FDA’s Broken Drug Approval System Continues Hurting Patients

Published February 12, 2020

Innovative developments in unapproved medical therapies are paving the way for the new frontier and the future of medicine. One patient in particular, Judi Polak, a former medical professional herself, is an example who puts into question the endpoints required by the gold standard Food and Drug Administration (FDA) drug approval process.

FDA determines the standards of two categories for drug approval: safety and efficacy. All new drugs coming to market must be put under rigorous testing to meet FDA standards, yet these standards vary from drug to drug, and therefore, there are often no objective standards that apply to the drug approval process. One might ask how we would know if a drug ought to be approved or not if the standards by which they are measured constantly vary?

Judi’s story is an example that begs this very question.

Judi spent her life serving others and taking care of patients as a nurse at West Virginia University (WVU) Children’s Hospital Neonatal Intensive Care Unit. She was diagnosed with early onset Alzheimer’s at 51 years old, about six years ago. The devastating news of being diagnosed with an incurable disease like Alzheimer’s was fortunately met with the opportunity to enroll in a clinical trial through West Virginia University Rockefeller Neuroscience Institute, led by Dr. Ali R. Rezai, a neurosurgeon at WVU. Results from this trial have been promising for Judi, and her condition has drastically improved.

Yet, the FDA drug approval process is running on an antiquated system that requires, on average, 12 years and $2.9 billion to bring a drug from lab to patients. Even though there are instances in which patients undergo unapproved therapies through clinical trials and see success, and with the FDA’s process, there are often lengthy and costly waiting periods that leave many patients not enrolled in clinical trials patients with few or no options.

The main issue with these testing standards is that there is no definition of value, meaning there is no single objective standard. Thus, the process by which drugs and therapies are approved varies between disease groups, payers, and patients. With the transformative nature of medicine and the ability of medical innovation to continue to develop, there is a growing need for reform of the current approval process of life-saving medical treatments. The Heartland Institute has a solution called, Free to Choose Medicine which would apply free market principles to the current drug approval process. Allowing patients to have access to therapies before they have received the gold stamp of approval from the FDA would bring drugs to market sooner, and additionally lower cost by saving time and money on unnecessary efficacy testing.

The goal of FDA should be to allow good drugs to succeed sooner, and bad drugs fail faster. There is no question that a means testing regime ought to be in place, but by what standards and who makes that determination is what must be reexamined and changed.