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2018 Agenda

  • 7:45 am

  • Registration & Breakfast

  • 8:30 am

  • Co-Chair’s Opening Remarks: The Evolution of Patients as Partners and What’s Next?

    We often look at patients while they are in the trials, their experience there, getting their information back to them, but that is not enough. We need to ask “Why did this participant even participate in the first place, and did they get out of it what they would?” Then internally look at all the different parts and ask why it’s valuable to partner with that team at that stage, how does this impact them beyond the features of inspiration? When it isn’t inspiring, what instruction do you get that is really helpful in making directional decisions? What does engagement truly look like and how can we do it more efficiently?

  • 8:45 am

  • Patient Fireside Chat

    Cancer survivor Stefanie Joho was diagnosed in 2013 with colon cancer. At the age of 24, after two surgeries and two aggressive rounds of chemotherapy failed to cure Stefanie, her oncologist sent her home to die. This was not acceptable and Stefanie’s sister helped her find an immunotherapy trial that ultimately saved her life. Stefanie joins us to talk about her immunotherapy journey, what she has learned and how she now advocates to help others shape their thinking on become their own patient advocate and an active participant in helping save their own lives.

    Stefanie Joho

    Cancer Survivor

  • 9:05 am

  • Overview of Key Results and implications of the CISCRP 2017 Perceptions and Insights Study

    In this session, Ken Getz, Director of Sponsored Research Programs Tufts CSDD and founder of CISCRP will share the results and implications of the 2017 Perceptions & Insights study.  The study gathered responses from nearly 12,500 people world wide.  Trends and insights into public and study volunteer attitudes, perceptions and experiences with clinical research will be reviewed with a look at a number of subgroup differences including by geographic region, age and disease condition. The 2017 Perceptions & Insights study also introduces a number of new areas of inquiry including the use and perceived value of patient engagement initiatives and overall public and patient receptivity to the integration of clinical research into clinical care.

    Ken Getz, MBA

    Director of Sponsored Research Programs / Chair, Tufts CSDD / CISCRP

  • 9:35 am

  • A New Innovative Approach to Integrate Patient Preferences into the Statistical Design for Clinical Trials: A Collaborative Case Study

    For chronic debilitating and terminal illnesses with no effective treatments, the standard statistical threshold for determining therapeutic effectiveness in clinical trials may be too conservative and may not reflect patients’ perspectives on the potential benefits and risks of a therapy. This session will present a novel method for incorporating patient preference information as an explicit means to set significance levels in clinical trial designs. This project, in collaboration with MIT, MDIC, the Michael J Fox Foundation and RTI Health Solutions, focuses on Parkinson’s disease but may be generalizable to other disease areas. The primary outcome of the project is a method to determine an appropriate p-value threshold and trial size using patient input. This method helps remove barriers to access by giving patients a pathway to breakthrough, lifesaving technologies based on their risk tolerance and the resulting potential for reduced clinical trial size. This session will present some of the results of the study, including key attributes identified as important to patients, how the patient preference information could be used to design a patient-centered clinical trial, and lessons learned from this unique collaborative partnership between patients, regulators and the medical devices industry.

    Stephanie Christopher

    MA Program Director, Medical Device Innovation Consortium (MDIC)

    Lauren McLaughlin
    Associate Director, Research Partnerships, The Michael J Fox Foundation for Parkinson’s Research
  • 10:05 am

  • Connections for Patient Care Pathway in Clinical Trials

  • 10:20 am

  • Networking Break

  • 11:00 am
    - 12:15 pm

  • Large Pharma Case Studies: Applying Patient Engagement Initiatives and Demonstrating Impact

    Choice of six case studies.

    • Track A

    • 11:00 am

      How Tesaro is Integrating the Patient in the Clinical Development Process

      In this session, Beth Zaharoff, Director, Patient Focused Clinical Trial Engagement of Tesaro will walk the audience through how they integrated patient input throughout multiple stages of the development process.

