Clinical Trials Explained for Participants

What a trial is and is not

• A trial tests whether a product is safe and effective for a stated use. It may involve a placebo or an approved comparison treatment; see Randomization, Blinding and Placebo.
• Some trials need healthy volunteers to help answer research questions. [1]
• A listing in ClinicalTrials.gov, or a firm's registration with the FDA, does not mean a product is legally marketed. [2] A listing also says nothing about whether the treatment works. See Registered Trials vs Published Results.
• You can look for studies at ClinicalTrials.gov, and the FDA suggests talking to your health care provider. [1]

What the phases mean for you

The FDA describes the usual sequence. [3]

Before a trial starts, the sponsor submits an IND and must wait 30 calendar days before starting. [4] The FDA can also place a clinical hold to delay or stop an investigation. [3] More detail: Clinical-Trial Phases Explained.

Informed consent: what you should be told

You should have the study explained to you in an informed consent process before you agree to join. [1] Under U.S. rule 21 CFR 50.25, the information given to each participant includes: [5]

• That the study involves research, its purpose, how long you would take part, and the procedures, including any that are experimental.
• Any reasonably foreseeable risks or discomforts.
• Any benefits to you or others that may reasonably be expected.
• Alternative procedures or courses of treatment that might be advantageous to you.
• How confidentiality of your records is handled, and that the FDA may inspect the records.
• For research with more than minimal risk, whether compensation or medical treatment is available if you are injured.
• Whom to contact with questions about the research and your rights, and about a research-related injury.
• That taking part is voluntary, and that refusing involves no penalty or loss of benefits to which you are otherwise entitled. [5]

When appropriate, you should also be told that a treatment may involve currently unforeseeable risks, and about any additional costs to you. [5] The consent form also identifies the Institutional Review Board (IRB), the group that assures protection of participants' rights and welfare. [6] For more, read Informed Consent in Emerging Treatments.

Questions worth asking

• What is being tested, and what stage is it?
• What happens if I get a placebo or a comparison treatment?
• What are the known and unknown risks, and who treats me if something goes wrong?
• Are there approved or other options I should compare?
• What will it cost me, and who pays for study-related care?
• Who do I call with concerns, and what happens if I leave?

Why representation matters

The FDA says people from underserved communities are underrepresented in clinical research, and that people of different ages, races and ethnicities may react differently to medical products. [1]

Red flags

Being charged for an unapproved product, or told a "trial" is a way to buy a treatment, calls for caution. The FDA says that for regenerative medicine products offered outside a clinical trial, being charged means you are likely being deceived and offered a product illegally. [2] See Research Participation and Therapeutic Misconception. To report a reaction to an FDA-regulated product, see Adverse-Event Reporting for Emerging Products.