Phase-Appropriate Drug Development – Validation Process

By Dr. Thomas S. Ingallinera, R.Ph., Ph.D., Vice President Technical Support

The drug development process is among the most complex, costly, and regulated of human pursuits and the statistical chances of success are horribly low. According to Derek Lowe’s blog, “In The Pipeline”, published by Science Translational Medicine, the chances of success for a drug compound entering Phase 1 trial has remained at slightly under 10% for the last two decades. Mr. Lowe challenges us to consider any other major business category that operates with such a high expectation for failure.

According to an MIT study, of those drugs that enter clinical trials, nearly 14% will ultimately gain FDA approval. Vaccines perform significantly better with a 33.4% chance of success, but investigational oncology is much lower at 3.4%.  Despite these dismal chances for success, there is some hopeful news. I know what you’re thinking, please get to the good news?

The study further found that clinical trials employing companion diagnostics tended to be more successful than those that did not. As a deeper understanding of patient populations continues to advance, fueled by healthcare data technology, the chances of success are expected to get better. When we couple this trend in patient disease knowledge with a disciplined phase-appropriate process for drug development, a more cost effective, success-oriented model emerges.  Let’s consider phase-appropriate development by applying it to process validation.