Commercial Health Plan and Enrollee Out-of-Pocket Spending on Accelerated Approval Products in 2019.

This cross-sectional study examines spending by health care plans and enrollees on products with accelerated approval.

Time to Confirmatory Study Initiation After Accelerated Approval of Cancer and Noncancer Drugs in the US.

This cross-sectional study examines and compares the time taken from the accelerated approval of cancer and noncancer drugs to the initiation of confirmatory studies in the US.

Association between low-income subsidies and inequities in orally administered antimyeloma therapy use.

OBJECTIVES: The Medicare Part D low-income subsidy program drastically reduces patient cost sharing and may improve access to and equitable use of high-cost antimyeloma therapy. We compared initiation of and adherence to orally administered antimyeloma therapy between full-subsidy and nonsubsidy enrollees and assessed the association between full subsidies and racial/ethnic inequities in orally administered antimyeloma treatment use. STUDY DESIGN: Retrospective cohort study. METHODS: We used Surveillance, Epidemiology, and End Results-Medicare data to identify beneficiaries diagnosed with multiple myeloma between 2007 and 2015. Separate Cox proportional hazards models assessed time from diagnosis to treatment initiation and time from therapy initiation to discontinuation. Modified Poisson regression examined therapy initiation in the 30, 60, and 90 days following diagnosis and adherence to and discontinuation of treatment in the 180 days following initiation. RESULTS: Receipt of full subsidies was not associated with earlier initiation of or improved adherence to orally administered antimyeloma therapy. Full-subsidy enrollees were 22% (adjusted HR [aHR], 1.22; 95% CI, 1.08-1.38) more likely to experience earlier treatment discontinuation than nonsubsidy enrollees. Receipt of full subsidies did not appear to reduce racial/ethnic inequities in orally administered antimyeloma therapy use. Black full-subsidy and nonsubsidy enrollees were 14% less likely than their White counterparts to ever initiate treatment (full subsidy: aHR, 0.86; 95% CI, 0.73-1.02; nonsubsidy: aHR, 0.86; 95% CI, 0.74-0.99). CONCLUSIONS: Full subsidies alone are insufficient to increase uptake or equitable use of orally administered antimyeloma therapy. Addressing known barriers to care (eg, social determinants of health, implicit bias) could improve access to and use of high-cost antimyeloma therapy.

Intensifying approaches to address clinical inertia among cardiovascular disease risk factors: A narrative review.

OBJECTIVE: Clinical inertia, the absence of treatment initiation or intensification for patients not achieving evidence-based therapeutic goals, is a primary contributor to poor clinical outcomes. Effectively combating clinical inertia requires coordinated action on the part of multiple representatives including patients, clinicians, health systems, and the pharmaceutical industry. Despite intervention attempts by these representatives, barriers to overcoming clinical inertia in cardiovascular disease (CVD) risk factor control remain. METHODS: We conducted a narrative literature review to identify individual-level and multifactorial interventions that have been successful in addressing clinical inertia. RESULTS: Effective interventions included dynamic forms of patient and clinician education, monitoring of real-time patient data to facilitate shared decision-making, or a combination of these approaches. Based on findings, we describe three possible multi-level approaches to counter clinical inertia - a collaborative approach to clinician training, use of a population health manager, and use of electronic monitoring and reminder devices. CONCLUSION: To reduce clinical inertia and achieve optimal CVD risk factor control, interventions should consider the role of multiple representatives, be feasible for implementation in healthcare systems, and be flexible for an individual patient's adherence needs. PRACTICE IMPLICATIONS: Representatives (e.g., patients, clinicians, health systems, and the pharmaceutical industry) could consider approaches to identify and monitor non-adherence to address clinical inertia.

Medicaid and Accelerated Approval: Spending on Drugs with and without Proven Clinical Benefits.

Many state Medicaid officials are concerned about rising prescription drug spending, particularly drugs approved through the Food and Drug Administration's (FDA) accelerated approval pathway. The authors examined how much of Medicaid programs' accelerated approval spending is attributable to products that have demonstrated clinical benefits versus those that have not. Their findings provide support for states' concerns that pharmaceutical companies often fail to complete their required postapproval confirmatory studies within the FDA's requested timeline. But the findings also highlight one issue that policy stakeholders have not yet devoted substantial attention to: the use of surrogate endpoints involved in the postapproval confirmatory studies for most of the products in this study's sample. The granularity of the study's results enabled an analysis of the impact of different policy recommendations on both the accelerated approval pathway and Medicaid programs. These findings inform the current policy debate, suggesting that policy stakeholders might focus attention on products converting their approval on the basis of surrogate outcomes rather than on clinical outcomes.

