patients as partners in clinical Newsletter

Janssen: Summer 2021 Lessons in Making a the Patient Data Patient Data Return issue Return Portal

ReveraGen: GSK: Patient Returning Feedback to Results in a Create a Rare Disease Patient Hub Trial

MRCT: Clincierge: How to Move Understanding Forward with Trial Genetic Data Participation Return Barriers

Kaiser Pfizer: Permanente: Empowering Qualitative Patients with Design Indivual ApproachesPatients as Partners Newsletter Summer 2021 Issue Result Return 1 Welcome Letter

Welcome to the Patient Data Return issue of the Patients Patients as Partners as Partners newsletter. This issue highlights R&D pioneers Newsletter who are working to return individual results to patients, The Conference Forum navigating the hurdles of health literacy, security, data 1430 Broadway portals and : Suite 1510 New York, NY 10018 (646)-350-2580 David Leventhal, Pfizer, describing Pfizer’s work in data return, as well as the multi-faceted challenges Danny McCarthy individual result return faces. Content Editor

Suzanne Gaglianone, ReveraGen, taking the audience Valerie Bowling through how their rare disease therapeutic organization Executive Director piloted returns for a Duchenne Muscular Dystrophy trial. Meredith Sands Executive Director, Scott Gray, Clincierge, discussing details can have big impacts on how patients experience . Business Development

Kate Woda Kristin Blouch, GlaxoSmithKline, outlining how her GSK Conference Director team put together a patient portal with patient insights for the impactful return of data. BreAnna Bugbee Marketing Manager Kafayat Babajide, Janssen, giving lessons learned from creating a patient data return portal. Mark Scherzer Patients as Partners Europe Sandra Prucka, Indiana University, discussing the Conference Producer complexities of genetic data return and how it can empower patients in healthcare decision-making.

Leah Tuzzio, Kaiser Permanente Washington Health Research Institute, advocating for the qualitative design approach and learning from real patient insights to return results and design research trials.

The Patients as Partners newsletter is the official publication of the Patients as Partners in Clinical Research conference, designed for R&D executives and patient advocacy. Enjoy the Summer 2021 issue.

Patients as Partners Newsletter Summer 2021 Issue 2 Featured Guests Recommended Media Enjoy more media of patient 4 David Leventhal, Senior Director, engagement in clinical research. Clinical Trial Experience, Pfizer

6 Suzanne Gaglianone, Patient Coordinator, ReveraGen BioPharma webinar Patient Data Access Initiative: Introduction to Patient Data Return Scott Gray, CEO, Clincierge 8 webinar Patient Data Access Initiative: Optimizing the Patient Experience in Data Return Processes 10 Kristin Blouch, Director, Molecular Disease Characterization & Strategy, webinar Patient Data Access Initiative: GlaxoSmithKline Patient Data Return Pilots: Lessons Learned

webinar Patient Data Access Initiative: , Associate 12 Kafayat Babajide How to Get Started in Patient Director, Patient Portals, Janssen Data Return

blog Decentralized Trials: Considerations for Building a New , Assistant Professor 14 Sandra Prucka Patient-Centric Model of Clinical Medical & Molecular Genetics, Indiana University podcast How to Utilize Technology to Truly Increase Diversity in Clinical Trials

Leah Tuzzio, Senior Research podcast 16 Solving for the Patient Retention Associate, Kaiser Permanente Problem in Clinical Research Washington Health Research Institute podcast Using Sensor Technology to Deploy Decentralized Trials and 18 Returning Patient Data Webinar Series Improve Patient Care

Patients as Partners Newsletter Summer 2021 Issue 3 How to Return Individual Data to Empower Patients

David Leventhal, Senior Director, Clinical Trial Experience at Pfizer, is responsible for a portfolio of innovative approaches to address vital patient engagement challenges across Global Product Development. These challenges span the entire patient journey from before an individual might consider a clinical trial, their experience during a trial, and after they complete their participation experience.

