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Jason Shafrin,1 Katalin Bognar,1 Katie Everson,1 Michelle Brauer,2 Darius N Lakdawalla,3 Felicia M Forma4 1Policy and Economics, Precision Health Economics, Los Angeles, CA, USA; 2Policy and Economics, Precision Health Economics, Boston, MA, USA; 3School of Pharmacy, Sol Price School of Public Policy, Leonard D. Schaeffer Center for Health Policy and Economics, University of Southern California, Los Angeles, CA, USA; 4Health Economics and Outcomes Management, Otsuka Pharmaceutical Development & Commercialization, Inc., Princeton, NJ, USA Background: New digital technologies offer providers the promise of more accurately tracking patients’ medication adherence. It is unclear, however, whether access to such information will affect provider treatment decisions in the real world.Methods: Using prescriber-reported information on patient non-compliance from health insurance claims data between 2008 and 2014, we examined whether prescribers’ knowledge of non-compliance was associated with different prescribing patterns for patients with serious mental illness (SMI). We examined patients who initiated an oral atypical antipsychotic, but were later objectively non-adherent to this treatment, defined as proportion of days covered (PDC)

Prior literature has documented improvements in cancer survival over time. However, ambiguity remains over the relative contributions of improved treatment and earlier detection to survival gains. Using registry data, we developed a novel framework to estimate the relative contributions of advances in treatment and detection. Our approach compares changes in the probability of early detection, which we interpret as the effects of advances in detection, to improvements in stageconditional survival, which we interpret as the effects of treatment. We applied this methodology using SEER data to estimate probabilities of early detection and stage-conditional survival curves for several cancers, by race, between 1988 and 2000. Survival increased for all of the cancers we examined, with blacks experiencing larger survival gains than whites for all cancers combined. Our baseline analysis found that treatment advances account for the vast majority of survival gains for all the cancers examined: breast cancer (83%), lung cancer (85%), colorectal cancer (76%), pancreatic cancer (100%), and non-Hodgkin's lymphoma (96%). Compared to whites, treatments appear to explain a lower percentage of survival gains for blacks for all cancers combined; breast cancer, NHL, and pancreatic cancer show a higher percentage of survival gains than lung cancer; and roughly the same percentage for the colorectal cancer. These results are robust to sensitivity analyses examining potential length and lead time bias. Overall, our results suggest that while improved treatment and early detection both contributed to the recent gains in survival, the majority of gains from 1988 to 2000 appear to have been driven by better treatment, manifested by improved stage-conditional survival. These results have important policy implications regarding investment in research and development and the evaluation of efforts to improve cancer screening. [ABSTRACT FROM AUTHOR]

Jason Shafrin,1 Suepattra G May,1 Anshu Shrestha,1 Charles Ruetsch,2 Nicole Gerlanc,2 Felicia Forma,3 Ainslie Hatch,4 Darius N Lakdawalla,1,5 Jean-Pierre Lindenmayer6 1Precision Health Economics, Los Angeles, CA, 2Health Analytics, Columbia, MD, 3Otsuka Pharmaceutical Development & Commercialization, Inc., 4ODH, Inc., Princeton, NJ, 5Schaeffer Center for Health Policy and Economics, University of Southern California, Los Angeles, CA, 6Department of Psychiatry, New York University School of Medicine, New York, NY, USA Objective: Overestimating patients’ medication adherence diminishes the ability of psychiatric care providers to prescribe the most effective treatment and to identify the root causes of treatment resistance in schizophrenia. This study was conducted to determine how credible patient drug adherence information (PDAI) might change prescribers’ treatment decisions.Methods: In an online survey containing 8 clinical case vignettes describing patients with schizophrenia, health care practitioners who prescribe antipsychotics to patients with schizophrenia were instructed to choose a preferred treatment recommendation from a set of predefined pharmacologic and non-pharmacologic options. The prescribers were randomly assigned to an experimental or a control group, with only the experimental group receiving PDAI. The primary outcome was the prescribers’ treatment choice for each case. Between-group differences were analyzed using multinomial logistic regression.Results: A convenience sample (n=219) of prescribers completed the survey. For 3 nonadherent patient vignettes, respondents in the experimental group were more likely to choose a long-acting injectable antipsychotic compared with those in the control group (77.7% experimental vs 25.8% control; P

