Assessing Payers' Preferences for Real-World Evidence in the United States: A Discrete Choice Experiment.

OBJECTIVES: To rank the US payers' preferences for attributes of real-world evidence (RWE) studies in the context of chronic disease and to quantify trade-offs among them. METHODS: We conducted a discrete choice experiment in which 180 employees from payer organizations were tasked to choose between 2 RWE studies assuming they were assessing evidence to inform formulary decisions for chronic disease treatment. Each RWE study was characterized by 7 attributes with 3 levels each: very informative, moderately informative, and not measured. We used a D-optimal main-effects design. Survey data were fitted to a conditional logit model to obtain a relative measure of the ranking of importance for each attribute. RESULTS: Clinical outcomes were the most preferred attribute. It was 4.68 times as important as productivity outcomes-the least preferred attribute. It was followed by health-related quality of life (2.78), methodologic rigor (2.09), resource utilization (1.71), and external validity (1.56). CONCLUSIONS: This study provides a quantification of the value payers place on key RWE attributes. Across attributes, payers have higher preferences for clinical and health-related quality of life outcomes than the other attributes. Between attributes' levels, payers prefer high levels of information in clinical outcomes and methodologic rigor but are indifferent in other attributes. Our results bridge the gap between the information that payers seek and the attributes that RWE studies prioritize and effectively guide future research design.

Trends and Factors Associated With Insurer Approval of Proprotein Convertase Subtilisin/Kexin Type 9 Inhibitor Prescriptions.

OBJECTIVES: Proprotein convertase subtilisin/kexin type 9 inhibitors (PCSK9is)-innovative yet costly cholesterol-lowering agents-have been subject to substantial prior authorization (PA) requirements and low approval rates. We aimed to investigate trends in insurer approval and reasons for rejection for PCSK9i prescriptions as well as associations between patients' demographic, clinical, pharmacy, payer, and PCSK9i-specific plan/coverage factors and approval. METHODS: We examined trends in PCSK9i approval rates and reasons for rejection using medical and prescription claims from 2015 to 2017 for individuals who received a PCSK9i prescription. We used multinomial logistic regression to estimate quarterly risk-adjusted approval rates for initial PCSK9i prescriptions and approval for any PCSK9i prescription within 30, 90, and 180 days of the initial PCSK9i prescription. For a 2016 subsample for whom we had PCSK9i-specific plan policy data, we examined factors associated with approval including PCSK9i-specific plan formulary coverage, step therapy requirements, and number of PA criteria. RESULTS: The main sample included 12 309 patients (mean age 64.8 years [SD = 10.8], 52.1% female, 51.5% receiving Medicare) and was similar in characteristics to the 2016 subsample (n = 6091). Approval rates varied across quarters but remained low (initial prescription, 13%-23%; within 90 days, 28%-44%). Over time, rejections owing to a lack of formulary coverage decreased and rejections owing to PA requirements increased. Lack of formulary coverage and having ≥11 PA criteria in the plan policy were associated with lower odds of PCSK9i prescription approval. CONCLUSIONS: These findings confirm ongoing PCSK9i access issues and offer a baseline for comparison in future studies examining the impact of recent efforts to improve PCSK9i access.

A Novel Point-of-Care Solution to Streamline Local Wound Formulary Development and Promote Cost-effective Wound Care.

OBJECTIVE: To develop and implement a point-of-care digital solution to streamline the creation and maintenance of wound care product formularies and promote cost-effective wound management. METHODS: Researchers used Design Thinking methodology to develop the Formulary Module, a point-of-care digital solution within a clinical and reimbursement decision support web application for wound care and hyperbaric clinicians. The module was implemented in a US hospital-based outpatient wound clinic as follows: A baseline list of products was established, with brands automatically grouped by product category. Brands within each dressing category were compared, and redundancy eliminated. Study authors assessed the financial impact of formulary implementation in the wound clinic by comparing inventory expenditure before and after implementation. RESULTS: Implementation of the digital Formulary Module resulted in a 36% decrease in products (67 to 43 across 22 types), 38.73% decrease in the monthly average dollar spent on chargeable products, 29.56% decrease in the average dollar amount spent on chargeable products per patient visit, and increased staff efficiency. CONCLUSIONS: The Formulary Module has the potential to increase the adoption of cost-effective practices in wound care significantly.

