Step 2.
Determine Who Should Join

2.0 Overview

Establish criteria to help you determine who is eligible to join your registry. Depending on your registry’s goals and constraints, you may want to recruit broadly or have a narrower focus. Your decisions will affect the cost and complexity of your registry, so keep in mind your short- and long-term goals. You may want to refer to RaDaR Tool: Participant Criteria Checklist.

2.1 Set Language Requirements

Decide what language(s) you will use for your registry. The simplest option is to use one language, but given that you’ll want to recruit as many people living with the rare disease as you to your registry,  consider translating your questions into multiple languages.

Consider:

  • What languages are spoken by patients and their caregivers?
  • How often is English used as either as a first or second language in the countries in which you are recruiting for your registry?
  • What language(s) will you use to contact registry participants?
  • Are you able to afford professional translators or find qualified volunteers?

One Language:

  • PROs: No translation is needed. Using a professional to translate your registry questions into another language can be costly. Using a native speaker as an informal translator can introduce errors or inconsistencies. Cultural or linguistic differences can also impact the way people interpret the registry questions. Work out all the “bugs” in your questions in one language before you translate them into other languages (otherwise, you’ll spend time and money using the translation services each time a change is needed as you work out the kinks). If you work out all the “bugs” first before translating the questions, you may be able to work with international rare disease patient advocacy groups to help fund translation into their language.
  • CONs: Some patients may not be able to participate. Multiple languages are used for registries involving participants all over the world. Especially when the patient population is very small, it can be important to include as many people as possible in the registry. If a participant does not speak the language, he or she will not be able to participate unless a friend or relative can complete the registry questions.

Multiple Languages:

  • PROs: More patients will be able to participate. Multiple languages are used for registries involving patients all over the world. Especially when the patient population is very small, it can be important to include as many people as possible in the registry.
  • CONs: Translation increases the cost and complexity of your registry. It may be difficult to identify translators for each language and assess their ability to be medically and technically proficient. It is important to ensure that the registry questions are translated consistently across the different languages. A translator will also need to convert all the answers back into one language.

2.2 Determine Geographic Location

Decide whether your registry will be national or international. To start, it may be easier to recruit only participants who live in the same country as you. To meet your registry goals, you may need to expand the geographic area to include participants from many countries (especially for an ultra-rare disease).

Consider:

  • How many people have the disease?
  • Where do participants live (which countries)?
  • How many participants do you need to meet your registry goals?
  • Are you familiar with the data security and privacy laws in other countries? (e.g., The EU General Data Protection Regulation (GDPR))

National:

  • PRO: An option if there are many participants living in one country.
  • CON: People from other countries may not be able to participate.

International:

  • PRO: An option if there are few participants in any one country.
  • CON: Multiple languages, time zones, and cultural differences will create additional challenges.

2.3 Establish Criteria for Diagnosis

Determine what type of diagnosis is required for joining your registry. Start with the requirement that participants have been diagnosed by a clinician. If needed, include additional diagnostic criteria.

Consider:

  • What is the typical method for making the diagnosis?
    • Clinical evaluation (medical history and physical examination)
    • Laboratory tests
    • Imaging tests
    • Diagnostic procedures
  • Do patients need to be diagnosed by a particular specialist (neurologist, geneticist, etc.)?
  • Are there any specific test results or clinical criteria required for an official diagnosis?

This type of information is considered protected health information (PHI), also known as personal health information, which is defined as all personally identifiable information (PII) such as demographic data (e.g., age, sex, ethnicity/race), medical histories, test results, doctor’s notes, and other information that could be used to identify a patient. The Privacy Rule of the Health Insurance Portability and Accountability Act of 1996 (HIPAA) creates a set of requirements and restrictions for the handling of PHI by certain “covered entity.” “Covered entities” include health plans, health care clearinghouses, and health care providers. In the case of most contact registries, HIPAA does not apply because patients or their parents or legal guardians are the ones providing the information directly to the registry. HIPAA may become important when collecting PHI for research registries. To read more about HIPAA and PHI, visit the U.S. Department of Health & Human Services (HHS) website.

