NCMRR/ REACT Workshop: the Future of Medical Rehabilitation Clinical Trials

On September 29-30, 2016, REACT cosponsored a clinical trials ;workshop with NIH/National Center for Medical Rehabilitation Research (NCMRR) and the REACT Center, at the National Institutes of Health headquarters in Bethesda, Maryland.

Clinical Trials Workshop Participants
A stellar group of researchers from around the nation gathered to take part in the NCMRR/REACT Clinical Trials Workshop, Sept. 29–30, in Bethesda, MD.
Left to right, front row: Dr. Alison Cernich, Dr. Edelle Field-Fote, Dr. Sean Savitz, Dr. Mona Fouad, Dr. Mary Ellen Michel, Dr. Carolee Winstein, Dr. Catherine Lang, Dr. Stephanie DeLuca
L to R, back row: Dr. John Whyte, Dr. Rob Motl, Dr. Kenneth Saag, Dr. Darcy Reisman, Dr. Anne Linblad, Dr. Gary Cutter, Carla Perna, Dr. Katie Schmitz, Dr. Marcas Bamman, Dr. Jennifer Lapsley-Stevens, Dr. John Chae, Dr. David Brienza, Dr. Daniel Corcos

This collaborative workshop aimed at bringing together researchers focused on clinical trials in medical rehabilitation to provide guidance for the field. It resulted in lively discussions about clinical trials in general, and specifically how clincial trials in medical rehabilitation are unique and can be most effective. Presentations also touched on how to support these trials, where criteria and interventions can prove challenging. Some concerns discussed included: How can we blind the trial; why do we need a comparison group and what to use; what outcome(s) to assess; or when to intervene or assess? A white paper addressing& some best practices in medical rehabilitation clinical trials will be released as a result of the conversations at this meeting.

As emphasized by the NIH, rehabilitation research is at an important juncture regarding clinical trials. Clinical trials research priorities and policies at NIH are evolving rapidly. The NIH recently published ;guidelines for receipt, review and management of all types of clinical trials.

This event was recorded and the videos of the workshop are available here:    Day 1        Day 2

The workshop resulted in lively discussions about clinical trials and specifically how clincial trials in medical rehabilitation are unique and can be most effective, as well as how to support these trials, which can be unique in their criteria and interventions. A white paper touching on some best practices in medical rehabilitation clincical trials will be released as a result of the conversations at this meeting.

See the event's full agenda below:

Thursday, September 29                                                    DAY 1 AGENDA

 

 

Morning Schedule: ON THE GROUND—Clinical Trials 101

Discussion Leaders

Session

Topic

 

 

8:00–8:20 a.m.

Welcome

Alison Cernich

NCMRR

Marcas Bamman, Ph.D.

University of Alabama at Birmingham (UAB)

8:20–8:30 a.m.

Intro

Mary Ellen Michel, Ph.D.

Gary Cutter, Ph.D.

1

 

8:30–10:00 a.m.

Design Considerations/Stages of Development?

  • Intention-to-treat principle
  • Control group: Standard of care? What is being tested? Active ingredient?
  • Is a run-­‐in valuable to wean the faint of heart? Pros and cons
  • Alternative and innovative trial designs (e.g., adaptive)
  • Are “n of 1” trials of any value in medical rehabilitation?
  • When is a crossover or delayed-­‐start design optimal/ appropriate?
  • Multi-­‐modal interventions
  • How many pilot data are enough (to move forward with a more definitive trial)?
  • Randomization and stratification
  • Blinding
  • Subgroup analyses; inference

Gary Cutter, Ph.D.

UAB

Edelle Field-Fote, Ph.D.

Shepherd Center

2

 

10:00–11:00 a.m.

Evolving Topics and Considerations in Clinical Trials

  • Efficacy vs. effectiveness
  • Pragmatic trials and adherence
  • Implementation science and group randomization
  • Multi-site standardization and IRB sharing
  • Rigor, reproducibility, and transparency
  • Evolving NIH priorities

Stephanie DeLuca, Ph.D.

Virginia Polytechnic Institute

Ken Saag, M.D., M.Sc.

