D. Ross Camidge, M.D., Ph.D.

Clinical trials establish an unbiased, scientific means to determine the best treatments for specific conditions. But when dealing with a diagnosis of cancer, a suggestion from your doctor that you consider joining a trial can be a source of enormous stress. Understanding more about trials can help alleviate that stress.

Most clinical trials for cancer are in the advanced disease setting. Although there can be trials of anything — from a diagnostic test to surgery to counseling &mdash; most anti-cancer clinical trials involve the development and integration of new drugs. A clinical drug trial can test:

> A new treatment added to a standard treatment.

> A new treatment on its own, or added to standard treatment, compared with standard treatment alone (a randomized study).

Randomized studies may be open-label — where you know which of the available treatments you are getting &mdash; or it may be “blinded.” A blinded study means that you, and probably your doctor, won’t know which treatment you are getting, or if you are even getting a real medicine or a placebo (dummy treatment).

Most trials ask specific questions and do not have much room to bend their eligibility rules. Sometimes eligibility or ineligibility are easy to determine — for example, if a study is designed for people with a certain type or stage of cancer. The three most common reasons for trial ineligibility are:

> the patient has not had the number or type of prior treatments required (either too many or too few)

> the patient is not fit enough to participate, based on physical health or other factors

> the patient’s health insurance does not cover treatment at the center running the trial

To help you decide whether to enroll, all clinical trials offer potential participants a detailed consent form that outlines what is known about the study and any alternative treatments. It describes what being in the study might entail and the risks associated with entering.

Anyone can withdraw consent at any time, although that also means a patient must stop taking any experimental treatment the trial is testing. Most study teams try to be flexible if patients need to miss an appointment or two, but participants are expected to comply with trial rules, including attending when needed and reporting any side effects and missed medication doses — acting like a responsible “test pilots” and accurately letting the “control tower” know how things are going. Patients who fail to comply can be removed from a study by researchers.

If a trial includes elements of standard care, such as standard chemotherapy drugs or routine doctor’s visits or scans, these will be billed to your insurance, and your regular copays will apply. The cost of “extras” associated with the study, such as research-related blood tests, experimental drugs or extra visits to the clinic, are usually absorbed by the sponsor of the study (typically either a pharmaceutical company or an institution working under a grant from the government, a charity or another organization).

What if you need emergency care because of something directly related to the study? That’s something you need to ask about up front. Another thing to clarify is whether you will still be able to receive the medication for free (assuming it is working) if it ultimately gets licensed and enters the marketplace.

What are the potential advantages of being in a clinical trial?

Broadly speaking, there are three main advantages to being on a study:

1. What is learned may help establish the best available treatment for a particular condition in the future, helping others.

2. On a study, you may get access to a more effective and/or less toxic treatment than those that are currently approved. However, it is important to remember that a new treatment may not be better than what is already out there. Also, in a randomized study, you may end up getting the same standard treatment that you would have gotten off-study.

3. Being in a clinical study involves forming a close relationship with a dedicated team of experts focused on your care, with a higher level of personal attention than you would receive in regular care. Some trial participants state that they don’t feel at all like “guinea pigs,” but more like “prize poodles” — with their own entourage of people fussing over them.

How do I know if a particular trial is a dumb idea?

Many people will join a trial to get access to something new, but without being a doctor or a molecular biologist, how can someone know whether new will be better? We should be reassured that clinical trials are very carefully regulated. International guidelines are regularly updated and expected to be followed. Once a trial is written, and before any patient can be enrolled, the design has to be approved by local scientists and ethicists.

However, it is still vitally important to ask your doctor two questions about any particular study:

 How much is known about this new treatment?

A phase 1, phase 2 or phase 3 study design tells you how much is already known about the best way to give the drug, its side effects and whether, and in whom, it might work. It can also give you a sense of the intensity of doctor’s visits that you will experience on the study and the chances that the trial will be randomized. But it doesn’t unequivocally tell you whether the study treatment is going to work for you. Potentially, you could access the right drug for you at any study phase. An important caution is that the center where you are being treated will not have all possible trials available. You can search for trials specific to your tumor type and a particular line of therapy by visiting clinicaltrials.gov. But before you travel, speak to the most knowledgeable physician you can find for help deciding if the trial is likely to represent a true breakthrough and is really worth the trip.

What are my options if I don’t go on this study?

It’s important to understand your alternatives (now and in the future), especially when considering a randomized study. More than almost anything else, randomization — a computer tossing a coin and determining which treatment you will get &mdash; causes patients stress when trying to decide whether to enter a clinical study. Therefore, if a study is randomized, try to clarify your chances of getting the different options; whether you will be told which you have been allocated to up front, at a later date or never; and whether, if you don’t get one option initially, you can “cross over” to get it at a later date. If there is no placebo, just a comparison of two different treatments, it is vital to ask if the “standard” treatment is the same one you would be offered if you weren’t on the study.

If I join a study, how long will I be involved?

In general, your study treatment will continue until either it is proven to not be working for you, until side effects make it intolerable, until you’ve completed a fixed amount of treatment, or until you change your mind and withdraw consent.

Clinical trials are essential for progress, to help each of us know the best treatments for different diseases at any given point in time. Sometimes, the information we generate from being involved in clinical trials helps others. Sometimes, there are general and specific benefits for participants, too. Entry into a trial is sometimes the right thing to do and sometimes not, and that may change over time — in part due to the specifics of the trial, the alternatives available and where you are in your own treatment journey. However, when you find yourself at a treatment crossroads, it is often something worth discussing with an expert.

Without the test pilots and prize poodles (and hopefully not too many guinea pigs) who have gone before, we would still be listening to snake-oil salesmen on the backs of wagons. Progress will come as we all work toward the same goal — doctors, scientists, drug companies, study teams, test pilots and prize poodles all pulling together to make cancer a footnote, rather than a headline, in people’s lives.

D. Ross Camidge, M.D., Ph.D., is professor of medicine/oncology and the Joyce Zeff Chair in Lung Cancer Research at the University of Colorado Cancer Center in Aurora, Colorado. This piece is an abbreviated version of his op ed, “Who are Clinical Trials For: Guinea Pigs, Test Pilots or Prize Poodles?” published by the Bonnie J. Addario Lung Cancer Foundation in the “Navigating Lung Cancer 360 Degrees of Hope Handbook” (2nd edition). The full version is available at lungcancerfoundation .org/handbook/.

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Expert D. Ross Camidge, M.D., Ph.D., offers tips for dealing with the stress of navigating clinical trials for cancer treatment.

D. Ross Camidge, M.D., Ph.D.

Who are Cancer Clinical Trials For: Guinea Pigs, Test Pilots or Prize Poodles?

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