Protocol for a Noninferiority Comparative Effectiveness Trial of Home-Based Palliative Care (HomePal)

Abstract

Introduction:

As health care systems strive to meet the growing needs of seriously ill patients with high symptom burden and functional limitations, they need evidence about how best to deliver home-based palliative care (HBPC). We compare a standard HBPC model that includes routine home visits by nurses and prescribing clinicians with a tech-supported model that aims to promote timely interprofessional team coordination using video consultation with the prescribing clinician while the nurse is in the patient's home. We hypothesize that tech-supported HBPC will be no worse compared with standard HBPC.

Methods:

This study is a pragmatic, cluster randomized noninferiority trial conducted across 14 Kaiser Permanente sites in Southern California and the Pacific Northwest. Registered nurses (n = 102) were randomized to the two models so that approximately half of the participating patient-caregiver dyads will be in each study arm. Adult English or Spanish-speaking patients (estimate 10,000) with any serious illness and a survival prognosis of 1-2 years and their caregivers (estimate 4800) are being recruited to the HomePal study over ∼2.5 years. The primary patient outcomes are symptom improvement at one month and days spent at home. The primary caregiver outcome is perception of preparedness for caregiving.

Study Implementation—Challenges and Contributions:

During implementation we had to balance the rigors of conducting a clinical trial with pragmatic realities to ensure responsiveness to culture, structures, workforce, workflows of existing programs across multiple sites, and emerging policy and regulatory changes. We built close partnerships with stakeholders across multiple representative groups to define the comparators, prioritize and refine measures and study conduct, and optimize rigor in our analytical approaches. We have also incorporated extensive fidelity monitoring, mixed-method implementation evaluations, and early planning for dissemination to anticipate and address challenges longitudinally. Trial Registration: ClinicalTrials.gov: NCT#03694431.

Introduction

Background

Multiple evidence-based guidelines^1-3 affirm the need to provide palliative care (PC) services across the care continuum to alleviate unnecessary suffering and improve quality of life for both patients and caregivers. For patients with a high symptom burden and significant functional limitations that make current clinic-based PC models unsuitable,⁴ telephone-based support inadequate,⁵ and hospice enrollment not yet tenable, home-based palliative care (HBPC) may be the only viable option. The decisional dilemma for health care systems and clinicians lies in how best to optimize delivery of high-impact HBPC services that meet the growing demand for such care while also remaining affordable for all payers including patients, caregivers, employers, public purchasers, and health systems.

To address this, our study compares a standard HBPC model that includes routine home visits by a nurse and prescribing clinicians (physicians and nurse practitioners) with a more efficient tech-supported HBPC model that aims to promote timely interprofessional team coordination through synchronous video consultation with the prescribing clinician while the nurse is in the patient's home. If the more efficient tech-supported model yields short-term reductions in symptom burden and helps patients spend more time at home that is comparable with standard HBPC, this could lead to redefining PC practice and policy for dramatically expanded, affordable access to HBPC. The purpose of this invited article was to describe the study protocol, stakeholder engagement efforts before and during study start-up, and early implementation challenges and learnings.

Objectives and hypotheses

Aim 1

Determine if tech-supported HBPC is noninferior to standard HBPC as measured by the co-primary patient-and-family-centered outcomes of symptom improvement (short term) and days at home in the last 180 days of life (long term). We will also measure several secondary outcomes: quality of life, care experience-satisfaction, acute care utilization, and survival.

Hypothesis

People with serious illness who receive tech-supported HBPC will have short-term, comparable improvements in symptoms from baseline to one month and will spend a similar number of days at home in their last six months of life compared with standard HBPC. Secondary outcomes are also expected to be similar between care models.

Aim 2

Compare the effects of tech-supported HBPC and standard HBPC on the primary outcome of caregiver perception of preparedness for caregiving and secondary outcomes of quality of life, anxiety-depression, caregiving burden, care experience-satisfaction, and health care utilization (for those caregivers who give permission to access their utilization data from the health system).

Hypothesis

Caregivers receiving standard and tech-supported HBPC will report similar perceptions of preparedness for caregiving.

Aim 3

Examine the effectiveness of HBPC across several subgroups, for example, cancer, organ failure, neurodegenerative diseases, multiple chronic conditions (three or more diseases/conditions), participation of a caregiver in the patient's care, racial/ethnic minorities, and low income.

Aim 4

Describe the longitudinal pattern of PC service exposure, intensity of acute health service use, and quality of end-of-life care in patients with serious illness who have been referred for HBPC services but did not enter the HBPC pragmatic trial (passive population cohort).

Aim 5

Describe barriers and facilitators to implementation of tech-supported HBPC.

Methods

Study design

Setting

Two Kaiser Permanente regions (Southern California [KPSC] and Oregon/Washington [KPNW]).

Timing

This five-year study started on January 1, 2018. The protocol, operating procedures, systems, tools, workflows, and trainings were completed in the first year. Participant enrollment commenced on January 7, 2019 with 14 sites launched in a staggered manner through February 4, 2019 and will extend through June 2021.

