Clinical Research

Understanding Participation in Clinical Research

Questions & Answers to Help You Determine if Clinical Research is Right for You

Clinical research is the backbone of medical discovery helping us to better understand how to prevent, diagnose and treat disease. Volunteer participants play a vital and important role in clinical research to evaluate whether a therapy is safe and effective before it can be approved by the U.S. (FDA) or in Europe (EMA). Every medication and breakthrough discovery to treat rare disorders is the result of clinical research made possible by volunteers like you.

These terms are often interchangeable. Clinical research is an umbrella term that has become more popular. The one element they all have in common is volunteer participants. There are two general types of clinical research; an interventional study, often referred to as a clinical trial that involves evaluating a drug, medical device or procedure and an observational study such as natural history studies and survey studies where participants are observed or certain outcomes measured, but there is no treatment given.

Participation in clinical research is a personal decision ‒ one that can only be made by you. Participants who volunteer in clinical research believe it’s an opportunity to give back and help advance the science to ultimately treat the rare disorder. Participating in rare disease clinical research allows participants to play a more active role in their own health, as well as the potential to offer a brighter future for their children and others. Whatever your motivation or reason for participation, researchers need volunteers to help bring innovative and breakthrough therapies to treat rare disorders.

It’s important to have all your questions answered before you can determine if clinical research is right for you. Consider these aspects of the research:


• The length of the clinical trial

• The investigational therapy

• Potential benefits and risks


As a trial is starting a list of the clinical sites that are participating can be found on or by contacting patient advocacy groups or contacting the company that is sponsoring the research. Talking to your healthcare provider about the study can also provide an additional perspective. It’s important to know that each step in the drug development pathway is strictly governed by regulatory authorities like the FDA and the EMA to ensure the most rigorous medical safeguards are put in place for the ethical well-being of all volunteer participants.

The journey to bring a new therapy to market has phases (or stages) to ensure it is both safe and effective. Even before a therapy makes it to human testing in clinical trials, many years of animal testing and other studies have already been conducted. Traditionally, there are three phases guiding clinical research and each phase is designed to answer a specific question about the treatment. In some cases, there may be a fourth phase of clinical research.


Phase 1: How safe is the therapy by examining possible side effects or other issues?
Phase 2: Is the therapy effective in a small group of patients determining effective dosages of the investigational drug along with possible side effects and risk factors?
Phase 3: How safe and effective is the investigational in a larger number of patients?
Phase 4: What more do we need to know about the therapy over time after the drug is approved?

All volunteers must be eligible in order to participate in clinical research. Age, gender and the severity of disease are typical eligibility criteria. Eligibility criteria are developed to ensure the volunteers’ safety and that the objectives of the research can be properly evaluated. Research in rare diseases has its unique set of challenges including small patient populations that are geographically diverse. To increase participation, the FDA & EMA have worked together with clinical researchers to overcome these obstacles through the development of novel protocol designs and soliciting valuable input from the ‘patient perspective.’

In some circumstances, it may be necessary for participants and caregivers to travel to a clinical research site. Travel assistance including transportation and hotel is typically provided for patients and caregivers. Home study visits may be offered in some clinical to greater meet the needs of the study volunteers. A participant has the right to leave a clinical research study at any time.
The answer is a resounding YES; your privacy will be protected, and your personal information and medical records will be kept confidential. Patient information collected in clinical research is “de-identified,” meaning that participants in the study are assigned a unique code that cannot be linked back to their names. By law, the study site is not allowed to share participant names without written permission by the participant.
In some cases, a stipend is provided in recognition of the time required to participate in a study.. Compensation is based on the type of research, length of the research, the number of clinic and home visits. If travel is necessary, travel assistance including transportation and hotel is provided for patients and caregivers.

If you are considering participating in clinical research, your healthcare provider is a great resource to help explain and answer your questions. Listed below are several questions to get the conversation started.


1. What are the possible benefits and risks associated with participating in clinical research?

2. Will I be guaranteed that I will be on the investigational drug?

3. When the clinical research trial is completed, can I stay on the therapy?

4. Can you help me identify the nearest study site?

5. What should I anticipate as far as travel and time away from home?

There are many trusted resources to help guide your decision making about participating in clinical research. is the largest and most comprehensive clinical trials database provided by the United States National Library of Medicine at the National Institutes of Health (NIH).  


Additionally, you might check out the following trusted sources:


Rare Disease Clinical Research Network (RDCRN) is an initiative of the Office of Rare Diseases Research (ORDR) at the National Institutes of Health’s National Center for Advancing Translational Sciences (NCATS). You can view the network of 23 active consortia or research groups.

• By answering a few short questions, can help you find a list of studies that may interest you.

Orphanet provides information on ongoing and unpublished research projects explicitly focused on a rare disease or on a group of rare diseases.  

Exit interviews play an important role at the end of the trial and allow researchers to listen to feedback from study participants about their experience participating in the research.
The informed consent process whereby you learn about the details of the study before it begins will inform you about the process of treatment availability options once the trial is completed. Where appropriate, an open-label extension study provides participants with long-term access to the available treatment. You’ll want to ask the study coordinator during the informed consent process.