A Research Framework to Improve Health Disparity Evidence Gaps in Value Assessments.

A value assessment is intended as a tool for evaluating healthcare treatments to gauge value and inform decisions. Economic value assessments typically incorporate a cost-effectiveness analysis, focusing on costs and health outcomes important to payers, missing important information to ensure existing markets optimize resource allocation. Despite frequent calls for more explicit consideration of health equity impacts in value assessments, health economists continue to develop models informed by traditional cost and quality-of-life data that do not capture differences experienced by health disparity populations. This conceptual paper proposes a research framework to enhance data collection and analysis to address these gaps and better quantify the value of a health innovation, and better assess how a new intervention impacts health disparities. The framework comprises three distinct phases that build on one another: (1) contextualization of lived experiences for disadvantaged communities; (2) individual-level quantification of health disparities for cost and quality-of-life measures; and (3) quantifying community-level impacts.

Exploring Pharmacy Student Experiences with Student Debt and Perspectives on Future Burnout and Loan Relief.

OBJECTIVE: Pharmacy students with substantial educational debt are at risk for excessive workloads, burnout, and clinical errors. During the COVID-19 pandemic, policies addressing economic hardships for all student debt borrowers included temporary suspension of monthly payments and 0% interest during the pause. This study aimed to understand student-level factors regarding student debt from the lived experiences of current pharmacy students and aimed to understand how current pharmacy students view temporary loan relief. METHODS: We used semi-structured interviews of pharmacy students across 4 years of progression in their pharmacy program to better understand student experiences with debt, different factors that may influence the impact of student debt on short-term and long-term outcomes for students, and student perspectives on debt relief policies and potential solutions. Our thematic analysis was grounded in existing evidence and a conceptual framework, while also allowing codes to emerge directly from the data. RESULTS: A total of 20 pharmacy students were interviewed with a median student debt of $77,000, with debt amounts ranging from $0 to $209,000. Students described what mediating factors influenced their experiences, the influence of student debt on clinician burnout, and other outcomes impacted by student debt. Six overarching themes emerged relevant to current students: student debt influences education and career decisions, debt is risky given the saturated pharmacy market, debt is an accepted burden, debt will inhibit starting a life, the COVID-19 loan relief is revealing, and early financial education is needed. CONCLUSION: Pharmacy students burdened with debt described a variety of different experiences and attitudes toward that debt and provided their perspectives on how student debt influences short-term education and career decisions. While students accept the trade-off of debt for their education as an inevitable burden, reported coping mechanisms and strategies shared suggest some solutions may be available to ameliorate this burden.

Pharmacy Benefit Managers: History, Business Practices, Economics, and Policy.

Importance: Pharmacy benefit managers (PBMs) play a major role in the provision of pharmacy services by acting as intermediaries between pharmacies, plan sponsors (insurance companies and employers), pharmaceutical manufacturers, and drug wholesalers. As their role and visibility have increased, PBMs have come under increased scrutiny from policymakers. However, no prior literature has systematically described the history, business practices, and policymaking of PBMs. Objective: To provide an overview of the PBM industry, including its history, the evolution of services provided by PBMs, an assessment of the current policy landscape, and analysis of how proposed policies could affect PBM practices and patient care. Evidence: This work reviews historical events; previous and current industry practices and publications; prior academic literature, existing statutes, regulations, and court cases; and recent legislative reforms and agency actions regarding PBMs. Findings: Pharmacy benefit managers evolved in parallel with the pharmaceutical manufacturing and health insurance industries. The evolution of the PBM industry has been characterized by horizontal and vertical integration and market concentration. The PBM provides 5 key functions: formulary design, utilization management, price negotiation, pharmacy network formation, and mail order pharmacy services. Criticism of the PBM industry centers around the lack of competition, pricing, agency problems, and lack of transparency. Legislation to address these concerns has been introduced at the state and federal levels, but the potential for these policies to address concerns about PBMs is unknown and may be eclipsed by private sector responses. Conclusions and Relevance: Pharmacy benefit managers are intermediaries in the pharmaceutical supply chain and perform multiple roles in the management and distribution of pharmaceuticals to patients. When regulating PBMs, it is important to adopt policies that address market failure problems by improving PBM competition as opposed to policies designed to serve the narrow financial interests of other market participants (eg, pharmacies, pharmaceutical manufacturers) without meeting the needs of consumers.