Population

English and Spanish-speaking patients (target enrollment 10,000) with a serious illness and prognosis of one to two years who have a high symptom burden and significant functional limitations that precludes clinic-based care and their caregivers (estimate 4800).

Design

This is a pragmatic, noninferiority cluster randomized trial to compare tech-supported HBPC with standard HBPC at 14 sites in two Kaiser Permanente regions that have existing HBPC programs. Registered nurse (RN) case managers across these sites were randomized (stratified by site) to tech-supported or standard HBPC so that approximately half the patient-caregiver dyads receive routine in-home nurse and prescribing clinician visits (standard HBPC; patients: 5000/caregivers: 2400) and the other half receive nurse visits coupled with remote prescribing clinician consultation-supervision (tech-supported HBPC; patients: 5000/caregivers: 2400) (Fig. 1). Features of the study align with the pragmatic-explanatory continuum indicator summary (PRECIS) guidance for pragmatic trials (Table 1).^6,7

FIG. 1.

HomePal study design.

Table 1.

PRECIS Criteria for Pragmatic Trials

Domain	Criteria for pragmatic trials	Standard vs. tech-supported HBPC trial
Participants	All eligible participants enrolled, regardless of risk, responsiveness, comorbidities or past compliance	All adult health plan members who meet the HBPC program admission criteria are eligible for the HBPC trial and all others are passively followed in the parallel observational cohort study
Intervention condition	Interventions are highly flexible, offering providers leeway in formulation and application	Beyond efforts to assure the standard and tech-supported HBPC patients and caregivers receive routine prescribing clinician home visits or remote consultation, respectively, HBPC services are tailored to patients' and caregivers' needs and preferences. Varying levels of participation are expected
Intervention practitioners	Interventions are applied by the full range of practitioners in the full range of settings with only ordinary attention to dose and side effects	HBPC clinicians are drawn from the existing local workforce of prescribing clinicians, nurses, social workers and other supporting staff. The sites are responsible for selection and supervision of clinicians (using standard quality control tools)
Comparison condition	“Usual Practice” (or the best alternative), offering practitioners considerable leeway in application	The tech-supported HBPC service is being compared to standard HBPC service that includes routine in-home provider visits
Comparison practitioners	The control intervention is applied by the full range of clinicians in the full range of settings, with only ordinary attention to training, experience, and performance	Standard HBPC is implemented by real-world clinicians under usual practice conditions. Basic training to ensure PC competency across all clinicians was provided at study start-up and yearly, with refresher workshops as would be provided in real-world health systems
Follow-up assessments	There are no research assessments; existing databases are searched for outcomes	All utilization and clinical data are collected from existing EMR and insurance claims data. Patient/caregiver reported outcome data (PRO surveys) are collected by telephone because not all PROs are collected during routine practice
Outcome definition	The primary outcome is objectively measured, meaningful to study participants, and does not depend on central adjudication	The primary patient outcomes include a PRO (symptom burden) that is captured at baseline and at one month and an EMR-based outcome of days at home in the last six months of life. Other secondary patient and caregiver outcomes require additional data collection by telephone
Intervention compliance	There are no special strategies to improve compliance, and compliance is unobtrusively measured	Interventions are supported by tools embedded in the EMR, but there is no central supervision of intervention providers. Quality of implementation is being assessed using EMR data
Practitioner adherence	There are no special strategies to maintain practitioner adherence, and adherence is unobtrusively measured	Interventions are supported by tools embedded in the EMR, but there is no central clinical supervision of intervention providers
Primary comparison	The analysis includes all patients regardless of compliance, eligibility, or others	All outcomes will be analyzed according to initial assignment—regardless of intervention participation or compliance

EMR, electronic medical record; HBPC, home-based palliative care; PC, palliative care.

We are also conducting a parallel observational cohort study to passively examine through electronic medical record (EMR) information, the patterns of PC service exposure, and overall health service use for all patients who have been referred for HBPC services but did not enter the trial.

Randomization

RN case managers (n = 102) who are full or part-time employees are the unit of randomization. The randomization scheme was stratified by geographical regions within the 14 clinical sites.

HBPC comparators

Consistent with standards from the National Consensus Project for Quality Palliative Care^8,9 and models of interdisciplinary PC,^4,5,10,11 HBPC services across the two comparators are encouraged to adhere to best practices addressing bio-psycho-social-spiritual needs and providing comprehensive PC support regardless of delivery method. The team may assume a co-management, primary, or mixed role. Patients remain on HBPC if the service continues to meet patients' needs. Both delivery models include biweekly interdisciplinary group (IDG) meetings to review current cases and discuss progress toward meeting patients' goals (Table 2).

Table 2.

