Report

The Biopharmaceutical Pipeline

Innovative Therapies in Clinical Development

PhRMA July 17, 2017

The biopharmaceutical pipeline contains thousands of significant and innovative new treatments with the potential to address unmet medical needs, save lives and improve patients’ health. A new report by the Analysis Group, “The Biopharmaceutical Pipeline: Innovative Therapies in Clinical Development,” examines the state of the drug development pipeline and provides insights into new approaches researchers are pursuing.

Key findings: 

  • 74 percent of medicines in clinical development are potentially first-in-class medicines, meaning they represent a possible new pharmacological class for treating a medical condition.
  • 822 projects – defined as unique molecule-indication combinations – are designated by the U.S. Food and Drug Administration (FDA) as orphan drugs, which is critically important given only 5 percent of rare diseases have an approved medicine.
  • A range of novel scientific approaches are being pursued, including cell and gene therapies, DNA and RNA therapeutics and conjugated monoclonal antibodies.

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