- As Alnylam Pharmaceuticals cheers Thursday's European approval of Onpattro, an influential U.S. drug pricing group called the first RNA interference therapy grossly overpriced, in a report published Wednesday evening.
- The Institute for Clinical and Economic Review concluded the drug's list price "far exceeds commonly cited cost-effectiveness thresholds" and would have to be slashed 90-95% to fall in line. That would drop the list price from $450,000 to between $24,700 and $46,488.
- Alnylam responded in a statement to BioPharma Dive, firing back at the report's "sensationalist conclusions ahead of ICER’s own public meeting and presentation of evidence." The drugmaker's stock was up roughly 1% in the first hours of trading Thursday morning.
The ICER analysis sets up the next battle for Alnylam, after completing a 16-year journey to take RNAi from a concept to marketed product in the U.S. and now Europe.
The European Commission approved Onpattro (patisiran) for treatment of hereditary ATTR amyloidosis in adults with stage 1 or 2 polyneuropathy. Earlier this month, the Food and Drug Administration OK'd the drug in the U.S. It is the first-ever RNA interference therapy to be approved in either market.
The question now becomes if Alnylam can extend its laboratory accomplishment into a commercial hit.
Using quality-adjusted life years (QALY), ICER found the $450,000 list price sails well above the group's target pricing of $50,000-$150,000 per QALY, coming in at $850,000 per QALY gained.
Dan Ollendorf, ICER's chief scientific officer, lauded the data on Onpattro along with Ionis and Akcea's Tegsedi (inotersen), calling them important scientific advancements.
"However, the announced price of patisiran, even taking into account expected discounts, far exceeds commonly cited cost-effectiveness thresholds," Ollendorf said in a statement.
Alnylam set a list price at $450,000 per year with an effective net price expected to be $345,000, following rebates and discounts. The company defended its price point in an emailed statement to BioPharma Dive.
Beyond calling ICER's conclusions sensationalist, a company spokesperson warned "against the use of broad generalizations and a questionable framework based on a myriad of assumptions to assign value to Onpattro."
Additionally, Alnylam adopted its own value-based system for Onpattro, offering rebates to insurers if it does not provide a defined clinical benefit, according to a company presentation.
Nearly simultaneously with the ICER report, the European Commission approved Onpattro on Thursday. Alnylam's head of Europe called the approval "the start of a new chapter in the treatment of this rare, rapidly progressive, fatal disease."
ICER's analysis may foreshadow concerns that European regulators might have with the pricing as well. In particular, England's National Institute for Health and Care Excellence recently deemed many high-profile drugs too pricey for coverage in the U.K., including Biogen's Spinraza (nusinersen), Sanofi and Regeneron's Dupixent (dupilumab) and Gilead's Yescarta (axicabtagene ciloleucel).
Value-based deals, where rebates are tied to how well a drug performs in the real world, may help alleviate some of the the fighting over high-priced therapies.
Ian Reynolds, manager of the Drug Spending Research Initiative at Pew Charitable Trusts, told BioPharma Dive he has observed an increased payer interest in value-assessment frameworks, including not just ICER but ones from places like the American Society of Clinical Oncology and the National Comprehensive Cancer Network.
"It's unclear what the impact of this particular report will be," said Ian Reynolds, "but there's certainly payer interest going forward."
Beyond pricing concerns, competition is coming for Onpattro.
The FDA is expected to make an approval decision on Tegsedi in October. Pfizer's tafamidis could be a competitor in the space too, as earlier this week it announced impressive data showing the drug cut the risk of death by 30% in ATTR cardiomyopathy.
ICER did find less clinical value in Tegsedi than Onpattro, setting Tegsedi's value-based price range lower than Onpattro's, with a range of $15,300 and $25,400 per year. Ionis and Akcea have not disclosed pricing plans.
"We are hopeful that inotersen’s manufacturer takes the report and meeting discussions under consideration when setting its price," Ollendorf said.
An ICER public advisory committee will hold a public meeting in Chicago on Sept. 13 to discuss this analysis and policy solutions.
"We welcome the opportunity to discuss ICER's evidence, how we arrived at the value of Onpattro, and our work to align with payers on value-based agreements at ICER's upcoming public meeting," an Alnylam spokesperson said.