Discovery vs Clinical Application

Two different questions

Discovery asks: what happens in a cell, a tissue or an animal? Clinical application asks: does a specific product, device or procedure help people with a specific condition, and at what risk? A finding can answer the first question without coming close to answering the second. This page lays out the path between them. It is information only, not medical advice.

What "translational research" means

The National Cancer Institute describes translational research as the process by which results of laboratory research are used to develop new ways to diagnose and treat disease. [1] It is often summed up as moving "from bench to bedside." One review describes medicine's progress as depending on moving basic science from the bench to clinical use, and notes that this has become harder in practice. [2] Translation is work, with many places to fail.

The stages, in order

1. Discovery. The FDA says researchers find possible drugs through new insight into disease, large tests of molecular compounds, unexpected effects of existing treatments, or new technologies. At this point thousands of compounds may be candidates, but after early testing only a small number look promising. [3]
2. Development. For a promising compound, researchers study how it is absorbed and cleared, its potential benefits and mechanisms of action, side effects, and how it compares with similar drugs. [3]
3. Preclinical research. Before testing in people, researchers must learn whether a drug could cause serious harm, using in vitro (laboratory) and in vivo (animal) work. [4]
4. Clinical research. Studies in people begin only after the developer submits an Investigational New Drug application that includes animal and toxicity data. [5]
5. Review. A developer can file an application that must show the drug is safe and effective for its intended use in the population studied. [6]
6. After approval. Phase 4 studies look at side effects over time in people using the treatment, which can reveal problems not seen earlier. [7]

The phases of human testing are explained in clinical-trial phases explained.

Where wellness claims skip steps

• Straight from the lab to the shelf. A cell result is described as if it were a benefit. Cell and animal findings are labeled preclinical (cells/animals only) here and are never carried over to people. In one review, corticosteroids helped in animal models of head injury but not in trials, and tirilazad improved animal stroke outcomes but was associated with a worse outcome in patients. [8]
• Plausible reasoning treated as proof. See mechanism vs demonstrated benefit.
• A registered trial or a clinic treated as approval. Starting a study shows only that one was planned. Dietary supplements are not reviewed this way: the FDA says it does not have the authority to approve dietary supplements before they are marketed. [9]
• A lab marker treated as a health result. NCI notes that surrogate endpoints are not always true indicators of how well a treatment works. [10]

Why many findings stop along the way

Most candidates are filtered out. The FDA reports that approximately 70% of drugs move from Phase 1 to Phase 2, approximately 33% from Phase 2 to Phase 3, and approximately 25 to 30% from Phase 3 onward. [5] The reasons vary: unexpected harm, no benefit in people, or results that do not hold up. For a broader view, see why promising findings sometimes fail.

Rules differ by product type

The FDA path above describes drugs. Other products are handled differently. Dietary supplements are not approved before marketing in the United States. [9] The NIA notes that trials testing a behavior change advance through similar steps but are not regulated by the FDA. [11] We name the product, country and regulator whenever a page makes a regulatory statement. To place a topic, ask which stage it has actually reached, then check the label on its page, starting with how we label evidence.