You get data-driven results that some food allergy organizations say miss the mark, and which therapy manufacturers call “premature.”
The Institute for Clinical and Economic Review (ICER), the U.S. non-profit pharmaceutical drugs and devices watchdog, undertook a time-consuming review of two peanut allergy therapy products in development. At a stakeholder discussion in Oakland, CA on June 11, 2019 therapy manufacturers, allergists and patient advocates weighed in with their responses to ICER’s evidence report on the two products: Aimmune Therapeutics’ AR101 and DBV Technologies’ Viaskin Peanut patch. These are, respectively, oral immunotherapy (OIT) and skin patch treatments for peanut allergy.
ICER revealed at the meeting that it is grading the two therapies as “P/I” – “promising but inconclusive.” In a joint press release a day later, Food Allergy Research and Education (FARE) and the Allergy & Asthma Network (AAN) expressed disappointment in the review and urged the ICER panel to broaden patient considerations in its final report.
“We are discouraged that ICER did not more thoroughly consider the complexity and nuances of available research on immunotherapy in evaluating the benefit these therapies can have on patients managing peanut allergy,” they said.
Comparing to Avoidance
ICER crafts its reports from data to analyze the effectiveness and value of new drugs and medical devices, and evaluate both the long-term impact on patients and costs to the overall U.S. health system. “It is challenging to fully evaluate the impact of these therapies without placebo-controlled assessments of the change in quality of life,” the evidence report noted.
In the review, each of the yet-to-be approved peanut allergy therapies were compared against the longstanding practice of avoidance of peanut to prevent reactions. ICER’s “promising but inconclusive” rating represents a slight downgrade for both treatments from an original “moderate certainty” of a net health benefit.
An ICER spokesperson told Allergic Living that this change was based in part on adding a new Lancet review of peanut allergy OIT studies to the data under consideration. That study, which has raised controversy in the food allergy community, favored the practice of avoidance, finding that OIT raised the odds of having a peanut-allergic reaction (of varying degrees). What the review in The Lancet didn’t analyze was food allergy families’ willingness to accept some level of informed reaction/anaphylaxis risk.
Of the ICER review, FARE and AAN said: “These therapies shouldn’t be assessed without a deep understanding of the patient journey, including the trade-offs that patients are willing to make for the potential benefit they will gain. Patient preference and expectations around outcomes must be paramount to consider in [ICER’s] analysis.”
ICER Review Too Soon?
“The silver lining is that they concluded that it is promising,” said Dr. Stephen Tilles, Aimmune’s senior medical affairs director, of ICER’s review of AR101, which the FDA is considering for approval.
“We’ve said all along felt that ICER’s review of our treatment is premature, and actually inappropriate at this time, due to this lack of published quality of life data in this space,” he told Allergic Living.
Dr. Todd Green, vice president of medical affairs (North America) for DBV Technologies, similarly dubbed the review “premature”. DBV won’t even submit its application for approval to the FDA until the fall. He told Allergic Living that “we still don’t understand the rationale for changing our rating based on The Lancet article,” since it assessed OIT, not the skin patch treatment.
ICER stressed the need for quality of life outcomes with the therapies. Recent results from an extension study of the AR101 PALISADE trial did find significant quality of life improvements on the therapy. This was raised at the June 11 meeting, but Tilles says ICER is seeking longer-term data.
This again is premature – with therapies this new, such data are not yet available. And Green points out that it’s not easy to be assessing patients’ quality of life while they’re taking part in a blinded, controlled study where they are not allowed to know whether they’re on the actual therapy or receiving a placebo version of it.
As for costs to consumers, he and Tilles told Allergic Living these are not set on either of the products. “It’s really too early,” said Green. ICER’s final report on the two peanut allergy therapies will be released on July 10.
Real Life Experience
The ICER meeting did allow advocates, allergists and patients to bring forward the food allergy community experience as part of the process.
“On a positive side, ICER’s review has clearly brought to light that peanut allergy has a major impact on caregivers,” said Kenneth Mendez, president and CEO of the Asthma and Allergy Foundation of America (AAFA). “They just need more data to quantify that.”
ICER’s evidence report cites AAFA’s April 2019 survey of 853 caregivers of children with peanut allergies. The survey found that 86 percent of caregivers said the peanut allergy was constantly on their minds, while over half of participants were fearful of an accidental peanut exposure. The personal burden is significant.
While desensitization treatment won’t be for everyone, Mendez says “it should really be up to an informed patient and their doctor to understand whether the patient wants to take the risk. There’s a risk of anaphylaxis, just like when you take [environmental] allergy shots.”
Patient advocate Caroline Moassessi* appreciated the chance to voice her views on why the status quo of peanut avoidance isn’t the most encouraging and why many families are eager for a therapy. “I wanted them to understand that just ‘avoiding the food’ isn’t that easy. It means that every single morsel must be scrutinized to know if it’s allergy-safe,” she said.
“Families like mine with food allergies must count on food manufacturers and restaurants to take care of us. Avoidance means, we must rely on the kindness of strangers,” she says.
The fact that ICER is encouraging drug companies to bring forward quality of life data may be a positive down the road, according to Mendez. “The FDA does not ask for that data, they ask: ‘Is it safe and is it effective?’ So ICER is trying to tell the drug companies, ‘you need to go beyond that.’ Clearly there are quality of life and caregiver issues, but we need quantitative data to prove that for ICER,” he says.
Tilles isn’t against this conversation either. “We look forward to continuing to discuss the importance of this emerging treatment area.”
*Patient advocate Caroline Moassessi is a contributor to Allergic Living, but was not representing our organization at this meeting.