ICER Analysis of Vertex’s 4 CFTR Modulators Hails Benefit of Trikafta but Suggests 73% Discount

PRICENTRIC BRIEF:

• The Institute for Clinical and Economic Review (ICER) published its assessment of the comparative clinical effectiveness and value of Vertex’s four CFTR modulators for cystic fibrosis (CF): Trikafta (elexacaftor/tezacaftor/ivacaftor), Kalydeco (ivacaftor), Orkambi (lumacaftor/ivacaftor), and Symdeko (tezacaftor/ivacaftor)
• David Rind, MD, ICER’s Chief Medical Officer, explained that findings showed Trikafta is even more “remarkable” than Kalydeco and could alter the course of disease for the majority of patients with CF, transforming their lives and the lives of caregivers, but its health-benefit price benchmark (HBPB) should be between $67,900-$85,500 per year, a 73% reduction on the treatment’s current list price
• Overall, ICER’s Rind said that the prices set by Vertex for these drugs, which amount to millions of dollars over a patient’s lifetime, are “out of proportion for their substantial benefits,” despite their positive impact on patients’ lives

THE DETAILS

BOSTON, Massachusetts, The United States – The Institute for Clinical and Economic Review (ICER) published its assessment of the comparative clinical effectiveness and value of Vertex’s four CFTR modulators for cystic fibrosis (CF): Trikafta (elexacaftor/tezacaftor/ivacaftor), Kalydeco (ivacaftor), Orkambi (lumacaftor/ivacaftor), and Symdeko (tezacaftor/ivacaftor).

David Rind, MD, ICER’s Chief Medical Officer, explained findings that showed while Kalydeco provides “great advance for a small segment of the CF population,” Trikafta is even more “remarkable” and could alter the course of disease for the majority of patients with CF, transforming their lives and the lives of caregivers. “It is critical that all eligible patients be able to access CFTR modulators,” said Rind.

However, the prices set by Vertex for these drugs can amount to millions of dollars over a patient’s lifetime and are therefore “out of proportion for their substantial benefits,” commented Rind.

“Patients who receive the treatments will benefit, but unaligned prices will cause significant negative health consequences for many unseen individuals – those throughout society who will experience financial toxicity and may have to delay care, forego care, or even abandon insurance as their out of pocket costs and premiums are driven upward,” Rind added.

Trikafta scored the highest rating from ICER for the indicated populations where clinical data exist, the evidence provides high certainty that Trikafta provides a substantial net health benefit over standard care and over Symdeko. And while Trikafta has not yet been studied in patients who are heterozygous for the F508del mutation and a residual function mutation, ICER determined that using Trikafta to treat that sub-population is likely to be at least as good as treating with Symdeko, possibly better even.

ICER’s recommended health-benefit price benchmark (HBPB) for Trikafta is $67,900-$85,500 per year, which would require at least a 73% discount on the treatment’s current list price.

For the other modulators from Vertex, ICER’s evaluation of new evidence since its 2018 assessment confirms previous evidence ratings” Kalydeco provides a substantial net health benefit, Orkambi provides a small net health benefit, and Symdeko provides at least a small net health benefit with the potential for a substantial benefit.

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