Association of High-Deductible Health Plan Enrollment With Spending on and Use of Lenalidomide Therapy Among Commercially Insured Patients With Multiple Myeloma.

Importance: High-deductible health plans (HDHPs) require high upfront cost-sharing, which has been associated with suboptimal anticancer medication uptake and adherence. Whether HDHP enrollment has limited the affordability and use of lenalidomide therapy among commercially insured patients with multiple myeloma is unknown. Objective: To assess the association of HDHP enrollment with out-of-pocket spending on and adherence to lenalidomide therapy. Design, Setting, and Participants: In this cohort study, data were obtained from the IBM MarketScan Commercial Claims and Encounters Database for adults aged 18 to 64 years with multiple myeloma who newly initiated lenalidomide therapy between April 1, 2013, and June 30, 2017. Quantile regression and modified Poisson regression evaluated out-of-pocket spending, and group-based trajectory models and multinomial logistic regression examined patterns of and factors associated with adherence. Analyses were conducted from April to August 2020. Exposures: High-deductible health plan enrollment in the 3 months before and 6 months after initiation of lenalidomide therapy. Main Outcomes and Measures: Distribution of out-of-pocket spending, the probability of paying more than $100 for the first and any lenalidomide prescription fill, and monthly lenalidomide therapy adherence using the proportion of days covered (≥80%). Results: Of the 3163 commercially insured patients who initiated lenalidomide therapy (median age, 57 years [IQR, 53-60 years for HDHP enrollees and 52-61 years for non-HDHP enrollees]), 328 (10.4%) were enrolled in HDHPs and 1769 (55.9%) were women. Among the highest spenders (95th percentile), HDHP enrollees paid $376 (95% CI, -$28 to $780) and $217 (95% CI, $106-$323) more for their first and any lenalidomide prescription fill, respectively, compared with non-HDHP enrollees in the 6 months after initiation. High-deductible health plan enrollment was also associated with an increased risk of paying more than $100 for the initial (adjusted risk ratio [aRR], 1.30 [95% CI, 1.13-1.50]) and any (aRR, 1.26 [95% CI, 1.12-1.42]) lenalidomide prescription fill. Three adherence trajectory groups were identified: those with high adherence (n = 1273), late nonadherence (n = 1084), and early nonadherence (n = 805). High-deductible health plan enrollment was not associated with adherence group assignment. Conclusions and Relevance: In this cohort study, HDHP enrollment was associated with higher out-of-pocket spending per lenalidomide prescription fill; however, no statistically significant differences in adherence patterns between HDHP and non-HDHP enrollees were observed. Patient (eg, perceptions of treatment benefits), payer (eg, out-of-pocket maximums), and clinician (eg, counseling patients on disease severity) factors may have limited the potential for nonadherence among commercially insured patients who initiated lenalidomide therapy.

Predictors of Self-repackaging of Antiretroviral Therapy in Northern Tanzania.

Objectives: In this study, we explored determinants of "researcher-observed" patient-initiated antiretroviral therapy (ART) repackaging practices among people living with HIV (PLHIV) in Northern Tanzania. Methods: We used a quasi-experimental design to describe the prevalence of ART self- repackaging; we conducted face-to-face surveys to determine factors associated with ART self- repackaging practices. Data collection sites included the Kilimanjaro Christian Medical Centre and the Mawenzi Referral Hospital. We used study-specific numerical identifiers assigned to ARTs packaging to determine self-repackaging behavior. Self-repackaging was defined as a binary variable where participants who discarded antiretroviral drugs packaging in at least 2 clinic visits were classified as self- repackagers. We used multivariable logistic regression to assess the determinants of patient-initiated repackaging practices. Results: Among 590 study participants, 57.6% self-repackaged based on researcher observation and 55.6% self-repackaged based on patient report. Researcher-observed self- repackaging was associated with gender (AOR = 1.590; 95% CI: 1.011, 2.502), employment status (AOR = 0.475: 95% CI; 0.239, 0.942), and study site (AOR = 0.218; 95% CI: 0.134, 0.355). Conclusions: A substantial proportion of patients self-repackage their ARTs for various reasons. Health system interventions should focus on addressing the attributes of repackaging among men and unemployed patients.

