Health Care Reform: 2 Views

First, new medicines come almost exclusively from industry. I trained as a physician and molecular biologist, focused on the translation of science to innovative medicines “from bench to bedside.” It became clear to me early on that the resources and infrastructure to develop new medicines simply do not exist outside of industry.

A 2001 study conducted by the National Institutes of Health found that of the 47 FDA-approved drugs meeting the study’s threshold of $500 million or more in annual sales, none had been developed entirely by NIH and only four had been developed in part with technologies from NIH funding.¹ Furthermore, as the Congressional Budget Office has noted, most of the costs involved in bringing a new drug to patients come not from the initial discovery research but from clinical testing and regulatory submission—the costs that industry tends to bear.

Second, society has seen a healthy return on its investment in biopharmaceutical innovation. Many studies show that advances in medical technology, including biopharmaceuticals, have resulted in significant gains in patient survival, reductions in hospital use and nursing home admissions, and decreases in other healthcare expenditures.² That said, we still need more comprehensive answers and a fuller understanding of the costs and benefits of different treatment options. It’s important to define value not only in dollar terms, but in terms of quality of life and the amelioration of human suffering.

At Amgen, we are aware that we have an obligation to demonstrate the value of our innovative medicines, and collecting outcomes research data is a vital part of our clinical development activities.

Third, the impact of decisions made now will be felt a generation later and will be very difficult to reverse. Many of us or our loved ones have benefited from recent innovations that have slowed the more crippling forms of arthritis, given more time to people suffering from common cancers such as colon and breast cancer, staved off some dangerous complications of diabetes, and successfully fought off viral hepatitis and other infections. We cannot take for granted that medical progress will continue to march forward. None of these advances would have happened without sustained investment and an environment conducive to innovation. Ten or 20 years from now, when we or perhaps our children are faced with a dreaded diagnosis, will effective new treatments be available to us, or will we find ourselves wondering why we have nothing new to offer? This will clearly be a moment of truth for us as a society.

Now is the time to engage in dialog and share our best ideas for solutions that will both improve our healthcare system and allow the life science industries to grow and flourish. Is it possible to have the best of both worlds? I believe it is. In fact, I believe that both must happen together.

I encourage you to seize the opportunity. Contact your Congressional representative and urge him or her to support bills that offer the greatest benefits for patients today and that keep medical innovation alive and well tomorrow.

Sean Harper is Amgen’s chief medical officer.

Science can guide the determination of what works—and what doesn’t—in healthcare.

By Dennis J. Cotter, Mae Thamer, and Yi Zhang

Epogen (epoetin) therapy for chronic anemia among end-stage renal disease (ESRD) patients is the archetype for costly policies formulated without scientific underpinnings.

Before epoetin was introduced, 16% of anemic ESRD patients were treated with blood transfusions. In 1989, the FDA approved epoetin hormone for treatment of anemia in transfusion-dependent dialysis patients because it elevated red cell levels, or hematocrit (Hct), alleviating the need for transfusions. Although the trials were conducted on ESRD patients who were transfusion dependent, and not on the remaining 83%, proponents saturated the ESRD population with epoetin. By 2005, 99% of all hemodialysis patients received it.

Epoetin’s unbridled prescription was coupled with broad overdosing. In 1991, when Congress imposed a fee-for-dose policy on Medicare, it created a perverse financial incentive to overprescribe epoetin, resulting in a threefold increase in dose. By 2007, 43% of patients exceeded the upper bound of the FDA’s approved target Hct, at a cost of $2 billion/year. In addition to Medicare payment incentives, volume-based discounts and product rebates, misguided quality measures fueled overuse of the drug, which has cost well over $20 billion since its introduction.

Now, in a muddled effort to curtail egregious overuse, Congress wants to establish a fixed epoetin payment based on historically high dosages, offering a $1 billion/year windfall to providers and a serious cost concern to taxpayers.

Despite the FDA’s recent removal of epoetin’s quality-of-life (QoL) claim and a Black-Box warning to providers to use the lowest possible dose, Medicare continues to promote the notion that elevation to higher Hct via epoetin (sold by Amgen) means better QoL. By paying for treatment up to Hct 39% and above—despite documented risks at such high Hct levels—Medicare has tacitly eclipsed FDA’s evidence-based safe Hct target range of 30–36%, while doubling costs to all taxpayers. Four recent clinical trial analyses consistently report high risk at higher targeted Hct.^{1 2 3 4} One analysis demonstrated a relationship between epoetin dose and poorer outcomes; high-dose epoetin was associated with a 57% increased hazard of death or an adverse cardiovascular event.⁴ Controversy continues regarding the benefits of its universal use.⁵ To date, no study has reported a safe and clinically appropriate target Hct—a serious safety concern.

The Institute of Medicine’s 2008 report, Knowing What Works in Health Care, admonished, “… The nation must significantly expand its capacity to use scientific evidence to know ‘what works’ in health care.”⁶ The story of epoetin being written today, at the expense of patient welfare and taxpayer billions, illustrates why science must play a central role in policy decisions. The inchoate actions by Congress and Medicare to fix this policy are not evidence based and highlight the need for science in decision-making. If policy makers continue to disregard the warning signs and show indifference to overuse, wasteful costs, and unsafe care of ESRD patients, then there is a distinct possibility that healthcare reform will not achieve its lofty goals.

The authors are at Medical Technology and Practice Patterns Institute, Bethesda, MD.