Q. Are clinical trials riskier than FDA-approved drugs?
A. If you are eligible for a particular trial, there is already scientific evidence that the therapy on a trial has as good a chance for success as “standard of care” treatments. In fact, a clinical trial may give you an early opportunity to receive a promising treatment before it is FDA-approved and available in the clinic.
Q. If I start a trial and then feel like it is not the right choice for me, can I stop my participation?
A. A clinical trial is never more important than an individual patient’s care. You can stop your participation on a clinical trial ANY TIME and for ANY reason. While patients have to meet strict eligibility criteria to enroll in a clinical trial, the patient can stop participating at any time.
Q. Who pays for a clinical trial?
A. “When it comes to clinical trials, who pays for it? The “standard care” part in any clinical trial is paid by your insurance company or your third party payer. The investigational part, the part that’s not considered “standard care” whether it be the new drug, whether it be the studies that are done because you are on the new drug, are paid by the study.
Generally speaking, the medical care that standard is paid by your third party payer or should be paid by your third party payer and the investigational stuff, the drug, if it’s not commercially available, is provided for free.”
Q. Who pays for my travel for a clinical trial?
A. “That is something that patients need to discuss with the study staff and their doctors because that changes differently from one study to another. Each study will have different support for patients in regards to extra travel and the extent of the support may be different.”
Q. In a randomized trial, will I be given a placebo, or no treatment?
A. Sometimes patients may be worried that they will be placed on a placebo but most of the studies that are done for myeloma, most of them do not have placebos and the patients are placed on the fully active drug. It’s usually a good question to ask, but there is usually no placebo involved.
A. The question of a Phase III trial is this: Is this new drug better than the standard of care? This is the Phase III study is a trial, where half the people in clinical trial get the new treatment, and half the people get the standard of care. Then you see if the new treatment is better than the standard of care or not. It is the ultimate decision, and it’s going head-to-head against the best that we have. What we’re saying is this new treatment really poised to push us forward, or in the long run does it turn out to be the same?
In randomized trials, patients are either given the “standard of care” or the new drug. There aren’t many times in myeloma when the standard of care is “do nothing.” (or a “placebo” group that gets no treatment. In most myeloma trials, the standard of care is the best we’ve got and in fact there are some Phase III trials where the new drug is randomized against investigator’s choice, which I think is a great trial design. In other words, you have your drug, your very promising drug, and you randomize it against whatever the doctor wants to give that patient. You think you got something better, give it. That’s the trial design. Then you ask, “Is this better than everything anyone else has come up with?”
Q. If my doctor doesn’t talk about clinical trials, does that mean they’re not right for me?
A. Well, that depends. Some doctors don’t get involved in clinical studies or don’t promote them so it could be just an issue with the doctor. On the other hand, often there aren’t clinical trials that are open in the neighborhood near you that might be appropriate to your stage of your cancer. So clinical studies in newly diagnosed myeloma are fewer — there are less of them than clinical studies in relapsed refractory myeloma which are generally open just about anywhere that has an academic center because there’s always a need for the clinical studies in that population group.
It could be either of those scenarios. Either your doctors are not necessarily interested in clinical studies or perhaps if you’re newly diagnosed or doing well with the standard care treatment, perhaps you don’t need to be on a clinical study just yet.
Q. Why participate in a clinical trial?
A. The only way we can make further progress in the treatment of myeloma is by entering new frontiers. It is the mission of university hospitals to move the field forward. To do so, new concepts need to be explored. There are two major groups of clinical trials:
- Entirely new and experimental drugs. These trials are typically performed by phamaceutical companies, because they require large financial resources. They include Phase I ( trying to find the optimal dose) and Phase II trials (trying to see how effective the experimental drug is in a given cancer). Once those questions are answered, the company may want to see if a combination of the experimental drug with a FDA-approved drug is better than the experimental drug alone, or if the new experimental drug (alone or in combination) is better than the standard of care. Most patients participating in Phase I and II trials will not derive any benefit from the treatment, but a lot can be learned from such trials for future myeloma patients.
- Using FDA-approved drugs (no experimental drugs) in a new combination or at a different dosing. These trials are typically performed in university hospitals. Although there is good reason to think that these new combinations will give us better outcomes, there is no guarantee that they will be better. However, the probability that patients will benefit from such treatments is much higher than that of experimental drugs.We still need to learn a lot about what is the best treatment for myeloma. Participating in clinical trials with strict rules and regulations, where everybody is being treated according to a study protocol is the best way to ultimately improve outcomes in myeloma. Patients should remember that participation in a clinical trial is completely voluntary and that they have the right to stop further participation if they feel that the trial is no longer in their best interest.
Q. I have a great relationship with my doctor but should I consider participating in a clinical trial somewhere else if it ever looked promising enough?
A. I think if it was something that appears appropriate to you, then often that’s something that’s quite hard to determine just by looking at the headline for the clinical study. Often there’s a lot of fine print that goes into these clinical studies as to who can go on them and what stage their myeloma has to be at. But I think if you’re interested in enrolling in a clinical study, you can of course always discuss it with your physician and if you’re not satisfied with the response you could always get a second opinion.