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.