Institute for Clinical and Economic Review’s Final Report on CFTR Modulators for Cystic Fibrosis Calls for Manufacturer Restraint and Increased Transparency in Pricing Decisions

– Pricing overreach harms people with CF and threatens the health care system’s ability to support future innovation –

BOSTON, June 7, 2018 – The Institute for Clinical and Economic Review (ICER) today released a Final Evidence Report and Report-at-a-Glance on cystic fibrosis transmembrane conductance regulator (CFTR) modulators. The report includes combination therapies tezacaftor/ivacaftor (Symdeko, Vertex Pharmaceuticals) and lumacaftor/ivacaftor (Orkambi®, Vertex Pharmaceuticals), and monotherapy ivacaftor (Kalydeco®, Vertex Pharmaceuticals), for the treatment of cystic fibrosis (CF).

ICER’s analysis was reviewed at a public meeting of the Midwest Comparative Effectiveness Public Advisory Council (Midwest CEPAC), where a majority of the Council voted that, in their specified indications, Kalydeco, Orkambi, and Symdeko in combination with best supportive care all offer a net health benefit compared to best supportive care alone.

Many members of the Midwest CEPAC also noted that the therapies offer other benefits beyond those examined in clinical trials, such as improved ability for patients to return to work, school, or other activities, and that the new treatments are the first in many years to offer significant clinical improvement. Council members noted that contextual considerations, such as a high severity of disease with a high lifetime burden of illness, must also be considered in determining the long-term value for money of the therapies.

Despite these positive findings, however, a majority of the Council ultimately voted that the therapies represent a low long-term value for money, due in large part to the high price of the drugs. ICER’s analysis suggested that discounts of up to 77% would be necessary to bring the prices into alignment with the drugs’ clinical value to people with CF and their families.

“Discussions at our meeting highlighted that, while these therapies offer important benefits for those living with CF, the $300,000 annual price is far too high to pay year after year, harming patients and families today while threatening the health care system’s ability to maintain access to important future clinical advances,” noted Dan Ollendorf, PhD, ICER’s Chief Scientific Officer. “As the sole manufacturer of CFTR modulators, Vertex should be rewarded for its innovation; however, the company should also be willing to make their pricing decisions fully transparent, and fully engage in independent, multi-stakeholder assessments of the value of these medications.”

Kalydeco, Orkambi and Symdeko were developed with significant financial and scientific support from the Cystic Fibrosis Foundation. During the public meeting, however, a representative from the Foundation noted that the organization was not included in the manufacturer’s discussions around how the products would be priced.

Key Policy Implications

Following the voting session during the Midwest CEPAC meeting, a policy roundtable of experts including CF families, patient advocates, clinicians, and public and private payers, convened to discuss the implications of the evidence for policy and practice. Key recommendations stemming from the roundtable discussion include:

  • The manufacturer bears a significant social responsibility to exercise pricing restraint during its period of monopoly pricing power. Vertex should abandon vague claims that prices are justified by the need to invest in future research, and instead join the growing number of biotech innovators that provide a transparent, explicit justification for their prices based on the ability of new treatments to improve the length and quality of patients’ lives.
  • Public and private payers should continue to affirm their commitment to provide access to important clinical advances for CF and should remove superfluous requirements for coverage approval and continuation.
  • Patient organizations that have a leading role in funding, organizing, promoting, and otherwise fostering innovative research into new treatments should demand commitments from pharmaceutical manufacturers for sustainable pricing of those products.
  • Professional societies should fully exercise their responsibility by bearing witness to the impact on their patients of failed pricing and insurance policies and by demanding to be part of the public process that should guide pricing to balance the needs for affordability and for investments in future innovation.

A full list of policy implications and detailed discussions of each are available in the final 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.