A new report by the Analysis Group, shows that 74 percent of medicines currently in clinical development are potentially first-in-class, meaning that they represent a completely new approach to fighting disease. The report, Biopharmaceutical Pipeline: Innovative Therapies in Clinical Development, examines the state of the drug development pipeline, providing insights into the new approaches that researchers are actively exploring.
Today’s pipeline of new medicines is vibrant, diverse and incredibly promising for patients. The analysis found that there are more than 4,000 projects – defined as unique molecule-indication combinations – in clinical development for oncology, 450 for cardiovascular disease and 700 for neurology, including 143 in Alzheimer’s disease, 67 in Parkinson’s disease and 29 in amyotrophic lateral sclerosis (ALS).
The report also found that 822 projects currently in development have been designated as orphan drugs by the U.S. Food and Drug Administration (FDA). This is critically important as there are about 7,000 rare diseases and only 5 percent have an FDA approved medicine.
Driving this intensive level of discovery is an array of emerging scientific approaches, including cell therapy where healthy, function cells are used to treat a disease or condition or gene therapy where patient’s genes are modified to treat or prevent a disease.
"We are in a new era of medicine where breakthrough science is transforming patient care and enabling us to find treatments for the world’s biggest health care challenges," said Stephen J. Ubl, president and CEO of PhRMA. "And as this new report underscores, we’re on the cusp of even more incredible breakthroughs that could further revolutionize how we prevent, treat and even cure disease for patients."
For more information, view the report synopsis and download the full report.