Evaluating patient-reported adherence and outcomes in specialty disease states: A dual-site initiative.

BACKGROUND: Patient-reported outcomes (PROs) are often used by clinicians to evaluate patient response to specialty medications used to treat multiple sclerosis (MS) and rheumatologic conditions. Identifying associations among PROs and patient characteristics could inform patient-centered treatment monitoring. OBJECTIVE: To examine the association among patient characteristics and PROs, including patient-reported adherence (defined as no missed doses), medication tolerance, patient perceived effectiveness, and health care resource utilization (HCRU; defined as emergency department visits or hospitalizations), for patients prescribed specialty medications in 2 health system specialty pharmacies. METHODS: A dual-center, retrospective review of monthly medication assessments completed by Vanderbilt Specialty Pharmacy and University of Illinois Hospital and Health Sciences System specialty pharmacy was conducted. Patients were included if they received at least 3 fills of a specialty medication from rheumatology or MS clinics from October 2019 to March 2022, excluding patients with more than a 30-day supply. Primary outcomes were the PROs of patient-reported adherence, medication tolerability, perceived effectiveness, and HCRU. For each of the 2 primary outcomes (adherence and tolerability), a mixed-effects logistic regression model was used to test for associations with age, sex, race, clinic, site, and the other PROs. RESULTS: A total of 61,926 assessments were completed from 3,677 patients (Site 1 = 3,346; 91.0% and Site 2 = 331; 9.0%). Patients were predominantly White (75.6%) and female (71.7%) with a median age of 50 years (IQR = 37-61). Assessments most frequently originated from rheumatology (76.0%). Nonadherence was reported 4.0% of the time, with the most common explanations being forgetfulness (33.1%) and medication being held because of a procedure or illness (29.5%). Most responses indicated perceived effectiveness as good/excellent (93.9%), with 98.5% of responses indicating no issues with tolerability. Patients who reported tolerability issues were 2.5 times more likely to report a missed dose (95% CI = 1.87-3.23, P < 0.001). An effectiveness rating of fair was associated with a 61% increase in the odds of a missed dose compared with a rating of good/excellent (95% CI = 1.33-1.94). CONCLUSIONS: Patients filling rheumatology or MS specialty medications within health system specialty pharmacies reported high rates of medication effectiveness and adherence and low rates of issues with tolerability and HCRU. Patients who report tolerability issues or lower perceived effectiveness may benefit from additional monitoring to prevent nonadherence.

An expert panel Delphi consensus statement on the use of palliative care in the management of patients with pulmonary arterial hypertension.

Mortality in pulmonary arterial hypertension (PAH) remains high and referral to palliative or supportive care (P/SC) specialist services is recommended when appropriate. However, access to P/SC is frequently a challenge for patients with a noncancer diagnosis and few patients living with PAH report P/SC involvement in their care. A modified Delphi process of three questionnaires completed by a multidisciplinary panel (N = 15) was used to develop expert consensus statements regarding the use of P/SC to support patients with PAH. Panelists rated their agreement with each statement on a Likert scale. There was a strong consensus that patients should be referred to P/SC when disease symptoms become unmanageable or for end-of-life care. Services that achieved consensus were pain management techniques, end-of-life care, and psychosocial recommendations. Palliative or supportive care should be discussed with patients, preferably in-person, when disease symptoms become unmanageable, when starting treatment, when treatment-related adverse events occur or become refractory to initial intervention. Care partners and patient support groups were considered important in improving a patient's overall health outcomes, treatment adherence, and perception of care. Most patients with PAH experience cognitive and/or psychosocial changes and those who receive psychosocial management have better persistence and/or compliance with their treatment. These consensus statements provide guidance to healthcare providers on the "who and when" of referral to palliative care services, as well as the importance of focusing on the psychosocial aspects of patient care and quality of life.

Comparison of provider satisfaction with specialty pharmacy services in integrated health-system and external practice models: A multisite survey.

PURPOSE: The purpose of this study is to obtain insight into providers' satisfaction with services offered by health-system integrated specialty pharmacies and to determine whether providers' perceptions of services offered under an integrated model differ from perceptions of external specialty pharmacy services. METHODS: A multi-site, cross-sectional, online survey of specialty clinic healthcare providers at 10 academic health systems with integrated specialty pharmacies was conducted. The questionnaire was developed by members of the Vizient Specialty Pharmacy Outcomes and Benchmarking Workgroup and was pretested at 3 pilot sites prior to dissemination. Prescribers of specialty medications within each institution were identified and sent an email invitation to participate in the study that included a link to the anonymous questionnaire. Respondents were asked to rate their agreement with 10 statements regarding quality of services of integrated and external specialty pharmacies on a 5-point scale (1 = strongly disagree, 5 = strongly agree). An analysis to determine differences in providers' overall satisfaction with the integrated and external specialty pharmacy practice models, as well as differences in satisfaction scores for each of the 10 statements, was performed using paired-samples t tests. RESULTS: The mean (SD) score for overall satisfaction with integrated specialty pharmacies was significantly higher than the score for satisfaction with external specialty pharmacies: 4.72 (0.58) vs 2.97 (1.20); 95% confidence interval, 1.64-1.87; P < 0.001. Provider ratings of the integrated specialty pharmacy model were also higher for all 10 items evaluating the quality of services (P < 0.05 for all comparisons). CONCLUSION: The study results confirm that the health-system integrated specialty pharmacy practice model promotes high rates of provider satisfaction with services and perceived benefits.

