Patient Centered Outcomes Research (PCOR) Project Workshop

May 18, 2018Clinical, Patient Centered Outcomes Research (PCOR), Regulatory Decision, SPI Guiding Principles

Patient Introduction

In May 2018, MDIC convened a public workshop to review and discuss the outcomes from MDIC’s Patient Centered Outcomes Research (PCOR) project. The workshop featured presentations and panel discussions involving experts from across the medical device ecosystem, including representatives of the Michael J. Fox Foundation for Parkinson’s Research, FDA CDRH, MIT, and RTI Health Solutions. These organizations all collaborated with MDIC on this effort to investigate how patient preferences could be used within medical device trial design.

By reviewing lessons learned from this project, the MDIC PCOR public workshop focused on how incorporating patient perspectives on risk tolerance into the process of developing clinical trial design could accelerate delivery of lifesaving technologies. Patients may be interested in reviewing the workshop summary, as well as various slide presentations and video recordings from the workshop, to better understand the concept of patient-centered outcomes research and how patient preference information can be used to expedite successful development of medical devices.

Summary:

MDIC’s May 18, 2018 public workshop provided an overview of the Patient Centered Outcomes Research (PCOR) project focused on Parkinson’s disease (PD) and an opportunity for experts from across the medical device community to assess how the lessons learned from this effort can be applied broadly in the field of medical device development. Workshop participants (including representatives from across healthcare stakeholder groups) focused on the central objective of the PCOR project: investigating how patient preferences could be used to set statistical significance levels in a clinical trial design. PD was selected as a useful test case for exploring the most important benefits and risk to patients and developing a model to design clinical trials based on disease-specific patient preferences. While this project focused on a model for using patient-preferences to set significance levels in a hypothetical clinical trial, the overarching goal was to test a model for optimizing actual clinical trial designs to benefit patients with a variety of disease and health conditions.

During the one-day workshop, participants discussed results from the four specific aims of the PCOR project, which were to: (1) identify the outcomes important to patients, family members, and caregivers; (2) design and conduct a patient preference assessment study; (3) design methods for clinical trials approval based on explicit patient input; and (4) assess medical device stakeholder acceptance of clinical trial designs based on patient preference.

MDIC PCOR Project: From P-Values to Patient Values in Parkinson’s Disease Workshop Summary

Following the public workshop on the PCOR project, MDIC released a report summarizing the key topics and points that were discussed. This summary compiles a succinct review of the presentations from the May 18, 2018 event and provides a useful resource for any stakeholder interested in evaluating the PCOR project and the PD pilot for insights about how to incorporate patient preference information into clinical trial design.

Jump directly to a specific note within the Workshop Summary:

Incorporating Patient Preferences into Medical Device Clinical Trials

The opening presentation of the PCOR public workshop set the stage for the entire meeting by posing the question: What if patients’ urgency for new therapeutic options and tolerance of uncertainty are considered when designing and sizing clinical trials? MDIC SPI Program Director Stephanie Christopher provided an overview of the purpose of the PCOR project, which was to design a new approach for developing and testing a method to incorporate patient perspectives on benefit-risk considerations as an explicit means to set significance levels in clinical trial design. This set of slides focuses on how unmet need and patients’ tolerance of uncertainty impact clinical trial design.

Moving from p-Values to Patient Values – Keynote Address

During the first keynote address of the workshop, MIT’s Andrew Lo discussed the history and meaning of p-values, the role of ethics and trade-offs in clinical trial design and FDA approvals, and a rationale for why we would want to examine how we set p-values. This slide deck provides a summary of Dr. Lo’s presentation, in which he also provided an overview of the Bayesian decision analysis model used in the PCOR project to inform the design for a clinical trial for hypothetical PD device.

Keynote Video:

Please note: due to technical difficulties, this is only a partial recording and does not include the introduction and initial slides.

Andrew Lo, PhD, Charles E. and Susan T. Harris Professor of Finance; Director, Laboratory for Financial Engineering, MIT

A Vision for Partnering with Patients: A Conversation with the Michael J. Fox Foundation for Parkinson’s Research – Keynote Address

The Michael J. Fox Foundation (MJFF) played a key role as a partner in MDIC’s PCOR project, helping to design and execute the project and engaging expert PD patients from its network to serve as advisors to the effort. During this keynote presentation, Sohini Chowdhury of MJFF described the organization’s vision for partnering with patients, emphasizing the importance of patient data to ignite a virtuous cycle of patient-centered research that can help drive:

  • Funding decisions
  • Education and public policy efforts
  • Payer relationship and industry collaboration

MJFF Patient Council representatives Margaret Sheehan and Anna Donnelley joined the conversation to present patient viewpoints within the discussion.

Keynote Video

  • Sohini Chowdhury, Deputy CEO, The Michael J. Fox Foundation for Parkinson’s Research
  • Margaret Sheehan, MJFF Patient Council
  • Anna Donnelley, MJFF Patient Council

PCOR Project Aim 2 Breakout Session: Designing and Conducting the Patient Preference Study

This breakout session focused on Aim 2 of the PCOR project, reviewing the process by which a patient preference survey for Parkinson’s disease was developed and the results were analyzed. This slide presentation reviews how attributes for the patient preference survey were selected and tested, including an overview of advantages and limitations of the threshold technique that was used. The slide presentation also reviews the demographic characteristics of the survey respondents and provides and overview the survey data.

PCOR Project Aim 3 Breakout Session: Designing Methods for Clinical Trials Based Explicitly on Patient Input

This breakout session focused on Aim 3 of the PCOR project, reviewing the methods used by the MIT Laboratory for Financial Engineering to incorporate patient preferences into statistical significance thresholds in clinical trial design. Shomesh E. Chaudhuri explained how the MIT team used a patient value model that provides the foundation for a Bayesian decision analysis (“BDA”) and illustrated the framework’s application in a case study for a neurostimulator device.

This slide presentation provides an overview of the process for developing a patient centered randomized clinical trial for Parkinson’s disease.

The clinical trial development process includes:

  • Patient value model
  • Bayesian decision analysis
  • Assumptions for trial design
  • Neurostimulator case study
  • The regulatory, patient, and industry perspective

Following the public workshop on the PCOR project, MDIC released a session summary highlighting the key topics and points that were discussed.

Aim 3 Discussion Video

Moderator: Shomesh Chaudhuri, MIT

  • Martin Ho, FDA – CDRH
  • Andrew Lo, MIT
  • Kevin Kwok, MJFF
  • Edward Karst, Abbott

Wrap-up and Closing Panel- What are the Implications of the Patient-Centered Approach to Clinical Trials?

During this breakout session recap and closing panel, experts summarized key implications of a patient-centered approach to clinical trial design, focusing on key aspects of the Parkinson’s disease PCOR project, its outcomes, and applicable lessons learned.

Video Summary:

Wrap-up and summary from the concurrent sessions 

  • Annie Saha, FDA CDRH
  • Shomesh Chaudhuri, MIT
  • Brett Hauber, RTI Health Solutions

Panel discussion: What are the implications of this patient-centered approach to clinical trials?

  • Annie Saha, FDA CDRH
  • Shomesh Chaudhuri, MIT
  • Brett Hauber, RTI Health Solutions
  • Margaret Sheehan, MJFF Patient Council
  • Anna Donnelly, MJFF Patient Council
  • Andrew Lo, MIT
  • Greg Molnar, University of Minnesota
  • Lauren McLaughlin, MJFF
  • Owen Faris, FDA CDRH