Development, implementation, and evaluation of a health outcomes and research program at an integrated health-system specialty pharmacy

Abstract

Purpose

Health-system specialty pharmacies (HSSPs) provide high-quality, efficient, and collaborative care to patients receiving specialty therapy. Despite proven benefits of the integrated model, manufacturer and payer restrictions challenge the viability and utility of HSSPs. Vanderbilt Specialty Pharmacy developed a health outcomes and research program to measure and communicate the value of this model, drive improvement in patient care delivery, and advocate for recognition of HSSP pharmacists’ role in patient care. The purpose of this descriptive report is to describe the development and results of this program.

Summary

The health outcomes and research program began as an initiative for pharmacists to evaluate and convey the benefits they provide to patients, providers, and the health system. Early outcomes data proved useful in communicating the value of an integrated model to key stakeholders and highlighted the need to further develop research efforts. The department leadership invested resources to build a research program with dedicated personnel, engaged research experts to train pharmacists, and fostered internal and external collaborations to facilitate research efforts. As of March 2021, the health outcomes and research program team has published 33 peer-reviewed manuscripts, presented 88 posters and 7 podium presentations at national conferences, and received 4 monetary research awards. Further, the program team engages other HSSP teams to initiate and expand their own health outcomes research in an effort to empower all HSSPs in demonstrating their value.

Conclusions

The health outcomes and research program described has pioneered outcomes research among HSSPs nationwide and has proven valuable to specialty pharmacists, the health system, and key specialty pharmacy stakeholders.

KEY POINTS.

• Health outcomes research is an effective tool to demonstrate the value of the integrated health-system specialty pharmacy care model to key external and internal stakeholders.

• The development and implementation of a health outcomes and research program within a health-system specialty pharmacy has led to successful completion, presentation, publication, and funding of research as well as important insights to drive practice improvement.

• Health-system specialty pharmacies should collaborate on outcomes research projects to further understand and expand the value of this integrated care model.

The rapid growth of specialty medications over the last decade has given rise to a new field in the pharmacy profession that is complex and challenging yet simultaneously offers significant opportunities for both patients and pharmacists.¹ The specialty pharmacist’s role combines responsibilities of traditional hospital- and clinic-based pharmacists while adding unique elements to address high medication costs, barriers to medication procurement and fulfillment, and significant technical and clinical patient support needs.^2,3 Various specialty pharmacy practice models have emerged across the marketplace, with notable recent growth in the number of integrated health-system specialty pharmacies (HSSPs).^4-6 The patient-centered HSSP model is innovative in that pharmacists work alongside prescribers and often face-to-face with patients, with access to the electronic medical record (EMR). Proven benefits of this model include improvements in medication access, adherence, and persistence, as well as patient satisfaction, faster time to treatment initiation, reduced provider burden, and lower patient out-of-pocket medication costs.^7-15

Vertical integration and consolidation of payers, pharmacy benefits managers, and pharmacies challenge the viability of the patient-centered HSSP model due to exclusion from limited distribution networks, decreased reimbursement rates, and cumbersome payer contracts.^4,16 It is therefore necessary to communicate our value by quantifying the benefits we convey to patients, providers, and the healthcare system. Until recently, literature regarding the design or benefits of the HSSP model was limited. Systematic evaluation and peer-reviewed publications were needed to help describe and measure the outcomes of this rapidly growing care model. At Vanderbilt Specialty Pharmacy, these needs and external pressures led us to develop a structured health outcomes and research program to demonstrate value and improved patient care at a HSSP.

The aim of this report is to describe the development, growth, and results of our health outcomes and research program (“outcomes program” hereafter).

Pharmacy practice model

The specialty pharmacy practice model at Vanderbilt University Medical Center (VUMC) has been described previously.¹⁷ In brief, specialty pharmacists and pharmacy technicians are embedded in outpatient specialty disease state clinics to help providers select appropriate specialty medications, ensure patients can access and initiate therapy, and monitor medication safety and efficacy. Patients are connected to a clinic-based specialty pharmacist who is familiar with their condition, medications, and healthcare team and is knowledgeable in navigating health insurance processes to secure treatment access. Following treatment initiation, the specialty pharmacist monitors the patient to ensure the desired outcomes of therapy are achieved. Pharmacists address patient-reported barriers to treatment adherence, address medication-related adverse effects, and perform necessary safety monitoring. Pharmacists consult with prescribers as needed, and pharmacists and prescribers collaborate to provide streamlined, continuous care. All relevant patient encounters are documented in the EMR and communicated to other healthcare providers, which facilitates seamless discussion of potential problems and patient needs.

