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

Hemophilia A Clinical Trial

— PASODOBLEDEMI2  

Official title:

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

Clinical Trial Details — Status: Completed

Administrative data

• NCT number: NCT05450640
• Other study ID #: PASODOBLEDEMI2
• Status: Completed
• Start date: January 9, 2023
• Est. completion date: January 9, 2023

Study information

• Verified date: March 2023
• Source: Hospices Civils de Lyon
• Contact: n/a
• Is FDA regulated: No
• Study type: Observational

Clinical Trial Summary

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

Recruitment information / eligibility

• Status: Completed
• Enrollment: 176
• Est. completion date: January 9, 2023
• Est. primary completion date: January 9, 2023
• Accepts healthy volunteers: No
• Gender: All
• Age group: 0 Years and older

Eligibility

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

Related Conditions & MeSH terms

• Hemophilia A

Intervention

Other:
• satisfaction survey
1 eQuestionnaire per patient completed during study

Locations

Country	Name	City	State
France	Hospices Civils de Lyon	Bron

Sponsors (1)

Lead Sponsor	Collaborator
Hospices Civils de Lyon

Country where clinical trial is conducted

France, 

Outcome

Type	Measure	Description	Time frame	Safety issue
Primary	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