NICE doesn't get the Soliris cost info it wants--but backs the drug anyway

Back in March, the U.K.'s cost watchdog went where it hasn't yet gone before, asking Alexion ($ALXN), maker of pricey orphan drug Soliris, to justify the drug's high sticker with information on the company's R&D costs. Now, it hasn't yet gotten the response it wants, it says, but it's backing Soliris regardless--once a few conditions are met.

Late last week, the National Institute for Health and Care Excellence (NICE) issued final draft guidance recommending the NHS fund Soliris (eculizumab) for treating atypical hemolytic uremic syndrome (aHUS)--despite the fact that Alexion hadn't provided sufficient reasoning for Soliris' cost per patient, BioCentury reports.

"The company's justification of costs were not exclusive" to Soliris and "would apply to all highly specialized technologies for very rare diseases," it said in the evaluation, as quoted by the publication.

But NICE's nod came with a caveat. The recommendation depends on a few arrangements, announced in September, that must be in place for the NHS to cover the drug, like a national protocol for starting and stopping Soliris for clinical reasons, a research program that evaluates when to stop treatment or adjust dosage, monitoring systems to record the number of aHUS patients, and coordination of Soliris through an expert center.

NICE CEO Sir Andrew Dillon

And in the meantime, NHS and Alexion "should consider what opportunities might exist to reduce the cost of eculizumab to the NHS," NICE chief Sir Andrew Dillon said in a statement.

The green light follows Dillon's March request for "clarification from the company on aspects of the manufacturing, research and development costs"--a first for the cost-effectiveness gatekeeper. While NICE has been known to ask for clinical data, price modeling and other info to help it decide whether a drug is worth its price tag, the body has paid special attention to Soliris. Aside from topping the list of the world's most expensive treatments, the med is also the first to be reviewed by NICE under a new process designed for drugs treating "ultraorphan" diseases.

But as Dillon noted, the very effective Soliris offers aHUS patients the chance to avoid late-stage kidney failure, dialysis and transplants, as well as other organ damage. "The committee accepted that eculizumab is a step change in the management of aHUS and can be considered a significant innovation for a disease with a high unmet clinical need," he said.

- read NICE's statement
- get more from BioCentury

Special Reports: Top 20 orphan drugs by 2018 - Soliris | The 25 most influential people in biopharma today - Sir Andrew Dillon

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