sponsors should discuss how the trail could be designed to avoid biased measurement of growth parameters.

• Novartis: State recommended growth measurement period of each study (at least 12 months). Puberty assessment should be flexible between men, women and non-binary gender. Provide example normative data chart for outside of USA trials.

• Otsuka: request different terminology applied on the proposed protocol, clarify on minimum trial period, revising statement about keeping discontinue study treatment participants in trial for their growth measurement data, inefficiency of rigid study visit timing, rephrase statement on the consistency of calibrate equipment, advising on error rate with the scales used, attire recommendation based on age-appropriate conditions, accessories and variance measurement at one time will negatively affected linear growth measurement and uses pragmatic approach for pubertal assessment

• Bristol-Myers Squibb (BMS): Ensure the involved site have proper measuring instruments, consider to do both study visit and growth measurement on same time, ensure scale instrument is standardized on site, provide standard sample of biomarker text and skeletal age evaluation.

• Biotechnology Innovation Organization (BIO): Suggesting FDA recommend accurate period to measure growth, providing guidance for timing of growth measurement, provide guidelines about the use of stretching and non-stretching methods for measuring height.

• Kidney Health Initiative (KHI): Recommend to consider nutrition and growth as an additional option of anthropometric measurements.

• Certara: Suggesting for reconsideration on a minimum period of growth measurement (12 months), leniency on specific equipment calibration, providing guidelines for diurnal variations for height and weight and reconsideration on puberty assessment as it might be personal and sensitive.

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