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Patients as Partners US2019 RECAP

Turning Discussion Into Action: Applying Patient Engagement Initiatives and Demonstrating Impact

OverviewThe Conference Forum is pleased to provide this recap of the 6th Patients as Partners US Summit held on March 11-12, 2019, in Philadelphia. The summit brought together industry, academia, individual patient advocates, federal officials and nonprofit organizations to discuss the ways in which patient input can be utilized in clinical trial development and throughout the product lifecycle for better outcomes and improved drug compliance.

The main themes of the summit were finding the best ways to capture patient input in clinical trial development, the increasing efforts by the Food and Drug Administration (FDA) to include patient preferences in drug, device and biologic product review, the efforts advocacy organizations can implement to prepare patients for enrollment in clinical trials, and the ongoing need to educate the general public of the value in participating in clinical trials.

Day OneThe program opened with remarks by program Co-Chairs Jan Nissen, VP, Patient Innovation and Engagement, Merck; Cynthia Verst, PharmD, President, Design and Delivery Innovation, IQVIA; and Suzanne Schrandt, JD, Director, Patient Engagement, Arthritis Foundation, on the evolution of Patients as Partners. Ms. Schrandt then shared her experience working with an esteemed research organization on a patient engagement effort that highlighted the need to engage patients early on in trial design.

Suzanne Schrandt, JD, Arthritis Foundation

Next, Mary Stober Murray, Associate Director, Diversity and Patient Engagement, BMS, moderated a VIP Fireside Chat with Judy Perkins, a Stage IV metastatic breast cancer veteran and patient advocate, who

1Patients as Partners US 2019 Recap

March 11-12, 2019Philadelphia, PA


shared her very personal and inspirational story of taking charge of her own treatment course by approaching the NIH about a clinical trial when she was “grasping at straws” to survive. She is the first person declared cancer free from metastatic breast cancer after a course of immunotherapy. When asked what advice she would give her younger self when diagnosed: “Go get a second opinion.” She added that there are two myths out there. “The first is that if you catch it early it will be okay and the more you cut out the better it will be. Neither are always true.” balanced story about the value of clinical

trial participation. They usually cover the same approach, focusing on one particularly astonishing case.” Getz pointed to the increasingly crowded media market and drive to sensationalize stories as a ‘challenging environment’ in which to educate the public. Newer initiatives he mentioned include Aware for All (educational campaigns geared towards local communities), crafting more enduring messages that play to personal relevance and ‘volunteerism,’ engaging healthcare professionals, and expanding into the international community. The morning continued with a robust panel discussion of the process patients go through when deciding whether to enroll in a clinical trial and what sponsors can do to personalize and support their decision. Panelists emphasize the plethora of smaller decisions and everyday lifestyle factors

The first is that if you catch it early it will be okay and the more you cut out the better it will be. Neither are always true.

- Judy Perkins, Metastatic Breast Cancer Veteran and Patient Advocate

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In the next session, Ken Getz, Director of Sponsored Research Programs, Tufts CSDD and Founder and Board Chair of CISCRP provided a review of global initiatives that have been implemented to raise awareness of the importance of clinical trials among the general public. He gave an update on Medical Heroes, a public awareness campaign, and spoke to the challenges of getting the word out in a fresh voice. “It’s rare to find a journalist writing a new and

Ken Getz, MBA, Tufts CSDD, CISCRP


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that are often overlooked by trial sponsors but can accumulate over time and dissuade patients from participating. The panel was moderated by Marilyn Metcalf, PhD, Patient Engagement Lead, GSK, who probed into common misconceptions about trial enrollment. Advice to patients included finding a specialist for their condition and gathering as much information as they can. According to Mary Stober Murray, Associate Director, Diversity and Patient Engagement, BMS, there are three questions patients ask themselves: 1) is it right for me? 2) Can I afford it? and 3) Does it fit with my life? She suggested pharma focus on decision support and better understanding the needs of different patient populations. Lilly Stairs, Patient and Advocate added, “Along the way, don’t lose that human touch.” The panel also spoke about the long-term impact of being in a clinical trial. Robert Long, Director, Rare Disease Engagement, Uplifting Athletes, shared his struggle with PTSD. “No one told me about the impact emotionally if you actually survive and beat the odds.”

