US biopharmaceutical research companies are stepping up their research into development of drugs for treating several rare diseases. There are 460 medicines for rare diseases in late stages of the pipeline. They are either in clinical trials or awaiting Food and Drug Administration (FDA) review, according to a report released by the Pharmaceutical Research and Manufacturers of America (PhRMA).
The report is intended to raise awareness of the problem and need for solutions. Of the 7,000 rare diseases, about half afflict children, according to the National Institutes of Health (NIH).
“A rare or “orphan” disease afflicts fewer than 2 lakh Americans. About 80 per cent of rare diseases affect fewer than 6,000 US patients, but taken together, rare diseases are common. An estimated 25 to 30 million Americans suffer from a rare disease. Often, patients suffer for years before getting a correct diagnosis, only to hear there is very little or nothing to help them,” said John J Castellani, president and CEO PhRMA.
“Researchers at biopharmaceutical companies are working to bring hope to patients with limited options”, he added.
According to Peter L. Saltonstall, president and CEO, National Organization for Rare Disorders (NORD), the lack of approved treatment for many rare diseases is a serious public health concern worldwide, but that much progress has been made in recent years.
A comparison of PhRMA's three previous reports on medicines in development for rare diseases reveals a steady increase in research that is bringing new medicines to the clinical trial stage and beyond. PhRMA's first report on the subject in 1989 found 133 medicines in development for rare diseases. The number jumped to 176 in 1991, to 303 in 2007 and to 460 today.
Researchers attribute the increases in part to progress made in basic science that provides clues that can be followed in the lab and in part to improved government policies like the Orphan Drug Act, which provides some incentives for research into rare diseases.
The Pharmaceutical Research and Manufacturers of America (PhRMA) represents the country's leading pharmaceutical research and biotechnology companies, which are devoted to inventing medicines that allow patients to live longer, healthier, and more productive lives. PhRMA companies are leading the way in the search for new cures. PhRMA members alone invested an estimated $45.8 billion in 2009 in discovering and developing new medicines. Industry-wide research and investment reached a record $65.3 billion in 2009.