The European Medicines Agency (EMA) has published a draft concept paper on the clinical evaluation of weight control medicinal products in children.
EMA adopted the existing addendum on weight control in children in 2008. In 2016, the agency revised the main guideline on the clinical evaluation of medicinal products used in weight control. Now, EMA is planning to revise the addendum to align the text with the 2016 guideline, where appropriate, and to “reflect recent developments in this rapidly evolving field.”
The agency wants to change the addendum due to the approval of new medicinal products for weight management and the current clinical practice guidelines. For example, EMA is proposing to update the introduction to the addendum to reflect current scientific knowledge and development.
Other planned changes relate to the definition of overweight/obesity in children and clinical trial design. Discussing the definition, EMA said the current addendum cites body mass index (BMI)-related metrics to determine overweight and obesity in children.
The agency has identified shortcomings with the current approach. BMI should be adjusted according to age and sex, EMA said, and the “metrics may not be ideal as they do not directly reflect adiposity nor the consequences of excess adiposity in an individualized way.” The agency said, “other staging tools may help to better characterize the target population for pharmacological treatment.”
On trial design, EMA listed five areas that it plans to change. The agency is considering dropping the requirement for a three-month run-in period, which it said may discourage people from participating in trials. It is also considering changes to the inclusion and exclusion criteria “in light of the intended target population.” EMA is also looking at the requirement for trials to observe patients for six months after stopping treatment.
“Stopping therapy may however have its drawbacks and may not add a lot of information. Therefore, the need for a follow-up after discontinuation and its length will be considered,” EMA said.
The other two trial design points relate to endpoints and comparators. EMA currently recommends trials use change in BMI standard deviation score or percentage change in BMI from baseline as the primary endpoint. The agency is considering alternatives and wants to capture “medical, mental and functional outcomes.” EMA is also looking at ways to address the dropouts seen in placebo-controlled trials.
The draft is open for comment until 28 February. EMA is aiming to release the draft addendum for a six-month consultation within six months of completing the concept paper consultation. The timeline, which includes six months to finalize the draft, suggests EMA could adopt the final addendum in the second half of 2026.
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