Next, Ken Getz, Tufts CSDD, moderated a lively fireside chat between two senior industry executives on how to create a corporate culture that promotes patient engagement in trial development. Jayne Gershkowitz, Chief Patient Advocate, Amicus Therapeutics spoke to the various ways patients are involved, including educational sessions, cross-functional roundtables, and Patient Advisory Boards, and shared Amicus’ commitment to analyzing the implications and sharing them with patient groups and the FDA. Julie Gerberding, MD, MPH, EVP & Chief Patient Officer, Strategic Communications, Merck, spoke candidly about the challenges industry often faces internalizing patient input. “It can be a struggle to share negative information internally. When your desire is to keep a drug moving through a pipeline you don’t want anything to slow down the process.” Marilyn Metcalf, PhD, GSK moderating the

panel: Mapping Out the Patient Decision Journey

It can be a struggle to share negative information internally. When your desire is to keep a drug moving through a pipeline you don’t want anything to slow down the process.

- Julie Gerberding, MD, MPH, Merck

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The morning concluded with a series of six pharma case studies on applying patient engagement initiatives and demonstrating their impact. In the first track, Linda Kollmar, Executive Director, Scientific Medical Patient Perspective – Oncology, shared Merck’s strategy for bringing on a patient, as an equal stakeholder of their clinical development team, to provide patient input in protocol and trial design. Jessica Scott, MD, JD, Head, R&D Patient Engagement, stated that Takeda has conducted over 25 patient engagement activities across R&D. The company requires those in R&D to be involved in three different activities related to patient engagement each year. “We also like to work with external groups to see that patients have some training ahead of time because most have no sense of drug development and we want them to be prepared to contribute to the discussion.” Tammy Guld, Senior Director, Janssen Clinical Innovation, spoke to the advantages of remote trials, such as Janssen’s Direct-to-Patient approach, including reduced travel burden for patients and increased retention. However, the potentially challenging aspects of remote trials include limited data collection, privacy considerations, potentially high screen failure rates, new infrastructures with new vendors, and compliance. In the second track, Alistair Lindsay, PhD, Director, R & D, discussed GSK’s approach to improving diversity in clinical trials. He spoke to the importance of picking trial sites carefully to ensure diversity in enrollment.

He noted that Lilly had launched the first-of-its-kind program to train, mentor and equip investigators to conduct clinical trials that are designed and relevant to minority populations. Douglas Brooks, Executive Director, Community Engagement, provided examples of Gilead’s efforts to engage patients and communities in clinical research design to ensure use of culturally competent language, protocol, and engagement.

Mary Stober Murray, MBA, BMS, leading the Building Patient Engagement into Systematic

Processes roundtable discussion

A series of roundtable lunch gatherings gave participants a chance to discuss issues of common interest. They included building patient engagement into systematic processes much like quality interventions; connecting employee-centric culture to patient-centric culture; and an interactive mock clinical trial protocol exercise on involving patients in protocol reviews.


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250+ 65+ 30+ 15+ 6+

PEOPLE IN ATTENDANCE

SPEAKERS

SESSIONS

HOURS OF DISCUSSION

HOURS OF NETWORKING

Patients as Partners US By The Numbers


Allowing patients to be partners in clinical trials often begins with interacting with CROs and/or trial sites. The afternoon started off with a panel discussion on how to build patient engagement in the process, creating greater efficiencies. “It’s an ecosystem, not a hierarchy,” said TJ Sharpe, Stage 4 Melanoma Survivor and Advocate. “Sites are literally the boots on the ground for sponsors when setting up the programs.” Much of the discussion was about the importance of building trust. “With pediatric patients we have to get innovative,” said Veronica Alcine, Global Head, Patient Recruitment and Engagement, Covance. “We’re also attuned to what parents need.” Sharpe believes having information that is digestible give sites an edge and builds patient confidence. Stephen Yates, Global Clinical Development & Medical Affairs, UCB, and Head, Patient Engagement Working Group, TransCelerate spoke to the difficulty of being ‘in the middle’ between clinical teams asking for additional funds and time and advocacy groups saying ‘you have to do it’. He recommended upfront planning to eliminate the haggling and infuse the process with patient input from the start. The seamless inclusion of patient input into clinical trials is a universal goal. Each step of the patient journey brings unique opportunities for researchers and sites to learn from the patient’s experience, emphasized Cynthia Verst, PharmD, IQVIA. “Well before a clinical trial is developed, we gleam insights in a contemporaneous manner and get insights from our advocacy

