http://www.raredr.com/news/canada-says-no-to-alexions-ahus-orphan-drug
Canada Says No To Alexion's aHUS Orphan Drug

James Radke, PhD

The Pan Canadian Pharmaceutical Alliance has called off the negotiations with Alexion Pharmaceuticals for the orphan drug Soliris (eculizumab) to treat Canadians with atypical hemolytic uremic syndrome (aHUS).
 
Soliris is often touted as the most expensive drug in the world with a price tag of $700,000 per year in some countries.
 
In 2011, the drug was approved in the United States for aHUS. The drug is also approved for paroxysmal nocturnal hemoglobinuria. Surprisingly, the drug is allowed in Canada to treat paroxysmal nocturnal hemoglobinuria for the price of $500,000. Just not for aHUS.

Its Not About the Price Tag

Details of the negotiation are unknown, but the problem appears to be the efficacy data and not the high price tag. According to a Canadian newspaper, Suzanne McGurn, executive officer of the Alliance said:

“(We) made every effort to expedite a decision on Soliris and negotiated for six months in good faith with Alexion to try and reach an agreement on clinical criteria.”

McGurn added:

“Patients and physicians want to try anything that may work to help them cope with their disease. We understand that and we can empathize with patients. However, public drug plans cannot provide coverage for all individuals who may wish to try Soliris or any drug – regardless of its cost - where the clinical evidence does not demonstrate improved health outcomes.”

The Pan Canadian Pharmaceutical Alliance does not decide if a drug will be allowed to be sold in Canada but they are involved in negotiating with drug companies on behalf of Canadian provinces. The Alliance relies on Health Canada’s Common Drug Review for its negotiations and in 2013, Health Canada recommended that drug not be listed.

According to Health Canada’s Common Drug Review, the “two uncontrolled prospective studies had several important limitations, including a lack of clear diagnostic criteria for atypical hemolytic uremic syndrome (aHUS), the absence of a comparator group to examine outcome differences, short duration of follow-up, and lack of data regarding clinically important outcomes for patients with aHUS. Therefore, the clinical benefit of eculizumab could not be adequately established.”
 
In contrast, the FDA approved the drug in 2011 and last year, England’s NICE also recommended that the drug be made available to all patients with aHUS.

Alexion Responds - "It is a tragedy that Canadian citizens with aHUS must suffer and die without the life-transforming benefits of Soliris."

In response to the end of negotiations, Alexion wrote:

"Alexion is disappointed that the Pan Canadian Pharmaceutical Alliance (pCPA) has unilaterally decided to end negotiations regarding funding of Soliris for the very few children and adults in Canada with aHUS. Since August 2015, Alexion worked in good faith with the pCPA to develop funding criteria, including a proposal that was submitted on January 5, 2016, and received no feedback until the pCPA decision was made public on February 16. Governments and private payers in nearly 50 countries around the world recognize the lifeā€transforming impact of Soliris on children and adults with aHUS, providing access and funding. We believe that Canadian children and adults with aHUS deserve the same level of care.  Without Soliris, more than half of the children and adults with aHUS will die, require dialysis, or have permanent renal damage within 12 months of diagnosis. With Soliris, 80% of children and adults on dialysis were able to eliminate dialysis. NICE in the UK established funding for Soliris for their citizens in England with aHUS and concluded that Soliris provides a substantial gain in quality adjusted life of a magnitude rarely seen for any new therapy. It is a tragedy that Canadian citizens with aHUS must suffer and die without the life-transforming benefits of Soliris.

 Alexion is one of the few companies in the world to take on the risks and challenges of drug development for severe diseases that affect a very small number of patients. We hold ourselves to the highest standards of medical innovation, and our three products, including Soliris, are transforming lives, as recognized by patients and policymakers globally. The responsibility now falls to the Canadian governments and payers to fulfill their obligation to their citizens by ensuring access to these life-transforming therapies for the few patients who need them."

About aHUS and Soliris

aHUS is a chronic, ultra-rare, and life-threatening disease that can lead to progressive renal  problems. A common problem in these patients is formation of complement-mediated thrombotic microangiopathy (TMA) – or blood clots in small blood vessels throughout the body. According to Alexion, 65% of all patients with aHUS die, require kidney dialysis, or have permanent kidney damage within the first year after diagnosis despite plasma exchange or plasma infusion (PE/PI). 

Soliris is a recombinant humanized monoclonal IgG antibody that targets the terminal portion of the complement cascade and has been found to inhibit TMA in patients with aHUS.
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