Rare disease research benefits from the active participation of patients and their families. Through tools like patient-driven registries, individuals and their families are no longer bystanders, passively watching the research process unfold. Today, patients are engaged and empowered to make personal contributions that accelerate discovery and research, and are critical for clinical trials, government intervention, funding, awareness, and advocacy. Patients and families are the key resources to the success of a registry and are the key beneficiaries of new knowledge.

After joining a registry and answering the questionnaire(s), a subset of the information will be shared with the GRDR data repository. Any data shared with GRDR is first de-identified, removing any personal identifiers such as name, address, or other information. The goal of the GRDR is to share medical information with scientists and other researchers, while still protecting patients' privacy. Approved scientists and researchers can use the GRDR data to:

• Study why individuals have different symptoms
• Learn whether treatments work
• Improve the medical care for patients
• Notify participants who may be interested in clinical research studies or clinical trials

Registries are encouraged to allow patients to state their preference regarding whether they wish to be contacted with information about new research studies/clinical trials from the scientists who use the GRDR data. Any such messages are sent from the registry coordinators, so that the patient's privacy is protected. Patients can then request additional information from the researcher, if they so choose.

Participants spend valuable time answering the survey questions, so in return de-identified, summarized data is provided in easy to understand graphs and charts. Patients and families can explore the data in their registry and learn about others with similar diagnosis, treatments or symptoms to better understand how they or their child fits within the disease community. The collected data can be also be used to develop standards of care or care considerations, thereby improving medical care available to patients.

Patients and their families can learn more about what it means to participate in clinical trials through the NIH Clinical Research Trials and You website.