Fraticelli Laurie, Freyssenge Julie, Promé-Combel Emilie, Agnellet Eléonore, Dargaud Yesim, Chamouard Valérie
Laboratory P2S (Health Systemic Process), UR 4129, University Claude Bernard Lyon 1, University of Lyon, Lyon, France.
Research on Healthcare Performance RESHAPE, INSERM U1290, University Claude Bernard Lyon 1, University of Lyon, Lyon, France.
JMIR Res Protoc. 2023 Mar 8;12:e43091. doi: 10.2196/43091.
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.
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.
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).
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.
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.
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.
艾美赛珠单抗（Hemlibra）自2019年商业化以来，仅在法国医院药房有售，用于预防有或无抑制物的甲型血友病。自2021年6月15日起，患者可以在医院药房和社区药房之间进行选择。护理途径的这些变化对患者、其亲属和卫生专业人员具有重要的组织影响。针对社区药剂师有两个培训项目：由国家血友病参考中心提出的“HEMOPHAR”培训项目和由该产品销售实验室提出的罗氏培训项目。
PASODOBLEDEMI研究旨在评估在艾美赛珠单抗配药背景下提供给社区药剂师的培训项目的直接影响，并评估患者对其治疗的满意度，无论他们选择从社区药房配药还是保留从医院药房配药。
我们基于四级柯克帕特里克评估模型设计了一项横断面研究：社区药剂师培训后的即时反应（反应）、培训期间获得的知识（学习）、社区药剂师在产品配药期间的专业实践（行为）以及患者对治疗的满意度，无论治疗是从医院还是社区药房配药（结果）。
考虑到单一结果指标无法充分反映这一新组织模式的复杂性，柯克帕特里克评估模型提供了4个不同的结果：HEMOPHAR培训项目后的即时反应、HEMOPHAR培训项目后获得的知识水平、培训对专业实践的影响以及患者对获得艾美赛珠单抗的满意度。我们为柯克帕特里克评估模型的4个级别分别制定了专门的问卷。所有参与艾美赛珠单抗配药的社区药剂师，无论他们是否参加了HEMOPHAR或罗氏培训项目，或两者都未参加，均符合纳入条件。所有重度甲型血友病患者均符合条件，无论是否使用抑制物、年龄、是否接受艾美赛珠单抗治疗以及他们是选择从社区药房配药还是保留从医院药房配药。
由于在罕见出血性疾病管理中存在严重和紧急出血情况的风险，在法国社区药房为甲型血友病患者配给艾美赛珠单抗的新组织模式必须伴有最佳的安全和质量条件。PASODOBLEDEMI方案的制定已经产生了积极影响，所有卫生专业人员、医生、医院和社区药剂师以及患者群体都参与其中。研究结果将在法国当局之间传播，并在必要时能够向其他罕见疾病推荐这种获取模式。
ClinicalTrials.gov NCT05449197，https://clinicaltrials.gov/ct2/show/NCT05449197?term=NCT05449197；ClinicalTrials.gov NCT05450640，https://clinicaltrials.gov/ct2/show/NCT05450640?term=NCT05450640。
国际注册报告识别码（IRRID）：DERR1-10.2196/43091。