Correction to: Medication Adherence and Healthcare Costs Among Patients with Pulmonary Arterial Hypertension Treated with Oral Prostacyclins: A Retrospective Cohort Study.

Changes within text are indicated in bold.

Medication Adherence and Healthcare Costs Among Patients with Pulmonary Arterial Hypertension Treated with Oral Prostacyclins: A Retrospective Cohort Study.

BACKGROUND: Given the improved convenience of oral prostacyclins, there is a shift toward their use in treating pulmonary arterial hypertension (PAH). OBJECTIVES: Our objective was to compare patient characteristics, medication adherence, healthcare resource use (HCRU), and costs among patients receiving oral treprostinil or selexipag. METHODS: We used Truven Health MarketScan Commercial and Medicare databases to identify patients with PAH with a diagnosis code for pulmonary hypertension (PH) plus a prescription for oral treprostinil or selexipag from July 2013 to September 2017. Medication adherence, persistence, and all-cause and PAH-related HCRU and costs were compared between cohorts during the 6-month follow-up. Adjusted healthcare costs were obtained using recycled predictions and bootstrapped samples. RESULTS: A total of 256 (130 oral treprostinil, 126 selexipag) patients fulfilled the study criteria. The oral treprostinil cohort was more likely to be male, to have previously used parenteral prostacyclins, and to have higher outpatient costs at baseline than the selexipag cohort. During follow-up, both cohorts had similar proportions of patients who were adherent to and persistent with their respective therapies. All-cause and PAH-related medical utilization was generally similar between cohorts. The oral treprostinil cohort had 66.9% lower total PAH-related healthcare costs (mean difference - $75,183; 95% confidence interval [CI] - 102,584 to - 49,771) and 70.6% lower PAH-related pharmacy costs (mean difference - $76,439; 95% CI - 104,512 to - 51,458) than the selexipag cohort, with similar differences in all-cause healthcare and pharmacy costs. CONCLUSIONS: Lower all-cause and PAH-related total healthcare and pharmacy costs were observed in patients receiving oral treprostinil compared with those receiving selexipag. It will be important to study longer-term costs and clinical outcomes.

The Role of Noninvasive Endpoints in Predicting Long-Term Outcomes in Pulmonary Arterial Hypertension.

BACKGROUND: Until recently, many clinical trials in patients with pulmonary arterial hypertension (PAH) evaluated exercise capacity with 6-minute walk distance (6MWD) as the primary endpoint. Common secondary endpoints include PAH functional class (FC), which assesses symptoms, and either brain natriuretic peptide (BNP) or the inactive N-terminal cleavage product of its prohormone (NT-proBNP), which assesses cardiac function. OBJECTIVE: Examine the relationships among 6MWD, FC, and BNP/NT-proBNP measured at baseline or follow-up with long-term outcomes in PAH studies. METHODS: Relevant literature from January 1990 to April 2018 were obtained by searching PubMed, Embase, and Cochrane. Articles in English reporting on associations between 6MWD, FC, or BNP/NT-proBNP and outcomes in PAH were identified. Each endpoint was evaluated individually. Prespecified inclusion and exclusion criteria were applied at level 1 (titles/abstracts) and level 2 (full-text review). RESULTS: The database search yielded 836 unique records; 65 full-text articles were reviewed. Twenty-five studies were eligible for inclusion. Findings supported the importance of measuring PAH noninvasive endpoints in predicting long-term outcomes. Patients with shorter or decreased 6MWD, poor (III/IV) or declining FC (e.g., from II to III), or elevated or increasing BNP/NT-proBNP had a higher risk of death and costly events (e.g., hospitalization, lung transplant). FC also predicted health care resource utilization and costs. Collectively, these endpoints establish risk groups that predict likelihood of complications from PAH or death. CONCLUSION: Assessment of 6MWD, FC, and BNP/NT-proBNP provides low-cost, efficient, and noninvasive means of predicting long-term health and economic outcomes in patients with PAH.

Navigating the Wild West of Medication Adherence Reporting in Specialty Pharmacy.

