Jim Omel is a myeloma patient and a cancer research advocate. He spoke at the 2017 Friends of Cancer Research Annual Meeting and was an author of the panel 2 whitepaper, Data Generation to Support Cross-Labeling of Indications for Combination Products. Below are his abridged remarks from the meeting. To view his remarks in full, click here. You may follow Jim on Twitter @IMFjimMYELOMA. 

Educated cancer patients understand the value of clinical trials in advancing our knowledge, but we all know that only a tiny percentage of patients actually participate in trials.

If you ask 10 patients, “What is the main purpose of a cancer clinical trial?” all 10 will say it is to help cancer patients.  However, they will all be wrong.  The principal reason for research studies containing clinical trials is to “Answer a Scientific Question”.  To obtain faster patient accrual, and better acceptance could we please make, “Helping Cancer Patients” the purpose of each trial?  That includes the final patients added to an arm to maximize and insure statistical validity even when the arm is already showing probable inferiority before final accrual.

I don’t want to be the last patient added to an inferior arm, and do poorly, just to prove that I should have been in the other arm in the first place.

I believe adaptive trial designs and Bayesian methods should be promoted.  Phase II single arm trials are important as we refine our targets and genomically identify patients who will benefit from a specific targeted therapy.  It is exciting to know that 19 of 30 new drugs in oncology, which received accelerated approval, were based on single arm trials.  Experts are seriously re-thinking the value of long, expensive Randomized Controlled Clinical Trials (RCT).  Another good approach is to determine whether currently existing data are already available, which can be rigorously and objectively evaluated.

Valuable health data continue to emerge, including information from electronic health records, and are providing real world outcomes for the 97% of cancer patients who will never be in a clinical trial.  Expanded patient registries, along with an increased willingness of patients to participate and share health information, are generating useful data.  While an RCT may show the benefit of a drug, large observational studies can be conducted to refine dosages and identify rare, adverse events.

New clinical trial strategies will increase enrollment, reduce costs and time to completion, and better identify populations that benefit from treatments.  Tables I and II in the Appendix of the Friends Annual Meeting Panel 2 Whitepaper explain several of these different trial designs for combination therapies.  Advances in genomic science allow for better understanding of unique patient characteristics that can affect specific treatment outcomes.

New products and standards of care are often developed before lagging clinical trials complete evaluation. The increasingly high costs, time constraints, and poor accrual to RCTs lead to wasted financial resources and valuable patient participation.  As the Whitepaper explains, conducting a clinical trial leading to regulatory approval in oncology averages 8 years. During those 8 years it is quite likely that Standard of Care for an indication in cancer can change, resulting in a completed trial with less value.

I end on a worrisome personal note. My myeloma was diagnosed in 1997, and I have had relapses in 2000, 2006, and 2010.  For 7 years I have been blissfully drug-free.

However, for the last few weeks I have had a deep, ominous, gnawing pain in my right posterior-lateral ribs.  My labs have been good but I recognize the pain.  Quite literally in my bones I know I’ll have to make a treatment decision about my 5th line of myeloma therapy.  I’ll want a triplet, perhaps with radiation.  While the official FDA product labels are important, I will make my decision based on other factors such as my past response history.

Both patients and their physicians need all relevant clinical trial data, NCCN guideline recommendations, personal experience, FDA regulatory data, and label information to make good treatment decisions.  I would not want my doctor to treat me based only on a product label.  Likewise I would not want any product label to be woefully out of date.  I don’t want an insurance company, or a third party clinical pathway company, or a group such as ICER to use an outdated FDA label as justification for not paying for my expensive therapy when my doctor and I feel we have chosen the best available treatment option.  Those of us with cancer want and deserve the best treatment for our disease.

There will always be an argument for more research and for better data, but waiting for more data is often its own implicit decision not to act.  Cancer patients don’t have time to wait for clear, obvious data.  We must decide NOW for the treatment we feel is appropriate NOW.