Recently Diagnosed or Relapsed? Stop Looking For a Miracle Cure, and Use Evidence-Based Therapies To Enhance Your Treatment and Prolong Your Remission
Multiple Myeloma an incurable disease, but I have spent the last 25 years in remission using a blend of conventional oncology and evidence-based nutrition, supplementation, and lifestyle therapies from peer-reviewed studies that your oncologist probably hasn't told you about.
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Are Myeloma Clinical Trials Too Good To Be True?
Are myeloma clinical trials too good to be true? Put another way, if you are a MM patient, can you think of a reason for another MM patient to be excluded from a clinical trial that you’re interested in? Have you ever read a clinical trial with a negative finding?
- The average age of a NDMM patient is 69 years of age-
- 80% of NDMM patients are diagnosed with bone involvement-
- 50% of NDMM patients are diagnosed with kidney involvement-
- 95% of NDMM patients are advanced aka stage 2,3
Can you think of a MM patient who should be excluded from a clinical trail that is testing for treatment efficacy that excludes one of the MM patients listed above?
I am a long-term MM survivor and MM cancer coach. Over and over again I find myeloma clinical trials to exclude patients who should be included in the results.
What are the possible negatives of myeloma clinical trials that exclude patients?
- Limited Generalizability:
- Narrower Patient Demographics: Trials excluding certain populations (e.g., elderly, those with comorbidities) may yield results that are not applicable to the broader patient population.
- Bias in Outcomes: The exclusion of diverse groups can lead to findings that do not represent the true effectiveness and safety of a treatment across all patient demographics.
- Missed Opportunities for Advancements:
- Lack of Data on Certain Groups: Excluding specific patients means missing out on understanding how new treatments affect these groups, potentially delaying advancements tailored to their needs.
- Stagnation in Treatment Options: Without inclusive trials, there may be slower progress in developing therapies suitable for a wide range of patients.
- Ethical Concerns:
- Inequity in Access to Experimental Therapies: Exclusion criteria may prevent some patients from accessing potentially beneficial treatments available through clinical trials.
- Undermining Patient Autonomy: Patients excluded from trials may feel their choices and autonomy in seeking innovative treatments are being limited.
- Skewed Safety and Efficacy Data:
- Incomplete Safety Profiles: Excluding patients with certain conditions or characteristics can result in an incomplete understanding of a treatment’s safety profile, as adverse effects may not be observed in the trial.
- Inaccurate Efficacy Measures: Efficacy outcomes may be overly optimistic if only the healthiest patients are included, failing to capture how treatments perform in a real-world, heterogeneous population.
- Impact on Health Disparities:
- Widening Health Inequities: Marginalized groups often face exclusion from clinical trials, perpetuating existing health disparities by not addressing their specific health needs.
- Lack of Tailored Treatments: Failure to include diverse populations may result in a lack of data needed to develop and optimize treatments for these groups.
- Regulatory and Approval Delays:
- Regulatory Hurdles: Regulatory bodies may require additional data from more diverse populations before approving new treatments, potentially delaying market entry.
- Post-Marketing Surveillance Burden: If a treatment is approved based on data from a narrow patient population, extensive post-marketing surveillance may be necessary to monitor its effects in the broader population, which can be resource-intensive.
- Patient and Caregiver Disappointment:
- Limited Options: Patients and caregivers may feel disheartened if excluded from trials, particularly when standard treatments have failed or are inadequate.
- Decreased Trust in Medical Research: Exclusion from clinical trials can lead to decreased trust in the medical research process and reluctance to participate in future studies.
Myeloma is a unique, rare blood cancer. Limiting myeloma clinical trials biases the trial results.
If you would like to learn more about multiple myeloma, email me at David.PeopleBeatingCancer@gmail.com
Thank you,
David Emerson
- MM Survivor
- MM Cancer Coach
- Director PeopleBeatingCancer
Should Cancer Trial Eligibility Become More Inclusive?
“Patients with treatment-refractory cancers who did not meet eligibility criteria for a pan-cancer clinical trial but received waivers allowing them to participate had similar outcomes to patients who participated without waivers, a new analysis revealed.
The study, published online in Clinical Cancer Research, highlighted the potential benefits of broadening eligibility criteria for clinical trials.
“It is well known that results in an ‘ideal’ population do not always translate to the real-world population,” …”Eligibility criteria are often too strict, and educated exemptions by experienced investigators can help individual patients, especially in a last-resort trial.”
Although experts have expressed interest in improving trial inclusivity, it’s unclear how doing so might impact treatment safety and efficacy.
In the Drug Rediscovery Protocol (DRUP), Gelderblom and colleagues examined the impact of broadening trial eligibility on patient outcomes. DRUP is an ongoing Dutch national, multicenter, pan-cancer, nonrandomized clinical trial in which patients are treated off-label with approved molecularly targeted or immunotherapies.
In the trial, 1019 patients with treatment-refractory disease were matched to one of the available study drugs based on their tumor molecular profile and enrolled in parallel cohorts. Cohorts were defined by tumor type, molecular profile, and study drug.
Among these patients, 82 patients — 8% of the cohort — were granted waivers to participate. Most waivers (45%) were granted as exceptions to general- or drug-related eligibility criteria, often because of out-of-range lab results. Other categories included treatment and testing exceptions, as well as out-of-window testing.
The researchers then compared safety and efficacy outcomes between the 82 participants granted waivers and the 937 who did not receive waivers.
Overall, Gelderblom’s team found that the rate of serious adverse events was similar between patients who received a waiver and those who did not: 39% vs 41%, respectively.
A relationship between waivers and serious adverse events was deemed “unlikely” for 86% of patients and “possible” for 14%. In two cases concerning a direct relationship, for instance, patients who received waivers for decreased hemoglobin levels developed anemia.
The rate of clinical benefit — defined as an objective response or stable disease for at least 16 weeks — was similar between the groups. Overall, 40% of patients who received a waiver (33 of 82) had a clinical benefit vs 33% of patients without a waiver (P = .43). Median overall survival for patients that received a waiver was also similar — 11 months in the waiver group and 8 months in the nonwaiver group (hazard ratio, 0.87; P = .33).
“Safety and clinical benefit were preserved in patients for whom a waiver was granted,” the authors concluded…
So, “although the clinical benefit rate of the patient group for whom a waiver was granted appears to be slightly higher, this difference might be explained by the selection process of the central study team, in which each waiver request was carefully considered, weighing the risks and potential benefits for the patient in question,” the authors explained.
Overall, “these findings advocate for a broader and more inclusive design when establishing novel trials, paving the way for a more effective and tailored application of cancer therapies in patients with advanced or refractory disease,” Gelderblom said…
However, “the paper overemphasized efficacy,” said Gyawali, from Queen’s University, Kingston, Ontario, Canada. The sample size of waiver-granted patients was small, plus “the clinical benefit rate is not a marker of efficacy.”
“The response rate is somewhat better, but for a heterogenous study with multiple targets and drugs, it is difficult to say much about treatment effects here,” Gyawali added. Overall, “we shouldn’t read too much into treatment benefits based on these numbers.””