Standard and Tech-Supported Home-Based Palliative Care Intervention Components

Key functions	Standard HBPC	Tech-supported HBPC
Start of care
RN visit	Home	Home
MD/NP visit	Home within 14 days of RN visit	Synchronous video visit with RN in patient's home
SW and Chaplain visit	As needed	As needed
Ongoing care
RN/LVN/SW/Chaplain/Aide visits	As per care plan	As per care plan
MD/NP visits	Home as needed	Video or home visits as needed
Interdisciplinary team meetings	Biweekly	Biweekly

Palliative care core practice across care models
Comprehensive bio-psycho-social-spiritual assessment	Emotional and spiritual support
Interdisciplinary care planning	Patient and family education/skills training
Pain and symptom management	Care coordination across settings
Communication on care priorities	Referrals to other resources as needed

LVN, licensed vocational nursing; MD, medical doctor; NP, nurse practioner; RN, registered nurse; SW, social worker.

To ensure a level of standardization across HBPC teams regardless of treatment arm, the health system required all HBPC team members to complete the CSU San Marcos Palliative Care Institute online PC essentials training before study launch. The HBPC staff completed the modules independently or in a group setting, coupled with debriefing sessions. Prescribing clinicians were encouraged to complete a list of recommended modules available from the Center to Advance Palliative Care. Our workforce training efforts are consistent with recommendations for conduct of pragmatic trials^6,7 to ensure sustainability and to minimize operational impact. All HBPC clinicians will be expected to complete life care planning training by the end of 2019.

Standard HBPC

The current standard HBPC^12,13 approach across our clinical sites is to utilize the RN case managers to initiate the evaluation and lead the team-based care. The RNs conduct the initial start of care visit in the patient's home. The prescribing clinician generally conducts a separate home visit up to 14 days later. The frequency of subsequent home visits is determined based on the care plan and emerging needs of the patient/caregiver.

Tech-supported HBPC

Tech-supported HBPC is also led by an RN case manager who conducts the initial start of care visit in the patient's home. The prescribing clinician participates in the visit remotely through video with the patient/caregivers while the RN is in the patient's home. We expect that early access to the prescribing clinician for discussion and concurrence on the treatment plan builds trust and confidence with the patient/caregivers, potentially reducing the need for an in-home prescribing clinician visit. The care teams may deviate from using video visits if it is clinically warranted or if the patient/caregiver declines. The teams use video visits at their discretion during follow-up touchpoints to address acute changes in condition, goals of care discussions, medication changes and review, recertification review, evaluation for transition off HBPC, or other uses primarily driven by the nurse in the home. The tech-supported clinicians have tablets and smartphones that are equipped with enterprise-wide secure, video-conferencing applications. We provided targeted training on interprofessional communications coupled with role play on use of the video application for the four-way nurse-patient-caregiver-prescribing clinician interaction before study launch.

Outcomes measures

We based our selection and timing of outcome measures for patients and caregivers in the literature, considering the hypothesized short and longer term impact of HBPC, and iterative feedback on what matters most from our diverse stakeholders. We also considered measure responsiveness, burden, likelihood that measures will be used in routine care, and harmonization with other Patient-Centered Outcomes Research Institute (PCORI)-funded PC studies (see Table 3 for a full summary).

Table 3.

Summary of Data Collection Schedule

Concept	Collection of patient- and caregiver-centered outcomes
		Data collection time points
	Instrument/source	Screen-baseline	1 Month	2 Months	6 Months	12 Months
Patients: primary		n = 10,000	n = 8500	n = 8000	n = 7000	n = 5000
Symptom burden	Edmonton Symptom Assessment Scale (10 items)	RS	DCC	RN/SW	DCC	RN/SW
Days at home	EMR/claims: Last 180 days of life					DCC
Patients: secondary
Distress	Distress thermometer	RS	DCC		DCC
Functional impairment	Palliative Performance Scale	RN/SW	RN/SW	RN/SW	RN/SW	RN/SW
Quality of life	PROMIS-10 (10 items)	RS^a	DCC		DCC
Care experience-satisfaction	Study specific (8 items)		DCC		DCC
Health care utilization^b	EMR/claims					DCC
Qualitative interviews	Semi-structured interview questions		RS		RS
Caregiver: primary		n = 4800	n = 4000		n = 3500
Preparedness	Preparedness for Caregiving Scale (8 items)	RS	DCC		DCC
Caregiver: secondary
Quality of life	PROMIS-10 (10 items)	RS^a	DCC^c		DCC^c
Burden/stress	Zarit Caregiver Burden (12 items)	RS^a	DCC		DCC
Care experience-satisfaction	Study specific (8 items)		DCC^c		DCC^c
Health care utilization	EMR/claims (only for Kaiser members)					DCC
Qualitative interviews	Semi-structured interview questions		RS		RS
EOL care/bereavement	Quality of end-of-life care experience		Administered by KP upon patient's death

Process measures are captured from the EMR: HBPC services include in-home visits (provider, RN, SW, and other ancillary staff), virtual encounters (telephone or video), and advice calls during nonbusiness hours and weekends; outpatient PC services includes clinic visits and virtual encounters (telephone or video); inpatient PC services; hospice enrollment and length of stay; use of physician aid in dying.

