Reimbursement to Pharmacies for Generic Drugs by Medicare Part D Sponsors.

This study evaluates whether organizations that offer Medicare Part D plans (referred to as Part D sponsors) overpay pharmacies for generic drugs.

Compulsory Licensing of Pharmaceuticals in High-Income Countries: A Comparative Analysis.

Policy Points Pharmaceutical trade organizations and media outlets in the United States regularly point to compulsory licensing-or even its threat-as the mechanism that peer countries use to control the price of prescription drugs. Our comparative analysis shows that compulsory licensing is not frequently employed in high-income countries outside the United States as a direct response to drug prices. When its use is threatened, a license is rarely issued and even less often does it lead to a price discount. Accordingly, compulsory licensing is unlikely to contribute to price discrepancies between the United States and other developed nations. In fact, of the 21 compulsory licensing petitions we identified outside the United States, over one-third were made by pharmaceutical companies themselves and only three were threatened by a government authority. CONTEXT: Compulsory licensing is a practice whereby national authorities can license a third party to produce a patented product, such as a pharmaceutical drug, effectively enabling the production of a generic before the original patent expires. The policy was designed-and has historically been used-to improve access to essential medicines in low-income countries and during public health crises. Although it was not intended to impact drug prices directly, the threat of compulsory licensing may indeed contribute to lower drug prices in high-income countries outside the United States. Our study sought to determine the plausibility of this claim. METHODS: We compiled a comprehensive database of compulsory licensing episodes in the United States and 17 comparator nations over the 20 years following the 2001 Doha Declaration, and we recorded the motivation and outcome of each instance. Our search began with publicly available reports compiled by organizations specializing in pharmaceutical intellectual property, expanded to a query of legal proceedings in Westlaw, and concluded with a comprehensive literature review on PubMed. FINDINGS: This strategy yielded 45 unique episodes of compulsory licensing, 24 in the United States and 21 outside. A minority (24%) of petitions outside the United States were motivated by high prices, and in all countries, only three cases were clearly associated with a price discount. CONCLUSIONS: We found no evidence to suggest that compulsory licensing is either frequently threatened or successfully implemented by countries outside the United States to secure price discounts for the most expensive pharmaceuticals, those that are newly patented and just entering the market.

Priceless Knowledge: Attitudes and Awareness Around Drug Pricing Among US Medical Students.

High US drug costs have garnered increasing attention, with multiple proposed reforms. While physicians are key stakeholders, medical education about drug pricing is not described, and medical students' understanding and attitudes are poorly understood. To assess students' awareness of drug pricing and its determinants, the authors conducted a cross-sectional, web-based survey of US medical students. Survey items included attitudes and knowledge around drug pricing and relevant education received (e.g., importance, quantity/quality of instruction). A composite knowledge score summed correct responses to 10 knowledge items. Descriptive statistics and t tests were used to evaluate associations. Among 815 viewers of the survey invitation, 361 visited the survey and 240 completed it (view rate 44%; participation rate 77%; completion rate 87%). Most participants were white (62%), in MD programs (82%), and female (53%). Nearly all (> 99%) said it was somewhat or very important to understand factors influencing drug pricing; over 90% were interested in learning more. Among year 3-4 students (n = 108), 59% reported receiving medical school instruction on pricing; few rated the quantity as adequate (7%) or the quality as excellent (3%) or good (8%). Among 10 knowledge questions, the median correct score was 6. Fewer than half (44%) knew that prices are uncorrelated with research/development costs. Knowledge was associated with year in school (p = 0.011) but not reported instructional quality or quantity. In sum, medical students report interest in drug pricing but inadequate instruction, and their knowledge is incomplete. Enhanced education is needed to equip future doctors to advocate effectively for patients around drug prices. SUPPLEMENTARY INFORMATION: The online version contains supplementary material available at 10.1007/s40670-020-01190-x.

Potential policy reforms to strengthen the accelerated approval pathway.

