On Aug. 27, the Institute for Clinical and Economic Review (ICER) hosted a virtual public meeting as part of their assessment of the clinical and cost effectiveness of CFTR modulator therapies, including elexacaftor/tezacaftor/ivacaftor (Trikafta®).
During the meeting, ICER reported its findings and provided a forum for patients and other stakeholders to share their experience with CFTR modulators. During the program's public testimony portion, Dr. JP Clancy, the Foundation's vice president of clinical research, provided his perspective on the value of these therapies.
“CFTR modulator therapies represent the most significant therapeutic advancement in the history of cystic fibrosis,” said Dr. Clancy. “Short of a cure for CF, modulators have the potential to dramatically alter the course of this chronic, life-shortening disease, particularly for those who start treatment at a young age.”
Dr. Clancy detailed the dramatic benefits already seen by people with CF taking CFTR modulators across key clinical outcome measures. However, he noted that a review of these modulators, particularly Trikafta less than a year after its approval from the U.S. Food and Drug Administration, does not allow time to collect data on the therapy's long-term implications. Without this data, he argued, any assessment would undervalue these treatments.
Mary Dwight, senior vice president and chief policy and advocacy officer, represented the CF Foundation during the meeting's policy roundtable. During the discussion, Dwight reiterated the Foundation's position on ICER's review, acknowledging the importance of these discussion but the serious reservations maintained by the Foundation about the model used to generate the findings.
“CFTR modulators have the potential to change the face of this disease and cost shouldn't get in the way of a person's ability to access them,” said Dwight. “Any analysis about cost should be centered around CF patients, reflecting the reality of their experiences, the complexity of the disease, and the clinical significance of these therapies.”
Throughout the nearly year-long assessment of these therapies, the Foundation has provided feedback to ICER in an effort to help them understand cystic fibrosis and the complexities of the disease, while also helping to ensure the assessment reflects the experiences and insights of people with CF, their loved ones, and their clinicians.
ICER's revised evidence report, published in April, concluded that while Trikafta is clinically effective, its price would need to be cut by 73 percent to be cost effective. Policymakers and payers may reference the report to understand the impact and significance of CFTR modulators. ICER's report is not intended to limit the coverage of treatments for people with CF.
ICER will release its final report in September 2020.