September 14, 2018: Advancing the Use of Mobile Technologies for Data Capture & Improved Clinical Trials (John Hubbard, PhD, Barry Peterson, PhD, Cheryl Grandinetti, PharmD)

Speakers

John Hubbard, PhD
Healthcare Strategic Advisory Board
Genstar Capital

Barry Peterson, PhD
Independent Consultant

Cheryl Grandinetti, PharmD
Office of Compliance, Office of Scientific Investigations, Division of Clinical Compliance Evaluation
Center for Drug Evaluation and Research
Food and Drug Administration

Topic

Advancing the Use of Mobile Technologies for Data Capture & Improved Clinical Trials

Keywords

Clinical trials; Mobile health technologies; Clinical Trials Transformation Initiative; CTTI; FDA; Data integrity

Key Points

  • The goal of CTTI’s Mobile Clinical Trials program is to develop evidence-based recommendations that affect the widespread adoption and use of mobile technology in clinical trials for regulatory submission.
  • Potential benefits of using mobile technology include higher quality, patient-centric endpoints and fewer barriers to participation in clinical trials.
  • Data access issues to consider before selecting a mobile technology include:
    • How will the data generated by the mobile technology be accessed and used by the manufacturer?
    • What data will be provided by the manufacturer to the sponsor?
  • The mobile era creates new data security demands.

Discussion Themes

CTTI’s recommendations aim to help sponsors determine the right device to use, how to write the protocol for remote data capture, and how to protect and analyze the data.

Know what you want to measure before selecting the mobile technology. The appropriateness of the selection should be justified through verification and validation processes.

Ensure the authenticity, integrity, and confidentiality of data over its entire lifecycle.

To reduce risk in large trials, conduct feasibility studies before full implementation.

Visit CTTI for more recommendations and resources for mobile clinical trials.

 

Tags

@CTTI_Trials, @PCTGrandRounds, #MobileTech, #pctGR

August 9, 2018: New Interview with Dr. David Shurtleff about the NIH Collaboratory

In a recent video interview, Dr. David Shurtleff, acting director of the National Center for Complementary and Integrative Health (NCCIH), discusses the unique work of the Collaboratory in bringing together multiple NIH Institutes and Centers to develop best practices, methods, guidance, and tools for conducting rigorous pragmatic research.

“The Collaboratory has been transformative in how it’s developed a novel, supportive infrastructure to conduct pragmatic research in a way that allows it to be embedded within healthcare systems.” David Shurtleff, PhD

June 14, 2018: NIH Collaboratory Members to Host Session on Embedded PCTs at AcademyHealth Annual Research Meeting

The upcoming AcademyHealth Annual Research Meeting, June 24-26, 2018, will feature a special session on Embedding Pragmatic Clinical Trials in Health Care Systems: Trials and Tribulations, Monday, June 25, at 5:15 pm, hosted by members of the Health Care Systems Interactions Core Working Group:

Chair: Cathy Meyers, MD, NIH/NICCH

Discussant: Eric B. Larson, MD, MPH, Kaiser Permanente Washington Health Research Institute

Speakers: Vince Mor, PhD, Brown University School of Public Health; Greg Simon, MD, MPH, Kaiser Permanente Washington Health Research Institute; and Lynn DeBar, PhD, MPH, Kaiser Permanente Washington Health Research Institute

Held at the Washington State Convention Center in Seattle, this large gathering of health services researchers and policy analysts will include workshops, poster and podium sessions, emerging issues panels, policy roundtables, and special topic sessions. Other NIH Collaboratory members planning to present at the meeting include Leah Tuzzio, MPH, Jerry Jarvik, MD, MPH, Miguel Vazquez, MD, Kathryn James, MPH, Gloria Coronado, PhD, and Beverly Green, MD, MPH.

 “Attendees will learn about the challenges of pilot testing, studying patient-reported outcomes, using existing data, and the multiple levels of implementation in dynamic systems.” – Leah Tuzzio, Kaiser Permanente Washington Health Research Institute

For details, visit the meeting event site at http://www.academyhealth.org/events/site/2018-annual-research-meeting.

June 4, 2018: New Article Explores Misleading Use of the Label “Pragmatic” for Some Randomized Clinical Trials

A recent study published in BMC Medicine found that many randomized controlled trials (RCTs) self-labeled as “pragmatic” were actually explanatory in nature, in that they assessed investigational medicines compared with placebo to test efficacy before licensing. Of the RCTs studied, one-third were pre-licensing, single-center, or placebo-controlled trials and thus not appropriately described as pragmatic.

Appropriately describing the design and characteristics of a pragmatic trial helps readers understand the trial’s relevance for real-world practice. The authors explain that RCTs suitably termed pragmatic compare the effectiveness of 2 available medicines or interventions prescribed in routine clinical care. The purpose of such pragmatic RCTs is to provide real-world evidence for which interventions should be recommended or prioritized.

The authors recommend that investigators use a standard tool, such as the CONSORT Pragmatic Trials extension or the PRECIS-2 tool, to prospectively evaluate the pragmatic characteristics of their RCTs. Use of these tools can also assist funders, ethics committees, and journal editors in determining whether an RCT has been accurately labeled as pragmatic.

