Become Part of the Solution: Participate in Clinical Research

Clinical research plays a critical part in advancing science and treatments. Volunteers who participate in this research are essential as new treatments for rare diseases cannot be approved without them.

Learn more about volunteering for a clinical study in this video from the National Institutes of Health (NIH) and read about why participating in clinical research could be beneficial on the NIH website.

Who can participate in clinical research?

EVERYONE! But it may take time to find the opportunity that’s right for you.

To participate in certain research studies, you must be eligible. Eligibility means you must meet certain criteria outlined in the clinical trial design such as your age, gender, medical conditions, overall health, and previous treatment history.¹ These are called the inclusion and exclusion criteria and they determine whether a volunteer can be considered for clinical research.

If you are not selected for a clinical research opportunity, don’t take it personally. Each one is different, and it’s important to find volunteers who fit the criteria. Just because you’re not eligible for one opportunity doesn’t mean you won’t be eligible for another or that you can’t contribute to research in other ways. Keep reading to learn how you can find the best opportunity for you!

Other kinds of research: There are many ways to get involved in advancing the science and understanding of a disease. Not all research is clinical, or involving a potential treatment, but it is just as important. Keep an eye out for our next blog that will focus on burden of disease surveys, patient interviews, natural history studies, and disease monitoring programs, which are often referred to as registries. This kind of research study can be open to anyone with a certain condition, regardless of the treatment they are on. It helps broaden understanding of a condition and the experiences of patient communities – a crucial part of delivering safe and effective medicines to the people who need them.

How does clinical research work?

Clinical research follows strict, government-regulated protocols to ensure the study is being conducted in a safe manner. A protocol is a set of guidelines that outline exactly what a research study will do, how it will work, and who can participate.²

Learn more about clinical research on the FDA’s website, PCORI’s website, and our About Clinical Trials page.

How does informed consent work?

It’s important to learn about the benefits and risks of clinical research opportunities before you participate. Potential trial participants receive information about the study in what is called an informed consent form (ICF). It tells you about the time commitment, the potential treatment being studied, the types of procedures involved, and much more. You will be asked to read and sign this form before you participate in clinical research. Understand that your participation is voluntary.³ Even after you sign an ICF, you can end your participation at any time, for any reason.

What questions should you ask?

Open communication between you and clinical researchers (also called investigators), study site coordinators, and study nurses is important. Write down questions you might have and take time to ask them at any point during your participation. You may want to ask:

• What is this research trying to accomplish?
• What are the possible benefits and risks of the study, and how does this compare to current therapies?
• What kinds of tests, treatments, and procedures are involved?
• How much of my time will the study take?
• How long will the program last?
• Will I be reimbursed for expenses like time, travel, meals, and childcare?
• Does the study use a placebo? If I receive the placebo in the study, will I receive the potential treatment at some point?
• Is there any follow-up after the study has ended?
• Is there an extension of the study?
• Will I continue to receive the treatment after the study ends?
• Will results of the study be provided to me? When?
• If the research is successful and this treatment is approved by the FDA or other regulatory agency, will I continue to have access to the therapy?

“Participating in a clinical trial is a very personal decision. Ask lots of questions. Talk to others who have participated in similar trials if you can. Always read the consent forms, even if you are not the type of person to normally read all the fine print. Create a support group for yourself of people who can help at home if the trial requires you to travel and be away.”

– Laura, mother to Jordan, a clinical trial patient living with Duchenne Muscular Dystrophy

What other tips should you keep in mind?

Once you have signed an ICF and have been accepted into a clinical study, there are a few things to keep in mind. Some of these guidelines will be discussed in the ICF, but they are important to remember for research purposes.

• Honesty is the best policy. It’s not your job to please the researchers. It is your job to tell them exactly how you are feeling, even if the feedback isn’t positive.
• Practice doesn’t always make perfect. Clinical research must follow specific procedures, and participants should try to do the same. Practicing for any tests that may be given could jeopardize the integrity of the study and negatively influence the results.
• Timing is everything. You can share some of your clinical research experiences with others, but it’s important to remember what you can share and when. During the study you should not share details or any results you feel you’ve had.
• Think first. It is especially important to think before you post about your clinical research experience on social media. Ask yourself, “Am I sharing information that could potentially harm the results of the research?” If you are unsure, check with your investigator(s). Wait until they give you the okay to share, which is usually once the study or trial is completed. Please note: some research studies may not allow you to post on social media.

Ready to make a difference?

“I was honest with Jordan that we didn’t know if the drug worked, but we were hopeful that it would. I’ve been so impressed with his attitude about how grateful he is to participate in the trial to be able to help others, not just himself.”

– Laura

Future therapies would not be possible without the help of volunteers. Participating in clinical trials, interviews, or observational studies are all impactful ways to drive research, treatment development, and disease education. These opportunities are great ways to advocate for yourself and your community, especially for those impacted by rare disease.

When you engage in research opportunities you are helping to advance treatments and help researchers better understand the needs of people living like you. Get involved!

CISCRP (Center for Information and Study on Clinical Research Participation) hosts multiple events each year to celebrate and honor the medical heroes who participate in clinical trials. Learn more here!

ASK your doctor or a patient advocacy organization if they know of any research opportunities.

SEARCH the ClinicalTrials.gov database of clinical trials around the world. (clinicaltrials.gov/ct2/about-studies/learn#ClinicalTrials)

GET MATCHED through ResearchMatch, a secure online tool that emails you research studies that might be a good fit for you. (researchmatch.org)

CONNECT over the phone at 1-877-MED HERO to CISCRP, an organization whose staff can help perform a clinical trial search for you. (Center for Information and Study on Clinical Research Participation: ciscrp.org)

BROWSE for other important patient engagement activities with pharmaceutical companies like advisory boards, burden of illness surveys, or interviews.