Drug Development for Rare Diseases - Biotech, Pharma and Life Science Channel
The economics of any industry relies on a large demand for a particular product; this applies to the pharmaceutical industry where efforts are focused on big and relatively straight-forward diseases such as hypertension and diabetes where there is great demand for the medications. This is why it is such a surprise and so encouraging to hear that a record number of medicines are in development for rare diseases. A rare disease (as defined by the U.S. government) is one that afflicts fewer than 200,000 Americans. In practice, 80% of rare diseases affect fewer than 6,000 U.S. patients, so although each rare disease in its self is rare, a total estimated 25-30 million Americans suffer from rare diseases, making them a cause for concern. Half of the 7,000 rare diseases affect children.
Rare diseases are a cause of great deal of suffering for the patients affected, but are often very complex, making research challenging and costly. this challenges include finding enough patients and medical experts for clinical trials, managing difficult safety issues (since the disease they are dealing with are often fatal) and sourcing funds to do the research. The later has been made easier by the Orphan Drug Act (USA), which provides some incentives for research into rare diseases. Although these challenges may put off some researchers, Chris Silber, M.D.,Chris Silber, M.D., vice president of U.S. Clinical Research and Medical Affairs for Lundbeck Inc, disagrees; “Finding a therapy that really works in an underserved population that has been through tremendous adversity is one of the most gratifying things I can imagine”
A record 460 medicines for rare diseases are in late stages of development (in clinical trials or awaiting FDA reviews). A comparison of previous reports shows a steady increase in research that is bringing new medicines to the clinical trial stage and beyond. In 1989, 133 medicines were in development for rare diseases, which increased to 176 in 1991, to 303 in 2007 and now to 460 today. Peter L. Saltonstall, president and CEO of the National Organization for Rare Disorders (NORD), expressed his concern about the lack of treatment for so many rare diseases, but was encouraged by the recent increase in research; “The patient community applauds those individuals and companies choosing the less-traveled research path. We are hopeful that many rare disease patients who currently have no treatment will someday benefit from these very important research efforts."
Source; Adapted from PhRMA
Last updated March 2011
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