Biotech Values

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From FDA blog.. good for SRPT...

http://blogs.fda.gov/fdavoice/index.php/2014/10/the-more-we-know-about-rare-diseases-the-more-likely-we-are-to-find-safe-and-effective-treatments

The more we know about rare diseases, the more likely we are to find safe and effective treatments

That’s why I am pleased about FDA’s support for an exciting new tool researchers are using to study rare diseases. It’s a new database with information about the diseases’ “natural history.”

“Natural history” is the scientific term to describe how a disease would progress with no treatment. Since a disease can affect different people differently, scientists must study many cases of a disease to acquire a thorough understanding of its natural history. Well-conducted studies of natural history can yield vital information about:

Biomarkers, demographic, genetic, and environmental variables that correlate with the course and stages of the disease;

Identification of patient subpopulations with different characteristics and effects of the disease;

Patient perspectives on what aspects of disease are most important to treat; and,

How to quantify those aspects so that they can serve as useful outcome measures for clinical trials.

But when it comes to rare diseases, their natural histories frequently are not fully understood because there are simply not enough cases that have been observed and studied. This lack of knowledge limits researchers’ ability to study rare diseases and develop new treatments. Knowledge of natural history is essential for developing more efficient clinical trial designs. It also could help reduce the length and cost of drug development and, possibly, contribute toward greater predictability of clinical development programs.