1. Introduction
Prophylactic treatment with coagulation factors in patients with hemophilia A and B reduces the number of bleeds and their consequences on joints and organs, thus increasing the length and quality of life of patients.1,2 Nevertheless, it is associated with significant costs to the health care system. In Canada, coagulation factors are purchased and distributed by Hema-Québec, for the Province of Québec, and by Canadian Blood Services for all the other Provinces and Territories. Funding is provided entirely by the Provincial and Territorial governments. Whether recombinant or plasma-derived therapies, factors are expensive drugs that follow complicated pharmacokinetic (PK) trajectories making it a challenge to use them in an optimal way.3
With the introduction of new extended half-life products (EHL), efficient management of coagulation factors has become even more challenging. Indeed, many of these newer products have considerable inter-individual variation in half-life,4,5 such as the new factor VIII EHL product, Adynovate™. Adynovate™ has been shown in clinical trials to have a half-life that ranges from 6 to 30 hours, depending on the patient. Thus, initiating therapy using the standard dosing regimen, as prescribed in the product monograph, rather than optimising the individual treatment plan is very cost inefficient. Effective management of these products therefore requires tailoring treatment to the patient. To do so, it is essential to assess and interpret the PK profiles of patients. Recently, the Web-Accessible Population Pharmacokinetic Service-Hemophilia (WAPPS-Hemo) web-based platform based at McMaster University, which allows individual PK assessments on a limited number of plasma samples, has become readily available for clinicians to use7. At Children’s Hospital of Eastern Ontario (CHEO), we have started to routinely perform PK assessments for hemophilia patients, particularly when the type of product is changed, but not systematically due to ressource limitations, including time constraints.
To be performed systematically, PK assessment and interpretation requires sufficient time to make extensive arrangements, ensure patient availability and coordinate a time to review the data and results with the care team. A dedicated pharmacist could greatly assist this process by standardizing the approach and assisting with the interpretation of the PK results. Since the therapy for patients with moderate to severe hemophilia costs $50,000-500,000 CAD/year/patient, small changes could result in significant cost savings. For instance, two US studies have shown that involvement of a pharmacist in the management of recombinant and plasma derived factor therapies for hemophilia improves patient management and reduces associated costs.8,9
As in Canada, in the US pharmacists are not typically part of the comprehensive care team for the hemophilia population. The Hemophilia Management Program8 and The Factor Stewardship Program9 were conducted to evaluate the impact of including a pharmacist in the management of hemophilia patients. This gave the pharmacist the opportunity to participate in rounds, multidisciplinary meetings and regular patient assessments. Due to this close contact with patients and collaboration with the team, the pharmacist was able to identify and propose therapeutic optimizations, including dose/frequency adjustments, to improve drug dispensing, drug administration and also reduce wastage. One of the pharmacist’s roles was to provide guidelines and training to the care team in order to increase and update the knowledge of each staff member involved in the management of patients with hemophilia. The pharmacist was also involved in coordinating the transition of care to the community by facilitating the flow of information with the pharmacy service. The combination of these activities allowed the pharmacist to improve the management of coagulation factors in patients with clotting disorders, which led to a reduction in the quantity of factor being used and significantly reduced the cost associated with therapy. Thus, the implementation of a pharmacist working as part of the transfusion medicine team in our institution could maximize patient outcomes while minimizing the cost of these expensive therapies.
The purpose of this pilot project was to determine whether a pharmacist working as part of the Hemophilia Treatment Centre (HTC) would render the management of coagulation factors more cost effective and to identify the pharmacist’s activities associated with this new role. The intent of incorporating a pharmacist as part of the care team was to optimise protocols and maximize cost savings through reduced wastage while maintaining patient outcomes.