Pharmacy Benefit Manager Pricing and Spread Pricing for High-Utilization Generic Drugs.

This cross-sectional study uses Medicare Part D claims for high-utilization generic drugs to analyze gross profits accumulated by pharmacy benefit managers, pharmacies, wholesalers, and manufacturers in the pharmaceutical supply chain.

Ethical and legal considerations in social media research for health technology assessment: conclusions from a scoping review.

OBJECTIVES: The objective was to identify and describe the published guidance and current academic discourse of ethical issues and standards related to the use of Social Media Research for generating patient insights for the use by health technology assessment (HTA) or health policy decisions. METHODS: A scoping review of the literature was conducted in PubMed and Embase and identified 935 potential references published between January 2017 and June 2021. After title and abstract screening by three reviewers, 40 publications were included, the relevant information was extracted and data were collected in a mind map, which was then used to structure the output of the review. RESULTS: Social Media Research may reveal new insights of relevance to HTA or health policies into patient needs, patient experiences, or patient behaviors. However, the research approaches, methods, data use, interpretation, and communication may expose those who post the data in social media channels to risks and potential harms relating to privacy, anonymity/confidentiality, authenticity, context, and rapidly changing technologies. CONCLUSIONS: An actively engaged approach to ensuring ethical innocuousness is recommended that carefully follows best practices throughout planning, conduct, and communication of the research. Throughout the process and as a follow-up, there should be a discourse with the ethical experts to maximally protect the current and future users of social media, to support their trust in the research, and to advance the knowledge in parallel to the advancement of the media themselves, the technologies, and the research tools.

Using social media research in health technology assessment: stakeholder perspectives and scoping review.

OBJECTIVES: The aim of this initiative was to examine collaboratively, in a multi-stakeholder team (health technology assessment (HTA) practitioners with patient involvement expertise, health technology industry, patient advocates, health policy experts, patient engagement experts), whether evidence generated through social media research (SMR) fills current information gaps relating to insights on specific aspects of patient experiences, preferences, or patient needs and delivers additional value to HTA. METHODS: The framing of the project was done in a co-creative, deliberative multi-stakeholder process. Challenge and refinement happened through discussions with 25 independent stakeholders from HTA bodies, industry, academia, and patient advocacy. For critical themes identified during the framing phase, scoping literature reviews were performed including the state of methods and examples for the use of SMR in HTA. RESULTS: The framing and stakeholder discussions specified a set of expectations and requirements, and the scoping reviews revealed the current state of methods and usage of SMR in health-policy decision making. CONCLUSIONS: The project concluded that SMR can contribute new, relevant evidence to HTA. It is however recommended to evolve the science through defining best practices when planning, conducting, and using SMR and to conduct multi-stakeholder pilot SMR projects to address questions relevant to current HTAs and to validate and improve the proposed practices.

Incorporating Added Therapeutic Benefit and Domestic Reference Pricing Into Medicare Payment for Expensive Part B Drugs.

OBJECTIVES: Identify expensive Part B drugs and evidence for each drug's added benefit and model a reimbursement policy for Medicare that integrates added benefit assessment and domestic reference pricing. METHODS: A retrospective analysis using a 20% nationally representative sample of 2015 to 2019 traditional Medicare Part B claims. Expensive drugs were defined as having average annual spending per beneficiary exceeding the average annual social security benefit ($17 532 in 2019). For expensive drugs identified in 2019, added benefit assessments conducted by the French Haute Autorité de Santé were collected. For expensive drugs with a low added benefit rating, comparator drugs were identified in French Haute Autorité de Santé reports. For each comparator, average annual spending per beneficiary in Part B was computed. Potential savings from 2 reference pricing scenarios were calculated: reimbursing expensive Part B drugs with low added benefit at the level of each drug's (1) lowest cost comparator and (2) beneficiary-weighted-average cost of all comparators. RESULTS: The number of expensive Part B drugs grew from 56 in 2015 to 92 in 2019. Of the 92 expensive drugs in 2019, 34 offer low added benefit. Implementing reference pricing for these expensive drugs with low added benefit could have saved an estimated $2.1 billion if prices were set based on spending for their lowest cost comparator, or $1 billion if prices were set based on the weighted average of spending for comparators. CONCLUSION: Reference pricing based on added benefit assessment could be used to address the launch prices for expensive Part B drugs with low added benefit.

