How Can We Encourage Participation in Clinical Trials?
Failed clinical trials come at a huge cost to their pharmaceutical sponsors. Many trial sites fail to enroll more than a single patient—up to 60% of oncology trials, according to Covance, for example. Yet they estimate it costs a sponsor $50,000 for a site start-up, with a loss of almost $2 billion between 2006-2010 from non-performing sites.
Did you know that only 3% of patients with cancer participate in clinical trials? Although we have an aging population and cancer rates increase with age, this dismal participation rate hasn’t budged in recent years. What factors are at play?
Enrolled Patient Numbers
Randomized Clinical Trials (RCTs) have constituted the foundation for new drug approvals for over fifty years. An Institute of Medicines (IOM) report concluded though in 2008 that RCTs may become unsustainable because of the time and expense involved. In addition, as therapies become more targeted, the financial calculus may make these RCTs even more problematic.
Some industry critics have argued that the increasing number of patients in Phase III clinical trials is actually an industry strategy to improve the statistical strength of clinical trial results and RCTs will actually have to continue to increase in size. As differences between active and non-active arms become more difficult to establish, larger sample sizes are used to improve the chances of reporting statistically significant differences.1 Rather than a response to scientific necessity, the growing number of enrolled patients is a conscious strategy to improve the chances of a favorable regulatory approval decision.
Enrollment of Patients With Lung and Colorectal Cancers Onto Clinical Trials
Clinical trials, both federally and industry sponsored, have allowed remarkable progress in the treatment of some cancers. However, a long-standing consensus exists that the enrollment of adult patients with cancer onto clinical trials is low. Enrollment fractions, defined as the number of enrollees divided by the population-based estimated cancers diagnosed, in therapeutic nonsurgical National Cancer Institute (NCI) Clinical Trial Cooperative Group studies are below 2% and are even lower, less than 1%, for surgical trials. When trial eligibility is taken into account, the rates rise but still may be as low as 10%.
A recent report by the Institute of Medicine recommended that participation of both patients and physicians in cancer clinical trials be expanded. This report generated extensive media coverage, leading to Congressional attention and a consensus that our system for cancer clinical trials is in crisis.