      • What patient focus means to TESARO and why/how we decided to jump in the deep end
      • How we gained internal support and tips for doing so
      • TESARO focus groups – what we learned from the experts
      • How TESARO solicited patient input to design a trial that is both relevant and scientifically rigorous
      • Patient input into clinical trial design = consumer input in the goods and services industry
      • How Tesaro is working to eliminate some of the burdens inherent in clinical trial participation

      Beth Zaharoff

      Director, Patient Focused Clinical Trial Engagement, Tesaro

    • 11:30 am

      How Janssen is Creating Strategic Collaborations that are Driving Patient Centered Trials

      An enormous amount of attention and resources have been dedicated to understanding what patient centricity means and how to truly engage patients and put them at the center of all R&D. It takes a village. In this case study, here how leading pharmas are collaborating to achieve patient-centered trials.

      Katherine Capperella

      Global Patient Engagement Leader, Janssen Pharmaceuticals

      Bennett Levitan, MD, PhD

      Senior Director, Benefit-risk Assessment, Department of Epidemiology, Janssen R&D, LLC

    • 12:00 pm

      How Takeda Used One or More of the Various Patient Engagement Frameworks Internally to Build Support and Cultural Shift for Patient Engagement

      There are many frameworks and models that exist from various organizations on aspects of patient engagement and may be challenging to navigate through when applying to your own initiatives. In this session, Takeda will walk the audience through how they used multiple patient engagement frameworks to create an internal R&D Patient Engagement Guide tailored for their drug development teams from discovery through marketed products. The audience will learn how the guide has help build support and a cultural shift for Takeda’s internal patient engagement initiatives and how it has helped each team at various phases of development with the following:

      • Identifying what the patient engagement activities are at the different phases of development
        • How does patient engagement apply to the work I am doing?
        • Identifying who is relevant to talk to, how to go about it
        • What is the impact?
      • Mapping the patient advocacy landscape
        • Identifying what all the ways are that teams can learn from patients
        • What you need to understand about patient advocacy groups before engaging with them
        • How to initiate relationships with patient advocacy
        • Understanding where patients fit in your development, where online communities fit in, etc
      • Implementing change and streamlining patient engagement initiatives internally
        • Identifying each team’s key patient engagement issues
        • Training teams on creating and documenting patient engagement plans
        • Demonstrating how to integrate patient engagement plans with overall development plans
        • Helping initiate activities

      Carol Meyer Sokolowski
      VP, Therapeutic Area Management and Operations, Takeda Pharmaceuticals

    • Track B

    • 11:00 am

      How an Entire Large Pharma Team Incorporates Patients at Every Stage of Development

      This session will feature a large pharma team case study where members of the clinical development and patient engagement groups demonstrate how at each phase, a patient was involved in helping research and plan, the impact that was made by doing this and the method behind implementing those initiatives as a result of patient involvement.

      Stephen Yates

      Global Clinical Development & Medical Affairs, UCB

    • 11:30 am

      Translating Patient Insight and Patient Experience Internally into Clinical Development Deliverables

      This session will look at applying quality systems framework (structure, processes, outcomes) to patient engagement initiatives to help create clinical development deliverables that meet industry goals while aligning with patients needs. Key focus:

      • Reviewing the framework
        • Structure: Designing appropriate protocols that align with patients needs
        • Processes: What process needs to be in place to help articulate what patients need and translate those needs into elements of a protocol?
        • Outcomes: What are the outcomes based on incorporating the patient insight into this framework?
      • How do you have those internal discussions so you can effectively translate a patient need into helping do something different in your program/protocol? What are some examples?
      • What does the internal communication plan look like to do this?

      Mary Stober Murray

      Associate Director, Diversity and Patient Engagement, R&D, Bristol-Myers Squibb

    • 12:00 pm

      How GSK Implemented Pivotal End-to-End Engagement Initiatives at Different Stages of Development

      In this session, Marilyn Metcalf, PhD, Senior Director of Benefit Risk Evaluation, GlaxoSmithKline, will discuss the pilot program on end-to-end engagement that is currently underway at GSK. Dr Metcalf will walk the audience through the plan, provide an update on the progress and share some examples of the engagement initiatives at several stages of their development.