Abstract Background Comprehensive, multi-level approaches are required to address obesity. One important target for intervention is the economic domain. The purpose of this study was to synthesize existing evidence regarding the impact of economic policies targeting obesity and its causal behaviours (diet, physical activity), and to make specific recommendations for the Canadian context. Methods Arksey and O'Malley's (2005) methodological framework for conducting scoping reviews was adopted for this study and this consisted of two phases: 1) a structured literature search and review, and 2) consultation with experts in the research field through a Delphi survey and an in-person expert panel meeting in April 2010. Results Two key findings from the scoping review included 1) consistent evidence that weight outcomes are responsive to food and beverage prices. The debate on the use of food taxes and subsidies to address obesity should now shift to how best to address practical issues in designing such policies; and 2) very few studies have examined the impact of economic instruments to promote physical activity and clear policy recommendations cannot be made at this time. Delphi survey findings emphasised the relatively modest impact any specific economic instrument would have on obesity independently. Based on empirical evidence and expert opinion, three recommendations were supported. First, to create and implement an effective health filter to review new and current agricultural polices to reduce the possibility that such policies have a deleterious impact on population rates of obesity. Second, to implement a caloric sweetened beverage tax. Third, to examine how to implement fruit and vegetable subsidies targeted at children and low income households. Conclusions In terms of economic interventions, shifting from empirical evidence to policy recommendation remains challenging. Overall, the evidence is not sufficiently strong to provide clear policy direction. Additionally, the nature of the experiments needed to provide definitive evidence supporting certain policy directions is likely to be complex and potentially unfeasible. However, these are not reasons to take no action. It is likely that policies need to be implemented in the face of an incomplete evidence base.

The Centers for Medicare and Medicaid Services' coverage with evidence development (CED) policy allows the agency to provide coverage for an item or service through a National Coverage Determination (NCD), conditional upon an agreement to collect evidence designed to address specific questions or uncertainties. The goals of this policy are to expedite beneficiary access to new items and services and to generate additional evidence on the impact of these items or services for Medicare beneficiaries. However, these goals have not been fully realized because of several issues with the way the policy has been implemented, including (1) a lack of clear criteria for when CED will be applied, (2) examples of CED data collection activities placing unnecessary burdens on clinicians and the potential for undue inducement on beneficiaries, and (3) a lack of clarity around the process and timeline for reconsidering and ending CED requirements. Additionally, there are cases in which the application of CED has failed to improve access to services for certain Medicare beneficiaries because no data collection activity was implemented in response to the CED requirement or because the NCD only allows the technology to be provided and studied in certain centers of excellence. We describe a roadmap for addressing these issues, which includes, for example, developing a framework to guide the application of coverage constraints in NCDs with CED requirements. Once these issues are addressed, the Centers for Medicare and Medicaid Services could consider expanding the use of CED to technologies that are not subject to NCDs., Competing Interests: Author Disclosures Author disclosure forms can be accessed below in the Supplemental Material section., (Copyright © 2024. Published by Elsevier Inc.)

This paper examines how direct-to-consumer advertising (DTCA) for prescription drugs influences utilization by exploiting a large and plausibly exogenous shock to DTCA driven by the introduction of Medicare Part D. Part D led to larger increases in advertising in geographic areas with higher concentrations of Medicare beneficiaries. We examine the impact of this differential increase in advertising on non-elderly individuals to isolate advertising effects from the direct effects of Part D. We find that exposure to advertising led to large increases in treatment initiation and improved medication adherence. Advertising also had sizeable positive spillover effects on non-advertised generic drugs. Our results imply significant spillovers from Medicare Part D on the under-65 population and an important role for non-price factors in influencing prescription drug utilization.

