The National Comprehensive Cancer Network (NCCN) recently released a treatment rating system they call Evidence Blocks for myeloma treatment. These grids add categories and definitions to the usual lists of recommended therapies provided by NCCN. The categories are: efficacy, safety, quality of evidence, consistency of evidence and expense.

Already you can see that there is a lot of complexity—something that would need to be interpreted by a treating physician to come up with a conclusion. But my immediate question is: Does it help the treating physician to provide the best care or better care for the patient? My answer is no, and let me explain why.

First, does this tool address the problem of costs? NO. Costs are too high. The “Evidence Blocks” just acknowledge that. Second, patients avoid recommended treatments because of co-pay or other costs. Do these “Evidence Blocks” help the patient navigate this problem? No. Sometimes patients do need to pay the costs directly. Other times, because of the coverage—health plan or treatment pathway or some other nuance—the costs are not a direct issue for the patient. But this is an individual thing. The MD has to navigate these issues with the patient and come up with what is, frequently, the best compromise: Skip that test. Do another one. Use this treatment for now, try another when you “qualify.”

Bring all parties to the table

Doctors know about costs. Patients, for sure, know about costs. Lowering drug and other treatment costs, such as for diagnostics and supportive care elements—which are equally (if not more) important—is a separate problem. Bringing all parties to the table to reduce costs is drastically needed. This includes doctors and, importantly, patients, as well as insurers, health care administrators, health economists and the like, in addition to the pharmaceutical companies. It is not going to be easy, but somehow or another the insupportable costs have to come down.

But this is a separate issue from the day-to-day management. Assuming high costs persist, how do we help our patients? The “Lists, Categories & Definitions” provided by NCCN really do not help. By systematizing the cost and other types of evidence, they actually make it easier for health care providers to limit access (to the “cheapest” available) and facilitate the development of strict “treatment pathways,” which can severely limit choices and even harm patients.

A snapshot in time

My additional concern is that NCCN has presented a snapshot in time. How will the myeloma treatment landscape change after the 2015 Annual Meeting of the American Society of Hematology (ASH) in December, for example, when we will hear about frontline combinations with or without transplant and comparisons of two-drug combinations versus three-drug combinations? Will there be a rapidly issued update with changes in categories?

What about different opinions? The NCCN team includes many experts. But maybe some other experts have different opinions. Will those opinions be addressed? And if so, when and how?

The problem with the NCCN lists is that they have many options but do not provide any focus on the concerns which doctors and patients deal with day-to-day in decision-making.  For example, the NCCN lists include many options but do not fully distinguish among Category 1 options, which makes it difficult to know which to use. The Evidence Block-approach focuses on tangential issues, including cost, rather than on key patient concerns, such as age, family and work factors, risk (FISH test results ), co-morbidities (diabetes, clotting problems, heart disease, need for many other medications, etc.) and practical monthly costs faced by an individual patient. For some patients, if drug costs are covered, diagnostic testing, being able to work or not and supportive care issues may be much more influential in decision-making.

In other words, the financial feasibility of a specific course of treatment is an individual patient issue, which has to be discussed as part of the selection process—not as part of a preformed grid.

Don’t remove choices based on costs

This is where several current efforts to integrate costs into the treatment paradigm fail (including the “Value Framework” issued by the American Society of Clinical Oncology in June this year). Costs are a separate issue. Forcing sick patients to be advocates to receive needed treatment is not fair. Many will choose to become advocates, perhaps. But for now, most will manage the best they can. I believe we should not remove choices based on costs.

Coverage for agents with more limited efficacy is an important challenge which deserves separate discussions. But for treatments which produce excellent outcomes access should be available. I favor addressing cost issues in the appropriate meetings and settings as an urgent matter, but not in a complex fashion which can negatively impact patients.

Dr. Durie sincerely appreciates and reads all comments left here. However, he cannot answer specific medical questions and encourages readers to contact the trained IMF InfoLine staff instead. Specific medical questions posted here will be forwarded to the IMF InfoLine. Questions sent to the InfoLine are answered with input from Dr. Durie and/or other scientific advisors and IMWG members as appropriate, but will not be posted here. To contact the IMF InfoLine, call 800-452-CURE, toll-free in the US and Canada, or send an email to [email protected]. InfoLine hours are 9 am to 4 pm PT. Thank you.


Dr. Durie -

Let me know if you would like to discuss this topic of NCCN “Evidence Blocks” as a #mmsm Twitter chat in the future.

Mike Thompson

Thanks for the invitation, Mike. The NCCN’s evidence block approach is certainly a topic that patients are concerned about and has the unfortunate consequence of curtailing access to treatment that might be best for patients. Unfortunately, my travel schedule in the near future is such that I’ll have to decline.

Sincerely, Brian Durie.

Dr. Durie,
As patients, we are so grateful for your speaking out about treatment protocols that are skewed by costs or other factors rather than keeping all options on the table with each individual patient and his/her doctor as the focus. Many of us are worried about not being able to obtain the best or possibly only treatment due protocols and calculations set by organizations or insurance companies. How could they possibly know what is best or "medically necessary" for an individual patient? Statistics and averages are no replacement for sound medical guidance that may keep a particular patient alive. Thank you for sharing your wisdom and being our advocate. Many of us are only here today because of you. Our gratitude...

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