RARE Daily

ICER Praises CF Drug’s Benefits, but Says It’s Overpriced

April 29, 2020

Rare Daily Staff

Drug price watchdog the Institute for Clinical and Economic Review said Vertex Pharmaceuticals’ cystic fibrosis combination therapy Trikafta provides substantial benefits for patients, family members, and society, but said that its price is out of proportion to its benefits.

In a new Evidence Report, ICER assesses the comparative clinical effectiveness and value of Trikafta (elexacaftor/tezacaftor/ivacaftor), as well as three related CF treatments from Vertex: Kalydeco (ivacaftor), Orkambi (lumacaftor/ivacaftor), and Symdeko (tezacaftor/ivacaftor). All four therapies are 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,” said David Rind, ICER’s chief medical officer. “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.”

Trikafta earned ICER’s highest evidence rating of “A.” It said for the indicated populations where clinical data exist, the evidence provides high certainty that Trikafta provides a substantial net health benefit over both standard care and 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 evaluated new evidence that has become available since its 2018 evaluation on these drugs. It said that the new evidence confirms its previous 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.

ICER’s recommended health-benefit price benchmark (HBPB) for Trikafta is $67,900-$85,500 per year, which would require at least a 73 percent discount off the treatment’s current list price. The HBPB is a price range suggesting the highest U.S. price a manufacturer should charge for a treatment, based on the amount of improvement in overall health patients receive from that treatment, when a higher price would cause disproportionately greater losses in health among other patients in the health system due to rising overall costs of health care and health insurance. It is the top price range at which a health system can reward innovation and better health for patients without doing more harm than good.

In response to its review, ICER said it has received nine public comments from patient advocacy organizations and 45 comments from individuals, caregivers, or family members. One comment noted that Trikafta provided such a high magnitude of treatment benefit that CF clinical experts consider them “highly effective modulator therapies.” ICER responded that it agreed that these drugs represent a “huge advance,” but “even the large magnitude of the benefit fails to justify the price.”

ICER said in addition to the demonstrated clinical effectiveness of these drugs, policymakers should be aware of the other potential benefits. Throughout its eight-month review, it said patients shared their personal stories about how CF affects all aspects of their lives. Patients shared how much Trikafta, Symdeko, Orkambi, and Kalydeco have helped them be more active, spend more time with family and friends, and plan for the future in a way that would have been unthinkable just a few years ago.

Due to public health concerns regarding meetings and travel during the ongoing COVID-19 situation, ICER said it was postponing its CF public meeting indefinitely.

Photo: David Rind, ICER’s chief medical officer.

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