Can you describe your involvement in individual So we knew that we had to do more. We wanted to make sure that we were modernizing the Blue Button patient data return? standard: we then started to see electronic health I’ve been involved in the whole concept of the return of records and the adoption of the FHIR standard. So individual data for nearly a decade now. Pfizer was one we needed to make sure we were modernizing in that of the first companies to really start to take seriously respect, but also that our approach to data return was the idea of returning individual clinical study data back one of maximally returning data. to back to participants, when we started our Pfizer Link alumni community, all the way back in 2011/2012. What does “maximally returning data” mean? And while we were not doing data return at the time, By that, we mean returning as much data as we the idea of creating an alumni community where past reasonably can, without jeopardizing the research that clinical trial participants can expect to receive the results we’re pursuing. Returning data at the end of the study of their clinical trial in some meaningful way, that’s where is something that we firmly believe in and is something Pfizer has been thinking seriously about this. And in the that we’ve been working on, and continue to work on intervening time, while individual results were out on the with our data transparency efforts. To provide a full data horizon, the concept of making sure that clinical trial set, at the end of the study, is very important. participants found out the results of their clinical trial written in language that they could understand took off. But there’s also the concept of returning data during a clinical trial, which a participant has the right to ask for. What we’ve been doing at Pfizer is advancing a lot of They can ask their principal investigator, “Can I have the thinking around what was known as the Blue Button my latest set of labs and my vitals?” What typically standard. We wanted to use that same idea, but thought, happens is that a PI will print that out for them, and give “Wouldn’t it be great if people could click the Blue Button them a piece of paper. As long as it’s not something and be able to download the data that was collected that will unblind the research, that’s typically okay and during their clinical trial?” acceptable for participants to ask for. The problem is that the data that they’re getting is not How did you start piloting off that standard? usable; they have a piece of paper. There’s a sense of We began a series of pilots based on the Blue Button empowerment in the patient communities: patients want standard, which was at the time HL7-compliant. We not just printouts of their data or to know the aggregate did it through Pfizer Link, which created the ability to results of the study. They want their data, and they want authenticate your identity, creating an account based on to be able to track the progress of their health. They your participant ID, study ID and site ID. want to share that data with their primary care physician after the research is completed. The security around this was really important. And then the pilots that we ran were really around very simple As a company, we feel that we have a responsibility to things, such as being able to download routine labs and fulfill that social contract with clinical trial participants. vitals after a study had closed. We were able to achieve We’ve been pursuing it at the speed that the that; we were able to do that work, around 2014/2015. complexity of this allows us. Each data element has a varying level of complexity. It’s everything from what But the realization was that it was essentially old their concomitant medicines are, to what their routine data, right? For someone who has participated in a is, what their medical history is – on the simple side – clinical trial, it could be a year and a half or two years, to genomics and biosamples at the very complex side depending on when they exit the study, before you were of things. able to give them back data that was relatively aged. We want to work towards maximally returning data and creating an infrastructure to be able to do it.