partner network. We believe it enables us to measure outcomes because we have proof points built in.” She offered four ways to maximize integration: 1) place data curation expertise on platforms made accessible and scalable; 2) utilize broad and deep real-world evidence data sets; 3) put in place digital technology solutions; and 4) provide advanced analytics. Another challenge, according to Verst, is what to do with patients who are excluded from one trial but who could possibly be considered for a different trial. “What if we had a catch basin,” she asked, noting that 81% of patients want to hear about upcoming clinical trials.

Cynthia Verst, PharmD, IQVIA

What does a patient-focused clinical trial really look like? The challenge is engaging with patients in ways that minimize the disruption to their everyday lives. One way is taking the study to the patient, as done by companies such as GlobalCare Clinical Trials, noted Gail Adinamis, CEO. Another is creating a more consumer-focused



approach. Scott Schliebner, SVP, Center for Rare Diseases, PRA Health Solutions challenged the audience to appreciate the changing consumer, but stressed that logistics such as travel time, loss of work, costs, cultural barriers, and strain on family and friends remain obstacles. He feels, that in the future, “clinical trials will evolve around the patient and technology will help us move the needle from old school to something that fits seamlessly.”

The afternoon continued with an interactive audience pop-up session that highlighted services and technologies that help diminish patients’ challenges within a clinical trial. The sessions that followed focused on ownership, access, and control of patient data.

The next session focused on the nuances of data ownership in clinical trials and the myriad of ways data flows. Currently, two states (MD and OR) have legislation pending that would expressly state that patients

own their health data. Mark Scrimshire, NewWave, Medicare Blue Button 2.0 Innovator, CMS, spoke on a Notice of Proposed Rule Making (NPRM) released in February 2019 that is focused on how patients can get their hands on their data. According to Mr. Scrimshire, honoring patients’ wishes to access their health data has been a goal of the current and past Administrations, and advances are underway that would allow Medicare beneficiaries to access their data. Providing insights on the patient perspective was Bray Patrick-Lake, MFS, Director of Stakeholder Engagement, Duke Clinical Research Institute. “We owe patients transparency and the possibility to consent to how their data will be used.” Some patients don’t know they have access to their own data or may be confused about data analytics.

We owe patients transparency and the possibility to consent to how their data will be used.

- Bray Patrick-Lake, MFS, Duke Clinical Research Institute

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Next up was a robust panel discussion focused on the feasibility of data sharing and improving patients’ access to data while in a clinical trial. Trial results and individual data are understandably important to patients. According to Jessica Scott, MD, JD, Head, R&D Patient Engagement, Takeda, “There Mark Scrimshire, NewWave, CMS


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are proprietary concerns but several pharma companies are working together to develop a playbook to address this issue. It’s not the technology that is the barrier, it’s the fear factor that releasing data will somehow interfere with the scientific integrity of the study.” Thomas Wicks, Chief Strategy Officer, TrialScope, added, “There’s a risk it will be viewed as promotional. It really depends upon the context in which you share data.” Scarlet Shore, Head of Product and Platform Lead, Project Baseline, Verily, shared they had set up a Returns and Results Committee to understand which results to return, when to contact the doctor, and when we could contact the patient directly. Cynthia Verst, PharmD, IQVIA also reminded participants that there is a level of pragmatism that has to be considered. “The Plain Language Guidelines will help level-set the content and generalize what would be acceptable,” she stated, but local country constraints make things more complex.

