Public Money for Research on Rare Diseases: Is it Fair?

by Carte Blanche on February 12, 2017 - 5:27pm

Governments most often invest in public medical research programs that are likely to benefit the large majority of the population. This puts research for rare diseases at a disadvantage, as the private pharmaceutical industry is reluctant in investing in research that would not result in a lucrative business, as its results would target only a small number of people. Thus, patients with debilitating rare disorders are left without treatment options and often a lack of even a basic scientific understanding of the disease. There is an ethical debate regarding whether taxpayer money should be invested in research for rare diseases through public research funding. The utilitarian approach would seemingly go against such an investment while virtue ethics would argue for public investment in research for rare diseases. I believe that part of public research funding should indeed be allocated to research for rare diseases because it is not only supported by ethical virtues of beneficence, professional virtues of scientific advancement and society’s moral obligation to care for these patients, but in this case, the utilitarian arguments against such an investment fail.

The ethical frameworks of utilitarianism and virtue ethics are in fact in conflict with one another in the debate of whether or not public funding should be allocated for research for rare diseases. The utilitarian view would suggest that it is not ethical to invest money in research that will benefit a small number of people and taking into account the reality of limited resources, it would also mean taking away from research that could benefit a larger proportion of the population. However, there are several points that make the utilitarian ethical arguments fail. The results of any drug research are quite uncertain and it is almost impossible to make a proper calculation of the end results of the research. This can be illustrated by a study that shows how on “average only one in 10 pharmaceutical compounds developed is successfully marketed.” Secondly, although the aim of the research may be to benefit patients with a rare disease, there could be downstream results that would benefit the advancement for a relatively common disease. For example, research on a rare genetic disease, “homozygous familial hypercholesterolaemia … led to the development of statins,” a now very common medication used to control high cholesterol in the general population internationally (Gericke 166). Thirdly, although each individual rare disease does not affect many people, an article published in 2012 states that there are “over 6000 known rare diseases and … 6% to 8% of the population is affected by a rare disease.”, illustrating that it is not uncommon to have a rare disease (Gupta "Rare Diseases: Canada’s “research Orphans”).

With virtue ethics as a basis, one would argue that society has a moral obligation towards those who are sick and that it would be unjust to abandon them just because they have a rare condition. This would also be supported by the social contract theory, which suggests “people are bound together by a sense of community veering in the direction of egalitarianism … [allowing] the state to play a more important part in making a fair and equitable system” (Merrill 36). Furthermore, doctors vow to always practice in accordance with the virtue and principle of beneficence, which, “requires that agents take positive steps to help others, not merely refrain from harmful acts,” (Gericke 166). In addition, medical professionals have a “professional obligation to advance scientific knowledge in pursuing new therapies,” suggesting that investing in research for rare diseases is a virtuous act (Gericke 165). Research activities contribute to education and experience for graduate and post-graduate students and results of seemingly futile basic science research may often contribute to future breakthroughs.

Overall, the utilitarian ethical framework does not provide strong arguments against government investments in research for rare diseases, as it is almost impossible to calculate benefits that may arise in the future, nor can you estimate how many people will end up benefiting the research. From a virtue ethics point of view, both society and medical professionals have moral and professional virtue-based obligations that would compel them to contribute to research.  Given that we have limited resources, a possible compromise could be public-private partnerships with government-based incentives for private pharmaceutical companies to pursue research for rare diseases. 

Works Cited

Gericke, C A, A. Riesberg, and R. Busse. "Ethical Issues in Funding Orphan Drug Research and Development." Journal of Medical Ethics 3 (2005): 164-68. PubMed. 31 Mar. 2005. Web. 9 Feb. 2017. <>.

Gupta, Samir. "Rare Diseases: Canada’s “research Orphans”." Open Medicine. Open Medicine Publications, Inc., 28 Feb. 2012. Web. 09 Feb. 2017. <>.

Merrill, John C. “Overview: Theoretical Foundations for Media Ethics.” Controversies in Media Ethics, A. David Gordon, John M Kittross, John C Merrill, William Babcock, and Michael Dorsher 3rd Edition. Routledge, 2011, p 36.