Polling and Q&A WiFi Network: ISPORGlasgow Password: IQVIA2017 https://myispor.cnf.io/ Step 2 ISPOR Congress Platform Step 3: Please select workshop W16 Tuesday 7 th November, 4.45pm Mobile App Search ISPOR in the App Store or on Google Play! Step 1 Patient engagement: The future for outcomes research and clinical trials? Bryan Bennett, PhD Director, Adelphi Values, Patient-Centered Outcomes 1
What we intend to cover in this workshop 1 2 3 4 5 What is Patient Engagement? Case Study 1: Oncology (and beyond) Case Study 2: Gout Discussion Where to next? 3 2
Patient engagement > A current buzz > What is it? > Why is it so important now? > Is it really beneficial to engage patients? 5 3
Patient engagement > The many faces of Patient Engagement (Gallivan et al 2012) > 15 different terms each with multiple definitions An active partnership between members of the public and researchers in the research process, rather than the use of people as subjects of research. (Snape et al, 2014 PiiAF) Persons with a relevant disease (including caregivers), who operate as active research team members on an equal basis with professional researchers, adding the benefit of their experiential knowledge to a research project (OMERACT- de Wit et al, 2011) Research that is informed by the perspectives, interests and values of patients throughout the research process, from the selection of research questions to the dissemination of research results. (Mayo clinic, 2012) 7 Evolution of patient engagement 6000 Google Scholar hits from 1999 to 2017 5000 4000 3000 2000 1000 0 1999 2000 2001 2002 2003 2004 2005 2006 2007 2008 2009 2010 2011 2012 2013 2014 2015 2016 2017 8 4
Patient engagement is not new! > Women's health movement (from early 1970 s) Information on gynaecological health for lay person. Woman had a right to be full partners with their doctors in making decisions about their bodies. Shift in power and decision making > AIDS activists (1990s) Campaigning for rights to public database of clinical trials which we now know as www.clinicaltrials.gov > Dave debronkart, cancer patient and blogger. E-patient (equipped, enabled, empowered and engaged). Activist for healthcare transformation through participatory medicine > Marginalised groups in society taking control > Re-distribution of power in health 9 Patient engagement in Pharma is evolving > 2014 Sanofi appointed a Chief Patient Officer Find better, more productive ways of integrating patient and caregiver needs into Sanofi activities Medical Intelligence and Patient Perspectives (MIPP) providing a platform for patient engagement throughout the drug development process > Pfizer: online crowd sourcing model and social media campaign Collaboration with patient advocacy groups Designed to engage patients with chronic pain and healthcare professionals > Many other pharma companies are engaging patients in the drug development process 10 5
Getting it right > "More often than not, pharmaceutical companies patient engagement is still done to, not done with patients. It s small wonder that they fail so often as a consequence. > The patient should be the engager; the company should be the engaged. How can genuine unmet needs be served otherwise? (Garces et al 2012) > Level of patient engagement has to be considered and justified, reflective of why patients are engaged in the research process. 11 What to expect from this workshop > Patient engagement in the development of patientreported outcomes (PROs) Getting to know what the patient thinks about clinical trials > Patient engagement in qualitative clinical outcome assessment research Specific to Gout Effective use of Patient Advisory Groups Novel approaches to data collection 12 6
Trial Feedback Questionnaire Stephanie Manson, Senior Director, HEOR Excellence, Novartis ISPOR Glasgow 7 Nov 2017 Disclaimer The opinions expressed in this presentation and on the following slides are solely those of the presenter and not necessarily those of Novartis Pharmaceuticals Corporation ( NPC ). NPC does not guarantee the accuracy or reliability of the information provided herein. ISPOR Glasgow 2017 14 Novartis Business Use Only 7
Trial Feedback Questionnaire Objective Understand Patient Voice: What is driving patient experience on trials? Feedback loop: Identify trial issues in near real-time to improve studies based on actionable feedback ISPOR Glasgow 2017 15 Novartis Business Use Only What is the Trial Feedback Questionnaire Brief patient questionnaire designed to capture patient trial experience Independent of disease and treatment Structured questions about key trial experience aspects Both protocol specified and site specific components Library of optional questions Questionnaire tested in 400+ trial experienced patients across disease and development spectrum ISPOR Glasgow 2017 16 Novartis Business Use Only 8
