What Is a Clinical Trial?
The basics of how research studies work, why they exist, and what they mean for patients seeking treatment.
Phases Explained
How a drug moves from first-in-human testing through FDA approval, and what each phase means for you.
Placebos, Randomization & Blinding
The #1 concern patients have about trials. What placebos actually mean, when they're used, and when they're not.
Risks & Benefits
An honest look at what trial participation involves, the potential upsides, and what could go differently than expected.
Cost & Insurance
What's covered by the sponsor, what might fall on your insurance, and financial assistance programs that exist.
Your Rights
Federal protections, informed consent, the right to withdraw, IRB oversight, and what the law guarantees you.
Questions to Ask
The specific questions experienced patient advocates recommend asking before you commit to a study.
Navigating a Diagnosis
How to build your medical case file, find the right specialist, and take action once you have a rare disease diagnosis.
Genetic Testing
How genetic testing works, which tests to ask for, and how results can unlock targeted treatments and clinical trials.
Orphan Drug Designation
What orphan drug status means for rare disease patients and how the Orphan Drug Act drives treatment development.
Compassionate Use
How to access investigational drugs outside of clinical trials when approved treatments have been exhausted.
Caregiver's Guide
Practical guidance for caregivers navigating trial logistics, advocating for a loved one, and sustaining yourself.
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