      Marilyn Metcalf, PhD

      Senior Director of Benefit Risk Evaluation, GlaxoSmithKline

  • 12:25 pm

  • Lunch

  • 1:15 pm

  • Afternoon Breakout Choices

    Attendees have the opportunity move freely between the two tracks to attend sessions of interest that will help with their own patient engagement initiatives.

    • Track A: Treatment Decisions, Early Development, Driving R&D

    • 1:15 pm

      Patient Perspectives: What is Your Decision Making Process When it Comes to Your Treatment?

      According to industry, there is still a need to better understand the decision making process a patient goes through when considering their treatment options, including clinical trials. Understanding these dynamics will help industry design appropriate initiatives that help meet its goals while aligning with patient’s needs. In this session, patients will lead a discussion that addresses:

      • What patients consider when determining to participate/not participate in a clinical trial?
      • What is the decision making process for patients?
      • How can pharma help support/provide tools that will help patients while deciding which treatment pathway they choose?
      • How can advocacy groups support this and how do they help inform patients?


      Jen Horonjeff, PhD

      Patient and Founder, Savvy Cooperative


      Tanika Gray Valbrun
      Patient and Founder, The White Dress Project

      Additional panelists TBD

    • 1:45 pm

      Involving Patients In Research and Early Clinical Development Phases

      Meeting with patients with unmet needs can help prioritize different indications. This session will provide case examples of how industry has involved patients in early R&D.

      • How do you involve patients to decide on indications/looking at unmet need and even formulation, dosing, etc?
      • How do you engage patients before Phase I or Phase II?
      • How do we need to work with patients in each of the clinical trial operation functions?


      Jodie Gillon

      Senior Director, Advocacy & Professional Society Affairs, Achillion Pharmaceuticals


      Jenny Ahlstrom
      Jenny Ahlstrom

      Myeloma survivor and Co-founder, CrowdCare Foundation

    • 2:15 pm

      How Patient Research Advocates are Driving R&D: A Patient Case Study

      In this session, patient and research advocate, Geraldine Blavat will walk the audience through 10 steps on how patient input is integrated through the entire clinical development continuum including:

      1. Developing the Study Concept
      2. Secure Funding
      3. Preparing the Study Protocol
      4. Creating Study Procedure
      5. Implementing the Study
      6. Monitoring the Study
      7. Analyzing the Data and Interpreting Results
      8. Disseminating Study Information
      9. FDA Review and Approval
      10. Post Approval Studies

      Geraldine Blavat

      Parkinson’s Foundation Research Advocate

      Karlin Schroeder

      Director, Community Engagement, Parkinson’s Foundation

    • Track B: Data and Metrics

    • 1:15 pm

      Engaging Patients By Giving Them What They Want…Their Data!


      Jenny Ahlstrom
      Jenny Ahlstrom

      Myeloma survivor and Co-founder, CrowdCare Foundation

      Cindy Geoghehan

      Cancer Survivor and Patient Advocate

      David Leventhal
      David Leventhal, MBA

      Director of Clinical Innovation, Pfizer, Inc

      Additional panelists TBD

    • 1:45 pm

      Measuring Patient Involvement

      In this session, pharma companies will present two case examples of patient-centered interventions that resulted in greater efficiencies/improved outcomes throughout the clinical development life cycle.

    • 2:15 pm

      Town Hall Meeting on Patient Engagement Metrics and Value

      This portion of the program will be a town hall style session that addresses the following questions:

      • What are companies measuring? What metrics will be used to measure success?
      • What do these measurements reveal and what can we do about it?
      • Are these metrics different depending on the stakeholder? Why and how to we close that gap?
      • What and how to benchmark and determine market value?
      • How do we determine Return on Engagement? What does that really mean?
      • How are we moving forward in a team approach- regulators, etc – on policies, guidance?
      • What are some of the centralized set of information about patient involvement?


      Bennett Levitan, MD, PhD

      Senior Director, Benefit-risk Assessment, Department of Epidemiology, Janssen R&D, LLC

  • 2:45 pm

  • Networking Break

  • 3:25 pm

  • Panel: Engaging with Communities to Educate and Enroll Diverse Populations

    This multi-stakeholder panel will discuss the complexities of diversity and what actions we can take to make sure that trial enrollment is reflective of the demographics of the populations.

