Patrick was invited to the second Brookings / FDA workshop:

The workshop focused on “Advancing Development and Use of Patient-Reported Outcomes in Drug Development: Near-Term Opportunities”.

This meeting was a follow-up from a previous workshop that took place on July 16, which focused on experiences with the FDA’s guidance on PROs, as well as on challenges in capturing the patient voice across the drug development continuum.

This time, sessions focused on:
– I.   Developing a Potentially Acceptable Endpoint List: Potential Value and Uses – Implementation Approaches
– II.  Identifying Emerging Themes from the Acceptable Endpoints Discussion
– III. Evidentiary Considerations for Making Existing PRO Instruments Fit for Purpose
– IV. Identifying and Prioritizing Next Steps
Washington DC, October 6, 2014

Patrick and Stefan Cano are partners in Modus Outcomes: Center of Excellence for the Development of Clinical Outcomes Assessments.

Modus Outcomes sets new standards for measuring patient outcomes and fostering patient-centered drug development. Together with Stefan, we bring complimentary skills and unique clinical, regulatory and measurement experience.. As a scientist and chartered psychologist, Stefan’s main research areas are in psychometrics and health measurement, and his specialties include psychological research methods and Rasch Measurement Theory.
Check our website!
For more information:
We are recruiting now: if you interested in joining our center of excellence for COA development, check our career page:

Patrick was invited to the first Brookings / FDA workshop:
“Enhancing the Development and Use of Patient-Reported Outcomes in Drug Development”:

The experience with FDA Guidance on PROs was discussed.
Sessions focused on:
– 1. Evidentiary Standards
– 2. Standardizing Communication Processes
– 3. Challenges to Capturing the Patient Voice Across the Drug Development
– 4. Next Steps in Promoting the Development and qualification of PROs
Washington DC, July 16, 2014

Both Stefan Cano and Patrick participated as faculty or speaker in ISPOR Montreal sessions focused on good COAs measurement practices and methods.

– Sunday June1, 2014: Short Course Program in Patient-Reported Outcomes Methods
Applied Mixed Methods to establish content validity of Patient -Reported ( PRO), Clinician- Reported (ClinRo) and Observer-Reported Outcome (ObsRo) Assessments.
Faculty: Donald Patrick, MSPH, PhD; Laurie Burke, RPh, MPH; Jeremy Hobart, PhD, FRCP; Stefan J. Cano, PhD

– Monday June 2, 2014: Ispor Forums – Session 1: Ispor Clinician Reported Outcomes Good Measurement Practices
Moderator: John H. Powers, III, MD, FACP, FIDSA
Speakers: Laurie B. Burke; Patrick Marquis, MD, MBA; Ashley Slagle, PhD, MS

TwoLegs Consulting has presented at the DIA Study EndpointsTargeting Patient-Centered Outcomes…

Workshop prepared by FDA and Pharma March 18-20, 2014, Bethesda North Marriott Hotel and Conference Center, MD.
Patrick Marquis was the speaker for “Practical Consideration when Planning the Evaluation of Measurement Properties”.
Session Chair: Paivi Miskala, MSPH, PhD, Endpoints Reviewer, SEALD, CDER, FDA

Please contact Patrick for copy of the slides:

TwoLegs Consulting has launched the Foresight Workshop Series:

Workshop 1:
Why is it imperative to take a patient-centered approach to demonstrate clinical value to payers?
The workshop presents an analysis of the new landscape, challenges and opportunities related to Patient-Reported Outcomes (PRO). The highlights include examples of recent HTA reviews and the analysis of guidelines (WellPoint, IQWiG, EunetHTA…). A discussion is proposed about the converging approach of regulators and HTA reviewers to better integrate the patient perspective and PRO in their decisions. Finally the workshop presents a roadmap for integrating an effective outcome assessment strategy focusing on clinical value to both regulators and payers.

Workshop 2:
Application of the Clinical Outcome Assessment model for labeling in the US and EU: why this is a must do.
The workshop presents the recent clinical outcome assessment paradigm, its foundation and the distinction between the four types of outcome assessments: Patient-Reported, Observer-Reported, and Clinician-Reported (respectively PRO, ObsRO, ClinRO), and Performance Outcome assessments (PerfO). Their role to demonstrate or support direct evidence of treatment benefit is reviewed as well as the newly developed good measurement principles for ClinRo. FDA and EMA similarities and differences in labeling are highlighted. Finally the workshop presents a roadmap for integrating an outcome assessment strategy which is truly patient-centered and targeted to labeling.

Both workshops are available in the US and EU and can be tailored to the therapeutic areas of interest to the sponsor.
Please contact

The patient perspective in healthcare decisions:  ISPOR Dublin Panel Issue chaired by TwoLegs Consulting…

TwoLegs Consulting has chaired a panel discussion at ISPOR Dublin: “Biopharma, Regulators, and Payers decisions: different processes but is there a convergence in the approach for integrating the patient perspective?”  Panelists: Laurie B. Burke, RPh, MPH, formerly Director, Study Endpoints and Labeling Development (SEALD), ONDIO, CDER, FDA), Mira Pavlovic, MD, Deputy Head, HTA Division, HAS, EUnetHTA;  Julia A. Gaebler, PhD, Director, Health Economics & Outcomes Research, Global Market Access, Biogen Idec); Session III on Tuesday, 5 November 2013 from 15:00-16:00.
For more information or copy of the slides, please contact

ClinRo: good measurement practices   

TwoLegs Consulting, member of the ISPOR task force leadership has presented at Dublin… ISPOR Clinician Reported Outcomes Good Measurement Practices Task Force has members from EMA, FDA, industry, and research. It has defined which outcome assessments should be considered ClinROs, and recommended ClinRO measures to document treatment benefit in clinical trials.  The ClinRO Forum has been hold on Monday, November 4th at 6:00PM.
Please contact for more information or copy of the slides.

Copyright © 2014 TwoLegs Consulting LLC, All Rights Reserved