Jean McCoy, SVP, Partnerships, Health Perspectives Group, LLC, closed out the afternoon with thoughts on how to bridge commercial and clinical efforts to enhance patient engagement, citing examples of other industries that are striving for excellence in consumer experience. “Stop looking at patients as end-users and instead see them as subject matter experts who are deeply invested as a valuable partner.” She encouraged industry to “look at what they’re doing on the commercial side of the business” to find ideas for better communicating and interacting with patients.

Day Two

Day two opened with discussions on FDA efforts to fully integrate the patient voice in drug development. Pujita Vaidya, Senior Advisor, Patient-Focused Drug Development Program Office of the Center Director (CDER) reviewed the agency’s approach to analyzing the risk-benefit profile of a drug under review and the progression of patient-centeredness through PDUFA legislation. The agency is soliciting applications for multiple grants to support the development of a publicly available standard core set(s) of COAs (Clinical Outcome Assessments) and their related endpoints for specific disease indications. Vaidya emphasized, “We are doing this to ensure confidence in the reliability and accuracy of product development, reduce regulatory uncertainty for the sponsor, promote rapid adoption, and make incorporating the patient’s experience in drug development the standard of care.”

FDA Patiet Engagement Synergistic Efforts Panel



During a panel presentation outlining FDA’s efforts in patient engagement across multiple centers, Morgan Moncur, MS, Senior Advisor, Science of Patient Input, Center for Biologics Evaluation and Research (CBER), stated that, “Patients can share perspectives with CBER through listening sessions organized through an agreement with NORD (National Organization of Rare Disorders).” Michelle Tarver, MD, PhD, Director, Patient Science & Engagement, Center for Devices and Radiological Health (CDRH), shared that the Center has a Patient Engagement Advisory Committee comprised solely of patients, caregivers, and patient organizations and will also implement Collaborative Communities this year. Pujita Vaidya, Senior Advisory, Patient-Focused Drug Development Program, reiterated the Center’s forthcoming Guidance Documents (2 & 3) and request for COAs in five designated areas.

ALL OF US is a US Precision Medicine program from the NIH that aims to enroll a million Americans and follow them for 10 years during which time participants will contribute biological, environment, and lifestyle data that will accelerate research and improve health. An update on the program was provided by Elise Felicione, MPH, Scientific Coordinator, Scripps Translational Science Institute/Senior Director, Janssen R&D. “Since its launch in May 2018, there are 150,000+ active participants, 100,000+ who have given measurements and bio-samples, and 455,000 online surveys have been completed.” The program has accomplished a representation of 77% from under-represented populations. The morning continued with a multi-stakeholder panel focused on the need to ensure diversity in clinical trials. Some of the techniques utilized by panel members’ organizations include one-on-one interviewing with patients to determine how they want to get clinical trial information, ensuring that patients know that trials exist, and working with organizations such as Foundation Medicine and those specializing in advocacy relations. Caroline Donovan, MPH, Manager, Patient Engagement, Lupus Therapeutics, described a pilot program at five academic centers in the US that have diverse communities. “We’ve trained 11 patients with lupus to do peer education. We’ve found that patients want to speak with someone who has gone through a clinical Audience members



trial. All of them are women of color from various backgrounds.” Patricia Roselle, Global Head, Patient Network Management, Sanofi, summarized, “We have to be prepared for a changing population. We will need the right representation and ways to overcome the barriers as they come up.”

online community where the company and patient or caregiver can engage with each other about the disease.”

We have to be prepared for a changing population. We will need the right representation and ways to overcome the barriers as they come up.

- Patricia Roselle, Sanofi

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The next session discussed mindfulness in design and how patient feedback can provide needed insights into technology selection and patient-friendly tools to support and optimize clinical trials. Abbe Steel, MSc, CEO, HealthiVibe, LLC, provided important advice. “Try to make the technology as real world as possible. Let patients take them home and play with them and try out different approaches or different controls.”

