Pharma Companies Developing 460 Orphan Drugs

(FDA.gov)

A report from the Pharmaceutical Research and Manufacturers of America (PhRMA) says there are now 460 medicines for rare  or “orphan” diseases in late stages of the pipeline, either in clinical trials or awaiting Food and Drug Administration (FDA) review. The group says this is the largest number of drugs for orphan diseases in development at later stages.

As defined by U.S. federal agencies, an orphan disease is one that afflicts fewer than 200,000 Americans. About 80% of rare diseases affect fewer than 6,000 U.S. patients, but while the number afflicted with specific diseases may be small, the total number of Americans with orphan diseases is substantial; an estimated 25-30 million Americans suffer from a rare disease.

Of the 7,000 rare diseases, about half afflict children, according to National Institutes of Health. Often, patients suffer for years before getting a correct diagnosis, only to hear there is very little or nothing to help them.

A comparison of PhRMA’s of previous reports on medicines in development for rare diseases reveals a steady increase in new medicines reaching the clinical trial stage and beyond. PhRMA’s first report on the subject in 1989 found 133 medicines in development for rare diseases. That number jumped to 176 in 1991, then to 303 in 2007, and to 460 today.

Some 217 drugs — nearly half (47%) of the 460 drugs in development — are related to cancer. Another 67 drugs, about 15 percent of the total, are for genetic disorders.

PhRMA  attributes the increases in the number of rare-disease drugs in part to progress made in basic science, as well as government policies like the Orphan Drug Act, which provides some incentives for research into rare diseases.

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