Clinical trials for Alzheimer's disease (AD) are slower to enroll study participants, take longer to complete, and are more expensive than trials in most other therapeutic areas. The recruitment and retention of a large number of qualified, diverse volunteers to participate in clinical research studies remain among the key barriers to the successful completion of AD clinical trials. An advisory panel of experts from academia, patient-advocacy organizations, philanthropy, non-profit, government, and industry convened in 2020 to assess the critical challenges facing recruitment in Alzheimer's clinical trials and develop a set of recommendations to overcome them. This paper briefly reviews existing challenges in AD clinical research and discusses the feasibility and implications of the panel's recommendations for actionable and inclusive solutions to accelerate the development of novel therapies for AD., (© 2022 The Authors. Alzheimer's & Dementia published by Wiley Periodicals LLC on behalf of Alzheimer's Association.)

Background: Fully assessing the mortality burden of the COVID-19 pandemic requires measuring years of life lost (YLLs) and accounting for quality-of-life differences., Objective: To measure YLLs and quality-adjusted life-years (QALYs) lost from the COVID-19 pandemic, by age, sex, race/ethnicity, and comorbidity., Design: State-transition microsimulation model., Data Sources: Health and Retirement Study, Panel Study of Income Dynamics, data on excess deaths from the Centers for Disease Control and Prevention, and nursing home death counts from the Centers for Medicare & Medicaid Services., Target Population: U.S. population aged 25 years and older., Time Horizon: Lifetime., Perspective: Individual., Intervention: COVID-19 pandemic through 13 March 2021., Outcome Measures: YLLs and QALYs lost per 10 000 persons in the population. The estimates account for the age, sex, and race/ethnicity of decedents, along with obesity, smoking behavior, lung disease, heart disease, diabetes, cancer, stroke, hypertension, dementia, and nursing home residence., Results of Base-Case Analysis: The COVID-19 pandemic resulted in 6.62 million QALYs lost (9.08 million YLLs) through 13 March 2021, with 3.6 million (54%) lost by those aged 25 to 64 years. The greatest toll was on Black and Hispanic communities, especially among men aged 65 years or older, who lost 1138 and 1371 QALYs, respectively, per 10 000 persons. Absent the pandemic, 38% of decedents would have had average or above-average life expectancies for their subgroup defined by age, sex, and race/ethnicity., Results of Sensitivity Analysis: Accounting for uncertainty in risk factors for death from COVID-19 yielded similar results., Limitation: Estimates may vary depending on assumptions about mortality and quality-of-life projections., Conclusion: Beyond excess deaths alone, the COVID-19 pandemic imposed a greater life expectancy burden on persons aged 25 to 64 years, including those with average or above-average life expectancies, and a disproportionate burden on Black and Hispanic communities., Primary Funding Source: National Institute on Aging.

Objectives: Policy makers have suggested increasing peritoneal dialysis (PD) would improve end-stage kidney disease (ESKD) outcomes and reduce Medicare spending compared with hemodialysis (HD). We compared mortality, hospitalizations, and Medicare spending between PD and HD among uninsured adults with incident ESKD., Methods: Using an instrumental variable design, we exploited a natural experiment encouraging PD among the uninsured. Uninsured patients usually receive Medicare at dialysis month 4. For those initiating PD, Medicare covers the first 3 dialysis months, including predialysis services in the calendar month when dialysis started. Starting dialysis later in a calendar month increases predialysis coverage that is essential for PD catheter placements. The policy encourages PD incrementally when ESKD develops later in the month. Dialysis start day appears to be unrelated to patient characteristics and effectively "randomizes patients" to dialysis modality, mitigating selection bias., Results: Starting dialysis later in the month was associated with an increased PD uptake: every week later in the month was associated with an absolute increase of 0.8% (95% confidence interval [CI] 0.6%-0.9%) at dialysis day 1 and 0.5% (95% CI 0.3%-0.7%) at dialysis month 12. We observed no significant absolute difference between PD and HD for 12-month mortality (-0.9%, 95% CI -3.3% to 0.8%), hospitalizations during months 7 to 12 (-0.05, 95% CI -0.20 to 0.07), and Medicare spending during months 7 to 12 (-$702, 95% CI -$4004 to $2909)., Conclusions: In an instrumental variable analysis, PD did not result in improved outcomes or lower costs than HD., (Copyright © 2021 ISPOR–The Professional Society for Health Economics and Outcomes Research. Published by Elsevier Inc. All rights reserved.)

