The Food and Drug Administration (FDA) is a federal agency that acts to protect public health across the United States by strictly ensuring investigational drugs, vaccines, biological products, and medical devices are safe, secure, and effective. Although exact success rates may differ depending on the disease indication, the overall chances of a product being granted FDA approval was estimated to be about 13.8% as of 2018. The entire journey of a therapeutic product from discovery to approval typically takes years to complete and requires millions of dollars in investment by the sponsor company.
Despite the complexities of this pipeline, we’ve examined and highlighted the five most important stages from start to finish. Read on to learn more about the path a new drug must take to get from the laboratory to a patient’s medicine cabinet.
Drug Discovery and Development
New drugs can be discovered in any number of ways. For example, our understanding of a disease or a piece of technology has expanded, and recent advances can help drive novel product designs. Regardless of the exact method with which a drug candidate is found, its development can involve extensive experiments on its mechanism of action, potential side effects, drug-drug interactions, and potential genetic differences in treatment response. At this point, early testing can usually narrow down potential therapies from numbering in the thousands to only a small handful.
Animal Research: Preclinical Studies
Although preclinical studies on animal subjects don’t tend to be too large, these results are often the basis for receiving approval to move to testing in human subjects. The most important investigations will thoroughly test the compound’s toxicity profile, the relationship between dosing and body weight, the impact of food consumption, the drug’s clinical pathology, and more.
Human Research: Phase I-III Clinical Trials
Despite the valuable information preclinical studies provide, most drug candidates will eventually have to undergo testing in humans to determine the most accurate performance data. Before any clinical trial can begin, sponsors must submit an Investigational New Drug (IND) application, which includes their animal efficacy and toxicity data, manufacturing information, study plans, and more. Within 30 days of submission, the FDA IND review team can choose either to approve the application or, in rare cases, place a clinical hold if further clarifications are required.
However, once approved, a new drug will be researched in humans at least through Phase I, Phase II, and Phase III clinical trials before the data can be presented to the FDA to potentially receive market approval.
- Phase I studies serve to demonstrate preliminary human dosage and safety data, typically in healthy volunteers, but may instead include individuals within the disease population in cases like oncology drugs. About 70% of new drugs will successfully make it past this stage.
- Phase II studies can include several hundred subjects with the disease indication, and their purpose is to review the efficacy, as well as identify adverse events. From here, 33% of drugs are estimated to reach Phase III testing.
- Phase III studies continue to investigate drug efficacy and side effects within the target population, often lasting several years in duration. Approximately 25% to 30% of novel drugs will move to the next stage.
FDA Review and Approval
Traditionally, clinical trials must reach at least Phase III testing in humans before a New Drug Application (NDA) can be submitted to the FDA prior to approval. After ensuring this application is complete, the FDA NDA review team will typically deliberate between 6 to 10 months. After a thorough assessment of all available data, approval of a new drug will be granted if the expert review panel finds sufficient evidence of its safety and efficacy. Following this, the FDA will continue to work with the sponsor to refine the medication’s labeling information.
What Comes After FDA Approval?
As important as clinical trials are for providing vital safety and efficacy data on a medication, they don’t provide a complete picture. Even after a medication hits the market, the FDA continues to monitor reports of any adverse event patterns from the general population. Over the period of a drug’s lifetime after approval, post-market surveillance, in part through Phase IV studies, provides more reliable long-term data on its safety and efficacy.
Although the path to FDA approval is a daunting one with no shortage of complexities to overcome, Vial CRO’s next-generation clinical trial services and our team of expert leadership are here to guide your study to its full potential. Visit our website to discover how our CRO services and products are delivering faster, more efficient trials at dramatically lower costs for sponsors. Connect with a team member today!
