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Save when you register for both the Advancing the Science of Study Endpoints: Seeking Practical Solutions and the Achieving Meaningful Regulatory and Clinical Outcomes for Patients: Strategies in Rare Disease Therapy Development
*You must register for both events at the same time.  Details in the registration section below.

Advancing the Science of Study Endpoints: Seeking Practical Solutions

Conference:  October 5-6

Selecting endpoints for use in medical product development programs involves both strategic and methodological/scientific considerations. The endpoints must provide a compelling view of how a new treatment benefits patients. The measures that support the endpoints must be well-defined and provide reliable and valid data in a specific context of use. Analyses of endpoints must address a variety of concerns, including questions regarding missing data, interpretability, and the clinical relevance of treatment effects. Carefully addressing these difficult study endpoint issues can dramatically increase the likelihood of a program’s success. This meeting will bring together stakeholders from the pharmaceutical industry, regulatory agencies, and expert workgroups and consortia to generate practical solutions to challenging questions associated with study endpoints. The primary objectives of the meeting are to:

  1. Discuss strategies for comprehensively demonstrating treatment benefit through the selection of optimal study endpoints (e.g., survival, clinical outcome assessments, biomarkers),
  2. Review key considerations regarding the development and implementation of assessments that underlie study endpoints, and
  3. Identify solutions related to the analysis and interpretation of study endpoints (in isolation and in assessing overall risk/benefit for a product).

These objectives will be addressed in three important product development contexts: oncology, pediatrics, and rare diseases. Attendees will learn more about study endpoint challenges in these areas, but the focus of the meeting will be on generating solutions that can be applied in clinical development programs.

The 2015 conference will feature sessions in each of the following areas:

Oncology (Program Committee Representatives: Stephen Joel Coons, Critical Path Institute and Katarina Halling • AstraZeneca, Sweden; Session Chair Alan Shields, Adelphi Values)
Despite the value of PRO data and regulatory encouragement for PRO endpoints in cancer trials, the promise of integrating the patient perspective into oncology drug approval and labeling remains largely unfulfilled.  The meeting session, Use and Usefulness of PRO Endpoints in Oncology Trials, chaired by Alan Shields, is designed to provide attendees with examples, insights, and practical knowledge regarding a) the value use of PRO-based endpoints as primary efficacy endpoints in oncology trials and b) the value of PRO endpoints in open label trials that are routinely conducted for cancer treatments.  This knowledge training session will include presenters to characterize the perspective of patients, drug developers, trial methodologists, clinicians, and the FDA.

Pediatrics (Program Committee Representative: Jean Paty, Quintiles; Session Chair: Diane Turner-Bowker, Quintiles)
There are a number of unique challenges and considerations when using COAs with pediatric patients.  While guidance documents provide a framework for pediatric COA assessment, this is a developing field of research, more information is needed to establish sound models for the development, validation, and implementation of COAs in pediatric clinical research.  This session is intended to provide a forum for in-depth discussion of methodological challenges involved in the development and use of pediatric COAs, and the pursuit of solutions that are practical yet still adhere to established principles of instrument development.

Rare Diseases (Program Committee Representative: Linda Deal, Pfizer; Session Chair: Susan Martin, RTI International)
Selecting and identifying study endpoints in Rare Disease (RD) clinical trials can present a challenges to drug developers. In many RDs there is a lack of precedence and even a void of existing instruments for evaluating treatment benefit in the context of a high unmet medical need for a small patient population.  As a result, standard methods for study endpoints development and use must be adapted in such a manner that preserves scientific best practices, e.g. of the FDA PRO Guidance, yet accommodates the special circumstances of RD. The following sessions will address these challenges and offer suggestions to be discussed by a panel of drug development specialists from Industry, FDA and patient advocacy groups.

This program has been developed in collaboration with the Study Endpoints Community.

Who should attend?

This workshop is for industry, academia, government, vendors, clinicians, statisticians, and health technology agency professionals involved in setting, executing or evaluating endpoint strategy for drug approval, labeling, promotion, translational science and market access.

Learning objectives

At the conclusion of this activity, participants should be able to:

  • Identify the challenges involved when developing and implementing clinical outcome assessments in oncology, pediatric, and rare disease clinical development programs
  • Implement strategies to facilitate the use of clinical outcome assessments, including patient reported outcomes, in oncology, pediatric, and rare disease clinical development programs
  • Describe how patient-reported outcomes can be used to understand treatment benefit in oncology trials
  • Outline challenges and solutions associated with clinical outcome assessments that are unique to pediatric programs
  • Apply innovative approaches to developing clinical outcome assessments in areas where large numbers of patients are not available

Program Committee

  • Chad  Gwaltney, PhD
    Chad Gwaltney, PhD President
    Gwaltney Consulting, United States
  • Annabel  Nixon, PhD
    Annabel Nixon, PhD PRO Consultant
    Chilli Consultancy, United Kingdom
  • Stephen Joel  Coons, PhD
    Stephen Joel Coons, PhD Executive Director, PRO Consortium
    Critical Path Institute, United States
  • Linda S. Deal, MS
    Linda S. Deal, MS Head of Patient-Centered Outcomes Measurement
    Pfizer Inc., United States
  • Katarina  Halling
    Katarina Halling PRO Group Director
    AstraZeneca, Sweden
  • Jean  Paty, PhD, MS
    Jean Paty, PhD, MS Vice President, Patient-Centered Science
    IQVIA, United States
  • Keith  Wenzel
    Keith Wenzel Senior Director, Scientific Data Organization
    Parexel International, United States
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