Is the "Best Therapy" Always Necessary Even If the Patient Can't Afford It?

Maurie Markman, MD
Published: Saturday, Oct 29, 2016
Maurie Markman, MD

Maurie Markman, MD

In a most provocative commentary published in a recent issue of The Lancet, two authors present a strong argument that it is sometimes ethical to provide therapy that is known to be validated but less effective than the standard of care if this therapy is also so significantly less expensive that access is substantially enhanced.1

The specific point being made in this analysis is that, if affordability is not taken into consideration, then neither the more nor the less effective strategy will be employed in the population in question since the resources required for the more effective—and more expensive—approach will realistically simply not be available.

Not surprisingly, the specific example discussed by the authors of this commentary was the use of antiviral therapy in the management of HIV/AIDS in developing world countries where the current costs of the most effective medications are unquestionably prohibitive both at the individual patient level and for certain countries as a whole.1

But what does this interesting and possibly ethically distressing discussion have to do with the United States, the richest country in the world?

Today, the objective answer is preciously little since, despite increasing evidence for the lack of reality in the rhetoric, it continues to be argued that patients with cancer should receive the “best therapy”—for example, as approved by the FDA—regardless of cost. That families are increasingly facing financial meltdown due to insurance co-pays or exceeding a lifetime drug benefit allowance seems to have continued to escape the notice of those who hold this viewpoint. However, the future regarding the costs of innovative pharmaceuticals often appears nothing short of bewildering and potentially catastrophic.

Examples of High Costs

Consider, for example, a recent report examining the potential impact of an exciting new class of agents, PCSK9 inhibitors, in the management of patients with familial hypercholesterolemia or atherosclerotic cardiovascular disease.2 This therapy recently received regulatory approval and existing data suggest the potential for widespread use of this approach.

In examining the cost effectiveness of this strategy, the investigators conclude that “at 2015 prices, PSCK9 inhibitor use in all eligible patients was estimated to reduce cardiovascular care cost by $29 billion over 5 years, but drug costs increased by an estimated $592 billion (a 38% increase over 2015 prescription drug expenditures).”2 To be clear, that is not a typo—that is the estimated size of the increase in societal expenditures for this single class of drugs.

What new wonder drug for one (or more) of a number of common chronic conditions is on the horizon that will subsequently trump even this stunning degree of cost escalation?

In the oncology sphere, there also appears to be absolutely no moderation in the rapidly escalating costs of novel antineoplastic therapy approved by the FDA based on the results of well-conducted clinical studies. One might hope that reason would somehow prevail and the government might intervene in some manner, such as increasing competition among biotech/pharma companies, reducing costs/timelines associated with drug development, limiting the ability to delay generics/biosimilars, or permitting the FDA and the Centers for Medicare & Medicaid Services to consider costs in their deliberations.

Today, however, there does not appear to be any realistic expectation that the situation will change substantially anytime soon despite increasingly strident rhetoric to the contrary.

What Can We Afford?

So, is it perhaps time to begin to inquire, even in the United States, if our society can afford the so-called evidence-based “most effective” anti-cancer therapy—at least in light of the continued profound paralysis of the government, with its complete inability to come to grips with this escalating fiscal disaster.

Perhaps we will truly be forced as a society to begin to deal with the fact it is no longer possible to accept the statement that we must be willing to pay whatever price is demanded for all strategies determined to be “most or more effective,” and to consider alternative interpretations and solutions for what has been observed in clinical studies.

For example, would it be irrational to elect to deliver novel antineoplastic agents in sequence, based on the observed response or toxicity in the individual patient, rather than in combination, if that approach would result in a 50% reduction of the nondiscounted cost of more than $300,000 a year to an insurer, employer, health system, or patient?

Further, is it reasonable to consider making this decision even if the company-sponsored study that revealed that the utility of using two drugs was only conducted as a combination rather than in sequence? Or, perhaps, if “Drug A” delivered at 10 mg/kg is shown to be statistically significantly better with a modest improvement in progression-free survival (HR, 0.78; P <.05) than when administered at 1 mg/kg, but the price of the agent is now $80,000 a month rather than $8000 a month, a decision might need to be made to only pay for the lower cost “less effective” but still clinically beneficial strategy.

Clearly, what is described above is unpleasant, and it must be hoped that the multiple involved parties will somehow finally bring reason into the current era of stunningly irrational pharmaceutical pricing. But if that does not happen—and happen soon—there may be no choice but to consider alternative approaches such as those briefly highlighted in this commentary.
Maurie Markman, MD, editor-in-chief, is president of Medicine & Science at Cancer Treatment Centers of America, and clinical professor of Medicine, Drexel University College of Medicine.


  1. Persad GC, Emanuel EJ. The ethics of expanding access to cheaper, less effective treatments. Lancet. 2016;388(10047):932-934. doi:10.1016/S0140-6736(15)01025-9.
  2. Kazi DS, Moran AE, Coxson PG, et al. Cost-effectiveness of PCSK9 inhibitor therapy in patients with heterozygous familial hypercholesterolemia or atherosclerotic cardiovascular disease. JAMA. 2016; 316(7):743-753. doi:10.1001/jama.2016.11004.

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