Developing Meaningful Endpoints for Pain Clinical Trials Workshop
Thu, 10/8/2020 - 10:00am - 3:30pm
Thu, 10/15/2020 - 11:00am - 1:30pm
View the Agenda PDF | 71.46 KB
View the Day 1 Videocast
View the Day 2 Videocast
View the Pre-workshop talks
More than 25 million Americans suffer from daily chronic pain, a highly debilitating medical condition that is complex and difficult to manage. In recent decades, there has been an overreliance on the prescription of opioids for chronic pain, contributing to a significant and alarming epidemic of opioid overdose deaths and addiction. Innovative scientific solutions to develop non-opioid, non-addictive alternative treatment options are thus urgently needed.
One of the goals of the Helping to End Addiction Long-term (HEAL) Initiative is to accelerate the discovery and preclinical development of new medications and devices to treat pain. Two recent NINDS workshops focused on (1) identifying endpoints in pre-clinical pain models and (2) discovering biomarkers to enhance pain therapy development. A logical next step is to identify the scientific gaps and development challenges in moving candidate therapeutics through proof-of-concept studies, early phase clinical trials and to later phases of development including regulatory approval.
One major challenge facing the development of non-opioid alternative pain medications in each pain condition is the heterogeneity in patient populations combined with high variability in individual responses to any given intervention. This is further compounded by the diversity of specific disorders associated with pain. Consequently, it is often difficult to define reliable endpoints, both within specific pain conditions and across pain conditions, for evaluation of novel therapeutics.
The workshop, being held on 2 days, one week apart, is designed to address these critical scientific gaps and identify novel approaches to study outcome measures spanning acute, transitional and chronic pain studies, collaboratively with academic, biopharmaceutical industry and government scientists. The first day will include breakout/subgroup discussions focused on acute pain, transition, and chronic pain followed by subgroup reports and recommendations the 2nd day. Participants are invited to sign up for the breakout group of their choice. Brief pre-recorded presentations will be available for registrants to view online 2 weeks prior to the meeting.
- To explore the state-of-the-science clinical trial outcome measures for pain and identify the research needed to stimulate the development of new outcomes in a manner that will withstand rigorous validation
- To review end points currently in use in phases 1 and 2 studies that could potentially be validated and used in Registration studies.
- To include broad and diverse representation of academic, biopharmaceutical industry, and government scientists working on the development of critical pain measures
- To share approaches, tools, and lessons learned that may apply across pain disorders
- To identify opportunities to help advance the development of outcome measures for therapeutics being developed across the pain chronological spectrum, including those for acute and chronic pain and for therapies that may mitigate the acute-to chronic pain transition.
- Recommendations for novel outcome measures and endpoints for clinical trials of drug and device pain therapeutics
- Recommendations/guidelines for approaches to identifying improved outcome measures and endpoints for clinical trials of pain therapeutics
- Publication of a white paper summarizing workshop findings/guidelines
Robert Dworkin, Ph.D., University of Rochester
Amy Chappell, MD, Pharmaceutical Product Development
Workshop Co-chair Bios PDF | 166.12 KB
Pain Regulatory Development Pathway
Endpoints for Pain Clinical Trials: Current approaches and accepted endpoints- drugs/biologics
Dr. Naomi Lowy discusses current approaches and accepted endpoints for drug and biologic therapeutics in pain clinical trials.
Current Approaches and Accepted Endpoints: Drugs and Biologics PDF | 502.97 KB
Endpoints for Pain Clinical Trials: Current approaches and accepted endpoints- devices
Dr. Carlos Peña discusses current approaches and accepted endpoints for devices in pain clinical trials.
Endpoints for Pain Clinical Trials: Current approaches and accepted endpoints- biomarkers
Dr. Christopher Leptak discusses current approaches and accepted endpoints for biomarker validation in pain clinical trials.
Endpoints for Pain Clinical Trials: Updated guidance on qualifying outcome measures
Dr. Elektra Papadopoulos discusses updated FDA guidance on qualifying outcome measures for pain clinical trials.
Clinical Outcome Assessment: A Regulatory Perspective PDF | 670.88 KB
Dr. Heather Fitter discusses disease-specific approaches for pain clinical trials, with specific examples from the approval pathway for migraine and headache therapeutics.
Approval Pathway for Migraine and Headache Products PDF | 296.84 KB
Pain Therapeutics Development Pathway
Endpoints for Pain Clinical Trials: Academic/European Perspective
Dr. Nadine Attal provides an academic/European perspective on the pain therapeutics development pathway and developing meaningful endpoints for pain clinical trials.
Endpoints for Pain Clinical Trials: Industry Perspective
Dr. David Hewitt provides an industry perspective on the pain therapeutics development pathway and developing meaningful endpoints for pain clinical trials.
Endpoints for Pain Clinical Trials: Patient Perspective
Lynne Matallana provides a patient perspective on the pain therapeutics development pathway and developing meaningful endpoints for pain clinical trials.
Pain Clinical Endpoints Landscape
Endpoints for Pain Clinical Trials: Landscape for clinical outcomes assessment
Dr. Bryce Reeve describes the current landscape for clinical outcomes assessment, including qualifying outcome measures, limitations and flexibility, in pain clinical trials.
Landscape for Clinical Outcome Assessments (COAs) for Pain PDF | 464.68 KB
Endpoints for Pain Clinical Trials: Patient Reported Outcomes
Dr. Robert Edwards describes the current landscape for patient reported outcomes in pain clinical trials.
Patient Reported Outcomes PDF | 4.76 MB
Endpoints for Pain Clinical Trials: Biomarkers
Dr. Petra Schweinhardt describes the current landscape for biomarkers in pain clinical trials.
Pain Clinical Endpoints - Biomarkers PDF | 965.08 KB