CF Foundation’s Statement on ICER’s Revised Evidence Report on Treatments for Cystic Fibrosis

Cost effectiveness analysis must be used carefully and as part of a comprehensive evaluation of the value a treatment, such as highly effective modulators, provide to people living with cystic fibrosis.

April 28, 2020 | 2 min read

Highly effective CFTR modulators represent the most significant therapeutic advancements in the history of cystic fibrosis. These medicines treat the underlying cause of this progressive, sometimes fatal disease and are known to have a transformative effect on the health and wellbeing of individuals eligible to take them. People with CF who are eligible for modulator treatment must have access to the most effective therapy. Patients should not bear undue cost and coverage burdens to access life-changing drugs.

We welcome ICER's efforts to add more data and transparency to the important discussions on the value and cost of drugs and services. Like many innovative medicines for severe, chronic, and rare disease, modulators are expensive. The Cystic Fibrosis Foundation hears frequently from people living with cystic fibrosis who are concerned about their ability to afford these therapies as well as insurers with concerns about the budget impact of care for cystic fibrosis. There must be a balance between rewarding groundbreaking innovation and preserving access to lifesaving therapies.

We are pleased that ICER acknowledges the transformative impact of highly effective modulators, and their essential role in the CF treatment model, in its response to comments from the CF community. However, we continue to have reservations about ICER's model and do not believe the evidence report reflects the full value these therapies will provide over time. In particular, ICER's focus on lung function may undervalue and under-predict the long-term, systemic benefits that people with CF derive from CFTR modulators.

We urge payers and policymakers to bear in mind that cost-effectiveness analyses must be used carefully and as part of a comprehensive evaluation of the value a treatment provides. Given the significant limitations of the ICER model it should not be used as the sole source of decision-making when determining coverage.

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