Are Payers Ready, Willing, and Able to Provide Access to New Durable Gene Therapies?

OBJECTIVE: To explore payer feedback regarding awareness of new gene therapies, sustainability of current financing mechanisms, unique challenges by payer segment, and need and preference for new financial models. STUDY DESIGN: Qualitative interview with standardized interview guide. METHODS: Sixty-minute telephone interviews were conducted with financial decision makers from 15 US payers between August and September 2017. RESULTS: One-third of payers interviewed (n = 5) were newly aware and learning about new gene therapies, 40% (n = 6) described watchful waiting, whereas 26.7% (n = 4) were engaged in active management. New payment models-specifically, performance-based agreements and risk-pooling-were supported by 47% (n = 7) of payers, whereas the current payment model was supported by 53% (n = 8). Major challenges included uncertainty related to utilization, cost, and duration of cure. Payers cited regulation, plan turnover, and ability to track long-term outcomes as barriers to implementation of new models. CONCLUSIONS: Access to new gene therapies may be impacted by payer ability to absorb the cost of coverage. Variation exists in awareness of new gene therapies and level of incorporation of new costs into future plan coverage. The sustainability of current financing mechanisms varies by payer segment, profitability, and size; smaller plans and Medicaid are likely to be impacted first. Government reinsurance, commercial reinsurance, and stop-loss insurance backstop current reimbursement models, dampening the need for urgent action. The tipping point for action may be severe premium inflation in stop loss and reinsurance. Payers are open to innovative financing models that improve financial predictability and reward clinical performance.

Fair, just and compassionate: A pilot for making allocation decisions for patients requesting experimental drugs outside of clinical trials.

Patients have received experimental pharmaceuticals outside of clinical trials for decades. There are no industry-wide best practices, and many companies that have granted compassionate use, or 'preapproval', access to their investigational products have done so without fanfare and without divulging the process or grounds on which decisions were made. The number of compassionate use requests has increased over time. Driving the demand are new treatments for serious unmet medical needs; patient advocacy groups pressing for access to emerging treatments; internet platforms enabling broad awareness of compelling cases or novel drugs and a lack of trust among some that the pharmaceutical industry and/or the FDA have patients' best interests in mind. High-profile cases in the media have highlighted the gap between patient expectations for compassionate use and company utilisation of fair processes to adjudicate requests. With many pharmaceutical manufacturers, patient groups, healthcare providers and policy analysts unhappy with the inequities of the status quo, fairer and more ethical management of compassionate use requests was needed. This paper reports on a novel collaboration between a pharmaceutical company and an academic medical ethics department that led to the formation of the Compassionate Use Advisory Committee (CompAC). Comprising medical experts, bioethicists and patient representatives, CompAC established an ethical framework for the allocation of a scarce investigational oncology agent to single patients requesting non-trial access. This is the first account of how the committee was formed and how it built an ethical framework and put it into practice.

An early examination of access to select orphan drugs treating rare diseases in health insurance exchange plans.

BACKGROUND: Patients with rare diseases often face significant health care access challenges, particularly since the number of available treatment options for rare diseases is limited. The implementation of health insurance exchanges promises improved access to health care. However, when purchasing a plan, patients with rare diseases need to consider multiple factors, such as insurance premium, access to providers, coverage of a specific medication or treatment, tier placement of drug, and out-of-pocket costs.  OBJECTIVE: To provide an early snapshot of the exchange plan landscape from the perspective of patients with select rare diseases by evaluating the degree of access to medications in a subset of exchange plans based on coverage, tier placement, associated cost sharing, and utilization management (UM) applied.  METHODS: The selection of drugs for this analysis began by identifying rare diseases with FDA-approved treatment options using the National Institutes of Health Office of Rare Diseases' webpage and further identification of a subset of drugs based on select criteria to ensure a varied sample, including the characteristics and prevalence of the condition. The medications were categorized based on whether alternative therapies have FDA approval for the same indication and whether there are comparators based on class or therapeutic area. The list was narrowed to 11 medications across 7 diseases, and the analysis was based on how these drugs are listed in exchange plan outpatient pharmacy benefit formularies. This analysis focused on 84 plans in 15 states with the highest expected exchange enrollment and included a variety of plan types to ensure that variability in the marketplace was represented. To best approximate plans that will have the greatest enrollment, the analysis focused on silver and bronze plan formularies because consumers in this market are expected to be sensitive to premiums. Data on drug coverage, tier placement, cost, and UM were collected from these plans beginning October 1, 2013, with the launch of the open enrollment period. RESULTS: Coverage and use of UM for selected medications vary within and across states. This study found that bronze plans were far less likely than silver plans to cover the 11 products included in this analysis. Results also showed that select drugs identified as the only FDA-approved product indicated for a certain rare disease experienced relatively robust coverage (at least 65% of plans) but often included some form of UM. However, coverage of selected rare disease therapies also is complicated by the fact that plans cover certain products under the medical benefit versus the pharmacy benefit. At the time of this analysis, transparency of medical benefit coverage for these products in exchange plans was limited.Selected medications are most likely to appear on the highest tiers of 4-tier formularies or are not covered at all. Although there are no requirements to designate certain tiers as "specialty tiers," more than 70% of plans in this study use coinsurance for the highest tiers of their formularies. Rates of coinsurance for medications on highest tiers range from 10% to 50% in silver plans and 15% to 50% in bronze plans. Among those plans utilizing copayments rather than coinsurance, ranges of copayments for these select products vary between $20 and $250 per prescription across both silver plans and bronze plans. CONCLUSIONS: This preliminary analysis of access to treatments for patients with select rare diseases revealed the complexities involved for patients with specific needs when selecting a plan with appropriate coverage. For patients with rare diseases, the process of identifying and selecting a plan centers on understanding if and how the plan covers a specific treatment or set of treatments. Access factors will likely vary substantially across plans, as demonstrated by the findings from this analysis. With limited treatment options and the potential for cost sharing and UM barriers, increased data transparency to assist patients in navigating formularies will be a critical step for patients to fully understand their access to needed therapies in each plan.

