Alexion's 430,000 euros-a-year drug set for UK state funding

LONDON, Jan 28 (APM) - The National Health Service will shortly fund Alexion's highly expensive Soliris (eculizumab) in the orphan indication of haemolytic uraemic syndrome (aHUS), after NICE on Wednesday issued final guidance recommending its use.
The cost-effectiveness body said in a statement it has has issued final and binding guidance for Soliris in aHUS - the first guidance produced as part of NICE’s highly specialised technologies programme to evaluate treatments for very rare conditions, it said.
Final guidance follows its second draft guidance in November 2014 that state funding should be available for the 340,000 pounds (430,000 euros) per patient per year agent. (APMHE 40606)
NICE’s estimates the drug will cost the NHS up to 58 million pounds (77.6 million euros) in the first year, rising to 82 million pounds (109.7 million euros) after five years.

Step change in treatment of orphan indication

NICE's chief executive Andrew Dillon said: "(NICE's evaluating) 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. It offers people with aHUS the possibility of avoiding end-stage renal failure, dialysis and kidney transplantation, as well as other organ damage.
“The drug is, however, very expensive. The committee felt that the budget impact of eculizumab would be lower if the potential for adjusting the dose of the drug and stopping treatment was explored. This is reflected in the guidance which recommends eculizumab should be funded only if important conditions are met, including the development of rules for starting and stopping treatment for clinical reasons. In the meantime National Health Service England and the company should consider what opportunities might exist to reduce the cost of eculizumab to the NHS.”
It repeated its conditions from the previous November guidance:
:: Co-ordination of use of eculizumab use through an expert centre.
:: Monitoring systems to record the number of people with a diagnosis of aHUS and the number who have eculizumab, and the dose and duration of treatment.
:: A national protocol for starting and stopping eculizumab for clinical reasons.
:: A research programme with robust methods to evaluate when stopping treatment or dose adjustment might occur.



Interviews with KOLs/senior executives amongst the Regulators, Payers, Health, Medical & Pharmaceutical organisations

Events coverage with a unique focus on Market Access & sustainability of healthcare systems

6 European bureaus : Berlin, Brussels, London, Madrid, Milan & Paris

Ask for a Free trial and get access to the latest stories

Our coverage includes:
  • Health Care
  • Market Access
  • HTA – policies & practices
  • European medicine regulations
  • Drug safety issues
  • Pricing & Reimbursement
  • International medicines agencies

If you are a Payer, Pharmaceutical or Consulting professional our premium data will keep you informed on the regulatory, pricing, market access and cost-effectiveness issues that impact all stakeholders.


an initial 10 day temporary access of APM Health Europe.