Frequently Asked Questions
What is a patient registry?
A registry is a program for collection, storage, retrieval, and dissemination of clearly defined information for a specific purpose. Data collected in this registry includes diagnosis and treatment, management of care, quality of life and longitudinal information for Refsum Disease.
Why a Refsum Disease patient registry?
In one word, PROGRESS. Having a registry specifically for individuals with Refsum Disease allows approved researchers to have easy access to useful medical data on many individuals with Refsum Disease. Since we will have already collected this data and obtained your consent to share it, researchers can begin to develop better studies much more quickly, and more easily find participants for these studies.
The Global DARE Foundation has our own goals for this registry as well. In addition to recruiting patients for medical studies in the future, we hope it will help us to provide better support to families NOW. Over the long term, our goals are to document trends across the Refsum Disease population, to bring about quality research and treatment efforts and, yes, to develop an official worldwide number of documented Refsum Disease cases. NUMBERS MATTER when it comes to driving research and better treatments.
Who should participate?
Everyone with Refsum Disease! Participating in the Global DARE Foundation Registry at CoRDS is a great way for participants to take part in helping to identify the specific causes, symptoms, and treatments of Refsum Disease.
Is my personal information safe?
Internet polls, questionnaires, and surveys are often used to collect information quickly and easily from respondents. However, to publish their results, researchers must only use information obtained in a specific way. The patient data they use must have written approval from patients to use their data this way and must adhere to strict privacy regulations. The registry is compliant with the European Union General Data Protection Regulation (GDPR). CoRDS has worked hard to ensure that researchers can use the information you provide.
CoRDS submits every registry to their Scientific Advisory Board (SAB) for approval. A SAB is an independent group that reviews the ethics of medical research studies. The SAB at Sanford Research has approved the Refsum Disease questionnaire. Researchers requesting to use the information in the registry must first obtain approval from their own Internal Review Boards and then a second approval from the Sanford SAB.
Informed consent also safeguards participant data. When registering to participate in CoRDS, participants (or their representatives) are given a chance to read the consent documentation before filling out the questionnaire. For any questions, participants can call CoRDS at + 1 877 658 9192 or send an email firstname.lastname@example.org..
The information you provide in the registry is kept strictly confidential. The questionnaire that you complete will never be associated with your name. Instead, an anonymous ID number is assigned to your identifiable information (name, address, etc.) and to the responses you provide in the questionnaires. If researchers receive approval to look at the registry or questionnaire responses, they will be given only the anonymous data without the identifiable information. The Global DARE Foundation can only access your identifiable information if you choose to share it with us when you complete your registry questionnaire.
Your privacy is also protected even if you indicate that you are willing to be contacted for additional research. For example, a researcher might contact CoRDS to ask for additional information from all participants who have a specific symptom, such as loss of hearing. CoRDs would then contact every participant in the registry who said that they had loss of hearing and that they would like to participate in additional studies. CoRDS would then provide these people with the researcher’s contact information, and it would be up to the individual participants to contact the researcher to participate in additional research. CoRDS will never provide your contact information to anyone.
When should I update registry information?
Updates can be done any time and should only take 10-15 minutes. But updates should be done at least every year, after any significant change in the participant’s health, and immediately after their 18th birthday. If the participant was enrolled in the registry as a minor, their data becomes inaccessible 30 days after their 18th birthday unless and until they are re-enrolled.
CoRDS will send a reminder by email to participants once a year to make any updates. For mail-based participants CoRDS will send a letter and questionnaire in the mail once a year for them to make any changes.
What is the purpose of the registry?
Researchers will be able to compare your answers with those of others. The researchers will then be able to detect patterns, identify common symptoms and gain insights into effective and ineffective treatments. Your answers will provide a better overall understanding of Refsum Disease and how the disease develops/progresses over the course of a lifetime.
Our goal is to collect and provide valuable information and to speed up research studies of Refsum Disease and help start clinical trials for treatments.
The information you provide to this registry can help to:
Drive research towards better treatments and possibly a cure
Allow healthcare professionals and researchers to learn more about the disease
Create a strong patient and research community
Enhance treatment guidelines for patients to improve quality and management of care
Can data be collected worldwide?
Yes, the registry can be accessed all over the world with the link provided on our website. International participation is highly encouraged.
How is my personal information used and protected?
The information you provide will be provided to researchers studying Refsum Disease. CoRDS has put many safeguards into place to ensure that this information will be kept safe and confidential. The registry is compliant with the European Union General Data Protection Regulation (GDPR).