    Key focus areas include:

    • What are sponsors doing currently doing to include diverse populations in trials?
    • What are some examples of how industry has approached diversity internally such as developing partnerships, education and awareness to identify and address the barriers of diversity?
    • How does industry assess the capacity of the sites they are working with and the ability to recruit diverse participants?
    • Why potential participants that are aware/not aware are not participating. What are their concerns of participation?
    • What are the clinical site teams challenges/issues on recruiting diverse participants? How do you incentivize sites to engage in diverse communities?
    • How has advocacy helped with outreach, awareness and recruitment of diverse populations for clinical trials?
    • Additional examples of what have been done that has worked and is scalable.


    Luther Clark
    Luther Clark, MD

    Global Director, Scientific Medical and Patient Perspective, Office of the Chief Patient Officer, Merck


    Diane Gross
    National Director, Advocacy and Programs, Lupus Research Alliance

    Additional panelists TBD

  • 4:00 pm

  • What Can We be Doing Better on the Site Level to Engage Patients?

    (Patient/ investigator/scientist relationship)

    • How can the pharma clinical trial team become an expert in the patient on a personal level?
      • Humanistic qualities and personal knowledge: When a patient commits to you for a study, learn who they are as a person and not a subject
      • Post study patient follow-up call to determine any adverse events
      • Constant communications throughout the trial
    • How can pharma ensure CROs/sites are including these aspects into their SOP?
    • What to measure at the site level? What is the Return on Engagement at the site level?

    Moderated by:

    Christine Pierre

    President, The Society for Clinical Research Sites

  • 4:35 pm

  • When Average Isn’t Enough: Harnessing The Expertise Of Patient Opinion Leaders

    As pharma struggles to turn the concept of patient engagement into more than just a feel-good catchphrase, there is a growing trend to partner with patients in a more meaningful way to impact development throughout the drug life cycle. What if you could create a Patient Opinion Leader (POL) Advisory Panel – a collaborative group of patients and caregivers possessing not only the knowledge and first-hand experience of living with their health condition, but also having expertise in a variety of subjects that intersect nicely with pharma to result in more valuable insights than the average patient could provide? Their collective experience in patient advocacy, pharmaceutical marketing, clinical trials and patient assistance programs among other areas of expertise converge to create the quintessential panel of POLs who can read a protocol without fear, perform UAT/UX testing and give actionable feedback and collaborate in a highly effective and broadly impactful way. In this session, attendees will learn:

    • How set up, select, and leverage the right POLs and employ a long-term advisory board to significantly impact the entire drug development lifecycle
    • Utilize an emerging model for an in-depth vetting process to develop a robust, sustainable, long-term relationship with key POLs to gain their unique combination of perspectives and expertise to support the design and development of a variety of initiatives across the product lifecycle

    Abbe Steel, MSc

    Founder and CEO, HealthiVibe LLC

  • 4:55 pm

  • Patient Engagement Sources and Solutions

  • 5:30 pm

  • Networking Reception

2018 Agenda

  • 8:00 am

  • Breakfast

  • 8:30 am

  • Co-Chair’s Opening Remarks

  • 8:45 am

  • Community Hospital Center of Excellence Program Case Study: Engaging and Educating Communities on Therapeutic Treatment Options

    Addario Lung Cancer Association has created, with physicians, a treatment pathway protocol that maps out the steps that every patient should have access too, including clinical trials.

    They are working with an elite team of specialists to create an unsurpassed paradigm for Lung Cancer treatment worldwide – a patient-centric, collaborative model to provide all patients, regardless of where they live, access to the newest and most effective diagnostic and therapeutic techniques. This is called the Community Hospital Center of Excellence Program. This new “standard of care” established at Community Hospitals will be accompanied by a formal seal of excellence awarded by the ALCF and will ensure that no Lung Cancer patient is left behind.