Patients as Partners Newsletter Summer 2021 Issue 4 That’s where the greatest challenges are. For many Therefore, so that you’ve made selections and said, sponsors of research, getting data out of systems can “These are the data elements that we expect to return, be difficult: the systems could be quite disparate and and when we expect to return them.” the technology can be aged and difficult to be able to do in an automated way. And so if you’re going to be You know then if it’s all this data at the end of the study, able to do this right, you must have a plan for returning or there are a couple of data elements that you may data at the study’s conclusion, as well as during the decide to return during the study. Also, we collect a lot study, before the study even starts. We’ve been very of data in the course of clinical trials that may or may not progressive in our views around returning data, in be important to an individual. The feedback that we’ve trying to come up with an automation schema and an gotten from patients and patient advocacy groups is that infrastructure that’s conducive to doing this, as well as if it’s happening to my body, I want to know. That’s a making sure we have solid operational processes, which reasonably good starting place around data return. are part of protocol development, that allow for the return of data to participants. Additionally, there’s all of this other data that’s being collected over the course of a clinical trial. If you have an electronic diary in a vaccine clinical trial, for example, or other patient-reported outcomes, we think that that data Expectations need to be really is particularly important, along with adverse event data clear about what data is going potentially being returned. to be available, and when. If If you as an individual can see the adverse events, and be able to see in your electronic diary that you were feeling there’s data that is not going a certain way on that day, it allows people to take control of their story. They then have the data to support their to be made available, there experience in the clinical trial. Being in a clinical trial can should be a reason why. have some life-changing effects. If the outcome of the study was positive, or even if the outcome of the study was negative, it was still a big thing. The decision process What has been the response from patients who for an individual to take those cognitive steps to muster received their data? the courage to say, “I’m going to do this, and here are my reasons for doing it,” – and the reasons why people It’s early to tell, and different sponsors have their own participate are quite variable – but all of this data helps an experiences. At least in our pilots, I think it was certainly individual own their story around their trial experience. appreciated. People like getting their data back. The feedback that we have gotten through advisory boards, raising the question, “Would getting your data back, What would be your guidelines for how to either during or after the study concluded, affect your communicate clearly what patients can or cannot retention in the clinical trial in any way?” expect, data-wise? Most of the responses that we’ve gotten have been This is simply good communication upfront. When positive, that they would feel a sense of being well- you’re providing participant materials in the clinical trial, considered and cared for as part of their study be really clear. There’s still discussions that need to be participation. I don’t have any hard evidence that had around if you put this in the informed consent, or if suggests that it affects retention rates in any way, but it’s it should be put in patient-facing materials, for example. still the right thing to do. But however it’s done, expectations need to be really It’s important that we’re being responsive to the demands clear about what data is going to be available, and of the patient community, and they’ve told and they’ve when. If there’s data that is not going to be made spoken pretty loudly that they would like access to their available, there should be a reason why. “Well, we can’t data. We should respond positively to that. return this data, because this data could be indicative of some therapeutic effect in the clinical trial? That could invalidate the research in some way.” Or “It could have What are some of the operational you take action that could affect the outcome of the recommendations for pursuing this in a larger research.” company? This is really just being clear, utilizing our best health The plumbing components of this – simply being able to literacy principles in how we’re communicating these pull data out of data repositories – is very difficult. But things. Once you’ve set expectations, meet them. The it becomes exponentially more difficult the later in the last thing that you want to do is have a mismatch of process you are considering data return. Having a data expectations at the end of the trial, where an individual return plan as part of your protocol development process, thought they were getting these data types and they is a best practice and vital. We have no compunction ended up not. of having a conversation about what our data management plan is for a particular clinical trial, or what That is very important and could make or break any our statistical analysis plan is for a particular clinical trial. customer experience. I don’t think clinical trials are really A new activity is a data return plan. That plan needs to different in any way than any experience that they might be created at the same time as those other plans. have in healthcare elsewhere. You say what will happen and you try to meet or exceed those expectations.

Patients as Partners Newsletter Summer 2021 Issue 5 How One Study Team Piloted Individual Result Return in Their Rare Disease Trial Suzanne Gaglianone is the Patient Coordinator at ReveraGen BioPharma. She was the lead of the study team for the VISION-DMD vamorolone Duchenne Muscular Dystrophy trial that returned individual results to the caretakers of child participants in trials, serving as the liaison and firewall to ensure the secure passage of data. How was the process for returning results (via a How did you get this data return off the ground? mailed, encrypted drive) developed? Reveragen is a very small, eight-employee company and It was a back-and-forth conversation, between myself, the company has always been quite patient-focused in Laurie Conklin, our CEO Eric Hoffman and others, their approach. This included engagement of parents of mostly centered around the fact that we needed a new DMD children in study design, attention to the burden in recruitment and informed consent process because trials on families by limiting child visits, and even seeing we did not include return of results in our clinical trial what parents felt was important in quality-of-life and protocols. We also wanted to centralize the process, looking at what endpoints should be included in trials. which involved my running it from ReveraGen, rather And then hiring me – they hired me as the international than burdening the 12 recruitment sites following patient travel coordinator for their clinical trials in 11 the patients. Of course we needed to pay attention countries, as a Duchenne mom. to GDPR as well. We have to say, “How can we do this making sure that patient data is secure, and not I had experience with my own son being in trials and felt compromise trial integrity?” that return of clinical trial data on my son was something that I would greatly appreciate, but was lacking. We had I should emphasize that our process was centralized, conversations with non-profit foundations and all of them and did not return results through the individual encouraged us to develop an approach to return clinical clinical sites following the patients. The decision trial results to participating families. was made to simplify the process, using a single WIRB ethics review that covered all countries, As I recall, Laurie Conklin, a pediatric gastroenterologist rather than individual site-specific ethics approval. at ReveraGen, saw a NIH request for applications for This reduced costs associated with designing and research, and she came to me and asked, managing the return of results program. Plus we felt “Could we do this return of results? Is this possible to do that the sites were already burdened. However, our under this NIH grant?” The NIH grant was then funded, protocol includes surveys of clinical site physicians and that got us off to the races to develop a centralized so we can obtain their feedback and preferences on mechanism for return of clinical trial results to families. mechanisms of return of results as well.

