This news release about a test of combination drug therapy in people newly diagnosed with multiple myeloma blood cancer slathers on hopes and promises, while neglecting caveats, potential harms and essential context. The release neglects to point out that even after this phase 2 trial has been completed, researchers won’t know if the experimental drug cocktail is better or worse than standard treatments, because it does not include any direct comparison. The release puffs up the threat of multiple myeloma, which accounts for less than 2 percent of cancer cases in the US. It does not explain that patients would be responsible for many of the costs of this treatment, in part because of overlaps with standard care. The release does report support from pharmaceutical companies that make two of the drugs to be used.
The lead investigator of this trial, Luciano Costa, MD, gave an interview in which he clearly pointed out much of the important information that is missing from the news release. The interview shows that the researchers are aware of the limitations of the evidence and of their trial. Costa pointed out that patients considering enrolling would be responsible for a share of the costs of treatment. Researchers have a responsibility to ensure that news releases issued about their work give readers a fair summary, not the kind of rosy depiction in this release.
It’s also important to point out that this is a phase 2 trial, which identifies safety and effectiveness of a treatment. The primary endpoint is a surrogate that is not clearly associated with overall survival for this disease.
In some experiments, it may be too early to discuss treatment costs, but this trial involves drugs that are all already approved for other uses, which means their prices are known. Also, while trial participants typically are not charged for experimental drugs, in this case it appears they will be billed for many treatment costs… and there would likely be costs for some (including travel and lodging) that insurance would not cover. In fact, the lead investigator acknowledged the costs when asked in an interview about what patients should know about the trial:
“[T]he study, like any other clinical trials, that our components are considered standard of care and they are charged to the patient or to the insurance. For example, the costs of seeing the doctor, the cost of doing the main test for a traditional disease assessment, the cost of stem cell transplant, but there are components that are considered experimental and are covered by the study, in this case, the daratumumab, the carfilzomib and the MRD testing by itself.”
The release should have also noted these costs.
By definition, the benefits of an experimental treatment are unknown. The release should not dangle the potential of unproven benefits, including eradicating multiple myeloma, without acknowledging that this experimental treatment may turn out to be no better, or even less beneficial, than standard treatments. In the interview mentioned above, the lead investigator noted, “There is no proof that a new therapy is better, otherwise would be no need for the clinical trial.” The release should have included that fundamental caveat.
We think it’s important to stress that a trial to determine the best therapy would ideally compare National Comprehensive Cancer Network (NCCN) recommended treatments such as revlimid, dexamethasone and velcade with a proposed therapy.
There is no mention of risks or harms of the experimental approach, but part of the function of a phase 2 trial is to evaluate harms. Not only does each drug in the experimental cocktail include its own risks (including increased risk of heart attack and stroke, nerve damage, blood clots, low white blood cell and platelet counts, fatal spikes in blood pressure), but the effect of the combination of drugs could exacerbate the harms or even create new ones. While multiple myeloma patients may be aware of and acknowledge the risks of individual drugs as part of standard treatment, the unknowns of the experimental drug combination should have been pointed out in the release.
Although the news release notes that this is a phase 2 clinical trial, it should have pointed out that because the trial does not directly compare the experimental treatment to standard care, another (phase 3) trial will be needed to test whether the experimental approach is better (or worse) than standard care.
The release cites an accurate number of new multiple myeloma cases (more than 25,000/year), but the lead phrase “Multiple myeloma is the second most common type of blood cancer in the United States” creates an exaggerated impression. The American Cancer Society calls multiple myeloma “a relatively uncommon cancer.” The ACS estimates it accounts for about 2 percent of all cancer cases and deaths.
The release reports that this trial is supported by Amgen and Janssen, makers of two of the drugs it uses. (Executives at Janssen may not be happy that the release misspelled the company’s name as “Janseen”.) The disclosure would have been more complete if it had noted that the lead investigator, Luciano Costa, MD, PhD, has also received speaking fees and other personal payments from Amgen and Celgene (which sells another one of the drugs being tested).
