Adding a Clinical Hemophilia Pharmacist to the Hemophilia Comprehensive Care Model Improves Health Care–Related Outcomes and Drug-Related Costs in an Integrated Health Care System

Abstract

Introduction

The National Hemophilia Foundation has advocated for a comprehensive care model in caring for patients with hemophilia. Currently pharmacists are not included as part of the team, and the impact of specific team members on patient outcomes is unknown. The authors designed an intervention to add a clinical pharmacist to this care model. The authors evaluated the impact of this intervention on bleeding outcomes, medication access and adherence, and cost within an integrated health care system.

Methods

The authors performed a multicenter, retrospective analysis of all hemophilia A and B patients enrolled in the hemophilia pharmacy service between March 2017 and February 2019. The authors analyzed data prior to and after this quality improvement project, encompassing the period between March 2016 and February 2020. Primary outcomes included an evaluation of the annualized bleed rates, emergency department visits, and hospitalizations.

Results

In the cohort of patients with hemophilia A and B, the overall bleed rate was reduced from 40 per 100 patient-years to 37.6 per 100 patient-years. Similarly, emergency department visits were reduced from 22.7 to 18.3 per 100 patient-years, and hospitalizations reduced from 6.4 to 3.0 per 100 patient-years (p values >.05). This was augmented by the use of a unique factor recycling program that saved $900,000 in medication costs over a 12-month period.

Discussion

This study shows that the addition of a clinical pharmacist to the core hemophilia team leads to positive trends toward improved bleeding outcomes for patients, improved medication access and adherence, and substantial cost savings to the health system.

Introduction

The National Hemophilia Foundation (NHF) has advocated for a comprehensive care model when caring for patients with hemophilia. The core members of this team include a hematologist, nurse hemophilia specialist, physical therapist, social worker, and specialized coagulation laboratory. ¹ The NHF–McMaster Guideline on Care Models for Hemophilia Management provided a conditional recommendation, with low certainty of evidence, suggesting utilization of a care team involving these specialists over an integrated care team that does not include these components. ² The goals of such programs are reducing overall bleed rates, patient and family education on appropriate factor dosing, addressing barriers to factor use, coordinating perioperative care, and improving quality of life and overall survival. ³ However, there is little published evidence evaluating the impact of these team members on clinical outcomes, and pharmacists are often not included in the above list of core hemophilia specialists. Here the authors describe the experience of a systems-based quality improvement initiative where clinical hemophilia pharmacists were added to a core hemophilia management team, and the impact of this initiative on patient care, medication access, drug adherence, and cost within an integrated health care system.

Methods

The setting is a multispecialty care delivery system encompassing 15 medical center service areas, each with their own hematology/oncology department. The care of hemophilia patients was facilitated through collaborative networks across medical centers with variability in patient follow-up, based on geography and care team composition. In 2017, hemophilia pharmacists were added to all the core teams, with pharmacist operations based in a centralized location providing telemedicine services to patients. The pharmacists served as an additional point of contact for patients and as a resource for education, and were able to streamline access to financial assistance programs. The pharmacists’ role augmented the work of the care team by taking extensive bleeding histories and developing comprehensive plans to provide appropriate factor replacement during bleeding episodes, prophylaxis of bleeding events, and individualized perioperative plans. The added clinical services included enhanced intensive management such as additional touch points in between routine follow-ups, ordering specialized coagulation labs on behalf of practitioners, and tailoring a patient’s medication selection. With each added encounter, patients were assured of timely medication refills, and medication adherence was emphasized. The pharmacist specialists had access to the medication vendor’s inventory and were able to personalize therapy by selecting factor vial sizes closest to the patient’s target dose while minimizing factor product waste, a feature of the program facilitated by the integrated nature of the health care system. A unique part of the service was the deployment of a factor recycling program, which involved regionwide pharmacy inventory tracking and dispensing of unused, soon-to-expire products to patients on routine prophylaxis to minimize factor product waste.

To evaluate the impact of this intervention on patient-related outcomes and drug costs, the authors performed a multicenter, retrospective analysis of all hemophilia patients enrolled in the hemophilia pharmacy service between March 2017 and February 2019. The Kaiser Permanente Northern California institutional review board approved the study and waived informed consent according to the Common Rule. The authors analyzed data prior to and after the quality improvement intervention began, encompassing the period between March 2016 and February 2020. The bleed rate was calculated as the incidence (new cases) of bleeds per 100 patient-years. Medication possession ratio (MPR) measures the percentage of time a patient has access to medication, with the calculation capped at 1.0. A MPR value of 0.80 means that patients had medication supply available for 80% of days within the period based on medication dispensed. Comparisons of pre and post bleed rates were performed using a paired t-test for continuous data and McNemar test for paired nominal data. The level of significance was established a priori with a p value of <0.05. These primary outcomes included an evaluation of the annualized bleed rates, outpatient encounters, emergency department (ED) visits, and hospitalizations. The study included male patients diagnosed with hemophilia A or B. The authors excluded patients who did not have health plan membership coverage, had a diagnosis of acquired hemophilia, or who were carriers for hemophilia. The study was comprised of 110 patients who met the inclusion criteria and were representative of hemophilia patients in the general population. Baseline characteristics included a mean age of 31.9 years, and the ratio of hemophilia A to B was 4:1, with 21% having severe disease requiring prophylaxis (Table 1). The vast majority (95.5%) of patients had a postperiod follow-up of 365 d. The mean postperiod follow-up was 327 d (range 88–365), with a median of 365 d (Interquartile range 337, 365).