I think we just had a sense, and there has been a lot of Throughout the process, there was a lot of reaching talk in the industry about, “Can this be done?” Because out to patient groups to get some feedback and we’re a relatively small company with a relatively small perspective. The key to our centralized consent and trial, it lent itself to trying to see what we could figure return process is an internal firewall within ReveraGen. out, if we could make this work and, and lay a blueprint Parents contact me directly if they wish to have results for other companies to follow suit. We did that in the returned, and only I have identifying information on hopes that we will be the first of many. the family, no one else at ReveraGen (Sponsor). We started to build around that firewall. It was perhaps a unique situation; I already had a lot of contact with the patients, because I was arranging their travel and stay for clinical visits. Participants really want this data. It’s our obligation to find As for the methods of data return to families, we opted for an encrypted memory stick that was sent to the a way to give it to them after family home. We decided that building an on-line, they have provided their time secure patient portal was outside the scope of our initial effort, but we are planning on this in future trials. We and effort. decided we would load data onto an encrypted USB memory stick, and mail it to them.

Patients as Partners Newsletter Summer 2021 Issue 6 We did have to, of course, obtain informed consent Also, the clinical and research infrastructure at each from each family. Once a family requested data, I would clinical trial site can support the return of results. transfer only the subject number to Dr Conklin, and she Including answering any questions the family may would prepare all the clinical outcomes and laboratory have. Disadvantages are the costs associated with this data for that participant. This included both individual decentralized process, and the burden on clinical trial and aggregate data, so the family could see how their site staff and resources. As return of data must be done son did relative to others in the trial. I would then send after data lock and unblinding of the trial results, this this data directly to the family. may extend the contracts with sites for additional years, adding to cost and burden.

What were the provisions created to help patients In the centralized, Sponsor-driven process we are using, understand their data? costs and complexity are reduced considerably although we have only returned results on 23 families to date. The Making every effort to ensure parents understood the family initiates the request for data return. That said, our data was a top priority of our project. We queried the foundation social media approach to outreach to families parents in a pre-return survey what types of data they is imperfect and likely missed some families. wanted returned and in what format. As we expected they wanted as much information as possible and in all Another growing desire of families is to have the data formats. And so, we presented the data in a few different in a format that is transportable to others of the family’s formats: tabular, graph, etc. We tried to present it in ways choosing? Our approach of returning data via a memory that would be clear to anybody. We are also conducting stick in the mail was effective, but not transportable. post return surveys to understand parents’ satisfaction with the process and data returned. Future efforts will focus on a secure web-based interface for return of trial participant data that is also We also prepared a separate document that I sent interoperable and transportable. We plan to work with called “To Learn More.” This document went through the stake-holder foundations on this next-generation return different types of data returned, and worked to teach of results pipeline. This may also lead to easier scale-up the parents what that data means, how clinical visits to larger clinical trials. and care are different from results from a research study, and that what we are trying to capture is how the drug If we could have built return of results into the clinical works. It clearly explains the differences. ReveraGen has trial protocol, that would have solved some of our been quick to publish trial results, so we also provided outreach problems, and made it a little bit easier to aggregate trial data and links to the publications from the reach the patients through the sites. That said, there VBP15-002/003 studies that were published. are drawbacks to doing the return through the sites. If you contracted with the sites to do this, this could In the data itself, we decided that we were going to be an ongoing, longer term contract with the sites return some exploratory biomarker data. Before the after the trial has concluded. That could be costly and return of results, Dr Conklin created educational materials burdensome to the site, with an ongoing requirement for including graphs and text explaining what each biomarker site staff that has to deal with this. does, explaining normal ranges and also explaining whether or not any specific tests are used in clinical care. We anticipate that our pre- and post-surveys of parents and site physicians will teach us about their preferences For biomarker outcomes and laboratory test results, our and guide us in our second generation return of results educational materials were quite explicit in stating, “This that can achieve a balance between desires of families is not something your doctor orders,” or “This is,” so and cost/benefit/time. We hope that our ‘lessons that they would understand the distinction of the clinical learned’ will be important going forward for other relevance of data returned. Also, during the informed companies to consider, including in trial design. consent process, I emphasize my availability to answer any questions they may have. We try to make sure that if there’s any question, they have a resource to go to What would be your takeaways from your and ask. I also told them that they may see something experience that you can impart with our audience? that may be troublesome to them; “You may look at the graph, and you see your son may not be as fast as all the I would encourage companies to really try to do this. other children.” I talked to them about that. And it’s easy Sponsors should consider writing the return of results for me to talk to them about that, being that I have a son into clinical trial protocols. I think that would make it a with the same condition. lot easier to think through how it can be implemented at that point. It gives them time, because you’re not going to implement it until the end of the trial. What would be your recommendations for a study looking to do individual patient data returns? I think it’s just part of the evolution of a patient-focused drug development. We’re trying to learn about this on First, participants in clinical trials have a right to obtain a small scale, but I think our lessons are going to be their data. This is something that all clinical trials applicable. It can be done safely and securely. I really need to move towards. Second, there are different want to emphasize that all indications are that the approaches to data return, and there are pros and participants really want this data. It’s our obligation to cons of each. Data return can be included in the initial find a way to give it to them after they have provided clinical trial protocols. Advantages of this are that their time and effort. patients and families can opt in or opt out with the initial informed consent process for the trial.

