FA Patient Registry

Frequently Asked Questions

+ What is the purpose of the FA Global Patient Registry?

The FAGPR aims to:

Characterize the spectrum of symptoms and heterogeneity of disease presentation of Friedreich’s ataxia.
Establish rate of disease progression in the context of patient self-reporting of symptoms and use of assistive devices.
Enable rapid clinical trial launch through informing eligible patients.

+ How is the FA Global Patient Registry (FAGPR) different than EFACTS (European Friedreich’s Ataxia Consortium for Translational Studies) and FA-COMS (FA Clinical Outcome Measures)?

The differences between the three databases, FAGPR, EFACTS and FA-COMS, are

Management of the databases – FAGPR is managed by international advocacy organizations, EFACTS is managed by a consortium of clinicians based in the EU, and FA-COMS is managed by the Collaborative Clinical Research Network under the guidance of the Friedreich’s Ataxia Research Alliance (FARA).
Person entering data – FAGPR is designed to house patient-entered data; EFACTS and FA-COMS are designed to house clinician-entered data.
Type of data collected – FAGPR data reflects the patients' perspectives of living with FA; EFACTS and FA-COMS data are based on clinical assessments, including FARS or SARA, quality of life assessment measures, and biological specimen collection.

The goals of all three databases are to more fully understand disease progression and to provide a framework for facilitating therapeutic interventions.

The FAGPR is available to all persons affected with FA and parents of children affected with FA. The FAGPR has a more global reach than EFACTS and FA-COMS because data entered into the FAGPR is not dependent on visiting an FA-specialty clinic.

+ Is the registry available internationally?

Yes. Participation is available to all FA patients regardless of country of residence. Initially available only in English, the registry is available since August 2022 in Dutch, French, German, Greek, Italian, Portuguese, and Spanish.

+ Why is a patient registry vital to advancing knowledge about Friedreich’s ataxia?

When pharmaceutical companies begin the journey of developing a drug for FA, one of the first questions asked is “Where are FA patients located around the world?” The answer to this question is important because:

Pharmaceutical companies will need to plan for clinical trials and eventual drug distribution.
Pharmaceutical companies will need to be aware of regulatory approval processes in each country where FA patients are treated.
Pharmaceutical companies will need to plan for physician education about drug availability in any country where FA patients are being diagnosed and treated.

+ What does the patient registry not do?

Patient registry information is never used for fundraising campaigns.

Patient registry information is never shared with researchers and pharmaceutical companies without appropriate protocol review of how the registry data will be used.

Data shared with researchers does not contain individual patient contact information.

+ Is my data secure in the registry?

Your data is secured through the Pulse Infoframe platform. The platform adheres to regulations for securing protected health information (PHI).

+ Was the FA Global Patient Registry reviewed for ethics approval?

FAGPR was created with input from a Steering Committee comprised of international FA advocacy organization representatives, clinicians, FA patients, FA parents, representatives from industry, and a representative from a muscular dystrophy advocacy organization.

The consent document, the Patient Profile, and the survey forms were all reviewed and approved by WIRB, an international ethics review board company.
(http://wirb.com/Pages/default.aspx)

+ How do I join the registry?

When you click the "Enroll / Login" button, you will be directed to the homepage for the FA Global Patient Registry. You will answer a series of questions in order to create an account – this process takes less than 5 minutes. Once completed, you will receive an automated email containing a link to create a password for your account. Please check your spam folder if you do not receive the automated email within 10 minutes after submitting your initial information.

Once logged in, please complete the Informed Consent process, found under the “Consents” tab on the left side menu of your account dashboard. The Informed Consent process must be completed before you can begin completing the Patient Profile (demographic information) and survey forms. Each survey takes 5-20 minutes to complete.

View the FAGPR User Guide (English)

+ How often should I update my information in the registry?

You can access your Patient Profile at any time to update your contact information and your preference for being contacted about research studies.

You can update your information on the survey forms every 12 months. You will receive an email notification to remind you to review and update the survey forms.


