Satisfaction Regarding the Dispensing of Emicizumab (HEMLIBRA®) for Patients With Haemophilia A in France (PASODOBLEDEMI2)

Evaluation of the Evolution of the Patient Care Pathway in the Context of the Implementation of the DOuBLE EMIcizumab Dispensing Circuit in France

Haemophilia A (HA) is a rare constitutional haemorrhagic disease whose drug management is based on the use of chronic lifelong replacement therapy. The occurrence of an inhibitor is a dreaded complication that impacts conventional management, consisting in using factor VIII (FVIII)-based replacement therapy, most often for prophylaxis. Although effective, these treatments can only be administered intravenously, leading to accessibility constraints and significant mental burden for patients and their relatives.

Before June 15, 2021 in France, the emicizumab (HEMLIBRA®) was available only in hospital pharmacies for the prevention or reduction of bleedings. The introduction of the dual dispensing circuit in hospital or community pharmacies, left to patient's choice, is effective from this date. These changes have important organizational consequences for patients and health professionals alongside the pathway of care. Therefore, the effectiveness of this new organization requires to be evaluated with a national French study, called PASO DOBLE DEMI. The aims of this study are twofold :

I. To evaluate the direct impact of the training programs provided to the new placeholders of the dispensing circuit ; the community pharmacists, II. To evaluate satisfaction of patients or their relatives regarding the emicizumab treatment whether they chose dispensing in the community pharmacy, or kept the dispensing at the hospital pharmacy.

The methodology was based on the 4-level of the Kirkpatrick's evaluation model; 1) the immediate reaction of community pharmacists following the trainings (Reaction), 2) their knowledge acquisition (Learning), 3) their professional practice (Behavior) and 4 ) the patients' satisfaction related to their treatment whether dispensing in hospital or in community pharmacies (Results).

The PASO DOBLE DEMI II study was based on the fourth level of the evaluation model and particularly to evaluate to what extent the dispensing of Emicizumab (HEMLIBRA ®) treatment in community pharmacies has contributed to the improvement of the satisfaction of patients with HA.

The availability of the treatment in community pharmacy assumes an improvement of the treatment accessibility for the patient at several levels :

• Global accessibility: what choice has the patient expressed to access his treatment from a practical point of view?
• Adaptation: Has the patient adapted to the new treatment delivery organization?
• Availability of resources: are the special needs of the patients taken into consideration?
• Social acceptability: is the patient willing to follow the proposed care pathway? The issue of treatment cost is not considered in this study because the cost of antihemophilic treatments is totally covered by the French government for all patients regardless of their insurance coverage

Study Overview

• Status: Completed
• Conditions: Hemophilia A
• Intervention / Treatment: Other: satisfaction survey

• Study Type: Observational
• Enrollment (Actual): 176

Contacts and Locations

Study Locations

• France
  • Bron, France
    • Hospices Civils de Lyon

Participation Criteria

Eligibility Criteria

• Ages Eligible for Study: 1 second and older (Child, Adult, Older Adult)
• Accepts Healthy Volunteers: No
• Genders Eligible for Study: All

• Sampling Method: Non-Probability Sample

Study Population

With approximately one in 6,000 men with haemophilia A, we estimated about 2,900 patients in France with 35% of cases with severe haemophilia, 15% of cases with moderate haemophilia and 50% of cases with minor severity. The eligible population for inclusion in the study considered all patients, regardless of age, treated with Emicizumab (HEMLIBRA®), whether they choose dispensing in community pharmacies, or retained dispensing in hospital pharmacy. The Laboratory Roche-Chugai which commercialize Emicizumab (HEMLIBRA®) estimated about 1'000 patients eligible to the inclusion criteria. We assumed that the uptake of the dispensing circuit, and therefore the number of patients choosing dispensing in community pharmacies, approximated the number of trained pharmacists.

Description

Inclusion Criteria:

• Patient with hemophilia type A with or without inhibitors
• Patients treated with Emicizumab (Hemlibra®)
• Age over 18 years old or legal representative of a minor haemophilia type A patient or his/her relative/caregiver for a non-autonomous adult patient

Exclusion Criteria:

• Patient under guardianship / curatorship
• Opposition expressed to use personal data

Study Plan

How is the study designed?

Design Details

• Observational Models: Other
• Time Perspectives: Cross-Sectional

Number of groups / cohorts

2

Cohorts and Interventions

Group / Cohort	Intervention / Treatment
Patients with haemophilia A with dispensing Emicizumab (HEMLIBRA®) in community pharmacy; This group of patients have chosen the dispensing of Emicizumab (HEMLIBRA®) in community pharmacy. This choice involves the contribution of the community pharmacist in the dispensing circuit who does not replace the hospital pharmacist but interact together for prescriptions renewals, emergency treatment or sharing information if necessary.	Other: satisfaction survey; 1 eQuestionnaire per patient completed during study
Patients with haemophilia A with dispensing Emicizumab (HEMLIBRA®) in hospital pharmacy; This group of patients have kept the dispensing of Emicizumab (HEMLIBRA®) in hospital pharmacy	Other: satisfaction survey; 1 eQuestionnaire per patient completed during study

What is the study measuring?

Primary Outcome Measures

Outcome Measure	Measure Description	Time Frame
satisfaction levels reported through a specific questionnaire	This questionnaire is composed of 40 questions for an estimated time to fulfil not exceeding 8 minutes. The overall satisfaction levels consists in collecting the patient's perception based on the 4-item of the Likert-scale (strongly dissatisfied, rather not satisfied, rather satisfied, strongly satisfied) taking into account the 4 dimensions of accessibility (global accessibility, adaptation, availability of resources, social acceptability).	6 months

Collaborators and Investigators

• Sponsor: Hospices Civils de Lyon

Study record dates

Study Major Dates

• Study Start (Actual): January 9, 2023
• Primary Completion (Actual): January 9, 2023
• Study Completion (Actual): January 9, 2023

Study Registration Dates

• First Submitted: July 5, 2022
• First Submitted That Met QC Criteria: July 5, 2022
• First Posted (Actual): July 11, 2022

Study Record Updates

• Last Update Posted (Actual): March 3, 2023
• Last Update Submitted That Met QC Criteria: March 2, 2023
• Last Verified: March 1, 2023

More Information

Terms related to this study

• Keywords: Patient satisfaction; Hemophilia A; Accessibility; Emicizumab (HEMLIBRA®)
• Additional Relevant MeSH Terms: Hematologic Diseases; Blood Coagulation Disorders, Inherited; Coagulation Protein Disorders; Hemorrhagic Disorders; Genetic Diseases, Inborn; Blood Coagulation Disorders; Hemophilia A
• Other Study ID Numbers: PASODOBLEDEMI2

Plan for Individual participant data (IPD)

• Plan to Share Individual Participant Data (IPD)?: NO

Drug and device information, study documents

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