465 - Is it permissible to present efficacy results from real patient cases (provided the cases are anonymous and align with the TMA)?
Jennifer Carroll last edited by
Case studies and testimonials are not considered “evidence” in and of themselves per PAAB code s3.1.3. They may, however, be used to illustrate the findings of a randomized, controlled trial (RCT) that is presented in close proximity within the APS. In such cases, note the following provisions.The case must be aligned with the RCT with regards to the following provisions relating to “What?”, “How much?”, “When?”, and “Who?”:
"What: The outcome in the case is the endpoint in the study presentation
How much: The magnitude of effect conveyed in the case is similar to the magnitude in the study (e.g. degree of efficacy)
When: Time points or durations conveyed in the case are consistent with time points for which the corresponding data presentation in the APS shows statistical significance"
Who: The patient type presented in the case is representative of the study population. Please note that putting emphasis on a patient characteristic (e.g. race, gender, baseline value/status, treatment or disease history…etc) would require that the presented RCT data reflect that subpopulation (i.e. statistically significant data for the group which is emphasized).These same provisions would apply for a completely fictitious case. It is the disclaimer wording which distinguishes real from fictitious patients.For more information, see the tip following tip document on the types of evidence required for particular types of claims: