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

• Author: Sarah Burningham
• Position: External Research Fellow, Health Law Institute, University of Alberta; PhD Candidate
• Pages: 317-350

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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).

318

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.

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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 .cadth.ca>.

7. Cooper, supra note 4.

8. Laucius, supra note 3 (it appears that Soliris may be covered for aHUS in

Quebec).