RESUMO
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.