NICE slaps unusual rules on Alexion's pricey Soliris after atypical cost review

Despite its well-publicized rejections of several high-priced cancer drugs, the U.K.'s National Institute for Health and Care Excellence (NICE) has just recommended what's widely considered to be the most expensive drug in the world--Alexion's ($ALXN) Soliris. The drug, which NICE sanctioned to treat the rare blood disease atypical hemolytic uremic syndrome, is expected to cost £340,200 per year ($569,000) in the U.K.

NICE did accompany its recommendation of Soliris (eculizumab) with a warning for Alexion, however. The agency's guidance says the drug should only be funded if "important conditions are met," according to a press release from NICE. Those conditions include coordinating Soliris prescriptions through an "expert center," which will monitor diagnoses of the disease, as well as the dosages Soliris patients receive. The center will also collect data "to evaluate when stopping treatment or adjusting the dose of the drug might occur," NICE CEO Sir Andrew Dillon said in the release.

Atypical hemolytic uremic syndrome (aHUS) affects about 200 people in England, NICE estimates. About 70% of patients develop end-stage kidney failure, which can be life-threatening. NICE determined that the drug is worth funding because it can prevent the disease from damaging the kidneys and other organs, making it "a significant breakthrough," Dillon said in the statement.

Still, NICE hasn't exactly been easy on Alexion. In March, the agency demanded the company provide information on whether the drug's total cost was reasonable when compared to the R&D and manufacturing expenses associated with it. It was a major departure from NICE's usual approach, which generally focuses on weighing such factors as quality-of-life years against a drug's price.

NICE CEO Sir Andrew Dillon

NICE's calculations have worked against several drugmakers of late, particularly in the oncology field. Roche's ($RHHBY) breast cancer treatment Kadcyla, and Johnson & Johnson's ($JNJ) prostate cancer pill Zytiga were recently rejected, causing a worldwide outcry. In August, Dillon responded by telling the press that the U.K.'s National Health Service simply can't afford every expensive drug that comes down the pike, and that he expects pharma companies to respond with more reasonable pricing decisions.

The agency's guidance on Soliris seems to offer a cost-saving strategy for the U.K.'s health system without forcing Alexion to reduce the price. In short, if the Soliris expert center's data shows that dosing can be lowered or even interrupted without harming patients, NICE could develop treatment protocols that specify when doctors should stop treatment or reduce doses, according to NICE's statement.

In spite of overseas cost pressures, analysts expect Soliris sales to skyrocket from $1.55 billion last year to $3.4 billion by 2018.

Alexion plans to provide comments to NICE about its plans for the Soliris expert center, according to a statement from the company. But it's pretty clear Alexion would prefer if NICE didn't meddle in treatment decisions.

"We believe that it is important that NICE work within its remit and that decisions regarding continuation of eculizumab should be made by the treating physician based on best clinical judgment," the company says in its statement.

- here's the NICE statement
- read Alexion's response here
- get more at Pharmaphorum

Special Report: Top 20 orphan drugs by 2018 - Soliris