PHAREO study: Perceived and observed accessibility to therapeutic drugs used for treating patients with inherited bleeding disorders

Abstract

What Is Known and Objective The dispensing of clotting factor concentrates in hospital pharmacies imposes accessibility constraints on patients and their caregivers, thereby increasing the disease burden. Very few studies have addressed these issues so far in terms of individual perceptions and actual difficulties. The PHAREO study aims to report patient's perception of treatment accessibility and evaluate spatial accessibility. Methods The PHAREO study is an observational survey based on a questionnaire specifically designed for the study purpose in collaboration with patients' representatives in the second demographic and economic French region. Results and Discussion We collected 293 responses (participation rate of 64.1%) which show that 89.8% of respondents were either very or rather satisfied with regard to access to treatment. However, respondents reported difficulties in accessing the hospital pharmacy. The data also showed that 79.2% of respondents tended to over‐estimate travel time which was reported above their acceptable threshold for 39.2% of them. The main determinants of dissatisfaction were parental burden (OR 2.5 [1.3; 4.8], p = 0.008) and waiting time at the hospital pharmacy (OR 1.5 [1.1;2.0], p = 0.016, per 10 min increase). What Is New and Conclusion The PHAREO study provides subjective and objective data regarding satisfaction levels of persons with haemophilia and other coagulation deficiencies, with a high representativeness rate for patients on prophylaxis (87.5%). Both respondents and hospital pharmacists pled for an evolution of the current dispensing circuit to improve access to treatment and reduce the burden for patients. Currently, the community pharmacists are apart from the dispensing circuit. The authors propose improvements in the pathway of care for patients and their caregivers by including the community pharmacists alongside the hospital pharmacists in a centralized coordination scheme.

Evaluation of the care pathway in the context of the dispensing of emicizumab (Hemlibra) in community and hospital pharmacies in France: A patient satisfaction survey

“…A recent regional observational study reported that the main determinants of dissatisfaction were parental burden (OR 2.5 [1.3; 4.8], p = .008) and waiting time at the hospital pharmacy (OR 1.5 [1.1;2.0], p = .016, per 10 min increase). 5 With 77.2% of patients without inhibitor in community pharmacy compared to 61.5% in hospital pharmacy, the comparison is borderline significant (p = .0521). 24 This difference is probably due to the fact that patients without inhibitor are less severe, and consequently more incentive and confident to choose the community pharmacy and away from the hospital professionals.…”

“…Significant changes in treatment accessibility are expected by patients community and carers 2 to reduce territorial constraints 3,4 . As observed in a previous French study, both patients and hospital pharmacists pled for an evolution of the current dispensing circuit to improve access to treatment and reduce the burden of the disease, especially for active workers, parents and for patients living in rural areas 5 …”

“…3,4 As observed in a previous French study, both patients and hospital pharmacists pled for an evolution of the current dispensing circuit to improve access to treatment and reduce the burden of the disease, especially for active workers, parents and for patients living in rural areas. 5 Emicizumab (Hemlibra) is the first prophylactic treatment that can be administered subcutaneously in haemophilia A with or without antifactor VIII inhibitors. 6,7 These new administration modalities are easier to implement than previous Factor VIII treatments, which required sometimes frequent intravenous injections.…”

Evaluation of the Care Pathway in the Context of the Dispensing of Emicizumab (Hemlibra) in Community Pharmacies in France: Protocol for a Cross-sectional Study Based on the Kirkpatrick Model

“…The availability of emicizumab in community pharmacies represents a significant advancement of the care pathway for patients with hemophilia A. The direct expected benefit concerns the improvement of the patients’ satisfaction by reducing the mental burden due to the geographical and organizational constraints to hospital pharmacies [ 8 ]. As observed in the management of other diseases, distance to the pharmacy may play a role in pharmaceutical management, leading, for example, to inappropriate and inadequate medication and management of asthma for patients living further from their preferred pharmacy [ 25 ].…”

“…To maintain a sufficient level of clotting factor in the bloodstream to prevent bleeds, patients with severe hemophilia A are typically prescribed prophylactic treatment that consists of regular administration of FVIII, for example, every day, week, or month, according to how long the factor lasts in the body. Although effective, this treatment can only be administered intravenously, and in France, as well as in other countries [3][4][5][6], dispensation FVIII is only available from hospital pharmacies; they both contribute to organizational and geographical constraints [7] as well as a significant mental burden for patients and their caregivers, particularly for the parents of children with hemophilia [8].…”