Introduction to Confirmatory Trials

If it has been demonstrated that exposure is related to a relevant clinical outcome, such as efficacy or toxicity, and that exposure can be optimized by PK-guided dosing, it could be logically assumed that PK-guided dosing would result in better treatment outcomes without the need for randomized confirmatory trials.

If it has been demonstrated that exposure is related to a relevant clinical outcome, such as efficacy or toxicity, and that exposure can be optimized by PK-guided dosing, it could be logically assumed that PK-guided dosing would result in better treatment outcomes without the need for randomized confirmatory trials.

Therefore, to accelerate the development process and better characterize the treatment effect of new investigational therapies, other TTE endpoints such as progression-free survival and event-free survival (EFS) are applied as primary efficacy endpoints in some confirmatory trials, either as a surrogate for OS or as a direct measure of clinical benefits.

Therefore, to accelerate the development process and better characterize the treatment effect of new investigational therapies, other TTE endpoints such as progression-free survival and event-free survival (EFS) are applied as primary efficacy endpoints in some confirmatory trials, either as a surrogate for OS or as a direct measure of clinical benefits.

5 Thus, ARD spearheaded the field by providing new, thoroughly peerreviewed findings at an early stage of our understanding of COVID-19 treatment so that others could perform confirmatory trials and patients could benefit timely from this advance.

ADs can be applied from early-phase trials to confirmatory trials.

5 Thus, ARD spearheaded the field by providing new, thoroughly peerreviewed findings at an early stage of our understanding of COVID-19 treatment so that others could perform confirmatory trials and patients could benefit timely from this advance.

ADs can be applied from early-phase trials to confirmatory trials.

Assessment of efficacy in important subgroups – such as those defined by sex, age, race and region – in confirmatory trials is typically performed using separate analysis of the specific subgroup.

Assessment of efficacy in important subgroups – such as those defined by sex, age, race and region – in confirmatory trials is typically performed using separate analysis of the specific subgroup.

As with other approvals under the Accelerated Approval Program, continued approval for this indication may be contingent upon verification of a clinical benefit in confirmatory trials.

As with other approvals under the Accelerated Approval Program, continued approval for this indication may be contingent upon verification of a clinical benefit in confirmatory trials.

In this study, we describe the methods and designs of breast cancer confirmatory trials published between 2001 and 2020.

In this study, we describe the methods and designs of breast cancer confirmatory trials published between 2001 and 2020.

Findings can inform the development of evidence-based guidance to facilitate design decisions for confirmatory trials.

Findings can inform the development of evidence-based guidance to facilitate design decisions for confirmatory trials.

This model was applied to predict the performance of the entire set of 3024 accessions, and the top predicted performers were sent to Madagascar for confirmatory trials.

This model was applied to predict the performance of the entire set of 3024 accessions, and the top predicted performers were sent to Madagascar for confirmatory trials.

The FDA's Accelerated Approval program is under scrutiny after four drug companies announced plans in recent months to withdraw indications for checkpoint inhibitors that, despite showing early promise, failed to demonstrate a survival benefit in confirmatory trials.

The FDA's Accelerated Approval program is under scrutiny after four drug companies announced plans in recent months to withdraw indications for checkpoint inhibitors that, despite showing early promise, failed to demonstrate a survival benefit in confirmatory trials.

Blinded, placebo-controlled confirmatory trials of higher methodological quality are warranted, especially using oral, short-term BCAAs-enriched supplements within the context of recent ERAS programs.

Blinded, placebo-controlled confirmatory trials of higher methodological quality are warranted, especially using oral, short-term BCAAs-enriched supplements within the context of recent ERAS programs.

Further investigation of PB-201 100 mg twice daily in confirmatory trials is warranted.

Further investigation of PB-201 100 mg twice daily in confirmatory trials is warranted.

Although these findings require replication in both retrospective and prospective confirmatory trials, they emphasize the need to incorporate similar genetic analyses of choline-related polymorphisms in other FASD-choline trials, and to test for similar associations within the general FASD population.

Although these findings require replication in both retrospective and prospective confirmatory trials, they emphasize the need to incorporate similar genetic analyses of choline-related polymorphisms in other FASD-choline trials, and to test for similar associations within the general FASD population.

As part of this approval, the manufacturer must conduct post-approval studies—known as phase IV confirmatory trials—to “verify the anticipated clinical benefit.

These results warrant further validation in larger confirmatory trials.

As part of this approval, the manufacturer must conduct post-approval studies—known as phase IV confirmatory trials—to “verify the anticipated clinical benefit.

These results warrant further validation in larger confirmatory trials.

Over the past 6 years, the FDA has approved six ICIs for more than 75 oncological indications (35 through an accelerated pathway), and the term “dangling accelerated approvals” was coined: duplicates or similar indications for which the required confirmatory trials failed to show a clear benefit, and yet their marketing authorization continued [3].

However, one of the barriers to the uptake of Bayesian adaptive designs for confirmatory trials is limited experience with how they may perform compared to a frequentist design.

Over the past 6 years, the FDA has approved six ICIs for more than 75 oncological indications (35 through an accelerated pathway), and the term “dangling accelerated approvals” was coined: duplicates or similar indications for which the required confirmatory trials failed to show a clear benefit, and yet their marketing authorization continued [3].

However, one of the barriers to the uptake of Bayesian adaptive designs for confirmatory trials is limited experience with how they may perform compared to a frequentist design.

Positive findings from modern trials are catalysing developments, but it is questionable whether current confirmatory trials are sufficient for advancing our understanding of safety and best practice.

Positive findings from modern trials are catalysing developments, but it is questionable whether current confirmatory trials are sufficient for advancing our understanding of safety and best practice.

In confirmatory trials, the rates of missed residual cancer [false-negative rates (FNRs)] were unacceptably high (> 10%).

In confirmatory trials, the rates of missed residual cancer [false-negative rates (FNRs)] were unacceptably high (> 10%).