Clinical trials are a key step in the development and approval of new drugs to treat cancer and often a lifeline to patients seeking options when other treatments have failed.
Reflecting its importance, the clinical trials process is getting a closer look from the medical research community for ways to improve efficiency and effectiveness. Two organizations, Enacct and CTTI, are working on projects that will help identify best practices in clinical trials.
Margo Michaels, executive director of Enacct, which stands for Education Network to Advance Cancer Clinical Trials, said her organization’s National Cancer Clinical Trials Pilot Breakthrough Collaborative project is aimed at finding systemic changes in the process that will increase clinical trial accrual.
Enacct points out some of the shortcomings of clinical trials that led to the project on its website, www.enacct.org:
- While 20% of adult cancer patients are eligible to participate in cancer clinical trials, only 3% of those eligible actually participate, and the percentage is even lower for the minority and elderly populations.
- About 15-30% of NCI Coooperative group trials are closed early due to poor accrual.
- Accrual varies greatly: 15-20% of local sites involved in cooperative group studies never enroll a single patient, while 30% of sites enroll 70% of evaluable patients.
“Participation in cancer treatment clinical trials is a key measure for delivery of quality cancer care,” Enacct asserts. Yet that is not happening.
For example, among some recent GIST clinical trials, the Phase III regorafenib trial was very successful in enrolling patients and led to the approval by the Food and Drug Administration of Bayer Pharmaceutical’s drug Stivarga for third-line treatment of GIST earlier this year. However, the Phase III Gleevec dose escalation study opened in January 2010 and closed in May 2011. It had planned to recruit 400 patients but only recruited five. It was not positioned in sites that saw newly diagnosed patients.
The Enacct project hopes to “get centers to implement changes to make accrual more possible,” Michaels said.
One of the main issues the first phase of the project identified was the lack of systematic screening for participants at cancer centers, she said.
“Systematic screening is not happening in the majority of places,” she said. Part of the project was working with cancer centers to change policies and procedures and put processes into place that will improve accrual.
“We have to make sure all eligible patients are approached,” she said.
She also said a more strategic approach is needed in choosing trial sites geographically to reach a greater number of participants. “We have to make sure things like travel costs are built into the process,” she said. “If there is a trial going on in New York and a person lives in Omaha, how are they going to get there?”
Michaels said clinical trial design is another critical issue. She would like to see more engagement of patient groups in the design process to make sure the right kinds of questions are being asked in studies.
Bray Patrick-Lake, Director of Stakeholder Engagement at CTTI, which stands for the Clinical Trials Transformative Initiative, said her organization “has just launched a formal recruitment and retention project.” CTTI is a public-private partnership that works to create a better clinical trials process in terms of quality and safety.
“We are beginning to identify issues,” she said. “The goal is systemic improvement, making the process better for all.”
She added, “A lot of times trials never enroll, which is terrible for patients.”
Jeff Allen, executive director of Friends of Cancer Research, said his group is also “very interested in how to improve efficiency” of clinical trials.
He sees the need for better physician education on what trials are available to patients. He also thinks patient groups can play a role in “shepherding treatments,” particularly as targeted approaches become more common.
He acknowledged that an issue in clinical trials is the difficulty of doing combination studies with drugs from two different companies. “It’s really hard to get two different companies to collaborate because of the financial implications,” he said.
“But I would hope there would be more collaboration going forward.”
He pointed to a successful lung cancer project with the Food and Drug Administration as an example of how collaboration can be helpful.
Dr. Patricia Keegan, who is Division Director of Oncology Products 2 of the FDA’s Center for Drug Evaluation and Research, also thinks collaboration is becoming more important.
She said the FDA offers guidelines for clinical trials but “not so much specific best practices” because “we would not want to be prescriptive in order to provide flexibility.”
She said with more targeted treatments being developed, clinical trials “in some ways have to change.” Part of that will be greater collaboration among groups.
“To the extent that groups can partner together” that will help, she said, adding that rare diseases such as GIST “require many minds” to find a cure.
She said an advantage to collaboration is that findings can be more easily confirmed and built upon. She also pointed out that “the development of tests to identify subpopulations needs to get the same kind of attention” as drugs to treat the mutations they identify.
She sees an important role for advocacy groups such as the Life Raft Group in “getting out the word and promoting participation” in clinical trials.
The LRG is working to develop a clinical trial model that would even go beyond that. The project aims to bring together pharmaceutical companies and cancer centers and leading researchers in a consortium that would have access to the LRG’s Patient Registry and Tissue Bank, a valuable source of data for both trial design and recruitment.
The model would go a long way toward improving recruitment, particularly for targeted treatments that are aimed at limited populations.