NICE result for Soliris, but no justification for high manufacturing costs

By Dan Stanton

- Last updated on GMT

Related tags Monoclonal antibodies

NICE recommends Alexion's orphan mAb drug Soliris
The monoclonal antibody drug Soliris has been recommended by NICE despite Alexion failing to justify the manufacturing costs contributing to the £3,150 ($5,140) price per vial. 

In March, NICE – the National Institute for Health and Care Excellence which governs drugs used by the National Health Service (NHS) in England and Wales – asked Alexion to justify the cost of its Haemolytic Uraemic Syndrome (aHUS) treatment.

The key question was why Soliris is up to 30 times more expensive than other monoclonal antibodies​, with NICE asking specifically if the £340,200 price tag was a result of any manufacturing processes unique to the drug.

Alexion’s response, which was included in the NICE recommendation issued yesterday, is that the price is justified because there are so few aHUS patients – approximately 200 in the UK – and that there is “a higher level of financial risk involved in entering the market for very rare diseases.”​ 

The firm also told NICE, with further the price tag is part of its effort to claw back costs accrued during trial recruitment, investment in clinical and patient education, and resources for new indications.

“The company also stated that there is a higher risk of failure associated with discovering new treatments for very rare diseases,” the guidance​ said.

No justification

The recommendation does not include full details of the justification as a result of Alexion’s request that they not be disclosed, which NICE told us at the time​ was unusual for a technology appraisal.

What NICE was able to disclose is that the details provided of Alexion’s manufacturing processes were not exclusive to the production of eculizumab and would apply to the production of all mAbs.

NICE spokesman Phil Ranson told “Despite the Committee concluding that it had not been presented with a justification of why the overall cost of eculizumab was materially higher than the overall cost of other highly specialised technologies, it did consider that investigation around dose adjustment or stopping treatment could potentially improve the value for money offered by eculizumab for aHUS.”

Soliris is made at Alexion's Smithfiled, Rhode Island facility - recently hit with a 483 after a US FDA reinspection​ - and two facilities made by contract manufacturer Lonza. Alexion spokesman Irving Adler did not respond to requests for more information when contacted by this publication.

Highly Specialised Technologies Programme

The recommendation of Soliris falls under NICE’s Highly Specialised Technologies programme, set up in 2013 as a result of the reforms outlined in the Health and Social Care Act 2012.

The programme has “a distinct methodology specifically designed to evaluate high cost, low volume drugs for very rare conditions,” ​Ranson told us, “and there is no cross-over into our methodology for assessing mainstream technologies.”

We asked him whether the Soliris result, therefore, would be a springboard for positive recommendations of other orphan mAb drugs. “No,”​ he said, “not unless such technologies are also able to demonstrate clinical effectiveness and value-for-money.”

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