QOL FastBlast: Clinical Trials

This is the first in a series of presumably shorter posts about cancer stuff. But don't hold me to that.

I don’t expect this to be a long post, but I’ve thought that before. Mostly I want to say a few things about what clinical trials for cancer actually are because I feel like they are misunderstood by most. Before, I get started, I feel the need to point out out that I am decidedly PRO clinical trial despite what my tone ahead may imply. I just think it’s important to understand them before going all in. You know, like how a science experiment is supposed to work. More on what I mean by that later on.

I think most recognize the basic concept of clinical trials in that they test the effectiveness of new treatments. But that’s not really the full picture.

For all intents and purposes, there are basically three phases to any clinical trial study that matters to most patients… though technically there are four. Here they are:

Phase I: These are small and essentially look to see if a treatment is safe to play around with. Around 70 percent of Phase I trials are successful.

Phase II: These are slightly bigger and look at whether the drug will actually do anything of interest. This is where most trials go to die as only 30-35% succeed.

Phase III: For most people, this represents the big one. Basically a successful Phase IV means a drug gets approval by a national regulating body like the FDA. Only 25-30% of the trials that start Phase III make it out alive.

Phase IV: These are a bonus round of sorts and look at long-term issues AFTER a drug is approved. It’s rare, but sometimes treatments are pulled from the market after thousands of patients have been tracked for effectiveness and side effects over several years. These are often not required. That’s one reason why Phase IV isn’t considered part of The Big Three.

There is such a thing as Phase 0 and even Phase V trials but those aren’t often required and are much more non-standard in structure. Phase 0 trials are very, very small. They test a treatment protocol in a very low dose to see if there’s any promise to someone’s well-educated guess while Phase V is related mostly to marketing and pricing issues.

So what does all of this mean? It means clinical trials aren’t really what most think. Maybe it was just my experience and the experience of nearly every cancer patient I’ve encountered, but it seems remarkably common for patients, caregivers, and muggles to ask about any available clinical trials upon first learning of a diagnosis. That infers that it is generally thought among the general public that clinical trials holds a world of miracle cures just waiting to be had. The ugly truth is that 9 out of 10 trials fail. That’s 90% of promising new treatments that are disproven.

And of the few that get approval, most only improve survival stats moderately. The lack of required Phase IV research explains why I know so many cancer patients who survive their original bout with cancer only to develop other serious health issues (ie heart/organ failure or different types of cancer) years later.

I mentioned earlier that I am decidedly pro clinical trial and made a comment about how science works. One reason to do a clinical trial is that, though rare, miracles do happen. And desperate people are looking for desperate answers. There’s no shame in that.

The other reason to participate in a trial is SCIENCE.

My take (which may not be yours) is that clinical trials very rarely do much to help the individual patients in the trial, but do a lot to help future patients by advancing knowledge and showing realistic odds of desirable outcomes. Over time, multiple trials lead to consequential results beyond a few extra months of survival or decreased side effects. That is absolutely something great and the main reason why I am a fan of trials.

And while we’re at it, can the medical community stop saying that patients have failed a trial? The patients don’t fail. The drugs didn’t do what it was thought it would do. THE TRIAL FAILED THE PATIENT.

Finally, here are my blunt thoughts (again, possibly not your own) on each phase to potentially consider if you or someone you know is looking to be involved in a clinical trial.

Phase I: Whenever I see a headline touting some new miracle treatment that will solve everything and then I read an article discussing a successful Phase I trial, I can’t help but roll my eyes. Yes, that is great news and I really do hope it pans out, but the Phase I bar is kinda low. It’s still important, but a patient is unlikely to garner significant results from a reduced dosage that’s just as much administered to see if something is safe as much as it is to see if the drug does… well.. anything.

Phase II: This is where things get much more serious and results really start to mean something. It is definitely possible that a patient could get positive results for themselves, but that success rate is oh so dismal that I personally wouldn’t bet the farm on it. Still, I take Phase II way more seriously than Phase I trials when I read about them.

Phase III: This is when shit gets real. These are widely administered and already have a significant track record. But, the reason most Phase III trials fail is because they don’t prove to be any more effective or “well-managed” (a comically vague medical phrase) than existing approved treatments. And even if a drug does get approval, that doesn’t mean it’s free of long-term consequences and so…

Phase IV: I personally feel all drugs should go through this. As a society, we are very fortunate that long term survivorship numbers are consistently rising (and thus explaining why I’m not dead yet), so it’s important that patients have more knowledge about what to possibly expect, ya know, if the drugs keep you alive. I think the overwhelming majority would be willing to roll the dice if it meant not dying pretty soon. Give drugs a generous provisional approval but let patients know what’s what if the drug has yet to pass Phase IV. What if a doctor doesn’t have time to explain the subtleties of the process? I honestly don’t care. You will ABSOLUTELY read a future post from me about how making time for better doctor/patient communication is among the highest priorities in healthcare. Both parties need to figure out more ways to make time.

Again, I’m not trying to bum anyone out but I think it’s better to have realistic expectations of what something is really about before going all in with hopes.

Click here if you are in the USA and looking for a potential clinical trial to participate in.