Importance: Prior research has documented the increase in prescription drug rebates and the coincident increase in out-of-pocket burden for patients paying coinsurance tied to list prices., Objective: To describe the out-of-pocket burden on patients with coinsurance and assess its association with pharmaceutical competition, which increases payers' leverage to seek higher rebates., Design, Setting, and Participants: This retrospective cohort study used branded prescription drugs with US sales reported by publicly traded companies. The study included drugs with nonmissing, nonnegative rebates between 2014 and 2018 from SSR Health. Data analysis was conducted from June to December 2020., Exposures: Level of branded and generic competition and calendar year., Main Outcomes and Measures: Retail price markup (ie, the ratio of rebate to net price) paid by patients at the point of sale and effective out-of-pocket share (ie, coinsurance multiplied by list price divided by net price) of a standard Part D plan. Trends in these outcomes were examined and then stratified by degree of competition., Results: There were 3322 unique National Drug Codes in the analysis, representing 232 distinct molecules from 138 therapeutic classes in 34 disease areas. The ratio of rebate to net prices was higher and increased faster for drugs with branded and generic competitors (from 83% to 172%) than for drugs with only branded competitors (from 61% to 115%) and those without generic equivalents (from 33% to 49%). Hypothetical patients paying standard Part D coinsurance on drug list prices would have experienced an effective out-of-pocket share increase from 48% to 64% in the initial coverage phase, and from 10% to 13% in the catastrophic coverage phase between 2014 and 2018. In the coverage gap, the share increased from 92% in 2014 to 98% in 2016 and then decreased to 90% in 2018. Compared with drugs with no competition, effective out-of-pocket share paid by patients grew 50% faster for drugs with branded competitors and 100% faster for those with branded and generic competitors., Conclusions and Relevance: This study found substantial increases in cost-sharing burden for patients paying coinsurance on drug list prices between 2014 and 2018, especially in markets with more pharmaceutical competition. Payers passing rebates through to patients at the point of sale could restore the benefits of competition and rebates.

Background: Evaluation of patients with serious mental illness (SMI) relies largely on patient or caregiver self-reported symptoms. New digital technologies are being developed to better quantify the longitudinal symptomology of patients with SMI and facilitate disease management. However, as these new technologies become more widely available, psychiatrists may be uncertain about how to integrate them into daily practice. To better understand how digital tools might be integrated into the treatment of patients with SMI, this study examines a case study of a successful technology adoption by physicians: endocrinologists' adoption of digital glucometers., Objective: This study aims to understand the key facilitators of and barriers to clinician and patient adoption of digital glucose monitoring technologies to identify lessons that may be applicable across other chronic diseases, including SMIs., Methods: We conducted focus groups with practicing endocrinologists from 2 large metropolitan areas using a semistructured discussion guide designed to elicit perspectives of and experiences with technology adoption. The thematic analysis identified barriers to and facilitators of integrating digital glucometers into clinical practice. Participants also provided recommendations for integrating digital health technologies into clinical practice more broadly., Results: A total of 10 endocrinologists were enrolled: 60% (6/10) male; a mean of 18.4 years in practice (SD 5.6); and 80% (8/10) working in a group practice setting. Participants stated that digital glucometers represented a significant change in the treatment paradigm for diabetes care and facilitated more effective care delivery and patient engagement. Barriers to the adoption of digital glucometers included lack of coverage, provider reimbursement, and data management support, as well as patient heterogeneity. Participant recommendations to increase the use of digital health technologies included expanding reimbursement for clinician time, streamlining data management processes, and customizing the technologies to patient needs., Conclusions: Digital glucose monitoring technologies have facilitated more effective, individualized care delivery and have improved patient engagement and health outcomes. However, key challenges faced by the endocrinologists included lack of reimbursement for clinician time and nonstandardized data management across devices. Key recommendations that may be relevant for other diseases include improved data analytics to quickly and accurately synthesize data for patient care management, streamlined software, and standardized metrics., (©Suepattra G May, Caroline Huber, Meaghan Roach, Jason Shafrin, Wade Aubry, Darius Lakdawalla, John M Kane, Felicia Forma. Originally published in the Journal of Medical Internet Research (http://www.jmir.org), 03.02.2021.)

