For parents and caregivers
When someone you love has a rare disease, the search for trials is personal. This guide is written for you - the parent, partner, or family member doing the research at midnight.
Where to start
01
Before spending weeks researching trials alone, find the patient advocacy organization for your condition. These communities - Facebook groups, forums, foundation newsletters - are where families share trial experiences in real time. You will learn more in one conversation with another parent than in ten hours of reading medical papers.
Use our Disease Directory to find the right organization for your condition.
02
Many rare diseases have natural history studies and patient registries. Registering ensures that when a trial opens and researchers are looking for participants, they may be able to contact you directly. It also contributes to research that eventually leads to treatments.
This is often the single most important step a rare disease family can take. Researchers use registries to recruit for trials, and small patient populations mean every registered patient matters.
03
Most trials require a confirmed diagnosis using the specific criteria the researchers defined. If your diagnosis is clinical (based on symptoms) rather than genetic, ask your specialist about genetic or enzyme testing. Many trials require a specific mutation or biomarker to be documented. Getting this done early avoids delays when a trial becomes available.
04
When you contact a trial site, they will ask for records. Having these ready saves weeks:
05
Participating in a trial with a child or dependent adult requires real commitment. Before you apply, honestly evaluate:
These are not reasons to avoid trials - they are conversations to have with your family and your doctor before committing.
Pediatric trials
For children, you as the parent or legal guardian provide consent. Children aged 7 and above are also typically asked for "assent" - their agreement to participate in language they can understand. The trial team will explain this process before any procedures.
Many pediatric trials have narrow age eligibility - for example, only children aged 2 to 5. If your child is approaching an age cutoff, do not wait. Contact the study site now, even if the trial is not yet recruiting at your nearest site. Sites fill quickly and waiting lists form.
Frequent clinic visits may mean missing school. Contact your child's school early to arrange medical leave. Many schools have formal policies for children in clinical trials. Ask the study team for documentation to give to your school administrator.
If travel is required, Ronald McDonald Houses near major medical centers offer free or low-cost family lodging. Most large academic medical centers have one nearby. See our financial assistance page for more.
For you, the caregiver
Caregiving for someone with a rare disease is one of the most exhausting things a person can do. The research, the appointments, the insurance battles, the emotional weight - it is a second job with no end and no manual.
Connecting with other caregivers who share your disease is not just emotionally valuable - it is strategic. They know which trial sites are actually responsive. They know which study coordinators return calls. They know what to pack for long clinic days and which hotels are near the hospital.
Find your community through your disease's patient advocacy organization. It is one of the first things we recommend for every caregiver, every time.