Orphan Diseases

Orphan Diseases are extremely rare, with a prevalence of less than 5 per 10,000 (fewer than 200,000 in the U.S.). Fibrodysplasia Ossificans Progressiva is an orphan disease. The problem with orphan diseases is that few people do research on these diseases. The rarity not only makes the process of research difficult, but it also makes funding scarce. In particular, it is not cost effective for drug companies to develop medications to treat diseases with very low prevalence; from a business standpoint, there's no demand. However, the Orphan Drug Act of 1983 aids these companies to develop drugs for these patients by giving them longer patent rights, tax benefits, etc. In fact, drugs for multiple myeloma, cystic fibrosis, and snake venom came out of this Orphan Drug Act.

In any case, there is an ethical question: how much resources should be spent on low prevalence diseases? If the goal of research is to maximize health benefits for the most number of people (a highly utilitarian approach), then it seems that few resources should be allocated to orphan diseases. After all, 2500 people in the world have fibrodysplasia ossificans progressiva. Yet "some 7,200,000 men and 6,000,000 women are living with some form of coronary artery disease" (Wikipedia). If you take a completely objective, utilitarian approach - which might seem the most "scientific" - how do you justify spending any resources on FOP? But if no one researches FOP, then those patients are doomed to a disease which we won't do anything about.

I find these ethical dilemmas particularly interesting, but they are not by any means novel. Philosophical ethical theory has focused for centuries on this tension between utilitarianism and Kantian ethics, but developing practical guidelines from first principles is always a difficult endeavor.