Early efforts

Figure 1 shows the timeline of development for the outcomes program. In 2015, the department leadership recognized the need to solidify our value proposition and prepare for new market trends, such as value-based contracting and population health management. These discussions led to a call for interested specialty pharmacists to develop a Health Outcomes and Research Committee (“the outcomes committee”) tasked with generating research from specialty pharmacy practice. Led by 3 specialty pharmacists, the committee convened monthly to review ongoing and future projects. Most projects were mentored pharmacy practice resident projects or were generated by clinic-based specialty pharmacists interested in research. Data generated from these research efforts were increasingly used by the departmental leadership to communicate our model’s successes to external stakeholders, highlighting the utility of this research. Findings also inspired other specialty pharmacists to become involved with research in their respective clinics.

Figure 1.

Development of the Vanderbilt Specialty Pharmacy (VSP) health outcomes and research program.

We found that while many pharmacists were interested in outcomes research, most were deterred from conducting projects due to lack of time, research training, or research structure. To address these concerns, the outcomes committee engaged institutional resources to provide research training to pharmacists. In early 2016, pharmacists received training in reviewing literature (from the institution’s librarian), designing and collecting data using Research Electronic Data Capture, an online web-based tool for data collection (from a specialty pharmacist), writing manuscripts (from the institution’s librarian), and navigating the institutional review board (from an institutional review board analyst).¹⁸ Many new project ideas were reviewed by the Vanderbilt Institute for Clinical and Translational Research, an entity funded by the National Institute of Health’s Clinical and Translational Science Awards Program, which offered guidance on various aspects of research, including optimal study design.¹⁹

We then further developed the outcomes committee into a more formal health outcomes and research program, with the following few key components: a specialty pharmacist, serving as chair, who facilitated generation, organization, and completion of all projects; a data specialist, who provided feedback on study design and extracted necessary data from multiple systems utilized in practice; core outcomes committee members, who mentored other pharmacists and pharmacy students participating on research projects; and a research pharmacist, who assisted with all ongoing projects. We outlined a standard process to guide research projects from design through execution. We also established collaborations with the 3 schools of pharmacy in Nashville, TN, to engage student pharmacists in our research, which led to the origination of a structured student research program. In 2017, as research efforts progressed, we realized the need for dedicated program resources to support research and a well-defined program strategy.

Gaining organizational support and program resources

In addition to developing the outcomes program’s strategy, we began forming a team with the skills necessary to perform high-integrity outcomes research. In June 2017, the outcomes committee chair gave a presentation on the progress, future directions, and resource needs of the outcomes program to the pharmacy steering committee, which is composed of leadership members including the chief pharmacy officer; medical director; leaders in the areas of business operations, 340B Drug Pricing Program compliance, specialty pharmacy, and outpatient and retail pharmacy; representatives from the departments of finance and information technology and office of legal affairs; and external legal counsel. The chair requested 3 new positions (program director, project manager, and research pharmacist) as well as consultation support in biostatistics and bioinformatics. In exchange, the outcomes program promised sustainable research infrastructure, quality publications and presentations, multisite collaborations, extramural funding, and national presence and recognition. These new positions were approved by the pharmacy leadership and funded by the institution. Finally, the department leadership decided to provide floater coverage for clinic-based pharmacists up to 1 day per week, as needed, to work on outcomes projects.

Thus, an initiative that began as a volunteer-based, clinical pharmacist–led effort became a structured department by early 2018. Currently, the team is composed of 3 dedicated staff and supplemented by collaboration with diverse institutional resources (Table 1). An organizational chart is presented in Figure 2. The outcomes committee, still composed of volunteer pharmacists interested in outcomes research, remains an important element of the structure and function of the newly redesigned outcomes program.

