Life-Saving At a High Cost: The Case for Provision of Rare Disease Treatments Being a Human Right

by | Mar 6, 2023

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About Alice Viera

Alice Viera is a JD student at New York University School of Law. At NYU, she has been named an International Law and Human Rights Fellow, a Human Rights Scholar, and a Robert McKay Scholar. She currently works as a Research Assistant for the Earth Rights Advocacy Clinic at the Center for Human Rights and Global Justice. Before law school, she received a Bachelor of Arts in English with Distinction from Stanford University. Her primary interests are human rights —particularly in relation to health, the environment, and technology— as well as Latin American law, politics, and policy.

Earlier this year, Brazilian authorities added Zolgesma, once “the world’s most expensive medical treatment to date,” to the country’s mandatory coverage list. The medication treats Spinal Muscular Atrophy, a rare genetic disorder whose sufferers are likely to die before the age of two. Zolgesma’s addition to Brazil’s essential medicine list resulted from the more than one hundred lawsuits filed invoking the constitutional right to health. Although this case is a success story, its replicability is limited given that 86 countries lack a similar constitutional provision. In the absence of such a right, the question arises: can people with rare diseases use international human rights law to mandate government coverage of expensive, life-saving medications?

The right to health presents a possible avenue to human rights litigation. Article 12 of the International Covenant on Economic, Social and Cultural Rights (ICESCR) guarantees the right “to the enjoyment of the highest attainable standard of physical and mental health”, which implies the obligation to “provide essential drugs”.

Unfortunately, a state’s failure to provide treatments for rare diseases probably would not constitute a violation of this duty. “Essential” medicines appear to be those that treat communicable, widespread diseases, which, by virtue of being primarily genetic and having low prevalence rates, rare diseases are not. Furthermore, states receive a high degree of deference when defining essential drugs. Accordingly, when a state justifies the exclusion of a medication on the basis of other priorities and financial constraints, it is unlikely to be acting in contravention of the law.

Claims framed under the right to life and the freedom from discrimination would also likely fail. On the right to life, consider Nitecki v Poland, a case before the European Court of Human Rights. It concerned a man who suffered from a life-threatening disease, for which a treatment was available. However, he could not pay his state-ordained 30% share of the drug, and Poland refused to exempt him from payment. The Court did not find a violation of the right to life, but it did admit that, had Nitecki “received no medical treatment . . . or [been made to pay] less than” 50% of the drug’s cost, “Article 2 may have been violated.” Nevertheless, subsequent case law has emphasised the state’s authority to determine how to allocate its limited resources in the healthcare realm.

Similarly, various human rights fora excuse what would otherwise be violations of the prohibition of discrimination when a state acts in pursuit of a “legitimate aim”. Depending on the forum in question, these aims can include economic considerations and the protection of health, among others. This indicates that, even if a state’s denial of medications to certain populations is discriminatory, such a denial may be excusable under human rights law.

Notwithstanding these dead ends, lesser-explored avenues present opportunities for prospective litigants. The right to health also requires states to “adopt and implement a national public health strategy and plan of action” that “give[s] particular attention to all vulnerable or marginalized groups”. People with different rare diseases could join to allege a collective claim, arguing that the state’s systemic ignorance of their health needs of people constituted a violation of the right to health.

Such groups could also invoke ICESCR Article 12(1)(c), which requires that all existing medicines be made available and accessible to prevent, treat, and control “epidemic, endemic, occupational and other diseases”. This obligation appears directed toward diseases that afflict larger proportions of a state’s population, but by collectively pointing to a phenomenon of neglect, people with rare diseases may triumph.

Lastly, claimants could allege a violation of the right to benefit from scientific progress (ICESCR Article 15(1)(b)). Within this right lies the obligation “to take steps necessary ‘for the diffusion of science,’ . . . in a non-discriminatory manner”. Consequently, people with rare diseases could point to the failure of a state to expand its patent legislation framework in a manner favorable to the development of affordable, widespread treatments as a violation. Although it could not address the failure to provide specific medications, this approach could “facilitate the manufacture of genetic gene therapies” in the future.

Ultimately, human rights jurisprudence on rare disease treatments is slim, so these strategies’ chances of success are unclear. Nevertheless, the General Assembly’s aim of universal health coverage for “all persons living with a rare disease by 2030” may signal increased willingness by human rights bodies to impose more stringent demands on states with respect to rare disease treatments.

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