    David LeDuc

    Executive Director, Bonnie J Addario Lung Cancer Foundation

  • 9:15 am

  • FDA & Regulatory Perspectives

    • Impact of PDUFA VI and what to expect next?
    • 2018 Guidance on engaging patients
    • What is it that we want and don’t want when it comes to patient engagement metrics?
    • Patient reported outcomes

    Theresa Mullin, PhD

    Director, Office of Strategic Programs, CDER, FDA

  • 9:45 am

  • Overcoming Emerging Legal Challenges

    While the discussion around legal challenges is not new, there are still challenges and perceptions around legal that impede industry, patients and advocacy to engage one another.

    • What are the legal perceptions and fears that exist about patient engagement and how can we overcome them?
    • Patients want to know how can they contribute to industry without encountering a legal roadblock?
    • How is industry interpreting the laws around patient engagement? Each company interprets it differently.
    • How can we include patient engagement within the current laws that do exist?
    • How can we work with internal lawyers on what they need to know before you start your patient engagement initiative?
    • How do you collaborate cross functionally (commercial, regulatory, medical) to create compliant but patient friendly language?


    Roslyn Schneider, MD

    Global Patient Affairs Lead, Pfizer Medical


    T.J. Sharpe

    Melanoma Survivor & Patient Advocate, Patient Power

    Additional panelists TBD

  • 10:15 am

  • Networking Break

  • 11:00 am

  • How to Strategically Move an Effort Beyond Early Adopters

    Marc Boutin, JD

    CEO, National Health Council

  • 11:30 am

  • Case Study: Externally-Led FDA Patient Focused Drug Development Meeting with The National Kidney Foundation and Achillion Pharma

    As the FDA has stated, patient input is critical in helping them understand the context in which regulatory decisions are made on new drugs. Patients, advocates and caretakers can share through the PFDD meetings the impact of their disease on their daily lives, what matters most to them and their experience with current drug treatments. In turn, according to the FDA, “this input can inform FDA’s decisions and oversight both during drug development and during our review of a marketing application.”

    In this session, the National Kidney Foundation and Achillion Pharma discuss their experience organizing the externally-led PFDD meeting, what role they each took on, how they collaborated on what has become the gold standard in PFDDs.


    Meghana Chalasani
    Meghana Chalasani

    Operations Research Analyst, Office of Strategic Programs/Center for Drug Evaluation and Research (CDER), FDA

    Feldman David
    David Feldman, PhD

    Medical Project Director, National Kidney Foundation

    Jodie Gillon

    Senior Director, Advocacy & Professional Society Affairs, Achillion Pharmaceuticals
  • 12:00 pm

  • How FARA and Horizon Pharma Partnered to Improve Clinical Development and Trial Design- Case Study

    This session will feature how the Friedreich’s Ataxia Research Foundation collaborated with Horizon Pharma on aspects of clinical development and trial design to better understand patients living with the disease and how to better develop medicine to help manage their disease. Key areas of discussion include how they collaborated on the following:

    • Focus groups to better understand drug administration
    • Clinical trial design to ensure feasibility and efficiency
    • Recruiting patients for clinical trials
    • Open discussion at FDA meetings

    Kyle Bryant

    Director of the Bicycle Ride Fundraiser, rideAtaxia for the Friedreich’s Ataxia Research Alliance (FARA)

    Robert Metz

    SVP, Global Business Operations and External Affairs, Horizon Pharma

  • 12:30 pm

  • Lunch

  • 1:30 pm

  • FasterCures Report on How Patient Advocates Engage Industry

    Kim McCleary

    Acting Executive Director and Managing Director, FasterCures

  • 1:50 pm

  • Advocacy/Industry Partnerships:

    • How can I help bring the appropriate patient voice or representative patient voice to industry?
    • How can we work together? How do we set up a system?
    • How can we do match making with Patients and Industry?
    • How can patient advocacy help industry with recruiting and retaining clinical trial participants?
    • How are patient advocates engaging industry?
    • Examples of industry/patient advocacy group collaborations


    Kim McCleary

    Acting Executive Director and Managing Director, FasterCures


    Kyle Bryant

    Director of the Bicycle Ride Fundraiser, rideAtaxia for the Friedreich’s Ataxia Research Alliance (FARA)

    Farmer Jen
    Jennifer Farmer, MS, CGC

    Executive Director, Friedreich’s Ataxia Research Alliance

  • 2:30 pm

  • End of Conference