Strensiq: In sight but out of reach for Canadians
Most Canadians are proud of their universal healthcare system which certain developed countries, including the U.S., still do not have. Unfortunately, this universal healthcare is not as universal as it may seem; it does not have any provisions in place to help Canadians that need it most: rare disease patients. Canada is one of the few developed nations which does not have a federal orphan drug act.1 The lack of an orphan drug plan has proven to be a major pitfall in Canada's healthcare system. The orphan drug Strensiq is a prime example of how patients can suffer when there is not a federal orphan drug policy in place. Strensiq was designed and developed in Canada to treat hypophosphatasia (HPP), a rare disease that was first correctly diagnosed in Canada.2 Despite these linkages to Canada, when Health Canada approved it in 2015, it was still inaccessible to many rare disease patients because of its exorbitant price tag. The lack of funding for orphan drugs like Strensiq has made life for Canadians suffering from HPP even more difficult knowing that a treatment exists but is out of reach.
HPP is a rare inherited disorder that affects the development of teeth and bones by disrupting mineralization, which is key to their formation.3 Scientists believe the defective mineralization is due to abnormal alkaline phosphatase, an enzyme important in mineralization. Abnormal alkaline phosphatase results in a buildup of substances such as inorganic phosphate. Depending on the severity of the disease, HPP can lead to skeletal abnormalities and can even be life-threatening for young children.3 In adults, HPP often leads to the softening of bones, which results in recurrent fractures leading to chronic pain. HPP is considered a rare disease because its frequency across Canada is 1 in 100,000 newborns, but in one Manitoba Mennonite community, its frequency is alarmingly high at 1 in 2,500 newborns.3
As previously mentioned, HPP has a strong connection with Canada, where it was first correctly diagnosed, and where the molecule that led to its treatment was invented. Asfotase alfa is the molecule that Dr. Philippe Crine developed at the Université de Montréal. Dr. Crine originally designed the molecule to treat X-linked hypophosphatemia (XLH), a type of rickets. After realizing it could not treat XLH, Dr. Crine set out to find a disease it would treat. He came across HPP and discovered that his molecule should be an effective treatment for the rare disorder.4 Dr. Crine developed the drug through the small pharmaceutical company Enobia, and after some initial studies showed success, it was sold to the big pharmaceutical company Alexion for over $1 billion.4 As Alexion continued clinical trials, Strensiq showed positive results and was approved in the US, Germany, Japan, and Canada in 2015.2
Despite Alexion’s approval from Health Canada to sell Strensiq, the struggle is not over for those suffering from HPP. Currently, in Canada, Strensiq is only available to children involved in the clinical study and infants who would die without the drug. Any other patient that wants Strensiq will either have to pay the exorbitant price for the drug or hope that their private insurance company is willing to cover its costs.5 The last two cases are unlikely because of the astonishing price Alexion Pharma Canada has set for Strensiq, costing 2,545,920 CAD per year for an adult dose of the drug.6 Orphan drugs come with a high price tag because often there are no alternative treatments for comparison. In Canada, drug coverage is conducted at a provincial level, and while some provinces such as Ontario, Alberta and New Brunswick have coverage for selected orphan drugs, other provinces do not, creating an uneven distribution of access to expensive drugs. The cost of Strensiq provides the perfect example as to why Canada needs a federal orphan drug plan so that all Canadians can access the medication they require.
The lack of an orphan drug plan has direct effects on patients suffering from rare diseases. In the Luckevich family, siblings Michael, Theresa, and Rosanne all suffer from HPP. Theresa and Rosanne participated in the clinical trials for Strensiq which they benefited from significantly for years. Unfortunately, now that the trial is over, without coverage from the government, the sisters cannot afford to continue taking Strensiq. On the other hand, Michael, who lives in the US, has the drug covered for him from his company’s private insurance and can live free of severe chronic pain. After finding out how much the drug costs, Michael decided to stop taking the drug, in hopes that Alexion Pharmaceuticals will lower the drug price, making it affordable for his sisters.5 This is just one family’s battle with the exorbitant cost of a rare disease drug, but there are many more like them who have been anxiously awaiting a federal orphan drug plan.
