Encouraging and Paying for Clinical Trials, Right to Try, and Expanded Access: Part One

A Q&A with Mark Shapiro, PhD,Vice President of Clinical Development at xCures, Inc.Partner at Pharma Initiatives; mshapiro@xcures.com

Q: Treatment of Americans with advanced cancer is complex and challenging and can be very expensive. Many urge greater participation of such patients in clinical trials. In general, who pays the expenses of clinical trials? And, specifically, how are the costs for Right to Try and expanded-access approaches reimbursed?

A: Incorporating clinical research into the clinical care of cancer patients may provide more options, and better outcomes, but participation is quite low. In 2004, only about 3% of American cancer patients participated in clinical trials.

More recent data suggest that the number may now be about 5%, although it is lower for women, children, minorities, and patients in community settings. The low figure should be of concern for a couple of reasons. First, patients are the scarcest resource in cancer research. Low participation in clinical trials represents a lost opportunity to learn and improve care. If every patient were part of systematic research, we could greatly accelerate the pace of cancer research findings. Second, most cancer treatment guidelines recommend a clinical trial as the standard-of-care at some stage in the course of disease. So, with current levels of participation, as many as 95% of American cancer patients are NOT receiving standard-of-care treatment at some point in their care. This deficit is partially attributed to the presence of comorbidities or poor function. Recent research suggests that liberalizing inclusion and exclusion criteria in clinical trials could increase enrollment by about 45%. In the study of common cancers, enrollment of patients with solid tumors could be increased from about 7% to 11%.

In my work at xCures, in partnership with Cancer Commons, we recently had a patient who traveled out of state to be screened for enrollment into a targeted-therapy clinical trial at an academic center. At screening, the patient was deemed ineligible because the disease had not yet progressed. A few months later, that patient began to progress, made another trip out of state, was re-screened and found eligible for the trial, but developed an acute and disqualifying comorbid condition the night before starting treatment. This illustrates a common challenge to enrolling patients in cancer clinical trials: The need to thread the needle during a window where the patient is “sick enough but not too sick.” This period can be days or weeks in diseases like glioblastoma and pancreatic adenocarcinoma. We would like to see more use of the U.S. Food and Drug Administration (FDA)’s expanded access programs for the 90% to 95% of cancer patients who can’t participate in a trial. Of course, the results of such participation would have to be reported so as to increase shared knowledge. Expanded access is a mechanism for doctors to access investigational treatments outside of a clinical trial for patients who have serious or life-threatening conditions without satisfactory therapeutic options.

Recently, the national Right to Try Act became law in May of 2018 (Public Law 115-176) following the passage of right-to-try laws in 40 states. The first patient treated under the federal Right to Try Act was in California last year. The sponsor, ERC-USA, notified the FDA that it would make their brain cancer vaccine available in June, shortly after the passage of the law, and treated a patient in November. While the stated goal was to move quickly, the time to treatment was longer than what we typically see for glioma patients with expanded access. However, this is a new alternative for treatment of cancer patients with an investigational therapy and may supplement clinical trials and expanded access programs as options for those patients who lack other treatment options.

All three of these approaches to treatment—clinical trials, expanded access, and Right to Try—raise issues of both cost and equitable access to care, which I’ll discuss next week in part two of this Q&A.


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