–Report also evaluates evidence on inotersen; During public meeting of Midwest CEPAC on September 13th, a policy roundtable will discuss possible contextual factors that may affect decision-making for ultra-rare conditions such as amyloidosis —

BOSTON, August 29, 2018 – The Institute for Clinical and Economic Review (ICER) today released an Evidence Report assessing the comparative clinical effectiveness and value of therapies to treat hereditary transthyretin (hATTR) amyloidosis. The report includes inotersen (Akcea Therapeutics) and patisiran (OnpattroTM, Alnylam Pharmaceuticals). Patisiran was recently approved by the FDA. An approval decision on inotersen is expected in October of 2018.

“Available data on clinical effectiveness suggest that these therapies offer important advancements for patients with hATTR amyloidosis,” noted Dan Ollendorf, ICER’s Chief Scientific Officer. “However, the announced price of patisiran, even taking into account expected discounts, far exceeds commonly cited cost-effectiveness thresholds. During our public meeting, the Midwest CEPAC will discuss evidence on these therapies with a policy roundtable of experts, working to identify policy solutions to ensure appropriate patient access despite the extremely high costs. We are hopeful that inotersen’s manufacturer takes the report and meeting discussions under consideration when setting its price.”

This Evidence Report will be the subject of an upcoming public meeting of the Midwest Comparative Effectiveness Public Advisory Council (Midwest CEPAC) in Chicago, IL on September 13, 2018. The Midwest CEPAC is one of ICER’s three independent evidence appraisal committees comprising medical evidence experts, practicing clinicians, methodologists, and leaders in patient engagement and advocacy.

A draft version of this report was previously open for a four-week public comment period. The updated Evidence Report and voting questions reflect changes made based on comments received from patient groups, clinicians, drug manufacturers, and other stakeholders. Detailed responses to public comments can be found here.

Key Findings

The two new therapies have not been compared to each other, and statistical comparisons were not possible given differences in trial design, study populations, and outcome measures. Each drug was compared only to the supportive care (i.e., placebo) arm of its clinical trial. Evidence provides high certainty that inotersen is at least clinically comparable to best supportive care, and there is moderate certainty that inotersen provides a small or substantial net health benefit. Evidence on patisiran provided moderate certainty of a substantial net health benefit, with high certainty of at least a small net health benefit compared to best supportive care.

Economic analyses assessing the long-term cost-effectiveness of patisiran compared to best supportive care, concluded that, at a net price of $345,000, the therapy far exceeds commonly accepted thresholds of $50,000-$150,000 per quality-adjusted life year (QALY) at $850,000 per QALY gained. To align costs with the added benefits for patients, the list price of patisiran would need to be discounted by 90%-95%. While a list price has not yet been announced for inotersen, ICER’s analyses suggest that the therapy would align with the benefit to patients if priced between $15,300 and $25,400 per year.

ICER’s report notes that decision-makers often give special considerations to therapies for ultra-rare diseases such as amyloidosis that may lead to coverage and funding decisions at higher thresholds for cost-effectiveness.

About ICER

The Institute for Clinical and Economic Review (ICER) is an independent non-profit research institute that produces reports analyzing the evidence on the effectiveness and value of drugs and other medical services. ICER’s reports include evidence-based calculations of prices for new drugs that accurately reflect the degree of improvement expected in long-term patient outcomes, while also highlighting price levels that might contribute to unaffordable short-term cost growth for the overall health care system.

ICER’s reports incorporate extensive input from all stakeholders and are the subject of public hearings through three core programs: the California Technology Assessment Forum (CTAF), the Midwest Comparative Effectiveness Public Advisory Council (Midwest CEPAC), and the New England Comparative Effectiveness Public Advisory Council (New England CEPAC). These independent panels review ICER’s reports at public meetings to deliberate on the evidence and develop recommendations for how patients, clinicians, insurers, and policymakers can improve the quality and value of health care. For more information about ICER, please visit ICER’s website.