Can be completed within seven days of HBPC admission visit if not completed previously.

Health care utilization includes: all-cause hospitalizations, observational stays, emergency department visits, skilled nursing facility (SNF) days, home health days, and use of outpatient services (primary and specialty care).

If patient is deceased, we will try to obtain these two measures from the caregiver.

DCC, obtained through telephone by the Data Coordinating Center; RN/SW, per routine care if available via EMR; RS, research staff (obtained by telephone).

Patients

We are measuring symptom burden with the total Edmonton Symptom Assessment Scale (ESAS) score.^14,15 The ESAS measures physical and emotional symptoms and overall well-being using a 0 to 10 point scale; financial distress and spiritual pain are also measured but not included in the total score. Days at home is a co-primary measure.^16,17

Caregivers

Caregivers have told us that feeling well prepared in caring for their loved one is what matters most and this measure closely relates to the education, skills training, and support they would receive from the HBPC team; thus we are using the eight-item Preparedness for Caregiving Scale.^18,19

Recruitment and Study Procedures

Eligibility criteria

Patients

Trial cohort

Given the pragmatic real-world study design, the target patient population is broadly inclusive of all health plan members who are 18 years and older, of all race and ethnicities, English and Spanish speakers who are enrolled into HBPC including those with nonmalignant (e.g., heart failure, chronic obstructive pulmonary disease (COPD), end-stage kidney or liver disease, and neurodegenerative diseases) and malignant serious illness^20-23 who have an expected survival of one to two years, and are homebound.

Caregivers

Trial cohort

We identify adult family or other caregivers (at least 18 years old) during the telephone screening/intake as someone “who helps you with your care.” We are excluding professional caregivers who are hourly wage employees; we estimate ∼80% of patients will have a caregiver and 60% of those caregivers will agree to participate.

Patients

Observational cohort

We are tracking patients who are referred to HBPC but who are not immediately enrolled in the trial through their EMR-based data through the end of the study or until they enroll in the trial. The observational cohort also includes non-English and non-Spanish speakers.

Recruitment

Because of minimal risks expected with study activities, we have a waiver of written consent to administer surveys and a waiver of consent and HIPAA to passively collect EMR-based data for all patients unless patients explicitly request to opt out of the study. Patients/caregivers also receive information letter that describes the study activities and how to opt out in their HBPC program packet.

We are identifying patients from either routine physician-initiated referrals or a population-based proactive case finding process. As per routine workflows, HBPC referrals are routed to the respective clinical sites where administrative and clinical staff determine programmatic and insurance eligibility. After the administrative and clinical staff complete their review and are scheduling the start of care home visit, research staff initiate recruitment calls to obtain verbal consent from the patient and designated caregiver and to complete the baseline assessments. Clinical programs that are expanding to proactive outreach may additionally identify eligible patients from an existing health system risk stratification model that uses EMR data to flag frail patients with an expected prognosis of one to two years.²⁴ Patients and caregivers are considered enrolled in the trial once they provide consent, complete the baseline surveys, and the patient is admitted to HBPC.

Study procedures

Figure 2 summarizes the overall participant flow and Figures 3 and 4 are the CONSORT diagrams for patients and caregivers, respectively. Recognizing that survey completion can be challenging in this population, we have prioritized collecting the ESAS to capture patient symptom severity followed by the distress thermometer, Caregiver Preparedness Scale, and then other secondary surveys. Administering all surveys takes ∼30 to 40 minutes. Surveys can be completed on more than one call within a seven-day window of start of care. Within two to five days of the clinical referral, either a standard or a tech-supported RN makes a start of care home visit. If patients are not initially admitted to HBPC, they are not included in the trial cohort. However, they are eligible to join the trial if they are admitted to HBPC at a later point. Consistent with intent-to-treat (ITT) principles, we follow patients for at least 12 months regardless of service transitions (e.g., to hospice) or until death; research staff contact the patients/caregivers 1 and 6 months after the start of HBPC to administer the surveys as given in Table 3.

FIG. 2.

HomePal participant flow.

FIG. 3.

HomePal patient CONSORT diagram.

FIG. 4.

HomePal caregiver CONSORT diagram.

Qualitative assessment of HBPC implementation

To obtain formative evaluations and help with midcourse corrections, we are seeking feedback from tech-supported patients/caregivers and HBPC team members. We are inviting a small randomly selected sample of patients/caregivers to participate in 15-minute telephone interviews about their video visit experience after completion of their one- and six-month assessments. We will invite clinicians to complete annual web-based surveys of their experiences in implementing HBPC services.²⁵ We are also gathering data from embedded research staff participating in IDG meetings and through weekly feedback from multiple clinical stakeholders during operational meetings.

Data Management and Analysis Plan

Data management, quality control, and monitoring

The KP Northwest Center for Health Research Data Coordinating Center (DCC) is responsible for central coordination and management of study data. The DCC developed and maintains a central database integrating EMR and survey data from both regions and supports a centralized web-based electronic data capture system that tracks all aspects of participant flow and provides for remote data entry and management. The DCC uses rigorous quality control procedures to protect participant safety and confidentiality.

