Posted 28 April 2015
By Alexander Gaffney, RAC
Patients and their advocates appear to be getting more prominent attention from the US Food and Drug Administration (FDA) if a recent release of documents in advance of an upcoming advisory committee meeting is any indication.
This story has been updated to include additional information from FDA and to correct one inaccuracy: Patient testimonials have always been made available to advisory committee members in advance of meetings, even if they are not publicly posted on FDA's website. We regret the error.
FDA's advisory committees are convened by the agency to address matters of regulatory importance. For example, FDA might ask an advisory committee to recommend whether a new drug should be approved by its regulators. While the recommendation of the committee's experts is influential, it is not binding on the agency.
Advisory committee meetings at which a drug is seeking a recommendation for approval are extremely important for a product's sponsor. A positive recommendation for approval can ease regulators' concerns that a drug might not be ready for approval. A recommendation against approval can all but ensure a drug's application is denied and that its sponsor must conduct tens—even hundreds—of millions of dollars in additional testing.
So several days prior to each advisory committee meeting, when FDA releases materials to be used by its committee members, industry watchers pay close attention in an attempt to determine whether a drug might be approved or rejected.
To date, most experts rely primarily on a single document: FDA's Briefing Document. The document, which can run more than 100 pages in length, is authored by FDA reviewers and contains a thorough analysis of the drug and whether FDA's review staff is confident in its approval.
A Focus on Patients
The briefing document is, along with several other documents, used by the experts on FDA's advisory committees to inform their discussion and debate about whether a drug should be approved.
But one set of documents seen by committee members but not the public has this week been posted by FDA in advance of its 29 April 2015 meeting of the Cellular, Tissue and Gene Therapies Advisory Committee (CTGTAC).
For this meeting—and perhaps the first time ever—FDA has posted three "testimonials" submitted to patients and patient advocates. While such testimony has always been available to members of the advisory committees, they have not, as a matter of course, been made available to the public prior to advisory committee meetings taking place.
Two of those patients’ testimonials are specific to the drug being considered by the committee, Amgen's T-Vec (talimogene laherparepvec). One emotional testimonial referred to it as a "miracle drug" that cured his/her father of melanoma. A second testimonial was more clinical in its approach, offering experts a concise breakdown of his/her experience (and success) using the drug.
"I am very thankful (an understatement) that I was able to participate in the trial and would like to share my experience with the committee," the patient added.
A third testimonial, authored by the National Patient Advocate Foundation (NPAF), spoke in general support of genetically modified therapies. "Melanoma patients are a prime example of a patient population that would benefit from a virus that targets only specific cancer cells, infects them and replicates continuously within the cell until it explodes to promote an overall immune system boost against their cancer," it wrote.
However, FDA included a note in the group's testimony that its advocacy contained a "general statement" that did "not appear to be specific to one product."
UPDATED: What Will the Effect Be?
The inclusion of patient testimony is not entirely novel. FDA advisory committees have long permitted patients to speak at FDA advisory meetings on behalf of specific products and their own experience.
When reached for comment by Regulatory Focus, FDA spokeswoman Tara Goodin confirmed that while FDA "does not typically post comments from third-party advocacy groups … these comments are always available for public viewing at the meeting itself (in a binder)."
"There is not and will not be a standard posting protocol moving forward for all FDA advisory committee meetings," she added, noting that the decision to post patient advocacy materials is a decision which is left to each FDA official overseeing a particular advisory committee meeting.
While other FDA advisory committees may choose to make these patient testimonials more widely available to the public in advance of their meetings, it is not required, Goodin confirmed.
What remains to be seen, however, is whether this decision might open up regulatory floodgates at the agency. By increasing the visibility of patient testimonials, FDA might encourage further participation by and input from patient groups and, by extension, calls for the testimonials to be more readily available to the public.
But the effectiveness of the testimonials may be another matter entirely. Most patient testimony is short on the sort of thing that tends to dominate advisory committee discussions: data. While anecdotes can serve as useful examples, they are rarely enough to sway expert opinion.
Cellular, Tissue and Gene Therapies Advisory Committee