Clinical Trials & Research

Clinical Trials & Clinical Research

Medical research is essential for developing new diagnostic tests and treatments to improve the lives of people with rare diseases. Many people living with rare diseases have an interest in advancing research for their disease through participation in clinical trials and clinical research.

Clinical trials are a type of clinical research designed to evaluate and test new interventions, such as medications or medical devices, using human volunteers who meet specific study criteria.

A clinical research study is any type of clinical research involving people, regardless of whether it is testing a specific intervention; clinical studies might investigate other aspects of care, such as improving quality of life.

Great advances are being made in understanding the basis for many rare diseases. This has the potential to generate new approaches for treatment. In some cases, a drug may be tested that is already approved by the FDA for some other indication. New information may suggest that it also could be helpful for people with a rare disorder.

Physicians can prescribe an FDA-approved drug for reasons other than those for which it was initially approved. This is referred to as “off-label use.” In such cases, it is unknown whether the drug will work or whether side effects may occur; however, the physician and patient may judge that the risks are worth taking to see if there may be a benefit. Sometimes, new knowledge about the biological basis for a rare disorder might lead to the development of entirely new drugs that have never been used to treat any disease.

Clinical trials can be conducted with existing FDA-approved drugs to see if they are safe and effective in treating a rare disease. Clinical trials are mandatory if FDA approval is being sought for a brand new drug.

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