The accelerated approval pathway for new drugs in the United States is often praised but faces growing criticism of whether it is finding the appropriate balance between uncertainty, access and cost. To support efforts to strengthen the pathway, this paper provides an analysis of key concerns and the advantages and disadvantages of ten potential policy reforms - those achievable through the US FDA action alone, and those that would require a combination of government, payer and life science industry actions. Accelerated approval sits at the heart of many of the controversies regarding drug approvals and pricing, and this analysis provides perspectives on how best to strengthen the pathway within the broader landscape of an innovative US healthcare system.

Tweetable abstract New ICER policy analysis evaluates ten potential policy reforms to strengthen FDA's accelerated approval pathway for prescription drugs.

The future of physician advocacy: a survey of U.S. medical students.

BACKGROUND: Advocacy is a core component of medical professionalism. It is unclear how educators can best prepare trainees for this professional obligation. We sought to assess medical students' attitudes toward advocacy, including activities and issues of interest, and to determine congruence with professional obligations. METHODS: A cross-sectional, web-based survey probed U.S. medical students' attitudes around 7 medical issues (e.g. nutrition/obesity, addiction) and 11 determinants of health (e.g. housing, transportation). Descriptive statistics, Kruskal-Wallis tests, and regression analysis investigated associations with demographic characteristics. RESULTS: Of 240 students completing the survey, 53% were female; most were white (62%) or Asian (28%). Most agreed it is very important that physicians encourage medical organizations to advocate for public health (76%) and provide health-related expertise to the community (57%). More participants rated advocacy for medical issues as very important, compared to issues with indirect connections to health (p < 0.001). Generally, liberals and non-whites were likelier than others to value advocacy. CONCLUSIONS: Medical students reported strong interest in advocacy, particularly around health issues, consistent with professional standards. Many attitudes were associated with political affiliation and race. To optimize future physician advocacy, educators should provide opportunities for learning and engagement in issues of interest.

Value-based management of specialty drugs: practical considerations and implications for pharmacy.

OBJECTIVES: Concerns about high and rising drug prices have prompted a call to manage prescription drugs according to their value. Although not all proposals referred to as "value based" are well suited to advance this mission, health plans must select among them under the influence of competing demands and constraints of their market and nonmarket environments. To understand the implications for health policy, we sought to explore how health plans might select among and implement these approaches for specialty pharmacy (SP) under the incentives and barriers that these conditions create. STUDY DESIGN: An experienced research team conducted a qualitative study with Blue Cross Blue Shield health plans interested in implementing value-based SP management. METHODS: Plans'objectives, operational strategies, and factors influencing their ability to execute on these strategies were elicited in 3 focus groups. RESULTS: Four business objectives were identified, centering on spending levels, spending variability, access to new treatments, and evidence generation for new treatments. Supporting operational strategies included increased utilization management (UM), provider and patient engagement, expanded data analytics, and adjustments to staffing models. Factors that influence their ability to act on these strategies include regional and national scale, strength of provider network relationships, disease management capabilities, business and data silos, and potential legislative actions to limit UM. CONCLUSIONS: Health plans' preferences for different forms of SP management may not be aligned with policy objectives, particularly those that advance innovation. Policy makers should consider market and nonmarket factors that influence these preferences, including the need to mitigate spending variability and generate evidence to guide coverage decisions.

Pharmaceutical Products and Their Value: Lessons Learned and the Path Ahead.

Steep increases in prices and spending on prescription drugs in the United States have triggered public outrage and questions over their value. Value-based pricing has emerged as a preferred alternative to prices determined by what the market will bear. In response, manufacturers and health plans have begun to publicize their efforts to engage in outcomes-based contracts and long-term financing agreements, which they describe as value-based. Nevertheless, both contracting approaches perpetuate existing distortions in the financial incentives of supply chain and prescribing intermediaries, and fail to realign the prices of drugs to their value to patients, the healthcare system, or society. This commentary describes the challenges of managing drugs according to their value, and describes several alternatives that promise greater impact than contracting strategies.

Evolving Medicaid Coverage Policy and Rebates.

