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

Table 1 Eligibility criteria for subject participation in the 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 moderate^a/severe^b 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 VIII^c
	• 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 hemophilia^b/ 25% moderate hemophilia^a
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 relationship^d 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

^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
^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
^cAn immediate family member or other non-professional caregiver (e.g. friend/neighbor)
^dWorks for/consults with/serves on the advisory board of/holds any ownership position (excluding stock)

ISSN: 1750-1172