The Jett Foundation Fighting Duchenne Muscular Dystrophy

Pink Sheet: Duchenne Group’s Presentation Is Milestone For Patient Involvement

May 4, 2016

Duchenne Group’s Presentation Is Milestone For Patient Involvement
Pink Sheet
By Derrick Gingery
May 2 2016 12:00 AM

Executive Summary
Advocacy group gets portion of Sarepta’s formal presentation period to present eteplirsen patient experience data, believed to be a first for an advisory committee meeting. Will it become commonplace?

FDA may have reached a new milestone in its effort to incorporate patient views into the drug review process when a Duchenne muscular dystrophy patient group obtained a prominent position in the eteplirsen advisory committee meeting presentations.

During the April 25 Peripheral and Central Nervous System Drugs Advisory Committee meeting, the Jett Foundation, a Duchenne advocacy group, was allotted the final 10 minutes of Sarepta Therapeutics Inc.’s formal presentation on the proposed Duchenne treatment.

Jett Foundation Executive Director Christine McSherry presented data on eteplirsen patient reported outcomes that the foundation collected through interviews and surveys. In the agenda (PDF) it was called an “Applicant Guest Speaker Presentation.”

McSherry’s talk is believed to be the first time a patient group has been allowed time to speak during the sponsor presentation, a point at the beginning of the meeting when the most positive aspects of the product in question and the need for the treatment generally are described.

The foundation unearthed “several key findings, all things that you would never expect to see in the normal progression of Duchenne,” McSherry told the committee.

Jett’s interviews with patients and caregivers found that spontaneous falls decreased or stopped among the boys taking eteplirsen. Boys also saw weakness and fatigue decrease or stabilize over time while on eteplirsen.

McSherry hopes it gave additional context to the clinical endpoints, which FDA had questioned after concluding that the historical data that Sarepta had relied on as a control did not in fact differ that much from the results of patients who were on treatment. “FDA now has the freedom to apply the patient stories and our report to the data,” she said.

FDA officials encouraged the advisory committee to take patient experiences into account when considering the strength of the clinical data and the questions associated with it.

Duchenne’s small and organized patient community also had a strong presence in the meeting room compared to other committee reviews of other orphan drugs. “I think with the majority of the patients here we have an incredible advantage” in assessing the drug, said Ellis Unger, director of the Office of New Drugs’ Office of Drug Evaluation I.

“One of the things that you can do is try to reconcile what we’ve heard from the patients with the data that we’ve seen presented by the company,” Unger said.

Weighing that evidence did not appear easy. Some said they were torn between the clinical data and the patient testimony.

Committee member Chiadi Onyike, associate professor of psychiatry and behavioral sciences at Johns Hopkins University School of Medicine, said the patient comments were meaningful evidence, but not properly measured in Sarepta’s studies.

The Jett Foundation presentation may be a high-water mark of patient involvement in drug reviews so far, but there are waves of activity in the area underway at FDA. The agency has conducted a number of disease-specific meetings with patients to gather patient perspectives on available treatments and unmet needs. It also has consulted patients for their thoughts during an application review.

The agency wants to better systematize patient-experience data so it can become a more formal part of its calculus in the next iteration of the user fee program.

Presentation Was Sarepta’s Choice

McSherry said in an interview with “The Pink Sheet” that she presented the data to FDA and suggested Sarepta use it in their presentation, but the company told her it was more appropriate for her to present it.

Sarepta offered to give her the time in their presentation. McSherry asked FDA if that was acceptable and Center for Drug Evaluation and Research Director Janet Woodcock agreed.

Woodcock sent a letter to her saying she had the time, but did not give any rules or restrictions, McSherry said.

“Because it had never happened before, I don’t think they knew what they were expecting,” McSherry said.

FDA told “The Pink Sheet” that it was “solely Sarepta’s choice” to include McSherry’s presentation and in the future those choices will remain with the sponsor.

“It would be up to a particular sponsor whether they would allot part of their time to a patient group or not,” the agency said.

Now that it has been shown to be possible, an advocate presentation, or at least a request for one, may soon become commonplace.

“To have [patient-reported outcomes] as part of the presentation is a big deal for FDA,” McSherry said. “They’re listening.”

An approval of eteplirsen may go a long way toward encouraging similar patient presentations. Approval of this product appears to be in doubt, however. FDA said during the meeting that it thought Sarepta could have completed a well-controlled clinical trial, and advisory committee members wondered why a placebo-controlled study was not done.

Hearing Length Did Not Affect Message Delivery, Group Says

Audience members gave McSherry a standing ovation when she completed the presentation.

It fed into the massive organized patient effort at the meeting that was also designed to help refute concerns that efficacy was lacking.

Between 800 and 1,000 people were expected at the Hyattsville, Md., conference center where the meeting was held. An overflow room was booked for participants who could not find seats in the main meeting area.

The open public hearing lasted more than 2.5 hours. Each speaker was allotted about three minutes and in many cases several people participated in each time slot.

By the end there appeared to be some fatigue among the committee members and others in attendance. But McSherry and Debra Miller, CEO and founder of the advocacy group CureDuchenne, said their message was received.

Miller said in an interview that the public hearing went well and she was not worried about its length.

The large basket of testimonials made it clearer that it wasn’t just a few patients that believed in eteplirsen, Miller said.

It also appeared that some may have stepped over a line in their efforts to push for an approval.

FDA said outside organizations had sent some committee members information considered germane to the proceedings prior to the meeting. The agency had to remind them that they could only consider FDA and Sarepta documents.

Patient Groups Not Giving Up

McSherry and Miller said they are not giving up on eteplirsen and were quick to remind advocates and patients that FDA has not made a decision on the NDA.

Eteplirsen’s review goal is May 26.

“I believe we have a drug here,” Miller said. CureDuchenne also helped fund early eteplirsen development.

The advisory committee meeting was the second for Duchenne groups. Their first foray into the process, the recent discussion of BioMarin Pharmaceutical Inc.’s drisapersen, proposed trade name Kyndrisa, did not go well.

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