Assessing the Added Therapeutic Benefit of Ultra-Expensive Drugs.

OBJECTIVES: While the United States does not have a method for assessing the added therapeutic benefit of drugs, France, Canada, and Germany do. We examined the added therapeutic benefit of the most expensive drugs prescribed to Medicare Part D beneficiaries in the United States. METHODS: We identified ultra-expensive drugs with annual Medicare spending that exceeded $62 794 (United States GDP per capita in 2018) using Medicare Part D Prescription Drug Spending and Utilization Data. We used added therapeutic benefit ratings assessed by health technology assessment agencies in France, Canada, and Germany. RESULTS: We identified 122 ultra-expensive drugs in 2018. Sixty-five percent of these drugs (n = 79) were assessed by at least one of the countries. Based on these assessments, approximately 75% received a low added therapeutic benefit rating. CONCLUSIONS: Most ultra-expensive drugs prescribed in the United States and assessed by France, Canada, and Germany provide low added therapeutic benefit. Policy reforms in the United States could use added therapeutic benefit to inform coverage and pricing decisions for ultra-expensive drugs. Similar to Germany, one approach would be to allow the company to set a market price for a limited period of time before requiring a price reduction if the added therapeutic benefit is below a certain threshold. Another approach would be to identify when drug prices are substantially more expensive in the United States and conduct an added therapeutic benefit assessment and price review on these drugs.

Comparative Approaches to Drug Pricing.

The United States relies primarily on market forces to determine prices for drugs, whereas most other industrialized countries use a variety of approaches to determine drug prices. Branded drug companies have patents and market exclusivity periods in most industrialized countries. During this period, pharmaceutical companies are allowed to set their list price as high as they prefer in the United States owing to the absence of government price control mechanisms that exist in other countries. Insured patients often pay a percentage of the list price, and cost sharing creates some pressure to lower the list price. Pharmacy benefit managers negotiate with drug companies for lower prices by offering the drug company favorable formulary placement and fewer utilization controls. However, these approaches appear to be less effective, compared with other countries' approaches to containing branded drug prices, because prices are substantially higher in the United States. Other industrialized countries employ various forms of rate setting and price regulation, such as external reference pricing, therapeutic valuation, and health technology assessment to determine the appropriate price.

Applying knowledge translation tools to inform policy: the case of mental health in Lebanon.

BACKGROUND: Many reform efforts in health systems fall short because the use of research evidence to inform policy remains scarce. In Lebanon, one in four adults suffers from a mental illness, yet access to mental healthcare services in primary healthcare (PHC) settings is limited. Using an "integrated" knowledge framework to link research to action, this study examines the process of influencing the mental health agenda in Lebanon through the application of Knowledge Translation (KT) tools and the use of a KT Platform (KTP) as an intermediary between researchers and policymakers. METHODS: This study employed the following KT tools: 1) development of a policy brief to address the lack of access to mental health services in PHC centres, 2) semi-structured interviews with 10 policymakers and key informants, 3) convening of a national policy dialogue, 4) evaluation of the policy brief and dialogue, and 5) a post-dialogue survey. RESULTS: Findings from the key informant interviews and a comprehensive synthesis of evidence were used to develop a policy brief which defined the problem and presented three elements of a policy approach to address it. This policy brief was circulated to 24 participants prior to the dialogue to inform the discussion. The policy dialogue validated the evidence synthesized in the brief, whereby integrating mental health into PHC services was the element most supported by evidence as well as participants. The post-dialogue survey showed that, in the following 6 months, several implementation steps were taken by stakeholders, including establishing national taskforce, training PHC staff, and updating the national essential drug list to include psychiatric medications. Relationships among policymakers, researchers, and stakeholders were strengthened as they conducted their own workshops and meetings after the dialogue to further discuss implementation, and their awareness about and demand for KT tools increased. CONCLUSIONS: This case study showed that the use of KT tools in Lebanon to help generate evidence-informed programs is promising. This experience provided insights into the most helpful features of the tools. The role of the KTP in engaging stakeholders, particularly policymakers, prior to the dialogue and linking them with researchers was vital in securing their support for the KT process and uptake of the research evidence.

A roadmap to parity in mental health financing: the case of Lebanon.

BACKGROUND: Inadequate access to mental health (MH) services in Lebanon, where prevalence is noteworthy, is a concern. Although a multitude of factors affects access to services, lack of financial coverage of MH services is one that merits further investigation. AIM OF THE STUDY: This study aims at providing a systematic description of MH financing systems with a special focus on Lebanon, presenting stakeholder viewpoints on best MH financing alternatives/strategies and recommending options for enhancing financial coverage. METHODS: A comprehensive review of existing literature on MH financing systems was conducted, with a focus on the system in Lebanon. In addition, key stakeholders were interviewed to assess MH organizational and financing arrangements. Finally, a national round table was organized with the aim of discussing findings (from the review and interviews) and developing an action roadmap. RESULTS: Taxation and out-of-pocket payments are the most common MH financing sources worldwide and in the Eastern Mediterranean Region. In Lebanon, all funding entities, except private insurance and mutual funds, cover inpatient and outpatient MH services, albeit with inconsistencies in levels of coverage. The national roundtable recommended two main MH financing enhancements: (i) creating a knowledge-sharing committee between insurers and MH specialists, and (ii) convincing labor unions/representatives to lobby for MH coverage as part of the negotiated benefit package. DISCUSSION: There are concerns regarding the equity, effectiveness and efficiency of the MH financing system in Lebanon. The fragmented system in Lebanon leads to differences in MH coverage across different financing intermediaries, which is inequitable. The fact that one out of four Lebanese suffer a mental disorder throughout their lives and very low percentages of those obtain treatment signals a problem in effectiveness. As for efficiency, the inefficient fragmentation of MH financing among seven intermediaries is a problematic characteristic of the healthcare financing system as a whole. Moreover, the orientation of the general healthcare system towards curative rather than preventive care is reflected in MH financing as well. Limitations of the study include the lack of access to data about the MH expenditure of every financing intermediary in Lebanon; therefore it was not possible to calculate a total annual MH spending on a country level. Another limitation was the inability to map the sources of funding with the MH service provision sector, as more extensive data about the MH services provided by each of the public, private, voluntary and informal sectors is needed. IMPLICATIONS FOR HEALTH POLICIES: Providing a clear description of the current MH financing system helps policymakers recognize the disparities present in the coverage of MH, guiding them into making informed decisions on allocation of funds. This study therefore constitutes the first step towards achieving more equitable and socially just coverage, advances knowledge and provides well-needed locally relevant research. Findings are expected to inform policymaking and have already contributed to influencing a change in the policy of the Internal Security Forces Health Fund. As a result of the roundtable discussion and follow up that ensued, the fund has removed the suicide attempt exclusion from its insurance policy.

