Evaluation of Patient and Physician Reported Reasons for Switching Factor VIII Replacement Therapies (PARkER)

Evaluation of Patient and Physician Reported Reasons for Switching FVIII Replacement Therapies

This US study will assess hemophilia A patient characteristics, health history and reasons for switching or not switching from both patient/caregiver and physician perspectives. For this purpose, this research study will include hemophilia A: 1) patients who have switched from conventional therapy to new FVIII products with an improved PK profile. 2) patients who remain on conventional therapy (who have never switched) but have considered switching, including those patients who switched from conventional therapy to new FVIII products with improved pharmacokinetics and then subsequently "switched back" to conventional replacement therapy. In doing so, real world evidence will be obtained from both patient and physician perspectives offering key insights for effective therapeutic management of patients with hemophilia A and to more fully understand what drives patient switching from a patient perspective and a physician perspective.

Study Overview

• Status: Completed
• Conditions: Hemophilia A
• Intervention / Treatment: Drug: FVIII products; Drug: Conventional FVIII replacement therapies

• Study Type: Observational
• Enrollment (Actual): 160

Contacts and Locations

Study Locations

• United States
  • New Jersey
    • Whippany, New Jersey, United States, 07981
      • Many Locations

Participation Criteria

Eligibility Criteria

• Ages Eligible for Study: 12 years and older (Child, Adult, Older Adult)
• Accepts Healthy Volunteers: No
• Genders Eligible for Study: All

• Sampling Method: Non-Probability Sample

Study Population

Study Population for the Patient/Caregiver Study:

Hemophilia A patients/caregivers will be recruited via opt-in patient panels and/or research databases.

Patients with hemophilia A (≥ 18 years of age) or caregivers of children with hemophilia A (≥12-<18 years of age)

Study Population for the Physician Chart Study:

The study population for the physician chart review study will be patients, who initiated FVIII products with improved half-life for the treatment of hemophilia A during the eligibility period. In order to obtain a broad patient population that is representative of real world prescribing; all eligible patients will be included in the study irrespective of the FVIII product with improved half-life received.

Description

Inclusion Criteria:

Inclusion criteria for patients/caregivers in the Patient/Caregiver Study:

• Patients with hemophilia A (≥ 18 years of age) or caregivers of children with hemophilia A (≥12-<18 years of age)
• Group 1: Hemophilia A patients/caregivers who have switched to FVIII products with improved half-life for the treatment of hemophilia A during the eligibility period. These patients can also include those who have switched back from FVIII products with improved half-life to conventional FVIII replacement therapy within the Data Collection Period
• Group 2: Hemophilia A patients/caregivers who are considering switching to FVIII products with improved half-life within 12 months of the Start of the Documentation period and have been prescribed prophylaxis regimen of at least 2x/week
• Able to understand, read, write and speak English
• Provide electronic informed consent
• Able to access the Internet for at least 20 minutes per day during the Data Collection Period

Inclusion criteria for physicians:

• At least 60% of time spent in direct patient care
• Board-certified or eligible with a Specialty in Hematology or Hematology-Oncology
• Physicians with a specialty in Hematology-Oncology must have at least 10% of their practice dedicated to treatment of hemophilia
• A minimum of 2 years' experience treating hemophilia A patients

Inclusion criteria for patients in the physician chart study:

• Hemophilia A patients age 12 year and over
• Prior treatment with one of the following FVIII replacement products: Adynovate, Afstyla, Eloctate, or Kovaltry
• Patients that have 12 months of medical chart data available; 6 months on conventional therapy and 6 months after switching to FVIII products with improved half-life.

Exclusion Criteria:

Exclusion criteria for patients/caregivers in the Patient/Caregiver Study:

- Hemophilia A patient initiated FVIII products with improved half-life for the treatment at time of diagnosis with hemophilia A.

Exclusion criteria for physicians:

- Unwilling to comply with the study protocol

Study Plan

How is the study designed?

