New sickle cell drugs from Novartis, GBT need big discounts: ICER draft

Drug prices
A draft report concluded new sickle cell disease drugs from Novartis and Global Blood Therapeutics are too expensive. (Getty/Tero Vesalainen)

When Global Blood Therapeutics won approval for its new sickle cell disease drug Oxbryta last year, execs predicted a “paradigm shift” in the way patients are treated. Now, the company is running into a common hurdle in today's U.S. launch paradigm—pushback from cost watchdog ICER.

In a draft report, ICER concluded that sickle cell disease drugs from GBT, Novartis and Emmaus Medical are too expensive to meet traditional cost-effectiveness measures. To fall within one measure of cost-effectiveness—below $150,000 per quality-adjusted life year—the companies would have to dramatically cut their prices, ICER said.

For GBT’s Oxbryta, the cut would have to be almost 90%, ICER says. A cost of $9,218 per year would be more appropriate, the watchdog says—down from an estimated list price of $84,000 per year. And for Novartis’ Adakveo, ICER’s calculations suggested an annual cost of $25,410, less than one-third of its existing cost of $88,000 per year, as estimated by ICER. 

ICER, or the Institute for Clinical and Economic Review, said the costs were the “main driver” for the cost-effectiveness results in the draft report. The group said the drugs can provide relief for patients, but that patients "will continue to suffer from other acute and chronic conditions that will have a significant impact on their quality of life.” 

Novartis disputed the findings, which could be changed after input from the drugmakers and others. 

A spokesman said Adakveo is a “significant advance in sickle cell disease care, which has seen little innovation in decades.” The med reduced vaso-occlusive crises compared with placebo and patients on the med spent fewer days in hospitals than those taking placebo, according to trial data.

Vaso-occlusive crises, or VOCs, are the “hallmark” of sickle cell disease, the spokesman said. They’re sudden, painful episodes that can lead to irreversible organ damage; patients suffering from VOCs often end up in the hospital.  

“We look forward to gaining a better understanding of the approach ICER used to make its assessment and to helping them better understand the value of Adakveo as they prepare the final assessment,” Novartis’ spokesman added. 

RELATED: Novartis eyes $1B sales with FDA nod for targeted sickle cell disease drug Adakveo 

A GBT representative said ICER’s review is “flawed," "premature" and that it "risks adversely impacting access to new, potentially transformative therapies." The company plans to provide feedback and hopes ICER will consider its input for the final report, he added.

There are about 100,000 sickle cell disease patients in the U.S. and millions more throughout the world, according to GBT. The disease primarily affects people whose ancestors are from sub-Saharan Africa, but it also hits several other groups. In the U.S., the disease mostly affects African Americans.

The disease is an inherited blood condition that eventually affects “virtually every organ” in patients’ bodies, a GBT exec told FiercePharma.

RELATED: Global Blood Therapeutics scores blockbuster FDA nod for Oxbryta, forecasts 'paradigm shift' in sickle cell disease 

ICER has been conducting drug pricing reviews for years and has often run into pushback from the pharma industry when it concludes new drugs are too pricey. But it has also endorsed new drug prices in some cases, including the multimillion-dollar sticker on Novartis' gene therapy Zolgensma.

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