Factors regarding post-authorisation interaction with regulators that facilitate or impede the feasibility of post-authorisation randomised controlled trials as identified from the focus groups
| Facilitating factor | Illustrative quote | Impeding factor | Illustrative quote |
| Post-authorisation interaction with regulators | |||
| Optimisation trials in the authorised line(s) of treatment allow for randomisation (eg, different dose, a shorter treatment period) | For example, you have a medicine that you have used for a stage 4 but you think ‘okay, but is that dose okay? Can we lower that dose or can we shorten it? Or can it be administered another way?’ Those are excellent studies that you could perform post-authorisation. (Physician) | Possibility for alternatives to RCTs | An RCT, of course, that’s the gold standard, but the key message, or the key question behind, is that you need more data. And if you can generate the data in a different way, maybe that that’s open for discussion. (…) We also could replace them (RCTs) by registries or managed access programs, provided you do proper data gathering in an unbiased way of course. So it’s not the proper replacement of an RCT, but in the case that these are actually not possible, you can think of alternatives. (Industry representative) |
| Trial in an earlier line of treatment could lead to an extension of the indication | So for me, the standard situation we should face is that the confirmatory trial is actually put into a different setting, into a slightly different population, and then things from a recruitment and from a trial design point of view become easier. (Industry representative) | ||
RCT, randomised controlled trial.