Institute for Clinical and Economic Review Final Report Highlights Uncertainty in Long-Term Safety and Effectiveness of New Treatments for Hereditary Transthyretin Amyloidosis, Discusses Options for Insurance Coverage Criteria

Boston – October 4, 2018 – The Institute for Clinical and Economic Review (ICER) today released a Final Evidence Report and Report-at-a-Glance on inotersen (Akcea Therapeutics) and patisiran (Onpattro™, Alnylam Pharmaceuticals) for the treatment of hereditary transthyretin amyloidosis (hATTR).

ICER’s report was reviewed at a September 2018 public meeting of the Midwest Comparative Effectiveness Public Advisory Council (Midwest CEPAC), one of ICER’s three independent evidence appraisal committees. During the meeting, the Council found that both inotersen and patisiran provide a substantial net health benefit when compared to best supportive care alone, but evidence is insufficient to distinguish between the two treatments. However, current pricing far exceeds commonly cited thresholds for cost effectiveness.

During the deliberation, Council members weighed the therapies’ other benefits and contextual considerations. The Council unanimously recognized that the novel mechanism of action was an important other benefit for treating individuals with such a high lifetime burden of illness. A majority also recognized that the new treatments may reduce family and caregiver burden and may improve a patient’s ability to return to work. These Council members emphasized that the burden that a hereditary disease places on families cannot be understated, and that these new treatments may also have a positive psychological effect on multiple generations of a family.

“Hereditary amyloidosis is an insidious condition that gravely affects patients and their entire families over generations, and it is exciting to have two new treatment options that, based on short-term evidence, appear to provide important benefits,” said Steven D. Pearson, MD, MSC, President of ICER. “Unfortunately, the pricing vastly exceeds a level commensurate with our best estimates of the treatments’ ability to improve patients’ lives, and is likely to cause financial toxicity for patients and ultimately reduce access. Despite this concern, insurers crafting coverage policy to help maintain affordability of health insurance for all patients now and in the future should consider the treatments’ broader benefits to patients, caregivers, and society.”

The Council voted unanimously that, despite the net health benefit, both inotersen and patisiran represent a low long-term value for money. The votes were influenced heavily by the $450,000 annual list price of patisiran, and the assumption that inotersen would be priced similarly.

Key Policy Recommendations

Following the voting session, a policy roundtable of experts – including patient advocates, physicians, and payers – convened to discuss the implications of the evidence for policy and practice. Key recommendations stemming from the roundtable discussion include:

  • Given that newly approved treatments for hATTR have new mechanisms of action, lack long-term safety and efficacy data, and are very expensive, it is reasonable for insurers and other payers to develop prior authorization criteria to ensure prudent use of these treatments.
  • Manufacturers should bring the price for innovative treatments for hATTR down to a level that aligns fairly with the added benefits for patients.
  • Patient organizations that have a leading role in funding, organizing, and promoting innovative research on new treatments should demand commitments from manufacturers for reasonable value-based pricing of the products patients helped bring to the market.
  • Future research should address the durability of improvements in neurological function, longer-term safety, and cardiac outcomes provided by treatments for hATTR.

ICER’s full set of policy implications, along with more detailed explanations of each, are available in the full report.

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.