Transportation as a barrier to colorectal cancer care.

BACKGROUND: Transportation barriers limit access to cancer care services and contribute to suboptimal clinical outcomes. Our objectives were to describe the frequency of Veterans reporting and the factors associated with transportation barriers to or from colorectal cancer (CRC) care visits. METHODS: Between November 2015 and September 2016, Veterans with incident stage I, II, or III CRC completed a mailed survey to assess perceived barriers to recommended care. Participants who reported difficulty with transportation to or from CRC care appointments were categorized as experiencing transportation barriers. We assessed pairwise correlations between transportation barriers, transportation-related factors (e.g., mode of travel), and chaotic lifestyle (e.g., predictability of schedules), and used logistic regression to examine the association between the reporting of transportation difficulties, distance traveled to the nearest Veterans Affairs (VA) facility, and life chaos. RESULTS: Of the 115 Veterans included in this analysis, 18% reported experiencing transportation barriers. Distance to the VA was not strongly correlated with the reporting of transportation barriers (Spearman's ρ = 0.12, p = 0.19), but chaotic lifestyle was both positively and significantly correlated with experiencing transportation barriers (Spearman's ρ = 0.22, p = 0.02). Results from the logistic regression model modestly supported the findings from the pairwise correlations, but were not statistically significant. CONCLUSIONS: Transportation is an important barrier to or from CRC care visits, especially among Veterans who experience greater life chaos. Identifying Veterans who experience chaotic lifestyles would allow for timely engagement in behavioral interventions (e.g., organizational skills training) and with support services (e.g., patient navigation).

Comparison of Anticancer Medication Use and Spending Under US Oncology Parity Laws With and Without Out-of-Pocket Spending Caps.

Importance: By 2020, nearly all states had adopted oncology parity laws in the US, ensuring that patients in fully insured private health plans pay no more for orally administered anticancer medications (OAMs) than infused therapies. Between 2013 and mid-2017, 11 states implemented parity with out-of-pocket spending caps, which may further reduce patient out-of-pocket spending. Objective: To compare OAM uptake and out-of-pocket and health plan spending on OAMs in states with parity with and without spending caps, as well as to assess out-of-pocket spending for caps that apply predeductible vs postdeductible. Design Setting and Participants: This cohort study analyzed OAM users enrolled in commercial health plans offered by Aetna, Humana, and United Healthcare in the US from 2011 to 2017, aggregated by the Health Care Cost Institute, using difference-in-difference-in-differences (DDD) analysis. Data analysis was conducted between June and August 2020. Exposures: Time (before vs after parity), whether the state parity law included an out-of-pocket spending cap, and whether the plan was fully insured (subject to parity) or self-funded (not subject to parity). Among states with caps, out-of-pocket spending was also compared by whether the cap was applied predeductible and postdeductible vs only postdeductible. Main Outcomes and Measures: Monthly OAM prescription fills per 100 000 enrollees, per-OAM prescription-fill out-of-pocket spending, and annual per-user health plan spending on OAMs. Results: In this study of 23 states (11 with caps and 12 without) and 207 579 OAM prescription fills, caps were associated with a modest increase in OAM use (DDD, 7.40 [95% CI, 3.41-11.39] per 100 000 enrollees). There was no difference in mean out-of-pocket spending comparing fully insured and self-funded enrollees in states with vs without caps (DDD, -$17 [95% CI, -$57 to $24), but caps were associated with lower spending among OAM users in the 95th percentile of out-of-pocket spending by $831 (95% CI, -$871 to -$791) per OAM prescription fill. Caps applied predeductible were associated with greater out-of-pocket savings relative to caps applied only postdeductible. This included per-OAM prescription-fill savings at the 75th, 90th, and 95th percentiles. Postparity, mean annual spending on OAMs among users was $113 589 in states without caps and $102 252 in states with caps, with no differences between groups (DDD, $9799 [95% CI, -$4230 to $23 829). Conclusions and Relevance: In this cohort study, among states adopting oncology parity laws between 2013 and 2017, mean out-of-pocket spending per OAM prescription fill and mean health plan spending among OAM users was similar in states with and without caps. However, enrollees in states with parity plus out-of-pocket caps had greater reductions in out-of-pocket spending among the highest spenders. Caps may offer improved financial protection for the highest spenders without increasing mean health plan spending on OAMs.

