Innovative Trials
CTTI Examines Stakeholder Perceptions of Mobile Clinical Trials: Latest Expert Meeting Summary Now Available
The use of mobile technology holds the promise for higher quality, more efficient clinical trials, allowing remote participation in study activities, reducing patient burden, and facilitating the involvement of a more diverse population.
Critical to the widespread adoption of mobile technology in clinical trials is positive reception by key stakeholders. However, until recently, very little was known about the possible benefits and barriers perceived by investigators and trial participants. To address this issue, CTTI conducted qualitative and quantitative research to better understand perceptions, and reviewed the findings in a multi-stakeholder expert meeting (see one-page summary) that included investigators, patient partners, regulators, technology experts, sponsor representatives, and other groups.
At the meeting, experts underscored that successful adoption of mobile technology requires
rethinking how trials are designed and conducted. At the same time, they noted that many of the factors that define high-quality trial design remain the same, whether or not mobile technology is used. They also agreed on:
- The importance of involving patients early and often in trial planning and design;
- Ensuring that participants remain engaged and are able to easily communicate with site staff;
- Selecting and tailoring devices based on study design; and
- Addressing the unique concerns that stakeholders have around mobile clinical trials, including patient confidentiality, missing or inaccurate data, maintaining patient engagement, and a lack of uniformity among processes, devices, and platforms.
As a next step, the CTTI MCT Stakeholder Perceptions Project team will use the findings from this meeting to inform the development of recommendations for designing and conducting trials with mobile technology to best meet the needs of patients and sites.
CTTI to Lead Breakout Session and Offer Insights into Using Mobile Tech and Novel Endpoints in Clinical Trials at 2018 SCOPE Meeting
This year’s Summit for Clinical Ops Executives (SCOPE) conference will feature a notable presence from CTTI as part of the annual meeting’s first-ever Sensors, Wearables and Digital Biomarkers in Clinical Trials track. The meeting will take place in Orlando from Feb. 12-15.
CTTI, which recently published a set of recommendations and resources for developing and incorporating novel technology-derived endpoints into clinical research as part of its larger Mobile Clinical Trials (MCT) Program, will lead a breakout session focusing on findings from this project and what they mean for site investigators and patients. Discussion will center on use cases developed from four therapeutic areas—Parkinson’s disease, heart failure, diabetes, and Duchennes muscular dystrophy—and will describe strategies for identifying, developing, and implementing novel endpoints derived from new and emerging technologies, including mobile devices.
The breakout session will be accompanied by a pair of related CTTI presentations taking place over the next two days. The first will provide insights from CTTI’s MCT Stakeholder Perceptions Project exploring patient and investigator views of research involving mobile technologies, and will examine ways to develop a successful patient-centered approach to leveraging mobile technology in clinical trials. The second presentation will offer expert perspectives from CTTI’s MCT Novel Endpoints Project, which addresses uncertainties about how best to incorporate mobile technologies into clinical research and offers pathways for leveraging data derived from such technologies to accelerate research and therapeutic development.
We hope you will join us in Orlando on Feb. 13-15 as we explore evidence-based approaches to integrating novel technologies and endpoints into clinical research.
Interactive Breakout Discussion: Selecting, Developing and Incorporating Novel Endpoints, Generated from Data Captured by Mobile Technologies, for Use in Clinical Trials
Date and time: Tues., Feb. 13, 4:00 p.m. EST
Presenters: Jennifer Goldsack (CTTI), Rob DiCicco (GSK), Amy Calvin (Eli Lilly), and Christian Gossens (Roche)
Presentation: New Findings on Patient and Site Perspectives from the Clinical Trials Transformation Initiative
Date and time: Thurs., Feb. 15, 12:25 p.m. EST
Presenter: Hassan Kadhim (Boehringer Ingelheim Pharmaceuticals)
Presentation: Identifying, Developing and Incorporating Technology-Derived Endpoints into Clinical Trials: A ‘How-To’ Guide
Date and time: Wed., Feb. 14, 11:15 a.m. EST
Presenter: Rob DiCicco (GSK)
New CTTI Article Investigates Mobile Device Use to Measure Outcomes in Clinical Research
A CTTI article recently published in Digital Biomarkers investigates the use of mobile devices to measure outcomes in clinical research from 2010-2016. The article provides a detailed accounting of where the field is currently, allowing researchers to see what measures exist for using or developing technology-derived endpoints, how they are being used, and how to access relevant literature.
The review found that, while mobile devices are widely used to assess outcomes in observational research, their use in interventional research is limited. Additionally, the absence of standardization across the measures used to assess outcomes of interest, units of measurement, sampling rate, device placement, and the technologies themselves indicates a pressing need for standards to interpret and compare results across studies and across therapeutic areas.
