Why joining a clinical trial is a gift to future generations
I’ve joined a clinical trial as a gift to future generations.
A potential bonus is that I might benefit, although the chances of this are low and the downsides in the form of side effects are significant. But I feel the pull to put my body and my data on the line for the greater good.
Let me explain.
New drugs and therapies come to market because millions of people have been willing to participate in medical trials testing new treatments.
These trials proceed in several ‘phases’ to ensure the new drug or medical device does no harm and has proven efficacy.
We know, right? The always breaking news is the latest on the ups and downs of taking up COVID19 vaccines. And the emerging successes have just produced a rush-order for silver-bullet protection against a virus that we haven’t faced before as a global community. Stand by for more headlines.
Every vaccine is being subjected toseveral layers of testing, peer reviewed research items, together with ratification by global and local government, including agencies like the WHO, CDC and other expert bodies.
Briefly, Phase one trials assess how a drug is metabolised, excreted and tolerated, and whether it has any toxic effects on human health.
Maybe the best illustration of why we need phase one clinical trials is the example of thalidomide — a 1950s drug prescribed for nausea during pregnancy — which caused more than 10,000 birth defects worldwide. Thalidomide wrought havoc because testing for ill effects of the drug weren’t detected when it was released to the market.
Phase 2 trials gather preliminary information about the efficacy of a device or drug based on testing in a small group of people.
Phase 3 trials, which are usually randomised control trials, often need up to 3,000 or more patients to compare a new drug or device with the current best available therapy or standard of care.
These trials are done to gather evidence about the effectiveness and safety of interventions and they’re essential in drug company applications to regulatory authorities like the US Food and Drug Administration before drugs can come to market.
But better drugs and therapies have been bought with the lives and suffering of millions.
The best available evidence reveals that new treatments tested in Phase 3 randomised control trials improve patients’ outcomes barely more than 50 per cent of the time compared to existing treatments, and few are substantially better.[i] [ii]
The finding that patients who receive new treatments do little better than those on existing therapies includes data on both morbidity and mortality outcomes. That is, regardless of whether we look at results measuring quality of life or the survival of sick or terminally ill patients assigned to new treatments tested in clinical trials, the results are barely better than established therapies.
What’s more, this conclusion is based on the average result from the analysis of 860 published and unpublished Phase 3 randomised control trials performed by academics and pharmaceutical companies over the past 50 years.
The results of any given randomised control trial are impossible to predict. In fact, the ethical underpinning of these trials is that they should be done only when there are genuine uncertainties about the relative merits of alternative treatments.
Therefore, sick or terminally ill people who are given an opportunity to try a new treatment as part of a clinical trial have no way of knowing their fate. And their chances of seeing an improvement in how they feel or of living longer are actually far less than 50 per cent.
Why? Because the finding that Phae 3 three medical trials improve patients’ outcomes in barely more than 50 per cent of cases is based on the average result of many hundreds of trials.
For example, while five decades of controlled experimentation has seen cure rates for childhood leukemia improve from almost zero to more than 80 per cent, only two to five per cent of any individual trial of a new treatment provides a real life-saving breakthrough.[iii]
Which means this: if you, a loved one or a friend think you want to join a clinical trial of a ‘hopeful’ new wonder drug, think again.
DREAMM 7
I’m participating in a clinical trial called DREAMM 7. Great name, right? DREAMM 7 is a phase 3, randomized study designed to evaluate the safety and efficacy of belantamab mafodotin (Arm A: B-VD) in combination with bortezomib/dexamethasone versus daratumumab in combination with bortezomib/dexamethasone (Arm B: D-VD) in participants with relapsed recurrent multiple myeloma.
Some 478 participants are being randomised to Arm A or Arm B of the study. Treatment continues in both arms until progressive disease, death, unacceptable toxicity, or the end of study, whichever comes first.
Belantamib mafodotin is an antibody-medication combination. This means that the antibody part of the medication identifies and then binds to multiple myeloma cells and then the medication part of the drug kills the targeted cells. B-cell Maturation Antigen (BCMA) is a protein on the surface of multiple myeloma cells that the antibody binds to. Belantamib mafodotin targets BCMA to find and then destroy myeloma cancer cells.
It’s an experimental treatment being tested in DREAMM 7 and other studies. It’s not currently is an approved treatment for multiple myeloma in Australia and has a long list of possible negative side effects. In many years’ time, the study’s outcomes will tell us whether it can improve outcomes in multiple myeloma patients.
Daratumumib is an approved cancer medication indicated for patients with multiple myeloma. Like Belantamib mafodotin, it is an antibody-medication combination that targets a protein called CD38, which is found on the surface of multiple myeloma cells. Once daratumumab attaches itself to the cells expressing CD38, it summons the body’s immune system to attack and destroy those cells. Like Belantamib mafodotin, it has a long list of possible negative side effects.
So joining a clinical trial is really a gift to future generations. There’s little upside for the individual and possibly a lot of downside — the biggest of which are side-effects of an experimental drug or device, and the avoidable and sometimes catastrophic effects of peddling false hope.
False hope says hey, thanks for all you did. Thanks for putting your body on the line. We appreciate you making the long trips to the clinic for years on end. And for enduring the protracted waiting times. And for filling in the forms and dealing with the waivers and the legal fine print. And suffering the side effects of a new drug we are only beginning to understand. But we’re sorry to say it didn’t work. You’re still sick, you’re still terminal, and your precious time is running out.
Dan Gaffney is a teacher and author. His book and podcast series, ‘Journey Home — Essays on Living and Dying’ was published in 2019
[i] Djulbegovic, BJ et al (2012). New treatments compared to established treatments in randomized trials. Cochrane Database Syst. Rev, 10: MR000024
[ii] Djulbegovic, BJ et al (2013). Treatment Success in Cancer: Industry Compared to Publicly Sponsored Randomized Controlled Trials. PLoS ONE, 8, e58711
[iii] Djulbegovic, BJ et al (2012). Op cit