Does Antiretroviral Therapy Packaging Matter? Perceptions and Preferences of Antiretroviral Therapy Packaging for People Living with HIV in Northern Tanzania.

INTRODUCTION: Despite improvements in treatment (eg, reduction in pill intake), antiretroviral therapy (ART) is dispensed in socially inefficient and uneconomical packaging. To make pills less conspicuous and decrease the risk of being stigmatized, people living with HIV (PLWH) often engage in self-repackaging - the practice of transferring ART from original packaging to alternative containers. This behavior has been associated with ART nonadherence and failure to achieve viral load suppression. While much of the literature on ART packaging has centered around medication adherence, patients stated preferences for ART packaging and packaging attributes that influence the observed ART nonadherence are understudied. METHODS: We conducted a qualitative study to elucidate perceptions of ART packaging among PLWH at two large referral hospitals in Northern Tanzania. Interviews were conducted until thematic saturation was reached. Interviews were audio-recorded, transcribed and coded. RESULTS: Of the 16 participants whose data were used in the final analysis, a majority were between 36 and 55 years of age (Mean 45.5 years SD: 11.1), had primary-level education (n=11, 68.8%), were self-employed (n=9, 56.3%), reported that they had self-repacked ART (n=14, 88%), and were taking ART for more than 6 years (n=11, 68.8%). Participants identified three attributes of ART packaging that increased anticipated HIV stigma and prompted self-repackaging, including visual identification, bulkiness, and the rattling noise produced by ART pill bottles. CONCLUSION: Given the drastic reduction in the number of pills required for HIV treatment, there is an opportunity to not only assess the cost-effectiveness of innovative ART packaging but also evaluate the acceptability of such packaging among PLWH in order to address stigma and improve ART adherence.

Primary Care Providers' Acceptance of Pharmacists' Recommendations to Support Optimal Medication Management for Patients with Diabetic Kidney Disease.

BACKGROUND: Patients with diabetic kidney disease (DKD) often struggle with blood pressure control. In team-based models of care, pharmacists and primary care providers (PCPs) play important roles in supporting patients' blood pressure management. OBJECTIVE: To describe whether PCPs' acceptance of pharmacists' recommendations impacts systolic blood pressure (SBP) at 36 months. DESIGN: An observational analysis of a subset of participants randomized to the intervention arm of the Simultaneous risk factor control using Telehealth to slOw Progression of Diabetic Kidney Disease (STOP-DKD) study. PARTICIPANTS: STOP-DKD participants for whom (1) the pharmacist made at least one recommendation to the PCP; (2) there were available data regarding the PCP's corresponding action; and (3) there were SBP measurements at baseline and 36 months. INTERVENTION: Participants received monthly telephone calls with a pharmacist addressing health behaviors and medication management. Pharmacists made medication-related recommendations to PCPs. MAIN MEASURES: We fit an unadjusted generalized linear mixed model to assess the association between the number of pharmacists' recommendations for DKD and blood pressure management and PCPs' acceptance of such recommendations. We used a linear regression model to evaluate the association between PCP acceptance and SBP at 36 months, adjusted for baseline SBP. KEY RESULTS: Pharmacists made 176 treatment recommendations (among 59 participants), of which 107 (61%) were accepted by PCPs. SBP significantly declined by an average of 10.5 mmHg (p < 0.01) among 47 of 59 participants who had valid measurements at baseline and 36 months. There was a significant association between the number of pharmacist recommendations and the odds of PCP acceptance (OR 1.19; 95%CI 1.00, 1.42; p < 0.05), but no association between the number of accepted recommendations and SBP. CONCLUSIONS: Pharmacists provided actionable medication-related recommendations. We identified a significant decline in SBP at 36 months, but this reduction was not associated with recommendation acceptance. TRIAL REGISTRATION: NCT01829256.