The data safety monitoring plan includes monitoring recruitment procedures and progress and potential adverse events, although minimal risks are expected with study activities. The data safety and monitoring board (DSMB) comprises a biostatistician and three subject matter experts and are meeting semi-annually to monitor, review, and assess study progress and safety data.

Power calculation

We relied on published minimal clinically important difference (MCID) criteria for the ESAS²⁶ and data on days at home in the last 180 days of life from a cohort of patients who received HBPC in KP to inform our power calculation for the primary study hypothesis of noninferiority between standard and tech-supported HBPC. For the primary short-term outcome of improvement in symptoms (ESAS score) from baseline to one month, assuming a noninferiority MCID margin of 3 points for a difference-in-differences (DID) comparison of change in total ESAS and within-patient change standard deviation (SD) of 11.6, and a conservative power of 0.90, type I error of 0.025, and intraclass correlation coefficients ranging from 0 to 0.05, we should be able to detect a noninferior absolute DID as large as 2.2 and as small as 1.3 with anticipated data from ∼8500 patients (15% attrition at one month).^27,28 Similarly, for the primary long-term outcome of days at home in the last 180 days of life for patients who are followed for at least 180 days after starting HBPC, based on our historical data (mean = 165 days, SD = 24) we should be able to detect a noninferior absolute difference as large as 18.8 and as small as 3.2 for margins ranging from 7 to 21 days, expecting ∼50% (n = 5000) of patients across both groups to die over the study period.

Analytical plan

The analyses will follow an ITT strategy. Our analysis for the short-term outcome of improvement in symptom burden (ESAS) from baseline to one month will be an ITT DID model, which will compare the average within-patient change in symptom burden between the two HBPC groups in patients surviving for at least one month. We will include as covariate adjustments in a generalized linear mixed-effects regression model any baseline covariates for which the standardized difference between HBPC groups is >0.1, as well as relevant RN and site characteristics. To accommodate such covariates and possible clustering of patients-within-RNs-within-sites, we will analyze the DID by a linear mixed-effects regression model with RN- and possibly RN-within-site-level random intercepts. We will test for both levels of clustering and will also test whether the DID cluster in the same manner. To declare noninferiority, we would need the lower 95% confidence limit for the regression parameter corresponding to tech-supported versus standard HBPC to be above our margin of −3.

For our long-term outcome of days at home in the last 180 days of life, we will use linear mixed effects to model counts of days as a function of relevant patient characteristics. The outcome of days at home will be aggregated at the patient level, but similarly allows for patient-within-RN-within-site clustering. To declare noninferiority we would require the lower 95% confidence limit on the parameter comparing tech-supported versus standard HBPC to be above our margin of −7.

We will adapt the generalized linear mixed-effects modeling framework described previously, using different link functions to address the different types of secondary outcomes (continuous, dichotomous, or counts). We will determine whether the intervention effects differ by prespecified subgroups using the same models described previously and will include an interaction term. To describe the different longitudinal patterns of PC service exposure, intensity of acute care use, and quality of end-of-life care for the observational, passive cohort, we will use latent class regression^29,30 to identify clusters of individuals following similar trajectories.

We are assessing barriers and facilitators to tech-supported HBPC implementation through quantitative and qualitative methods. We are tracking video visit uptake, refusal rates and reasons, crossovers with prescribing clinician visits and reasons, and patient/caregiver complaints. Data from qualitative interviews of patients/caregivers, site visits, and clinician surveys will be analyzed using standard procedures for thematic analysis.^31,32

Ethics Determination

The study was approved by the KPSC and KPNW Institutional Review Boards.

Study Implementation: Challenges and Contributions

Stakeholder engagement

Our Stakeholder Advisory Committee (SAC), comprising patients, family and other caregivers, professional subject matter experts, and health systems partners, actively collaborates with the research team by providing input on the study design and methods, conduct (recruitment and retention, data collection and analysis, and other activities), and dissemination efforts. Table 4 provides a detailed summary of how the SAC engagement informed the design and approach during the study start-up.

Table 4.

