Vertex Pharmaceuticals Inc. should significantly slash prices for three cystic fibrosis drugs or risk harm to patients and future innovation, the U.S. cost advisory group the Institute for Clinical and Economic Review said in a final report.
Vertex's Symdeko, Orkambi and Kalydeco would need to be discounted up to 77% to be cost-effective for their clinical value, ICER concluded. The report comes months after a draft document from the institute recommending that prices be halved, even when the highest cost-effective range is assumed.
Kalydeco has a wholesale price of $426.72 per dose and patients generally take two doses a day — racking up an annual cost of nearly $310,000.
Symdeko has a list price of $400.08 and an estimated annual cost of more than $282,000, while Orkambi lists for $186.78 and approaches $265,000 a year.
ICER has no jurisdiction over pricing, yet its analysis has increasingly attracted notice from drugmakers and investors. Sanofi and Regeneron Pharmaceuticals Inc. recently heavily discounted their cholesterol medicine following consultation with the institute, while Amgen Inc. and Novartis AG launched a new migraine therapy at a lower-than-expected price of $6,900, months after an ICER report on the new class of migraine treatments used $8,500 as a placeholder and deemed it cost-effective.
Benefits vs costs
All three of Vertex's therapies provide patients some benefit compared to supportive care alone and give patients a range of less quantifiable benefits, such as the ability to go to school or work, a regional ICER panel, the Midwest Comparative Effectiveness Public Advisory Council, concluded in a public meeting.
However, these benefits do not outweigh the drugs' costs, ICER said, adding in a press release that pricing overreach could harm people with the chronic disease and curb the healthcare system's ability to support future medicines.
An expert roundtable at the meeting — including doctors, patients, advocates and payers — concluded that "Vertex should abandon vague claims that prices are justified by the need to invest in future research, and instead join the growing number of biotech innovators that provide a transparent, explicit justification for their prices based on the ability of new treatments to improve the length and quality of patients' lives."
Sole manufacturer
Also at the event, a representative from the Cystic Fibrosis Foundation said that the three therapies were developed with significant financial and scientific support from the group, yet it was not included in pricing discussions.
"While these therapies offer important benefits for those living with CF, the $300,000 annual price is far too high to pay year after year, harming patients and families today while threatening the healthcare system's ability to maintain access to important future clinical advances," Dan Ollendorf, ICER chief scientific officer, said in a statement.
Vertex is the sole manufacturer of this class of therapies, known as CFTR modulators, Ollendorf noted. Because of that, it should be willing to make its pricing decisions transparent, he said.
The expert roundtable also concluded that payers should cut down on coverage requirements for important cystic fibrosis therapies; that patient organizations should continue supporting funding, promoting and organizing research efforts; and that professional societies "should fully exercise their responsibility by bearing witness to the impact on their patients of failed pricing and insurance policies."
The company has also been under pressure from the state of New York to lower Orkambi's price after the state passed a drug pricing law last year. New York's Drug Utilization Review Board recommended in late April that the Medicaid program pay about a third of the drug's current unit price.
