Seventy-four percent of medicines in clinical development around the world are potentially first-in-class medicines, meaning they use a completely new approach to fighting a disease, according to a new report by The Analysis Group, commissioned by the Pharmaceutical Research and Manufacturers of America (PhRMA). The Biopharmaceutical Pipeline: Innovative Therapies in Clinical Development report examines the global state of the drug development pipeline and provides insights into new approaches biopharmaceutical researchers are pursuing.
The pipeline report, which updates a previous analysis, highlights new treatment approaches such as cell therapy and gene therapy, as well as DNA or RNA therapeutics and conjugated monoclonal antibodies. 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).
"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."
The report also found that 822 projects in clinical development are designated by the FDA as orphan drugs, which is critically important given only 5% of rare diseases have an approved medicine.