Thursday, January 12, 2017

FDA Has Just Released Its Draft Guidance for Industry "Multiple Endpoints in Clinical Trials"

For those who are interested in the design and analysis of clinical trials, FDA has just released its long-anticipated draft guidance for industry: Multiple Endpoints in Clinical Trials. The release has been delayed for about 4 years. I have heard several years ago this guidance has been completed and ready to be released. For example, in a presentation by Dr Huque in 2013, it was mentioned "The Draft Guidance, Multiple Endpoints in Clinical Trials, is near completion.  It is expected to be released soon for public comments."

In contrast, EMA issued its guidance “Points to Consider on Multiplicity Issues in Clinical Trials” more than a decade ago. I believe that EMA is considering to update its guidance and its guidance released in 2002 is obsolete now. This can be reflected in its "Workshop on multiplicity issues in clinical trials" organized in 2012. Perhaps this will be delayed due to Brexit.

Similarly to other statistical heavy FDA guidance (such as the guidance on adaptive design and non-inferiority clinical trials), the guidance "Multiple Endpoints in Clinical Trials" provided a lot of details, not only about the situations the adjustment for multiplicity is needed, but also the various procedures for handling the multiplicity issues.

The next FDA guidance in statistical area should be in missing data and Bayesian clinical trial. For missing data, EMA published its "Guidance on Missing Data in Confirmatory Clinical Trials". FDA sort of relies on the report "the Prevention and Treatment of Missing Data in Clinical Trials" as its general guidance. For Bayesian clinical trials, FDA CDRH division is ahead of CDER/CBER and has published its "Guidance for the Use of Bayesian Statistics in Medical Device Clinical Trials". This makes sense since Bayesian approach is more often used in medical device trials and CDRH is more amenable to accepting the Bayesian designs.

       RAPS (2016) Multiple Endpoints in Clinical Trials: FDA Issues Draft Guidance

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