I have been spending time identifying highly qualified and reputable resources in health care. My business is an independent health advocacy for people that want to be proactive in their health planning. During this initial period of resource exploration, I have encountered several true ‘best of class’ health care individuals. I have been so impressed with their knowledge and philosophy on improving health and treating disease, that I want to share with anyone who will listen. I have asked several of these individuals to participate in a Q&A session. Here is Part One of the Series entitled, ‘Wisdom for Better Health.’

D. Ross Camidge qualified in Medicine from Oxford University in the UK, with a PhD in Molecular Biology from Cambridge University. He trained in both Medical Oncology and Clinical Pharmacology and is an expert in the development of new anti-cancer drugs. He joined the University of Colorado in 2005, initially as a Visiting Professor and then as full-time Faculty from October 2007. He is the Clinical Director of the Lung Cancer Program, as well as being a specialist Attending Physician within the Developmental Therapeutics Program, at the University of Colorado.

(Mary) You and your team at UCCC are performing groundbreaking work in regards to personalized medicine based on the type of cancer and the molecular structure of a specific patient. 99.9% of all humans have the same genetic make-up. However, that .1% makes the difference in how we each respond to drugs. More specifically, your work with lung cancer has made vast improvements over the past several years in which some patients are able to live with the cancer as a chronic illness, sometimes for years. I only wish that my family had access to that knowledge and the corresponding tests and treatments when we were dealing with my mother’s lung cancer. In our situation, we knew how to research the treatments and we were actively seeking answers. Unfortunately, the expert advice and treatments weren’t available to us locally. We did the best we could with the resources available to us locally, but it just wasn’t enough.

Question #1:  What advice do you have for a newly diagnosed cancer patient in regards to receiving the most up-to-date treatment options available?  As I have heard you say before, while everyone with a diagnosis of cancer wants to get the best treatment, how does anyone know what the ‘best’ is?

(Dr. Camidge) Being cared for in a center where the doctors have expert knowledge of your disease and a large palette of available studies to choose from in order to select the best drug for you at each line of treatment is something to be strongly considered. There are many good generalists, who can deliver standard therapies close to your home. However, if you want to have access to the latest developments in care, that may only be available in clinical trials, you may wish to seek out a big center. Ideally, you should work directly with an oncologist that specializes in your specific type of cancer. Often the specialists who are really at the cutting edge can be found in National Cancer Institute – designated comprehensive cancer centers. These centers receive their designation due to scientific excellence. They provide leadership for clinical trials worldwide as well as provide advances in healthcare to the public. Also, if a cancer center is associated with an academic institution, there may be a stronger focus on research as well as new technologies and treatments. There are approximately 40 NCI comprehensive cancer centers located throughout the U.S. You can visit www.cancer.gov to locate one that is closest to you.

(Mary) When it comes to clinical trials, it seems that some doctors are well aware of key trials that affect their patient base, and unfortunately, like in any business, there are doctors that are not well-informed.

Question #2: What are the potential advantages of being in a clinical trial and how can a patient find the one that is most appropriate for their situation?

(Dr. Camidge) Your physician will not know every trial that is going on around the country, so it’s perfectly acceptable to do some homework in your own time and ask your doctor’s opinion on the different studies. You can review current trials at www.clinicaltrials.gov . It offers a complete search engine to allow you to narrow down to your particular tumor type and line of therapy.

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

1)      The evolution of new knowledge that may help others know what is the best available treatment for their condition in the future.

2)      An individual on a study may get access to a better (more effective or less toxic) new treatment than is currently available outside of the clinical study. However, it is important to remember that a new treatment may NOT be better than what is already out there (otherwise we wouldn’t need to do the trial to prove it). It is also important to remember that, if you are considering a randomized study, you may end up getting the same standard of treatment that you would get off-study and not getting the new treatment at all.

One key thing about randomized studies – And I cannot emphasize this point enough. Ask your doctor, ‘If I don’t go into this study, what would you treat me with?” If it’s very different from the supposed ‘standard’ you might be getting in the randomized trial, this might make you think twice before accepting the study. Simply put, some studies are using yesterday’s standard not today’s because of when they were written. Only when you have asked this question (and are happy with the answer!) can you truly weigh the pros and cons of being entered into a randomized clinical study.

3)      Being in a clinical study involves forming a close relationship with a dedicated team of experts focused on your care that may bring many general health benefits – such as having a larger number of named individuals to contact for help or advice, or spotting and acting on other conditions, symptoms or side-effects earlier than might happen with standard medical care. For this reason, I clearly recall one trial participant commenting that in her clinical trial she didn’t feel at all like a guinea pig, but more like a prize poodle – with her entourage of people fussing over her and making sure everything was just as good as it could possibly be.