Establish criteria to help you determine who is eligible to join your registry. Depending on your registry’s goals and constraints, you may want to recruit broadly or have a narrower focus. Your decisions will affect the cost and complexity of your registry, so keep in mind your short- and long-term goals. You may want to refer to RaDaR Tool: Participant Criteria Checklist.

Decide what language(s) you will use for your registry. The simplest option is to use one language, but given that you’ll want to recruit as many people living with the rare disease as you to your registry,  consider translating your questions into multiple languages.

Consider:

  • What languages are spoken by patients and their caregivers?
  • How often is English used as either as a first or second language in the countries in which you are recruiting for your registry?
  • What language(s) will you use to contact registry participants?
  • Are you able to afford professional translators or find qualified volunteers?

One Language:

  • PROs: No translation is needed. Using a professional to translate your registry questions into another language can be costly. Using a native speaker as an informal translator can introduce errors or inconsistencies. Cultural or linguistic differences can also impact the way people interpret the registry questions. Work out all the “bugs” in your questions in one language before you translate them into other languages (otherwise, you’ll spend time and money using the translation services each time a change is needed as you work out the kinks). If you work out all the “bugs” first before translating the questions, you may be able to work with international rare disease patient advocacy groups to help fund translation into their language.
  • CONs: Some patients may not be able to participate. Multiple languages are used for registries involving participants all over the world. Especially when the patient population is very small, it can be important to include as many people as possible in the registry. If a participant does not speak the language, he or she will not be able to participate unless a friend or relative can complete the registry questions.

Multiple Languages:

  • PROs: More patients will be able to participate. Multiple languages are used for registries involving patients all over the world. Especially when the patient population is very small, it can be important to include as many people as possible in the registry.
  • CONs: Translation increases the cost and complexity of your registry. It may be difficult to identify translators for each language and assess their ability to be medically and technically proficient. It is important to ensure that the registry questions are translated consistently across the different languages. A translator will also need to convert all the answers back into one language.

Decide whether your registry will be national or international. To start, it may be easier to recruit only participants who live in the same country as you. To meet your registry goals, you may need to expand the geographic area to include participants from many countries (especially for an ultra-rare disease).

Consider:

  • How many people have the disease?
  • Where do participants live (which countries)?
  • How many participants do you need to meet your registry goals?
  • Are you familiar with the data security and privacy laws in other countries? (e.g., The EU General Data Protection Regulation (GDPR))

National:

  • PRO: An option if there are many participants living in one country.
  • CON: People from other countries may not be able to participate.

International:

  • PRO: An option if there are few participants in any one country.
  • CON: Multiple languages, time zones, and cultural differences will create additional challenges.

Determine what type of diagnosis is required for joining your registry. Start with the requirement that participants have been diagnosed by a clinician. If needed, include additional diagnostic criteria.

Consider:

  • What is the typical method for making the diagnosis?
    • Clinical evaluation (medical history and physical examination)
    • Laboratory tests
    • Imaging tests
    • Diagnostic procedures
  • Do patients need to be diagnosed by a particular specialist (neurologist, geneticist, etc.)?
  • Are there any specific test results or clinical criteria required for an official diagnosis?

This type of information is considered protected health information (PHI), also known as personal health information, which is defined as all personally identifiable information (PII) such as demographic data (e.g., age, sex, ethnicity/race), medical histories, test results, doctor’s notes, and other information that could be used to identify a patient. The Privacy Rule of the Health Insurance Portability and Accountability Act of 1996 (HIPAA) creates a set of requirements and restrictions for the handling of PHI by certain “covered entity.” “Covered entities” include health plans, health care clearinghouses, and health care providers. In the case of most contact registries, HIPAA does not apply because patients or their parents or legal guardians are the ones providing the information directly to the registry. HIPAA may become important when collecting PHI for research registries. To read more about HIPAA and PHI, visit the U.S. Department of Health & Human Services (HHS) website.

Continue to Step 3. Develop the Right Questions to Ask after you finish Step 2