Project: Patient Centered Outcomes Research

Over two years of collaborative work among various stakeholders, the MDIC Patient Centered Outcomes Research (PCOR) project developed and tested a method for incorporating patient preference information as an explicit means to set significance levels in clinical trial design. While the pilot focused on a specific disease state, Parkinson’s disease (PD), the method was generalizable to other diseases and has the potential to remove barriers to therapy access by giving patients a pathway to breakthrough, lifesaving technologies based on their risk tolerance.

Full Summary

For chronic, debilitating, and terminal illnesses with no effective treatments, the standard statistical threshold for determining therapeutic effectiveness in clinical trials may be too conservative and may not reflect patients’ perspectives on the trade-off between the risk of endorsing an ineffective therapy and the risk of rejecting an effective therapy.

Clinical trials designed and sized with consideration of patient preference information may expedite the development process while incorporating patient perspectives. By designing clinical trials that reflect patients’ urgency and risk tolerance, scarce resources can be allocated more efficiently, speeding up progress in bringing forward innovative therapies.

The specific aims of the project focusing on Parkinson’s disease 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 based on explicit patient input
  4.  Assess medical device stakeholder acceptance of clinical trial designs based on patient preference, with a particular focus on regulatory and reimbursement stakeholders

This project yielded several important outcomes, including incorporation of patient preference information as an explicit means to set significance levels in clinical trial design. While this project focuses on a specific disease state (Parkinson’s disease), the method used may be generalizable to other diseases. Using this method to incorporate patient preferences into clinical trial design will address the longstanding conundrum that, in an enterprise devoted to easing the burden of disease, patients often have little or no direct input into the study design process.

Articles in

Patient Centered Outcomes Research (PCOR)

Patient-Centered Identification of Meaningful Regulatory Endpoints for Medical Devices to Treat Parkinson’s Disease (PCOR Project Aim 1 Paper)

Patient-Centered Identification of Meaningful Regulatory Endpoints for Medical Devices to Treat Parkinson’s Disease (PCOR Project Aim 1 Paper)

A growing body of literature has developed identifying outcomes that matter to patients. The study that these published articles (Aims 1, 2 & 3 of MDIC’s PCOR project) are based on demonstrates an approach to identifying outcomes of medical devices for Parkinson’s...

Parkinson’s Patients’ Tolerance for Risk and Willingness to Wait for Potential Benefits of Novel Neurostimulation Devices: A Patient-Centered Threshold Technique Study (PCOR Project Aim 2 paper)

Parkinson’s Patients’ Tolerance for Risk and Willingness to Wait for Potential Benefits of Novel Neurostimulation Devices: A Patient-Centered Threshold Technique Study (PCOR Project Aim 2 paper)

A growing literature has developed identifying outcomes that matter to patients. The study that these published articles (Aims 1, 2 & 3 of MDIC’s PCOR project) are based on demonstrates an approach to identifying outcomes of medical devices for Parkinson’s disease...