Table 1.

Vanderbilt Specialty Pharmacy Health Outcomes and Research Program Team Members and Collaborators

	Role and Responsibilities
Team member
Program director	Develop and execute annual strategic priorities
	Develop and maintain relationships with external stakeholders and other health-system specialty pharmacies
	Provide support for creation, coordination, and execution of outcomes research studies
	Assist in budgetary management
	Collaborate internally to enhance outcomes data capture and capabilities
	Provide oversight of student research program
Research pharmacist	Design and execute outcomes research studies
	Coordinate efforts with pharmacist and physician champions
	Develop, support and execute collaboration opportunities with internal and external stakeholders
Project manager	Assist with planning, development, and execution of all projects to meet established goals, objectives, and timelines
	Provide professional and/or technical leadership in the execution of day-to-day project activities
	Communicate within and across areas to maximize information sharing around progress, needs, interdependencies, and/or accomplishments
	Facilitate identification of gaps in service levels and/or processes to identify potential areas for improvement
	Assist with student research program coordination and orientation
	Support collaboration with sponsors for outcomes research
Collaborators
Biostatistician	Review research project design and methods
	Analyze research data
	Assist with writing and reviewing grants
	Consult with manufacturers on potential studies
	Provide continuing education for pharmacists
	Participate on Outcomes Committee
Health information   technology personnel	Extract data from medical and pharmacy databases
	Design data collection instruments
	Develop methods for prospective data collection
Providers and clinic staff	Contribute guidance per clinical expertise
Vanderbilt Institute for  Clinical and Transla-  tional Research	Consult on study design and implementation
	Assist with institutional review board applications
	Review study protocols and manuscripts

Figure 2.

Health outcomes and research program organizational chart. FTE indicates full-time employee; VSP, Vanderbilt Specialty Pharmacy.

Setting a strategy and defining goals

We developed our strategy by defining our purpose and asking “Who are our stakeholders and how can we employ outcomes research to communicate our value to them?” We identified internal stakeholders as patients, providers, specialty pharmacists, and the health system (Table 2). External stakeholders included payers, the pharmaceutical industry, and other HSSPs. Our strategic priorities included employment of research findings in stakeholder discussions to gain access to limited distribution medications and inclusion in payer contracts; use of outcomes research to inform specialty pharmacist practice improvements; and positioning the HSSP model as the optimal care model by showing superior quality of healthcare delivery and outcomes in this practice setting.

Table 2.

Vanderbilt Specialty Pharmacy Stakeholder Analysis

	Vested Interest(s)	Strategic Priorities
Internal stakeholders
Patients	High-quality, seamless healthcare experience	Use research findings to demonstrate better patient outcomes as a result of integrated care
		Use research findings to continually evaluate and improve VSP patients’ medication access, adherence, and health outcomes
Physician champion	Optimal patient outcomes, reduced staff burden from assistance with utilization management, patient satisfaction	Use research to highlight benefits of integrated specialty pharmacist role in reducing provider and clinic burden and improving medication access, medication adherence and persistence, and patient satisfaction
Clinical pharmacist	Optimal patient outcomes, job security	Use research findings to support expansion of pharmacist integration across the health system
		Use research to support and enhance VSP specialty pharmacists as thought leaders in their fields
Health system	Optimal care delivery, revenue generation	Use research findings to secure access to patients through payer contracts and limited distribution drugs through manufacturers to sustain growth of the specialty pharmacy
External stakeholders
Payers	High quality, cost-effective care; prudent spend of healthcare dollars	Use research findings to demonstrate how integrated pharmacies provide better care quality and continuity, closer monitoring of medication appropriateness and optimization, and lower healthcare costs than external pharmacies
Pharmaceutical industry	Optimal real-world medication utilization and outcomes	Use research findings to evaluate the impact of integrated care on the patient journey and clinical outcomes
Other HSSPs	Recognition as a best practice healthcare delivery model in a climate of continual consolidation and vertical integration	Use research findings to support integrated model as the ideal care model

Abbreviations: HSSP, health-system specialty pharmacy; VSP, Vanderbilt Specialty Pharmacy.

