San Francisco, CA—The National Comprehensive Cancer Network (NCCN) has added a new value criterion to its clinical practice guidelines in the form of “Evidence Blocks” for the evaluation of treatment options. These Evidence Blocks add a new affordability component for available therapies included in the NCCN clinical guidelines.
“NCCN Evidence Blocks will educate providers and patients about the efficacy, safety, and affordability of systemic therapy, starting as a starting point for shared decision-making based on the individual patient’s value system,” said NCCN Chief Executive Officer Robert W. Carlson, MD. These criteria are intended to empower patients “to identify, alongside their physician, optimal treatment based on clinical and economic considerations that are of most value to them,” he said.
The first Evidence Blocks issued in October are for the treatment of chronic myelogenous leukemia (CML) and multiple myeloma, which have been presented at the 2015 NCCN hematologic malignancies annual congress and are included in the most current versions of the guidelines for CML and multiple myeloma. The Evidence Blocks were developed, in part, in response to patients’ requests for “additional information that will allow them to participate in decision-making and make better, more informed choices among therapy options included in the guideline,” said Dr Carlson.
Value for the Patient
“Value has many definitions, and the individual patient definition of value, we believe, is most important,” Dr Carlson said. “Value for one patient may be different than value for another patient.” The Evidence Blocks are displayed on “flash cards” that include the 5 measures for the guideline recommendations, as shown in the Figure. Each of these measures is scored from 1 to 5, with a score of 1 being unfavorable and a score of 5 being most favorable.
“The affordability measure has generated the most discussion among the NCCN experts and other stakeholders,” said Dr Carlson. “Affordability is the NCCN expert panel members’ estimate of the total cost of care surrounding a specific recommendation. This would include drug costs, required supportive care such as antinausea medications, treatment administration, toxicity monitoring, and cost of care associated with management of toxicity.”
In cancer care, the most important value perspective is that of the individual patient, said Dr Carlson. Some patients want an emerging therapy, even when the data are limited, others are most concerned about the expected side effects indicated in the safety column of the Evidence Block, and yet others may be more sensitive to cost, he said.
For example, a woman aged 25 years with high-risk breast cancer who has 2 children will likely focus on efficacy, an 85-year-old woman with the same type of cancer may focus on safety, quality of life, and affordability.
“We view the Evidence Blocks not as an answer, but rather as a conversation starter for shared decision-making between patient and physician,” Dr Carlson said. “They are not a means of restricting choice, or of giving specific, definite answers.”
The new Evidence Blocks list the following primary treatment options for a patient with multiple myeloma who is a candidate for transplant:
- Bortezomib plus dexamethasone
- Bortezomib plus cyclophosphamide and dexamethasone
- Bortezomib plus doxorubicin and dexamethasone
- Bortezomib plus lenalidomide and dexamethasone
- Bortezomib plus thalidomide and dexamethasone
- Lenalidomide plus dexamethasone.
Because short-term and long-term complications are not obvious in the Evidence Blocks, they should only serve as a starting point in the decision-making process, noted Dr Somlo. “In myeloma, even though there is evidence to use the drug lenalidomide after autologous transplants…that essentially doubles the likelihood of survival in patients who get it after transplant, there is a risk for secondary malignancies.”
For a patient for whom preservation of hand sensation is important, “I would have a discussion about using a less neurotoxic agent, with the implication that price has to be in there, because you have to have approval from whatever insurance company to be able to do this,” Dr Somlo added. “If I can justify it based on medical needs, then that regimen would be moved, for that patient, to the preferred one.”
Demystifying Cost Discussions
Cost issues are rarely raised by patients, according to Dr Carlson, because they are reluctant to acknowledge any financial difficulties related to treatment. Physicians hesitate to raise affordability, because it implies that patients who have difficulty paying for treatment may not be offered the most effective therapy.
“The Evidence Block…demystifies the discussion of cost, because the affordability issue is there in front of you, and so it gives people permission to talk about cost and affordability,” said Dr Carlson.
Few physicians believe that they are competent to talk about affordability, he said, “but almost all physicians have financial counselors or resources within their practices that can have that discussion with a fair amount of accuracy in terms of what the out-of-pocket expenses are likely to be.”
The Power of the Evidence Block
Drs Carlson and Somlo cited the new Evidence Block for multiple myeloma to support the primary treatment options over a regimen containing carfilzomib, which has shown impressive survival advantage over other treatment options. They said that phase 3 data for carfilzomib plus lenalidomide and dexamethasone are lacking, relegating this regimen to nonpreferred at the moment, but this could change rapidly.
“It’s knocking on the door, but we want to see the full set of evidence, which is how NCCN works,” said Dr Somlo. “However, when the phase 2 study was published, and clearly showed that this drug is very effective in a salvage setting for controlling this disease—again not survival but controlling disease—that drug made it immediately into a potential choice. Then it comes down to not just evidence-based blocks but toxicity profile.”
Dr Carlson added, “That specific regimen does have the highest efficacy rating, but if you look to the right [of the Evidence Block], you’ll also see that the level of evidence supporting that rating is lower. To some degree, it’s a question of how much confidence do you have to have in the data to make the leap of faith that that increase in efficacy is worth it, or is real.”
More Evidence Blocks Expected
By the end of 2015, NCCN expects to publish NCCN Evidence Blocks for breast, colon, lung, and rectal cancers. The Evidence Blocks for systemic therapies are expected to be available in the complete library of the NCCN Guidelines by the end of 2016.