Step 1.
Create Your Registry Plan

1.0 Overview

Planning ahead is essential to establish clear goals for your registry and a strategy for collecting quality data. In this step you will define specific, detailed, and attainable goals for your registry; define your constraints; plan for roadblocks and identify possible solutions to them; and establish milestones to track your success.

1.1 Set Your Goals

Before creating a registry and setting your goals, determine whether a registry has already been created for your rare disease. Partnering with an existing registry allows for you to combine efforts, avoid “reinventing the wheel,” and reduce redundancy. There are many ways to find out whether a registry currently exists for your rare disease.

After confirming that no other registry exists for your disease, you can start creating your registry.

Be specific, detailed, and realistic about the goals for your registry and clear about what you plan to do with the information you collect. Focus on how you can use your registry to organize your patient community and connect patients and researchers. Have a long-term vision for capturing detailed participant medical information to support the development of new treatments. Below are some goals to consider. Use the RaDar Tool: Registry Plan Template for help getting started.

Short-term registry goals:

  • Identify patients who are interested in participating in research studies.
  • Describe the personal characteristics of participants in your registry.
  • Contact participants to inform them about new studies.

Long-term registry goals:

  • Document patient medical history.
  • Discover trends and common needs of participants.
  • Improve scientific understanding of the disease.

Visit the Resources section of this step for resources that provide additional information for setting up your registry.

1.2 Consider Your Constraints

Budget
When first building your registry, start with volunteers and free or low-cost methods for collecting information. At this early stage, your registry is similar to a contact list or member database. If your patient advocacy group has more financial resources, consider the option of setting a higher budget to cover more sophisticated data collection methods or staff. Over time, you will build your registry to include medical information, at which point most patient advocacy groups pay for a service, or link with a research center or vendor to operate their registry.

Knowledge, Skills, and Time
You may need additional skills or experience to collect contact and demographic (e.g., age, race, ethnicity, sex) information. Many patient groups spent a few hours a week getting their registry up and running. Consider how much time you have available and look for one or more partners to join you if your time is limited. Consider the importance of obtaining Institutional Review Board (IRB) approval before collecting participant information. Without prior IRB approval, researchers may not be able to use your registry’s data to publish the results of their study.

1.3 Plan for Roadblocks

Any project is bound to face some roadblocks along the way. Create a list of things that might slow down your progress and include some ideas to move past the obstacles.

Potential Roadblocks:

  • Lack of time
  • Lack of interest within the patient community
  • Competing ideas over the direction of the registry
  • Difficulty making decisions
  • Existence of other registries for the disease
  • Difficulties of working alongside a research group versus doing it by yourself
  • Too many “cooks in the kitchen”

Possible Solutions:

  • Find others in your patient advocacy group to help share the workload.
  • Use social media to create buzz by sharing stories, videos, infographics, and articles. Focus on the needs of the community and how your registry can address them.
  • Work to stay unified by compromising and focusing on shared goals.
  • Get the advice of other patient advocacy groups that have established similar registries.
  • Define roles and responsibilities of registry staff.

1.4 Create Milestones to Track Progress

Draft a timeline for achieving registry milestones and for staying motivated. The timeline may change, but it will help you track your progress. For example:

  • Registry will be set up and ready to collect information by [Date].
  • Complete first recruitment push within [#] months.

1.5 Develop a Governance Plan

Registries should also create and follow a written governance plan that includes defining objectives, identifying stakeholders, building your registry team, establishing Ethical Legal and Societal Issue (ELSI) and privacy guidelines, and sustainability. You can read more about governance plans by referring to the paper “Recommendations for Improving the Quality of Rare Disease Registries.”  A good governance plan will also provide guidance for making decisions about how the registry is run and how and with whom data will be shared.

Visit RaDaR Step 7.3 Manage Data Requests to learn about issues pertaining to data access, security, and privacy.

Resources

Name Source Type Size
1.1 Set Your Goals
Section 1: Creating Registries Registries for Evaluating Patient Outcomes: A User’s Guide: 3rd Edition link --
Article: The Fair Guiding Principles for scientific data management and stewardship Nature: Scientific Data .pdf 9 MB
RaDaR Tool: Registry_Plan_Template RaDaR .pdf 131 KB
Frequently Asked Questions (FAQs) - Registries NIH Clinical Research Trials and You link --
RARE Webinar: Collaboration Among Foundations Global Genes link --
Patient Registries in the Field of Rare Diseases European Commission Expert Group on Rare Diseases link --
Rare Disease Registry: Potential Applications Toward Impact on Development of New Drug Treatments Orphanet Journal of Rare Diseases link --
Rare Toolkit: Rare Disease Registries: Advancing Disease Understanding, Treatments, and Cures Global Genes link --
PCORI Training: A Program for Rare Disease Patient Advocates Patient-Centered Outcomes Research Institute (PCORI) link --
Frequently Asked Questions (FAQs) - Registries World Federation of Hemophilia link --
Registries Tool Kit TREAT-NMD Neuromuscular Network link --
Fact Sheet - Rare Disease Patient Registries EURORDIS Rare Disease Europe link --
Developing a Patient Registry: A Practical Guide Medical Research Charities Group link --
1.2 Consider Your Constraints
Video: Institutional Review Boards (IRBs) U.S. Department of Health and Human Services link --
1.5 Develop a Governance Plan
Recommendations for Improving the Quality of Rare Disease Registries International Journal of Environmental Research and Public Health link --
Section II. Legal and Ethical Considerations for Registries Registries for Evaluating Patient Outcomes: A User's Guide [Internet]. link --

Planning ahead is essential to establish clear goals for your registry and a strategy for collecting quality data. In this step you will define specific, detailed, and attainable goals for your registry; define your constraints; plan for roadblocks and identify possible solutions to them; and establish milestones to track your success.

