2017 Agenda

  • 7:45 am

  • Registration & Breakfast

  • 8:30 am

  • Co-Chairs’ Welcome and Introduction of Patient Engagement Toolbox

    Ken Getz, MBA
    Director of Sponsored Research / Founder, Tufts CSDD / CISCRP

    Judith Ng-Cashin, MD
    Chief Scientific Officer, INC

    Suzanne Schrandt, JD
    Director, Patient Engagement, Arthritis Foundation

  • 8:45 am

  • A Cancer Survivor Story: Jamie Goldfarb’s Journey

    Jamie Goldfarb survived Stage IV melanoma as a result of an immunotherapy clinical trial. She was diagnosed with Stage IV melanoma in January 2011, when her son was just 11 weeks old. Upon diagnosis, her oncologist told her that he would do everything in his power to extend her life by six more months. Jamie joined an immunotherapy clinical trial at the National Cancer Institute (NCI) and is now, six years later, completely disease-free. We are delighted to welcome Ms Goldfarb and her insights into the patient POV of a participant in IO clinical trials.

    Jamie Goldfarb
    Patient Survivor and Advocate

  • 9:15 am

  • How The Role of The Patient Advocacy Function within Industry/Pharma Evolved and What it Should Be

    There are misconceptions on what the role of the patient advocacy function within pharma should be. While some guidelines exist they are general and this is a grey area. Some think it should be about enrolling patients in clinical trials or focusing on ROI while others believe it’s to incorporate patient’s voice in development. In this session, we will focus on:

    • What is the role of the patient advocacy function in industry? What should it be? What should it not be?
    • Are there best practices in the grey area?
    • How do we define ROI in patient engagement?
    • Should we be measuring ROI in Patient Engagement and if so how?

    Kathy Gram
    VP, Patient Advocacy, Snow Companies

    Amy Fisher, MS, CGC
    Patient Advocacy Lead, Spark Therapeutics

    Jodie Gillon
    Senior Director, Advocacy & Professional Society Affairs, Achillion Pharmaceuticals

    Sharon Hesterlee, PhD
    Director of Patient Relations and Public Affairs, Bamboo Pfizer

  • 9:55 am

  • Large Pharma Case Studies on the Progress of Patient Involvement

    This session will feature four 15-minute case studies from large pharma that demonstrate how patient involvement ideas have been put into action by providing tangible examples of what, where, when and how those strategies have been implemented. A 10-minute panel Q&A will take place at the end of all four case studies.

    The focus area will be on the following:

    1. CT design
    2. Roll out
    3. Early Drug Development


    • What that looks like? What are the models of working together with patients?
    • What are some best practices in how we are actually engaging patients to get feedback into the trials?
    • What differences has it made having the patient involved in these processes?
    • What are the challenges and hurdles to overcome?
    • What did they have to do internally to sell it to the team?
    • What ROI did they see as a result?

    Victoria DiBiaso, MPH
    Associate Vice President, Global Head Clinical Operations Strategy & Collaboration, Sanofi

    Paulo Moreira
    VP, Global Clinical Operations, Head of External Innovation, EMD Serono

    Michelle Vichnin, MD
    Executive Director, Oncology, Office of the Chief Medical Officer, Merck

    Tom Wallace
    Senior Director, Global Advocacy & Professional Relations, Eli Lilly & Company 

  • 10:55 am

  • Networking Break

  • 11:30 am

  • The Science of Patient Input

    Maureen Japha, JD
    Director, Regulatory Policy, FasterCures

  • 11:50 am

  • Value Framework Developments: Patient Involvement and Progress of Models

    Several organizations have recently developed or intensified their development of frameworks that attempt to objectively assess the value of individual therapies and other health‐care services. Those organizations include the Institute for Clinical and Economic Review (ICER), Memorial Sloan Kettering Cancer Center (MSKCC), National Comprehensive Cancer Network (NCCN), American Society of Clinical Oncology (ASCO). This session will focus in on the progress of the value framework developments, how patients have been included in this process and other framework developments. Key areas that will be addressed:

    • The NHC value model rubric that was designed for framework developers to assess whether or not they are engaging patients and if so, how well. What does the value model rubric look like and what are the objectives/outcomes?
    • How are the developers revising and updating their frameworks to include significant input from patients or patient organizations to their models or definitions of value.
    • FasterCures progress on developing the first value framework that includes patient perspectives and addresses individual patient value considerations.

