DIA 2025 Global Annual Meeting

Component Type: Session
Level: Intermediate
CE: ACPE 1.00 Knowledge UAN: 0286-0000-25-513-L04-P; CME 1.00; RN 1.00

Despite advances, patient engagement (PE) in post-approval access decisions remains limited, complex, and unclear. This session examines how patients can offer value-based insights and explores pathways to strengthen PE in product access decisions.

Learning Objectives

Identify existing opportunities to incorporate patient input into access determinations; Recognize patient-contributed information and data that could broaden the evidentiary base for CMS and other payers; Discuss the resources and frameworks needed for CMS to systematically and scientifically engage with patient communities.

Chair

June Cha, PhD, MPH

Speaker

Vital Voices from Patients in Access Determination
June Cha, PhD, MPH

Patients Engaging with CMS: Opportunities, challenges, and lessons learned
Kristi Martin, MA, MPA

Patient Perspectives: Are They Missing from Commercial Payer Drug Coverage Policies?
James Chambers, PhD, MPharm, MSc

How Breakthrough T1D has Engaged CMS
Campbell Hutton, MPH

Component Type: Session
Level: Intermediate
CE: ACPE 1.00 Knowledge UAN: 0286-0000-25-539-L04-P; CME 1.00; RN 1.00

A multi-stakeholder panel will discuss: regulatory pathways and consideration for early engagement with regulators, harmonization efforts to standardize use of PED, present frameworks to support Industry planning, and explore future opportunities.

Learning Objectives

Discuss regulatory pathways for early engagement to streamline PFDD; Examine regulatory considerations and harmonization efforts to standardize the collection and use of Patient Experience Data; Recognize how to evaluate frameworks available to support industry planning of PFDD studies; Describe best practices for ensuring successful patient-focused study planning and regulatory decision-making.

Chair

Pujita Vaidya, MPH

Speaker

FDA Perspective
Valentina Mantua

Patient-Experience Data in Development Programs and Regulatory Decision-Making
Juan Garcia-Burgos, MD, PhD

Patient Representative Perspective and Resources for Advancing PFDD
Silke Schoch, MA

Industry Perspective
Samantha Roberts, PhD

Component Type: Session
Level: Intermediate
CE: ACPE 1.00 Knowledge UAN: 0286-0000-25-540-L04-P; CME 1.00; RN 1.00

Addressing data gaps in drug development, especially capturing patient experiences in both rare and non-rare diseases, is crucial. From Proof of Concept to Proof of Value, integrated evidence planning and incorporating patient voices in Target Product Profile (TPP) creation drive comprehensive evidence generation.

Learning Objectives

Discuss the utility in global drug development context, including post-marketing efforts; Identify strategies to create a patient informed comprehensive Target Product Profile; Recognize strategies outlined in the interactive guide to align PAG and industry efforts in creating a comprehensive Target Product Profile.

Chair

Agathe Le Lay, PhD, MSc

Speaker

What are we Missing: Potential Data Gaps in Drug Development and the Importance of Capturing Lived Experience in rare and non-rare Diseases
Kristin Hatcher, MEd

From Proof of Concept to Proof of Value: An Industry Approach to Integrated Evidence Planning Throughout the Product Lifecycle
Agathe Le Lay, PhD, MSc

New Strategies and Practices to Incorporate the Patient Voice in TPP Creation
Patricia Roselle

New Strategies and Practices to Incorporate the Patient Voice in TPP Creation
Bruce E. Miller, PhD

Panelist
Martine Zimmermann, PharmD

Component Type: Workshop
Level: Intermediate
CE: ACPE 1.00 Application UAN: 0286-0000-25-552-L04-P; CME 1.00; RN 1.00

Imagine a future where AI measures patient burden from provided inputs. In this interactive workshop, participants will design input logic for AI using mock data and protocols, then present their rationale to foster collaborative learning.

Learning Objectives

Define and categorize key factors affecting patient burden in clinical trials; Analyze patient-related data to design effective AI inputs; Evaluate and justify the role of human insight in enhancing AI-driven patient burden assessments for patient-centric trial outcomes.

Chair

Mihail Tanev, DMD, PhD

Speaker

Panelist
Karen Correa, PhD

From Measuring to Modeling the Patient Burden: An Interactive Take on What Defines the Patient Experience in the AI-Age
Mihail Tanev, DMD, PhD

Component Type: Forum
Level: Intermediate
CE: ACPE 1.00 Knowledge UAN: 0286-0000-25-583-L04-P; CME 1.00; RN 1.00

This session examines the relationship between protocol complexity, participant burden, and enrollment challenges, alongside the development and impact of participant feedback questionnaires. Patient advisors and attendees will interpret results and discuss improvements in trial performance.

Learning Objectives

Discuss patient participation burden based on protocol design characteristics and requirements; Identify design elements driving elevated levels of burden and impacting clinical trial performance; Recognize how to manipulate results into practical improvements for clinical research projects; State the value of participant feedback and compose rationale for inclusion of feedback in future studies; Describe strategies to reduce and address clinical trial and portfolio-wide participation burden.

