Consistency with TMA
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Would like some clarity to ensure my understanding of consistency with TMA is correct in relation to the patient population attribute.
Example: Drug X is indicated for Disease Y. The pivotal trial for this indication only included patients with stable metastases Z. It excluded (exclusionary criteria) patients with active metastases Z. However, the indication for Drug X does not specify any restrictions with use of Drug X in those with Disease Y and active metastases Z.
Would an RWE paper where the overall patient population consists of patients with stable AND active metastases Z be considered not consistent with the the TMA?
The overall study population is not "exceeding the product’s indication" (wording taken from PAAB RWE guidance) but it is exceeding the patient population of the pivotal trial which resulted in the indication for Drug X.
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Would like some clarity to ensure my understanding of consistency with TMA is correct in relation to the patient population attribute.
Example: Drug X is indicated for Disease Y. The pivotal trial for this indication only included patients with stable metastases Z. It excluded (exclusionary criteria) patients with active metastases Z. However, the indication for Drug X does not specify any restrictions with use of Drug X in those with Disease Y and active metastases Z.
Would an RWE paper where the overall patient population consists of patients with stable AND active metastases Z be considered not consistent with the the TMA?
The overall study population is not "exceeding the product’s indication" (wording taken from PAAB RWE guidance) but it is exceeding the patient population of the pivotal trial which resulted in the indication for Drug X.
Hey @tk2022
It sounds like this might be a specific question about a specific case. We’d suggest submitting for review as the forum is guiding principles. Each case is assessed based on it’s merits in the context of all relevant documents. The answer provided below is with the caveat that it is a general response.
In general, an RWE study is not excluded from advertising based on a pre-planned evaluation of a subgroup which would be within the limitations of the indication. “Consistent with the TMA” with respect to patient population states “1.1.2 Patient population: The APS presentation must be derived from analysis of patients that fall within the indicated population and are aligned with any relevant contraindications from the TMA. In instances where an overall study population exceeds the product’s indication, it may be possible to present data from a pre-planned patient subset that reflects the indicated patient population or relevant subset thereof.”