The article offers suggestions for incorporating mobile technology into interventional research—such as consolidating evidence supporting the clinical meaningfulness of specific technology-derived endpoints, and standardizing the use of mobile devices in clinical research to measure these endpoints.
The review also found that:
- The majority of technology-derived novel endpoints are currently being used in cardiac studies. However, in randomized controlled trials, technology-derived novel endpoints are most commonly used in Type I diabetes studies.
- Physical activity measures are the most commonly used technology-derived endpoints in clinical studies. Other commonly used endpoints are related to sleep, mobility, and pill adherence, as well as biomarkers such as cardiac, glucose, gastric reflux, respiratory measures, and intensity of head-related injury.
- There is a scarcity of technology-derived measures being used as actual outcome assessments in studies of neurological diseases such as Parkinson’s and Alzheimer’s, which have a considerable unmet need for measures. Oncology and nephrology are two other key therapeutic areas with unmet need for better assessments, but a dearth of technology-derived measures.
New Results on Cost Drivers in Phase 3 Drug Trials for HABP/VABP
Drug-resistant infections are on the rise, and one type of serious infection that can be caused by drug-resistant bacteria is hospital-acquired and ventilator-associated bacterial pneumonia (HABP/VABP). Despite the desperate need for effective new therapies for HABP/VABP, clinical trials for this condition are lagging due to difficulties in conducting these trials and low expected return on investment. A new study from CTTI and the Tufts Center for the Study of Drug Development, published in Clinical Infectious Diseases, calculates the cost of phase 3 trials for HABP/VABP and identifies areas of opportunity to lower costs and boost investment in these critically important studies.
“To meet the urgent public health need for new antibacterial drugs, clinical trials must be more efficient, cost-effective and feasibly executed. Our study identified several factors driving clinical trial costs that can be targeted to optimize the development of new treatments,” said Ken Getz, an associate professor and researcher at Tufts University involved in the study.
“Leveraging evidence to direct meaningful changes in clinical trials is what CTTI is all about,” said Pamela Tenaerts, CTTI executive director. “We hope this information will help target efforts to streamline HABP/VABP trials, which will ultimately benefit patients.”
Key findings include:
- The cost of a phase 3 HABP/VABP clinical trial averages $89.6 million, which is over double the average cost of a phase 3 oncology trial
- Per patient, phase 3 HABP/VABP clinical trials were $2,300 more expensive than oncology clinical trials and $31,900 more expensive than endocrine trials
- Screening failure rates and the cost of screening failures were the biggest drivers of cost, suggesting sponsors and research sites should consider ways to increase the number of potentially eligible patients
CTTI released recommendations for streamlining HABP/VABP trials and is testing novel approaches to make these trials more feasible, such as using predictive models to find and consent patients earlier in the disease process.
Finding Solutions to Legal and Regulatory Challenges of Conducting Mobile Clinical Trials
Mobile clinical trials (MCTs) offer several potential advantages, such as greater patient convenience, efficiencies in data collection, potential for novel endpoints, and enhanced participant retention.
However, despite the successful conduct of several MCTs for regulatory submission, the MCT model has yet to be widely adopted. This is due in part to presumed legal and regulatory issues in the U.S., such as the complexity of navigating different state medical licensing laws, patient and investigator oversight, and issues with the drug supply chain of custody.
In July 2017, CTTI convened a multi-stakeholder expert meeting to explore the legal and regulatory challenges inhibiting more widespread practice of MCTs, and to identify potential solutions. A meeting summary is now available highlighting main points from the discussion, including the following:
- Highly varied state laws and regulations need to be thoroughly understood and recorded in an accessible location, e.g., a public database.
- Guidance from regulatory bodies is needed to define principal investigators’ responsibilities regarding patient care oversight and potential delegation of activities,
- Trials with a mobile clinical component should not be held to higher standards than traditional trials.
- Patients and regulatory agencies should be engaged in trial design early in the development phase.
CTTI’s MCT Legal and Regulatory Project team will leverage themes and specific solutions from the multi-stakeholder discussions to develop recommendations and tools that will help address legal and regulatory challenges currently associated with MCTs.
This work is part of CTTI’s MCT Program, which is also exploring other topics and barriers related to planning for and conducting MCTs.