Background: Uninsured patients with end-stage renal disease face barriers to peritoneal dialysis (PD), a type of home dialysis that is associated with improved quality of life and reduced Medicare costs. Although uninsured patients using PD at dialysis start receive retroactive Medicare coverage for required predialysis services, coverage only applies for the calendar month of dialysis start. Thus, initiating dialysis later in the month yields longer retroactive coverage., Objectives: To examine whether differences in retroactive Medicare were associated with decreased long-term PD use., Research Design: We exploited the dialysis start date using a regression discontinuity design on a national cohort from the US Renal Data System., Subjects: 36,256 uninsured adults starting dialysis between January 1, 2006 and December 31, 2014., Measures: PD use at dialysis days 1, 90, 180, and 360., Results: Starting dialysis on the first versus last day of the calendar month was associated with an absolute decrease in PD use of 2.7% [95% confidence interval (CI), 1.5%-3.9%], or a relative decrease of 20% (95% CI, 12%-27%) at dialysis day 360. The absolute decrease was 5.5% (95% CI, 3.5%-7.2%) after Medicare established provider incentives for PD in 2011 and 7.2% (95% CI, 2.5%-11.9%) after Medicaid expansion in 2014. Patients were unlikely to switch from hemodialysis to PD after the first month of dialysis (probability of 6.9% in month 1, 1.5% in month 2, and 0.9% in month 4)., Conclusions: Extending retroactive coverage for preparatory dialysis services could increase PD use and reduce overall Medicare spending in the uninsured.

Background. Value assessments and treatment decision making typically focus on clinical endpoints, especially overall survival (OS). However, OS data are not always available, and surrogate markers may also have some value to patients. This study sought to estimate preferences for progression-free survival (PFS) relative to OS in metastatic breast cancer (mBC) among a diverse set of stakeholders-patients, oncologists, and oncology nurses-and estimate the value patients and providers place on other attributes of treatment. Methods. Utilizing a combined conjoint analysis and discrete choice experiment approach, we conducted an online prospective survey of mBC patients and oncology care providers who treat mBC patients across the United States. Results. A total of 299 mBC patients, 100 oncologists, and 99 oncology nurses completed the survey. Virtually all patients preferred health state sequences with contiguous periods of PFS, compared with approximately 85% and 75% of nurses and oncologists, respectively. On average, longer OS was significantly ( P < 0.01) preferred by the majority (75%) patients, but only 15% of nurses preferred longer OS, and OS did not significantly affect oncologists' preferred health state. However, in the context of a treatment decision, whether a treatment offered continuous periods of stable disease holding OS constant significantly affected nurses' treatment choices. Patients and providers alike valued reductions in adverse event risk and evidence from high-quality randomized controlled clinical trials. Conclusions. The strong preference for observed PFS suggests more research is warranted to better understand the reasons for PFS having positive value to patients. The results also suggest a range of endpoints in clinical trials may have importance to patients.