Question #3: Is it free to participate in a clinical trial or are there out-of-pocket costs that will be incurred by the patient?

(Dr. Camidge) In general, the payment of costs associated with most clinical trials tends to follow the same basic principles. Firstly, if the trial includes elements of standard care – for example, standard chemotherapy drugs in addition to, or as an alternative to, any experimental drugs, routine visits to the doctor or routine scans to assess whether the treatment is working – these will be billed to your insurance. If you normally have co-pays for these things then that will not change. For ‘extra’ things associated with the study – research blood tests or research scans, any experimental drugs, even any extra visits to the clinic – usually these are not billed to your insurance but are absorbed by the sponsor of the study. Some insurance programs will not cover any aspects of clinical trials. However, this is the exception rather than the rule. If it happens, sometimes your doctor can explain matters to your insurance company, sometimes they can’t. One thing that is important to ask is if you need emergency care because of something directly related to the study – from an extra visit to your doctor to address side effects, to admission to hospital because of the severity of these side effects – would this care be perceived as standard, or as study-related costs. The other thing to clarify is that, if you are receiving benefit from continued use of the study drug, you will still be able to receive the drug for free, even if it ultimately gets licensed and other people starting on it are then being billed for it.

(Mary) I have always thought that participating in a Phase III clinical trial was better than being in a Phase I or II trial. I assumed that if a drug was being tested, the drug manufacturer improved the drug over the course of each phase based on participants’ responses.  However, I have heard you say that the drug does not change at all from Phase I to Phase III. Furthermore, participating in a Phase I trial may be just as effective for the right person instead of waiting for the Phase III trial.

Question #4: Please explain the differences between each phase so a potential candidate understands what to expect?

(Dr. Camidge) The Phase of the study really only tells you how much is already known about the drug and the level of intensive investigation/extra visits/extra tests and/or the chances of the study being a randomized study. It doesn’t tell you if a drug will work or not and an expert physician may seek out the best drug for you in studies of any Phase.

All drugs, when they are first given to humans, have to explore the correct dose to give – either on their own or in combination with other drugs – these dose-finding studies are called Phase I studies. Because they happen early on in the life of a new drug, there are more unknowns than in later Phase studies, and they are, by definition, the most experimental of studies. Phase I studies tend to be open to anyone with any type of advanced cancer at any line of therapy. Participants have to be fairly fit to enter Phase 1 studies in order to cope with the unexpected. Also, the number of study-specific visits and tests tend to be more than in any other Phase of clinical studies.

Once a Phase I study is complete, the drug – at the doses determined as appropriate to take forward based on the results of the Phase I study – is then explored in a series of Phase II studies to get a good feel of its activity in different cancers at that dose. If you started on the Phase I study and the drug is working for you, you stay in the Phase I study. It is the drug that expands to start a new study, not you. Phase II trials focus on answering the question ‘does it work in a specific cancer?” Within Phase II studies all patients receive the same dose of drug and, because more is known about the side effects and tolerability of the drug, the fitness requirements for entry tend to get more relaxed and the number of study-specific visits and tests also get less. However, at this point the manufacturer of the drug is starting to look for a specific license for the drug, so Phase II studies are usually restricted both by tumor type (there may be several parallel Phase II studies, each in a different tumor type) and by line of therapy.

Once a drug has (a) its dose determined from a Phase I study and (b) some signal as to which tumor type it might work in from the Phase II studies, in order to get a license from the FDA, it has to be shown to be at least as good or better than what is already available for treating a particular cancer. This kind of large comparative study, almost always randomized against some current standard treatment, is called a Phase III study.

The question that the Phase III study focuses on is ‘does it work better than what we have?’As this may be the final step before a drug is licensed, Phase III studies are the most restrictive in terms of tumor type and line of therapy.

Question #5: What would be a reason that someone cannot qualify for a clinical trial?

(Dr. Camidge) The three most common reasons that cause people with cancer to fail to qualify for clinical trials are inappropriate line of therapy, inadequate fitness for participation, and, less frequently, inappropriate insurance coverage.

Note: More information on this topic can be found in a recent article written by D. Ross Camidge, MD PhD., entitled, “Who are clinical trials for: Guinea Pigs, Test Pilots or Prize Poodles?’ This article can be viewed in its entirety on this blog as well as www.lifelongwellnessadvocates.com. To contact Dr. Camidge directly, he can be reached at the University of Colorado Cancer Center located in metro Denver on the Anschutz Medical Campus in Aurora. The University of Colorado Cancer Center is the Rocky Mountain region’s only National Cancer Institute – designated comprehensive cancer center. Phone: 720-848-0392