By Tandy Tipps, MEd, MPA, PhD, Vice President, Precision Oncology Site Network
Today’s competitive oncology drug development space is evolving rapidly, with high stakes for sponsors and participants alike. An increasing number of novel new oncology drugs are in development—precision medicines and immunotherapies such as immune checkpoint inhibitors, CAR-T cell therapies, and oncolytic viruses—that leverage genomic insights. Many more applications than in the past are also qualifying for the FDA’s expedited approval programs. Needless to say, in this race for novel drug approvals, the complexity of oncology trial design and execution has increased dramatically.
For biotech drug developers, moving oncology treatment innovations from the lab to clinical research can be daunting. One of the most common bottlenecks in the development pathway is study start-up. According to a survey completed by the Tufts Center for Drug Development, the industry average time for clinical trial activation, defined as the time from when the primary investigator receives the final protocol to the date of first patient in for academic and community sites is 7.9 to 12.9 months.1 Many studies get passed on to new partners in the start-up phase, leading to costly delays.
Selecting the right research development partner from the outset is essential to streamline study start-up. When the development partner utilizes the following best practices, achieving expedited trial activation within 90 days is a realistic goal.
1. Experienced guidance for oncology trial design. Clinical trials are best accepted by the research community when the study protocols have been vetted by researchers. Insight gleaned from experienced oncology investigators within a trusted network sets the trial on course for feasible implementation and enables a “ready-to-triage” study.
Most oncology protocols will integrate biomarker identification and patient stratification approaches from the outset, providing sponsors with the best opportunity to identify responders, optimize doses, and narrow results for further study. There is no room for error in the implementation of these complex studies. A poorly designed oncology study can slow patient access to lifesaving therapeutics. Real-world design input from seasoned oncology researchers is a critical success factor.
2. Optimal site selection. Rapid trial start-up is more likely to occur when sponsors work with a development partner with an established clinical trial network of prequalified oncology research sites. Sites should be selected and vetted up front, building a network based on a track record of quality in oncology research and a commitment and ability to follow expedited start-up processes. The network should cover a wide geographic area and include major academic cancer centers as well as community sites in rural regions, enabling recruitment from a large catchment.
When a development partner knows each site’s therapeutic “sweet spot”—a combination of the site’s ability to recruit well for a cancer type and a track record of excellence conducting specific phases of oncology research—selection of the right study sites for a sponsor’s novel compound are quickly identified. For example, many new study designs in oncology are combination dose escalation/dose expansion. Careful site selection must be pursued to ensure a site can readily transition an “all-comers” trial into tumor type–specific cohorts.
Sponsors save a significant amount of time and money when expedited start-up shortens the overall length of their studyA network consisting of pre-established sites that provide master service and confidential disclosure agreements also expedites the start-up process. Eliminating much of the up-front administrative work and matching the right site to the right study are critical steps to expedited start-up.
3. Build the right enrollment strategy. Patient recruitment is an industry-wide challenge—only about 3 percent of American cancer patients participate in clinical trials.2 The recruitment and retention of qualified participants are essential for meeting timelines, budgets, and overall trial success. The development partner’s project management and clinical trial monitoring staff must work closely with the sponsor to build and implement site-specific patient enrollment plans to ensure projections are met with the right patient populations and within site recruitment budgets.
Experienced oncology clinical monitors operate as site champions who not only provide on-site and remote monitoring, but also ensure that the sites are supported through all aspects of the enrollment plan. They are ready to resolve issues that arise with rigorous attention to detail, ensuring that the trial start-up is as efficient as possible. This single point of contact is a valuable resource to even the most experienced oncology sites.
The study inclusion and exclusion criteria inform the enrollment strategy, but a development partner should go well beyond those factors when advising and supporting sites. Site-specific enrollment strategies and support, combined with practical protocols and the “right” sites, are essential for achieving first-patient-in and beyond.
When the best practices for study start-up are employed by an experienced development partner, along with a trusted and proven site network, expedited study start-up is an achievable goal for even the most complex oncology. With the right trial design, site selection, and enrollment strategy from the outset, sponsors set themselves on the right pathway for quickly obtaining the robust data needed for proof-of-concept.
Tandy Tipps is vice president of the Precision Oncology Site Network. She established the network with a focus on consistently optimizing start-up at both academic and community-based sites across the United States and Europe. Before joining Precision Oncology, she oversaw the world’s largest phase 1 program for cancer research and treatment at the renowned University of Texas MD Anderson Cancer Center.
- Tufts Center for the Study of Drug Development. 2012 survey. https://csdd.tufts.edu. Accessed January 10, 2017.
- Institute of Medicine (US) Forum on Drug Discovery, Development, and Translation. Transforming Clinical Research in the United States: Challenges and Opportunities: Workshop Summary. Washington DC: National Academies Press; 2010. 6, Clinical trials in cancer. https://www.ncbi.nlm.nih.gov/books/NBK50895/. Accessed December 29, 2016.