The release repeatedly touts the promise that this experimental therapy will prove to be superior to standard treatments, without clearly stating that even after this trial is done the answer to that fundamental question won’t be known, because there is no direct comparison. What’s more, the release highlights problems with and risks of standard care, even as it neglects the potential risks of the experimental treatment.
The release notes that the trial uses drugs that are already available, though some are approved for only patients who have relapsed after initial treatment. It makes clear that the “minimal residual disease” testing regime to be used is not routinely available.
Most readers of this release will get the impression that this trial is unique, when actually several groups are testing the same drug combination. The release highlights the researchers’ plan to use “next generation sequencing technology to detect minimal residual disease” (MRD) and to use those MRD results to guide treatment. There is much discussion of using MRD to monitor response to therapy but it is certainly not ready for widespread use.
Some of the other similar trials also track minimal residual disease, though a quick review of trial descriptions on www.ClinicalTrials.gov does not make clear whether this trial is unique in using the MRD test results to guide treatment decisions or whether this feature is important.
Similar trials include:
A Study of Daratumumab in Patients With Newly Diagnosed Multiple Myeloma
Selinexor and Backbone Treatments of Multiple Myeloma Patients
Aggressive Smoldering Curative Approach Evaluating Novel Therapies and Transplant
2015-12: A Study Exploring the Use of Early and Late Consolidation/Maintenance Therapy
A quote from the lead investigator captures how this release dangles promises that go well beyond the evidence:
“I believe for the first time that we have treatments that are effective enough to make it possible to eradicate multiple myeloma definitively in a substantial proportion of patients, along with having the technology to detect that the disease has been targeted and that treatment can be stopped,” said Luciano Costa, M.D., Ph.D.
In the interview noted above, Costa was careful to note that there is no proof this approach is better than existing treatments. The release should have highlighted similar basic caveats.
This news release about a test of combination drug therapy in people newly diagnosed with multiple myeloma blood cancer slathers on hopes and promises, while neglecting caveats, potential harms and essential context. The release neglects to point out that even after this phase 2 trial has been completed, researchers won’t know if the experimental drug cocktail is better or worse than standard treatments, because it does not include any direct comparison. The release puffs up the threat of multiple myeloma, which accounts for less than 2 percent of cancer cases in the US. It does not explain that patients would be responsible for many of the costs of this treatment, in part because of overlaps with standard care. The release does report support from pharmaceutical companies that make two of the drugs to be used.
The lead investigator of this trial, Luciano Costa, MD, gave an interview in which he clearly pointed out much of the important information that is missing from the news release. The interview shows that the researchers are aware of the limitations of the evidence and of their trial. Costa pointed out that patients considering enrolling would be responsible for a share of the costs of treatment. Researchers have a responsibility to ensure that news releases issued about their work give readers a fair summary, not the kind of rosy depiction in this release.
It’s also important to point out that this is a phase 2 trial, which identifies safety and effectiveness of a treatment. The primary endpoint is a surrogate that is not clearly associated with overall survival for this disease.
In some experiments, it may be too early to discuss treatment costs, but this trial involves drugs that are all already approved for other uses, which means their prices are known. Also, while trial participants typically are not charged for experimental drugs, in this case it appears they will be billed for many treatment costs… and there would likely be costs for some (including travel and lodging) that insurance would not cover. In fact, the lead investigator acknowledged the costs when asked in an interview about what patients should know about the trial:
“[T]he study, like any other clinical trials, that our components are considered standard of care and they are charged to the patient or to the insurance. For example, the costs of seeing the doctor, the cost of doing the main test for a traditional disease assessment, the cost of stem cell transplant, but there are components that are considered experimental and are covered by the study, in this case, the daratumumab, the carfilzomib and the MRD testing by itself.”
The release should have also noted these costs.
By definition, the benefits of an experimental treatment are unknown. The release should not dangle the potential of unproven benefits, including eradicating multiple myeloma, without acknowledging that this experimental treatment may turn out to be no better, or even less beneficial, than standard treatments. In the interview mentioned above, the lead investigator noted, “There is no proof that a new therapy is better, otherwise would be no need for the clinical trial.” The release should have included that fundamental caveat.
