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Fact Sheets

DID YOU KNOW?

America’s Pharmaceutical Research and Biotechnology Companies are Helping Patients Conquer Rare Diseases

DID YOU KNOW THAT THERE ARE 6,000 KNOWN RARE DISEASES AFFECTING NEARLY 30 MILLION AMERICANS?
  • A rare disease is defined as a condition affecting fewer than 200,000 patients in the United States.
  • One in every 10 Americans receives a diagnosis of a rare disease, according to the National Institutes of Health.
  • The Food and Drug Administration (FDA) estimates that 85-90 percent of rare diseases are life-threatening.

DID YOU KNOW THAT THERE ARE MORE THAN 300 MEDICINES IN DEVELOPMENT TO HELP TREAT OR PREVENT RARE DISEASE?
  • Advances in science, such as better understanding of molecular and genetic causes of disease, have given researchers important new insights to better tackle rare diseases, which are often more complex than other diseases.
  • Between 1995 and 2005, more than 160 medicines were approved by the FDA, compared to 108 in the decade before and fewer than 10 in the 1970s.
  • Today, with more than 300 medicines in human clinical trials or awaiting approval by the FDA, it is clear that significant progress is being made to bring new therapies to patients.

DID YOU KNOW THAT AMAZING THERAPEUTIC ADVANCES HAVE BEEN MADE IN THE AREA OF RARE DISEASES?

Some examples include:
  • The first treatment for ALS or Lou Gehrig’s disease.
  • Five new treatments for pulmonary hypertension.
  • A genetically engineered antibody that is the first treatment for Crohn’s disease.
  • The first medicine that treats the cause of Fabry disease, rather than its symptoms.
  • The first in a new class of medicines to treat acromegaly, a disorder in which excess growth hormone causes enlarged hands, feet and facial features.

DID YOU KNOW THAT SINCE THE ORPHAN DRUG ACT WAS FIRST PASSED IN 1983, 325 ORPHAN DRUGS HAVE RECEIVED FDA APPROVAL?
  • The Orphan Drug Act of 1983 provided tax relief and some market exclusivity to provide incentives for innovators to research and develop orphan drugs.
  • In the 1970s – before the Orphan Drug Act passed – fewer than 10 orphan drugs were approved. 
  • The dramatic increase in the number of medicines approved for rare diseases after the Orphan Drug Act passed shows the great impact of the legislation on the lives of patients.

DID YOU KNOW THAT IT COSTS MORE THAN $1 BILLION, ON AVERAGE, TO DEVELOP A NEW MEDICINE?
  • It is a long, risky and costly process to develop a new medicine. Besides costing more than $1 billion, on average, to develop a new medicine, it can take anywhere from 10 to 15 years to get final FDA approval.
  • Most of the compounds investigated as potential new medicines never make it out of the lab. Only one of every 5,000-10,000 potential compounds investigated are ever approved for use as a medicine.
  • In short, the odds of a medicine ever making it out of the lab is daunting but the risk is worth taking because America’s pharmaceutical research companies operate in an environment that encourages investment, innovation and risk-taking.

DID YOU KNOW THAT THERE IS HELP OUT THERE FOR UNINSURED AND FINANCIALLY-STRUGGLING PATIENTS WHO HAVE TROUBLE ACCESSING MEDICINES?
  • The Partnership for Prescription Assistance (PPA) is a clearinghouse of information on more than 475 public and private patient assistance programs that can help provide eligible uninsured and financially-struggling patients with free or nearly-free medicines.
  • Spearheaded in 2005 by America’s pharmaceutical research companies, PPA has helped more than 5 million patients nationwide.
  • There are many medicines that help treat patients suffering from rare diseases available through PPA. 
  • For more information about PPA, please call 1-888-4PPA-NOW or log online at www.pparx.org.