Questionnaire administration 17 questions divided between 3 time points trial enrollment (n=4), during (n=8) and trial end (n=5) Electronic questionnaire Administration via epro system or URL given on postcard Usage guidance available User guide and FAQ to answer common implementation questions ISPOR Glasgow 2017 17 Novartis Business Use Only Data Handling Data monitored and shared with study teams in real time Study dashboard for automated data visualization Where feasible, improvements implemented directly into study Anonymized study feedback provided to sites, sitespecific feedback given once they have 5+ patients Database to be created to benchmark across trials ISPOR Glasgow 2017 18 Novartis Business Use Only 9
Free Text Free text key in patient testing, currently being tested Box monitored for 24hr spontaneous AE reporting Small text entry field to prevent excessive comments Specific comments allow study adaptation to be more patient-centric Monitoring benefit vs. burden of free-text In testing, free text was used in 22% of responses (n=72/324) with 0 spontaneous AE reports ISPOR Glasgow 2017 19 Novartis Business Use Only Which Trials Would Benefit? Trials with long duration Prevent early discontinuation due to practical concerns Trials with high recruitment targets Optimize enrollment process, keep patients engaged Trials with high patient commitment Understand patient reaction to requirements Trials with novel design Understand how new design aspects are perceived by patients ISPOR Glasgow 2017 20 Novartis Business Use Only 10
Learnings to Date Act of measuring makes study teams and sites more vigilant Micro-issues can weigh heavily on patients Diverse reactions from stakeholders ISPOR Glasgow 2017 21 Novartis Business Use Only Interested in learning more? Questionnaire is open access, with no usage fees for other parties Contact Stephanie.manson@novartis.com for questionnaire and additional documentation ISPOR Glasgow 2017 22 Novartis Business Use Only 11
A Patient-centric approach to qualitative clinical outcome assessment research: a case study in gout Katja Rϋdell, currently employed by Shire/ QMUL but at the conduct of this research employed by Pfizer Sophi Tatlock, Patient-Centered Outcomes at Adelphi Values Rüdell R, Tatlock S, Panter C, Arbuckle R, Symonds T. Assessing the methodological value of digital real-time collection of qualitative content in supporting in-depth qualitative interviews exploring the symptoms and impacts of gout on healthrelated quality of life. ISPOR 17th Annual European Congress, Amsterdam, 8-12 November 2014 Tatlock S, Rüdell K, Panter C, et al. What Outcomes are Important for Gout Patients? In-Depth Qualitative Research into the Gout Patient Experience to Determine Optimal Endpoints for Evaluating Therapeutic Interventions. The Patient. 2017;10(1):65-79. doi:10.1007/s40271-016-0184-x. 12
Background and objectives for case study To employ innovative and patient-centric methods: To gain an understanding of patients experience of gout symptoms, flares, and the broader HRQoL impact of gout To develop a conceptual model of this condition Assess the appropriateness of existing instruments in measuring the symptoms and impact of gout 25 Overview of methodology Real-time data capture Patient interviews (Concept elicitation and cognitive debriefing) Engagement of expert clinicians and patient advisory group (PAG) Modification of existing measure/ Development of new measure 26 13
Real-time data collection (N=9) Example tasks The picture shows when the foot is inflamed and the swelling is so bad and it hurts so bad you can t even get up off the bed and hop down the steps just to get something to drink so you can take a pill RTDC Pictures and videos *Nine patients participated in the RTDC one of the original ten patients had to drop-out due to medical reasons. Of note, some patients are yet to complete all of the tasks therefore data for each task may not be based on all nine patients. This picture of my right hand in a paralysed state, after chopping up some onions and bell peppers and seasoning for my food, it s not a great feeling. I am in a lot of pain, whats more it delays me from cooking 27 Engagement of Patient Advisory Group and expert clinicians in case study Patient-advisory group Interview guides and informed consent form Conceptual comprehensiveness Relevance and understanding /interpretation Patient perspective on findings Relevance and suitability of existing instruments Study documents Conceptual model Instrument selection Interim and final results Decision to modify or develop Inclusion/ exclusion criteria Clinical relevance of concepts Clinician engagement Clinical relevance of concepts Clinical input on measurement strategy Clinical input on measurement strategy 28 14
