Though President Trump's statements supporting compassionate use of medicines were meant to criticize the FDA's so-called “burdensome” drug-approval process, his mention of right-to-try laws propelled the issue of fair use back into the limelight. Trump has argued that the way FDA operates has stunted innovation and has restricted access to investigational medicines for terminally ill patients.
But the FDA already has an excellent track record for compassionate use; according to FDA calculations, the agency authorized 99% of single-patient expanded-access applications in FY2010–2015. Despite the high approval percentage, FDA authorization does not always correlate with improved patient clinical trial enrollment, as barriers such as insurance coverage could still restrict some patients from trying life-saving medications. A 2010 study revealed that nearly 14% of patients in cancer trials from 2003 to 2008 were denied coverage for those trials.
For oncology trials, in particular, decisions about trial participation need to occur quickly, as disease can progress in a short amount of time.
While Medicare has covered the routine costs of clinical trial participation for enrollees, private insurers are not required to cover investigational therapies, and whether the insurer will cover treatment often depends on where the patient resides and a host of other factors. The Patient Protection and Affordable Care Act (ACA) contains a provision that requirs private insurance plans to allow trial participation and cover some of the costs related to standard of care.
A new study, published online on July 20, 2017 in Clinical Cancer Research, a journal of the American Association for Cancer Research, shows that expanded insurance coverage as a result of the passage of the ACA, and the provision therein allowing trial participation and coverage, contributed to an increase in clearance rates for clinical trials among privately insured patients. According to the study, approval rates to enter trials rose steadily for privately insured patients, from about 85% prior to the ACA mandate to approximately 95% after the mandate. In other words, more cancer patients got access to investigational medicines and could benefit from FDA’s compassionate use policy after the ACA was passed than before the law was introduced.
Investigators looked at 2404 referrals from the departmental database of the Clinical Center for Targeted Therapy at MD Anderson and examined clearance rates during three separate time periods: July 2012–June 2013, July 2013–December 2013, and January 2014–June 2015.
Additionally, patients received decisions about whether their treatment in a trial would be covered more quickly than they did prior to the passage of the ACA. And even though overall clearance rates for Medicare enrollees to get into clinical trials were not affected by ACA coverage, the researchers found that Medicare/Medicaid enrollees experienced faster time to clearance (i.e., a decision about coverage was made more quickly) than in the reference period.
Some limitations of the study exist, however. The authors noted that improved efforts to prescreen patients occurred when the program underwent a leadership change, and it is difficult to assess whether this increase in insurance coverage for trials was because of the leadership change or because of the ACA provision, which went into effect in January 2014. Details such as institution exclusion from provider networks could not be measured, which served as another limitation. In addition, the researchers point out that some older private plans that existed before the ACA were not required to change their benefits; thus, the changes to clearance trends in this cohort could not easily be measured.
But the institutional-based changes identified as a limitation do not present a huge problem, Darius N. Lakdawalla, Ph.D., Quintiles Chair in pharmaceutical development and regulatory innovation at the University of Southern California—who was not involved in the study—told GEN. “This is a standard limitation of analyses like these, and is not a fatal issue.” He added, “More generally, this is an interesting finding that suggests the important role that health insurance policy can play in supporting the study and development of new medical innovation.”
Patient-specific therapies, and those that rely on the identification of a disease biomarker, are increasingly entering early-stage clinical trials. Thus, increased enrollment in these trials will likely fuel future drug discovery and the identification of disease mechanisms. As study author Kenneth Kehl, M.D., told GEN, “Facilitating access to clinical trials and optimizing accrual rates is more important than ever for drug discovery and personalized medicine, given the increasing number of targeted therapies and immunotherapies under investigation.”
The investigators did not evaluate whether the increased rate of acceptance in clinical trials actually helped improve overall patient survival. Noted Dr. Kehl, “We did not include assessment of these clinical endpoints in our study. Patients with insurance that declined clinical trial enrollment would not be actively followed by the early-phase clinical research program at MD Anderson thereafter.” However, Kehl told GEN this is something that could warrent further study, considering that prior evidence suggests that participation in clinical trials is associated with improved outcomes. “Such an analysis would need to be carefully designed, given heterogeneity in patient populations and tumor types within a broad drug-development program such as the one at our institution.”
*Updated on 7/20 to include comments from Kenneth Kehl, M.D.