Chair

Lani Hashimoto

Speaker

Addressing Participation Burden in Clinical Trials: Evidence, Participant Feedback, & Signal Interpretation to Drive Impact
Sabina Kineen

Addressing Participation Burden in Clinical Trials: Evidence, Participant Feedback, & Signal Interpretation to Drive Impact
Patrick Gee, SR, PhD, MS

Addressing Participation Burden in Clinical Trials: Evidence, Participant Feedback, & Signal Interpretation to Drive Impact
Stephanie Manson, PhD

Addressing Participation Burden in Clinical Trials: Primary Causes, Outcomes and Mitigation Strategies and Practices
Kenneth Getz, MBA

Component Type: Session
Level: Intermediate
CE: ACPE 1.00 Knowledge UAN: 0286-0000-25-602-L04-P; CME 1.00; RN 1.00

With several regulatory and ethics authority initiatives to encourage patient input to trial design, how should sponsors communicate this information in submissions and how can authorities provide aligned review, guidance and expectations towards these activities?

Learning Objectives

Discuss US and EU initiatives for systematic conduct and documentation of patient input to trial design; Explain learnings from survey on sponsor submission/advice experience and guidance needs; Identify gaps and opportunities for authority (regulatory and ethics committee) harmonization in expectations and guidance

Chair

Melissa Herman, MSc

Speaker

Industry Perspective
Maria Apostolaros, JD, PharmD, MS, RPh

Industry Perspective
Lesley Maloney, PharmD

Industry Perspective
Elizabeth Rosenkrands-Lange, MPH, MS

Component Type: Session
Level: Intermediate
CE: ACPE 1.00 Knowledge UAN: 0286-0000-25-656-L04-P; CME 1.00; RN 1.00

Patient experience data (PED) is sought by regulators, payers, and value/health technology assessment (V/HTA) bodies globally and used in different ways. Actively including PED in scientific, regulatory, and V/HTA processes has been a recent feature of regulatory and HTA reform, notably FDA guidance on patient-focused drug development (PFDD) and the EU Joint Clinical Assessment framework. Evolving perspectives on V/HTA have brought attention to the role of PED and there is a need to more systematically integrate PED into payer decision-making. Yet, the PED needs and perspectives of different stakeholders must be better understood for PED to have meaningful impact on product development, evaluation, and related decision-making. Many questions remain. Where do patients believe they have the most impact? What is industry experience gathering and leveraging PED globally? Do government agencies believe their efforts to include patient perspectives are being impactful on evidence they finally review? How can the impact of PED on V/HTA increase and be optimized globally? We will explore options to increase the impact of PED on development, V/HTA, and access decisions through optimal coordination and information sharing, and highlight options and good practices for integrating PED into regulatory, V/HTA, and payer decisions. We will discuss learnings across various opportunities for leveraging PED through policies and processes that affect patient access. Perspectives on PED from patient-group, EMA, payer, and industry panelists will showcase differences and similarities in stakeholder views and evidence needs to inform PFDD, V/HTA, and payer decisions, ultimately impacting patient access. Panelists’ experiences provide a lens on how current processes are working in concert or opposition, and opportunities for meaningful multi-stakeholder collaborations maximize PED impact on access decisions.

Learning Objectives

Discuss patient-group, payer, regulator, and industry perspectives on PED, in general, and regarding how to optimize PED use in decision-making in their respective roles; List the benefits and challenges to integrating PED in regulatory, V/HTA, and payer decision-making, from various stakeholder perspectives; Describe two examples of good practices and recommendations for integrating PED into regulatory, V/HTA, and payer decisions.

Chair

Eleanor M. Perfetto, PhD, MS

Speaker

Panelist
Maria Mavris, PhD

Panelist
Maxwell Isaacoff

Panelist
Magdalena Harrington, PhD

Panelist
Anna Hyde, MA

Component Type: Forum
Level: Advanced
CE: ACPE 1.00 Knowledge UAN: 0286-0000-25-657-L04-P; CME 1.00; RN 1.00

Join us for an engaging discussion on an ongoing DIA study on enhancing the integration of tolerability patient experience data into immune-oncology clinical trials. This session will explore the study’s efforts to address regulatory requirements for the collection of core tolerability patient experience data and the need for a comprehensive, multidimensional approach to assessing tolerability in cancer treatments. With a multistakeholder focus, the study aims to define tolerability more holistically by incorporating treatment-related factors, convenience, and patient beliefs. Learn about the study’s progress, next steps, and its potential to align with regulatory needs while addressing real-world patient concerns.

Learning Objectives

Recognize the significance of advancing patient-centric tolerability measurement in immune-oncology trials to enhance patient outcomes; List insights into the ongoing study and future steps aimed at addressing the real-world needs of patients through a multistakeholder framework.

Chair

Maria Paula Bautista Acelas, MSc

Speaker

Panelist
Alison Cave, PhD

Moderator
Rohini Sen, PhD, MS

Unlocking Insights: Progress and Key Findings from the Tolerability Study
Ryan Murphy, PhD

Panelist
Catherine Coulouvrat, DrMed

Panelist
Lia Ridout

Component Type: Forum
Level: Intermediate
CE: ACPE 1.00 Knowledge UAN: 0286-0000-25-660-L04-P; CME 1.00; RN 1.00

This session will provide an opportunity for key stakeholders, including regulators, product developers and patients to discuss best practices around patient experience data, including planning, collecting, and submitting such data to regulators.

Learning Objectives

Discuss current expectations for regulator/sponsor interactions related to patient experience data that will be used in regulatory submissions; Recognize common pitfalls for submission of patient experience data in regulatory applications, and how to avoid them.

Chair

Robyn Bent, BSN, MS, RN

Speaker

Panelist
Pujita Vaidya, MPH

Panelist
Brett Hauber, PhD, MA