Some of the possible goals of a patient registry are:

• Creating a list of patient contacts

• Engaging the rare disease population by obtaining their perspective, opinions, and experiences

• Recruiting patients for clinical trials

• Studying natural history data, which involves understanding the progression of a disease over time so that the full picture of the condition can be understood (1).

Pros:

• Contribute to the overall knowledge of the condition/disease

• Help further the understanding of the perspectives and opinions of those affected

• Inform healthcare providers and the public

• Contribute to building the community of those affected by the same condition/disease

• Feel good knowing their data helps research, treatment, or cure for their disease

• Possibility of contact for clinical trials

Cons:

• Participating in a patient registry does not guarantee a cure, treatment, or participation in a clinical trial

• Patients may never hear back or know if data was used for anything, which can be disappointing (2)

• Time commitment is required in order to complete the questionnaires

• Who owns the data?

• How much of a time commitment would participation in the registry require?

• Who enters the data? (patient, doctor)

• What will the data be used for?

• How is my data protected?

• Who can see my data and for what purpose?

• Who will see my de-identified vs. personally identifiable (name, email, etc..) information?

• Will I be contacted with updates regarding the use of my data?

• What happens to my data if the registry closes?

• What type of data will be collected?

• Who can I ask questions about my participation in the registry?

• Who can I contact if I have any concerns?

Here are some key steps and things to keep in mind when starting a patient registry:

Define goals of the registry.

In order to build a successful registry, it is crucial that goals of the registry are defined and understood from the beginning and clearly communicated to all stakeholders.

Create proper governance structure and oversight.

Consider including roles such as researchers, technical experts, patients, ethical oversight and legal adviser (3).

Select a registry platform.

A registry platform is the software service that will host your patient registry. It can vary from a simple electronic spreadsheet or a large interactive database collection service that can be integrated with electronic medical records (4).

As described in the Patient Registry Design Guide for Rare Disease Foundations, some considerations in selecting a registry platform are:

• Can data be entered by patients, clinicians, or both?

• What data standards are used, and will the platform comply with regulatory agencies such as the Food and Drug Administration (FDA) and European Medicines Agency (EMA)?

• Should data be entered and collated into the same registry by multiple organizations that support the same disease?

• How is ownership of the data decided and governed?

• Is there a need to accept electronic health records?

• Is informed consent capability included?

• What types of devices (phone, tablet, laptop, desktop, etc.) can be used?

• Are alerts and reminders available to prompt data collection?

Technical expertise

An enormous amount of technical expertise is needed in order for your registry to result in useful and meaningful data that can be used for analysis, research, and drug development. Therefore it is absolutely vital to include necessary technical resources from the very beginning in order to evaluate and select an appropriate platform, design surveys, and look at the data from the start of data collection in order to identify any issues early on.

Transparency, responsibility, and trust

It is important to remember and recognize the participants that place their hope and trust in the registry. Open, honest, and transparent communication with the patient community should be fundamental to running a registry and is a major responsibility (5).

Independent Ethics Review

In the U.S., all studies involving human participants require oversight by an Institutional Review Board (IRB). The role of an IRB is to protect human rights. Specifically they:

• Assure protection of the rights and welfare of human subjects

• Review and approve the initiation of, and periodically revisit, biomedical research involving human subjects

• Assess Risk-Benefit of the procedures being used

Even if your registry is not collecting medical information or records, or studying any intervention, IRB approval may be needed to ensure adequate steps are taken to protect the privacy of participants (6).

General Information​​​

• Patient Registry Design Guide for Rare Disease Foundations by Sophia Zilber and Jason Colquitt

• RaDaR (nih.gov)

• Patient Registries - Registries for Evaluating Patient Outcomes - NCBI Bookshelf (nih.gov)

• Three Questions to Consider when Future-Proofing a Registry - Pulse Infoframe Inc.

• What is Data Governance? - RARE-X (rare-x.org)

Data Standards

• Data Standards and RARE-X - RARE-X (rare-x.org)

Ethics, Data Ownership, Privacy, and Transparency​

• Principles of Registry Ethics, Data Ownership, and Privacy

• Registry Transparency, Oversight, and Data Ownership​

Patient Perspectives

• Patients are more than just data points - Seth Rotberg

• Bringing Data Back To Patients - RARE-X (rare-x.org)