    Marc Boutin, JD
    Chief Executive Officer, National Health Council

    Maureen Japha, JD
    Director, Regulatory Policy, FasterCures

    Deb Maskens
    Vice Chair, International Kidney Cancer Coalition

    Dana Wollins
    Senior Director of Health Policy, American Society of Clinical Oncology (ASCO)

  • 12:30 pm

  • Lunch & Group Workshop Exercise: Best Practices on How to Keep Patients Well Informed Throughout a Trial

    What we need to do as an industry is find appropriate ways to keep patients updated. Not just return data to them, but how to keep patients informed throughout the trial. The conference co-chairs will moderate the group exercise.

    Workshop Format:

    • Each group table will discuss the assigned overall topic and questions.
    • Each table will nominate one person to collect the group feedback and share it in an open setting.

    Workshop Questions & Objectives:

    • What is the appropriate information that we can give patients and in what format?
    • How are we thinking about innovative ways to keep patients updated so they are included throughout the entire trial from screening onward?
    • Ask the patients at your table about how they want to be informed? What is important to them? Share your initiatives to see if what you are working on matters to the patients?
  • 1:30 pm

  • Workshop Review

    In this short session, the co-chairs will review with the entire delegation the shared ideas that have been submitted by each workshop table on how to keep patients well informed throughout a clinical trial.

  • 1:45 pm

  • Patient Fireside Chat: How Do Patients Want to be Informed? How Do They Want to Learn?

    This fireside chat will feature a short video by Cancer 101’s CEO Sarah Krüg on how patients want to be informed followed by interviews with patients directly to gain their perspectives first hand. Patients will address the following questions:

    • How do you tailor information for each patient? What do they want to learn?
    • How can patients communicate to us what they want to learn beyond surveys, focus groups and interviews? How do they see that relationship and informing us happening?
    • How do we customize information for different types of patients? How do measure that? How do you even know how engaged the patient is in learning?
    • How much information is too much? How to avoid patient burnout? At what point is too much? How can patients be motivated to be involved?

    Sarah Krüg
    CEO / President, Cancer 101 / Society of Participatory Medicine 

    Fireside Guests:
    Geraldine Blavat
    Parkinson’s Foundation Research Advocate and Women and Parkinson’s Advocate

    John Creveling
    Patient and Parkinson’s Foundation Research Advocate

    Kaamilah Gilyard
    Patient and advocate

  • 2:00 pm

  • The Medicine Information Exchange – What Patients and other Stakeholders Need to Say to One Another

    Communication among patients and researchers during medicine development is a vital part of creating medicines that matter to patients’ health.  Continuing a dialogue when medicines are approved and available to be prescribed or bought over the counter is essential for patients’ decision making.  Patients, caregivers, and healthcare providers need clear, scientifically objective information to choose the treatment course that best meets the patient’s health goals.

    • Where and how should information about medicines and their effects be available?
    • What information do patients and other stakeholders find the most helpful? What other information is needed?
    • Researchers who developed medicines and patients who tested medicines have valuable knowledge and experience.  How can the learning best be shared?
    • What sources of information are trustworthy, and how do we know?
    • How is it easiest to access information now? How do stakeholders wish they could access information?
    • What change in the way stakeholders exchange information would make the biggest difference to patients if it could be accomplished in 2017?

