Thanks. Naive question: should the FDA grant or take away approval based on efficacy? I get it for safety but why not let docs decide efficacy? I can see that reimbursement should be based on two phase III trials, as people should not pay for drugs that do not work. But approval (or not) for efficacy seems to be heavy handed
Regulators generally require positive evidence of efficacy before authorising a drug. If negative trials were considered equally acceptable, on the grounds that efficacy ultimately did not matter, then what would be the purpose of conducting a comparative randomised trial in the first place?
Actually, two randomised studies aren’t necessarily needed for reimbursement. Many drugs are being reimbursed based on a small single randomised study... The important thing for both payers and regulators is whether the pre-specified hypothesis of the study was actually demonstrated.
For example, if the trial is designed as a non-inferiority study, the drug doesn’t need to be better than the control. It just needs to show that it is within the pre-specified non-inferiority margin. But if the trial was designed to show superiority and superiority isn’t demonstrated, then the trial is negative.
In areas of high unmet need though, regulators may make an exception for a negative trial and consider additional evidence or approve the drug for a certain subgroup.
Thanks. Naive question: should the FDA grant or take away approval based on efficacy? I get it for safety but why not let docs decide efficacy? I can see that reimbursement should be based on two phase III trials, as people should not pay for drugs that do not work. But approval (or not) for efficacy seems to be heavy handed
Thanks, what about that : would you approve a 30 000 $ per month sugar-(free)-pill if it's safe?
Why not. So long as no reimbursement. If you want to pay for a $30k sugar pill you should have that option, no? Who am I to say you shouldn't
Regulators generally require positive evidence of efficacy before authorising a drug. If negative trials were considered equally acceptable, on the grounds that efficacy ultimately did not matter, then what would be the purpose of conducting a comparative randomised trial in the first place?
See my original comment. Very important for reimbursement.
Actually, two randomised studies aren’t necessarily needed for reimbursement. Many drugs are being reimbursed based on a small single randomised study... The important thing for both payers and regulators is whether the pre-specified hypothesis of the study was actually demonstrated.
For example, if the trial is designed as a non-inferiority study, the drug doesn’t need to be better than the control. It just needs to show that it is within the pre-specified non-inferiority margin. But if the trial was designed to show superiority and superiority isn’t demonstrated, then the trial is negative.
In areas of high unmet need though, regulators may make an exception for a negative trial and consider additional evidence or approve the drug for a certain subgroup.
That makes sense. Thank you
That makes sense. Thank you