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...
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.
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:
- Identify the outcomes important to patients, family members, and caregivers
- Design and conduct a patient preference assessment study
- Design methods for clinical trials based on explicit patient input
- 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 could be leveraged to other diseases. Using this method to incorporate patient preferences into clinical trial design will address the longstanding challenge that, in an enterprise devoted to easing the burden of disease, patients often have little or no direct input into the study design process.
Patient-Centered Outcomes Research (PCOR)
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...
Incorporating Patient Preferences in Clinical Trial Design and Research
This whitepaper was authored by Margaret Sheehan and Anne Cohn Donnelly, who live with Parkinson’s disease and have participated as Patient Scientists on the MDIC PCOR project team along with representatives from the Michael J. Fox Foundation for Parkinson’s Research...
MDIC PCOR Project: A brief summary of a novel method for incorporation of PCOR in clinical trial design
MDIC developed a novel method for incorporating patient-centered outcomes research (PCOR) into clinical trials designed with consideration of patient preference information. The goal is to expedite clinical trials and elevate the voices of smaller patient populations...
Patient Centered Outcomes Research (PCOR) Project Workshop
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,...