Friday, June 6, 2014

The Rationale for Funding Rare Disease Research

This week, my latest book entitled Rare Diseases and Orphan Drugs: Keys to Understanding and Treating the Common Diseases was published by Academic Press, an imprint of Elsevier.

The book builds a rationale for increased funding for the rare diseases, and begins with the assertion that it has been much easier to find effective targeted treatments for the rare diseases than for the common diseases. Furthermore, treatments that are effective against rare diseases almost always find a place in the treatment of one or more common diseases. These assertions are not based on wishful thinking, and they are not based on extrapolation from a few past triumphs wherein some treatment overlap has been found in rare and common diseases. These assertions are based on the general finding 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. A smart way to fund medical research is to increase funding for the rare diseases, with the ultimate goal of curing the common diseases.


Part I. Understanding the Problem.

Chapter 1. What are the Rare Diseases, and Why do we Care?

Chapter 2. What are the Common Diseases?

Part II. Rare lessons for Common Diseases.

Chapter 3. Aging.

Chapter 4. Metabolic Diseases.

Chapter 5. Diseases of the Heart and Vessels.

Chapter 6. Infectious Diseases and Immune Disorders.

Chapter 7. Cancer.

Chapter 8. Gastrointestinal and Renal Disorders.

Part III. Fundamental Relationships Between Rare and Common Diseases.

Chapter 9. All Diseases are Complex

Chapter 10. Rare Diseases and Common Diseases: Understanding their fundamental differences.

Chapter 11. Rare Diseases and Common Diseases: Understanding Their Relationships.

Chapter 12. Future Directions.


I urge you to read more about this book. There's a good preview of the book at the Google Books site. If you like the book, please request your librarian to purchase a copy of this book for your library or reading room.

Jules J. Berman, Ph.D., M.D.

tags: biology of rare diseases, common diseases, genetic disease, orphan diseases, orphan drugs, rare disease organizations, rare disease research, rare diseases, rare disease funding, rare disease research, funding for rare diseases, importance of rare diseases, funding opportunities, books about rare diseases, books about orphan drugs, orphan drug development