Press Release

PhRMA Statement on Orphan Diseases

PhRMA April 30, 2013

Washington, D.C. (January 22, 2010) — Pharmaceutical Research and Manufacturers of America (PhRMA) President and CEO Billy Tauzin today issued the following statement about treatments for rare diseases:

“This week’s report from Tufts University about the growing number of orphan drug designations granted by the Food and Drug Administration underscores the commitment that America’s pharmaceutical research and biotechnology companies have made to patients suffering from uncommon diseases.

“According to the National Institutes of Health, 25 million Americans have a rare disease. Because of the relatively small number of patients affected by these rare diseases, many have been historically under-treated.

“However, as the Tufts Report demonstrates, hope is on the horizon. The 425 orphan drug designations for products in development between 2006 and 2008 represent more than twice the number of designations – 208 – for the period from 2000 to 2002.

“The illnesses being targeted include rare cancers, autoimmune disorders, cardiovascular disorders, and more. According to a PhRMA survey, some examples of medicines being developed for rare diseases are: a monoclonal antibody for chronic sarcoidosis, an immune system disorder; a medicine for Lennox-Gastaut syndrome, a severe form of epilepsy; a gene therapy for cystic fibrosis; and a medicine for Friedreich’s ataxia, a genetic disorder.

”We find hope for the future of rare disease treatment by remembering the successes of the past. In a 2006 report on rare diseases – conducted with the National Organization for Rare Diseases – we found that more than 160 new medicines for these illnesses had been approved in the decade between 1995 and 2005, compared with 108 such approvals in the decade prior, and fewer than 10 in the 1970s. Many of the new medicines were the first approved to treat a particular disease.

“That’s why this work is so important – through it, researchers at America’s pharmaceutical research and biotechnology companies are helping to provide hope and relief to patients who previously may have had no treatment options. That’s why the research will continue. We’ve made a good start, but there’s a lot of work still to be done.”


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 $50.3 billion in 2008 in discovering and developing new medicines. Industry-wide research and investment reached a record $65.2 billion in 2008.

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