Abstract
BACKGROUND: Commercialized since 2019, emicizumab (Hemlibra) was available only in French hospital pharmacies for prophylaxis of hemophilia A with or without inhibitors. Since June 15, 2021, patients can choose between a hospital and community pharmacy. These changes in the care pathway have important organizational consequences for patients, their relatives, and health professionals. Two training programs are available for community pharmacists: the "HEMOPHAR" training program proposed by the national reference center for hemophilia and the Roche training program proposed by the laboratory that markets the product. OBJECTIVE: The PASODOBLEDEMI study aims to evaluate the direct impact of the training programs provided to community pharmacists in the context of the dispensing of emicizumab, and to evaluate patients' satisfaction with their treatment whether they choose dispensation from a community pharmacy or retained dispensation from the hospital pharmacy. METHODS: We designed a cross-sectional study based on the 4-level Kirkpatrick evaluation model: the immediate reaction of community pharmacists following training (Reaction), the knowledge acquired during the training (Learning), the professional practice of community pharmacists during dispensing of the product (Behavior), and patients' satisfaction related to the treatment whether it is dispensed from a hospital or from a community pharmacy (Results). RESULTS: Considering that single outcome measures cannot adequately reflect the complexity of this new organization, the Kirkpatrick evaluation model provides 4 distinct outcomes: the immediate reaction after the HEMOPHAR training program, the level of knowledge acquired after the HEMOPHAR training program, the impact of training on professional practice, and patient satisfaction with access to emicizumab. We developed specialized questionnaires for each of the 4 levels of the Kirkpatrick evaluation model. All community pharmacists involved in dispensing emicizumab, whether they have followed the HEMOPHAR or the Roche training program or neither, were eligible for inclusion. All patients with severe hemophilia A were eligible, irrespective of inhibitor use, age, treatment with emicizumab, and whether they chose dispensation from a community pharmacy or retained dispensation from a hospital pharmacy. CONCLUSIONS: The new organization for dispensing emicizumab to patients with hemophilia A in French community pharmacies must be accompanied by optimal safety and quality conditions due to the risk of serious and urgent bleeding situations in the management of rare bleeding diseases. The elaboration of the PASODOBLEDEMI protocol has already a positive impact with the commitment of all health professionals, physicians, hospital and community pharmacists, and the patient community. The results will be disseminated among the French authorities and will enable, if necessary, proposing this access model to other rare diseases. TRIAL REGISTRATION: ClinicalTrials.gov NCT05449197, https://clinicaltrials.gov/ct2/show/NCT05449197?term=NCT05449197; ClinicalTrials.gov NCT05450640, https://clinicaltrials.gov/ct2/show/NCT05450640?term=NCT05450640. INTERNATIONAL REGISTERED REPORT IDENTIFIER (IRRID): DERR1-10.2196/43091.