Thursday, February 18, 2016

Rationale for Funding Rare Disease Research

Funding for the rare diseases has always been small, relative to the common diseases. Funding agencies find it impractical to devote large portions of their research budget to the rare diseases, while so many people are suffering from the common diseases. As it turns out, direct funding of the common diseases has not been particularly cost-effective. With relatively little research funding, progress in the rare diseases has greatly outpaced progress in the [highly funded] common diseases.

Laypersons advocating for rare disease research almost always appeal to our charitable instincts, hoping that prospective donors will respond to the plight of a few individuals. Supplications are unnecessary and misdirect attention from more practical arguments. When rare diseases are funded, everyone benefits. It is much easier to find effective targeted treatments for the rare diseases than for common diseases. Furthermore, treatments that are effective against rare diseases will almost always find a place in the treatment of one or more common diseases. This assertion is not based on wishful thinking, and is not based on extrapolation from a few past triumphs wherein some treatment overlap has been found in rare and common diseases. The assertion is based on the observation that rare diseases encapsulate biological pathways that drive, in the aggregate, our common diseases. This simple theme is described and justified throughout many of my blogs, this month.

When we increase funding for rare diseases, we move closer to curing the common diseases. This is the scientific rationale for increasing our funding for rare disease research.

Rare Disease Day is coming up February 29 (a rare day for rare diseases). In honor of the upcoming event, I'll be posting blogs all month, related to the rare diseases and to rare disease funding.

- Jules Berman (copyrighted material)

key words: rare disease, orphan drugs, orphan diseases, zebra diseases, rare disease day, disease complexity, common diseases, funding, jules j berman