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Table 1 Eligibility criteria for subject participation in the study

From: Optimizing language for effective communication of gene therapy concepts with hemophilia patients: a qualitative study

Hematologists Nurses
• Primary specialty in either hematology or hematology/oncology • Qualified as a Registered Nurse or Nurse Practitioner
• Board certified/eligible (applies to US physicians only) • Actively involved in the management of hemophilia
• Has been in practice for 2–35 years • Has been in practice for between 4 and 30 years
• Spends a minimum 50% of time in direct patient contact • Spends a minimum 75% of time in direct patient contact
• More than 25% of patients are moderatea/severeb hemophilia patients  
• 50% of patients must receive prophylactic treatment with factor VIII  
Hematologists and nurses
• Is involved in the care of 10 or more hemophilia A patients (in US; 5 or more in other countries)
• Spends more than 20% of work time in a hemophilia treatment/care center
Target quotas
At least 50% of hematologists and nurses to be involved predominantly in treating adult patients. No more than 2 hematologists to be involved in a clinical trial of gene therapy or emicizumab
Patients Caregivers
• Age 18 to 60 years • Age 18 to 60 years
• Diagnosis of hemophilia A • Is primary caregiver of someone diagnosed with hemophilia A
• Must be on prophylactic standard or extended half-life factor VIII • Is caring for someone on prophylactic standard or extended half-life factor VIIIc
  • Is not a professional caregiver
  • Has not been a physician, physician’s assistant, pharmacist, mental health professional, nurse practitioner, or registered/licensed nurse
Age distribution to be evenly split between 18–30, and 31–60 years Aimed for 75% caregivers of severe hemophiliab/ 25% moderate hemophiliaa
No more than 1(2) US (EU5) participants with factor VIII inhibitors No more than 1 participant caring for someone who has developed Factor VIII inhibitors
Target quotas
No more than 1 patient and 1 caregiver of a patient per country enrolled in emicizumab trial. No more than 10% of patients and caregivers to be highly involved in hemophilia patient groups (as organizer, advocate, or speaker); no more than 30% of patients to be regular attendees of patient group activities
All participants
• Does not (and close family members do not) have a relationshipd with any of the following types of companies
 - Medical equipment manufacturer
 - Market research or advertising firm
 - Marketing or healthcare consulting firm
 - Local, state, or federal government
• Has not participated in market research in the past 12 months
  1. aModerate defined as factor VIII activity between 1 IU/dL and 5 IU/dL (normal range 50–150 IU/dL); bleeds occur upon injury; occasional breakthrough/spontaneous bleeds; treatment can be on-demand or prophylactic
  2. bSevere defined as factor VIII activity less than 1 IU/dL; bleeds occur upon injury; generally have frequent breakthrough/spontaneous bleeds without treatment (once a month or more); on prophylactic treatment to control bleeds
  3. cAn immediate family member or other non-professional caregiver (e.g. friend/neighbor)
  4. dWorks for/consults with/serves on the advisory board of/holds any ownership position (excluding stock)