Formulary Coverage of Brand-Name Adalimumab and Biosimilars Across Medicare Part D Plans.

This study examines formulary coverage of brand-name adalimumab and biosimilars across Medicare Part D plans.

Prior Authorization Policies and Preferred Drug Lists in Medicaid Plans: Stakeholder Perspectives on the Implications for Youth with ADHD.

This qualitative study describes how Medicaid policies create challenges for the delivery and receipt of mental health treatment for low-income youth in Georgia. We conducted focus groups with caregivers of Medicaid-enrolled children with ADHD and semi-structured interviews with providers and administrators at four safety net clinics that provided mental health care to these youth. Stakeholders reported that prior authorization policies for psychosocial services, restrictiveness of preferred drug lists, and changes in preferred drug lists in Medicaid plans created barriers to treatment continuity and quality for youth with ADHD and led to more administrative burden for safety-net clinics serving these youth.

Reference Pricing in Germany: Implications for U.S. Pharmaceutical Purchasing.

Issue: The German health care system resembles that of the United States in important ways ­ it is financed by multiple private payers and relies principally on negotiation rather than regulation to establish prices. New drugs that offer minimal benefits compared with existing alternatives within a therapeutic class are subject to reference pricing; those with incremental benefits are subject to price negotiations. Together, the reference and negotiated pricing systems have held German prices substantially below U.S. equivalents. Goal: To describe the German reference-pricing system and compare it to tiered formularies and consumer cost-sharing in the United States. Methods: Document review and interviews with leaders in payer, policy, and pharmaceutical industry organizations in Germany. Key Findings and Conclusions: The German pharmaceutical pricing system uses modest levels of consumer cost-sharing to influence consumers' choices for drugs with therapeutically equivalent alternatives. Manufacturers are free to set the prices of their products, but insurers will not pay more for a new drug than for its comparators unless it offers an additional clinical benefit. For drugs covered by reference pricing, the insurers' payment maximum is set at a level that ensures sufficient choices of low-priced options. These models offer an alternative to the U.S. system of tiered formularies.

Pharmacy Benefit Managers: Practices, Controversies, and What Lies Ahead.

Issue: Pharmacy benefit managers (PBMs) are responsible for negotiating payment rates for a large share of prescription drugs distributed in the U.S. Recently, policymakers have expressed concern that certain PBMs' business practices may not be consistent with public policy goals to improve the value of pharmaceutical spending. Goal: We sought to explain key controversies related to PBM practices and their roles in driving value in the pharmaceutical market. Methods: Literature review and feedback from top experts on PBM business practices and potential policy solutions. Key Findings and Conclusion: In some cases, PBMs' use of rebates has contributed to high pharmaceutical costs, yet proposed solutions to the rebate controversy--including passing the rebate through to payers or patients--will not on their own reduce overall pharmaceutical spending without other policies that drive toward value. Policymakers seeking to reform pharmaceutical reimbursement beyond the practice of rebates will need to consider these changes in light of the recent mergers between PBMs and insurers and the entry of new market competitors.

Computational health economics for identification of unprofitable health care enrollees.

Health insurers may attempt to design their health plans to attract profitable enrollees while deterring unprofitable ones. Such insurers would not be delivering socially efficient levels of care by providing health plans that maximize societal benefit, but rather intentionally distorting plan benefits to avoid high-cost enrollees, potentially to the detriment of health and efficiency. In this work, we focus on a specific component of health plan design at risk for health insurer distortion in the Health Insurance Marketplaces: the prescription drug formulary. We introduce an ensembled machine learning function to determine whether drug utilization variables are predictive of a new measure of enrollee unprofitability we derive, and thus vulnerable to distortions by insurers. Our implementation also contains a unique application-specific variable selection tool. This study demonstrates that super learning is effective in extracting the relevant signal for this prediction problem, and that a small number of drug variables can be used to identify unprofitable enrollees. The results are both encouraging and concerning. While risk adjustment appears to have been reasonably successful at weakening the relationship between therapeutic-class-specific drug utilization and unprofitability, some classes remain predictive of insurer losses. The vulnerable enrollees whose prescription drug regimens include drugs in these classes may need special protection from regulators in health insurance market design.

