Pharmacogenetic trials and RWE
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Assuming that a trial met the criteria of the RWE guidance, would a pharmacogenetic study evaluating a specific population be acceptable if the approved indication of the product was non-specific i.e. general population vs. select demographic? As a secondary point, given that it's a pharmacogenetic study and not an efficacy driven study, would there be any limitations to including data in APS if the results are only pharmacokinetic in nature?
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Assuming that a trial met the criteria of the RWE guidance, would a pharmacogenetic study evaluating a specific population be acceptable if the approved indication of the product was non-specific i.e. general population vs. select demographic? As a secondary point, given that it's a pharmacogenetic study and not an efficacy driven study, would there be any limitations to including data in APS if the results are only pharmacokinetic in nature?
Hey @cchristopoulos
We would have to assess the study given the indication and study design (including the nature of the pharmacogenetic parameter). In general, subgroup studies can be presented when the subgroup would fall within the indicated population and there are no other factors which would make it inconsistent with the TMA for use in that population. For example, a product indicated in adults could show a study in the subgroup of adults 40-60 years old. Similarly, it could show efficacy data for a subgroup of patients with renal impairment UNLESS the TMA has a warning or other cautionary copy against use in renally impaired patients.
For the second part of the question, it would not be possible to present RWE pharmacogenetic pharmacokinetic data that is not clinically relevant.
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Hey @cchristopoulos
We would have to assess the study given the indication and study design (including the nature of the pharmacogenetic parameter). In general, subgroup studies can be presented when the subgroup would fall within the indicated population and there are no other factors which would make it inconsistent with the TMA for use in that population. For example, a product indicated in adults could show a study in the subgroup of adults 40-60 years old. Similarly, it could show efficacy data for a subgroup of patients with renal impairment UNLESS the TMA has a warning or other cautionary copy against use in renally impaired patients.
For the second part of the question, it would not be possible to present RWE pharmacogenetic pharmacokinetic data that is not clinically relevant.
@jennifer-carroll Thank you for your response! As a follow up question, can you clarify what defines"clinically relevant"?
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Hello @cchristopoulos
The primary reason for the implementation of the RWE guidance document was to provide some flexibility around presenting evidence that was not previously accepted, in order to allow additional clinical data. Code section 3.1.4 states “Claims based upon laboratory or animal testing reports should be separated and cannot be used to imply clinical significance, unless there is evidence of a valid clinical correlation”. Unless the PK/laboratory-type data are unequivocally shown to have clinical relevance reflected by widely-accepted medical opinion and practice (e.g. authoritative consensus guidelines) and determined to be a valid endpoint (an example of this would be viral load in HIV), then a PK presentation would also not fall under the scope of the RWE guidance document.
