NICE backs Novartis' Adakveo via special channel despite 'high uncertainty' about cost, long-term efficacy

When England’s drug economics reviewers say they’re not sure about a drug’s cost-effectiveness, that typically results in a rejection of coverage. But that’s not the case for Novartis’ sickle cell disease drug Adakveo.

Tuesday, Adakveo won backing from the National Institute for Health and Care Excellence (NICE) as the first new therapy for sickle cell disease therapy in 20 years.

Clearly, the agency recognizes the unmet need in the blood disorder as it's allowing Novartis a special arrangement for coverage despite “high uncertainty” about Adkveo’s long-term effectiveness and cost-effectiveness. In exchange, the Swiss pharma is offering a confidential discount.

Adakveo will be available through a Managed Access Agreement (MAA) to patients aged 16 or above. NICE expects more than 300 people to get Adakveo via the program, growing to more than 450 people later.

Compared with routine coverage, such a deal allows patients to access treatment while officials collect additional data to assess the long-term benefits. NICE and NHS England have previously offered the pathway to other breakthrough drugs, such as Biogen’s spinal muscular atrophy drug Spinraza, whose MAA status remains in place.

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As for Adakveo, the drug showed in a phase 3 trial dubbed Sustain that it could significantly reduce the annual rate of sickle cell-related pain crisis over placebo. But NICE’s drug appraisal committee pointed to the study’s small sample size and duration of a little over a year, arguing that the long-term effectiveness of Adakveo is unproven.

To resolve the committee’s concerns, Novartis has proposed two sources for additional data. A phase 3 U.K.-specific trial dubbed Stand will study Adakveo, with or without traditional hydroxyurea therapy, in patients aged 12 years and over with a history of vaso-occlusive crisis. The trial is expected to report a primary analysis in 2023 and will provide data for up to three years. NICE has agreed that it will revisit its guidance when the new evidence becomes available.

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In addition to the U.K. trial, Novartis also suggested that the U.K.’s National Haemoglobinopathy Registry could help collect real-world data.

NICE said it considers Adakveo cost-effective under the terms of the MAA. In making the recommendation, the organization also took into account “the high unmet need for treatments for sickle cell disease and NICE’s aim of reducing health inequalities.” The inherited disorder disproportionally affects Black people.

Approved by the FDA in November 2019, Adakveo brought in sales of $42 million in the second quarter of 2021. A recent doctors’ survey by JPMorgan showed U.S. physicians were complaining about Adakveo’s reimbursement status compared with hydroxyurea therapies