The BMC Medicine article cites NIH Collaboratory publications by Ali et al. and Johnson et al., as well as the Living Textbook, in its discussion of pragmatic RCTs and the tools available to assess their relevance for real-world practice.

“Submissions of RCTs to funders, research ethics committees, and peer-reviewed journals should include a PRECIS-2 tool assessment done by the trial investigators. Clarity and accuracy on the extent to which an RCT is pragmatic will help [to] understand how much it is relevant to real-world practice.” (Dal-Ré et al. 2018)

April 24, 2018: NIH Collaboratory ePCT Training Workshop Resources Now Available

Interested in learning more about the complexities of designing and conducting embedded pragmatic clinical trials (ePCTs)? Now you can view and download the slide sets and handouts from the first NIH Collaboratory ePCT Training Workshop. Held on February 20-21, 2018, at Duke University, this pilot workshop offered training in the conduct of ePCTs to 27 mid- and senior-level investigators. Also present at the workshop were representatives from the NIH, NIH Collaboratory Demonstration Projects and Core Working Groups, and Coordinating Center staff. NIH Collaboratory subject matter experts used experiences from the program and chapters from the Living Textbook to develop workshop content.

The workshop comprised 10 topics across the 2 days and included hands-on exercises and case studies from the Demonstration Projects illustrating the particulars of designing and conducting ePCTs and providing resources for planning and overcoming challenges. Among the topics covered were Engaging All Stakeholders and Aligning with Healthcare System Partners, Design and Analytic Considerations, Pilot and Feasibility Testing, and Developing a Compelling Application. The NIH Collaboratory will be making additional training resources available to the public in the near future.

The training was funded as an NIH Roadmap Initiative 3U54AT007748-05S2.

April 9, 2018: PPACT Study Design Paper Published

Congratulations to Dr. Lynn DeBar and the investigators of the Collaborative Care for Chronic Pain in Primary Care pragmatic trial for recently publishing their study design paper. One of the NIH Collaboratory Demonstration Projects, the trial is designed to test whether a primary care–based behavioral intervention the Pain Program for Active Coping and Training (PPACT)—will provide a “more effective, safer, and more satisfactory alternative to opioid-based chronic pain treatment” than usual care for patients on chronic opioid treatment (Debar et al 2018). Learn more about this innovative trial in the article in Contemporary Clinical Trials. You can also download a trial snapshot.

Full Citation: DeBar L, Benes L, Bonifay A, et al. Interdisciplinary team-based care for patients with chronic pain on long-term opioid treatment in primary care (PPACT) – Protocol for a pragmatic cluster randomized trial. Contemporary Clinical Trials. 2018;67:91-99. doi:10.1016/j.cct.2018.02.015

January 19, 2018: New Research Methods Resources Website on Group- or Cluster-Randomized Studies

The National Institutes of Health (NIH) Office of Extramural Research has released new clinical trial requirements for grant applications and contract proposals due on or after January 25, 2018. In anticipation of these new requirements, the NIH modified the Application Guide and the Review Criteria to address methodological problems common to many clinical trials. As group- or cluster-randomization designs are increasingly common in both basic and applied research, the new Application Guide includes links to the new Research Methods Resources website, which provides resources for investigators considering these group- or cluster-randomized designs, including lists of NIH webinars, key references, and statements to help investigators prepare sound applications and avoid methodological pitfalls.

December 7, 2017: Dr. Greg Simon Explains Individual, Cluster, and Stepped-Wedge Randomization in a New Prop Video

In a new video in the Living Textbook, Dr. Greg Simon describes the differences between individual, cluster, and stepped-wedge randomization using props, including marbles, Play-Doh, and glassware.

“In the end, it’s all about randomly assigning who gets which treatment, or who gets which treatment when, so that we’re able to make some un-biased judgement about which treatment is really better.” —Greg Simon, MD

November 20, 2017: NIH Collaboratory Core Working Group Interviews: Reflections from the Regulatory/Ethics Core

We recently asked Drs. Jeremy Sugarman and Kevin Weinfurt, Co-chairs of the Regulatory/Ethics Core, to reflect on the first 5 years of the Core as well as on the challenges ahead. The regulatory and ethical landscape for pragmatic clinical trials was not well defined when the Core began 5 years ago, and the Core helped to map and navigate the emerging landscape to enable the implementation of Demonstration Projects in ways that satisfied ethical and regulatory criteria.

“The Core’s work has led to the creation of a substantial body of scholarship contributing to the ongoing policy and ethics debates about pragmatic clinical trials.” – Drs. Sugarman and Weinfurt

Download the interview (PDF).

October 18, 2017: NIH Collaboratory Core Working Group Interviews: Reflections from the Biostatistics and Study Design Core

We recently asked Dr. Liz DeLong, Chair of the Biostatistics and Study Design Core, to reflect on the first 5 years of the Core’s work and the challenges ahead. She says the biggest impact of the Core has been working with the individual Demonstration Projects to provide a sounding board to discuss statistical challenges. Further, Core members have contributed to new knowledge through manuscripts that address key methodological issues related to pragmatic clinical trials. She’s hoping the Core will continue to push the boundaries of statistical methods in the coming years.

“The statisticians on the individual trials have not only developed excellent statistical methods for their own studies, but also contributed substantively to the Core.” Dr. Liz DeLong

Download the interview (PDF).