Making Hepatitis C a Rare Disease in the United States

By Victor RoyDave ChokshiStephen Kissler, and Prabhjot Singh

New breakthrough medicines for Hepatitis C present an important choice about setting goals and taking systemic action to achieve public health advances in the United States. Despite appearing to offer cure rates greater than 90 percent, high-priced Hepatitis C drugs have driven treatment rationing since their approval over two years ago. Gaps in the screening, diagnosis, and treatment of Hepatitis C pose significant public health consequences.

In May, the Centers for Disease Control and Prevention (CDC) identified Hepatitis C as the leading infectious killer in the United States in 2014—the first year in which new medicines for the disease were available—claiming more lives (nearly 20,000) than 60 other infectious diseases, including HIV/AIDS, pneumococcal disease, and tuberculosis, combined.

However, recent steps have improved the prospects for many patients waiting for treatment. In March, the Veterans Affairs department announced access to Hepatitis C medicines for all patients regardless of disease stage. Last month, New York State Medicaid also lifted treatment restrictions. A newly organized Institute of Medicine committee on viral hepatitis proclaimed in its interim April report that the “elimination of hepatitis C as a public health problem in the United States is feasible.” Notably, the committee stopped short of setting a national goal for the elimination of Hepatitis C, as they continue to study the challenge for their final report due in 2017.

While advances in access to Hepatitis C treatment for some individuals could mark an important shift, we suggest that increased access can be leveraged in the direction of a larger, strategic aim: an explicit public health target.

Target: A ‘Rare Disease’ By 2025

Multiple indicators could be used to define such a target for Hepatitis C, such as deaths averted, percent treated and/or cured, incidence, or population prevalence. We demonstrate one possible approach, using a model published last November in Health Affairs by Van Nuys and colleagues. With a baseline scenario of using older interferon-based therapies, the model considered three treatment scenarios for Hepatitis C with the newest medicines: treat patients with advanced disease, treat all diagnosed patients regardless of disease stage, or treat 5 percent of diagnosed patients across all disease stages. They found that the option of treating 5 percent of the infected population annually would best balance potential benefits with affordability.

Here, we apply this model to a specific time-bounded public health goal: curing 95 percent of all Hepatitis C patients by 2025 to reduce Hepatitis C prevalence below the Food and Drug Administration’s (FDA) “rare disease” designation of 200,000 patients (Figure 1). The Van Nuys model forecasted the consequences of disease progression and transmission dynamics across genotypes and exposure groups (from drug injection, sexual transmission, and blood transfusion) as well as the impact of a cure on those who are uninfected but susceptible.

To realize our rare-disease target, we modeled that all patients in the advanced (F3, F4, decompensated cirrhosis, and hepatocellular carcinoma) stages of disease would be treated in the first two years of analysis (586,947 patients). A progressive strategy for patients in earlier stages (F0-F2) was also required: we began with a baseline treatment rate of 10 percent in the first year with rate increases of 3 percent annually as screening and delivery strategies improve. Realizing these treatment rates will require providing access for patients with earlier stage disease alongside identifying the approximately 50 percent of those chronically infected with Hepatitis C who remain undiagnosed. We believe this strategy is clinically feasible, as demonstrated by the recent announcements by the Department of Veterans Affairs and New York State Medicaid to expand access.

Though this target would not end all new transmissions, it would set the country on a path towards elimination and significantly reduce the morbidity and mortality from Hepatitis C. In comparison to our suggested scenario, treating only advanced patients would lead to a declining but persistent epidemic.

A time-bounded target carries two central advantages. First, it can spur measurable action and accountability to reduce avoidable mortality and morbidity from Hepatitis C through promising medicines. In April 2015, Gilead Sciences and the CDC announced a joint partnership in the country of Georgia for the elimination of Hepatitis C where Gilead’s drug Harvoni would be provided to patients at no charge. Over the past year, Egypt and Scotland have also launched Hepatitis C elimination-oriented efforts. These examples indicate that rather than being fixed, price and cost considerations can be responsive to public health priorities.

Second, it can better direct further resources in service of a strategic population health goal. Since the beginning of 2014, US health systems have spent over $25 billion on Hepatitis C treatments.

Overcoming Three Central Barriers

Making Hepatitis C a rare disease by 2025 is an ambitious goal and requires overcoming three central barriers related to pricing and payment, delivery coordination, and vulnerable groups.

Pricing And Payment

This target-driven approach would shape the parameters of an important debate on options for drug pricing and payment. While the new Hepatitis C medicines have been deemed cost-effective compared to prior, patented therapies with poorer outcomes, the high prevalence of the disease means that population-level spending is still a major obstacle particularly for public payers. Under one approach to address this challenge, proposed in the May issue of Health Affairs by Amy Kapczynski and Aaron Kesselheim, the federal government would use an existing “government patent use” law to procure generic versions of patented drugs. In exchange, patent-holding companies would receive royalties to compensate for research and development.

Another approach could involve state-based coordination for purchasing medicines, with the creation of a voluntary Medicaid negotiating pool to increase volume-based discounts. Any funding plan must balance achieving public health goals for Hepatitis C, limiting opportunity costs in other areas of health and social spending, and determining fair remuneration to manufacturers.

Delivery Coordination

Gaps in the treatment cascade for Hepatitis C between testing and cure could be addressed by a scale-up strategy coordinated by multiple stakeholders including the Centers for Medicare and Medicaid, Veterans Affairs, state-based payers and prison systems, and the CDC. This strategy would pair “treatment-as-prevention” approaches that combine testing, linkages to care, and availability of medicines with “prevention as prevention” programs, including harm reduction programs such as needle exchange, particularly in settings of recent outbreaks. Newer pan-genotypic drug regimens could also lead to simpler “test and cure” models of care delivery and accelerate scale-up.

Vulnerable Groups

Vulnerable populations disproportionately affected by Hepatitis C—including those also infected with HIV/AIDS, people who inject drugs, and incarcerated populations—must move from the margins to the center of any strategy. Strategies such as opt-out Hepatitis C testing in prisons and population-based case finding (that is, targeting resources at groups who are at risk for the disease), when linked to treatment, can help break the chains of transmission and drive deeper reductions in incidence and prevalence.

Approximately 30 percent of all Hepatitis C-infected persons in the United States spend at least part of the year in correctional facilities. Yet three quarters of state prisons offer no screening or targeted testing of inmates reporting high-risk behavior, thereby missing many potential patients.

In sum, we suggest working backwards for Hepatitis C: defining a specific public health target to drive appropriate financing options and delivery coordination solutions. We recognize that these steps will raise vexing questions about the role of private firms in helping realize a social good. But addressing these questions can also set us on a trajectory that will secure public health gains that are rarely so achievable as with these new, curative therapies.

Figure 1: Hepatitis C Prevalence Under ‘Treat Advanced Only’ Vs. ‘Rare Disease By 2025’ Scenarios

hepatitis c prevalence

Authors’ Note

We are grateful to Will Shrank, MD (CVS Health), Charles Roehrig, PhD (Altarum Institute), Abdul El Sayed MD PhD (City of Detroit), and Anna Stapleton (Arnhold Institute for Global Health) for their insightful comments on an earlier version of our post.