With a team and strategy in place, we defined our vision, mission values, and goals for the outcomes program:

• Vision: A department that produces meaningful research benefiting patients, pharmacists, providers, the health system, and the field of specialty pharmacy

• Mission: To improve patient care through sound research, measure and expand the value of an integrated specialty pharmacy practice model, and foster research training in the field of specialty pharmacy

• Values: To be a patient-centered model that fosters innovation and collaboration and promotes both scientific integrity as well as teaching and training excellence

• Purpose: To identify and measure the link between specialty medications, healthcare delivery, and patient outcomes and to perform research encompassing the complete patient journey from clinical evaluation to treatment outcomes, including medication access and medication adherence as well as clinical, financial, and patient-reported outcomes

Three outcomes program areas of focus emerged: research, training and development, and outreach. We developed short-term and long-term goals and an action plan for each focus area. Our research goals targeted 3 objectives: improve patient care delivery and health outcomes, communicate the value of HSSP model to key stakeholders, and enhance the role of integrated specialty pharmacists. To accomplish our training and development focus, we outlined measurable training opportunities for pharmacists and students in the outcomes program. To achieve our outreach focus, we aimed to engage with the pharmaceutical industry, other HSSPs, our healthcare community, specialty patients, and payers with our outcomes program and research efforts.

Outcomes program deliverables and realized value

Improving patient care delivery and health outcomes

Health outcomes research encompasses a wide range of study types and topics (Figure 3). Our initial efforts focused on tangible, objective health outcomes, such as medication adherence, medication access, and patient costs. Across multiple clinics, we demonstrated a remarkable impact on these outcomes.^7,8,15,17,20 However, these studies also illuminated areas for us to improve patient care delivery. Although most of our patients achieved high adherence and persistence and incurred low out-of-pocket costs, research findings stimulated internal discussions on how to best use outcomes research to identify and address modifiable barriers to these outcomes.

Figure 3.

Potential outcomes research topics of interest to health-system specialty pharmacies throughout the patient journey. The ochre boxes represent steps in the patient care experience within the Vanderbilt University Medical Center health system (top left). Gray-shaded boxes provide noncomprehensive lists of potential research topics.

From early adherence and access research our projects were expanded to address more complex issues, with a focus on improving patient outcomes. These issues included navigating insurance approval, standardizing calculations for primary and secondary nonadherence, identifying risk factors for medication nonadherence, and establishing quality measures for specialty medication use. Study design complexity and rigor also progressed to prospective, randomized and nonrandomized studies to evaluate novel targeted interventions that optimized specialty medication use.

We developed a standardized adherence calculation and measured the rates of adherence in each of our specialty disease clinics.^21-23 We evaluated medication adherence in adult patients with inflammatory bowel disease, finding that 4 factors increased the risk of nonadherence: smoking, narcotic use, previous biologic use, and a psychiatric diagnosis.²⁴ Building on this outcome, we designed a prospective study to evaluate the impact of health coaching for moderate- and high-risk patients.

Through collaboration with the medical center’s gastroenterologists, we developed quality measures for the utilization of biologic therapy in the management of adult inflammatory bowel disease.²⁵ Following the positive response to this initiative, we engaged the medical center’s oncology clinic to develop quality metrics for the use of tyrosine kinase inhibitors in non–small cell lung cancer.²⁶

To ensure the continued growth, rigor, and ease of outcomes research efforts, we needed to advocate for prospective data capture and documentation. The outcomes program team engaged the department’s clinical operations and health informatics teams to implement outcomes data collection within various databases. To encourage documentation of outcomes as part of routine clinical practice, we modified our pharmacist intervention form, medication refill questionnaire, and specialty pharmacy encounters documentation within the EMR.

