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

September 10: NIH Collaboratory Launches New ePCT Training Resources

The NIH Collaboratory is pleased to announce new training resources available on the Living Textbook. These resources are being shared with the research community to provide guidance about building partnerships with health systems and overcoming the challenges of conducting embedded pragmatic clinical trials (ePCTs). The materials reflect the knowledge, insight, and best practices acquired by the NIH Collaboratory program and its Demonstration Projects.

Resources include:

  • Materials from the inaugural ePCT Training Workshop held in February 2018 to provide training to mid- and senior-level investigators interested in conducting ePCTs
  • A slide presentation of the NIH Collaboratory’s goals and organizational structure along with a brief introduction to each Demonstration Project
  • An infographic introducing the elements of the NIH Collaboratory program and the value of engaging in pragmatic research
  • A slide presentation and worksheet on essential things to think about and do when designing, conducting, and disseminating ePCTs
  • An introductory video from NIH Collaboratory leadership on the rationale and aims for the ePCT training resources

“The workshop reinforced that there is a demand for these kinds of training opportunities across the clinical research community.” Lesley Curtis, PhD

Visit the new training resources webpage.

September 7, 2018: Spotlight on a New Demonstration Project: HiLo

Kidney transplantation is the preferred treatment for patients with end-stage renal disease (ESRD), but an insufficient organ supply renders dialysis the only viable treatment option for most patients. Though clinical outcomes among patients receiving dialysis have improved modestly in recent years, annual rates of hospitalization and mortality remain unacceptably high, and quality of life is poor. Poor outcomes are driven primarily by increased risk of cardiovascular disease (CVD), but interventions that improve outcomes in the general population by targeting traditional CVD risk factors have mostly failed in patients with ESRD. Current clinical practice guidelines advocate aggressive treatment of high serum phosphate to near-normal levels using dietary phosphate binders and restrictive diets. The benefits of this approach, however, are unproven, the optimal serum phosphate target remains unknown, and potential harms of aggressive treatment have not been definitively identified.

The Pragmatic Trial of Higher vs. Lower Serum Phosphate Targets in Patients Undergoing Hemodialysis (HiLo) plans to address these clinically important questions in a large, pragmatic, cluster-randomized trial that will evaluate the effects of liberalizing the serum phosphate target (“Hi”) versus maintaining aggressive phosphate control (“Lo”) for patients receiving treatment with maintenance hemodialysis.

 “The question at hand is something we grapple with on a daily basis in every dialysis facility across the country. Either answer will be important new information that will help us do a better job taking care of patients and hopefully improve their quality of life.”

HiLo is led by Myles Wolf, MD, of Duke University with support from the National Institute of Diabetes and Digestive and Kidney Diseases. Read more about HiLo.

August 20, 2018: Spotlight on a New Demonstration Project: EMBED

The NIH Collaboratory is pleased to introduce the Pragmatic Trial of User-Centered Clinical Decision Support to Implement Emergency Department-Initiated Buprenorphine for Opioid Use Disorder (EMBED). Led by co–principal investigators Dr. Ted Melnick and Dr. Gail D’Onofrio of Yale University, and supported by the National Institute on Drug Abuse, EMBED is a pragmatic, multicenter, group-randomized trial that will implement and evaluate a user-centered clinical decision support tool to facilitate the initiation of buprenorphine/naloxone therapy for opioid use disorder in emergency departments.

The intervention consists of computerized treatment guidance for emergency department physicians and is embedded in the existing care delivery workflow. By conducting the study under real-world conditions and employing passive collection of structured data from the electronic health record, EMBED will use an innovative approach to address public health concerns about opioid use in the United States. Watch a video interview with Dr. Melnick, and read more about EMBED.

“This is an area where there is already good efficacy data for the practice of ED-initiated buprenorphine treatment for patients with opioid use disorder, but the practice is not part of routine care right now.” — Ted Melnick, MD, MHS

 

EMBED is 1 of 6 new large-scale clinical trials launched by the NIH Collaboratory in 2018. The Demonstration Projects are multicenter pragmatic trials that engage healthcare delivery systems in research partnerships to gather real-world evidence and answer clinical questions of major public health importance. Learn more about the Demonstration Projects.

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

July 30, 2018: Learn More About the New NIH Collaboratory Demonstration Projects

Check out the new program materials from the Collaboratory:

Study information for the 6 new UG3 Demonstration Projects:

  • ACP PEACE: Improving Advance Care Planning in Oncology: A Pragmatic, Cluster-Randomized Trial Integrating Patient Videos and Clinician Communication Training
  • EMBED: Pragmatic Trial of User-Centered Clinical Decision Support to Implement Emergency Department-Initiated Buprenorphine for Opioid Use Disorder
  • GGC4H: Pragmatic Trial of Parent-Focused Prevention in Pediatric Primary Care: Implementation and Adolescent Health Outcomes in Three Health Systems (Guiding Good Choices for Health)
  • Nudge: Personalized Patient Data and Behavioral Nudges to Improve Adherence to Chronic Cardiovascular Medications
  • PRIM-ER: Primary Palliative Care for Emergency Medicine
  • HiLo: Pragmatic Trial of Higher vs. Lower Serum Phosphate Targets in Patients Undergoing Hemodialysis

In addition, the May 2018 Steering Committee Meeting materials are now available, including presentations from the UG3 and UH3 Demonstration Projects and the full-day intensive workshop “Embedded Pragmatic Clinical Trials of Therapeutic A vs. B Interventions.”

July 23, 2018: New Report Summarizes Patient-Reported Health Data and Metadata Standards in the ADAPTABLE Trial

A new report in the Living Textbook describes results of a literature review of data standards and metadata standards for variables of interest to the ADAPTABLE trial. Based on the review, the authors recommend standards for ADAPTABLE, also known as the Aspirin Study, which is the first major randomized comparative effectiveness trial to be conducted by the National Patient-Centered Clinical Research Network (PCORnet). The trial aims to identify the optimal dose of aspirin therapy for secondary prevention in atherosclerotic cardiovascular disease.

Because the ADAPTABLE trial relies on patients to report key information at baseline and throughout follow-up, it represents a unique opportunity to develop, pilot, and evaluate methods to validate and integrate patient-reported information with data obtained from electronic health records (EHRs). In 2016, the National Institutes of Health implemented a project with the goal of using the ADAPTABLE study to develop methods to (1) assess the quality of patient-reported data and (2) integrate the data with existing EHR data. It is hoped that this project will inform future efforts to synthesize potentially inconsistent data from patient-reported and EHR sources and identify opportunities to streamline data.

Download the report.

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)