Help is Available for Patients Suffering from Rare Diseases;
More Than 300 Orphan Drugs in Development Pipeline

Washington, D.C. (July 24, 2008) — Recognizing the importance of access to innovative therapies for patients suffering from rare diseases, America’s pharmaceutical research and biotechnology companies today reinforce their commitment to ongoing scientific research in this area, and highlight the help available for existing therapies through patient assistance programs.      

“With nearly 30 million patients battling rare diseases in America, it is important – now, more than ever – that the innovation pipeline continues to grow so that patients can better arm themselves in their fight against disease,” said Billy Tauzin, President and CEO of the Pharmaceutical Research and Manufacturers of America (PhRMA).   “While pharmaceutical scientists are currently developing new medicines to add to this expanding arsenal, we recognize that innovative medicines do no good if they sit on pharmacy shelves and out of reach of patients who need them the most.  

“America’s pharmaceutical research companies are committed to helping make sure that these medicines are within reach of America’s most vulnerable patients and to show this commitment they created the Partnership for Prescription Assistance (PPA),” Tauzin stressed. “The good news is that PPA has helped patients suffering from rare diseases find patient assistance programs that offer free or heavily discounted medicines.”

PPA is a clearinghouse of information on more than 475 public and private patient assistance programs that offer free or nearly-free medicines to eligible uninsured and financially-struggling patients.  Since 2005, when America’s pharmaceutical research companies created PPA (www.pparx.org or 1-888-4PPA-NOW), more than 5 million patients nationwide have been helped. 

“Incredible advances have been made in the development of orphan drugs that can help patients conquer rare diseases,” Tauzin stressed.  “In the 1970’s, fewer than 10 medicines were approved to treat rare diseases, but thanks to the passage of the Orphan Drug Act in 1983 and the investment and dedication of pharmaceutical research companies, 325 orphan medicines have been approved by the Food and Drug Administration to treat rare diseases.  And importantly, more therapies are on the way.”

There are currently more than 300 new orphan medicines in the pipeline to treat diseases such as rare cancers, muscular dystrophy and cystic fibrosis.  “These medicines offer great hope for future patients and their families,” Tauzin said.

Besides costing more than $1 billion, on average, to develop a new medicine, it takes anywhere from 10-15 years to develop one.  Most of the compounds investigated as potential new medicines never make it out of the lab, and only one of every 5,000-10,000 potential compounds investigated are ever approved for use as a medicine.

Read the backgrounder.

Contact: Jennifer Page
202-835-3460

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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 $44.5 billion in 2007 in discovering and developing new medicines. Industry-wide research and investment reached a record $58.8 billion in 2007.

PhRMA Internet Address: www.phrma.org

For information on stories of hope and survival, visit: http://sharingmiracles.com/

PhRMA en Español: www.nuestraphrma.org

For information on how innovative medicines save lives, visit: www.innovation.org

For information on the Partnership for Prescription Assistance, visit: www.pparx.org

For information on the danger of imported drugs, visit: www.buysafedrugs.info