Equity vs. Efficacy: The Case of Orphan Diseases

While reading through Paul Farmer’s article entitled “Pathologies of Power: Rethinking Health and Human Rights,” I was particularly struck by the following:

Over the past decade and against a steady current of naysaying, we have     channeled significant resources to the destitute sick in Haiti, Peru, Mexico, and   Boston. We didn’t argue that it was “cost-effective,” not did we promise that such efforts would be replicable. We argued that it was the right thing to do. It was the human rights thing to do.

It is quite natural to think that there is only so much money available in each country to be spent on the health of its populace. In this mind frame, proper allocation of resources is crucial in order to lower mortality and morbidity by the greatest possible amount. But what happens to those members of the population who suffer from diseases and conditions that affect a small proportion of the population? Should they simply go by the wayside, as it would be too expensive to treat them? What if a disease affects a relatively small proportion of a wealthy population but a large proportion of a lower income population?

An orphan disease is defined not only as “diseases that are neglected by doctors,” but has also been used to “designate diseases that affect only small numbers of individuals.”[i] Exact definitions of orphan diseases vary from nation to nation. The US National Organization for Rare Disorders (NORD) identifies tuberculosis as a rare disorder, despite the fact that “globally, TB is second only to HIV/AIDS as a cause of illness and death of adults, accounting for nearly nine million cases of active disease and two million deaths every year.”[ii] The definition of an orphan disease can also vary within a nation (NORD recognizes about 1200 diseases, while the NIH’s Office of Rare Diseases publishes a list of over 6000).[iii] The development of orphan drugs has been deemed to not be very  cost-effective.[iv] In this case, one must weigh equity against what is deemed to be affordable. Lately, countries such as Australia, Japan, the US, as well as the European Community have begun passing measures to increase the development of drugs for these rare and neglected diseases. The US Orphan Drug Act (1983) seems to have been particularly effective, where from 1983 to 2003 nearly 1100 orphan drugs and biological products were developed and 231 approved.[v] Compare this to the “handful of orphan drugs available before 1983.”[vi]

At first glance, this seems to be a victory for human rights. If it is not cost-effective for pharmaceutical companies to invest in orphan drug development, the government will make it worthwhile. However, these companies are still looking to maximize their profit. Drug companies will tend to concentrate on producing drugs for the most affluent markets, even if their morbidity and mortality rates do not reflect global rates. This is especially clear when looking at the case of infectious and parasitic diseases, “which account for a third of the worldwide disease burden” but comprise “only 5% of the disease burden in high-income countries.”[vii] A study by Trouiller et al. found that from 1975 to 1999, the “number of new chemical entities per million DALYs” was two to three times higher for the main diseases of the high-income countries compared to values for infectious and parasitic diseases.[viii] While legislation similar to that which has been passed in the US is a great first step, it has seemingly not done enough to encourage pharmaceutical companies to think globally. If we truly want to promote equity and “do the right thing,” this must be remedied. Personally, I doubt that pharmaceutical companies will take up this task on their own. Here, national governments will have to work on passing legislation that is not only similar to the US Orphan Drug Act, but that also aims to incentivize drug development for diseases that disproportionately affect lower-income nations.


[i] J.K. Aronson, “Rare diseases and orphan drugs,” British Journal of Clinical Pharmacology 61.3 (2006): 243.

[ii] “WHO Declares TB an Emergency in Africa.” World Health Organization. 26 Aug. 2005. <http://www.who.int/mediacentre/news/releases/2005/africa_emergency/en/index.html&gt;.

[iii] Aronson 244.

[iv] Aronson 244.

[v] Aronson 244.

[vi] United States, Office of the Inspector General, The Orphan Drug Act: Implementation and Impact (May 2000) 9.

[vii] Trouiller et al., “Drug development for neglected diseases: a deficient market and a public-health policy failure,” The Lancet (Jun 22, 2002): 2188.

[viii] Trouiller et al. 2189.

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