Daniel Donovan, CEO and Co-Founder, rareLife Solutions, then discussed the value of building a digital home to provide patient support education and clinical trial awareness. “Patients with rare diseases don’t want to go to Instagram and Facebook to talk about their disease. They want pharma to establish an

Patients as Partners Attendees

How might biopharma help patients become knowledgeable partners? Industry is eager to provide patients with as much information about clinical trials as possible, but are concerned of the appearance of undue influence. On the other hand, patient advocacy organizations do not have the financial resources or staff to adequately prepare and train members for participation in a clinical trial. Kevin Freiert, Owner and Principal, Salem Oaks, facilitated an honest look from a patient and sponsor perspective. Nicole Horvath, Regulatory Manager, Global CMC – New Products, Pfizer, (also a self-identified patient) said, “There’s a misnomer that patient education is needed. There are many well-educated patients but what is needed is an understanding of how industry works, all the acronyms, and how the development process works so they can more fully participate.” Mr. Freiert added, “Everyone


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else is a professional who has been doing the work for many years. A patient may be new to their disease. Education is key.” The morning concluded with a session on mobile clinical trials. Hassan Kadhim, Director, Clinical Trial Business Capabilities/Project Team Member, BMS/CTTI described CTTI’s evidence-based recommendations for engaging patients and investigative sties through the planning process and execution when utilizing mobile technology. He identified three important areas: the informed consent process and attention to literacy, including technical literacy; explanation of privacy risks; and providing patients with easy access to technical support. “Most patients want to get help from their physician or a study site, not someone in tech support.” Cindy Geoghegan, Patient Advocate and Project Team Lead, CTTI, added: “Find ways to return value to patients. They will expect something out of their participation and most of them want it in real time.”

The afternoon started off with a discussion of one approach used to accelerate timelines in a global Phase III program. According to Mark Summer, President, Patient Engagement, WCG Clinical, “Most recruitment models focus on doing some type of outreach to patients and use different channels to achieve that. We flip that upside down and start with the site because the site has been treating these patients. We put an enrollment assistant on-site at the research site to engage directly with patients.”

Cindy Geoghehan, CTTI (left) and Hassan Kadhim, CTTI (right)

It’s not only important to engage, it’s how you engage that’s important.

- Stephen Yates, UCB/TransCelerate

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The vision of the TransCelerate Patient Experience Initiative is to increase engagement and partnerships between sponsors and patients and to create better experiences for clinical study participants. In the next session, Stephen Yates, Head, Patient Engagement Working Group, described two new toolkits TransCelerate is developing: the P-PET (Patient Protocol Engagement) Toolkit and the SPFQ (Study Participant Feedback Questionnaire) Toolkit. These toolkits include a sponsor-facing user guide, resource guide, and various templates. “It’s not only important to engage, it’s how you engage that’s important,” he noted. “You need to ask the right questions. Otherwise we will not get actionable answers.”


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Next up was a panel discussion on how clinical research and trials could be improved through effective pharma-advocacy partnerships. Panelists took a deep dive into the elements of effective, sustainable relationships, emphasizing the need to build long-lasting relationships. Both sides have different top points for engagement and timing. According to Ellen Miller-Sonet, JD, Chief Strategist and Policy Officer, CancerCare, “Communication and transparency are key.” A continuous series of one-offs are not helpful. “We want to build upon prior findings.” Kristen Smedley, President, Curing Retinal Blindness Foundation, added that pharma must “be a giver in as many ways as possible” and patients don’t need to be ‘fixed.’ Asked what had they learned from something that didn’t go well, Keri Yale, Health Patient Affairs and Engagement, Boehringer Ingelheim, responded, “Fail and fail fast.” You learn something from this and should share those experiences more readily internally. Tiffany Westrich-Robertson, CEO and CO-Founder, International Foundation for Autoimmune & Autoinflammatory Arthritis, added, “We don’t call it failure. We call it learning. You can’t be afraid to give it a shot and from those failures we make progress.”

The afternoon continued with a panel discussion on how the device industry uses the science of patient input to impact the total product lifecycle. As with drug development, manufacturers and regulators are increasingly including patient perspectives in the design and testing of medical devices. However, consensus on when and how to incorporate this input varies. Stephanie Christopher, Program Director, Medical Device Innovation Consortium (MDIC), and moderator, opened with an overall description of the device industry’s collective work in patient engagement through MDIC. “MDIC coordinates the development of methods, tools, and resources used in managing the total product life cycle of a medical device to improve patient access to cutting-edge medical technology.” Whether or not to include a patient preference study

Communication and transparency are key.