Stakeholder Influence on Study Design and Approach

Topic	SAC engagement and impact on study
Study design and nomenclature
Cluster randomization at the RN level	The logistical complexity and inability to measure our distal outcome, with the individual patient randomization and stepped wedge designs, respectively, reaffirmed our original decision to use a parallel, cluster randomized controlled trial (RCT) design
Labels for comparators	Selection of the comparator labels “Standard HBPC” and “Tech-supported HBPC” most accurately reflects the core goals of the study
Study name	The study name, “HomePal” was proposed by a SAC-caregiver member
Measures/outcomes
Proximal outcome: symptom burden	Patients are primarily referred to HBPC for symptom management and thus, the primary goal of HBPC is to demonstrate its short-term effectiveness on symptom control
Distal health system outcome: days at home in last 180 days of life	As patients may transition in and out of HBPC depending on their needs to other PC/hospice programs within the health system, days at home reflect the overall health system's ability to care for and support patients in their homes toward the end-of-life
Inclusion of other caregiver related questions	Key questions to better capture the caregiving experience were added to the data collection protocol (length of caregiving, previous experience with being a caregiver, and living situation with care recipient)
Patient/caregiver semistructured interview questions	Concise, simple clear language is used. Questions for caregivers differ from patients to ensure that both perspectives are captured
Clinician surveys	Questions about the utility of video visits are framed in a neutral way. Inclusion of other domains, including how video visits impact clinicians' work life and team working relationships to capture potential moderators and secondary clinician outcomes
Recruitment materials
Information letters and telephone recruitment script	Concise, lay language to describe the overall study, the why and how of the video visits in the information letters. Telephone script is also concise with the right amount of detail for patients and caregivers to make an informed decision about completing the study surveys
Videos describing HBPC and other PC/hospice programs	Extensive engagement with multiple stakeholders to ensure the brief English and Spanish videos clearly captured key elements of the respective programs. The HBPC video describes the clinical program and the design and data collection for the HomePal study
Pilot data collection
Harmonizing HBPC admission criteria	Volume of ESAS data for each clinical site from the pilot was insufficient to provide structured guidance on the admission criteria at study launch. Concerns regarding patient and caregiver understanding of the scaling used to rate symptom severity over the telephone
Streamlining patient and caregiver surveys	Prioritize collection of the primary patient (ESAS) and caregiver (preparedness) outcomes over secondary outcomes. Dropped health literacy questions because of low percentage of patients who can independently respond to surveys

SAC, Stakeholder Advisory Committee.

Fidelity monitoring

We are assessing fidelity on a biweekly (year 1) to monthly basis through extraction of key process measures, for example, prescribing clinician visits (home visits for standard HBPC and telephone/video for tech-supported HBPC). In addition, the research RNs attend IDGs to elicit and listen for technical, logistical, and staffing issues and assist the teams with troubleshooting, as well, providing context for deviations that might be captured on the fidelity reports. We are providing site and individual feedback reports to the RNs and prescribing clinicians and the leadership at each site to inform implementation and reinforce the study protocol. Tech-supported HBPC teams may not feel comfortable with remote prescribing clinician supervision and consultation for certain patients and thus can choose to deviate from the protocol if the clinical situation calls for it. These crossovers could potentially contribute to artificially minimizing differences between groups. Our fidelity monitoring tracks variation in the timing and use of video visits and overall adherence to study arm allocation.

Implementation challenges and mitigation strategies

As much as the study team aims to plan in advance and apply rigor to the study conduct, pragmatic trials embedded within health systems must contend with the complex, dynamic, and evolving micro- and macroenvironments in which care is delivered. This is especially true for care of patients with serious illness.^6,33-35 Implementation challenges for the study fall under four domains: (1) clinician readiness and openness to integrating video visits in their practice, (2) participant enrollment and data collection, (3) HBPC program staffing and resources, and (4) the evolving telehealth landscape. These challenges and corresponding mitigation strategies are described briefly here and summarized in Table 5.

Table 5.

