Many Americans don’t realize that it can take up to 15 years for a drug they’re prescribed to go from initial discovery to the market. This lengthy process involves several phases to ensure the drug product is not only safe but also effective. Due to this rigorous criterion, only 10% of drugs in development will advance and become a drug on the market.
All chemical compounds developed by researchers for medical treatment must go through clinical trials. These trials are overseen by the Federal Drug Administration (FDA) to ensure ethical and outstanding laboratory practices are followed. These regulations set the minimum requirements for conduct, facilities, equipment, protocols and procedures and reporting. Each trial must move through all phases in order and cannot advance until the previous steps have been completed.
Pre-clinical trials: Ideation
The journey begins when scientists hypothesize an idea for a molecule with potential medical use. If this molecule shows potential, researchers must find a way to synthesize and purify it in preparation for pre-clinical trials where they’ll use cell or animal models. Pre-clinical trials test for dangerous effects, or toxicity, and dosing strategies to confirm it will not be harmful to humans.
Phase I: Drug safety
If the pre-clinical trials do not show any signs of toxicity, the substance, or drug, proceeds to phase I. This phase focuses on the safety of the drug in humans as well as appropriate dosing and possible side effects. Phase I typically has fewer than 100 participants that are healthy, do not have the disease state of interest and can participate in the trial for multiple months.
Phase II: Efficacy
Once phase I is complete, the drug moves onto phase II. Phase II consists of around 300 participants, conducting tests to determine if the drug will be effective for the intended disease state. This process can last upwards of a year. It is important to note that many trials end after phase I or II. This can happen for many reasons, with the most common being lack of funding, inconclusive results or a drug’s failure to show safety and efficacy.
Phase III: Large scale studies
Once the drug has gone through phase I and II with favorable results, phase III begins. Phase III is a large-scale study, including thousands of patients, with the goal of confirming the previous trials’ results. Multiple phase III trials can be conducted on the same drug to confirm safety and efficacy while comparing the drug to treatments currently used in the intended disease state. Phase III trials can last for years, especially if the drug is intended for chronic conditions like heart disease or diabetes.
FDA approval: Availability to providers and patients
After all phases are complete, the data, results and methods are sent to the FDA for review and approval. The FDA will assess the data and determine the risk versus the benefit of the drug. If the FDA deems the drug has significant benefits that outweigh any potential risk, it is approved. Once approved, manufacturing of the product begins, followed by access to prescribers and patients.
Phase IV and beyond: Ongoing monitoring
However, approval is not the end of the journey. Once the drug product begins to be prescribed and used by the public, phase IV begins. Phase IV is the ongoing monitoring of the drug and provides real world data and evidence.
Adverse effects of the drug can be reported to MedWatch, an FDA reporting program that is available to medical professionals, patients and consumers. To report an adverse event, you can access MedWatch here. If there are multiple serious adverse events reported, the FDA can conduct a review of the drug and can subsequently pull the drug off the market if it is found to be unsafe in the real world.
If you have any questions regarding new drugs or drugs currently in the clinical trial process, contact the Employers Health clinical team at [email protected].
