From Research Studies to Your Pharmacy Shelves: The Drug Development Process

When you’re feeling sick and your doctor prescribes you medication, you’re likely not thinking about everything that happened before that drug got into your hands. But the fact is that on average, it takes at least ten years for a new medicine to make it to market — from initial discovery to your local pharmacy. Of those ten years of drug development, the treatment is making its way through clinical trials for six to seven of them.

It’s important to understand all that goes into developing a drug in order to appreciate the role that patients play in helping bring drugs to market, so that future patients can benefit from new and better treatments.

The FDA outlines five steps that all drugs go through before making it to the public:

1. Discovery and Development: This is the stage wherein researchers first discover a new drug — through new insights into a disease, new tests, unexpected side effects of other medications, or new technology. There may be thousands of potential treatments or therapies discovered, but only a handful make it to the development stage, in which researchers begin initial tests on new compounds. While patients are not directly involved in this step, research is driven by patient need for new and better treatment options.

2. Preclinical Research: Of course, extensive testing needs to happen before a potential treatment can be administered to humans (even in a clinical trial). Preclinical research involves studying a potential drug both in test tubes and in living things, such as animals. These studies are small but conclude with detailed information on toxicity and dosing. Researchers then look carefully at this information to determine whether or not to move on to testing the drug in humans.

3. Clinical Research: Here is where the drug will be studied in people through clinical trials. At the onset of a trial, researchers design the study, mapping out how the study will be run and desired outcomes for each phase of the trial, and begin the Investigational New Drug Process (IND), which the FDA requires before any new compound is tested in humans. Clinical research includes all the phases of a clinical trial.

Some would-be clinical trial participants are concerned about the risks of taking part. Risks can include side effects, or that the potential drug doesn’t work for you. Those concerned about risks may feel more comfortable participating in a later-stage clinical trial. Phase 1 trials are conducted with just 20 to 100 volunteers, and test for safety, not effectiveness. These trials are the most likely ones to be paid clinical trials. Next, Phase 2 trials enroll several hundred volunteers to test a treatment for both safety and effectiveness. If the drug is found effective in Phase 2 trials, it moves to Phase 3, which needs several hundred to several thousand volunteers to take part.

4. FDA Review: Once a treatment moves successfully through all clinical trial stages and researchers believe it’s safe and effective, sponsors — the pharmaceutical companies or research organizations who ran the trials  submit a New Drug Application (NDA) to the FDA for approval. The NDA should demonstrate safety and efficacy, and include all data obtained to-date for review. It must also include things like proposed labeling, patent information, and directions for use. The FDA review team decides if the NDA application is complete, and if so, the FDA has six to 10 months to decide whether or not to approve the drug. The application is reviewed carefully, FDA inspectors visit clinical study sites, and a project manager pulls all of the information together. The review team makes a recommendation about approval, and a senior FDA official makes the final decision.

Once a drug is approved, the FDA will work with researchers to make sure the labeling is correct, and to schedule something called an FDA Advisory Committee meeting. This is a meeting to discuss additional considerations that may have come up during review. At an Advisory Committee meeting, researchers, patients, and advocacy groups are invited to weigh in on any remaining issues around the drug or its indication.

5. FDA Post-Market Safety Monitoring: The FDA’s work, and patient involvement, doesn’t end when a drug is approved. After a treatment hits the market, the organization continues to monitor the safety and efficacy of the drug. It may be that a drug interacts unexpectedly with an over-the-counter medication, that patients find the labeling confusing, or that a drug advertisement doesn’t offer enough side effect information. The FDA watches all of this and requires the research organization responsible for the drug to make corrections on an ongoing basis.

As you can see, patients are at the heart of the drug development process. Is the drug safe for them? Will it work to cure an illness or alleviate symptoms? But it takes patients participating in research to answer these questions and find new and better treatments for the future. If you’d like to be a patient who helps uncover the treatments of tomorrow, find your clinical trial match below.