Before creating a registry and setting your goals, determine whether a registry has already been created for your rare disease. Partnering with an existing registry allows for you to combine efforts, avoid “reinventing the wheel,” and reduce redundancy. There are many ways to find out whether a registry currently exists for your rare disease.

After confirming that no other registry exists for your disease, you can start creating your registry.

Be specific, detailed, and realistic about the goals for your registry and clear about what you plan to do with the information you collect. Focus on how you can use your registry to organize your patient community and connect patients and researchers. Have a long-term vision for capturing detailed participant medical information to support the development of new treatments. Below are some goals to consider. Use the RaDar Tool: Registry Plan Template for help getting started.

Short-term registry goals:

  • Identify patients who are interested in participating in research studies.
  • Describe the personal characteristics of participants in your registry.
  • Contact participants to inform them about new studies.

Long-term registry goals:

  • Document patient medical history.
  • Discover trends and common needs of participants.
  • Improve scientific understanding of the disease.

Visit the Resources section of this step for resources that provide additional information for setting up your registry.

Budget
When first building your registry, start with volunteers and free or low-cost methods for collecting information. At this early stage, your registry is similar to a contact list or member database. If your patient advocacy group has more financial resources, consider the option of setting a higher budget to cover more sophisticated data collection methods or staff. Over time, you will build your registry to include medical information, at which point most patient advocacy groups pay for a service, or link with a research center or vendor to operate their registry.

Knowledge, Skills, and Time
You may need additional skills or experience to collect contact and demographic (e.g., age, race, ethnicity, sex) information. Many patient groups spent a few hours a week getting their registry up and running. Consider how much time you have available and look for one or more partners to join you if your time is limited. Consider the importance of obtaining Institutional Review Board (IRB) approval before collecting participant information. Without prior IRB approval, researchers may not be able to use your registry’s data to publish the results of their study.

Any project is bound to face some roadblocks along the way. Create a list of things that might slow down your progress and include some ideas to move past the obstacles.

Potential Roadblocks:

  • Lack of time
  • Lack of interest within the patient community
  • Competing ideas over the direction of the registry
  • Difficulty making decisions
  • Existence of other registries for the disease
  • Difficulties of working alongside a research group versus doing it by yourself
  • Too many “cooks in the kitchen”

Possible Solutions:

  • Find others in your patient advocacy group to help share the workload.
  • Use social media to create buzz by sharing stories, videos, infographics, and articles. Focus on the needs of the community and how your registry can address them.
  • Work to stay unified by compromising and focusing on shared goals.
  • Get the advice of other patient advocacy groups that have established similar registries.
  • Define roles and responsibilities of registry staff.

Draft a timeline for achieving registry milestones and for staying motivated. The timeline may change, but it will help you track your progress. For example:

  • Registry will be set up and ready to collect information by [Date].
  • Complete first recruitment push within [#] months.

Registries should also create and follow a written governance plan that includes defining objectives, identifying stakeholders, building your registry team, establishing Ethical Legal and Societal Issue (ELSI) and privacy guidelines, and sustainability. You can read more about governance plans by referring to the paper “Recommendations for Improving the Quality of Rare Disease Registries.”  A good governance plan will also provide guidance for making decisions about how the registry is run and how and with whom data will be shared.

Visit RaDaR Step 7.3 Manage Data Requests to learn about issues pertaining to data access, security, and privacy.

Resources

Name Source Type Size
1.1 Set Your Goals
Section 1: Creating Registries Registries for Evaluating Patient Outcomes: A User’s Guide: 3rd Edition link --
Article: The Fair Guiding Principles for scientific data management and stewardship Nature: Scientific Data .pdf 9 MB
RaDaR Tool: Registry_Plan_Template RaDaR .pdf 131 KB
Frequently Asked Questions (FAQs) - Registries NIH Clinical Research Trials and You link --
RARE Webinar: Collaboration Among Foundations Global Genes link --
Patient Registries in the Field of Rare Diseases European Commission Expert Group on Rare Diseases link --
Rare Disease Registry: Potential Applications Toward Impact on Development of New Drug Treatments Orphanet Journal of Rare Diseases link --
Rare Toolkit: Rare Disease Registries: Advancing Disease Understanding, Treatments, and Cures Global Genes link --
PCORI Training: A Program for Rare Disease Patient Advocates Patient-Centered Outcomes Research Institute (PCORI) link --
Frequently Asked Questions (FAQs) - Registries World Federation of Hemophilia link --
Registries Tool Kit TREAT-NMD Neuromuscular Network link --
Fact Sheet - Rare Disease Patient Registries EURORDIS Rare Disease Europe link --
Developing a Patient Registry: A Practical Guide Medical Research Charities Group link --
1.2 Consider Your Constraints
Video: Institutional Review Boards (IRBs) U.S. Department of Health and Human Services link --
1.5 Develop a Governance Plan
Recommendations for Improving the Quality of Rare Disease Registries International Journal of Environmental Research and Public Health link --
Section II. Legal and Ethical Considerations for Registries Registries for Evaluating Patient Outcomes: A User's Guide [Internet]. link --

Continue to Step 2. Determine Who Should Join after you finish Step 1