Oral Oncology Parity Laws, Medication Use, and Out-of-Pocket Spending for Patients With Blood Cancers.

BACKGROUND: In this study, we sought to estimate the association between oral oncology parity law adoption and anticancer medication use for patients with chronic myeloid leukemia or multiple myeloma. METHODS: This was an observational study of administrative claims from 2008 to 2017. Among individuals initiating tyrosine kinase inhibitors (TKIs) for chronic myeloid leukemia or immunomodulatory drugs for multiple myeloma, we compared out-of-pocket spending, adherence, and discontinuation before and after parity among individuals in fully insured plans (subject to parity) vs self-funded plans (exempt from parity) using propensity-score weighted difference-in-differences regression models. RESULTS: Among patients initiating TKIs (N = 2082) or immunomodulatory drugs (N = 3326) there were no statistically significant differences in adherence or discontinuation associated with parity. The proportion of patients with initial out-of-pocket payments of $0 increased in fully insured plans after parity from 5.7% to 46.1% for TKIs and from 10.9% to 48.8% for immunomodulatory drugs. Relative to changes in self-funded plans, those in fully insured plans were 4.27 (95% CI = 2.20 to 8.27) times as likely to pay nothing for TKIs and 1.96 (95% CI = 1.40 to 2.73) times as likely to pay nothing for immunomodulatory drugs after parity. Similarly, the proportion paying more than $100 decreased from 30.3% to 24.7% for TKIs and 30.6% to 27.5% for immunomodulatory drugs in fully insured plans after parity. Relative to changes in self-funded plans, those in fully insured plans were 0.74 (95% CI = 0.54 to 1.01) times as likely to pay more than $100 for TKIs and 0.85 (95% CI = 0.68 to 1.06) times as likely to pay more than $100 for immunomodulatory drugs after parity. CONCLUSIONS: Among patients initiating TKIs or immunomodulatory drugs, parity was not associated with better adherence or less discontinuation of therapy but yielded decreased patient out-of-pocket payments for some patients.

Initiation of generic imatinib may improve medication adherence for patients with chronic myeloid leukemia.

PURPOSE: To compare adherence to tyrosine kinase inhibitors (TKIs) between patients with chronic myeloid leukemia (CML) who initiated branded or generic imatinib. METHODS: We used MarketScan commercial claims data (January 2011-June 2018) to identify patients with CML who newly initiated branded imatinib before 1 August 2015 or generic imatinib on or after 2 February 2016, and were continuously enrolled in health plans for 6 months before through 6 months following their initial fill. After inverse probability of treatment weighting, we compared adherence (proportion of days covered [PDC]) and persistence (no gaps ≥30 and ≥60 consecutive days in therapy) to TKI therapy. RESULTS: Patients initiating generic imatinib consistently had higher average PDC per month and over the 6-month follow-up period than initiators of branded imatinib. Average 6-month PDC was 92% (95%CI:89%-94%) for generic initiators and 85% (95%CI:83%-86%) for brand initiators. Compared with branded imatinib initiators, a larger proportion of generic imatinib initiators were adherent and persistent to TKI therapy (PDC ≥ 90%:78% versus 64%; no≥60-day gap:94% versus 86%). CONCLUSIONS: Patients initiating generic imatinib achieved clinically significant improvements in adherence to TKI therapy relative to branded drug users, presumably due to lower out-of-pocket costs. Given the importance of optimal adherence in CML, considering barriers to adherence (eg, patient-cost sharing and health benefit design) when selecting initial treatment may improve long-term medication adherence. Pharmacoepidemiologic studies should consider how best to account for expected cost-sharing and its impact on adherence and subsequent clinical outcomes.

Novel application of approaches to predicting medication adherence using medical claims data.