Medicaid covers approximately 1 in 5 Americans and accounts for one-sixth of US health care spending. Despite having to navigate increasing and variable spending on prescription drugs, Medicaid programs must balance their annual budgets, and they rely heavily on the Medicaid Drug Rebate Program (MDRP). The MDRP requires programs to maintain an open formulary covering all of a manufacturer's drugs in exchange for being given the lowest price in the market. Recent attempts by states to close their formularies signal that the benefit of this program might be attenuated by the lack of negotiating leverage in the rest of the market, exposing Medicaid to higher prices. Regardless of whether closed formularies would succeed in constraining Medicaid prescription drug spending, this trend raises important questions about the usefulness of a system that pegs Medicaid drug spending to net prices negotiated by others in the market.

Using Nominal Group Technique to Identify Key Attributes of Oncology Treatments for a Discrete Choice Experiment.

Background. Responding to rising oncology therapy costs, multiple value frameworks are emerging. However, input from economists in their design and conceptualization has been limited, and no existing framework has been developed using preference weightings as legitimate indicators of value. This article outlines use of the nominal group technique to identify valued treatment attributes (such as treatment inconvenience) and contextual considerations (such as current life expectancy) to inform the design of a discrete choice experiment to develop a preference weighted value framework for future decision makers. Methods. Three focus groups were conducted in 2017 with cancer patients, oncology physicians, and nurses. Using the nominal group technique, participants identified and prioritized cancer therapy treatment and delivery attributes as well as contextual issues considered when choosing treatment options. Results. Focus groups with patients (n = 8), physicians (n = 6), and nurses (n = 10) identified 30 treatment attributes and contextual considerations. Therapy health gains was the first priority across all groups. Treatment burden/inconvenience to patients and their families and quality of evidence were prioritized treatment attributes alongside preferences for resource use and cost (to patients and society) attributes. The groups also demonstrated that contextual considerations when choosing treatment varied across the stakeholders. Patients prioritized existence of alternative treatments and oncologist/center reputation while nurses focused on administration harms, communication, and treatment innovation. The physicians did not prioritize any contextual issues in their top rankings. Conclusions. The study demonstrates that beyond health gains, there are treatment attributes and contextual considerations that are highly prioritized across stakeholder groups. These represent important candidates for inclusion in a discrete choice experiment seeking to provide weighted preferences for a value framework for oncology treatment that goes beyond health outcomes.

An analysis of expenditures on primary care prescription drugs in the United States versus ten comparable countries.

OBJECTIVE: We sought to estimate size and sources of differences in per capita expenditures on primary care medications in the US versus ten comparable countries combined: Australia, Canada, France, Germany, the Netherlands, New Zealand, Norway, Sweden, Switzerland, and the United Kingdom. METHODS: Using market research data on year 2015 volumes and sales of medicines, we measure total per capita expenditures on six categories of primary care prescription drugs: hypertension treatments, pain medications, lipid lowing medicines, non-insulin diabetes treatments, gastrointestinal preparations, and antidepressants. We quantified the contributions of five drivers of the observed differences in per capita expenditures. RESULTS: We estimated that the US spent 203% more per capita on primary care pharmaceuticals than did the ten comparable countries. Despite the difference in spending levels, on average, Americans actually purchased 12% fewer days of related therapies than residents of the comparator countries. Most of the observed difference in expenditures was due to higher transaction prices of medicines and the use of a more expensive mix of medicines in the US. CONCLUSIONS: If utilization patterns and pharmaceutical prices in the US were similar to those in the 10 comparator countries combined, total spending on primary care pharmaceuticals would fall by 30% or more. Such evidence on the level and drivers of US pharmaceutical expenditures should inform policies in this sector.

Real-World Treatment Patterns among Patients with Advanced Gastric Cancer in South Korea.