Managing heterogeneity in prescription drug coverage policies.

Necessity and fairness require that health plans limit the products and services they cover. The basis for these decisions refers to population averages and related population parameters. However, individuals vary and may not be accurately represented by the parameters used to establish coverage policies. Health plans, therefore, are obligated to anticipate and manage heterogeneity among their member groups. This commentary offers considerations for managing heterogeneity in prescription drug benefits.

Greater adherence to diabetes drugs is linked to less hospital use and could save nearly $5 billion annually.

Improving adherence to medication offers the possibility of both reducing costs and improving care for patients with chronic illness. We examined a national sample of diabetes patients from 2005 to 2008 and found that improved adherence to diabetes medications was associated with 13 percent lower odds of subsequent hospitalizations or emergency department visits. Similarly, losing adherence was associated with 15 percent higher odds of these outcomes. Based on these and other effects, we project that improved adherence to diabetes medication could avert 699,000 emergency department visits and 341,000 hospitalizations annually, for a saving of $4.7 billion. Eliminating the loss of adherence (which occurred in one out of every four patients in our sample) would lead to another $3.6 billion in savings, for a combined potential savings of $8.3 billion. These benefits were particularly pronounced among poor and minority patients. Our analysis suggests that improved adherence among patients with diabetes should be a key goal for the health care system and policy makers. Strategies might include reducing copayments for certain medications or providing feedback about adherence to patients and providers through electronic health records.

Comparative effectiveness and personalized medicine: evolving together or apart?

Comparative effectiveness research and personalized medicine can at first appear to be at odds with each other. This research initially compares the overall benefits of one therapeutic approach with those of another for the majority of patients, while personalized medicine identifies the subsets of patients who could benefit based on personal characteristics such as genetics. But because comparative effectiveness research typically enrolls heterogeneous patient populations, it can uncover subpopulations that might benefit most from particular treatments. Thus, comparative effectiveness research can help discern the appropriate role of personalized medicine in improving health care outcomes and rationalizing costs.

Comparative effectiveness research and personalized medicine: catalyzing or colliding?

Comparative effectiveness research (CER) is generating intense attention as interest grows in finding new and better drug technology assessment processes. The federal government is supporting the expansion of CER through funding made available in the American Recovery and Reinvestment Act of 2009 (ARRA) and by establishing the Patient-Centered Outcomes Research Institute through the Patient Protection and Affordable Care Act of 2010. At the same time, personalized medicine is generating debate about its place in clinical medicine, and so, naturally, how CER can or cannot play a role in personalized medicine is part of these debates. At the heart of the debate around the role of CER in personalized medicine is the nature of personalized medicine and how it fits within contemporary clinical research concepts. We maintain in this article that CER can serve to catalyze personalized medicine, but we recognize that, for this to happen, researchers will need to embrace new data sources and new analytic approaches. We also recognize that drug technology assessment processes will have to undergo necessary adaptations to accommodate CER as configured for personalized medicine, and that clinicians will need to be educated appropriately and provided access to decision-support systems through health information technology to use the information coming from this research. To illustrate our argument, we describe two ongoing CER studies funded and managed in the private sector evaluating personalized medicine interventions that have important clinical and financial implications. One of the studies investigates the clinical and financial effects of pharmacogenomic testing for warfarin as prescribed in conditions of typical practice settings. The other study is also set in community practice settings and compares cardiovascular outcomes of patients receiving clopidogrel who are extensive metabolizer phenotypes for the cytochrome P450 2C19 hepatic isoenzyme with all patients receiving prasugrel.

A proposed ethical framework for prescription drug benefit allocation policy.

OBJECTIVES: To present an ethical framework that could aid prescription drug benefit design and to propose that such a framework could be organized around a benefit allocation hierarchy. SUMMARY: Four hierarchical levels of allocation are proposed, as follows: (1) drug categories and subcategories, (2) individual drugs within covered drug categories, (3) specific indications of covered drugs, and (4) special features of drug use, such as quantities covered for specific indications. Rationales for decision making should address the distinct ethical issues apparent at each level. CONCLUSION: Ethical issues associated with designing and managing a pharmacy benefit can be identified by using a hierarchical prescription drug allocation scheme. Articulating these issues is an important first step toward arriving at a fully developed framework that will serve to produce rationales acceptable to typical stakeholders in prescription drug benefits.

An ethical template for pharmacy benefits.

We propose an ethical template for pharmacy benefits and a fair process for using it. The template delineates four levels of decisions about pharmacy coverage, connecting ethically acceptable types of rationales for limits with decisions made at each level. It provides a framework for organizing ethically relevant reasons for coverage (or the tiered copayments). The process for using the template assures accountability for the reasonableness of benefit decisions. It requires transparency and relevance of rationales for limit setting and revisability of decisions, including through fair procedures for appeals. The template and the process facilitate broader public learning about fair limit setting.