Glycemic Improvement with a Fixed-dose combination of DPP-4 inhibitor + metformin in patients with Type 2 diabetes (GIFT study).

This study investigates changes in A1C following a switch from dual therapy of metformin and DPP-4 inhibitor to a fixed-dose combination (FDC) of metformin + DPP-4 inhibitor following the introduction of the FDC in the provincial formulary. The LMC Diabetes Registry was queried retrospectively for patients with type 2 diabetes, aged between 18 and 80 years with at least one A1C recorded prior and ≥3 months post-switch. Five hundred and sixty-eight subjects with mean age 64 ± 12 years and mean A1C 7.7% ± 1.2% met study criteria. Overall, A1C was 0.3% lower post-switch to FDC (P < .01). In stratified analysis, subjects with baseline A1C between 7% and 10% had 0.4% lower A1C (P < .01), with 31% of these subjects reaching target A1C ≤7%, post-switch. A1C reduction was greater among patients with a higher baseline pill burden: 0.4% among those using ≥10 pills/day vs. 0.1% for those with <10 pills/day (P = .02). In this real-world study, switching to FDC of metformin + DPP-4 inhibitor was associated with a significant improvement in A1C. Switching to FDC, especially in patients with high pill burden, can improve A1C goal achievement in clinical practice.

The 'top 100' drugs and classes in England: an updated 'starter formulary' for trainee prescribers.

AIMS: Prescribing is a complex skill required of doctors and, increasingly, other healthcare professionals. Use of a personal formulary can help to develop this skill. In 2006-9, we developed a core list of the 100 most commonly prescribed drugs. Our aim in the present study was to update this 'starter formulary' to ensure its continued relevance for prescriber training. METHODS: We analysed large contemporary primary and secondary care datasets to identify the most frequently prescribed medicinal products. Items were classified into natural groups, broadly following their British National Formulary classification. The resulting drug groups were included in the core list if they comprised ≥0.1% prescriptions in both settings or ≥0.2-0.3% prescriptions in one setting. Drugs from emergency guidelines that did not qualify by prescribing frequency completed the list. RESULTS: Over 1 billion primary care items and approximately 1.8 million secondary care prescriptions were analysed. The updated list comprises 81 drug groups commonly prescribed in both settings; six from primary care; seven from secondary care; and six from emergency guidelines. Eighty-eight per cent of the formulary was unchanged. Notable changes include entry of newer anti-epileptics and dipeptidyl peptidase-4 inhibitors and exit of phenytoin and thiazolidinediones. CONCLUSIONS: The relative stability of the core drug list over 9 years and the current update ensure that learning based on this list remains relevant to practice. Trainee prescribers may be encouraged to use this 'starter formulary' to develop a sound basis of prescribing knowledge and skills that they can subsequently apply more widely.

Selection of essential medicines for the prevention and treatment of cardiovascular diseases in low and middle income countries.