Patient advisory group: approach Patients identified through a patient recruitment agency In-depth training on PRO development methods and the specific aims of the project Input throughout the project through teleconferences and presenting via WebEx 29 Considerations regarding the methods of patient recruitment Patient advocacy group Market research recruiter Pros Cons Pros Cons Engaged and knowledgeable Less representative than typical patients? Potentially more representative of target population Greater costs in management of engagement Willing to dedicate time Identification of patients may impact timelines Purposive sampling range of characteristics May be less responsive and flexible 30 15
Patient Advisory Group: optimal number of participants Ease of scheduling and More representative + + obtaining input More time for participants to talk Reduced burden on specific participants Fewer participants More participants Are the views representative? Dynamics of group discussion may be hindered Ensuring all voices are heard How to reconcile divergent views Need to balance optimal methodology with feasibility, cost and timeline limitations 31 Training of the patient advisory group The patients were provided with background materials and participated in a 2 hour conference call to train them on PRO development methods and specifics of the project The sessions started with an ice breaker to help put the patients at ease More extensive training delivered face to face might be ideal, but was not feasible for this study 32 16
Output Input Methods of patient engagement Webex/Skype teleconference Face to face Digital/app engagement Greater access Cost efficient May bias sample Scheduling challenges still an issue In-depth data/feedback Scheduling challenges Costly Greater access Flexibility in scheduling Cost efficient May bias sample Discussion with research team more limited 33 How did patient engagement guide and drive the project to obtain patient-centric outputs? Patient-advisory group Interview guides and informed consent form (ICF) Conceptual comprehensiveness Relevance and understanding Patient perspective on presentation of findings Relevance and suitability of existing instruments Study documents Conceptual model Instrument selection Interim and final results Decision to modify or develop Revised language in ICF Revised question order Confirmation of relevance Confirmed relationships between concepts Completion burden Feedback on language /relevance/ recall Confirmed patientcentric findings Informed decision to not modify existing measure 34 17
Value of engagement: what was gained in this study by engaging patients? Additional depth of understanding for all stakeholders Sponsor Research team Clinicians Patients Patient-centric approach Increased confidence the results reflect the patient experience Increased credibility of research and selection of endpoints 35 Patient perspective I think the worse thing was trying to coordinate my work schedule and my time to assist with the manuscript [and] with research Seeing the results and listening to other peoples experiences and stuff I realised I was learning also and contributing I didn t realise by contributing, I was actually helping and that is what encouraged me to keep involved. Some of it was a little bit above my head as far as understanding some of the charts [but] it was pretty clear and there was very little confusion on my part as far as understanding what was going on 36 18
Key learnings, takeaway messages > Involvement of a patient advisory group definitely adds value to a research study, but Requires careful planning and consideration Important to educate patients and build in time to ensure they feel comfortable Likely to add timelines and requires flexibility in scheduling > The ideal in terms of involvement must be balanced with a practical approach for both sponsors and patients time and resources are not unlimited > However, a tokenistic approach is no longer acceptable 37 Acknowledgements and further information > Would like to acknowledge the contribution of all of the other team members (not least the patient advisors!): Patient advisors: Victor Villa and Katherine Hagarty Adelphi Values: Charlotte Panter and Rob Arbuckle Clinical Outcomes Solutions: Tara Symonds* Expert clinicians: Dr. Will Taylor, Dr. Lesley Harrold Rüdell R, Tatlock S, Panter C, Arbuckle R, Symonds T. Assessing the methodological value of digital real-time collection of qualitative content in supporting in-depth qualitative interviews exploring the symptoms and impacts of gout on health-related quality of life. ISPOR 17th Annual European Congress, Amsterdam, 8-12 November 2014 Tatlock S, Rüdell K, Panter C, et al. What Outcomes are Important for Gout Patients? In-Depth Qualitative Research into the Gout Patient Experience to Determine Optimal Endpoints for Evaluating Therapeutic Interventions. The Patient. 2017;10(1):65-79. doi:10.1007/s40271-016-0184-x *Tara and Katja both worked at Pfizer during the conduct of this project 38 19
Open Discussion > Thank you for your attention > Any Questions 39 20