This week, my latest book entitled Rare Diseases and Orphan Drugs: Keys to Understanding and Treating the Common Diseases was published by Elsevier.
The book develops a scientific rationale for funding research in the rare diseases. Currently, there is a vigorous lobbying effort, launched by coalitions of rare disease organizations, to attract research funding and donations. Funding for the rare diseases has always been small, relative to the 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. Readers of this book will learn that such supplications are unnecessary and misdirect attention from more practical arguments.
When rare diseases are funded, everyone benefits.
The book builds the case that 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 the many biological pathways that drive, in the aggregate, our common diseases. This simple theme is described and justified throughout the book. The world would benefit if we increased funding for the rare diseases, with the primary goal of curing the common diseases.
Jules J. Berman, Ph.D., M.D.
tags: rare diseases, orphan diseases, orphan drugs, rare disease funding, patient advocacy, rare disease advocacy, patient advocates, rare disease research, common disease, complex disease
