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PPMD Working with FDA to Plan Landmark Pediatric Advisory Committee Meeting to Improve Clinical Trial Experience

Later this spring – likely on or about May 18 – FDA’s Pediatric Advisory Committee will convene to consider the issue of allowing in-dwelling ports in Sarepta’s Essence trial.

Once again, our community is at a pivotal moment in our history where we can affect change and improve the clinical trial experience for our children.

The Backstory

Time and again in our Duchenne community, we see brave kids and their warrior parents work to change the landscape. After all, it’s that exact spirit upon which PPMD was founded. And with that same spirit that family after family has transformed their personal moments into broader movements that have benefited us all.

When the Bullers family screened into Sarepta's Essence study, they had no idea that their participation would soon become a policy issue before the FDA. But after enrolling in the study and several weeks of traumatic infusions for their son Nicholas, they inquired about the possibility of opting for a port rather than subject Nicholas to the difficulty of finding a vein for his infusions. Port placement facilitates direct access into the vein for delivery of the infusion, preventing repeated attempts to get into peripheral veins. What they learned was that the trial protocol did not allow for the placement of an in-dwelling port. But, like most families in our community, that answer didn't end their inquiry.

The Bullers kept asking questions and learned that the FDA does not allow for in-dwelling port placement in pediatric clinical trials with placebo controls, as the concern is that:

  1. Port placement for a placebo is unnecessarily invasive, and
  2. Only placing ports in participants on active drug would unblind the study.

The Bullers enlisted the support of the Principal Investigator at their study site who agreed with them – and together they appealed to their clinical trial institution’s IRB. They asked that the Essence trial protocol be amended to allow for in-dwelling port placement at the discretion of trial participants.

Their belief was that this trial was exceptional given the fact that:

  1. All study participants will eventually roll over to active drug (so port placement would not be unethical if someone were in the placebo arm at the time of port placement), and
  2. This is a longer study than most so the duration of time young boys are being asked to participate in infusions warrants to option of the port.

The IRB referred the case to the FDA and Erin Bullers sent a personal letter to Dr. Robert “Skip” Nelson. Dr. Nelson is the Deputy Director and Senior Pediatric Ethicist in the Office of Pediatric Therapeutics (OPT) in the Office of the Commissioner (OC). Dr. Nelson was working with PPMD on the planning of our upcoming Unique Burdens of Pediatric Clinical Trials meeting in April, when he received Erin’s letter. He agreed that this issue deserved immediate attention.

What is a Pediatric Advisory Committee?

According to the FDA website, a Pediatric Advisory Committee is:

“…advises and makes recommendations to the Commissioner of Food and Drugs regarding (1) pediatric research conducted under sections 351, 409I, and 499 of the Public Health Service Act and sections 501, 502, 505, 505A, and 505B of the Federal Food, Drug, and Cosmetic Act; (2) identification of research priorities related to pediatric therapeutics and the need for additional treatments of specific pediatric diseases or conditions, (3) the ethics, design, and analysis of clinical trials related to pediatric therapeutics, (4) pediatric labeling disputes as specified in section 3 of the Best Pharmaceuticals for Children Act (Public Law 107-109), (5) pediatric labeling changes as specified in section 5 of the Best Pharmaceuticals for Children Act (Public Law 107-109), (6) adverse event reports for drugs granted-pediatric exclusivity and any safety issues that may occur as specified in section 17 of the Best Pharmaceuticals for Children Act (Public Law 107-109), (7) any other pediatric issue or pediatric labeling dispute involving FDA-regulated products, (8) research involving children as subjects as specified in 21 CFR 50.54, and (9) any other matter involving pediatrics for which FDA has regulatory responsibility.”

We have learned that this is the first review in the history of the Pediatric Advisory Committee where the sponsor will be at the table. The first review that’s not academic. Where the outcome has bearing on a clinical trial underway. This is an open IND and the committee is open to reviewing the issue in real-time.

We Need YOU!

We are grateful to the FDA for understanding the importance and urgency of this issue and for giving it the attention it deserves. We are grateful to the Bullers and each family who has worked to raise this issue.

We are hopeful that this Pediatric Advisory Committee meeting will yield an outcome that allows for greater personal/provider choice.

We are grateful to the families and providers who have fought so hard to raise this issue up – to the boys who’ve endured despite great discomfort – and to the FDA for understanding that this issue matters.

Our community is grateful to all those who’ve participated in trials requiring infusions who would like to lend your expert insight into the proceedings – we need YOU.

If you have experience to lend we will be asking you in the coming weeks to submit your written testimony or join us in person at this Pediatric Ad Comm.

The Details – Coming Soon!

PPMD has been working closely with the FDA to plan this landmark meeting. Details, including date/time/location as well as instructions on submitting both written and oral testimony will be coming soon.

As always, PPMD will provide you the pertinent logistical information as soon as it is published by the FDA in the Federal Register.

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