BACKGROUND: The incidence and mortality of cardiovascular diseases (CVDs) in low and middle income countries (LMICs) have been increasing, while access to CVDs medicines is suboptimal. We assessed selection of essential medicines for the prevention and treatment of CVDs on national essential medicines lists (NEMLs) of LMICs and potential determinants for selection. METHODS: Only operational NEMLs were considered eligible for this study. A selection of medicines listed under "cardiovascular medicines" or "blood products and plasma substitutes" in the NEMLs were included if they were present on international guidelines for the prevention and treatment of CVDs (hyperlipidemia, hypertension, platelet inhibition, ischemic stroke, stable ischemic heart disease, acute coronary syndromes, heart failure, atrial fibrillation, peripheral arterial disease and acute limb ischemia). The number and diversity of essential medicines selected for CVDs were studied. Moreover, determinants of selection of essential medicines for CVDs at a national level were explored. Data analysis was done using univariate linear regression and non-parametric tests. RESULTS: All medicine groups listed by the international guidelines were selected by the majority of the 34 countries studied with the exception of adenosine diphosphate receptor inhibitors which appeared on less than half of the NEMLs studied (41% of countries). The total number of essential medicines for the prevention and treatment of cardiovascular diseases (median 24 (range 16-50)) differed significantly across income levels (median range: 19.5-25, p = 0.014) and across regions (median range: 20-32, p = 0.049). When recommendations of the international guidelines were considered, over 75% of the NEMLs contained essential medicines for the majority of CVDs. CONCLUSION: The main medicine classes for the management of CVDs were represented on NEMLs. Consequently, for the majority of CVDs, evidence-based guideline-recommended treatment is possible as far as selection of essential medicines is concerned. Selection will therefore not be the limiting step in access to medicines for cardiovascular diseases.

Patients' knowledge and attitudes to the Wise List - a drug formulary from the Stockholm Drug and Therapeutic committee.

BACKGROUND: Involving patients in decisions about their pharmacotherapy is crucial for a satisfactory treatment outcome. Information and opinions about medicines are available from a variety of sources. The Wise List is the drug formulary of recommended essential medicines for the Stockholm healthcare region and is issued by the Drug and Therapeutics Committee (DTC). To inform the public about treatment for common diseases and the concept of recommended medicines, a patient edition of the Wise List was developed. The aim of this study was to explore patients' knowledge, needs and attitudes to the Wise List, DTC and information about medicines in general. METHODS: To examine patient knowledge about recommended medicines a survey (n = 312) was carried out at four large primary healthcare centres in Stockholm, Sweden. To further elucidate the patients' needs of the information on recommended medicines and medicines in general, three focus group discussions (FGDs) were performed. RESULTS: Of the respondents 57% did not recognise the Wise List, 26% recognised but did not use it and 17% used it. A total of 63% reported that they search for information about medicines. The most common information source was "asking their doctor" (36%) followed by searching the internet (31%). The FGDs revealed that the patients were not interested in medicines in general, only in the medicines they use themselves. They did not understand the aim of the Wise List or how they could benefit from information about recommended medicines. The patients expressed a wish to access all information they need about their own care as well as public healthcare information at one location. CONCLUSION: The intended aim of the DTC with providing information to the public was not achieved as the patients have difficulties to understand the information and how they should use it. The patients were not interested in medicines in general, they wanted information tailored to their specific needs. The findings highlight the importance of creating tools for patients in collaboration with them and evaluate the concept continuously.

Pharmaceutical Reference Pricing: Does It Have a Future in the U.S.?

Issue: Reference pricing is an emerging health insurance benefit design aimed at reducing health costs. In this model, an insurer establishes a maximum payment that it will contribute toward covering the price of a product or service in situations where there is wide price variation for therapeutically similar drugs, diagnostics, or procedures. Experiences to date indicate that reference pricing can influence patients and physicians to switch to less costly options within each therapeutic class, reducing overall drug prices. Goal: Describe how reference pricing can be and has been applied to drugs in the United States and compare it to more conventional pharmaceutical benefit designs such as tiered formularies and coinsurance. Methods: Assessment of peer-reviewed research and the experiences of employers that have used reference pricing. Findings and Conclusions: To appropriately motivate price-conscious consumer choice, reference pricing must include up-to-date information. Consumers and physicians must have access to the prices charged at different distribution sites and for different drugs within each therapeutic class. Reference pricing also must include information on quality. Several modifications to the reference pricing model should be made before it can be adapted to specialty drugs, and those changes should be informed by comparative effectiveness research.

CVS and the $100,000 QALY.

The PBM unit of the health giant CVS decided that any new drug exceeding $100,000 per quality-adjusted life year (QALY) may be excluded from the formularies that are maintained by its clients. Patient groups (with the backing of pharma) say that the measure is discriminatory.