PURPOSE: The purpose of this study was to understand patient treatment patterns, outcomes, and healthcare resource use in cases of metastatic and/or locally recurrent, unresectable gastric cancer (MGC) in South Korea. MATERIALS AND METHODS: Thirty physicians reviewed charts of eligible patients to collect de-identified data. Patients must have received platinum/fluoropyrimidine first-line therapy followed by second-line therapy or best supportive care, had no other primary cancer, and not participated in a clinical trial following MGC diagnosis. Data were summarized using descriptive statistics. Kaplan-Meier analysis was used to describe survival. RESULTS: Of 198 patients, 73.7% were male, 78.3% were diagnosed with MGC after age 55 (mean, 61.3 years), and 47.0% were current or former smokers. The majority of tumorswere located in the antrum/pylorus (51.5%). Metastatic sites most often occurred in the peritoneum (53.5%), lymph nodes (47.5%), and liver (38.9%). At diagnosis, the mean Charlson comorbidity indexwas 0.4 (standard deviation, 0.6). The most common comorbidities were chronic gastritis (22.7%) and cardiovascular disease (18.7%). Most patients (80.3%) received second-line treatment. Single-agent fluoropyrimidine was reported for 22.0% of patients, while 19.5% were treated with irinotecan and a fluoropyrimidine or platinum agent. The most common physician-reported symptoms during second-line treatment were nausea/vomiting (44.7%) and pain (11.3%), with antiemetics (44.7%), analgesics (36.5%), and nutritional support (11.3%) most often used as supportive care. Two-thirds of inpatient hospitalizations were for chemotherapy infusion. Outpatient hospitalization (31.6%) and visits to the oncologist (58.8%) were common among second-line patients. CONCLUSION: Most patients received second-line treatment, although regimens varied. Understanding MGC patient characteristics and treatment patterns in South Korea will help address unmet needs.

Treatment patterns in patients with advanced gastric cancer in Taiwan.

AIM: To describe treatment patterns, outcomes and healthcare resource use in patients with metastatic and/or locally recurrent, unresectable gastric cancer (MGC) in Taiwan. METHODS: Patients who had received first-line therapy (platinum and/or fluoropyrimidine) followed by second-line therapy or best supportive care (BSC) only were eligible. Participating physicians provided de-identified information from patient charts. Data were summarized descriptively and Kaplan-Meier analysis was used to describe time to events. RESULTS: Overall, 37 physicians contributed 122 patient charts. Of the 122 patients (median age, 61 years; 62% male), 43 (35%) received BSC only following first-line therapy, whereas 79 (65%) received second-line therapy. There was heterogeneity in second-line treatment, although fluoropyrimidine with or without a platinum agent was most frequently used. Median survival was 12.5 (interquartile range [IQR], 8.2-20.8) months from MGC diagnosis for patients receiving second-line therapy and 8.0 (IQR, 5.6-not reached) months for patients receiving BSC only. The most common treatment-related symptoms were nausea/vomiting (58%); the most common cancer-related symptoms were pain (61%), ascites (35%) and nausea/vomiting (33%). Inpatient and outpatient hospitalizations were numerically more common for patients receiving second-line therapy than for those receiving BSC only; the prevalence of hospice and skilled nursing facility stays were numerically more common for patients receiving BSC only. CONCLUSIONS: In this Taiwanese MGC population, 65% received active second-line therapy with heterogeneity seen in the regimen used. Clinical outcomes suggest an unmet medical need in this population. This study may help inform clinical practice and future research to ultimately improve patient outcomes in Taiwan.

Care pathways in US healthcare settings: current successes and limitations, and future challenges.