Implementation Challenges and Mitigation Strategies

Implementation challenges	Mitigation strategies
Clinician readiness/openness for integrating video visits into routine practice
Variation in HBPC MD/NP readiness for change within and across clinical sites	Repeated assurance that use of video visits is ultimately based on the HBPC team's clinical judgment of whether video would adequately address patients/caregivers' needs and that MD/NP can still make home visits if neededAt start-up, leveraged experience and testimonials of the few MDs who were already using video visits in outpatient PC to share their lessons learned and positive experiences. These MDs emphasized that the RN-facilitated video visit was a definite plus as this arrangement does not require patient access to technology and thereby would increase access to careAfter study launch, early MD adopters shared their experiences and tips during cross-site webinars and in person panel discussionsAn updated video visit “frequently asked questions” document was circulated to all HBPC team members three months after study launchSince tech-supported RNs had difficulty coordinating and scheduling video visits with MDs/NPs who were reluctant to embrace change, MD/NP reassignment was necessary at two sitesAudit and feedback of video visit completion is shared with the site physician leadership on a weekly basis to foster competition across sitesSite MD and nursing leaders are working together to set targets and expectations for video visit completion with their teams
Variation in randomized tech-supported HBPC RN readiness and enthusiasm for change	One-on-one and group role-play trainings on facilitating four-way (RN-patient-caregiver-MD/NP) communicationsResearch RNs accompany HBPC RNs on their first video visit to assist with technical issues and provide coaching as neededResearch RNs attend IDGs as ethnographers, learning the culture of the teams, participating in care delivery discussions, provide coaching/mentoring, and helping to facilitate video visit implementationTech-supported RNs sharing their experiences and tips with peers during IDGs and during cross-site webinarsSupport from site nursing leadership to allow tech-supported RNs more time to coordinate, schedule, and conduct the video visit and to adjust their productivity expectations accordinglyAudit and feedback of video visit completion is shared with the site nursing leadership on a weekly basis to foster competition across sites
Participant enrollment and data collection
Sites already have existing program with a current census	Expect slow ramp up for some sites that already have a full census at study launch and adjust accruals accordingly. Research RNs encouraging teams to evaluate current census to open up capacity, as well, to leverage video visits to increase efficiency with existing patients
Suboptimal participation in telephone surveys by patients and caregivers	Because of the very short time frame from when a HBPC referral is made and when the first home visit is completed per CMS regulatory requirements, research staff have very little time to reach out to patients and caregivers (CGs) to complete the baseline telephone surveysIt was necessary to allow for a wider data collection window, balancing the need for obtaining baseline data before HBPC starts and optimizing data capture. Also, the number of contacts need to be balanced with other clinical calls that patients receive from the health systemMonthly cross-regional and site engagement workgroup for site research staff to discuss and share ideas to enhance patient/caregiver accrualThe HBPC programs have adopted the primary study measure, ESAS, into their workflow that would allow us to extract these data for analysis to supplement data collected by research staff
Interpretation of HBPC program admission criteria varies across sites and teams. System and clinical leaders in one region requested help with standardizing these criteria	The two HBPC programs operate under different CMS' regulatory structures, hospice (n = 3 sites) and home health (n = 12 sites). Sites and HBPC clinicians differ on their interpretation of CMS' home health “skilled need” criteria for admissionAnalyses of baseline ESAS data may provide structured admission guidance to the 12 sites that operate under CMS's home health license. This work is ongoing
Evolving HBPC program staffing and resources
RN case manager attrition/turnover and cross-coverage	To minimize confusion, RNs have been trained to implement the model to which they are assigned, even as they provide cross-coverage for other RNs. This will be tracked as part of our fidelity monitoring plan
Clinical site redistribution of RNs to address changing operational needs	Because of the strong labor partnerships in the health system, clinical sites may be asked over the course of the study to redistribute clinician assignments to ensure workload equity. The study has limited influence over the restructuring but will advocate for RNs to maintain their model assignment
“Soft” boundaries across home health, HBPC and hospice programs that are under one administrative structure	Patients may transition to HBPC from hospice or home health. However, whether because of the need for or request for continuity of care or in response to staffing challenges, the RN from the previous service may continue to manage the patient, even though patient is admitted to HBPC. These patients are not enrolled in the study because they are not linked to a randomized HBPC RN. Strategies to address this challenge are still under consideration
Reorganization of teams with greater integration of LVN staff	Clinical sites are undergoing a fair bit of transition with greater integration of LVNs although LVNs may bridge two RNs assigned to different models of care. Provision of video tools to LVNs (or to other HBPC team members) will help increase team efficiency but can create cross-contamination. However, in situations where an LVN works exclusively with a tech-supported RN, they will be trained to conduct video visits
Shifting HBPC resources to meet increasing demand/prioritization of hospice program	Because of HBPC being offered as part of the health plan benefits and hospice being a Medicare carve out, there are greater pressures to expand and prioritize resources for the hospice program. The research team recently published an article demonstrating the value for the health system to concurrently invest in both programs for patients with serious illness (Wang et al.¹³)
Coordinating HBPC with emerging services as individual sites or regional health system refines ecosystem for serious illness management	It is expected that the health system will introduce new programs or services for patients with serious illness over the course of the study as part of its commitment to optimizing end-of-life care. Research teams in each region are embedded in the discussions and will influence the design of these programs and services to ensure coordination with the study
Evolving telehealth landscape
Reliability, variation, and changes to the video visit application	Continually revised HBPC team “playbook” that includes tips to resolve common technical challenges with video visitsWe expect that, over the course of the study, there will be changes to the video application used in the study as more user-friendly tools become available. We plan to provide close support to the teams to ensure successful transition to any new toolSimilarly, we expect that clinical sites will continue to pilot emerging tools. The study is agnostic to the types of video applications that are used as long as teams use standardized documentation to facilitate data collection.
Use of publicly available unsecured video applications	Standard HBPC team members may use publicly available, unsecured video applications to meet patient needs. The research team needs to be mindful of these anecdotes and, in close collaboration with our health system partners, assess whether restrictions to use of video visits for half of the team could create other security risks and unintended consequences
Payment policy changes affecting home health services and Medicare Advantage plans	Proposed changes to payment policy around telehealth services in 2019 and 2020 may affect the conduct of the study if the health system chooses to respond rapidly to keep up with market pressures. We are working closely with our health system partners to understand how their preparations for these changes will affect HomePal and to strongly advocate for thoughtful, rigorous, and deliberate evaluations of any telehealth efforts, modeling the approach we have taken with HomePal to maximize learnings for practice and policy

CMS, Centers for Medicare and Medicaid Services; IDG, interdisciplinary group.

