The development of new therapies, for the past several decades, has followed a very clear, step-wise progression. First, there are phase 1 trials to establish a medication’s safety and dose. Next, phase 2 trials explore which cancers are appropriate for the medication. After that, phase 3 trials compare the treatment to existing treatment with the goal of earning FDA approval.
Recently, this timeline seemed to be turned on its ear. Back in 2011, the medication that would later be named Keytruda went through a different process called a first-in-human trial (as opposed to the phase 1, 2, 3 process) since it showed high potential value for cancers that had virtually no other treatment options. This expedited process allowed this beneficial medication to be used by patients (saving some of their lives) years before it could have been used if the standard process was followed.
Keytruda became a model of success. Currently there are at least 40 first-in-human oncology trials underway. This is potentially very good news for cancer patients. But it does need to be noted that not every medication – even if it shows early, positive results – will prove itself to be a therapeutic advance. New doesn’t always mean better. So while any cancer-fighting advances in the oncology pipeline are certainly welcome, health care providers will continue to provide the best options for each patient at the current time. It’s a dual process of hope and pragmatism.
Prowell TM, Theoret MR, Pazdur R. Seamless oncology-drug development. New Engl J Med 2016;374:2001-3.
Balasubramaniam S, Buzdar A, Flaherty K, et al. The blurring of phase 1, 2, and 3 trials in oncology: Expansion cohorts in phase 1 trials. Conf Clin Cancer Res November 2015.
Stephen C. Vogt, Pharm.D.
President and CEO
BioPlus Specialty Pharmacy
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