As our clinical trial pipeline unfolds and therapies become available, navigating the landscape is becoming increasingly complex. But today our landscape shone a bit brighter as the sun rose on the FDA campus early this morning and we watched members of our Duchenne community arrive.
Because today – our Duchenne community has again worked with the FDA to ensure that our community’s voice is heard.
Today, we are grateful to the FDA and the FDA Office of Pediatric Therapeutics (OPT), Office of the Commissioner (OC) for helping to facilitate our community’s dialogue by convening an advisory committee around an issue that has arisen in the course of patient participation in Sarepta’s ESSENCE study. The central question being asked to the committee is around the placement of an in-dwelling port in patients currently enrolled in a placebo controlled study – whether or not that should be allowed.
We are grateful to the members of the Duchenne community who have traveled to the FDA to share their personal experiences with port placement within Duchenne clinical trials. In his opening comments, Dr. Skip Nelson thanked the Duchenne community for raising this issue to the attention of the FDA, as well as the UCLA IRB for working with our community and the FDA. He also noted that this is the first time that a meeting of this type has involved a commercially-sponsored study (rather than a study sponsored by an academic institution) and acknowledged Sarepta’s willingness to work with the agency.
Following presentations today from Dr. Perry Sheieh, the UCLA IRB, the Bullers family, and Sarepta, there will be an Open Public Comment period. All of this testimony will be taken into consideration by the Advisory Committee – along with the written comments that were received prior to May 5 – during an afternoon discussion. The Advisory Committee will then vote on whether to amend the protocol of the Sarepta ESSENCE study to allow for the consideration of port placement. The outcome of today’s vote and discussion will be summarized into a recommendation and report for the FDA Commissioner. A final decision will be announced once that Commissioner has reviewed the summary report.
Today's questions before the committee:
Question One: Use of indwelling central venous access device in the ESSENCE clinical trial should be allowed.
- YES - There are circumstances in which an indwelling central nervous access device should be allowed in the ESSENCE clinical trial.
- NO - There are no circumstances in which an indwelling central venous access device should be allowed in the ESSENCE clinical trial.
Question Two (non-voting): If the ESSENCE protocol, as amended to include the use of an indwelling central venous access device, is allowed to proceed, please discuss the following issues:
- Should the choice and timing of placement of a clinically-appropriate central venous access device be left to the discretion of the study site investigator?
- Should the protocol include criteria for deciding when an individual study participant has difficulties with peripheral intravenous access (DIVA) such that use of a central venous access device may be appropriate?
- If the protocol should include such criteria, what type of criteria ought to be specified (e.g., number of failed attempts at establishing peripheral intravenous access, number of visits where there was difficulty establishing peripheral intravenous access, use of alternative visualization technologies)?
- How should the burden of undergoing multiple failed attempts at establishing peripheral intravenous access be taken into account (e.g., anticipatory anxiety, post-traumatic stress)
The Bullers Family - Testimony
If the FDA does ultimately recommend that the ESSENCE protocol be amended to allow for consideration of port placement, it would mean that each individual site’s IRB would then have the ability to review the local trial protocol to determine whether port placement within the ESSENCE study would be permitted for consideration by trial participants, in consultation with their principal investigator and treating clinician.
The written docket for comment on today’s proceedings will remain open through tomorrow (May 19). All comments submitted today and tomorrow WILL be considered in the final dossier prepared for review by the FDA Commissioner.
Patients deserve options. And we are grateful to all those who have worked to ensure that the experiences of our patient community are understood.
Today’s Advisory Committee meeting is being live streamed and can be viewed at: https://collaboration.fda.gov/pacm051817/.