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EMA backs DMD trial simulation models but calls for more data

Posted by on 31 January 2023
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European regulators have backed a simulation tech for Duchenne muscular dystrophy (DMD) drug trials and encouraging sponsors to share patient-level data its developers.

The EMA gave its verdict on the model-based Clinical Trial Simulation Platform (CTSP) in a letter that also acknowledged the development work undertaken by the Critical Path Institute’s Duchenne Regulatory Science Consortium (D-RSC).

“The EMA has issued this Letter of Support to encourage the further development and validation of the CTSP, as well as encouraging sponsors to share patient-level data with the D-RSC team.”

DMD is a rare, muscle-wasting, fatal disorder associated with mutations on the X chromosome. Efforts to come up with treatments have been hampered by the disease’s complexity, complexity, heterogeneity, and rarity, which make developing trial protocols very challenging.

The CTSP is designed to simulate disease progression using a series of clinically relevant outcome measures. It was developed using data from 1139 subjects with DMD over an age range of 4 to 34 years of age.

According to the Critical Path Institute the model “will help inform exploration of key design constructs including sample size, sampling scheme, patient enrollment criteria, dose and study design and accelerate medical product development.”

The EMA letter details feedback from the agency’s Committee for Medicinal Products for Human Use (CHMP) which includes some suggested improvements and development goals.

“The prospect of moving from the crude criteria to a more informative model for the design of the clinical trial is appealing. However, it is important to elaborate on the use cases, in order to make sure that such a model will be used to optimize trial design, including enriching the populations included in the trials.”

The CHMP also pointed out that the CTSP was developed using a limited data set, which it said should be expanded upon as the platform is further advanced.

“The adequacy of the database depends on what the most common features are, so far it is largely dominated by the ambulant DMD population, the 6-12 years age category, modal FU, time function tests, etc. So, the representativeness of the database for all DMD stages, coverage of endpoints, etc. needs additional data integration from further sources."

It added that “not all existing databases appear to have been shared with the Applicant. The overall dataset is largely dominated by the US region, data from Europe currently are scarce. Thus, the statement that the current database is representative of DMD patients and current DMD studies in general will need further justification.”


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