4:56 PM
Sep 13, 2018
 |  BC Extra  |  Company News

ICER panel: hATTR treatments have low value

The Institute for Clinical and Economic Review's Midwest Comparative Effectiveness Public Advisory Council (CEPAC) voted unanimously in separate tallies that the long-term value for money was low for hATTR treatments from Alnylam Pharmaceuticals Inc. (NASDAQ:ALNY) and Akcea Therapeutics Inc. (NASDAQ:AKCA).

The panel reviewed ICER's evidence reports for Alnylam's Onpattro patisiran and Akcea's Tegsedi inotersen, in which the group found that the treatments, at an annual price of $345,000, were not cost effective at the $150,000 per quality-adjusted life year (QALY) threshold. Alnylam launched Onpattro last month at a net price of $345,000. The biotech said it had agreements in principal with some insurers on a pay-for-performance arrangement for the RNAi therapeutic (see "Agreement to Differ").

ICER's cost-effectiveness analysis presented at the meeting did not take into consideration Alnylam’s outcomes-based arrangements.

Onpattro is approved in the U.S. to treat polyneuropathy caused by hereditary transthyretin (TTR)-mediated amyloidosis (hATTR) in adults.

Tegsedi is under FDA review with an Oct. 6 PDUFA date, and Akcea has yet to disclose a price.

Separate from the cost-effectiveness analyses, all 11 panel members felt that the burden of the disease on the caregiver and the ability of a new treatment to relieve that burden was an important factor to take into consideration in determining the value and access to these new drugs.

Patients who were taking Onpattro or Tegsedi spoke during the meeting about how the treatments improved their ability to care for themselves and reduce or eliminate reliance on their family members as well as assistive devices such as canes.

But patients also encouraged the drug companies as well as payers to come together to ensure patients will have access to both of these new treatments.

"We urge everyone, companies and payers to ensure patients have access to these treatments and that they have a choice. I can't underestimate how important choice is and both treatments have considerable value," said Kristen Hsu, executive director of clinical research at the Amyloidosis Research Consortium.

During a policy roundtable following the vote, Young Fried, VP of pharmacy plan services at HealthPartners, said that as a payer, HealthPartners can't afford to cover every new rare disease drug and called on patients to advocate for lower prices. "I understand that the drug companies have these patient access programs, but wouldn't it be great if instead of these, they just offered a lower price. Why don't they just decrease the price up front so everyone can have access to it and so we don't have to go through these debates," Young said.

Alan Eisenberg, VP of global government relations and public policy at Alnylam, said that the biotech took multiple factors into consideration when it set the price of Onpattro, including the effect of the treatment to halt or reverse polyneuropathy in patients with hATTR, as well as the rarity of the disease, the $2.5 billion R&D investment to get the drug across the approval line, and the need to invest in its next-generation treatment for the indication.

ICER will use the feedback to draft its final report on the therapies, which is expected on Oct. 4.

Onpattro is an IV RNAi therapeutic that targets the TTR gene using second-generation lipid nanoparticle (LNP) technology. Tegsedi is a TTR antisense inhibitor.

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