The Case for 'Safe' Step Therapy

David J. Kerr, CBE, MD, DSc


May 02, 2022

This transcript has been edited for clarity.

I'm David Kerr, professor of cancer medicine at the University of Oxford. I'd like to talk today about a rather provocative article that was published recently in The New England Journal of Medicine.

It had a rather strong focus on the US health care system, as is so often the case with the New England Journal, but I think there are broader lessons that we might learn and apply to how we deliver cancer care in low-, middle-, and of course, high-income countries.

The article was about how medical care is delivered in the United States and a particular set of circumstances in their insurance system for delivering care called "step therapy." It was also about a current push for legislation around "safe step care," which is an interesting juxtaposition of words.

Step therapy is a utilization management strategy where insurers, or those who are in control of paying for healthcare, implement tiered treatment pathways.

Patients and their physicians need to seek approval for restricted — that is, expensive — therapies. But they can only do this if they've documented that they've tried the various different levels of simpler treatments before they can apply for, and one would imagine, be granted access to the expensive medicines. Of course, all of these management practices stem from the fact that, as a society, we've been unable to deal with how we provide wider access to innovative, expensive medicines.

We do it in the United Kingdom in our socialized healthcare system using a health technology assessment group called the National Institute for Health and Care Excellence (NICE), in which we make a value proposition. We look at the cost and the benefits of new drugs, and we put that into a transparent model that allows us to work out relative cost-effectiveness to compare the benefits and disbenefits of cancer drugs vs hip replacements vs new imaging technologies vs new medical devices, so we can speak a common language and compare the societal benefits of each of these different interventions.

Step therapy is interesting, though. If one were to be assured that we could link step therapy to evidence-based guidelines, I don't see a problem with that at all, nor do the authors of the article in The New England Journal of Medicine. We would argue that step therapy could promote improved quality of care when it's used to steer patients toward evidence-based medicine and guideline-associated treatment.

One of the problems that they've found, though, is that when they've looked at the payment structure and how access to these restricted expensive medicines works — not only cancer cases, but across all of medicine — in as much as a third of cases, the tiered steps don't seem to map to evidence-based guidelines at all. Again, that may be a particular problem of the sometimes perverse incentives in the US health care system.

The idea of tiering different steps and levels of treatment seems sensible to me, and I think it could work very well. What's interesting, though, is that the article in the journal talks about new legislation around "safe step" medicine — variations to step care that should be legislated to protect the human rights of the individual patient. There were some quite interesting exceptions that have been built into this legislation.

One exception is based on demonstrating that required treatments have previously been ineffective.

Another is that delay of effective treatment would lead to severe or irreversible consequences; therefore, that would protect patients from harmful delays. This seems a no-brainer, but they've had to legislate for it.

[Another is if] required treatments are contraindicated. That, of course, would promote patient safety. Drug labeling would offer an established way for identifying contraindications. One would have thought that physicians would have been aware of these in the first case.

An exception can also be granted if the required treatments would prevent the patient from performing normal occupational responsibilities or activities of daily living. Therefore, this would promote the patient's quality of life.

This article made me think about how we deliver cancer care in a setting in which sometimes access to very basic care is unavailable or patchy or difficult to get a hold of. When we're seeking to make innovative medicines available to all strata of society, to all countries, to those who are rich and poor, the idea of this stepwise progression linked to evidence-based guidelines seems quite attractive to me.

We've seen many of the important guideline groups now incorporating some element of cost into their analysis of the benefits and disbenefits of any treatment. We see this with both NCCN and ASCO guidelines. ESMO has done its own work in terms of trying to bring a financial aspect so that we might better understand how we can deliver innovative cancer care cost-effectively to a wider spectrum in the community of patients that we see.

Have a look at the paper. I think it's thought-provoking. As I say, look beyond the immediate focus on the intricacies of the US health care system and consider its wider implications. As always, I'd be really grateful for any comments or feedback that you might have.

Thanks for listening, as always. For the time being, Medscapers, over and out. Thank you.

David J. Kerr, CBE, MD, DSc, is a professor of cancer medicine at the University of Oxford. He is recognized internationally for his work in the research and treatment of colorectal cancer and has founded three university spin-out companies: COBRA Therapeutics, Celleron Therapeutics, and Oxford Cancer Biomarkers. In 2002, he was appointed Commander of the British Empire by Queen Elizabeth II.

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