Communicating the value of the HSSP model to key stakeholders

Outcomes research findings were used in stakeholder discussions to illustrate how we achieved optimal health outcomes in a real-world practice setting. The goal was to convey the message that the best outcomes for patients receiving their medications could only be attained in an integrated, high-quality care delivery model that optimized coordination of care across the parties involved with medication selection, initiation, and ongoing utilization: the prescriber, the insurer, the pharmacy, and the patient. These discussions resulted in access to limited-distribution medications and generated dialogue around value-based contracting. In addition, communicating our success stories to colleagues in the pharmaceutical industry generated interest in collaborating on outcomes research. Partnerships with multiple pharmaceutical industry companies developed organically as we aligned on shared outcomes of interest. In these partnerships, we leveraged internal capabilities including access to comprehensive patient-specific data, documentation from high-touch patient interactions, and clinical expertise to position ourselves as an ideal setting for manufacturers to assess real-world outcomes of use of their medications.

Physician and nurse practitioner champions are key internal stakeholders and have participated in outcomes research alongside clinic pharmacists, offering valuable expertise and mentorship. As a result of internal recognition of our research efforts, additional providers expressed interest in working with the specialty pharmacy team to conduct research on real-world outcomes of specialty therapy within their clinical area. To date, we have completed collaborative projects with the departments of endocrinology, infectious diseases, hematology, gastroenterology, renal transplant, rheumatoid arthritis, asthma and allergy, and others, which have piqued the interest of both medical and pharmacy communities.

Outcomes committee members maintain active roles in professional organizations, including Vizient, the Pharmacy Quality Alliance, the American Society of Health-System Pharmacists (ASHP) Section of Specialty Pharmacy Practitioners, and the National Association of Specialty Pharmacy. Each of these organizations has shown increased focus on generating outcomes research, developing specialty pharmacy standards, and collaborating to improve specialty pharmacy care delivery.

We recognized early on that collaboration among HSSPs was vital to the continued growth and relevance of the integrated care model. In 2019, we initiated 2 multisite study initiatives: assessing rates of adherence to specialty medications in patients with rheumatoid arthritis and evaluating pharmacist interventions and outcomes in patients with multiple sclerosis. In addition, we formed a blog discussion on our website to allow HSSPs to share research ideas and find partners for research collaboration.²⁷

Enhancing the role of integrated specialty pharmacists

We conducted several studies to highlight integrated pharmacists’ success in navigating an often convoluted insurance approval process to optimize patient access to specialty medications.^7,28,29 Across multiple disease states, we found that integration of a pharmacist decreased time to medication access and provided valuable interventions related to safe and effective medication use. Realizing that our pharmacists are in an ideal position to directly interact with patients on a regular basis, we promoted the results of their work to advocate for integrated positions in other specialty disease state clinics.

We also designed an adherence clinic staffed by a dedicated specialty pharmacist who receives notification of patients falling below optimal adherence levels and proactively engages them to mitigate barriers. This effort demonstrated the ability and expertise of a specialty pharmacist to tackle barriers that are often overlooked. In addition to standard-of-care monthly refill calls and pharmacist interventions, the adherence clinic specialty pharmacist tailors care to patient-specific adherence barriers. For example, if a patient expresses difficulty attending clinic visits due to transportation, the adherence clinic pharmacist identifies and communicates transportation options for the patient.

Lastly, we helped our pharmacists present their work at key regional and national conferences, providing them with the platform to be recognized as experts and leaders in their field. Our specialty pharmacists have received high interest and recognition for our projects presented to local and national conferences. As of March 2021, 88 poster presentations and 7 podium presentations have been presented through the work of the outcomes program. Our projects have received awards from the Academy of Managed Care and Specialty Pharmacy, the American College of Gastroenterology, and the National Association of Specialty Pharmacy. The outcomes program and specialty pharmacy department were recognized as the 2019 Innovative Health-System Practice of the Year by the Tennessee Society of Health-System Pharmacists and as a recipient of an ASHP 2019 Best Practices Award. These honors help support further resource allocation, pharmacist motivation, and national recognition of the importance of outcomes research in specialty pharmacy.