Comparison of Price Index Methods and Drug Price Inflation Estimates for Hepatitis C Virus Medications.

Importance: Measuring drug price inflation is challenging because new drugs continually enter the market, some drugs transition from branded to generic, and current inflation indexes do not account for these market basket changes. Instead, they measure the price increases after new drugs have been launched. Therefore, the public pays the higher costs of newer and usually more expensive drugs, but the inflation indexes do not reflect the increases over existing drugs previously used to treat the same conditions. Objective: To assess how price index methods can affect estimates of drug price inflation using a case study of hepatitis C virus (HCV) medication and to explore other approaches for constructing a price index. Design, Setting, and Participants: This cross-sectional study used data from outpatient pharmacies to compile a list of all HCV medications that were ever on the market (brand and generic) from 2013 to 2020. Using National Drug Codes of HCV drugs, a 20% nationally representative sample of Medicare Part D claims from 2013 to 2020 was queried. Alternative drug price indexes, including product-level vs class-level product and quantity definitions were developed in which gross vs net price definitions were used and an adjustment was created and applied to capture treatment duration because newer drugs often required a shorter duration. Main Outcomes and Measures: Price index value and rate of inflation from 2013 to 2020 for each methodologic approach to constructing a drug pricing index. Results: In all, 27 different HCV drug regimens were identified in Medicare Part D claims in 2013 to 2020. A product-level approach for measuring inflation estimated a 10% gross drug price increase from 2013 to 2020 for HCV drugs, whereas a class-level approach including the higher prices of the new drugs showed a 31% gross price increase. After adjusting for manufacturer rebates to estimate net prices, the findings showed that HCV drug prices fell by 31% from 2013 to 2020. Conclusions and Relevance: The findings of this cross-sectional study indicate that the current product-level methods to estimate drug price inflation underestimated price increases for HCV drugs by failing to include the high launch prices of new market entrants. Using a class-level approach, the index captured higher spending on new products at launch. Prescription-level analyses, which did not consider shorter durations of treatment, overestimated price increases.

Cost-Related Medication Nonadherence and Desire for Medication Cost Information Among Adults Aged 65 Years and Older in the US in 2022.

Importance: Rising prescription drug costs and increasing prices for consumer goods may increase cost-related medication nonadherence. Cost-conscious prescribing can be supported by real-time benefit tools, but patient views on real-time benefit tool use and their potential benefits and harms are largely unexplored. Objective: To assess older adults' cost-related medication nonadherence, cost-coping strategies, and views on the use of real-time benefit tools in clinical practice. Design, Setting, and Participants: A weighted, nationally representative survey of adults aged 65 years and older administered via the internet and telephone from June 2022 to September 2022. Main Outcomes and Measures: Cost-related medication nonadherence; cost coping strategies; desire for cost conversations; potential benefits and harms from real-time benefit tool use. Results: Among 2005 respondents, most were female (54.7%) and partnered (59.7%); 40.4% were 75 years or older. Cost-related medication nonadherence was reported by 20.2% of participants. Some respondents used extreme forms of cost-coping, including foregoing basic needs (8.5%) or going into debt (4.8%) to afford medications. Of respondents, 89.0% reported being comfortable or neutral about being screened before a physician's visit for wanting to have medication cost conversations and 89.5% indicated a desire for their physician to use a real-time benefit tool. Respondents expressed concern if prices were inaccurate, with 49.9% of those with cost-related nonadherence and 39.3% of those without reporting they would be extremely upset if their actual medication price was more than what their physician estimated with a real-time benefit tool. If the actual price was much more than the estimated real-time benefit tool price, nearly 80% of respondents with cost-related nonadherence reported that it would affect their decision to start or keep taking a medication. Furthermore, 54.2% of those with any cost-related nonadherence and 30% of those without reported they would be moderately or extremely upset if their physicians used a medication price tool but chose not to discuss prices with them. Conclusions and Relevance: In 2022, approximately 1 in 5 older adults reported cost-related nonadherence. Real-time benefit tools may support medication cost conversations and cost-conscious prescribing, and patients are enthusiastic about their use. However, if disclosed prices are inaccurate, there is potential for harm through loss of confidence in the physician and nonadherence to prescribed medications.

