Clinical trial NCT05450640: Satisfaction Regarding the Dispensing of Emicizumab (HEMLIBRA®) for Patients With Haemophilia A in France
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
Study type: OBSERVATIONAL
Overall status: COMPLETED
Conditions: Hemophilia A
Keywords: Hemophilia A
Accessibility
Emicizumab (HEMLIBRA®)
Patient satisfaction
Lead sponsor: Hospices Civils de Lyon
Sponsor class: OTHER
First posted: 2022-07-11
Enrollment: 176
Sex: ALL
Minimum age (years): 0
Standard ages: CHILD
ADULT
OLDER_ADULT
Accepts healthy volunteers: no
Observational model: OTHER
Time perspective: CROSS_SECTIONAL
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.

Brief 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


Eligibility criteria: 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

Primary outcomes:
measure: satisfaction levels reported through a specific questionnaire
timeFrame: 6 months
Secondary outcomes:
Other outcomes:
Locations:
city: Bron
country: France
facility: Hospices Civils de Lyon
Study officials:
affiliation: Hospices Civils de Lyon
name: Valérie Chamouard
role: PRINCIPAL_INVESTIGATOR
References: