Vertex's cystic fibrosis drugs work, but they're too expensive, ICER finds
Earlier this month, Reshma Kewalramani took over the reins as Vertex Pharmaceuticals’ CEO — she will now be tasked with grappling with criticism from cost-effectiveness watchdog ICER, which on Monday issued a report suggesting that despite the health gains offered by its suite of cystic fibrosis drugs, their prices are far too high to be sustainable for patients and health care systems.
“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 in a statement.
The cystic fibrosis drugs made by Vertex are the first treatments that address the underlying genetic causes of CF, which is characterized by thick sticky mucus in the lungs, digestive system and other organs that reduces life expectancy. Vertex’s medicines target the cystic fibrosis transmembrane conductance regulator (CFTR) gene and are engineered to correct the malfunctioning protein it makes.
A little over 300 different mutations are known to cause CF, and patients typically carry pathogenic mutations in both copies of the CFTR gene — the most common such mutation is F508del. The company’s Orkambi and Symkevi focus on the more common F508del mutation. Vertex’s first-ever approved CF drug is Kalydeco. Its triplet regimen, Trikafta, which is designed to treat 90% of all CF patients, was approved by the FDA a spectacular five months ahead of its expected decision date in October.
“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,” said Rind.
There was no doubt that all three drugs, most notably Trikafta, offer a clinical benefit over standard supportive CF care — ICER’s analysis suggested that discounts of up to 77% would be imperative to align prices with the drugs’ clinical value.
The institute worked up a health benefit price benchmark (HBPB) — a price range indicating the highest price a manufacturer should charge for a treatment, based on the amount of improvement in overall health patients achieve on that therapy. Each of the four therapies, according to this analysis, requires a substantial discount, without which Vertex risks causing “disproportionately greater losses in health among other patients in the health system due to rising overall costs of health care and health insurance.”
Although these drugs were developed with significant financial and scientific support from the Cystic Fibrosis Foundation, a representative from the group suggested that it was not included in Vertex’s discussions around how the products would be priced, ICER said.
Akin to NICE in the UK, ICER is an independent body that analyzes the cost-effectiveness of drugs and other medical services in the United States. Unlike NICE, though, ICER is not government-affiliated, but its determinations are increasingly gaining traction with payers and policymakers. Manufacturers frequently attack the methodologies the institute employs in its analyses.
Social image: Reshma Kewalramani, Vertex CEO via YouTube