Courts, Challenges, and Cures: Legal Avenues for Patients with Rare Diseases to Challenge Health Care Coverage Decisions

AuthorSarah Burningham
PositionExternal Research Fellow, Health Law Institute, University of Alberta; PhD Candidate
(2015) 1 CJCCL
Courts, Challenges, and Cures:
Legal Avenues for Patients with
Rare Diseases to Challenge Health
Care Coverage Decisions
Sarah Burningham*
is paper examines the legal tools that may be available to patients with rare diseases
seeking to compel Canadian governments to provide funding for required or desired
treatments. In making health care coverage decisions, governments must decide whether
to extend funding to cover potentially expensive treatments that bene t relatively few
people, particularly when those treatments are experimental. If particular treatments
are not covered by health insurance, patients with rare diseases may turn to the courts
with claims based in constitutional, human rights, administrative, international or tort
law, in an e ort to compel the government to provide funding. Strategies that employ
the courts in this way are unlikely to be successful, as courts tend to defer to government
on these types of policy-driven decisions.
* External Research Fellow, Health Law Institute, University of Alberta; PhD
Candidate, University of Ottawa.  ank you to the Health Law Institute,
Timothy Caul eld, Professor Ubaka Ogbogu, Robyn Hyde-Lay, and Kalina
Kamenova, for support, insightful comments and feedback.  ank you to
Professor Barbara von Tigerstrom who read and provided comments on an earlier
draft.  is paper is made possible by the support of the Canadian Institutes of
Health Research (Emerging Team Grant on “Developing E ective Policies for
Managing Technologies for Rare Diseases”) and the support of Genome Canada,
Genome Alberta, CIHR, and Alberta Health and Wellness (PACEOMICS).
Burningham, Courts, Challenges, and Cures
I. I
II. O  H C C D-M
III. L M  C H C C D
A.  e Charter
B. Administrative Law
C. Human Rights Legislation
D. International Law
E. Tort Law
IV. C
I. Introduction
Canadian patients with rare diseases may face unanticipated
barriers in pursuit of treatment. Given the plethora of diagnostic
challenges associated with rare diseases, patients may go undiagnosed
or misdiagnosed for years.1 If and when they are correctly diagnosed,
e ective treatment may not exist, as characteristics of rare diseases may
render the study of these illnesses and the development of medication
di cult or unpro table.2 Even if treatment is available, it may not
1. Erik Tambuyzer, “Rare Diseases, Orphan Drugs and their Regulation: Questions
and Misconceptions” (2010) 9 Nature Reviews Drug Discovery 921 at 921;
Paola Pierucci et al, “A Long Diagnostic Delay in Patients with Hereditary
Haemorrhagic Telangiectasia: A Questionnaire-Based Retrospective Study
(2012) 7:33 Orphanet Journal of Rare Diseases (epub).
2. Erika F Augustine, Heather R Adams & Jonathan W Mink, “Clinical Trials
in Rare Disease: Challenges and Opportunities” (2013) 28:9 Journal of Child
Neurology 1142; Tambuyzer, ibid at 922; John Forman et al, “ e Need for
Worldwide Policy and Action Plans for Rare Diseases” (2012) 101:8 Acta
Paediatrica 805 at 805. Drug developers have tended to neglect rare diseases,
opting to focus on developing medications for more common diseases, though
many countries have introduced policies and legislation intended to stimulate
research in this area: Samir Gupta, “Rare Diseases: Canada’s ‘Research Orphans’”
(2012) 6:1 Open Medicine e23 at e23-e26; Abbas H Panju & Chaim M
Bell, “Policy Alternatives for Treatments for Rare Diseases” (2010) 182:17
Canadian Medical Association Journal E787; Mae  amer, Niall Brennan &
Rafael Semansky, “A Cross-National Comparison of Orphan Drug Policies:
Implications for the US Orphan Drug Act” (1998) 23:2 J Health Pol 265.
(2015) 1 CJCCL
be covered by the public health care system and the cost may put the
treatment out of reach for many patients.3
e recent controversy surrounding the drug eculizumab (brand
name Soliris) provides a helpful example.  e drug was approved by
Health Canada as treatment for two rare diseases: paroxysmal nocturnal
hemoglobinuria (PNH) and atypical hemolytic uremic syndrome
(aHUS).4 e drug, with a price tag of about half a million dollars every
year for each patient, is very expensive.5 e Canadian Drug Expert
Committee, which makes suggestions to provinces regarding which drugs
should be covered by their health insurance plans, recommended in both
cases that the drug not be funded. In the case of PNH, the Committee
believed the cost was too high, and in the case of aHUS, the Committee
doubted the e cacy of the drug.6 In 2011, the provincial governments
coordinated to cover Soliris for PNH.7 However, provincial health
plans, for the most part, do not cover Soliris for aHUS.8 As this case
demonstrates, patients with rare diseases may face agonizing uncertainty
and disappointment about the scope of coverage. Coverage of a particular
3. Media reports frequently feature personal stories of patients in need of
medication not covered by the public system. See e.g. Sarah O’Donnell, “Family
Wins Drug-Cost Coverage”, Edmonton Journal (13 August 2013) A1; Joanne
Laucius, “Rare Condition Could Leave 12-year-old a Drug Orphan”, Ottawa
Citizen (5 August 2013) online: Ottawa Citizen
com>; “North Vancouver Man Denied Life-Saving Drug”, CBC News (10 April
2011) online: CBC News .
4. Health Canada, “Product Monograph: Soliris (eculizumab)” (25 May 2009),
online: Health Canada ; Laucius, ibid. An estimated
90 people in Canada have PNH: Sam Cooper, “Drug-Funding Agreement Gives
Rare Disease Patients New Hope”, Edmonton Journal (26 July 2011) A2.
5. Laucius, supra note 3.
6. Canadian Drug Expert Committee, “CDEC Final Recommendation:
Eculizumab: (Soliris – Alexion Pharmaceuticals Inc.) New Indication: Atypical
Hemolytic Uremic Syndrome” (18 July 2013), online: Canadian Agency for
Drugs and Technologies in Health ; Canadian Expert
Drug Advisory Committee, “CEDAC Final Recommendation: Eculizumab:
(Soliris – Alexion Pharmaceuticals Inc.) Indication: Paroxysmal Nocturnal
Hemoglobinuria” (19 February 2010), online: Canadian Agency for Drugs and
Technologies in Health>.
7. Cooper, supra note 4.
8. Laucius, supra note 3 (it appears that Soliris may be covered for aHUS in

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