OBJECTIVE: To compare predictive analytic approaches to characterize medication nonadherence and determine under which circumstances each method may be best applied. DATA SOURCES/STUDY SETTING: Medicare Parts A, B, and D claims from 2007 to 2013. STUDY DESIGN: We evaluated three statistical techniques to predict statin adherence (proportion of days covered [PDC ≥ 80 percent]) in the year following discharge: standard logistic regression with backward selection of covariates, least absolute shrinkage and selection operator (LASSO), and random forest. We used the C-index to assess model discrimination and decile plots comparing predicted values to observed event rates to evaluate model performance. DATA EXTRACTION: We identified 11 969 beneficiaries with an acute myocardial infarction (MI)-related admission from 2007 to 2012, who filled a statin prescription at, or shortly after, discharge. PRINCIPAL FINDINGS: In all models, prior statin use was the most important predictor of future adherence (OR = 3.65, 95% CI: 3.34-3.98; OR = 3.55). Although the LASSO regression model selected nearly 90 percent of all candidate predictors, all three analytic approaches had moderate discrimination (C-index ranging from 0.664 to 0.673). CONCLUSIONS: Although none of the models emerged as clearly superior, predictive analytics could proactively determine which patients are at risk of nonadherence, thus allowing for timely engagement in adherence-improving interventions.

Opportunities to Leverage Telehealth Approaches Along the Hypertension Control Cascade in Sub-Saharan Africa.

PURPOSE OF REVIEW: To review the current literature on use of telehealth at different stages of the hypertension control cascade in sub-Saharan Africa (SSA) and to discuss opportunities to harness technology infrastructure in SSA to improve population-level blood pressure control. RECENT FINDINGS: Despite the high burden of hypertension in SAA, strategies to improve awareness, diagnosis, and management are inadequate. In high-income countries, telehealth has increased patient access to high-quality care at reduced costs. Notwithstanding the limited evidence on the use of telehealth at the different stages of the hypertension control cascade in SSA, the few published interventions in this review reported reduction of blood pressure and increase in the proportion of individuals with controlled blood pressure. Telehealth use across the hypertension control cascade in SSA is promising. These under-resourced settings provide opportunity to better understand the demand for these interventions in order to achieve meaningful clinical outcomes.

Summary of Veterans Health Administration Cancer Data Sources.

OBJECTIVES: The Veterans Health Administration (VHA) is a leader in generating transformational research across the cancer care continuum. Given the extensive body of cancer-related literature utilizing VHA data, our objectives are to: (1) describe the VHA data sources available for conducting cancer-related research, and (2) discuss examples of published cancer research using each data source. METHODS: We identified commonly used data sources within the VHA and reviewed previously published cancer-related research that utilized these data sources. In addition, we reviewed VHA clinical and health services research web pages and consulted with a multidisciplinary group of cancer researchers that included hematologist/oncologists, health services researchers, and epidemiologists. RESULTS: Commonly used VHA cancer data sources include the Veterans Affairs (VA) Cancer Registry System, the VA Central Cancer Registry (VACCR), the Corporate Data Warehouse (CDW)-Oncology Raw Domain (subset of data within the CDW), and the VA Cancer Care Cube (Cube). While no reference standard exists for cancer case ascertainment, the VACCR provides a systematic approach to ensure the complete capture of clinical history, cancer diagnosis, and treatment. Like many population-based cancer registries, a significant time lag exists due to constrained resources, which may make it best suited for historical epidemiologic studies. The CDW-Oncology Raw Domain and the Cube contain national information on incident cancers which may be useful for case ascertainment and prospective recruitment; however, additional resources may be needed for data cleaning. CONCLUSIONS: The VHA has a wealth of data sources available for cancer-related research. It is imperative that researchers recognize the advantages and disadvantages of each data source to ensure their research questions are addressed appropriately.

The Role of the FDA and Regulatory Approval of New Devices for Diabetes Care.

PURPOSE OF REVIEW: The Food and Drug Administration (FDA) is responsible for assuring the safety, effectiveness, and quality of medical devices in the USA. Extensive review times coupled with the demand for necessary treatments have prompted the policymakers to implement measures to speed medical devices to market.The purpose of this review is to summarize the evolution of the regulatory pathways through which medical devices utilized in diabetes care gain market access. RECENT FINDINGS: Regulatory pathways, ranging from premarket notification to premarket approval, require distinct, yet necessary ("least burdensome") evidence demonstrating a device's safety and effectiveness. Collaboration between manufacturers, regulators, and patients has resulted in the development and approval of novel diabetes care devices, including the first hybrid closed-loop artificial pancreas. Policy provisions, ranging from the least burdensome approach to the "breakthrough device" expedited pathway, aim to balance innovation, access, and safety. Clinicians must be aware of the evolving regulatory landscape and play an active role in enhancing patient safety.