Critical Care Beds With Continuous Lateral Rotation Therapy to Prevent Ventilator-Associated Pneumonia and Hospital-Acquired Pressure Injury: A Cost-effectiveness Analysis.

OBJECTIVES: Mechanical ventilation increases the risk of hospital-acquired conditions (HACs) such as ventilator-associated pneumonia (VAP) and pressure injury (PrI). Beds with continuous lateral rotation therapy (CLRT) are shown to reduce HAC incidence, but the value of switching to CLRT beds is presently unknown. We compared the cost-effectiveness of CLRT beds with standard care in intensive care units. METHODS: A cost-effectiveness analysis from the healthcare sector and societal perspectives was conducted. A Markov model was constructed to predict health state transitions from time of ventilation through 28 days for the healthcare sector perspective and 1 year for the U.S. societal perspective. Value of information was calculated to determine whether parameter uncertainty warranted further research. RESULTS: Our analysis suggested that CLRT beds dominate standard care from both perspectives. From the healthcare sector perspective, expected cost for CLRT was U.S. $47,165/patient compared with a higher cost of U.S. $49,258/patient for standard care. The expected effectiveness of CLRT is 0.0418 quality-adjusted life years/patient compared with 0.0416 quality-adjusted life years/patient for standard care. Continuous lateral rotation therapy dominated standard care in approximately 93% of Monte Carlo simulations from both perspectives. Value of information analysis suggests that additional research is potentially cost-effective. CONCLUSIONS: Continuous lateral rotation therapy is highly cost-effective compared with standard care by preventing HACs that seriously harm patients in the intensive care unit.

Predictors of orphan drug coverage restrictions in Medicare Part D.

OBJECTIVES: It is unclear on what basis Medicare drug plans impose coverage restrictions on orphan drugs. This study aims to investigate the factors associated with utilization controls in Medicare fee-for-service Part D formularies. STUDY DESIGN: Cross-sectional analysis. METHODS: We used multivariate logistic regression to assess the association between orphan drug characteristics and use of formulary utilization controls in 2016. We controlled for number of beneficiaries per drug, exclusivity expiration, and the number of plans and beneficiaries per formulary. We conducted sensitivity analyses using fixed and random effects. RESULTS: On average, 85% of orphan drugs on a formulary were placed on its highest cost-sharing tier and 76% were subject to prior authorization (PA). Orphan drugs with annual costs of $50,000 or more had twice the odds of having PA requirements compared with less expensive ones. Relative to orphan drugs with a single indication, drugs with multiple indications were more likely to have restrictions. Less effective drugs had 1.5 times the odds of highest tier placement relative to more effective drugs. The presence of black box warnings and patient assistance programs were associated with more restricted access. Orphan drugs with generics were less likely to undergo restrictions than those without generics (all P < .05). CONCLUSIONS: Plans are making evidence-based decisions by rewarding more clinically effective and safer orphan drugs. They are penalizing drugs with multiple indications. Surprisingly, plans place fewer restrictions on orphan drugs that have a generic equivalent, which may further discourage generic entry into the orphan space, where competition is already sparse.

Varying Trends In The Financial Viability Of US Rural Hospitals, 2011-17.

The financial viability of rural hospitals has been a matter of serious concern, with ongoing closures affecting rural residents' access to medical services. We examined the financial viability of 1,004 US rural hospitals that had consistent rural status in 2011-17. The median overall profit margin improved for nonprofit critical access hospitals (from 2.5 percent to 3.2 percent) but declined for other hospitals (from 3.0 percent to 2.6 percent for nonprofit non-critical access hospitals, from 3.2 percent to 0.4 percent for for-profit critical access hospitals, and from 5.7 percent to 1.6 percent for for-profit non-critical access hospitals). Occupancy rate and charge markup were positively associated with overall margins: In 2017 hospitals with low versus high occupancy rates had median overall profit margins of 0.1 percent versus 4.7 percent, and hospitals with low versus high charge markups had median overall margins of 1.8 percent versus 3.5 percent. Rural hospital financial viability deteriorated in states that did not expand eligibility for Medicaid and was lower in the South. Rural hospitals that closed during the study period had a median overall profit margin of -3.2 percent in their final year before closure. Policy makers should compare the incremental cost of providing essential services between hospitals and other settings to balance access and efficiency.