"Worth it if you could afford it": Patient perspectives on integrating real-time benefit tools into drug cost conversations.

BACKGROUND: Medication costs can lead to financial burdens for patients, creating barriers to effective medication use. Health care provider use of real-time benefit tools (RTBTs) may facilitate cost conversations with patients. We sought to explicate patient views on how RTBTs could be used to improve cost considerations in prescribing decisions. METHODS: We conducted focus groups to characterize patient perspectives on holding cost conversations with their physicians and to identify factors that would influence the value of RTBTs. We focused on adults aged 50+ who reported trouble paying for their prescriptions. Three groups included patients with conditions requiring high-cost treatments and one group included lower-income patients independent of their medical conditions. Focus groups were recorded, transcribed, coded, and categorized to salient themes employing inductive and deductive approaches using the Health Equity Implementation Framework. RESULTS: Focus groups were conducted from 09/2020-12/2020 including 18 participants representing cancer (n = 6), diabetes (n = 6), rheumatoid arthritis (n = 3), and lower income (n = 3). Participants were between 50-74, eight self-identified as Black, 10 as White, and eight reported earning <$50,000/year. We identified five themes regarding cost conversations (medication cost importance, past experiences with cost/cost conversations, perception of physician's role and knowledge, knowledge of existing resources, and influence on decision-making) and four RTBT-use-specific themes (advantages/disadvantages, perceived relevance, data quality concerns, and implementation considerations). CONCLUSION: Approaches that envision RTBTs as one-size-fits-all technological interventions may underestimate the complexity of incorporating price information into prescribing decisions. Nevertheless, patients highlighted the potential value of accurate, real-time information on medication costs to inform decision-making.

Developing Patient-Centered Real-World Evidence: Emerging Methods Recommendations From a Consensus Process.

OBJECTIVES: The Joint ISPOR-ISPE Special Task Force on Real-World Evidence included patient/stakeholder engagement as a recommended good procedural practice when designing, conducting, and disseminating real-world evidence (RWE). However, there are no guidelines describing how patient experience data (PED) can be applied when designing real-world data (RWD) studies. This article describes development of consensus recommendations to guide researchers in applying PED to develop patient-centered RWE. METHODS: A multidisciplinary advisory board, identified through recommendations of collaborators, was established to guide development of recommendations. Semistructured interviews were conducted to identify how experienced RWD researchers (n = 15) would apply PED when designing a hypothetical RWD study. Transcripts were analyzed and emerging themes developed into preliminary methods recommendations. An eDelphi survey (n = 26) was conducted to refine/develop consensus on the draft recommendations. RESULTS: We identified 13 recommendations for incorporating PED throughout the design, conduct, and translation of RWE. The recommendations encompass themes related to the development of a patient-centered research question, designing a study, disseminating RWE, and general considerations. For example, consider how patient input can inform population/subgroups, comparators, and study period. Researchers can leverage existing information describing PED and may be able to apply those insights to studies relying on traditional RWD sources and/or patient registries. CONCLUSIONS: Applying these emerging recommendations may improve the patient centricity of RWE through improved relevance of RWE to patient communities of interest and foster greater multidisciplinary participation and transparency in RWD research. As researchers gather experience by applying the methods recommendations, further refinement of these consensus recommendations may lead to "best practices."

Physician Perspectives on Implementation of Real-Time Benefit Tools: A Qualitative Study.

