New peanut allergy therapies aren’t as effective — or nearly as inexpensive — as abstinence, says ICER
While peanut allergy therapies from Aimmune and DBV Technologies are locked in a race to the finish line, influential cost-effectiveness watchdog ICER has determined that neither therapy offers superior net health benefit compared to strict peanut avoidance, in a final report published on Wednesday.
Traditionally, peanut allergies are managed by avoidance, but the threat of accidental exposure cannot be nullified.
Some physicians have also been dosing patients with peanut powder orally, albeit off label. Aimmune’s $AIMT AR101 and DBV’s $DBVT Viaskin Peanut are set to be the pioneering peanut allergy treatments approved by the FDA. AR101 is effectively peanut flour, which must be mixed into pudding, applesauce, or other foods. Dosing is escalated gradually, and the therapy must be continued daily to maintain desensitization. Viaskin Peanut is a patch applied daily to the upper back that delivers peanut antigen to desensitize the patient. The patch must be worn for longer periods as time passes and used every day to maintain desensitization.
While the ICER panel acknowledged that desensitization as a surrogate outcome is promising, they were looking for data to demonstrate that desensitization is linked to improved quality of life and reduced reactions to accidental exposure to peanuts.
Based on surrogate outcomes — oral food challenges — AR101 appears to be more effective than Viaskin Peanut, but carries more adverse effects. In addition, in studies of AR101, Viaskin Peanut, and off-label therapy (OIT), epinephrine use and systemic allergic reactions increased, ICER found.
Overall, the body of evidence is not strong enough to suggest that AR101 or Viaskin Peanut offer a superior net health benefit versus strict peanut avoidance, the panel said, adding that there is also uncertainty about the long-term effects of either therapy.
“There is hope that the rates of systemic allergic reactions, epinephrine use, and reactions to accidental exposure will decrease with continued therapy, but this remains to be demonstrated. The potential need for lifelong therapy raises issues about long-term adherence to treatment, particularly during adolescence and young adulthood,” ICER said.
ICER did acknowledge, as it did in its draft report, that one of the limitations of its analysis is that it assumed the utility of the two peanut allergy therapies on the basis of existing data on food allergies, but not specifically the peanut allergy patient population, due to “the paucity of preference-weighted health-related quality of life estimates in food allergy patients and their caregivers.”
“With both AR101 and Viaskin, patients still need to spend the rest of their life avoiding peanuts and still worried about accidental exposure and wondering if their peanut treatment will protect them from anaphylaxis,” said James Wallen, who owns AMW Consultants, which is focused on assisting foreign biotechnology companies understand the US allergy immunotherapy market.
“With OIT (off-label oral immunotherapy), the patient is eating peanuts daily so they no longer have that fear of avoiding accidental exposure. When you are dealing with children (since both therapies only looked at or “succeeded” in kids), the QOL (quality of life) differences also have to consider the parent’s level of comfort with their child’s desensitization success. This is where OIT is completely different and vastly superior from both AR 101 and Viaskin in my opinion,” he told Endpoints News.
Neither company has divulged its pricing plans, so ICER used analyst projections to evaluate each therapy’s long-term cost-effectiveness: AR101 at $4,200/year and Viaskin Peanut at $6,500/year.
One of the ways ICER makes these calculations is on quality-adjusted-life-years (QALYs), a measure of the state of health of a person or group in which the benefits — in terms of length of life — are adjusted to reflect the quality of life.
Treatment with AR101 resulted in 0.75 incremental QALYs, while treatment with Viaskin Peanut came up relatively short, resulting in 0.26 incremental QALYs — when both were compared to no immunotherapy treatment over a lifetime, ICER’s analysis suggested.
“These benefits are due to improved subjective quality of life despite the relative rarity with which serious events occur. The ultimate value of these products will be determined by the prices that are set by the manufacturers and their long-term effectiveness,” ICER concluded.
ICER’s analysis also indicated only 41% of eligible patients could be treated with AR101 and 71% of eligible patients could be treated with Viaskin Peanut in a given year without exceeding ICER’s budget impact threshold of $991 million.
Both Aimmune and DBV said ICER’s conclusions were premature.
The final report is generally biased against immunotherapy — and fails to capture the full value of AR101, Aimmune said, underscoring that ICER did not include in its analysis the long-term efficacy and quality-of-life data from an open-label follow-on study, as well as clinical outcomes data from the company’s European late-stage trial.
“We believe this final report raises more questions than it answers and should be viewed as an early starting point for future conversations—not the final word—about the value of AR101,” Aimmune chief Jayson Dallas said in a statement.
Meanwhile, DBV voiced similar concerns, suggesting it disagreed with ICER’s methodology as well as the timing of the report.
Among other points of contention, DBV took issue with ICER’s inclusion of a Lancet study on oral immunotherapy (OIT), which did not include Viaskin Peanut clinical trials. “DBV has concerns with grouping Viaskin Peanut’s rating along with this systemic OIT analysis,” a spokesperson told Endpoints News.
Aimmune effectively leapfrogged DBV when the latter rescinded an application to market Viaskin Peanut patch last year in response to FDA concerns about the state of manufacturing and quality control data submitted.
An FDA decision for AR101 is expected in January 2020, while DBV is expected to submit its marketing application later in 2019. The so far untapped market is expected to grow to $4.5 billion in 2027 globally, according to GlobalData.