Learn about Research

Research Advances Our Clinical Understanding of a Disease
Research Can Lead to the Discovery of New Diagnostics and Treatments
Patient Involvement is Key to Advancing Research
Research is Important for Rare Diseases

Rare disease research is needed to advance medical knowledge. Research can clarify the common symptoms of a disease and the course of the disease. It can also lead to a quicker diagnosis or improved treatment and medical care. It may even contribute to discoveries related to more common diseases.

Participate in Research

Joining a Clinical Study

Clinical studies include all research that involves people either through direct interaction or through the collection and analysis of blood, tissues, or other samples. Clinical trials are clinical studies involving participants who are assigned to an intervention, procedure, or potential treatment. They evaluate the effect of the intervention on the participant. It is important to review all aspects of the clinical study when making a decision about whether to participate.

Reasons to Participate in Clinical Studies

Clinical studies can benefit participants, the medical field, and the rare disease community in multiple ways. 

A person may decide to participate in clinical studies because clinical studies often provide: 
 

  • An opportunity to contribute information leading to a better understanding of their rare disease.
  • Advancements in medical knowledge for other rare diseases and more common diseases.
  • New ways to prevent, diagnose, and treat diseases.
  • Improved health care and quality of life for people who currently have a disease and for those who may develop a disease in the future.
  • An opportunity to access cutting edge research treatment before it is widely available to others and to receive medical care from knowledgeable providers. 
  • Hope for current and future patients and a sense of accomplishment for actively playing a vital role in the progress of research.

Information to Consider Before Joining a Clinical Study

Understanding as much as possible about participating in clinical studies is key to making an informed decision. 

Before joining a clinical study, it is important to understand that:
 

  • Participating in a clinical study is entirely voluntary. Participants do not have to start or finish a study
  • An Institutional Review Board (IRB) approves and monitors most studies in the U.S. to ensure that guidelines are followed, the study is ethical, and the participants’ rights and safety are protected
  • Clinical studies may involve some risks. It is important to understand these potential risks and the chance of harm occurring
  • All information obtained in the study is confidential
  • In many treatment studies, a participant may be randomly assigned to receive a placebo (a harmless, inactive substitute)
  • Even in studies involving treatments, a participant’s disease and symptoms may not improve, and there is a possibility they may get worse

How do you find the right clinical study?

For more information:

Clinical Research & You [NIH]

Provides information about clinical trials, participating in clinical trials, and finding clinical trials. Extra sections include personal stories by clinical trial participants and enrolling children in clinical trials.

Clinical Trials - What Patients Need to Know [FDA]

Offers information about specific topics related to participating in clinical trials, including the difference between clinical research and medical treatment, different types of clinical trials, and informed consent.

Want to Participate in a Clinical Study?

Search ClinicalTrials.gov

We strongly recommend talking to a trusted doctor before choosing to participate in any clinical study. Not all diseases will have an active clinical study.

NIH - National Center for Advancing Translational Sciences - GARD Genetic and Rare Diseases Information Center

Get in touch with us for help.

1-888-205-2311

GARD provides general information about rare diseases and clinical studies. We are unable to make a diagnosis or to give personal medical advice.

Contact Us

Stay Informed and Get Involved

Staying informed about research efforts can help patients and families make better medical decisions. Learning about different ways to become involved in research provides options for patients and families to join efforts outside of clinical studies.

Engaging in Research Efforts

NCATS Toolkit for Patient-Focused Therapy Development
NIH - National Center for Advancing Translational Sciences - Toolkit for Patient focused Therapy Develpoment

NCATS Rare Diseases Registry Program (RaDaR)
NIH - National Center for Advancing Translational Sciences - RaDaR Rare Diseases Registry Program

Last Updated: Nov. 8, 2021