Objectives: To estimate the impact of cures for chronic hepatitis C (CHC) infection on organ donation in the United Kingdom. Curing CHC infection reduces the need for liver transplants and enables cured individuals to donate organs of all types., Methods: We adapted a double-queuing model of organ allocation to estimate the effects of CHC infection cures on liver, lung, heart, and kidney transplants in the United Kingdom. We assumed that cured individuals would donate organs at similar rates as the general population and no longer require liver transplants because of CHC infection. We estimated how curing CHC infection influences waitlist lengths for each organ and the annual net present value to society on the basis of quality-adjusted life-years gained through additional transplants under opt-in and opt-out organ donation policies., Results: Curing CHC generates the most value for patients on the liver waitlist, because it increases the number of transplantable livers and reduces the need for transplants. Under the current opt-in policy, liver waitlist length falls by 24%, generating £34.3 million of annual net present value. Growth in the number of uninfected lungs, hearts, and kidneys generates an additional £19.2 million annually, with £18.7 million from kidneys. Implementing the opt-out policy, liver waitlist length would decrease by 75%, implying that treating CHC eliminates one-third of the excess liver waitlist due to an opt-in policy., Conclusions: Treating CHC has large positive spillovers to uninfected individuals by reducing the need for liver transplants and allowing cured individuals to donate organs. These spillovers have not been included in traditional value assessments of CHC treatment., (Copyright © 2019 ISPOR–The Professional Society for Health Economics and Outcomes Research. Published by Elsevier Inc. All rights reserved.)

Objectives: The causes of oncology drug price growth remain unclear. Analyzing corresponding trends in revenue can help understand these causes. This study seeks to assess changes over time in prices, patient counts, and drug-level revenues in the US market for oncology therapies and to investigate whether price growth is driven by an increased ability by pharmaceutical firms to capture profits., Study Design: Nineteen-year retrospective study (1997-2015)., Methods: We used panel regression to investigate trends in prices, patient counts, and revenues within a US national data set consisting of targeted oncology therapies launched in different eras., Results: We find that prices have roughly tripled, whereas average patient counts per therapy have fallen by 85% to 90% over this period. However, the entire distribution of annual revenues has fallen: For instance, median revenues for drugs launched in the early 2010s are about half of what they were for drugs launched in the late 1990s., Conclusions: Future research on the causes of quantity decline can help inform pharmaceutical policy.

Objectives: This study seeks to identify service categories that present the greatest opportunities to reduce spending in oncology care episodes, as defined by the CMS Oncology Care Model (OCM). Regional variation in spending for similar patients is often interpreted as evidence that resources can be saved, because higher-spending regions could achieve savings by behaving more like their lower-spending counterparts., Study Design: We used Surveillance, Epidemiology, and End Results Medicare data from 2006-2013 for this retrospective observational cohort study. Analysis focused on patients with non-small cell lung cancer, advanced (stage III or IV) breast cancer, renal cell carcinoma, multiple myeloma, or chronic myeloid leukemia., Methods: Episodes were identified for patients with the 5 included cancers, following the episode definition used in the OCM. We estimated standardized episode-level spending for a standard patient across subcategories of care for each hospital referral region (HRR) defined by the Dartmouth Atlas. The contribution of each subcategory to interregional variation in total spending reflects that subcategory's potential to yield savings., Results: Chemotherapy and acute inpatient hospital care tended to be the highest contributors to interregional variation. Imaging, nonchemotherapy Part B drugs, physician evaluation and management services, and diagnostics were negligible contributors to interregional variation for all 5 cancers., Conclusions: Chemotherapy and inpatient hospital care offer the most potential to reduce spending within OCM-defined episodes. Other sources of savings differ by type of cancer. Assuming patient outcomes are not compromised, low-spending HRRs may be models for lowering cost in cancer care.

