publication date: Sep. 16, 2016
White House: New Moonshot Initiatives On Clinical Trials Will Improve Speed, Access
By Matthew Bin Han Ong
Vice President Joe Biden Sept. 16 announced a series of initiatives to improve the safety, accessibility, and impact of clinical research—one of the central goals of the National Cancer Moonshot Initiative.
The programs, which span four federal entities—NCI, FDA, the Department of Health and Human Services, and NIH—would make existing clinical trial search sites more user-friendly, and ensure that results of clinical trials are shared in a timely fashion.
“Clinical trials are essential for developing new and more effective cancer diagnostics and treatments,” Biden said in a statement. “But right now, less than five percent of cancer patients enroll in a clinical trial, often because patients and doctors don’t know what trials are available. We can do better—and we are.”
According to the White House, these steps would:
• Make it easier for participants to find clinical trial opportunities as quickly as possible,
• Incentivize new ways of designing clinical trials to maximize participation while minimizing burden and risk, and
• Strengthen the transparency of clinical trials and trial results.
About 31 percent of all open trials listed in ClinicalTrials.gov are for cancer-related research, but approximately 20 percent of these trials are not completed due to low participation.
“Clinical trial participation is even lower for cancer patients who do not receive treatment at academic medical centers, and for older, underserved and minority populations,” according to a White House factsheet.
One of the initiatives involves a rethinking of patient eligibility criteria: a modification of FDA’s requirements could expand the number of people who qualify for clinical trials.
“We are working with stakeholders across government and industry to revisit the criteria used for determining whether a patient is eligible to participate in a trial,” said Richard Pazdur, director of the FDA Office of Hematology and Oncology Products and acting director of the agency’s new Oncology Center of Excellence. “Of course, regardless of adjustments, patient safety will remain paramount.
“We recently published our perspective on shifting away from the conventional phase one, phase two, and phase three drug development paradigm to a more seamless approach that could expedite the regulatory pathway, providing earlier access to highly effective therapeutic drugs,” Pazdur wrote in a Sept. 16 FDA blog post. “Adopting this approach could complement FDA’s expedited regulatory programs such as breakthrough designation and accelerated approval to get products to patients in the most efficient manner possible.
“Another initiative is the use of common control trials. These trials, sharing a common control arm, involve multiple different drugs for the same indication and may involve different companies. When a common control arm is used, it decreases the overall number of patients that need to be recruited and enrolled, optimizing clinical trial resources and potentially decreasing the time it takes to get a new study off the ground.
“Encouraging the use of large simple trials is another way to make more efficient use of clinical trial resources. These trials generally use easily-measured endpoints that are well understood, optimizing the collection of data for safety or secondary efficacy endpoints and thus reducing the amount of data needed compared to conventional randomized trials.”
The White House’s summary of the new moonshot initiatives follows:
• NCI Launches Redesign of Cancer Clinical Trial Information Available through Trials.Cancer.gov
The NCI and the White House Presidential Innovation Fellows have collaborated to re-design how patients and oncologists locate information about cancer clinical trials. At the Cancer Moonshot Summit in June, the team unveiled trials.cancer.gov, a streamlined, user-friendly website for searching for NCI-supported cancer clinical trials. The next big step announced today is making the information on trials.cancer.gov available through a new application programming interface (API) integrated within this site. This new API allows researchers and patient groups to tailor information from the site, in real time, to identify trials relevant to individual communities and specific cancers. Ultimately, the combination of a more easily navigable web interface, with a user-friendly API, will make it much easier for researchers to recruit new participants and for patients to find trials that make a difference for them.
• FDA will Encourage Use of Innovative Clinical Trial Designs
The FDA is announcing a series of efforts to bring together clinical researchers across government and private industry to design smarter and more efficient clinical trials by exploring ways to modify the criteria for who can participate in a trial or by sharing control groups across studies using different drugs for the same indication. These kinds of efforts can minimize the number of participants needed to achieve meaningful results while keeping participant safety at the forefront. Designing more efficient trials that produce better results can dramatically accelerate how quickly new therapies are brought to patients, and decrease the cost of doing so.
• HHS Enhances Clinical Trial Registration and Results Reporting through Final Rule
HHS is announcing the publication of a final rule that specifies requirements for clinical trial registration and summary results information reporting in ClinicalTrials.gov, a registry and results database of publicly and privately supported clinical studies of human participants conducted around the world. The rule extends the requirement for summary results information reporting to include applicable clinical trials of drug products (including biological products) and device products that are not approved, licensed, or cleared by the FDA. This will enhance transparency within the clinical trials enterprise, allowing people to make more informed decisions about participating in research. The rule will also help prevent unnecessary duplication of unsuccessful or unsafe trials, has the potential to help increase the efficiency of drug and device development processes, and build public trust in clinical research. Announced at the same time is a new NIH policy to help ensure that all NIH-funded clinical trials are registered and their results information shared in ClinicalTrials.gov.
• NIH is Working to Increase Usability of ClinicalTrials.gov
To expand on the final rule and policy for clinical trial registration and summary results reporting, NIH is committed to make ClinicalTrials.gov a more user friendly and accessible site. As such, it is working with technical experts at 18F, the Presidential Innovation Fellow’s counterparts, to explore ways in which a partnership between the two could accelerate these efforts. Enhancing the usability of the information within this database will make it easier for patients to find trials and information about interventions that may be relevant to their cancers.