Despite the broad organizational commitment and support of system and clinical leaders to the study design of allowing half of care teams to continue standard practice while innovating in the tech-supported arm, it still takes substantial effort to engage clinicians who are being asked to alter their usual patterns of care delivery or to hold back while others are innovating. There is notable variation within and across sites in clinician readiness and openness for integrating video visits into their practice. Change management strategies such as reassurance that use of video visits with individual patients is ultimately based on the clinical team's judgment, peer-to-peer sharing of positive experiences by early adopters, and ongoing technical training and coaching have been positively received.

Second, participant enrollment and data collection efforts must allow for inherent programmatic variations and integrate with existing workflows to improve value for the system, to avoid disruptions to current operations, and to minimize the burden on patients/caregivers and clinicians. Health system adoption of the ESAS as a standardized symptom measure across all PC services will enrich the study's data collection efforts. We also need to have realistic expectations for patient enrollment because some sites have limited capacity to enroll new patients. A cross-site engagement workgroup provides a forum for sharing implementation learnings across sites.

Third, with the dynamic and evolving demands on the HBPC program staffing and resources, care team structures will continue to pose challenges. We developed study-wide and local site manuals of operations to provide anticipatory guidance on issues such as handling attrition or reassignment of the randomized RNs. Other related challenges include reassignment of prescribing clinicians and RNs depending on clinical need, reorganization of teams and greater integration of licensed vocational nurses and shifting of staffing and resources from HBPC to hospice programs. Research staff RNs are attending IDG meetings as ethnographers, learning the culture of the teams, and participating in the care delivery discussions. Change happens at the speed of trust and through this mechanism, the research and clinical teams are collaboratively troubleshooting unfolding video visit implementation challenges.

Finally, the telehealth landscape will continue to evolve over the course of the study in tandem with programmatic changes described previously. Our priority is to maintain standardized procedures for collecting data across sites and encourage organizational commitment to rigorous evaluation of the two HBPC delivery modalities even as clinical teams further innovate. The cross-site coordination from the DCC also anticipates and has proactively established practices for monitoring and engaging the stakeholder advisory groups, cross-site research staff, and DSMB in activities such as monitoring and accounting for the impact of system differences and regulatory and policy changes. Maintaining effective collaborations with system leaders and clinicians by providing ongoing audit and feedback, sharing new information, and supporting processes introduced by the study is intended to help the HBPC program achieve greater efficiency, access, and improved care experience.

Plans for dissemination

A key facilitator to dissemination and implementation of the more efficient tech-supported HBPC model is our involvement of key stakeholders throughout the process of conceptualizing, developing, and evaluating this intervention. We have and are continuing to incorporate the perspectives of patients, caregivers, clinicians, administrators, high-level operational and executive leadership, payers, advocacy groups, and policy makers throughout the study to ensure the tech-supported model is acceptable to this diverse group of stakeholders.

Our dissemination plan and activities are guided by an emerging framework (derived from ongoing PCORI and non-PCORI studies) outlining a systematic, comprehensive approach for planning and conducting patient-centered outcomes research dissemination and implementation. This framework includes distinct phases of activity to (1) assess, in advance and continually, potential policy and practice implications of the study; (2) identify stakeholders likely to be affected by—and interested in—study results; (3) strategically involve these stakeholders in identifying potential implications and stakeholder groups' likely responses to findings; (4) proactively and systematically work to minimize any adverse reactions and maximize beneficial reactions and steps to benefit from the study; and (5) plan, design, and carry out specific communication, dissemination, and implementation activities to facilitate and encourage desired stakeholder response to the study. The approach includes numerous activities conducted well before study findings are available, in contrast to many dissemination approaches that begin only upon the availability of findings.

Conclusions

The HomePal study is highly responsive to the imperative from collaborating stakeholders to move beyond the four clinic walls and provide PC in patients' homes, especially when high symptom burden is coupled with significant functional limitations, making clinic visits too burdensome for both patients and caregivers. It also addresses gaps in the current state of the science in PC³⁶ and HBPC in particular,^37-44 with regard to rigorous evaluations of telehealth integration. In close partnership with stakeholders across multiple representative groups, we refined the comparators, prioritized, and refined measures and study conduct, and optimized rigor and statistical approaches. Our fidelity monitoring protocol, mixed-methods evaluations, and early planning for dissemination incorporated anticipated challenges and mitigation strategies. The comparison between standard HBPC and a potentially more efficient, tech-supported HBPC model will provide much-needed evidence and guidance to policy and practice leaders interested in providing effective, scalable, and sustainable seamless high-quality supportive care services.

Footnotes

Acknowledgments

The authors are greatly appreciative of the HomePal study participants, the entire HomePal Research Group, which includes investigators, clinical partners, and staff from Kaiser Permanente Southern California and Kaiser Permanente Northwest, consultants, SAC members, and members of the data and safety monitoring board. Kaiser Permanente Southern California, Pasadena, CA:

Author Disclosure Statement

None of the authors have a conflict of interest with the content of this paper.

All statements in this report, including its findings and conclusions, are solely those of the authors and do not necessarily represent the views of the PCORI, its Board of Governors, or Methodology Committee.

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