You probably know that clinical trials help bring life-saving treatments to patients. But for most people, that’s where their knowledge ends.
How safe are clinical trials? How much do they cost? And just how sick do you have to be to join one?
Jai Patel, PharmD, chief of pharmacology research for Phase 1 trials at Carolinas HealthCare System’s Levine Cancer Institute, spends his days overseeing clinical trial patients and monitoring how they respond to cancer drugs. Here, he gives you answers to common questions about cancer clinical trials.
So, what exactly is a clinical trial?
A clinical trial is a defined way to evaluate the effects of a new medication or combination of medications that haven’t been approved yet for a specific use. Since we’re testing non-approved medications, it’s all done in a highly regulated setting.
You’ve probably heard of clinical trials being different phases – Phase 1, Phase 2 and so on. The different phases refer to what exactly we’re testing and how far along we are in that process.
In Phase 1 trials, we’re testing medicines that aren’t approved yet for a given use, and are often being given to a patient for the first time. The goal here is to look at the drug’s side effects and how people are tolerating it, and to figure out the best dosage. More recently, clinical trials have shifted toward looking at how well the medication works much earlier into the Phase I setting, and even if there are certain factors that predict who might benefit.
Phase 2 trials start with that “best dosage,” and then look at its clinical activity against the cancer in a larger population. In Phase 3 trials, we start to test the new drug against the current standard of care drug to prove that it works better. In Phase 4, we’re testing the long-term effectiveness of the drug over a prolonged period of time in significantly larger numbers of people.
Are they safe?
Because we’re testing drugs in real people, there’s a lot of research that happens long before the patient ever takes a drug. You must have very good pre-clinical (before human) data to show your drug is safe and effective before you start a trial. So, yes, there’s a very high standard of safety that must be met.
That doesn’t mean patients won’t experience side effects on a trial. We’re honest with patients about what the risks are and what we expect them to encounter in terms of side effects. During Phase 1, we often start patients at a very low dose and slowly increase it in a controlled fashion to determine the most appropriate dose that doesn’t cause too many side effects. But often, patients are willing to accept the possibility of side effects because the alternative may be worse, or perhaps no treatment at all.
Are they really expensive?
The good news with clinical trials is that there can be little to no cost for the patient, depending on the trial design. Because most of the drugs aren’t approved yet, drug companies usually cover the costs of the trial. Sometimes patients can even be monetarily compensated for participating in trials.
If there’s anything within the trial that’s considered routine – things that would have been done as part of the patient’s standard of care – that may end up being billed to insurance. But overall, clinical trials can allow patients to get access to groundbreaking therapies at essentially no cost. That’s huge considering how expensive some of these medications are.
How accessible are they?
Traditionally, clinical trials have mostly been offered at large academic centers. That means that a lot of the time, patients do have to travel to participate, which is an unfortunate and unnecessary hassle.
At Levine Cancer Institute, we think patients should have the same access to trials no matter where they live. Our multi-site model lets us offer trials at most of our locations across the Carolinas. We have the physician staff, the research staff and the standardized process to bring clinical trials to many of our locations, so that patients don’t have to travel too far. Soon, we’ll actually be one of the few centers offering Phase 1 trials at multiple locations.
Patients with cancer are already going through so much. They have enough concerns, and travel shouldn’t be one of them.
How hard is it to get into a trial?
The criteria are different for every trial and sometimes it can be hard to get into a trial. Normally, the criteria are set by the drug maker. In general, you have to meet certain guidelines in terms of your age, type of cancer, lab values and organ function.
A lot of people think you have to be out of all options and close to dying to get on a clinical trial. That’s not true! There are many trials being offered – from the very first treatment a patient ever tries to the very last treatment. There are even clinical trials that address other symptoms that cancer patients experience, such as pain, fatigue or loss of appetite. There are a lot of clinical trials out there, and if you have cancer, there’s a good chance there’s a trial that you’re eligible for.