Clinical Research

"My son Brian has been involved in CF clinical trials literally from the week he was diagnosed. Now that he is 18, it's his decision whether or not to volunteer. As he says, 'If not me, who?'" Terry, Brian's mother

Opportunities to help develop new drugs from cystic fibrosis research have never been more promising.

More potential therapies are in the CF drug development pipeline today than in the entire history of cystic fibrosis research.

While that gives all of us great cause for hope, it also charges us with recruiting more people than ever before to help us test new drugs.

That’s the only way we’ll be able to get them on their way to the children and adults who desperately need them.

Participants in clinical studies and trials are the key to new treatments and a cure. Research cannot move forward without them.

In this section, you’ll learn more about clinical research, including:

• Why it’s so important to participate in clinical studies and trials;
• The types, phases and sponsors of clinical studies and trials; and
• What a participant in a clinical study or trial can expect.

What’s the difference between clinical research, studies and trials?

• Research is a general term used to describe efforts to explore and understand things that are not clear to us.

• A clinical study is a type of research—also called observational research—in which participants are observed. Drugs are not used. For example, CF clinical studies usually explore ideas about CF and the disease process.
  
  While they don’t test drugs or treatments, they are very important for developing new ideas about cystic fibrosis and how it might be treated.
  
• A clinical trial is a type of research—also called interventional research—that follows certain guidelines set up by the government to allow drugs or treatments to be tested with people.
  
  Researchers see how a drug works in the body under highly controlled conditions and whether a treatment is helpful.

What are the various types of research?

• Treatment trials test experimental drugs, new combinations of drugs, or new ways to use surgery or radiation therapy.
  
  
• Prevention studies look for better ways to prevent disease in people who’ve never had a disease or to prevent a disease from returning. They may include medicines, vitamins, vaccines, minerals, or lifestyle changes.
  
  
• Diagnostic studies try to find better tests or procedures for detecting certain diseases or health conditions.
  
  
• Screening studies test the best ways to detect certain diseases or health conditions.
  
  
• Quality of Life studies (or Supportive Care studies) explore ways to improve the comfort and quality of life for people with a chronic (continuing) illness.

Who sponsors clinical research?

Clinical research can be sponsored in part or entirely by any number of organizations or individuals. For example, medical institutions, universities, foundations, voluntary groups, drug companies, and federal agencies, such as the National Institutes of Health (NIH), all sponsor research.

The sponsor chooses doctors, called principal investigators, to run the studies or trials. Study-related medical care is often provided to the patient at no cost.

The Cystic Fibrosis Foundation is the primary sponsor of CF research. Almost all of the approved CF drug treatments available today were made possible because of research funded by the CF Foundation.

Why do people participate in clinical trials and studies?

Participating in a clinical trial or study can be a very satisfying and worthwhile experience. Click here to learn more about participating in clinical trials and studies.

back to top