Euro Roundup: EMA seeks feedback on regulation of weight control clinical trials in children
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.
Concept Paper
EMA starts consultation on data quality framework for EU medicines regulation
EMA is seeking feedback on the application of the data quality framework for European medicines to real-world data (RWD).
The agency has published a 36-page draft document that extends the existing framework to include recommendations for assessing the quality of RWD. EMA wants to improve the quality of RWD for regulatory use and enhance the assessment and documentation of RWD quality. The document provides background on RWD and the application of the framework before addressing the three core topics.
EMA has focused on characterizing the systems and processes that underpin data, establishing quality metrics for RWD and assessing the suitability of a dataset for answering a specific research question. The sections are intended to provide “actionable and focused recommendations” for assessing RWD quality.
The systems and processes section provides guidelines for ensuring the steps involved in the “collection or recording and transformation are reliable and offer the necessary guarantees.” EMA said RWD cannot be used for regulatory purposes, regardless of its source, in the absence of “reasonable evidence that the information provided is true and not accidentally or intentionally altered.”
EMA is accepting comments on the draft until 31 January.
Draft Guidance
Swissmedic publishes position paper on running first-in-human clinical trials
The Swiss Agency for Therapeutic Products (Swissmedic) has described the requirements sponsors and sites need to meet when running first-in-human (FIH) clinical trials.
Swissmedic created the position paper with the Swiss Association of Research Ethics Committees (Swissethics). The organizations said subjects in FIH trials may “be at unforeseen risk of serious adverse events including potential emergency situations such as uncontrolled immune cascades, anaphylactic shock or even cardiac arrest.”
To promote safety and mitigate risks, Swissmedic and Swissethics want sponsors and trial sites to meet certain requirements. The sponsor requirements include providing a detailed description of the dose escalation steps, having a clear definition of stopping criteria and selecting a suitable trial site via a quality control process.
Another section of the paper covers the requirements for investigator site personnel and facilities. The agencies said sites should organize “the possibility to transfer subjects to a hospital with access to an intensive care unit” before the start of a trial. Procedures between the site and intensive care unit need to be established to determine “responsibilities and undertakings of the transfer and care” of patients.
Swissmedic and Swissethics have based their recommendations on ICH GCP E6.
Position Paper
EMA finalizes guideline on clinical requirements for certain hemophilia therapies
EMA has finalized a guideline on the clinical requirements for non-replacement therapy in hemophilia A and B.
The agency released a draft for consultation in October 2023 after identifying a need to update its advice in light of the development of alternatives to plasma-derived or recombinant FVIII or FIX products. The new products are mainly developed for prophylaxis, typically have a different route of administration than factor replacement therapies and can potentially treat a broad patient population.
EMA addressed the implications of those characteristics in the draft. The final guideline hews closely to the draft. EMA has made small changes throughout the text, for example by clarifying that developers that want approval in moderate or mild hemophilia need data in both subgroups. The agency also added a line about the inability of its general recommendations to define all aspects of development programs.
Final Guideline
MHRA selects five participants for AI Airlock pilot scheme
The UK Medicines and Healthcare products Regulatory Agency (MHRA) has selected five technologies for its AI Airlock pilot scheme. MHRA designed the scheme to help its team and the companies understand the challenges of regulating the technologies.
Participants in the pilot include Philips, which will evaluate the generative AI function it has added to a system for storing and sharing radiology data. The function creates a summary of the key findings that a radiologist observes during the imaging examination. Philips will include the summary in a section of the report that includes the information the radiologist thinks is most important for the referring physician.
Philips will test the product “by generating synthetic radiology reports which the generative AI can then use to create the patient impression,” MHRA said, and the AI Airlock will “assess the use of synthetic data that mimics complex, different patient cases in training AI models to handle a wide array of clinical situations.”
Press Release, More
Other news:
EMA has published guidance on how marketing authorization holders and applicants can pay its fees and charges. The agency released the guidance one month before the new fee regulation takes effect. In the document, EMA addresses receiving, paying and querying its invoices. EMA Guidance
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