Epidemiology, health policy and outcomes

CRC: Optimizing RCTs in Rheumatology: Past, Present and Future

Saturday, November 21, 2020

10:00 AM – 4:45 PM ET

Moderator(s)

Jeffrey Katz, MD, MSc

Brigham and Women's Hospital
Michael LaValley, PhD

Boston University School of Public Health
Elena Losina, PhD

BWH
Kenneth Saag, MD, MSc

The University of Alabama at Birmingham

Speaker(s)

Jamie Collins, PhD

Brigham and Women's Hospital
RG

Robert Glynn, PhD

Brigham & Women's Hospital
NN

Nikolay Nikolov, MD

FDA
Jeffrey Katz, MD, MSc

Brigham and Women's Hospital
Christopher Swearingen, PhD

Samumed LLC
Eleanor Murray, DSc

Boston University
Maria Suárez-Almazor, MD, PhD

University of Texas MD Anderson Cancer Center
Kelli Allen, PhD

University of North Carolina
Robert Carter, MD

NIAMS/NIH
Katherine Liao, MD, MPH

Brigham and Women's Hospital
Yuchiao Chang, PhD

MGH

The 2020 ACR Clinical Research Conference is focused on optimizing randomized clinical trials (RCTs) in rheumatology and musculoskeletal disorders. The RCTs are providing the highest level of evidence, while they take a lot of time and carry nontrivial costs. Unfortunately, a number of such trials are inconclusive due to design deficiencies. The conference will feature thought leaders from the fields of epidemiology, outcomes research, biostatistics, clinical science and rheumatology, who will highlight the modern analytic toolset available to scientists who plan to design and conduct RCTs in rheumatology-related fields.

In addition, faculty will highlight successful examples of past RCTs. They will discuss the means of expediting the implementation of RCTs into clinical practice by using pragmatic trial and implementation science designs, as well as how to utilize data science to expedite RCT enrollment using electronic medical records. They will thoroughly review modern statistical methods for RCTs.

Learning Objectives:

Distinguish different designs of randomized clinical trials in rheumatology research
Review differing FDA and NIH perspectives as used to assess the potential impact of RCT findings
Describe ways to use biomarkers as outcomes of randomized clinical trial data
Appraise the heterogeneity of treatment effects
Utilize causal inference methods to derive proper inferences of long-term follow-up of RCT samples

Presentations:

10:00 AM – 10:25 AM ET

Navigating the Winding Road of RCT Designs: Superiority, Non-Inferiority, Adaptive Designs
Jamie Collins, PhD – Brigham and Women's Hospital
10:25 AM – 10:30 AM ET

Q&A
10:30 AM – 10:55 AM ET

Modern Statistical Toolset: Can We Fix Design Deficiencies with Statistical Tools?
Michael LaValley, PhD – Boston University School of Public Health
10:55 AM – 11:00 AM ET

Q&A
11:00 AM – 11:25 AM ET

Road to Success: Reflections of NIH Reviewer
Robert Glynn, PhD – Brigham & Women's Hospital
11:25 AM – 11:30 AM ET

Q&A
11:30 AM – 11:55 AM ET

Surrogate Outcomes & Indirect Comparisons: FDA Perspective
Nikolay Nikolov, MD – FDA
11:55 AM – 12:00 PM ET

Q&A
12:00 PM – 12:15 PM ET

Break
12:15 PM – 12:40 PM ET

Outcomes Selection: The Balance Between Objective and Subjective (Patient-Reported) Outcomes
Jeffrey Katz, MD, MSc – Brigham and Women's Hospital
12:40 PM – 12:45 PM ET

Q&A
12:45 PM – 1:10 PM ET

Long Run for a Short Gain: Pros and Cons of a Short Trial Duration
Christopher Swearingen, PhD – Samumed LLC
1:10 PM – 1:15 PM ET

Q&A
1:15 PM – 1:40 PM ET

How to Turn 'More' Into 'Better': Use Causal Inference Methods to Draw Proper Inferences Over the Long Term
Eleanor Murray, DSc – Boston University
1:40 PM – 1:45 PM ET

Q&A
1:45 PM – 2:00 PM ET

Break
2:00 PM – 2:25 PM ET

The Role of Mechanistic RCTs
Maria Suárez-Almazor, MD, PhD – University of Texas MD Anderson Cancer Center
2:25 PM – 2:30 PM ET

Q&A
2:30 PM – 2:55 PM ET

Need for Implementation RCTs in Rheumatology: Past, Present and Future
Kelli Allen, PhD – University of North Carolina
2:55 PM – 3:00 PM ET

Q&A
3:00 PM – 3:25 PM ET

NIAMS vision for RCT in rheumatic and musculoskeletal diseases: past, current and future
Robert Carter, MD – NIAMS/NIH
3:25 PM – 3:30 PM ET

Q&A
3:30 PM – 3:45 PM ET

Break
3:45 PM – 4:10 PM ET

Challenges and Opportunities in Using ML & NLP to Define Eligibility Pool
Katherine Liao, MD, MPH – Brigham and Women's Hospital
4:10 PM – 4:15 PM ET

Q&A
4:15 PM – 4:40 PM ET

Addressing Heterogeneity of Treatment Effect: Proper Techniques for Slicing and Dicing
Yuchiao Chang, PhD – MGH
4:40 PM – 4:45 PM ET

Q&A