Estimating medication adherence through the use of pharmacy claims-based adherence calculations such as medication possession ratio (MPR) and proportion of days covered (PDC) plays a significant role in specialty pharmacy practice. Although MPR and PDC are frequently used in clinical practice, calculation methodologies vary, making meaningful comparisons of adherence rates difficult. In addition, MPR and PDC are increasingly used by insurance companies, pharmacies, accrediting bodies, and drug manufacturers to demonstrate quality differences or clinical benefit across the specialty pharmacy industry. Therefore, recognizing the source and effect of calculation variability is necessary to fully understand reported adherence results. This article highlights the challenges in standardizing adherence methodologies, minimum methodology considerations that should be reported with MPR and PDC results, and key elements to consider when interpreting and applying adherence results. Further, recommendations are provided to promote a more consistent description of calculation methods and to aid pharmacies in adherence measure analysis, interpretation, and application to practice, with a focus on specialty pharmacy programs. A detailed description of methodology as outlined in this article must be provided to ensure reproducibility, external validation, and scientific rigor. In the absence of standardization, specialty pharmacies should be prudent in their use of adherence calculations as a clinical benchmarking tool or comparative quality indicator with outside organizations. Furthermore, specialty pharmacies should consider using current adherence measure calculations to identify and provide targeted interventions to patients with potential adherence problems and strive to better demonstrate ties between adherence measures and direct clinical and cost outcomes. DISCLOSURES: No outside funding supported the writing of this article. Anguiano is a speaker and research consultant for United Therapeutics. The other authors have nothing to disclose.

Identifying Patients with Pulmonary Arterial Hypertension Using Administrative Claims Algorithms.

Retrospective administrative claims database studies provide real-world evidence about treatment patterns, healthcare resource use, and costs for patients and are increasingly used to inform policy-making, drug formulary, and regulatory decisions. However, there is no standard methodology to identify patients with pulmonary arterial hypertension (PAH) from administrative claims data. Given the number of approved drugs now available for patients with PAH, the cost of PAH treatments, and the significant healthcare resource use associated with the care of patients with PAH, there is a considerable need to develop an evidence-based and systematic approach to accurately identify these patients in claims databases. A panel of pulmonary hypertension clinical experts and researchers experienced in retrospective claims database studies convened to review relevant literature and recommend best practices for developing algorithms to identify patients with PAH in administrative claims databases specific to a particular research hypothesis.

Managing the Risks of Medicines: An Examination of FDA's Application of Criteria for Requiring a REMS.

BACKGROUND: In September 2016, the Food and Drug Administration (FDA) published a draft guidance for industry, FDA's Application of Statutory Factors in Determining When a REMS Is Necessary, that detailed the factors the Agency considers in determining when a Risk Evaluation and Mitigation Strategy (REMS) is necessary. The objective of this study was to determine how the FDA has applied these criteria for newly approved drugs. METHODS: For the 3-year period, 2015-2017, which included a full year of FDA approvals both before and after the issuance of the draft guidance, publicly available FDA reviews were analyzed for all 113 approved products using the criteria outlined in the guidance. RESULTS: Of the 113 products approved, 5 required a REMS. The most cited reasons for not requiring a REMS for the remaining 108 drugs were that risks could be managed via professional labeling (87%), physicians (primarily specialists) were familiar with the management of the risks (76%), the risk profile was similar to other non-REMS marketed products (45%), products were used in a controlled setting (inpatient, infusion center) (30%), and/or safety concerns would be further evaluated by a postmarket study (14%). CONCLUSIONS: A review of Agency risk evaluations indicate that whether physicians are sufficiently familiar with and capable of managing a risk and that the health care setting where the product is administered is conducive to such management are leading factors in determining whether or not to require a REMS.

Medication adherence and persistence over time with self-administered TNF-alpha inhibitors among young adult, middle-aged, and older patients with rheumatologic conditions.

OBJECTIVE: Self-injectable TNF inhibitors are increasingly used early in the chronic treatment of moderate to severe rheumatologic conditions. We estimated medication adherence/persistence over time following initiation in young adult and older adult patients with rheumatoid arthritis, ankylosing spondylitis or psoriatic arthritis. METHODS: We conducted a retrospective cohort study of patients aged 18+ years newly initiating etanercept, adalimumab, certolizumab pegol, or golimumab using the Truven Health MarketScan Database between 2009 and 2013. Pharmacy dispensing data were used to calculate 12-month medication possession ratios (MPR) and determine adherence (MPR ≥ 0.80) for up to 3 years after starting therapy. Persistence over each 12-month interval was defined as not having a ≥92-day treatment gap. Multivariable generalized estimating equation models were used to calculate odds ratios (OR) and robust 95% confidence intervals (CI) for associations between patient characteristics and repeated adherence/persistence measures over time. RESULTS: Among 53,477 new users, 14% were young adults (18-34 years), 49% middle-aged (35-54 years), and 37% older adults (55+ years). Overall, 37% of patients were adherent and 83% were persistent in the first year of therapy. The lowest adherence (17%) and persistence (70%) were observed among young adult patients by Year +3. Compared to older adults, middle-aged (OR = 0.73, 95% CI: 0.71-0.76) and young adults (OR = 0.50, 95% CI: 0.47-0.53) were less likely to be adherent. Higher Charlson comorbidity scores, hospitalizations, and emergency department visits were associated with non-adherence/non-persistence. CONCLUSIONS: We observed low adherence to self-administered TNF inhibitors but most patients remained persistent over time. Further efforts to improve adherence in young adults and patients with greater comorbidity are needed.