Background: Previous research finds significant variation in spending and utilization across regions, with little evidence of differences in outcomes. While such findings have been interpreted as evidence that spending can be reduced without compromising patient outcomes, the link between spending variation and outcomes remains a critical question., Objective: To use evidence from geographic variations in spending and an individual-level survival analysis to test whether spending within oncology care episodes is associated with survival, where episodes are defined as in the Center for Medicare and Medicaid Innovation's Oncology Care Model (OCM)., Methods: In this retrospective cohort analysis, patient data from the Surveillance, Epidemiology and End Results Medicare (SEER-Medicare) database for 2007-2013 were linked to hospital referral regions (HRRs) using ZIP codes. Patients in the SEER program are a part of selected population-based cancer registries throughout the United States whose records are linked to Medicare enrollment and claims data (93% of elderly registry patients were successfully linked to Medicare data). Episodes of cancer care were defined as in the OCM: 6 months following a triggering chemotherapy claim. We analyzed episodes of care for 5 tumor types: advanced breast cancer (BC), non-small cell lung cancer (NSCLC), renal cell carcinoma (RCC), multiple myeloma (MM), and chronic myeloid leukemia (CML). We removed the effects of differentials in Medicare payment rates, which were mostly geographic. Regression analysis was then used to calculate standardized spending levels for each HRR, that is, spending adjusted for differences in patient and episode characteristics. To examine the effect of spending during OCM-defined episodes on individual-level survival, we used Cox regression with patient characteristics and standardized HRR spending per episode as covariates. To address concerns that may arise from multiple comparisons across the 5 tumor types, we used the Benjamini-Hochberg procedure to control the false discovery rate., Results: Our analysis showed significant differences in standardized spending across HRRs. Compared with spending at the 20th percentile episode, spending at the 80th percentile ranged from 25% higher ($57,392 vs. $45,995 for MM) to 47% higher ($36,920 vs. $24,127 for RCC), indicating practice style variation across regions. The hazard of dying for patients with NSCLC and MM statistically significantly decreased by 7% (HR = 0.93, P = 0.006) and 13% (HR = 0.87, P = 0.019), respectively, for a $10,000 increase in standardized spending (in 2013 U.S. dollars). For the 3 other cancers, spending effects were not statistically significant. After using the Benjamini-Hochberg procedure with a 5% false discovery rate, the effects of increased spending on improved survival for NSCLC and MM remained statistically significant., Conclusions: The association we found between spending and survival suggests caution may be warranted for physicians, pharmacists, other health care professionals, and policymakers involved in efforts to reduce across-the-board spending within OCM-defined episodes for at least 2 of the 5 cancers studied., Disclosures: Funding for this research was provided by Novartis Pharmaceuticals to Precision Health Economics in support of research design, analysis, and technical writing services. The funder provided input on study design and comments on the draft report. Baumgardner, Shahabi, and Linthicum are employees of Precision Health Economics (PHE), a health care consultancy to the insurance and life science industries, including firms that market oncology therapies. Vine was an employee of PHE at the time of this research. Zacker is an employee of and shareholder in Novartis Pharmaceuticals. Lakdawalla is a consultant to PHE and holds equity in its parent company, Precision Medicine Group.

Objectives: To estimate the long-run average cost (LAC) for a typical drug, accounting for the effects of generic competition and medical cost offsets., Study Design: Descriptive analysis of retrospective cross-sectional survey data., Methods: We estimated the LAC for a drug as the average price per unit paid over the lifecycle of the drug, discounted across all time periods using Medical Expenditure Panel Survey data, and accounted for the effects of generic competition and medical cost offsets attributable to the use of pharmaceuticals., Results: The average market-weighted price fell rapidly after generic entry. As a result, the brand price in the year prior to generic market entry was 39% (95% confidence interval [CI], 37%-43%) higher than the LAC per 30-day supply or package. When accounting for medical cost offsets, the brand price in the year prior to generic market entry was 75% (95% CI, 69%-79%) greater than the LAC per 30-day supply or package. The brand price at launch was 11% more than the LAC, and 40% more than the LAC net after adjusting for medical cost offsets., Conclusions: Branded drug prices might overstate the true long-run cost of pharmaceuticals by 40% to 75%, accounting for generic price reductions and medical cost offsets. To ensure that all drugs providing long-run value end up entering the marketplace, market access and other policy decisions should consider the full range of long-term costs-and not just prices-at a particular point in time.