    Marilyn Metcalf
    Head, Centre of Innovation, Global Clinical Safety and Pharmacovigilance, Global Medical, GlaxoSmithKline

    Janet Freeman-Daily
    Lung Cancer Patient & Activist

    Ken Getz, MBA
    Director of Sponsored Research / Founder, Tufts CSDD / CISCRP

    Jean McCoy
    SVP, Strategy and Innovation, Health Advocacy Strategies

    T.J. Sharpe
    Melanoma Survivor, Advocate and Cancer Blogger

    Durhane Wong-Rieger, PhD
    President and CEO / Founder and Head, Canadian Organization for Rare Disorders / Consumer Advocate Network

  • 2:30 pm

  • Patients View on Why They Are/Are Not Participating in a Clinical Trial

    This panel will include both clinical trial participants and patients who have not volunteered in order to explore the thought processes behind entering a clinical trial. For those who don’t sign on, is it lack of awareness or have they considered a study and consciously rejected the idea? For those who have participated, what compelled them to take part? This panel will dive deep into the patient perspective to discover what really works — or doesn’t — when it comes to the recruitment and awareness-raising efforts for your studies.

    Jennifer Kelly
    Senior Director of Operations, HealthiVibe LLC

    Cynthia Chmielewski
    Patient and Advocate

    Laura Failla
    Patient and Advocate

    Marie Recine
    Patient, Advocate and Medical Writer

    T.J. Sharpe
    Melanoma Survivor, Advocate and Cancer Blogger

    MarlaJan Wexler, RN
    Patient and Creator of Luckfupus.com

  • 3:15 pm

  • Networking Break

  • 4:00 pm

  • Partnering with Patient Organizations for Advancing Cures

    In this multi-stakeholder session, patients, advocates, industry and nonprofit organizations come together to discuss how they partnered and the outcomes of those partnerships.

    • What’s the roadmap?
    • What are the ways you have partnered with industry?
    • What are some best practices or rules of engagement?
    • How can the pharma industry better serve patient advocacy organizations?
    • How are we creating and ruthlessly maintaining transparency and trust?
    • Addressing global models of patient engagement:
      • Big companies are working in global environment and the Patient Engagement issues are quite different culturally, whether it’s China, Japan, Asia. What are the unique challenges and opportunities in countries outside US and EU?

    Lori Abrams
    Director, Diversity and Patient Engagement, Bristol-Myers Squibb

    Stephanie Christopher
    Program Manager, Patient Centered Benefit-Risk, Medical Device Innovation Consortium

    Lauren McLaughlin
    Associate Director, Research Partnerships, The Michael J Fox Foundation for Parkinson’s Research

    Suzanne Schrandt, JD
    Director, Patient Engagement, Arthritis Foundation 

    Nirmala Singh
    Former Patient Engagement Director, National Kidney Foundation

  • 4:40 pm

  • A Clinical Trial That’s the Right Fit: Bringing the Latest Therapies Close to Home

    Sandy Smith
    Senior Director, Site Services, US Oncology Research

  • 5:00 pm

  • Integrating Patient Needs Into a Shared Patient Community Beyond the Trial

    In this session, Thomas Klein, Founder and CEO of Be the Partner will discuss how they took a “patient first” perspective in building its platform, using patient input and feedback. Key focus areas include:

    • Results from an initial pilot of the platform in a study conducted by a large pharma sponsor
    • Leveraging the success of the pilot of learnings from it and further patient input, Be the Partner will discuss the future of the platform and the opportunity for pharma to create a pre-competitive, patient-centric asset

    Thomas Klein
    Founder and CEO, Be the Partner

  • 5:20 pm

  • Patient Engagement Sources & Solutions

    Helen Green
    Account Director, Langland

    Julie Walters
    Founder, Raremark

    Komathi Stem, MS
    Founder and CEO, MonARC Bionetworks

  • 6:00 pm

  • Networking Reception

2017 Agenda

  • 8:00 am

  • Breakfast

  • 8:30 am

  • Co-Chairs’ Welcome and Introduction of Patient Engagement Toolbox Review

    Judith Ng-Cashin, MD
    Chief Scientific Officer, INC  

    Ken Getz, MBA
    Director of Sponsored Research / Founder, Tufts CSDD / CISCRP

    Suzanne Schrandt, JD
    Director, Patient Engagement, Arthritis Foundation

  • 8:45 am

  • Patient Input & The Regulatory Process

    Richard Moscicki, MD
    Deputy Center Director for Science Operations, Center for Drug Evaluation and Research (CDER), FDA