- Ellen Miller-Sonet, JD, CancerCare

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Panel: How the Device Industry uses the Science of Patient Input to Impact

the Total Product Lifecycle


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is determined by the risk-benefit ratio of the device. Barry Liden, VP, Patient Engagement, Edwards Lifesciences spoke about patient engagement in the device industry, which depends on the therapeutic area as to how patients are engaged. MDIC surveyed device companies and found that about half never gain feedback from patients prior to finalizing a study protocol and 60% never gain feedback after the protocol is finalized. Suzanne Schrandt, Arthritis Foundation, said, “We’re struggling to get involved. If asked at all it’s usually in the late stages. Going forward we need to model what is going on in drug development. And don’t forget about the patients already implanted with a device. They are a great resource for information.” What can pharma learn from the device industry? According to Matt McCarty, Global Head, Patient Engagement at ICON, “Patient preference evidence is applicable but the

device industry has the best guidance from regulators on how to do that. We should be cross-sharing information on how to do this.” According to Mr. Liden, “Collaborating with the patient community is essential. Industry should support patient advocacy organizations and not look at it as ‘what’s in it for us’. We also need to see FDA as collaborators too.”

The final session of the day was a two-part session discussing what patients face when considering enrolling in a clinical trial as part of their course of treatment. The first session focused on barriers. According to Cindy Geoghegan, CTTI, pharma must be able to respond quickly to patients responding to trial advertisements or risk failure. Ellen Miller-Sonet, CancerCare shared details of patients who experienced hurdles, including one who did not have a primary care physician to help coordinate care while in the trial.

Panel: Better Strategies to Inform Patients on Clinical Research and Trial Participation

Collaborating with the patient community is essential. Industry should support patient advocacy organizations and not look at it as ‘what’s in it for us’. We also need to see FDA as collaborators too.

- Barry Liden, Edwards Lifesciences

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The second session was a panel discussion on how to collaborate and create better strategies to inform patients of clinical research and trial participation, and also how to inform physicians on getting involved. Moderator Irfan Khan, MD, CEO and Founder, TrialScout shared his personal experiences. “We need to make both points of contact a little easier. I’m excited to hear advocacy groups are giving voices to those without a voice. But the community of physicians I’m hearing about who refuse to refer a patient to a clinical trial is haunting.” Linnea Olson, Patient Advocate, now in a trial for lung cancer, shared her experience. “I did my own risk-benefit analysis and this time the trial was the only way to go.” However, she said, “I was offered the opportunity when I was first diagnosed but at the time, I had little interest. I was worried it would hasten my death or make it worse for my kids.” Kelly Wade, MD, PhD, Neonatologist, CHOP & CHOP Newborn Care at Pennsylvania Hospital spoke to the unique needs for research involving children. “We’ve identified four areas of importance: 1) it’s a team sport and we’re on the same team; 2) we operate a NICU like a family—center care model; 3) we have the luxury of time because we can anticipate, even before the baby is born, how to promote care; and 4) we share stories of former patients and trials.” In contrast, Alistair Lindsay, PhD, Director R&D and Cardiologist remarked, “When someone

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comes in with a heart condition, every second counts. We don’t have the luxury of time to always think about a clinical trial as a treatment option.” Sanjay Sethi, MD, Professor and Chief, Pulmonary, Critical Care and Sleep Medicine, University of Buffalo agreed that timing is not always open. “Not all physicians are against studies but we often do not have the luxury of timing. In our conversations sometimes it is in the last conversation that it comes up – ‘how about research’ – and it’s the last thing to ask.” Ms. Wade added, “The first thing you should say is ‘no matter what you decide about being in research will not impact the care you receive from me.”


Many thanks to Gwen Mayes for her reporting and to Marie Recine for

contributing to the Patients as Partners report summary.

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