Advances in foundational science, technology, and clinical knowledge are driving a revolution in patient care. Minimally invasive surgery has reduced rates of post-surgical complications, reduced hospitalization, and dramatically accelerated recovery; direct-acting antivirals have brought a cure for hepatitis C; and novel immunotherapies have brought the promise of increased survival to late-stage cancer patients. The list goes on.
At the same time, spending on these innovative drugs and devices has increased dramatically. Between 1980 and 2010, overall personal health care expenditures in the US grew nearly four-fold, driven in part by new technologies. Outside of the US, health care spending per capita is likewise increasing, along with international concern about the cost of new innovations.
The question is, what—if anything—should be done to regulate this innovation? To balance physician prescribing of state-of-the-art drugs and patient desires to access them with the fiscal realities of high treatment costs, many governments have turned to health technology assessment (HTA) bodies to identify “high value” treatments. In the United Kingdom, for example, the National Institute for Health and Care Excellence (NICE) conducts HTA to inform coverage and policy decisions made by the National Health Systems of England and Wales.
NICE measures a treatment’s value by comparing relative costs and benefits of new technologies. Even if a treatment has significant clinical benefits, NICE may decide not to cover it. NICE is perhaps the best-known example of a governmental HTA agency, but many other countries—including Germany, France, Japan, Canada, and Australia—have similar agencies. The United States, however, is conspicuously absent from this list.
The rise and fall of HTA in the US
Ironically, the US was the original HTA pioneer. To inform and improve policy making, Congress created the Office of Technology Assessment in the 1970s to provide objective assessments of “the physical, biological, economic, social, and political effects” of new technologies or their applications. OTA hired a multidisciplinary scientific staff to help in this effort and produced generally well-received reports on key technologies such as the CT scanner. The OTA went on to publish numerous assessments of medical technologies before its closure in 1995 due to political backlash towards its reports.
Other organizations tried to fill the void, especially in health care. The Institute of Medicine (now the National Academy of Medicine) often weighed in with reports on technology assessment, including “Knowing What Works in Health Care: A Roadmap for the Nation,” which called for establishing priorities, funding, and managing systematic reviews of clinical effectiveness.
Today, the US has multiple public organizations devoted to the study and evaluation of health care and health technologies. The US Food and Drug Administration evaluates clinical safety and efficacy. The Agency for Health Care Research and Quality (AHRQ) and the Patient-Centered Outcomes Research Institute (PCORI) study patient outcomes and conduct comparative effectiveness research. Nevertheless, none of these bodies has taken on actually conducting HTA; in fact, these organizations operate under mandates that they should not undertake full HTA.
Why European-style HTA will not work in the United States
The ideals of European-style HTA—to better distribute limited health care resources to maximize health—are unassailable. Its practice, however, is antithetical to several realities of our nation and American health care.
European-style HTA relies on a narrow conception of value
For a country with a single-payer health care system, where decision-making is truly centralized, it may make sense to distill value to a single measure (such as the incremental cost-effectiveness ratio). This allows a national health system to determine a uniform, efficient level of service which everyone in society agrees is the ‘right’ choice (although pressure is emerging to reform this model).
Stakeholders in the US system have much more diverse perspectives on value. Some value unrestricted choice in and of itself; as an example, they will pay additional health insurance premiums to access larger networks with fewer restrictions on care. Employers may place a high value on treatments that get their employees back to work, whereas Medicare may place more value on treatments that reduce risk of complications from aging (like osteoporotic fractures). Patients and families—vital stakeholders—may place greater value on treatments that reduce caregiver burden, such as those that might occur in treatment of Alzheimer’s disease. The point is that the American health system has always recognized that stakeholders may value treatments differently, and efforts to limit those choices with HTA are likely to fail.
European-style HTA has increasingly become a budget tool
Health care is rightly viewed as an investment. Some of the greatest sources of value from a new technology may not arise for years, and a proper assessment must go beyond short-term budgets. Novel treatments for hepatitis C, for instance, have a very long-tail before they pay off, but the returns are very large. When HTA was applied to these products, however, news stories captured headlines about short-term impact, rather than the costs and benefits of possibly eradicating the disease in the United States.
Such public debates are certainly useful as part of a price-negotiation among government payers, health plans, and pharmaceutical companies. However, as a society, a long-term perspective is essential to sustain innovation. Today’s treatments are the result of large investments in research and development, often over decades. To sustain the wave of scientific breakthroughs and clinical innovations that we are currently enjoying, it is essential that the investments in today’s innovation be rewarded.