  • 9:15 am

  • PDUFA VI: An Update on New Performance and Procedural Goal

    Theresa Mullin, PhD
    Director, Office of Strategic Programs, CDER, FDA

  • 9:45 am

  • Organizational Adoption of Patient Engagement: A CDRH Case Study on How They Have Instilled an Internal Culture of Patient Engagement

    The CDRH has committed to do a variety of activities within the agency to promote a culture of patient engagement. In this session, CDRH’s Associate Director, Kathryn O’Callahan addresses the following:

    • Addressing the differences of opinion when it comes to the scope and the right approach with incorporating patient input on the science side of things
    • Demonstrate how the staff in key decision making roles who may not have the opportunity to routinely interact with patients are affected by their [patient] decisions.
    • What has the impact been?
    • How has the agency evolved as a result of this internal culture shift?

    Anindita Saha
    Director, External Expertise and Partnerships, Center for Devices and Radiological Health (CDRH), FDA

  • 10:15 am

  • Networking Break

  • 11:00 am

  • What Patients Want: Addressing the Gap Between Clinical Care, Patient Involvement and What Patients Actually Want

    This session will discuss what patients want when it comes to their involvement in research, clinical trials and their care options.  Patients will lead a multi-stakeholder panel addressing the following:

    Closing the gap between clinical care and patients wants/needs:

    • Ask patients what’s going on in their lives
    • Ask patients what their goals are
    • Because no one asks patients these questions, the result is they get care that’s aligned what the doctor thinks is clinically relevant. Even though it may be completely out of context to what is important to the patient.

    What patients want and how pharma can help

    • Identify what patients and industry want respectively in terms of patient involvement and why that is meaningful for each
    • What can we do to enhance it across the development pipeline?
    • What is getting in the way? Identify hurdles?
    • How can we work thru this collaboratively?

    Mary Stober-Murray, MBA
    Associate Director, Diversity and Patient Engagement, Bristol-Myers Squibb

    John Creveling
    Patient and Parkinson’s Foundation Research Advocate

    Kaamilah Gilyard
    Patient and Advocate

    Paul Graves
    VP, Head, R&D Network Relationships and Strategic Communications, Takeda Pharmaceuticals 

    Deb Maskens
    Vice Chair, International Kidney Cancer Coalition

    Michelle Vichnin, MD
    Executive Director, Oncology, Office of the Chief Medical Officer, Merck

  • 11:40 am

  • Considering Diverse Patient Needs in Clinical Trials

    Clinical trials are often planned more for investigators and scientists than for patients. Even so-called “patient-centric trials” are likely to be designed for a single patient type. A more effective approach is to take into account the needs of patients from all of the geographic regions and cultures involved in the study.

    This session outlines an approach for obtaining actionable patient insights to design protocols and patient recruitment/retention programs. The method includes steps to verify and adapt findings regionally to meet the diverse needs of patients. Using examples and case studies, audience members will learn how incorporating cultural diversity into study design has been used to ensure successful patient experiences in multiple countries and regions.

    Kelly Franchetti, RN, CCRN, CEN
    VP, Global Patient Insights and Engagement, Mapi Group

  • 12:00 pm

  • Raising Public Awareness in Clinical Research: Lessons Learned

    This session led by Ken Getz will include a state of the union on lessons learned in raising public awareness in clinical research.

    Ken Getz, MBA
    Director of Sponsored Research / Founder, Tufts CSDD / CISCRP 

    Judith Ng-Cashin, MD
    Chief Scientific Officer, INC

  • 12:30 pm

  • Lunch

  • 1:45 pm

  • How are Patients Being Involved Earlier in the Preclinical Research Process?