OBJECTIVES: Care pathways are used widely in US healthcare settings and are expected to have greater influence on quality of care and patient outcomes in the future. We conducted qualitative research to evaluate the state of care pathways and their impact in the United States. STUDY DESIGN: Targeted literature review followed by an online survey and in-person interviews. METHODS: The PubMed and Cochrane databases were searched for publications on care pathways (January 1, 2005, to July 21, 2014) to inform the subsequent surveys and interviews with payers, providers, and pathway vendors regarding care pathway design, development, and management. A targeted hand search was completed in May 2015 to supplement the earlier review. RESULTS: Twenty-nine publications, posters, or abstracts on specific care pathways were identified; the online survey and follow-up interviews included 26 and 18 respondents, respectively. Positive trends in current care pathways development and implementation include prioritization of high-quality evidence, enhancing the role of providers in development and implementation, and flexibility for providers to tailor treatment decisions to patients' needs. Nevertheless, there are some limitations in methodology for development and implementation, in criteria for evaluation, and in the degree of transparency surrounding these activities. CONCLUSIONS: Our research confirms that high-quality evidence of efficacy and safety are expected to be central to future pathway development, and that physicians play a major role in development and implementation. To achieve the goals of improving quality of care while managing costs, further efforts are required regarding systematic development and evaluation, consistent implementation and compliance metrics, and transparency in implementation outcomes and financial motivators.

Glioblastoma treatment patterns, survival, and healthcare resource use in real-world clinical practice in the USA.

BACKGROUND: Glioblastoma (GB) treatment remains challenging because of recurrence and poorly defined treatment options after first-line therapy. To better understand real-world application of treatment paradigms and their impact on outcomes, we describe patterns of treatment, outcomes, and use of cancer-related healthcare resource for glioblastoma in the USA. METHODS: A retrospective, online chart-abstraction study was conducted; each participating oncologist contributed ≤5 charts. Patients were ≥18 years with biopsy-confirmed primary or secondary newly diagnosed GB on or after 1 January 2010, had received first- and second-line therapies, and had information collected for ≥3 months after initiation of second-line therapy or until death. Assessments were descriptive and included Kaplan- Meier analyses from initiation to end of second-line therapy, disease progression, or death. RESULTS: One hundred sixty physicians contributed information on 503 patient charts. During first-line therapy, patients most commonly underwent temozolomide monotherapy (76.5%). During second-line therapy, patients most commonly underwent bevacizumab monotherapy (58.1%). Median duration of second-line therapy was 130 days; median time to disease progression was 113 days. Median survival was 153 days. Use of supportive care was observed to be numerically higher in first- compared with second-line therapy except for anti-depressants, growth factors, and stimulants. Frequently used resources included corticosteroids (78.8% of patients in first-line and 62.6% in second-line therapies), anti-epileptics (45.8% and 41.5%) and narcotic opioids (45.3% and 41.4%). CONCLUSIONS: Most GB patients received temozolomide during first-line therapy and bevacizumab monotherapy or combination therapy during second-line therapy. Use of supportive care appeared to be higher in first- compared with second-line therapy for some agents.

Retrospective Analysis of Dose Titration and Serum Testosterone Level Assessments in Patients Treated With Topical Testosterone.

Patterns of care following topical testosterone agent (TTA) initiation are poorly understood. This study aimed to characterize care following TTA initiation and compare results between patients with and without a serum testosterone (T) assay within 30 days before and including TTA initiation. Adult men (N=4,146) initiating TTAs from January 1, 2011, to March 31, 2012, were identified from a commercially insured database. Patients were included if they initiated at recommended starting dose (RSD) and had ≥12 and ≥6 months of continuous eligibility preinitiation (baseline) and postinitiation (study period), respectively. Patients were stratified by preinitiation T assay. Maintenance dose attainment month was determined using unadjusted generalized estimating equations regression to compare dose relative to RSD month by month. Outcomes included maintenance dose attainment month, time to stopping of index TTA refills or a claim for nonindex testosterone replacement therapy (TRT), and proportion of patients with study period T assay or diagnosis of hypogonadism (HG) or another low testosterone condition, and were compared using chi-square and Wilcoxon rank-sum tests for categorical and continuous variables, respectively. Maintenance dose was attained in Month 4 postinitiation, at 115.2% of RSD. Approximately 46% of patients had a preinitiation T assay; these men were more likely to receive a diagnosis of HG or another low testosterone condition, to have a follow-up T assay, to continue treatment by filling a nonindex TRT, and less likely to stop refilling treatment with their index TTA. Differences in care following TTA initiation suggest that preinitiation T assays (i.e., guideline-based care) may be helpful in ensuring treatment benefits.