ICER deems unapproved Aimmune, DBV peanut allergy drugs unaffordable, sparking backlash

Peanuts in shell
Aimmune and DBV haven't yet disclosed their pricing plans for their experimental drugs to desensitize children against peanut allergies. (Pixabay / riteshman)

Two small companies, Aimmune Therapeutics and DBV Technologies, are racing to break into what analysts expect will be a billion-dollar market for drugs to treat peanut allergies. But neither of their experimental treatments, Aimmune’s AR101 and DBV’s Viaskin, has been approved by the FDA, nor have the companies said anything about pricing plans.

That didn’t stop the Institute for Clinical and Economic Review (ICER) from issuing a dreaded “affordability alert” on the drugs, however. The report, released earlier this week, blasted both drugs for being unaffordable based on analysts’ estimates that AR101 could cost $4,200 a year and Viaskin $6,500.

AR101 and Viaskin were designed to build an immune tolerance to peanuts in children with the allergy. With the help of an independent appraisal committee, ICER determined that there wasn’t enough evidence to prove that either drug offers a health benefit over simply avoiding peanuts. In order to avoid crossing ICER’s acceptable annual healthcare budget threshold of $819 million, Aimmune would need to capture 41% of eligible patients and DBV 71%, ICER concluded.

David Rind, M.D., ICER’s chief medical officer, acknowledged in a statement that the drugs have been proven to desensitize patients to the peanut allergy, but that they also raise the risk of “increased allergic reactions” and use of epinephrine—the shot kids and parents carry around to stop life-threatening reactions.

“Caregivers and patients must remain vigilant about avoiding peanuts even while on desensitization therapy, and future clinical trials will need to demonstrate whether long-term benefits of these approaches outweigh their short-term risks,” Rind said.

RELATED: DBV pulls peanut allergy filing after FDA faults data details

ICER’s report sparked an immediate backlash from the drug developers and the broader allergy community.

Aimmune issued a statement Thursday urging stakeholders not to draw any conclusions from the ICER report.

“The resulting final report is biased against immunotherapy, generally, and fails to specifically capture the full value of AR101,” the company said. Aimmune pointed to results from two phase 3 trials in which AR101 increased the median dose of peanut protein that patients could tolerate by 100-fold.

DBC provided feedback to ICER that the agency failed to incorporate in the final report, a company spokesperson said in an emailed statement. DBV listed several flaws in ICER’s methodology, including the fact that the agency relied heavily on a Lancet study on oral immunotherapy that didn’t include DBV’s treatment, and that there isn’t enough real-world data on quality-of-life improvements that children may ultimately enjoy once the treatments are available.

DBV said “the cost-effectiveness of Viaskin Peanut can only be meaningfully assessed after peanut allergy patient-reported quality of life data are available and analyzed with reliable approaches to calculate associated health state utility—neither of which are currently available.”

Patient advocates are up in arms, too. The Asthma and Allergy Foundation of America called the ICER review “premature” and blasted the organization in a statement for potentially raising concerns that could end up dissuading insurers from covering the drugs if they’re approved.

“ICER’s own economic model calculates between a 30-70% positive improvement in ICER’s quality of life measure from these therapies when considering the societal costs associated with peanut allergies,” the statement said. “This improvement demonstrates the importance and opportunity for development of new therapies to address all food allergies, not just peanut allergies.”

RELATED: Novartis' SMA gene therapy would not be cost-effective if priced over $1.5M: ICER

Aimmune is expecting an approval decision on AR101 by January, and the FDA has planned a public meeting to discuss the drug on September 13. DBV fell behind its rival in the peanut-allergy race last December, when the FDA faulted the company for providing inadequate data on manufacturing and quality control.

DBV withdrew its filing and is now working on a revised submission, but not under the most ideal of circumstances. The company is running out of cash, and in March, two top execs, COO Charles Ruban and CDO Laurent Martin, headed for the exit.

Analysts at Piper Jaffray who follow Aimmune weren’t fazed by the ICER review. In a note sent to clients Thursday, they called the report “clearly biased” and the conclusion that AR101 provides no benefit over peanut avoidance “laughable.” The analysts predicted that Aimmune’s negotiations with insurers would not be negatively impacted by the ICER report.

This is not the first time ICER has made a ruling on cost-effectiveness based on hypothetical prices. In April, it issued a report suggesting that Novartis’ then-experimental gene therapy to treat spinal muscular atrophy (SMA), Zolgensma, would not be a good value at any price over $1.5 million.

After the gene therapy was approved and priced at $2 million, ICER changed its tune, saying that new data and Novartis’ plan to tie payments to performance made Zolgensma worth that high price.

Whether or not a similar cooperative dynamic plays out between ICER, Aimmune and DBV remains to be seen. Meanwhile, ICER published a laundry list of policy recommendations on peanut-allergy treatments for all of the stakeholders, from patient advocates to payers.

For example, ICER said that companies developing the drugs need to develop biomarkers to help clinicians determine when patients should go on and off desensitization therapy. And the organization urged the FDA to require manufacturers to run additional trials to prove “clinically meaningful benefits for patients.”