Clinical Trials, Explained for Patients and Caregivers
14 plain-language guides in 4 parts, from what a trial is to placebos, costs, your rights and getting a drug outside a trial. Written for families living with a rare disease.
Start with your question
Tap a question for the short answer. Each one links to its full guide.
What is a clinical trial?
A research study that tests a medical treatment, device or approach in people, following a written plan that an ethics board called an IRB has reviewed. Most US trials are listed on ClinicalTrials.gov. Joining is voluntary, and you can leave at any time.
Read the full guide →Will I get a placebo?
Only if the trial has a placebo group, and the consent form has to tell you that before you join. Some trials give the placebo on top of standard care, so no one gives up their usual treatment, and some switch everyone to the study drug after a set period. Some trials use no placebo at all.
Read the full guide →Do clinical trials cost money?
The study drug is usually free, and many trials pay for study-only visits and tests, so ask for the details in writing. Most private health plans must cover your routine care during an approved trial for cancer or another life-threatening condition under the Affordable Care Act, and Medicaid must cover routine care in qualifying trials in every state since January 1, 2022. Some trials also repay travel costs.
Read the full guide →Can I quit a clinical trial?
Yes, at any time and for any reason. Federal rules protect that right, 21 CFR 50.25 for FDA-regulated trials and 45 CFR 46 for federally funded research, and leaving cannot cost you benefits you are otherwise entitled to. Some drugs should be stopped gradually, so tell the study team before you stop.
Read the full guide →How do I join a clinical trial?
Find studies for your condition, contact the site coordinator listed on the study, go through screening to confirm you qualify, then read and sign the informed consent form. Trial Friend's Match Me tool checks every open trial for a condition against your age, location and treatment history.
Read the full guide →What are the phases of a clinical trial?
Phase 1 tests safety and dosing, usually in 20 to 100 people. Phase 2 looks for early signs that the drug works, in up to several hundred. Phase 3 confirms the benefit and watches for side effects in 300 to 3,000 people, according to the FDA. Phase 4 follows a drug after approval. Rare disease trials are often much smaller.
Read the full guide →What if there is no trial for me?
Ask your doctor about expanded access, also called compassionate use, an FDA program that lets a company provide an experimental drug outside a trial to someone with a serious condition and no good options. The drug company has to agree, and your doctor makes the request.
Read the full guide →Is orphan drug designation an FDA approval?
No. Orphan drug designation is a status the FDA gives to a drug being developed for a disease that affects fewer than 200,000 people in the United States. It brings incentives such as 7 years of market exclusivity once the drug is approved, but the drug still has to prove it is safe and works.
Read the full guide →Start here
- 01What Is a Clinical Trial?10 minHow research studies work, why they exist and what they mean for someone looking for treatment.
- 02Clinical Trial Phases Explained11 minHow a drug moves from first-in-human testing to FDA approval, and what each phase means for you.
- 03Placebos, Randomization and Blinding10 minThe most common worry about trials. When a placebo is used, when it is not, and what you are told.
Getting in
Know before you go
- 06Risks and Benefits of Clinical Trials11 minAn honest look at what taking part involves, the possible upsides and what can go differently than hoped.
- 07Clinical Trial Costs and Insurance8 minWhat the sponsor pays for, what your insurance must cover and where to find help with travel.
- 08Your Rights in a Clinical Trial11 minInformed consent, the right to leave at any time, ethics board oversight and what federal law guarantees.
- 09Questions to Ask Before You Join8 minThe questions patient advocates recommend asking the study team before you say yes.
Living with a rare disease
- 10Navigating a Rare Disease Diagnosis4 minBuild a medical case file, find the right specialist and know what to do next.
- 11Genetic Testing for Rare Diseases4 minWhich tests to ask for and how a result can open targeted treatments and trials.
- 12Orphan Drug Designation12 minWhat orphan status means, what it does not mean, and how the Orphan Drug Act shapes rare disease drugs.
- 13Compassionate Use and Expanded Access4 minHow to ask for an experimental drug outside a trial when approved options have run out.
- 14A Caregiver's Guide to Clinical Trials10 minTrial logistics, speaking up for someone you love and keeping yourself going.