New Report Examines the Biopharmaceutical Pipeline and Innovation in Clinical Development

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Report notes that 74% of drugs that are currently in development are potentially first-in-class medicines, and 822 drugs in development are for rare diseases.

A new report by the Analysis Group, “The Biopharmaceutical Pipeline: Innovative Therapies in Clinical Development,” analyzed the drug developments and new techniques researchers are utilizing to discover new advancements in pharmaceuticals.

The report emphasized that 74% of medicines that are currently in development are potentially first-in-class medicines, meaning they present new treatment options than previously developed in their class and often provide greater efficacy with fewer adverse effects. These medicines are apparent in all stages of clinical development, but are less frequent in later stages due to the high levels of uncertainty.


The authors also found that 822 orphan drugs, designated by the FDA, are currently in clinical development or regulatory review to eventually treat a variety of rare diseases that can now be identified with new scientific advancements. The pipeline includes the orphan drug development in the categories of cancer, cardiovascular, immunology, infections, musculoskeletal, and neurology.

New scientific approaches and strategies are also being used in order to develop new and effective treatments. Four strategies demonstrated in the report include:

  1. Cell therapy in 529 projects that utilizes healthy cells to treat damaged or diseased cells.
  2. Gene therapy in 202 projects that modifies a patient’s genes to prevent or treat a disease.
  3. The use of DNA or RNA therapeutics in 173 projects, intending to target RNA and DNA rather than the direct proteins.
  4. The use of 188 conjugated monoclonal antibodies that combine with cytotoxic agents to attack tumor cells.

“RNA-based therapeutics […] have great potential to target a large part of the currently undruggable genes and gene products and to generate entirely new therapeutic paradigms in disease, ranging from cancer to pandemic influenza to Alzheimer’s disease,” stated Steven Dowdy of Nature Biotechnology.

The report overall emphasized the innovation and advancement in biopharmaceuticals that can help improve and potentially save patient lives.

“The continued discovery and development of newer, better treatments saves and improves lives,” concluded the report. “As the rapid pace of scientific and technological advances is propelling a new era in biopharmaceutical innovation, it is critical that we ensure a policy and regulatory system that continues to foster the substantial investments needed to support medical advances.”