Table 1:

Baseline characteristics

Characteristic	Study group (N = 110)
Age in years, mean (range)	31.9 (0.5–80.7)
Male gender, n (%)	110 (100)
Hemophilia A, n (%)	89 (80.9)
Hemophilia B, n (%)	21 (19.1)
Prophylaxis treatment, n (%)	23 (21)
Positive inhibitor status in preperiod, n (%)	2 (1.8)

Results

The primary outcomes are summarized in Table 2 and show that there was a trend toward a reduction in the annual bleed rate, hospitalizations, and ED visits. The overall bleed rate was reduced from 40 per 100 patient-years to 37.6 per 100 patient- years (p = 0.78). Similarly, ED visits were reduced from 22.7 to 18.3 per 100 patient-years (p = 0.48), and hospitalizations were reduced from 6.4 to 3.0 per 100 patient-years (p = 0.27). There was an increase in clinic-managed bleeds, likely because of higher reported joint bleeds. Additionally, there was a positive trend in the number of patients adherent to prophylaxis treatment, with MPR < 0.80. Among 18 patients with severe hemophilia on routine ongoing prophylaxis with outpatient prescription dispensing information available, 9 (50%) patients had an MPR < 0.80 in the prestudy period, whereas 11 (61%) patients had an MPR < 0.80 in the poststudy period (p = 0.32). The pharmacists were able to use available factor doses more efficiently through the factor recycling program and personalize vial size selection directly from vendors, leading to tighter inventory control, reduction in factor expiration, and improved formulary concordance. The estimated savings from this program was approximately $900,000 in medication costs over a 12-month period between September 2019 and August 2020. Of the 89 patients with hemophilia A, 10 converted their therapy to emicizumab, a novel subcutaneous fixed-dose weekly injection, during the study period. Emicizumab recently gained US Food and Drug Administration approval for prophylaxis in patients with hemophilia A and is a more convenient, though higher-cost, option for health care systems. ⁴ This additional increase in acquisition cost was factored into the benefits from the factor recycling program to arrive at the total overall medication cost savings of $900,000.

Table 2:

Outcomes summary

Outcome	Preperiod	Postperiod	p value
Annual bleed rate a	40.0	37.6	0.78
Annual joint bleed rate a	18.2	19.3	0.85
Utilization of services for bleed management a
Clinician outpatient encounters	22.7	29.5	0.34
ED visits	22.7	18.3	0.48
Hospitalizations	6.4	3.0	0.27
Prophylaxis treatment (n = 18)
MPR ≥ 0.80, n (%)	9 (50)	11 (61)	0.32

^a Incidence of new bleed cases per 100 patient-years.

ED, emergency department; MPR, medication possession ratio.

Discussion

This study shows that the addition of a clinical pharmacist with expertise in hemophilia leads to positive trends toward improved bleeding outcomes for patients, improved medication adherence, and substantial drug cost savings to the health system. Although clinical outcome improvements in this study were not statistically significant, this may have been because of the baseline use of a core comprehensive care team and the small sample size of this study. The improvement in communication and additional touch points added by the pharmacy specialists led to earlier identification of bleeds, and therapy being administered in the clinic rather than the ED. A reduction in ED visits reduces the overall cost of care for members by affecting copayments, as well as utilization of emergency services within the health care system. A unique feature of the program was personalized factor vial size selection and a factor recycling program, which allowed for more efficient use of valuable medication prior to its expiration. Emicizumab is a more convenient fixed-dose option for hemophilia A treatment with a cost higher than that of Factor VIII infusions, which likely blunted the total savings of the factor recycling program had conversions not taken place. Though emicizumab has had an impact on the current management of hemophilia A, and gene therapy is poised to alter the paradigm of care, these will continue to be taken up gradually as real-world experiences are published, especially in resource-poor environments. ^5–7 There will therefore continue to be a need for traditional factor infusion therapy and ongoing management through a traditional core team, because emicizumab is only for prophylaxis and not indicated for on-demand bleeding or perioperative management. The goal of this initiative was to utilize a systems-based approach to improve access to expertise in hemophilia care and utilize factor therapy in a more consistent and cost-effective manner, while improving patient outcomes. ^8–10 Pharmacists added tangible value to the care of hemophilia patients and were able to provide stability in patients’ care during transient periods of core team staffing turnover, given their expertise overlapped that of other core team members. Future studies may explore the value of core team members in a randomized fashion to establish the cost effectiveness of their impact on quality of life and clinical bleeding outcomes.

Conclusions

This experience shows the addition of a pharmacist to a hemophilia comprehensive care model leads to improved patient outcomes and drug cost savings, and this model may prove beneficial for other integrated health care systems.