Hello, everyone, and welcome to this week’s edition of Excelsior Solutions’ ICER Update.
Here is this week’s ICER recap:
ICER unexpectedly released an Evidence Report assessing the comparative clinical effectiveness and value of elexacaftor/tezacaftor/ivacaftor (Trikafta®, Vertex Pharmaceuticals) for the treatment of cystic fibrosis (CF). ICER also examined new data that have become available since its May 2018 review of three related CF treatments: ivacaftor (Kalydeco®, Vertex), lumacaftor/ivacaftor (Orkambi®, Vertex), and tezacaftor/ivacaftor (Symdeko®, Vertex). All four therapies are CFTR modulators.
“CF is a devastating disease that affects the lungs and other organ systems, typically requires substantial medical care, and robs patients and families of hours each day, before taking away full years of life,” said David Rind, MD, ICER’s Chief Medical Officer. “Kalydeco was a great advance for a small segment of the CF population, and Trikafta appears to be an even more remarkable advance for a much larger segment of the population. Trikafta is likely to 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.”
“Despite being transformative therapies, the prices set by the manufacturer –costing many millions of dollars over the lifetime of an average patient — are out of proportion to their substantial benefits. When a manufacturer has a monopoly on treatments and is aware that insurers will be unable to refuse coverage, the lack of usual counterbalancing forces can lead to excessive prices. 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.”
Trikafta earned ICER’s highest “A” evidence rating. 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. In addition, although 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, and possibly better.
For the other three related treatments — Symdeko, Orkambi, and Kalydeco — ICER’s evaluation of new evidence since their 2018 assessment confirms their previous evidence ratings. For their respective indicated populations, and compared to best supportive care, the evidence provides high certainty that 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.
EXCELSIOR ACTION POINT: This group of CF medications, with the emphatic addition of Trikafta, illustrates the conundrum that occurs when even drugs having the highest degree of clinical evidence supporting their effectiveness are launched with manufacturer-pegged pricing that dampens enthusiasm surrounding their use and benefit. Perhaps the ICER spotlight will bring some necessary light and heat to cause some reduction in current prices, especially for Trikafta whose future seems brightest for the widest population of CF patients.
As always, if you would like to discuss the potential impact of these, or any prior, ICER reports, or any other aspects of your Pharmacy Benefit Plan, simply reach out to your Excelsior Solutions account team and we will be happy to quickly set up a call.
Until next time!
Bob Kordella, RPh, MBA