Does a One-Size-Fits-All Cost-Sharing Approach Incentivize Appropriate Medication Use? A Roundtable on the Fairness and Ethics Associated with Variable Cost Sharing.

BACKGROUND: Tiered formularies, in which patients pay copays or coinsurance out-of-pocket (OOP), are used to manage costs and encourage more efficient health care resource use. Formulary tiers are typically based on the cost of treatment rather than the medical appropriateness for the patient. Cost sharing may have unintended consequences on treatment adherence and health outcomes. Use of higher-cost, higher-tier medications can be due to a variety of factors, including unsuccessful treatment because of lack of efficacy or side effects, patient clinical or genetic characteristics, patient preferences to avoid potential side effects, or patient preferences based on the route of administration. For example, patients with rheumatoid arthritis may be required to fail low-cost generic treatments before obtaining coverage for a higher-tier tumor necrosis factor alpha inhibitor for which they would have a larger financial burden. Little is known about stakeholders' views on the acceptability of greater patient cost sharing if the individual patient characteristics lead to the higher-cost treatments. OBJECTIVE: To identify and discuss the trade-offs associated with variable cost sharing in pharmacy benefits. METHODS: To discuss the trade-offs associated with variable cost sharing in pharmacy benefits, we convened an expert roundtable of patient, payer, and employer representatives (panelists). Panelists reviewed background white papers, including an ethics framework; actuarial analysis; legal review; and stakeholder perspectives representing health plan, employer, and patient views. Using case studies, panelists were asked to consider (a) when it would be more (or less) acceptable to require higher cost sharing; (b) the optimal distribution of financial burdens across patients, all plan members, and employers; and (c) the existing barriers and potential solutions to align OOP costs with medically appropriate treatments. RESULTS: Panelists felt it was least acceptable for patients to have greater OOP costs if the use of the higher-cost treatment was due to biological reasons such as step therapy (6 = unacceptable, 9 = neutral, 2 = acceptable) or diagnostic results (5 = unacceptable, 10 = neutral, and 2 = acceptable). In contrast, panelists felt it was more acceptable for patients to pay greater OOP costs when treatment choice was based on preferences to avoid a side-effect risk (1 = unacceptable, 3 = neutral, and 13 = acceptable) or the route/frequency of administration (1 = unacceptable, 1 = neutral, and 15 = acceptable). Five guiding principles emerged from the discussion: When patients have tried lower-cost therapies unsuccessfully, the benefits of higher-cost treatments were certain and significant, the cost difference between treatments was aligned with improved benefits, and penalties due to bad luck were mitigated, then cost-sharing differences should be minimized but not eliminated. CONCLUSIONS: Patient OOP costs can affect the use of both inappropriate and appropriate medications. This study identified 5 guiding principles to determine when it was more (or less) acceptable for patients with the same or similar conditions to have different OOP costs. Barriers that hinder the alignment of care and patient cost sharing exist. Policies that facilitate the alignment of patient cost sharing with appropriate care are needed. DISCLOSURES: Funding for this roundtable was provided by the National Pharmaceutical Council (NPC). Graff and Dubois are employed by the NPC. Shih was employed by the NPC at the time of this study. Barker, Dieguez, Sherman, and Larson received consulting fees for participation in this study. Larson also reports receiving grants and other payment from multiple major pharmaceutical manufacturers outside of this study. The NPC employees developed the study design and chose the case studies in collaboration with the white paper authors. The roundtable was facilitated by Dubois, and the meeting summary and manuscript were written by Graff and Shih, with revisions by all roundtable participants. The abstract for this article was previously presented as a poster at the following meetings: Stakeholder perspectives on balancing patient-centeredness and drug costs in the design of pharmacy benefits. Presented at: Academy of Managed Care Pharmacy 27th Annual Meeting & Expo; San Diego, California; April 8, 2015. Considering efficiency and fairness in the design of prescription drug benefits: seeking a balanced approach to improve patient access to medically appropriate medication and manage drug costs. Presented at: AcademyHealth Annual Research Meeting; Minneapolis, Minnesota; June 15, 2015. Study concept and design were contributed by Shih, Dubois, and Graff, along with Barker and Dieguez. Barker and Dieguez took the lead in data collection, assisted by Graff, Shih, and Dubois. Data interpretation was performed by Shih, Larson, Sherman, and Graff, with assistance from Dubois. The manuscript was written and revised by Graff and Shih, with assistance from the other authors.

The cost-effectiveness of rotavirus vaccination in Malawi.

BACKGROUND: Rotarix (GlaxoSmithKline), a newly licensed rotavirus vaccine requiring 2 doses, may have the potential to save hundreds of thousands of lives in Africa. Nations such as Malawi, where Rotarix is currently under phase III investigation, may nevertheless face difficult economic choices in considering vaccine adoption. METHODS: The cost-effectiveness of implementing a Rotarix vaccine program in Malawi was estimated using published estimates of rotavirus burden, vaccine efficacy, and health care utilization and costs. RESULTS: With 49.5% vaccine efficacy, a Rotarix program could avert 2582 deaths annually. With GAVI Alliance cofinancing, adoption of Rotarix would be associated with a cost of $5.07 per disability-adjusted life-year averted. With market pricing, Rotarix would be associated with a base case cost of $74.73 per disability-adjusted life-year averted. Key variables influencing results were vaccine efficacy, under-2 rotavirus mortality, and program cost of administering each dose. CONCLUSIONS: Adopting Rotarix would likely be highly cost-effective for Malawi, particularly with GAVI support. This finding holds true across uncertainty ranges for key variables, including efficacy, for which data are becoming available.

Diffusion of new technology and payment policies: coronary stents.

Medicare payment is often cited as a major driver of medical technology diffusion. Stakeholders claimed that beneficiaries would be denied access to stents because Medicare payment did not initially cover the cost of stents. Nevertheless, stents diffused rapidly, including to untested indications. Outcomes with stents improved over time, primarily because of a fundamental property of technology diffusion termed "reinvention," in which new technology is modified by users. The traditional system of regulatory approval and reimbursement does not account for this dynamic process. There has been no incentive for systematic collection of data to determine which modifications are most beneficial.

Improving depiction of benefits and harms: analyses of studies of well-known therapeutics and review of high-impact medical journals.

The issues of weighing benefits and harms and of shared decision-making have become increasingly important in recent years. There is limited knowledge and lack of adequate data on the most transparent method of communicating the information. In this article we discuss examples of communicating benefits and harms for well-known therapeutics, illustrating that relative risk estimates are not helpful for communicating the chance of experiencing adverse events. In addition, we show that asymmetric presentation of the data for benefits and harms is likely to bias toward showing greater benefits and diminishing the importance of the harms (or vice versa). We also present preliminary results of a brief review of high-impact medical journals that show limitations of current systematic reviews. In the review we found that every second published study does not discuss frequency data and 1 in 3 studies that report information on both benefits and harms does not report information in the same metric. We conclude that consistently depicting benefit and harm information in frequencies can substantially improve the communication of benefits and harms. Investigators should be requested to provide frequency data along with relative risk information in the publication of their scientific findings. Currently, even in the highest impact medical journals, evidence of benefits and harms is not consistently presented in ways that facilitate accurate interpretation.