UAB

 

11:00–11:15 a.m. Break

3

 

11:15–11:45 a.m.

Critical Staff

  • Research coordinators
  • Outcomes assessors
  • Who delivers intervention? Research staff vs. clinic staff
  • Fidelity of treatment; training

Carla Perna

UAB

Carolee Winstein, Ph.D., PT

University of Southern California

4

 

11:45 a.m.–12:30 p.m.

Recruitment and Retention

  • Monitoring
  • Planned enrollment/inclusion
  • Centralized support resource
  • Steps to take if recruitment falls below predicted rate
  • Retention and attrition

Jennifer Stephens-Lapsley, Ph.D., PT

University of Colorado Denver

Mona Fouad, M.D.

UAB

 

 

12:30–1:15 p.m. Lunch

 

Afternoon Schedule: OUTCOMES AND INNOVATION—Clinical Trials 201

Discussion Leaders

Session

Topic

 

 

5

 

1:15–2:00 p.m.

Data Collection and Management

  • Quality by design
  • Training
  • Case report forms and source
  • Data entry system choices
  • Electronic health record integration
  • Documentation
  • Data monitoring
  • Quality assurance

Darcy Reisman, Ph.D., PT

University of Delaware

 

Anne Lindblad, Ph.D.

EMMES Corporation

6

 

2:00–2:45 p.m.

Data and Safety Monitoring

  • Safety monitoring plan
  • Data & Safety Monitoring Board vs. Safety Monitoring Committee
  • Charter
  • Composition
  • Meeting format
  • Interim analysis
  • Reporting

Catherine Lang, Ph.D., PT

Washington University

Anne Lindblad, Ph.D.

EMMES Corporation

7

 

2:45–3:45 p.m.

Outcomes/Stages of Development

  • Phase II
  • Phase III
  • Clinically meaningful outcomes
  • Mechanistic/secondary outcomes

Daniel Corcos, Ph.D.

Northwestern University

 

John Whyte, M.D., Ph.D.

Moss Rehabilitation Research Institute

3:45–4:00 p.m. Break

8

 

4:00–4:45 p.m.

Follow-up: Sustaining Treatment Effects Over Time

  • Importance of retaining participants during follow-up: How do we integrate behavioral science?
  • Trajectory from pilot study to dissemination trials: When does it become appropriate to focus on sustainability?
  • How does a focus on sustainability alter our treatments/ interventions?
  • How long should we follow up to establish sustainability?

Katie Schmitz, Ph.D., M.P.H.

Pennsylvania State University

 

Rob Motl, Ph.D.

University of Alabama at Birmingham

 

4:45–5:30 p.m.

Discussion

 

 

 

Friday, September 30                                                           DAY 2 AGENDA

 

Morning Schedule: Clinical Trials 301

9

 

8:00–9:00 a.m.

Technology

  • What is the role of technology in modern rehabilitation trials?
  • Telemedicine/telerehabilitation
  • Robotics
  • Monitoring devices
  • Feedback devices (intervening and screening tools; outcome measures)
  • Validation
  • What have we learned so far?

David Brienza, Ph.D.

University of Pittsburgh

John Chae, M.D., M.E.

Case Western Reserve University

 

10

 

9:00–9:45 a.m.

Inter-individual Response Heterogeneity

  • Value of studying inter-individual response heterogeneity: what can we learn?
  • Predicting heterogeneity in the planning stages to optimize trail design and maximize yield
  • Sources of heterogeneity
  • Strategies to understand response heterogeneity
  • How to deal with evolving technology
  • Resource sharing: improving processes to maximize yield

Marcas Bamman, Ph.D.

UAB

 

Sean Savitz, M.D.

University of Texas

 

9:45-10:00 a.m. Break

 

 

 

10:00–11:30 a.m.

Review of Topics/Discussion

  • Design
  • Reproducibility
  • Critical staff
  • Recruitment and retention
  • Data entry and management
  • Data and safety monitoring
  • Outcomes
  • Follow-up
  • Technology
  • “Omics” and heterogeneity

Recommendations to investigators

Recommendations to NCMRR

 

 

11:30 a.m.–12:30 p.m.

White Paper Outline