Research!America and the Pancreatic Cancer Action Network held a meeting in New York this year to discuss ways to restructure clinical trials to find a cure for cancer. The meeting called, “A World Free From Cancers: Probable, Possible, or Preposterous?”, was held at the New York Academy of Sciences. Topics of discussion included revamping clinical trials, clinical trial regulation, the high cost of cancer drugs, and the role of Oncologists in treatment and clinical trials.
Panelist Clifton Leaf, author of The Truth in Small Doses: Why We’re Losing the War on Cancer — and How to Win It stated that “We need to be able to move faster in testing our ideas in cancer science. “The most remarkable thing is when innovation is coupled with freedom — freedom of ideas, bureaucratic freedom, regulatory freedom, and freedom to test those ideas to the marketplace more fluidly.”
Robert J. Hariri, MD, PhD, chair, founder, and chief scientific officer at Celgene Cellular Therapeutics shared that”using twentieth-century thinking and logic to solve twenty-first-century problems is a fundamentally flawed concept.”
Panelists spoke about one of their main hopes being getting patients back to their regular lives through clinical trials. Frank R. Lichtenberg, PhD, Courtney C. Brown professor of business at Columbia University stated that “these therapies for cancer are actually returning people to useful, highly productive lives.”
Amy P. Abernethy, MD, a hematologist/oncologist and palliative care physician and director of the Duke Center for Learning Health Care went on to say that “it is not just 1 person. It is often multiple people in a family; a person with cancer often has caregivers. When you get 1 person back to work, you often get the family back to work.”
Redesign of Clinical Trials
The demand for clinical trials is extremely high. Only 3% to 5% of adults are enrolled in clinical trials according to Julie M. Fleshman, president and CEO of the Pancreatic Cancer Action Network.
Scott Gottlieb, MD, resident fellow at the American Enterprise Institute stated that clinical trials are “contrived and hard to enroll,” and estimated that it costs about $100,000 to enroll 1 patient in a clinical trial for late-stage cancer.
Clinical Trial Regulators
Also discussed was the need for regulators to be absolutely certain about their approvals
Dr. Gottlieb stated that “If p doesn’t equal .05, then it is untrue; if it does, then it is all true. This forces us to develop highly contrived clinical trials that are geared to trying to get a statistical result and not geared to generating the kind of practical information that will allow us to better target therapies.” (editor’s note – p needs to be equal or less than .05 to prove statistical significance at a 95% confidence level.)
“We talk about how regulators are risk averse and are worried that they will approve a drug that has a certain side effect. Most side-effect profiles are usually very well established,” and patients are often willing to tolerate them because of the benefits.
“The risk that is paralyzing the [FDA] is the risk that they will approve a drug on the basis of an observation of benefit that isn’t 100% of certain. What they want is to say that if this trial shows that this drug shrinks tumors by 40%, I want to be 100% certain that it shrinks them by 40%.”
Costs of Cancer and Clinical Trials
The discussion also focused on the financial costs that patients face.
“In the clinical space, we don’t have any clue about what the costs looks like to the patients,” said Dr. Abernethy. “I think one of the issues is that physicians are scared of this space because they feel uncomfortable.” Traditionally doctors have not discussed the cost of treatment with their patients, but now “we have to,” she emphasized. “If the cost of a drug to a patient is going to mean their mortgage, you need to know that. The worst thing is to get a call from a patient standing at the pharmacy, saying ‘This drug is $3000. How should I pay for it?’ ” she said.
The LRG is extremely interested in clinical trials and recognizes how important they are to GIST patients and their caregivers. We also agree that the current clinical trial process is far from optimal and would love to see that change as soon as possible, and have met with various pharmaceutical companies to discuss this. We continuously monitor what clinical trials for GIST are available; this can be accessed via our website by clicking here.
You can read the full article about the meeting here.