Impact of a Value-based Formulary on Medication Utilization, Health Services Utilization, and Expenditures.

BACKGROUND: Value-based benefit design has been suggested as an effective approach to managing the high cost of pharmaceuticals in health insurance markets. Premera Blue Cross, a large regional health plan, implemented a value-based formulary (VBF) for pharmaceuticals in 2010 that explicitly used cost-effectiveness analysis (CEA) to inform medication copayments. OBJECTIVE OF THE STUDY: The objective of the study was to determine the impact of the VBF. DESIGN: Interrupted time series of employer-sponsored plans from 2006 to 2013. SUBJECTS: Intervention group: 5235 beneficiaries exposed to the VBF. CONTROL GROUP: 11,171 beneficiaries in plans without any changes in pharmacy benefits. INTERVENTION: The VBF-assigned medications with lower value (estimated by CEA) to higher copayment tiers and assigned medications with higher value to lower copayment tiers. MEASURES: Primary outcome was medication expenditures from member, health plan, and member plus health plan perspectives. Secondary outcomes were medication utilization, emergency department visits, hospitalizations, office visits, and nonmedication expenditures. RESULTS: In the intervention group after VBF implementation, member medication expenditures increased by $2 per member per month (PMPM) [95% confidence interval (CI), $1-$3] or 9%, whereas health plan medication expenditures decreased by $10 PMPM (CI, $18-$2) or 16%, resulting in a net decrease of $8 PMPM (CI, $15-$2) or 10%, which translates to a net savings of $1.1 million. Utilization of medications moved into lower copayment tiers increased by 1.95 days' supply (CI, 1.29-2.62) or 17%. Total medication utilization, health services utilization, and nonmedication expenditures did not change. CONCLUSIONS: Cost-sharing informed by CEA reduced overall medication expenditures without negatively impacting medication utilization, health services utilization, or nonmedication expenditures.

Variation in Prescription Drug Coverage for Triptans: Analysis of Insurance Formularies.

OBJECTIVES: To analyze triptan coverage by insurers to examine (1) possible disparities in coverage for different formulations (oral, intranasal, etc) and (2) quantity limits and stepped care requirements to obtain triptans. BACKGROUND: Triptans are FDA approved migraine abortive medications. Patients frequently state that they have difficulty accessing triptans prescribed to them. METHODS: We searched the 2015 drug formularies of commercial and government health insurers providing coverage in NY State. We created a spreadsheet with all of the commercially available triptans and included information about covered formulations, tier numbers and quantity limits for each drug. We then calculated the number of listed plans that cover or do not cover each triptan or triptan formulation, the total number of medications not covered by an insurance provided across all of its plans, as well as the percentage of plans offered by individual companies and across all companies that covered each drug. We also calculated the number and proportion of plans that imposed quantity limits or step therapy for each drug. RESULTS: Of the 100 formularies searched, generic sumatriptan (all formulations), naratriptan, and zolmitriptan tablets were covered by all plans, and rizatriptan tablets and ODTs were covered by 98% of plans. Brand triptans were less likely to be covered: 4/36 Medicaid plans covered brand triptans. Commercial insurers were more likely to cover brand triptans. All plans imposed quantity limits on 1+ triptan formulations, with >80% imposing quantity limits on 14/19 formulations studied. Almost all plans used tiers for cost allocation for different medications. Generic triptans were almost always in Tier 1. Brand triptans were most commonly in Tier 3. Approximately 40% of brand triptans required step therapy, compared with 11% of generic triptans. CONCLUSIONS: There are substantial variations in coverage and quantity limits and a high degree of complexity in triptan coverage for both government and commercial plans.

Constraints on Formulary Design Under the Affordable Care Act.

