The Right to Try
In Canada, the availability of drugs is mostly dependent on which pharmaceutical companies see profit potential and who are willing to invest in licenses to manufacture and market the drugs.1 While the theory of only investing in low-risk products is efficient economically for these companies, it builds significant inequalities for those suffering from rare or uncommon diseases, whether the disease is rare regionally or globally. Thankfully, the Canadian government has come up with a program to supplement this gap in the system: Health Canada's Special Access Programme (SAP) considers requests for access to drugs that are unavailable for sale in Canada from practitioners treating patients with serious or life-threatening conditions when conventional treatments have failed, are unsuitable or unavailable.2 The SAP has helped many Canadians receive treatments that may be commonly used in other countries but are not licensed in Canada, free of charge.2
While the SAP is a vital aspect of healthcare to many, the program itself is limited and constricted by budgets and application criteria.3 Physicians say the application process is lengthy and unpredictable: they will apply for the same medication for multiple patients and only one patient may be approved.3 This lengthy process paired with the unpredictable and unscientific method of approving applications, leads to frustration among physicians and patients around the country and ultimately can cost the lives of patients.3 Alberto Ceccarelli, an Alberta man, suffered undiagnosed for months with a rare parasitic infection and his physician applied for access to the drug albendazole, which is commonly used in Africa.4 Although the medication has been on the World Health Organization’s list of essential medications for over 20 years, the SAP rejected Mr. Ceccarelli’s application ultimately leading to him not surviving the infection.4 Mr. Ceccarelli was living with a disease that was only regionally rare, and the medication that could have saved his life costs just sixty USD over-the-counter in Zambia.4 Other Canadians who suffer from relatively rare diseases to which treatments exist in other countries have also met the same fate. Dr. Christie and colleagues determined that the Special Access Program uses its discretionary authority arbitrarily, based on subjective assessments of data and the SAP renders value-laden decisions camouflaged in the language of evidence-based decision-making.3 After being denied drugs for their HIV patients, Dr. Christie and colleagues confirmed that their rejection was a result of the SAP not making decisions about safety and efficacy, and the SAP exercising discretionary authority based on subjective assessments.3 Another vital component of the SAP’s weaknesses is the turnaround time between applying for and receiving a drug. With Canada’s increasing variety of immigration and globalization, tropical diseases are making their way into the country with the SAP as the only way for physicians and patients to access the common medicines that already exist.2 In the case of strongyloidiasis, another tropical parasite, Ivermectin is another globally used treatment that is generally accepted for use under the SAP, but a study by Dr. Boggild found that the average time from submitting the application to receiving the drug is five days, even in hyperactive cases.5 These five days are critical and can be too long of a time for some patients to wait.
Although useful, the SAP does not adequately suffice the need for medications of Canadians suffering from a disease that is relatively or globally rare in Canada. But there is another leg of the SAP that is also not adequately meeting the medical needs of Canadians: experimental drug use. A movement has begun in Canada by people with rare diseases like ALS to give them the “Right to Try.”6 This spin off of the recently passed “Right to Die” law is building momentum in the rare disease community. People who have rare diseases feel that it is their right to try drugs that are still experimental when no other treatments for their disease exist.6 Mr. Gino Sisera, a 34-year old Canadian man with three children has Amyotrophic Lateral Sclerosis (ALS) and has been bound to a wheelchair, only speaking via computer.4 “I think if anyone put themselves or a loved one in a terminal situation, then they would want as many options as possible… How can that be denied to someone who has no other option?" he said in an interview.6
Thirty-five-year-old Jeffrey Perreault also has ALS and is lobbying for legislation on the “Right to Try.” Mr. Perreault would do anything to halt the progression of this debilitating disease. In an interview with CTV News he said, "I know that if that law was in place, I could get that lifesaving treatment today and I would be around for my wife."6 While Canada's SAP offers some chance for patients with no alternative treatments to obtain non-marketed drugs for treatment, patients like Perreault say, “There are too many legal hoops to jump through in order to access those drugs… The SAP does not provide access to experimental stem cell therapies, which are considered a promising direction.”6
While the SAP does not sufficiently provide Canadians with life threatening rare diseases access to experimental drugs, the only other options these patients have is to join clinical trials. However, the criteria for clinical trials are contingent on numerous usually including no co-morbidities and early progression of the disease, which further widens the inequalities for those with rare diseases who often are in the late stages of disease. The right to try movement has already been in legislation in the United States in Colorado and Louisiana if the drug has passed phase I of a clinical trial.7 While the argument persists that the patients with rare, life-threatening illnesses should be granted the right to try anything to live, a series of legal and safety concerns arises at the possible legislation. Firstly, policy makers are wary to essentially create a “back door” for pharmaceuticals to make money off of drugs not approved by the FDA.6 In addition, since the proper standard of efficacy and safety concerns are not considered by the FDA, there is significant risk that rests on the prescribing physician’s decision that the risks of treatment do not outweigh the possible gains.7 If moving forward with the policy of Right to Try, important safeguards would need to be in place to ensure safety in the interest of the patient and not allow pharmaceutical companies to prey on the desperation of sick patients that are willing to pay for possible treatments.7
It seems that Canadians suffering from diseases to which there is no treatment available or just no treatment on the Canadian market have nowhere else to turn but to the SAP, and that this program can create inequalities with regards to access to care by many people. While the SAP does allow some of these patients to receive these treatments, the application process is too lengthy, unpredictable and unjust leaving many Canadians with a rare disease with no hope but to wait, and too often a treatment is not on the market fast enough. With a glimmer of hope, the “Right to Try” movement is progressing forward in Canada bringing about the possibility for a new era for those living with rare diseases.
1. Government of Canada. Canada’s pharmaceutical industry and prospects. Published April 8, 2014. https://www.ic.gc.ca/eic/site/lsg-pdsv.nsf/eng/hn01768.html.
2. Special Access Programme - Drugs. http://www.hc-sc.gc.ca/dhp-mps/acces/drugs-drogues/sapfs_pasfd-eng.php. Published March 18, 2015.
3. Christie T, Harris M, Montaner J. Special Access Denied: A Case Study of Health Canada's Special Access Program. Healthcare Policy | Politiques de Santé. 2006;2(2):27-34. doi:10.12927/hcpol.2007.18525.
4. Why world-beating tropical drugs are so hard to get in Canada. The Globe and Mail. http://www.theglobeandmail.com/news/national/why-world-beating-tropical-drugs-are-so-hard-to-get-incanada/article33469954/. Published January 2, 2017.
5. Boggild AK, Libman M, Greenaway C, McCarthy AE, on behalf of the Committee to Advise on Tropical Medicine and Travel (CATMAT). CATMAT statement on disseminated strongyloidiasis: Prevention, assessment and management guidelines. Can Comm Dis Rep 2016;42:12-19.
6. Patients demand 'right to try' experimental treatment. CTVNews. http://www.ctvnews.ca/health/patients-demand-right-to-try-experimental-treatment-1.3160133. Published November 14, 2016.
7. Begg CB, Kim K, Neaton JD. “Right to Try” laws. Clinical Trials. 2014;11(5):519-520. doi:10.1177/1740774514546574.
Cite This Article:
Vucenovic D., Zheng K., Chan G., Ho J. The Right to Try. Illustrated by H. Zhang. Rare Disease Review. February 2017. DOI:10.13140/RG.2.2.21801.60008.