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Clinical Trial Uncertainty in Myeloma
Is there clinical trial uncertainty in myeloma? In other words, do oncologists fully understand the risks and benefits of the drugs approved through clinical trials? According to the research below, no. Clinical trial uncertainties go unreported much of the time.
The problem, as I see it, is that oncologists prescribe MM therapies that can cause serious problems or “uncertainties” that the oncologists do not know of. MM patients taking these therapies can experience serious side effects as a result.
MM patients are constantly posting about the side effects they are experiencing, asking their fellow patients if such and such a health problem is “normal.”
The hidden side of clinical trials
I am not criticizing clinical trials. I am criticising the way that clinical trials are conducted.
The solution? Less is more. Take the “control” side of what Vincent Rajkumar calls the Cure vs. Control Debate in Myeloma.
I am a long-term MM survivor. I live with a host of long-term therapy-induced side effects. I wish I had known about Dr. Rajkumar’s debate when I was going through active therapies.
David Emerson
- MM Survivor
- MM Cancer Coach
- Director PeopleBeatingCancer
Trial Uncertainties With New Cancer Drugs Rarely Reported
TOPLINE:
FDA reviewers flagged trial uncertainties for nearly 80% of cancer drugs approved from 2019 to 2022, but few were reported in journals or National Comprehensive Cancer Network (NCCN) guidelines.
METHODOLOGY:
- The FDA approves most new cancer drugs through expedited pathways, which often leave uncertainties about efficacy and safety, such as reliance on unvalidated endpoints, limited follow-up, or approval based on a single trial. Although the FDA describes these uncertainties in its risk-benefit assessments, clinicians rely on journal publications and clinical guidelines, making accurate reporting of uncertainties in these sources critical for informed prescribing.
- Researchers used Drugs@FDA to identify 52 cancer drugs approved by the FDA from 2019 to 2022. By April 2025, journal publications and NCCN guidelines had referenced 51 pivotal trials covering 48 of these drugs.
- The researchers examined FDA review documents to identify uncertainties related to primary outcomes of pivotal trials, focusing on those included in the FDA’s Benefit-Risk Framework, which the agency considers important to its decisions.
- The primary outcome was the proportion of uncertainties reported in publications and NCCN guidelines. The study also assessed whether drugs with more uncertainties had lower evidence ratings and consensus recommendations in the guidelines.
TAKEAWAY:
- FDA reviewers highlighted a total of 94 uncertainties for 38 (79.2%) cancer drugs. Journal publications reported only 22% (21 of 94) of the FDA-identified uncertainties, and NCCN guidelines cited only 23% (22 of 94).
- For nearly half of the drugs, journals (20 of 38) and NCCN guidelines (18 of 38) omitted all FDA-identified trial uncertainties.
- NCCN guidelines recommended 37% (14 of 38) of cancer drugs with category 1 evidence and 58% (22 of 38) with category 2A evidence, while they did not recommend 5% (2 of 38). The two drugs that were not recommended had the greatest number of trial uncertainties.
IN PRACTICE:
The findings suggested that “clinicians may be unaware of important clinical trial limitations identified by the FDA when making prescribing decisions,” the authors of the study wrote. “To improve transparency and clinical decision-making,” they concluded, “the FDA should make its benefit-risk assessments more accessible and user-friendly. Reporting guidelines should consistently require disclosure of key clinical trial uncertainties, and guideline developers should systematically incorporate FDA assessments into their recommendations.”
SOURCE:
The study, led by Avi Cherla, MSc, London School of Economics and Political Science in London, England, was published online in JAMA.
Decoding uncertainty for clinical decision-making
“…Uncertainty can lead to diagnostic errors, including misdiagnoses or delayed diagnoses. Choosing the most effective treatment often involves navigating through uncertain outcomes. Clinicians must weigh the potential benefits and risks of various options, sometimes without clear evidence favouring one approach over another.
Uncertainty can compromise patient safety if it leads to inappropriate treatment choices or missed diagnoses. For instance, prescribing a medication without fully understanding its potential interactions with other drugs the patient is taking can lead to adverse effects.
Uncertainty can drive up healthcare costs due to the need for additional tests, treatments and hospitalizations resulting from diagnostic errors or ineffective treatments….”
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