We think it’s important to stress that a trial to determine the best therapy would ideally compare National Comprehensive Cancer Network (NCCN) recommended treatments such as revlimid, dexamethasone and velcade with a proposed therapy.
There is no mention of risks or harms of the experimental approach, but part of the function of a phase 2 trial is to evaluate harms. Not only does each drug in the experimental cocktail include its own risks (including increased risk of heart attack and stroke, nerve damage, blood clots, low white blood cell and platelet counts, fatal spikes in blood pressure), but the effect of the combination of drugs could exacerbate the harms or even create new ones. While multiple myeloma patients may be aware of and acknowledge the risks of individual drugs as part of standard treatment, the unknowns of the experimental drug combination should have been pointed out in the release.
Although the news release notes that this is a phase 2 clinical trial, it should have pointed out that because the trial does not directly compare the experimental treatment to standard care, another (phase 3) trial will be needed to test whether the experimental approach is better (or worse) than standard care.
The release cites an accurate number of new multiple myeloma cases (more than 25,000/year), but the lead phrase “Multiple myeloma is the second most common type of blood cancer in the United States” creates an exaggerated impression. The American Cancer Society calls multiple myeloma “a relatively uncommon cancer.” The ACS estimates it accounts for about 2 percent of all cancer cases and deaths.
The release reports that this trial is supported by Amgen and Janssen, makers of two of the drugs it uses. (Executives at Janssen may not be happy that the release misspelled the company’s name as “Janseen”.) The disclosure would have been more complete if it had noted that the lead investigator, Luciano Costa, MD, PhD, has also received speaking fees and other personal payments from Amgen and Celgene (which sells another one of the drugs being tested).
The release repeatedly touts the promise that this experimental therapy will prove to be superior to standard treatments, without clearly stating that even after this trial is done the answer to that fundamental question won’t be known, because there is no direct comparison. What’s more, the release highlights problems with and risks of standard care, even as it neglects the potential risks of the experimental treatment.
The release notes that the trial uses drugs that are already available, though some are approved for only patients who have relapsed after initial treatment. It makes clear that the “minimal residual disease” testing regime to be used is not routinely available.
Most readers of this release will get the impression that this trial is unique, when actually several groups are testing the same drug combination. The release highlights the researchers’ plan to use “next generation sequencing technology to detect minimal residual disease” (MRD) and to use those MRD results to guide treatment. There is much discussion of using MRD to monitor response to therapy but it is certainly not ready for widespread use.
Some of the other similar trials also track minimal residual disease, though a quick review of trial descriptions on www.ClinicalTrials.gov does not make clear whether this trial is unique in using the MRD test results to guide treatment decisions or whether this feature is important.
Similar trials include:
A Study of Daratumumab in Patients With Newly Diagnosed Multiple Myeloma
Selinexor and Backbone Treatments of Multiple Myeloma Patients
Aggressive Smoldering Curative Approach Evaluating Novel Therapies and Transplant
2015-12: A Study Exploring the Use of Early and Late Consolidation/Maintenance Therapy
A quote from the lead investigator captures how this release dangles promises that go well beyond the evidence:
“I believe for the first time that we have treatments that are effective enough to make it possible to eradicate multiple myeloma definitively in a substantial proportion of patients, along with having the technology to detect that the disease has been targeted and that treatment can be stopped,” said Luciano Costa, M.D., Ph.D.
In the interview noted above, Costa was careful to note that there is no proof this approach is better than existing treatments. The release should have highlighted similar basic caveats.
Systematic, Criteria-Driven
Excelente conversación entre @garyschwitzer de @HealthNewsRevu y @marynmck sobre exageración y #COVID19 en el excelente MOOC de @utknightcenter. Vale la pena verlo. https://www.youtube.com/watch?v=SkgXiPojtg4&feature=youtu.be
Words of real wisdom, as usual @garyschwitzer
And: “There were no details, charts, graphs, numbers, nothing published in a journal."
#Pharma chairman:“If you don’t put out data people will say why are you withholding the data?”
Well, yes, that is exactly what people are asking. https://twitter.com/garyschwitzer/status/1264572399523180548
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