Number of groups / cohorts

3

Cohorts and Interventions

Group / Cohort	Intervention / Treatment
FVIII products (prospective); Qualitative patient/caregiver study: Hemophilia A patients/caregivers (N=30) having initiated a FVIII products with improved half-life	Drug: FVIII products; Adynovate, Eloctate, Afstyla, Kovaltry; Drug: Conventional FVIII replacement therapies; Advate, Kogenate FS, Helixate, Novoeight, Nuwiq, Recombinate, Xyntha
Conventional FVIII replacement therapies; Qualitative patient/caregiver study: Hemophilia A patients/caregivers (N=30) receiving "conventional" FVIII replacement therapy for at least 6 months who are considering switching to a FVIII product with improved half-life within the next 1 year	Drug: Conventional FVIII replacement therapies; Advate, Kogenate FS, Helixate, Novoeight, Nuwiq, Recombinate, Xyntha
FVIII products (retrospective); Quantitative physician interview/ chart review study: Hemophilia A patients (N=100) who have switched from "conventional" FVIII replacement therapy to FVIII products with improved half-life.	Drug: FVIII products; Adynovate, Eloctate, Afstyla, Kovaltry; Drug: Conventional FVIII replacement therapies; Advate, Kogenate FS, Helixate, Novoeight, Nuwiq, Recombinate, Xyntha

What is the study measuring?

Primary Outcome Measures

Outcome Measure	Measure Description	Time Frame
The reasons of patients/caregivers "switch" from conventional FVIII replacement therapy to FVIII products with improved half-life	These patients/caregivers will participate in an asynchronous online discussion forum consisting of predetermined open ended and close ended questions for a series of 6 consecutive days completing approximately 20 minutes' worth of questions per day.	Up to 6 days
The obstacles of switching among hemophilia A patients who did not switch from conventional therapy to FVIII products with improved half-life but who are open to switching	These patients/caregivers will participate in an asynchronous online discussion forum consisting of predetermined open ended and close ended questions for a series of 6 consecutive days completing approximately 20 minutes' worth of questions per day.	Up to 6 days
The clinical characteristics of patients who switch from conventional FVIII replacement therapy to FVIII products with improved half-life	A retrospective patient medical chart review	Up to 4.5 months
The changes of treatment characteristics from 6 months prior to switching compared to 6 months after switching from conventional FVIII replacement therapy to FVIII products with improved half-life	A retrospective patient medical chart review	Up to 4.5 months
The changes of bleeding related outcomes from 6 months prior to switching compared to 6 months after switching from conventional FVIII replacement therapy to FVIII products with improved half-life	A retrospective patient medical chart review	Up to 4.5 months
The reasons for switching from conventional FVIII replacement therapy to FVIII products with improved half-life, from the physician perspective	A retrospective patient medical chart review	Up to 4.5 months

Collaborators and Investigators

• Sponsor: Bayer

Study record dates

Study Major Dates

• Study Start (Actual): February 14, 2018
• Primary Completion (Actual): June 13, 2018
• Study Completion (Actual): June 13, 2018

Study Registration Dates

• First Submitted: January 15, 2018
• First Submitted That Met QC Criteria: January 15, 2018
• First Posted (Actual): January 23, 2018

Study Record Updates

• Last Update Posted (Actual): September 27, 2018
• Last Update Submitted That Met QC Criteria: September 26, 2018
• Last Verified: September 1, 2018

More Information

Terms related to this study

• Additional Relevant MeSH Terms: Hematologic Diseases; Blood Coagulation Disorders, Inherited; Coagulation Protein Disorders; Hemorrhagic Disorders; Genetic Diseases, Inborn; Blood Coagulation Disorders; Hemophilia A; Coagulants; Factor VIII
• Other Study ID Numbers: 19529

Drug and device information, study documents

• Studies a U.S. FDA-regulated drug product: No
• Studies a U.S. FDA-regulated device product: No