Introduction: Prophylaxis treatment is recommended for haemophilia patients, but associated real-world economic costs and potential cost-savings associated with improved disease management are not fully known. This study aimed to assess haemophilia A-related resource use and cost by treatment type (prophylaxis versus non-prophylaxis) and any associated cost-savings., Methods: Truven MarketScan Commercial claims data (2004-2012) were used to identify haemophilia A-related healthcare utilization, healthcare costs and patterns of prophylaxis and non-prophylaxis treatment among 6- to 64-year-old males. We estimated bleeding-related resource utilization and costs in three age groups (6-18, 19-44, 45-64) by treatment types and assessed the extent to which early initiation of prophylactic treatment can mitigate them. T-tests and ordinary least squares regressions were used to compare unadjusted and demographics-adjusted cost estimates., Results: Among children, overall haemophilia- and bleeding-related non-pharmacy costs were substantially lower for patients receiving prophylaxis (haemophilia-related: $15,864 vs. $53,408; P < 0.001; bleeding-related: $696 vs. $2013, respectively; P = 0.04). Among younger adults (19-44), haemophilia-related non-pharmacy costs were lower for patients receiving prophylaxis ($22,028 vs. $56,311, respectively; P = 0.001). Among children, these savings fully offset the incremental pharmacy cost due to prophylaxis. Among younger adults, the savings offset approximately 34% of the incremental pharmacy cost. No differences were found for older adults (45-64)., Conclusion: These results suggest that initiating prophylaxis earlier in life may reduce the healthcare costs of bleeding events and their long-term complications. Future studies should strive to collect more detailed information on disease severity and treatment protocols to improve estimates of disease burden., (© 2017 John Wiley & Sons Ltd.)

Objectives: To quantify how adherence mismeasurement affects the estimated impact of adherence on inpatient costs among patients with serious mental illness (SMI)., Study Design: Proportion of days covered (PDC) is a common claims-based measure of medication adherence. Because PDC does not measure medication ingestion, however, it may inaccurately measure adherence. We derived a formula to correct the bias that occurs in adherence-utilization studies resulting from errors in claims-based measures of adherence., Methods: We conducted a literature review to identify the correlation between gold-standard and claims-based adherence measures. We derived a bias-correction methodology to address claims-based medication adherence measurement error. We then applied this methodology to a case study of patients with SMI who initiated atypical antipsychotics in 2 large claims databases., Results: Our literature review identified 6 studies of interest. The 4 most relevant ones measured correlations between 0.38 and 0.91. Our preferred estimate implies that the effect of adherence on inpatient spending estimated from claims data would understate the true effect by a factor of 5.3, if there were no other sources of bias. Although our procedure corrects for measurement error, such error also may amplify or mitigate other potential biases. For instance, if adherent patients are healthier than nonadherent ones, measurement error makes the resulting bias worse. On the other hand, if adherent patients are sicker, measurement error mitigates the other bias., Conclusions: Measurement error due to claims-based adherence measures is worth addressing, alongside other more widely emphasized sources of bias in inference.

Objectives: To illustrate a more comprehensive view of value associated with medicines treating a highly severe illness and to apply these insights to estimate the costs and benefits of 3 treatments for multiple sclerosis (MS): Avonex, Tysabri, and Tecfidera., Study Design: Retrospective study spanning 2002 to 2013. We used economic theory to derive the value of therapy to patients with MS and to individuals who face the risk of contracting MS in the future, under the alternative assumptions that therapies were fully insured or paid for out of pocket., Methods: Models were parameterized through secondary data analysis and targeted literature review. Estimates of individual value were aggregated to the societal level using therapy-specific treatment prevalence rates. Aggregate consumer value was compared with manufacturer revenue., Results: In the baseline model, Avonex, Tysabri, and Tecfidera generated $46.2 billion of total value to consumers, almost one-third of which accrued to those without MS. The total value to consumers was double manufacturer revenue. Results were qualitatively robust to the use of alternate epidemiological and economic parameters. We found that value to the healthy is positively related to disease severity, and that value to both the sick and the healthy are larger when costs are shared via health insurance., Conclusions: Theory predicts that treatments for severe disease provide "peace of mind" value to the healthy. Avonex, Tysabri, and Tecfidera have generated significant social value, a large majority of which accrues to consumers. Future economic valuations of medical technology should consider both the potential value to the healthy and the effects of insurance.