Patients as Partners Newsletter Summer 2021 Issue 7 Overcoming Clinical Trial Participation Barriers

Scott Gray is the CEO of Clincierge. In 2013, a client told Scott about the logistics challenges that make it difficult for patients to participate in clinical trials, and the Clincierge concept was born.

Clincierge is the leading provider of patient support services for clinical trials and manages participant logistics for trials around the globe.

What are some of the other hurdles? What are the challenges patients face that prevent For many patients, it’s the cost of being out of work them from participating in clinical research? or paying for food and necessities in a location that Once the research team has identified the patient has a higher cost of living. We try to meet the needs population for the trial, they enlist Clincierge to work of each individual, and in many cases, we’ve been with trial participants to help them navigate logistics able to arrange for upfront meals per diems or wage related to their site visits. Many of the patients we reimbursement to offset financial burdens. support have a rare disease, which means eligible patients are few-and-far-between and often have to Providing these types or services and accommodations travel great distances to get to the clinical sites. aids in both patient recruitment and retention. This shortens the timeline of the trial and means that fewer Many rare diseases are genetic, and patients are patients drop out – they don’t have any reason not to identified in utero or just after birth. In the case of participate when they stand to benefit from the trial and pediatric patients, sometimes an entire family needs to Clincierge makes it easy for them to do so. On the other be relocated from one country to another for a duration hand, finding new patients has high upfront costs and of time, longer than a typical visitor’s visa would extends the life cycle of the trial itself, delaying the time permit. We work through all the hurdles associated it takes to get new drugs and therapies to market. with medical visas, relieving the family and the research organization of those additional stressors. From your experience, what are small things that This can include coordinating local insurance really matter in terms of the patient experience? coverage, arranging prepaid housing and flights, and I would say, first of all, remember that patients are ensuring access to reliable ground transportation people. They’re not a number. They’re not subjects and other conveniences so they feel comfortable or a cluster of symptoms. They’re people. While in their new surroundings. If they don’t speak the clinical research requires anonymity, Clincierge has local language, we also arrange interpretation and the ability – the privilege – of treating trial participants translation services. like extended family. So when we relocate a family from one country to another, we don’t just rent an Our business model is unique in that we assign local apartment for them. We ask, “When you arrive, what patient coordinators to each patient. These coordinators do you need?” We’ll have things delivered to the are in the same time zone and speak the same apartment, such as fresh groceries or diapers or language as the patient, and in most cases, the same formula for families with infants. It’s these small things coordinator works with a patient and/or their caregivers that make a huge impact on patients and their families for the duration of their participation in the trial. and show them the people involved in the study care about them as people, not just numbers. Patients often develop strong bonds with their coordinators, looking to them not only as a resource for travel and accommodations, but also a To recruit and retain more diverse participants, source of support and reassurance amidst difficult circumstances. The coordinator also liaises with the what do you see as the bigger hurdles in terms site, communicating the patients’ travel plans and of participation logistics? How can we make it staying in close touch should those plans change at easier for people to participate? the last minute. Increasing diversity in clinical trials requires a combined effort. From the researcher’s perspective, that means investing in outreach and education for diverse populations. There needs to be a proactive outreach campaign from the study’s sponsors, saying “We want you! We want people just like you in our trial!”