OBJECTIVES: Congress and Medicare have required real-time benefit tools (RTBT) to provide patient-specific medication price information during prescribing to decrease the cost of medications. We sought physicians' perspectives on how these tools might most effectively improve their selection of low-cost medication. METHODS: We conducted 15 semi-structured interviews of physicians (6 oncologists, 1 endocrinologist, 4 rheumatologists, and 4 from internal medicine) and identified key themes across interviews during coding and analysis. RESULTS: Although physicians saw value in real-time medication price information, they were wary of the complexity of obtaining specific information and the potential for inaccuracies. Physicians described how medication price information would be used in various prescribing scenarios including from simple substitutions (different drug formulations) to more complex decisions (different drug classes). In more complex situations, physicians were concerned that price information might only be available after discussing options with the patient, which would be too late to inform decisions. Concern about adding more information to the electronic health record was common. CONCLUSION: While most physicians saw value in implementation of RTBTs, they also expressed concerns related to the accuracy of information, the availability of information at the right time in the clinical workflow, and the most effective format for information. Many concerns raised paralleled the "Five Rights of Clinical Decision Support" framework and indicate the need for additional design work to achieve benefit from RTBTs. Beyond the public policy that has supported the availability of RTBTs, substantial development will be required to ensure that information is used to improve prescribing decisions.

State-level policy efforts to regulate pharmacy benefit managers (PBMs).

Pharmacy Benefit Managers (PBMs) originated in the 1960s to address a market need providing services to insurance companies in managing the newly introduced prescription drug benefits. Since then, PBMs have been expanding their roles in the prescription drug supply chain and have come under scrutiny. As of November 2021, all 50 states have enacted some form of regulation on PBMs. These state-level regulations focus on a variety of different policy levers, many of which align with advocacy efforts led by community pharmacists that focus on business transactions between pharmacies and PBMs without evidence supporting how these policies would enhance the patient experience, improve population health, lower costs, or improve the experience of health care providers. Many state policies could help increase transparency, prevent anticompetitive behavior between PBM-owned and independently-owned pharmacies, and increased accountability of PBMs to plan sponsors. However, there were no direct mechanisms to ensure that the potential benefits of these policies would ultimately be realized as savings for the health care system or consumers, improved health outcomes, or increased quality of care.

Drug costs in context: assessing drug costs in cost-of-illness analyses.

Cost-of-illness (COI) studies attempt to measure and describe the costs associated or attributed to a specific disease, but there are several considerations for measuring and interpreting drug costs estimates. The complexity of the pharmaceutical supply chain and contractual relationships between manufacturers, wholesalers, pharmacies and payers create challenges for researchers attempting to include drug costs in COI analyses. This article aims to provide contextual information for a general audience interested in conducting or evaluating COI studies that may include drug costs and to describe key factors to consider when reviewing drug costs in the peer-reviewed literature.

A review of caregiver costs included in cost-of-illness studies.

INTRODUCTION: Economic evaluations typically focus solely on patient-specific costs with economic spillovers to informal caregivers less frequently evaluated. This may systematically underestimate the burden resulting from disease. AREAS COVERED: Cost-of-illness (COI) analyses that identified costs borne to caregiver(s) were identified using PubMed and Embase. We extracted study characteristics, clinical condition, costs, and cost methods. To compare caregiver costs reported across studies, estimated a single 'annual caregiver cost' amount in 2021 USD. EXPERT OPINION: A total of 51 studies met our search criteria for inclusion with estimates ranging from $30 - $86,543. The majority (63%, 32/51) of studies estimated caregiver time costs with fewer studies reporting productivity or other types of costs. Caregiver costs were frequently reported descriptively (69%, 35/51), with fewer studies reporting more rigorous methods of estimating costs. Only 27% (14/51) of studies included used an incremental analysis approach for caregiver costs. In a subgroup analysis of dementia-focused studies (n = 16), we found the average annual cost of caregiving time for patients with dementia was $30,562, ranging from $4,914 to $86,543. We identified a wide range in annual caregiver cost estimates, even when limiting by condition and cost type.