	RESOURCES	GOVERNANCE	ADVOCACY

Welcome! The goals of the FA Global Patient Registry (FAGPR) are to collect information on all FA patients in one registry, to develop the registry into a powerful resource for research, and to engage the FA community in studies aimed at advancing our knowledge of FA and the treatments being developed. This registry is only for people diagnosed with Friedreich's ataxia. Participation in this registry will require your consent to share your data. Participation in the FAGPR is voluntary. The registry is maintained by international patient advocacy organizations through a Governance Board. Please direct any questions to the registry coordinators at FAGPR@curefa.org Enroll / Login Frequently Asked Questions + What is the purpose of the FA Global Patient Registry? The FAGPR aims to: Characterize the spectrum of symptoms and heterogeneity of disease presentation of Friedreich’s ataxia. Establish rate of disease progression in the context of patient self-reporting of symptoms and use of assistive devices. Enable rapid clinical trial launch through informing eligible patients. + How is the FA Global Patient Registry (FAGPR) different than EFACTS (European Friedreich’s Ataxia Consortium for Translational Studies) and FA-COMS (FA Clinical Outcome Measures)? The differences between the three databases, FAGPR, EFACTS and FA-COMS, are Management of the databases – FAGPR is managed by international advocacy organizations, EFACTS is managed by a consortium of clinicians based in the EU, and FA-COMS is managed by the Collaborative Clinical Research Network under the guidance of the Friedreich’s Ataxia Research Alliance (FARA). Person entering data – FAGPR is designed to house patient-entered data; EFACTS and FA-COMS are designed to house clinician-entered data. Type of data collected – FAGPR data reflects the patients' perspectives of living with FA; EFACTS and FA-COMS data are based on clinical assessments, including FARS or SARA, quality of life assessment measures, and biological specimen collection. The goals of all three databases are to more fully understand disease progression and to provide a framework for facilitating therapeutic interventions. The FAGPR is available to all persons affected with FA and parents of children affected with FA. The FAGPR has a more global reach than EFACTS and FA-COMS because data entered into the FAGPR is not dependent on visiting an FA-specialty clinic. + Is the registry available internationally? Yes. Participation is available to all FA patients regardless of country of residence. Initially available only in English, the registry is available since August 2022 in Dutch, French, German, Greek, Italian, Portuguese, and Spanish. + Why is a patient registry vital to advancing knowledge about Friedreich’s ataxia? When pharmaceutical companies begin the journey of developing a drug for FA, one of the first questions asked is “Where are FA patients located around the world?” The answer to this question is important because: Pharmaceutical companies will need to plan for clinical trials and eventual drug distribution. Pharmaceutical companies will need to be aware of regulatory approval processes in each country where FA patients are treated. Pharmaceutical companies will need to plan for physician education about drug availability in any country where FA patients are being diagnosed and treated. + What does the patient registry not do? Patient registry information is never used for fundraising campaigns. Patient registry information is never shared with researchers and pharmaceutical companies without appropriate protocol review of how the registry data will be used. Data shared with researchers does not contain individual patient contact information. + Is my data secure in the registry? Your data is secured through the Pulse Infoframe platform. The platform adheres to regulations for securing protected health information (PHI). + Was the FA Global Patient Registry reviewed for ethics approval? FAGPR was created with input from a Steering Committee comprised of international FA advocacy organization representatives, clinicians, FA patients, FA parents, representatives from industry, and a representative from a muscular dystrophy advocacy organization. The consent document, the Patient Profile, and the survey forms were all reviewed and approved by WIRB, an international ethics review board company. (http://wirb.com/Pages/default.aspx) + How do I join the registry? When you click the "Enroll / Login" button, you will be directed to the homepage for the FA Global Patient Registry. You will answer a series of questions in order to create an account – this process takes less than 5 minutes. Once completed, you will receive an automated email containing a link to create a password for your account. Please check your spam folder if you do not receive the automated email within 10 minutes after submitting your initial information. Once logged in, please complete the Informed Consent process, found under the “Consents” tab on the left side menu of your account dashboard. The Informed Consent process must be completed before you can begin completing the Patient Profile (demographic information) and survey forms. Each survey takes 5-20 minutes to complete. View the FAGPR User Guide (English) + How often should I update my information in the registry? You can access your Patient Profile at any time to update your contact information and your preference for being contacted about research studies. You can update your information on the survey forms every 12 months. You will receive an email notification to remind you to review and update the survey forms. The FAGPR has been collaboratively built on Pulse Infoframe's healthie™ platform.

Welcome!

Frequently Asked Questions