Student research program

Student engagement and training, which we have offered though a structured student research program, has been a key focus of the program from its infancy, Vanderbilt University does not have a school of pharmacy, but Nashville is home to 3 schools of pharmacy whose students have participated in outcomes research. After students are accepted into the program, we provide orientation and research training and assign students a project and mentor. Students participate in various stages of a project, from collecting data to creating poster presentations and manuscripts. In the second year of our program (2017), as the number of student applications multiplied, we recognized the need for a structured plan for recruiting, selecting, and assigning students to projects, so we created application and scoring criteria to evaluate applicants. Students in our program attend biannual research forums to present their research to peers and learn practical research skills. To date, 44 pharmacy students have participated in this program, most of whom have completed national presentations and authored publications.

Outcomes summit and consortium

Over time, more HSSPs have implemented similar integrated models of specialty pharmacy care,⁴ resulting in an emergent need for a collective voice to communicate the value of this model and discuss how limited-distribution networks and payer contract exclusions pose barriers to patient care. We sought to empower other HSSPs to perform outcomes research, given our success and the lessons learned. In March 2019, we hosted a 3-day inaugural HSSP Outcomes Research Summit that attracted 144 attendees from the pharmaceutical industry, payer representatives, and 44 distinct health systems. Presenters and panelists disseminated tools and knowledge to other HSSPs looking to start or invigorate research efforts. Attendees expressed a desire to discuss and collaborate with other institutions on outcomes research; thus, we created an outcomes research consortium. The consortium is open to any HSSPs interested in collaborating on research efforts and currently has several projects in process.

Lessons learned and implications for HSSPs

In our experience building an outcomes program dedicated to researching and advancing patient care, we have learned valuable lessons that we recommend to HSSPs with minimal to several resources that wish to initiate outcomes research:

Start slow and with clear direction

In the early stages of our outcomes program, we eagerly accepted and attempted any and every project that sounded exciting, without understanding the time and effort required to design, execute, and present a research project. As a result, we started many projects but progressed slowly on each. Some of the projects did not serve the goals of our program, as we had not yet formed our focus areas and goals. We recommend starting with small, feasible projects that allow you to learn how to do research using available resources at your institution.

Develop an organized team

Identify a leader with some research experience (this is encouraged but not mandatory) and time to develop program direction. Engage a few pharmacists with some research experience (this is helpful, not required) and time to work on projects. Have monthly meetings to establish and maintain goals, identify projects, and stay on track. Identify health-system resources. These may be institutional resources or key opinion leaders with research experience who can provide research training, expertise, or services.

Formulate clear goals and reasons for undertaking outcomes research

Without direction, it is easy to take on projects that become time-consuming without meeting the needs of your HSSP. Consider developing criteria to evaluate the value of a potential project before deciding to undertake it. It is likely that internal and external stakeholders will become interested in partnering on research and generate a number of potential study ideas. Creating a plan for engaging other departments and external partners can be helpful when deciding whether to engage in potential projects.

Engage, but limit the number of learners

Early on, we accepted many students to our program and assigned them to underdeveloped projects. Students were eager to learn and help, but we realized we needed to develop our projects further before incorporating learners. We also developed clear expectations for work, timelines, and communication, which helped studies progress. Provide clear direction, expectations, and responsibilities to learners. Also, mentoring students and residents is time-consuming, so plan ahead to ensure mentors have sufficient time and resources.

Develop a strong collaboration with data partners

Data collection and extraction requires dedicated resources and time. Our team required education and practice to learn how to best communicate our data needs to informatics professionals. We also identified ways to improve pharmacist documentation in the EMR to reduce the need for extensive, manual chart review.

In its short existence, the outcomes program has created a productive research agenda (eAppendix).

Conclusion

Our health outcomes and research program has brought value and recognition to the institution and the field of pharmacy. HSSPs interested in starting research efforts should do so in a systematic, thoughtful manner, with a clear understanding of goals and resource requirements. Outcomes research should be aimed at evaluating and improving patient outcomes and care delivery models.

This article is part of a special AJHP theme issue on specialty pharmacy. Contributions to this issue were coordinated by Joseph Cesarz, MS, PharmD, and Scott Canfield, PharmD, CSP.

Disclosures

The authors have declared no potential conflicts of interest.

Additional information

The contents of this article are solely the responsibility of the authors and do not necessarily represent official views of the National Center for Advancing Translational Sciences or the National Institutes of Health.