Dr. Greenberg’s Synopsis

Providing an update on the status of Asfotase alfa for the treatment of HPP is both exciting and challenging at the same time. To me it is challenging to be the site lead for Canada for Asfotase alfa clinical trials and to remain objective, patient and realistic all at the same time. Every country is unique with respect to its approach to providing access to Asfotase alfa and to other drugs for rare and ultrarare disorders. In Canada there are a series of steps that are followed in the drug approval process .

  • First, Health Canada received and subsequently approved the new drug submission for Asfotase alfa in September 2015.
  • Subsequently the CADTH’s (Canadian Agency for Drugs and Technologies in Health) Canadian Drug Expert Review Committee (CDEC) systematically reviewed the data from 3 open-label pivotal studies, the pharmacoeconomic evaluation provided by Alexion and information submitted by the SBC patient group about the impact of HPP on their lives.
  • The CADTH CDEC committee recommended on March 23 2016 that Asfotase alfa be listed for enzyme replacement therapy in patients with a confirmed diagnosis of pediatric-onset HPP but at a drastically reduced price which was not quantified. CDEC also did not recommend listing Asfotase alfa for adult HPP patients who had pediatric-onset disease and who have never been on enzyme replacement treatment.
  • The next step involves the pCPA (Pan Canadian Pharmaceutical Alliance), a joint pan-Canadian body representing the provinces and territories, which is the process by which the public payers ie our provincial drug benefits branches, determine if they will add Asfotase alfa to their provincial formularies.
  • The pCPA process is in now in progress and it is hoped that a mutually acceptable agreement can be reached between the manufacturer Alexion and pCPA and its participating jurisdictions. In parallel, each participating jurisdiction makes its own final decision on funding the drug through its own public drug plan and entering into an agreement with Alexion.