The Pan Canadian Pharmaceutical Alliance (pCPA) has ended negotiations with Alexion.

The quest to get public funding of the drug eculizumab (Soliris) for aHUS patients in Canada (the only pharmaceutical treatment for them) has been ongoing since July 2013. After two years of advocating, the Ontario Ministry of Health and Long Term Care decided in February 2015 to provide interim funding of this drug for aHUS patients who meet defined criteria. In August of 2015, aHUS Canada was pleased to hear that the pCPA (pan-Canadian Pharmaceutical Alliance) finally began negotiations with eculizumab’s manufacturer, Alexion, and were hopeful that permanent funding criteria would be created for all of Canada.

After months of requesting,  aHUS Canada was granted a meeting on February 8, 2016, with the Executive Officer (E.O.) of the Ontario Public Drug Plans who is the lead negotiator for this indication on behalf of the pCPA.  We voiced our concerns from the patient perspective .  On Tuesday February 16 we had another telephone conversation with the E.O. and were told that the pCPA had ended negotiations with Alexion.  Below is a link to the statement from the E.O. on behalf of the pCPA.

Although grateful for Ontario’s decision to continue funding the drug for aHUS patients, we are still waiting to receive the details of the updated criteria to see which patients will actually qualify.  We are extremely concerned and disappointed that the pCPA ended negotiations.  This means that, besides Ontario and Quebec, the rest of Canada still does not have public access to this life changing and life saving drug.  This is unacceptable since an effective treatment exists and the patients remain in need.  According to the E.O., “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.”

To state that the clinical evidence for Soliris does not demonstrate improved health outcomes is denying science.  The clinical evidence has been proven in many leading scientific journals.  As stated in the New England Journal of Medicine published in June 2013, “Atypical Hemolytic Uremic Syndrome is a genetic, life-threatening, chronic disease of complement-mediated thrombotic microangiopathy. Affected patients have a lifelong risk of systemic clinical complications of thrombotic microangiopathy, including damage to multiple organ systems (e.g., the central nervous system, kidneys,heart, and gastrointestinal tract). Plasma exchange or infusion may transiently maintain normal levels of hematologic measures but does not treat the underlying systemic disease. End-stage renal disease (ESRD) or death occurs in approximately 33 to 40% of patients during the first clinical manifestation of atypical hemolytic–uremic syndrome. Within 1 year after a diagnosis of this syndrome, up to 65% of patients treated with plasma exchange or infusion sustain permanent renal damage, have progression to ESRD, or die. The results of eculizumab therapy  represent a substantial advancement in the treatment of patients who have this severe and life-threatening systemic disease.” The New England Journal of Medicine article proved that aHUS patients treated with eculizumab were able to discontinue plasma infusion/exchange and dialysis therapies, and saw improved kidney function, reduced blood vessel damage and decreased risk of blood clots.

 The effectiveness of eculizumab has revolutionized the management and outcomes of aHUS  and has been described as a break through treatment for aHUS where previously there was no approved treatment and has opened up the possibility of renal transplantation in patients with aHUS. It is about time that Canada’s Health Ministries acknowledge the proven scientific evidence and find a way to provide this treatment before any more patients fall victim to this severe and life threatening chronic disease.

aHUS Canada will continue to advocate and educate in order that aHUS patients receive the treatment they need.