CTTI Update: Advancing the Use of Mobile Health Technology to Transform Clinical Trials
Mobile technology has the potential to revolutionize clinical trials, but putting mobile clinical trials (MCTs) into action requires consideration of issues such as data integrity, legal and regulatory barriers, scientific and technological challenges, and perspectives of potential trial participants, investigators, and site staff. For the past two years, CTTI’s MCT Program has systematically explored barriers to MCTs, gathered evidence, and convened multiple stakeholders to tackle these issues.
By developing best practices on how to effectively integrate mobile technologies into clinical trials for regulatory submissions, CTTI intends to promote quality, efficient, and more cost-effective research. Recent developments include the following:
- CTTI’s Recommendations for Developing Novel Endpoints Generated by Mobile Technology for Use in Clinical Trials were released in June and are prominently featured in a recent mHealth Action Plan from the Duke-Margolis Center for Health Policy.
- CTTI’s recommendations are accompanied by several practical tools and case examples that describe the process of novel endpoint development for MCTs.
- A survey of potential research participants, as well as in-depth interviews with site investigators on issues related to MCTs, are underway. Analysis of these results will begin this fall and will inform recommendations to overcome barriers to the use of mobile technology in clinical trials as perceived by key stakeholders.
- CTTI presented “Turning Opportunity Into Reality: Real Data and Practical Guidance for Incorporating Mobile Technology in Clinical Trials” at the DPharm Disruptive Innovations Conference in September 2017. The presentation included—as it relates to mobile technology—the patient perspective, tips for dealing with large volumes of data, and engaging with the FDA during trial design, execution, and monitoring.
- Two expert meetings were held in 2017, one on legal and regulatory issues and one on scientific and technological challenges affecting MCTs. Official CTTI recommendations and resources on these topics will be issued in early 2018
CTTI Advances Mobile Clinical Trials at DPharm 2017
Mobile technology has been used in a variety of ways, but it has yet to be widely incorporated into clinical trials—despite its great potential to increase trial quality and efficiency. As part of its Mobile Clinical Trials (MCT) Program, CTTI has implemented four projects to identify and address the challenges related to planning for and conducting clinical trials that use mobile technology. This week, CTTI will present strategies and insights from the program at DPharm 2017 Conference, held in Boston on September 6-8.
At the 2-day conference themed “Disruptive Innovations US,” CTTI will bring together key industry leaders who are collaborating on recommendations for the use of mobile technologies in regulated clinical trials after the point of informed consent. This will include—as it relates to mobile technology—the patient perspective, tips for dealing with large volumes of data, and engaging with the FDA during trial design, execution, and monitoring.
Presentation: Turning Opportunity Into Reality: Real Data and Practical Guidance for Incorporating Mobile Technology in Clinical Trials
Date & Time: Friday, Sept. 8, 2:30–3:10 p.m.
Moderator: Komathi Stem (monARC Biotech)
Panelists: Ray Dorsey (University of Rochester), Cindy Geoghegan (Patient and Partners), and Gary Grabow (Genentech)
Also, at DPharm’s pre-conference program, “Mobile in Clinical Trials,” CTTI will share recommendations that clarify the pathway for developing novel endpoints from data generated using mobile technology in clinical trials.
Presentation: CTTI Findings Are in From Their Mobile Clinical Trials Program: Recommendations on Novel Endpoints
Date & Time: Wednesday, Sept. 6, 9:20–9:40 a.m.
Presenter: Jennifer Goldsack (CTTI)
In addition to Novel Endpoints, CTTI is also working on recommendations to address challenges and opportunities related to Mobile Devices, Legal and Regulatory, and Stakeholder Perceptions, as part of the MCT Program.
Addressing Challenges of Using Mobile Devices in Clinical Trials
Mobile devices hold great potential to transform clinical trials by their ability to capture objective, real-world health data directly from study participants. While this technology shows promise to improve the quality and efficiency of medical product development, questions remain on how to successfully integrate mobile devices for data capture into clinical trials.
On June 16, 2017, CTTI convened a multi-stakeholder expert meeting to further refine solutions to the scientific and technological challenges preventing widespread adoption of mobile devices in clinical trials. A meeting summary is now available, including the following:
- The recommended best practices for studies using mobile devices for data capture do not differ significantly from many of the best practices that we already apply to clinical trials.
- Studies using mobile devices for data capture should not be held to higher standards than traditional trials.
- Efforts to leverage mobile devices for data capture should strive to engage patients in study design and endpoint selection.
The MCT Mobile Technologies Project focuses on using mobile devices in FDA-regulated clinical trials after initial participant consent—in other words, the use of devices for data capture rather than for recruitment, retention, or as the study intervention. The project team will use findings from the expert meeting to develop recommendations and tools that guide the adoption of mobile devices for data capture in clinical trials.