The trial sponsor, often the pharmaceutical company that develops the therapy or medication, prepares the protocol for the clinical trial. A protocol is a plan that explains
how the trial will work, what will be done during the trial, and why. Each medical center that conducts the trial uses the same protocol.
Key information in a protocol includes:
- How many patients will participate
- Who is eligible
- What tests patients will get and how often
- What type of data will be collected
- Detailed information about the treatment plan, if appropriate
Study-related medical care is often provided to the patient at no cost.
Clinical researchers work to ensure that they avoid bias in clinical trials. Bias refers to human choices or other factors (unrelated to the protocol) that might affect the trial's results. For example, if doctors could choose which patients to assign
to comparison groups in a study, some might assign sicker patients to the treatment group and healthier patients to the control group (not receiving treatment). The doctors might not even realize they are doing this, and it could affect trial results.
Randomization helps ensure that researchers don't introduce bias into the trial. In many clinical trials that test the effectiveness of a medication, half of the participants receive the medication in question. The other half receive a placebo, which
contains no medication. Randomization involves assigning patients to these comparison groups by chance, rather than choice.
Researchers also may use “blinding” to help avoid bias. In a blinded trial, researchers will not know which patients are receiving treatment and which ones are receiving a placebo.
The Four Phases of Clinical Research
For any new drug to receive approval by the U.S. Food and Drug Administration (FDA) and become available to the public, it must pass through three phases
of interventional clinical trials to show that it is safe and effective in treating the disease. If the FDA approves the drug, it will continue to be monitored for safety and effectiveness in what is known as a Phase 4 study.
The infographic below shows a breakdown of the questions that researchers try to answer, the number of participants needed, and the time per participant for each phase of research.
Please note that the length of time refers to the time it takes to participate in a trial, not the entire length of the phase. It takes time to enroll participants and to process
and analyze the results of each phase. All told, it typically takes 10 to 14 years from the time a drug is discovered in a laboratory to its possible approval by the FDA for people with CF.
Who Can Participate?
All clinical trials have guidelines about who can join. Some enroll healthy people. Others enroll only people with certain conditions, such as CF.
A CF clinical trial may have other guidelines in addition to requiring that the study volunteer has CF. These guidelines are known as inclusion criteria and exclusion criteria, and they ensure that the research results are reliable and help to reduce
the risk for those enrolling in the trial.
Inclusion criteria and exclusion criteria refer to the standards used to decide whether a person may or may not enroll in a clinical trial. Criteria are based on such factors as age, gender, disease, previous treatment history, and other medical conditions.
The criteria depend on the type of trial. For example, the age that the person with CF must be to participate varies according to each trial because drugs work differently in young children than they do in adults. Before a drug can be tested in young
children, it must be shown to be safe and effective in adults with CF.
If researchers are testing how well a particular antibiotic works in fighting Pseudomonas aeruginosa, then the trial would have inclusion criteria
specifying that only people with CF who are infected with that bacterium can join the trial. Those who do not have that bacterial infection would be excluded from the trial.
We
call people with CF who participate in clinical trials, and all those who
support them, trailblazers. Help us
blaze a trail to better treatments and a cure for CF.
Who Sponsors Clinical Research?
Clinical research can be sponsored (i.e. paid for) 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 principal investigators to run the trials. Study-related medical care is often provided to the participant at no cost.
The Cystic Fibrosis Foundation is the primary supporter of CF research in the United States. Nearly all approved CF therapies available today were made possible because of research funded by the CF Foundation.
The Foundation facilitates and financially supports clinical trials through its
Therapeutics Development Network (TDN), which is made up of CF care centers with an expertise
in clinical research.