I study the effect of prescription drug essential health benefits (EHB) requirements from the Affordable Care Act on prescription drug formularies of health insurance marketplace plans. The EHB regulates the number of drugs covered but leaves other dimensions (cost sharing and utilization management) of the formulary unregulated. Using data on almost all formularies in the country, I demonstrate that requiring insurers to cover one additional drug adds 0.22 drugs (3.3%) to the average formulary, mostly owing to firms increasing the number of drugs covered to comply with the EHB requirement. The EHB requirement also increases the probability that a drug is subject to utilization management and is assigned to a higher (more costly) formulary tier. My results suggest that newly covered drugs are 22.3 percentage points more likely to be subject to utilization management, compared to 36.7% for the average covered drug. Using formularies for Medicare Advantage plans, which are subject to uniform, nationwide benefit design standards, and the formulary status of newly approved drugs that do not satisfy the EHB requirement, I reject the hypotheses that consumer demand or effects on plan entry can explain my results. Copyright © 2017 John Wiley & Sons, Ltd.

Antimicrobial stewardship in the Federal Bureau of Prisons: Approaches from the national and local levels.

OBJECTIVES: To determine the impact of national and local antimicrobial stewardship measures on overall antibiotic prescribing in the Federal Bureau of Prisons (BOP). SETTING: Care was delivered to more than 160,000 inmates in 122 BOP facilities in the United States and Puerto Rico. PRACTICE DESCRIPTION: Medical centers and health services clinics staffed by in-house medical staff, consultants, and specialists. Staffs include a variety of disciplines, including physicians, pharmacists, dentists, nurses, infection control personnel, therapists, health services administrators, and institution executive staff. PRACTICE INNOVATION: Innovations occurred on 2 levels: local components were used to reinforce national initiatives. Local institutions used a multidisciplinary team approach including education and focused evaluations of all antibiotic prescriptions before dispensing. National initiatives included the development of a closed formulary, clinical practice guidelines, an antimicrobial stewardship group led by pharmacy, development of tools and strategies for institutions, inclusion in the BOP strategic plan, and a drug utilization evaluation. EVALUATION: This was a study of antimicrobial stewardship within BOP and the resultant impact on antibiotic prescriptions. In addition, one institution's antimicrobial stewardship methods were reviewed to determine the impact on antibiotic prescribing practices. RESULTS: The total number of antibiotic prescriptions in BOP-managed institutions in fiscal year (FY) 2010 (October 2009 to September 2010) was 142,907 and progressively decreased to 105,832 in FY2015. The number of antibiotic prescriptions per 1000 inmates correspondingly decreased from 829 in FY2010 to 625 in FY2015. The overall number of antibiotic prescriptions as a percentage of total prescriptions decreased from 7.64% in FY2010 to 5.84% in FY2015. CONCLUSION: A robust multidisciplinary antimicrobial stewardship program has likely contributed to a decrease in both the total number and the rate of antibiotic prescriptions on a per-1000-patient basis in BOP.

The evolution of the cancer formulary review in Canada: Can centralization improve the use of economic evaluation?

Public reimbursement of drugs is a costly proposition for health care systems. Decisions to add drugs to the public formulary are often guided by review processes and committees. The evolution of the formulary review process in Canada's publicly funded health system is characterized by increased centralization and systematization. In the past, the review of evidence and recommendation was conducted at the regional level, but was replaced with the pan-Canadian Oncology Drug Review in 2011. We assess the extent to which centralization and systematization of the review process have responded to past challenges, focusing on the use of economic evaluation in the process. Past challenges with economic evaluation experienced by regionalized review committees were identified from literature and qualitative data collected in the province of Nova Scotia. We categorize these using a typology with a macro-, meso, and micro-level hierarchy, which provides a useful framework for understanding at which level change is required, and who has the authority to influence change. Using grounded theory methods, we identify approaches used by Nova Scotia past committee members to compensate for perceived shortcomings of the process. These include an undue reliance on other committee members, on the multidisciplinarity of the committee, and on past decisions. Using a policy analysis approach, we argue that centralization and systematization of the review process only partially address the shortcomings of the previous regionalized process. Lessons from Canada can inform policy discussions across all health systems, where similar challenges with the formulary review process have been identified. © 2016 The Authors. The International Journal of Health Planning and Management published by John Wiley & Sons Ltd.