By John Miller

ZURICH (Reuters) – Sickle cell disease drugs made by Novartis and Global Blood Therapeutics (GBT) may not be cost effective at current prices, a draft report published on Friday suggested, widening the debate over U.S. healthcare affordability.

Novartis and GBT disputed the preliminary conclusions by the

Boston-based Institute for Clinical and Economic Review (ICER). The institute scrutinised Novartis’s Adakveo and GBT’s Oxbryta, both approved last year, and Emmaus Medical’s Endari, to treat the inherited disease that disproportionately affects African Americans.

The finding that the costs of all three medicines would likely have to be slashed to achieve acceptable cost-effectiveness thresholds comes as Congress and U.S. President Donald Trump weigh action over high healthcare costs.

Insurers and governments use privately funded ICER’s conclusions to help set reimbursement rates.

ICER said it concluded that Adakveo’s annual cost should be $25,410, down from an estimated $88,000 bill, and that Oxbryta must be trimmed to $9,218 from about $84,000, to meet cost-effectiveness thresholds. Endari must be cut to $3,859 from $24,000 to be cost-effective, the group added.

“Treatment costs were the main driver of the cost-effectiveness results,” said ICER, which is taking public comment until Feb. 20 before final publication this year.

Shares in GBT fell more than 5.5% on Friday, while Novartis shares closed 0.3% lower.

ICER seeks to measure how many years of good health – a quality-adjusted life year, or QALY – may be gained with a new treatment, and the incremental cost for that health gain.

At the current estimated costs for Adakveo, Oxbryta and Endari, all the QALYs exceeded $1 million, ICER said, well above the $150,000 the group ordinarily considers an appropriate cost-effectiveness threshold.

Novartis countered that ICER had not appropriately weighed Adakveo’s benefits, including reducing pain crises suffered by sickle cell disease (SCD) patients or potentially helping cut hospital stays.

“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,” Novartis said.


San Francisco-based GBT contended that ICER’s review conflicts with the U.S. Food and Drug Administration’s goal of speeding medicines to patients.

“ICER’s review is flawed, premature and risks adversely impacting access to new, potentially transformative therapies,” GBT said.

U.S.-based Emmaus could not immediately be reached for comment.

ICER said its draft report may change based on public comments, including from drugmakers. It also acknowledged setting a drug’s price may go beyond traditional measures of clinical effectiveness and cost-effectiveness, to include other, non-financial considerations.

Recent advances in treating long-neglected sickle cell disease include the three medicines reviewed by ICER and an experimental gene therapy from Bluebird Bio Inc..

Still, costs are of particular interest. More than half of the roughly 100,000 U.S. sickle cell disease patients are covered by government health insurance.

(Reporting by John Miller; Editing by Susan Fenton)