    How are researchers, patients and industry coming together and identifying key preclinical research areas that are of importance to the patients? This researcher/patient led session will address the following:

    • How do we leverage that support?
    • How do we engage our patients?
    • What do patients want to see from researchers or industry? Visa Versa?
    • What is important for patients?
    • What is that magic “ah-ha’ that will make patients trust the patient advocacy groups connecting them with the researchers in the industry groups?

    Melissa Hogan, JD
    Rare Disease Patient Strategist, Founder/President, Saving Case & Friends 

    Douglas McCarty, PhD
    Principal Investigator, Center for Gene Therapy / Associate Professor, Department of Pediatrics, The Research Institute at Nationwide Children’s Hospital / The Ohio State University College of Medicine

  • 2:15 pm

  • Involving Patients in Clinical Trial Design for Targeted Evidence Creation: An Inspire Case Study

    As patients become increasing involved in decision making related to treatments, there are now two equally important audiences for clinical trials — physicians, with their technical understanding of drug mechanics, and patients.  Patients often look for different things in clinical trial design than physicians. Patients are interested in whether the treatment resolves their crucial complaint, whether it has bad side effects, and in some cases whether it’s blinded. Involving patients early in design helps guarantee that trials can effectively answer the questions of both populations. This session will address:

    • How to find patients
    • Ways to engage patients over the course of a clinical trial
    • Examples of successful recruitment stories

    Jeff Terkowitz
    Director, Product Management, Inspire

  • 2:30 pm

  • Including the Patient Voice in Mobile and Wearable Tech Development for Clinical Trials: A Case Study

    This session will explore how The Michael J. Fox Foundation (MJFF) and Intel have partnered to create a patient-centric research platform using a mobile application and paired wearable device to collect sensor data and patient reported outcomes (PROs) from people living with Parkinson’s disease (PD). This platform can be used in clinical trials or observational studies to collect robust, real-time data on the lived experience PD outside of a clinical setting. To ensure the platform development is patient-centric and aligned with needs and preferences of people with Parkinson’s, MJFF and Intel have formalized a Beta Testing program with people living with Parkinson’s (n= 15) to routinely and iteratively test upgrades to the hardware and software of the platform and ensure that tools are designed appropriately for PD patients and their needs. Through this, beta testers have identified bugs, suggested areas to improve the platform’s flow and functionality, and recommended new app features that are of interest to them as patients. Discussion will address how this Beta Testing program was designed and implemented and will provide specific examples of how patient insights have influenced the platform’s development. Lessons learned from this program can be applied across mobile or wearable tech development efforts in various disease areas.

    Followed by a technology panel discussion on this topic.

    Daisy Daeschler
    Research Partnerships Officer, The Michael J Fox Foundation for Parkinson’s Research

    Gary Ostroff, PhD
    Research Professor in Molecular Medicine, UMass Medical School

  • 2:50 pm

  • Putting Patients 1st at the Cleveland Clinic: Lessons Learned for Pharma

    Putting patients first requires more than world-class clinical care – it requires care that addresses every aspect of a patient’s encounter with Cleveland Clinic, including the patient’s physical comfort, as well as their educational, emotional, and spiritual needs. Our team of professionals serves as an advisory resource for critical initiatives across the Cleveland Clinic health system. In addition, we provide resources and data analytics; identify, support, and publish sustainable best practices; and collaborate with a variety of departments to ensure the consistent delivery of patient-centered care.

    Cleveland Clinic was the first major academic medical center to make patient experience a strategic goal, appoint a Chief Experience Officer, and one of the first to establish an Office of Patient Experience. This session will address:

    • How has the Cleveland Clinic changed their culture to focus on the patient?
    • What has made the Cleveland Clinic initiate these changes? What were the drivers?
    • How was it implemented?
    • How do we measure the overall patient experience? Was ROI measured? What were the results of the changes?
    • How can pharma learn from this to better the overall patient experience when we engage with patients?

    Rachel Taliercio, DO
    Director of Postgraduate Enrichment, Center for Excellence in Healthcare Communication, Office of Patient Experience, Cleveland Clinic

  • 3:20 pm

  • Conference Concludes