Orphan drug plans are not a new concept as many developed countries such as the US, UK, and Japan have had one in place for years.1 In Canada, the idea has been brought up in the past and dismissed. In 1996, Health Canada stated that Canadians do not need an orphan drug policy because they can access any orphan drugs they need in the U.S.7 Canadians accepted this response until 2012, when after significant campaigning from the Canadian Organization for Rare Diseases (CORD), Health Canada finally started to develop an orphan drug policy framework.7 Many patient organizations and CORD were optimistic that it meant there would be changes that would make it easier for Canadians to access orphan drugs. The framework continued to develop, until October of 2017 when it was erased from the Health Canada website.8
The mysterious deletion of any reference to the orphan drug policy framework is concerning to all who were anxiously awaiting its implementation. In an email to the National Post, Health Canada said, “Many elements initially proposed as part of an orphan drug regulatory framework are now being considered more broadly for all drugs as part of this initiative.”9 This is disappointing because the problem is that orphan drugs should not be treated the same as other drugs. Rare disease drugs have a more difficult time making it through development and entering the market, which is why countries that have a strong orphan drug policy have so much more success developing and selling them to patients.
Although the deletion of the orphan drug policy framework is a setback, advocates for rare disease patients in Canada will not stop their efforts to get a plan in place. The framework previously developed was approved by many experts who thought it would be very effective in Canada. If the Canadian government can focus on helping the approximately three million Canadians with a rare disease, it would have a big impact on each patient’s life.10
1. Gammie T, Lu C, Babar Z. Access to Orphan Drugs: A Comprehensive Review of Legislations, Regulations and Policies in 35 Countries. PLoS ONE. 2015;10(10):e0140002. doi:10.1371/journal.pone.0140002.
2. Cision. Health Canada Approves Strensiq™ (asfotase alfa), the First Therapy for Patients with Hypophosphatasia (HPP), an Ultra-rare, Life-threatening Disease. https://www.newswire.ca/news-releases/health-canada-approves-strensiq-asfotase-alfa-the-first-therapy-for-patients-with-hypophosphatasia-hpp-an-ultra-rare-life-threatening-disease-522085271.html. Published 2015.
3. Hypophosphatasia. Genetics Home Reference. 2018. Available at: https://ghr.nlm.nih.gov/condition/hypophosphatasia.
4. CTI Science. Closing of acquisition of Enobia by Alexion – Yet another successful portfolio exit for CTI’s Life Sciences Fund. http://www.ctisciences.com/?p=93. Published 2012.
5. Grant K. Why drugs for ‘orphan’ diseases are a booming cost with colossal costs for patients. The Globe and Mail. https://www.theglobeandmail.com/news/national/orphan-diseases-expensive-drugs/article34628538/. Published 2017.
6. Canadian Agency for Drugs and Technologies in Health. CDR Pharmacoeconomic Review Report For Strensiq. Common Drug Review; 2017. Available at: https://www.cadth.ca/sites/default/files/cdr/pharmacoeconomic/SR0443_Strensiq_PE_Report.pdf.
7. Wong-Rieger D. Canada’s long journey toward an Orphan Drug framework. Advocate. http://www.raredisorders.ca/content/uploads/Orphan-Drug-Framework.pdf. Published 2013.
8. Murphy R. Health Canada puts brakes on plan for orphan drug framework. Benefits Canada. http://www.benefitscanada.com/news/health-canada-puts-brakes-on-plan-for-orphan-drug-framework-105588. Published 2017.
9. Forrest M. Health Canada gives 'kiss of death' to planned policy for rare-disease drugs. The National Post. http://nationalpost.com/news/politics/health-canada-gives-kiss-of-death-to-planned-policy-for-rare-disease-drugs. Published 2017.
10. About CORD | Canadian Organization for Rare Disorders. Raredisordersca. 2018. Available at: https://www.raredisorders.ca/about-cord/.
Cite This Article:
Smith E., Chan G., Zhang B., Palczewski K., Lewis K., Ho J. Strensiq: In sight but out of reach for Canadians. Illustrated by M. Yao. Rare Disease Review. October 2018. DOI:10.13140/RG.2.2.23756.87684.