European-style HTA can adversely affect health and well-being
European-style HTA largely aims to identify a single “best” along a set of criteria, but such an assessment only tells us the best treatment on average for a given patient population. Patients are heterogeneous, and treatments that are high value to some patients may be less valued by others. Clinically, there are potentially serious risks to limiting physicians’ ability to match patients to the most appropriate therapies. Some cancer patients may have a strong preference for the treatment option with the strongest survival outcomes in trials, regardless of safety, while others prefer safer alternatives.
For example, NICE considered nivolumab—a new cancer-fighting immuno-oncology agent—to be clinically effective based on its “ability to increase median survival by almost 3 months” for patients with non-small cell lung cancer. However, NICE concluded that it would “not recommend nivolumab as a cost-effective use of NHS resources” due to the high cost of treatment. In the United States, guideline decisions recognized the heterogeneity in response. Researchers noted that the median response does not capture a more durable response in some patients, and the influential National Comprehensive Cancer Network (NCCN) recommended nivolumab for second-line therapy with their strongest grade of evidence.
Tailoring care to individual patients may also lead to better outcomes. For example, one study finds that limiting the choice of anti-psychotics in Medicaid populations—mental health is a large driver of spending—would lead to more hospitalizations (and more spending).
Clearly, Americans have not embraced a ‘one-size-fits-all’ approach to health care, even if it saves money. That is why we have very different care delivery models, including integrated models such as Kaiser, community physicians and hospitals, and academic medical centers.
European-style HTA gives up on health care markets
At launch, the $100,000 price of Gilead’s Harvoni for hepatitis C brought public and Congressional ire, and calls for price controls. Soon after, other companies—most notably AbbVie—introduced competing products that quickly drove down prices. As prices fell due to competition, real-world information about the effectiveness of this new class of drugs was collected that demonstrated an increasingly strong case for the benefits of treatment.
Vital data often accrues after the introduction of new treatments. As a drug disseminates into clinical practice, physicians learn critical information about which patients respond best to treatment, when and with whom to initiate treatment, and potential complications. This leads to new opportunities for more comprehensive analysis of optimal use of new treatments.
Our understanding of value must be dynamic if prices are to arrive at an accurate approximation of value. Prices vary after launch due to new evidence surrounding treatment value, as well as increased competition, either from other branded products or eventually from generic or biosimilar formulations. In addition, overall treatment value changes in the years following launch as new indications are identified and physicians and clinical researchers learn about the best use of a product for their patient population.
European-style HTA is static compared to these dynamic processes, and it risks harmful restrictions on access to valuable new therapies based on early clinical and pricing information. Because agencies evaluate innovative therapies before making an initial coverage determination, European-style HTA evaluates a new therapy soon after its launch — but this is precisely when we know the least about the product’s likely performance in the real world. With better data and clinical analytics, real world evidence is advancing at an accelerated pace and should better inform a dynamic process.
The future of HTA in America
While European-style HTA is a poor fit for the US, we do need better mechanisms for identifying value and allocating resources to more cost-effective interventions.
For example, we are making progress towards treating Alzheimer’s and dementia. An effective drug would be compelling to millions of patients and their families. On average, the prevented costs of caring for an individual living with dementia over eight to 10 years (the average life expectancy following diagnosis) would be approximately $375,000. For the 5 million people diagnosed each year and the approximately 15 million unpaid caregivers, there are also considerable productivity benefits from curing dementia. Thus, how do we think about value for an efficacious Alzheimer’s therapy?
More importantly, what if such an Alzheimer’s treatment is very expensive? If the debate plays out the way it has for other recent breakthroughs, then the net effect might be to limit access as a result of the immediate financial impact. What we need to do, however, is to figure out how to price such a drug to ensure early and broad access — perhaps with payment tied to intermediate outcomes (biomarkers).
This theme is broadly true for all new technologies. New pricing mechanisms are needed to effectively link prices to value; we need innovation in pricing, not just treatment. Potential pricing mechanisms include outcomes-based contracts and indication-specific pricing, but other approaches are possible, such as selling licenses for pharmaceuticals like we do for software.
Our challenge is to design a system that: (1) measures value appropriately; (2) links reimbursement to that value; and, (3) does so in a way that stimulates innovation in high-need areas. European-style HTA is not really up to the task. Moving forward will be daunting and will require multiple viewpoints, which is why we are helping to lead the Innovation and Value Initiative to advance the field. The result, we hope, will be an approach to technology assessment that reflects multiple perspectives and defines a new role for the United States as an engine of value-based innovation.
Author’s Note
Dana Goldman is a founder, member of the Value and Evidence Advisory Board, and Executive Economist at Precision Health Economics, a consulting firm to the health care industry.
from Health Affairs BlogHealth Affairs Blog http://ift.tt/2czBiEC
No comments:
Post a Comment