Patients as Partners Newsletter Summer 2021 Issue 8 Long before the pandemic, I was at an industry What do you see coming down the line in regards conference. After a pharma speaker ended their to how clinical research is done and how you thought, an FDA official asked “You’ve only tested your drug on 60-year-old bald white men; why don’t we might respond to that? approve your drug for 60-year-old bald white men and We’ll continue to offer and expand high-touch patient see if you’re able to recover your investment?” The FDA support services – the need will never go away. Many has not been shy about the need for broadening the of our clients have also asked us to manage less diversity of patients in clinical trials, so that we know complex support for all of their patients, even those the true efficacy – and the true risks – of what we’re who don’t need concierge-level support, for things bringing to market, across diverse patient populations. like short-distance ground transport or simple financial reimbursement. We are currently building a proprietary software platform that will allow us to efficiently support How has the pandemic impacted the hurdles that the full spectrum of patient needs. people are coming into contact with? We intentionally set a slow pace building out this The pandemic has forced us to be even more creative! technology to ensure we had enough experience We had to pivot and quickly come up with unique to truly, thoroughly understand the different ways solutions to support patients, keeping them safe while companies manage their trials. Our solution was ensuring they made it to their study visits. For example, designed to reflect our experience managing 180+ trials we had several immuno-suppressed oncology patients for 50 customers in 40 countries around the world. It and the only safe means of travel was by chartered will be flexible and scalable, so we can use it to manage jet. In another instance, we helped a single mother in the workflow of any trial. In this way, we’ll be able to Argentina bring her child to New York City at the height exponentially increase the volume of what we’re able to of the pandemic, and arranged a safe place for them to do for the pharma industry. quarantine for two weeks prior to the start of the child’s participation in the trial. What would you like to see pharma doing more of, Those were extraordinary examples. More commonly, in terms of making it known that it is possible for because rail and air travel within Europe was restricted, we arranged long-haul car travel, planning frequent patients to participate in clinical research? rest stops and overnight hotel stays for both the driver I’d like to see more of what I’ve seen over the past few and patient along the route from home to the research years, which is a mind shift in providing better support site and similar logistics for their return. In other cases, for patients. I see it a lot from biotech and gene therapy we were able to coordinate specimen collection and startups, especially those with VC funding. They are assessments at the patient’s home. more open-minded with their thinking and willing to adopt a new way of doing things. I would like to see One of the adjustments that we made during COVID more of the “big ships” start to turn and recognize that is that we were no longer always taking the patient all making things easier for the patient is not just more the way to a distant research site. Researchers were humane, it is also the right thing to do for their business. networking with their colleagues to find someone who could do basic assessments locally. Although the To make that shift, it will have to be a collaborative pandemic was a horrific experience for all of us, I do effort. We will continue to share stories of successful believe it pushed clinical trial professionals to make things trials that have invested in patient support services, but simpler and easier for patients. People are thinking more it may also take pressure from the FDA to impress some creatively about how research can be done differently, so urgency on the need to expand patient diversity and the patient faces less barriers to participation. reduce barriers to participation.

Are there any “common sense” changes that you What would be your final message to the clinical are surprised that clinical research still does, or research professional audience? something that patients commonly describe to you More than anything else, I would encourage as an issue? professionals who work in clinical trials to remember The most obvious change is not necessarily with trial who is on the other end. Participating in a clinical and protocol design, or even pharma’s mindset on what trial isn’t just a favor or paid vacation. In many cases, they should be providing in terms of patient support. it’s a patient’s only hope for life-saving, or at least We’ve learned the most surprising struggle is the impact life-extending, treatment, and they’re making heart- of varying regulations across the globe. What can be wrenching sacrifices to keep themselves or their family done in one country can’t be done in another. If we members alive. Let’s eliminate as many burdens as can’t gain access to a patient population, because possible so that patients can get the help they need and the IRB won’t approve it or we can’t provide a certain ensure we get effective treatments to market as quickly service to patients in a specific country, it thwarts our as possible. efforts to promote participant diversity. This seems to be a blind spot for the industry.

For more information, visit clincierge.com

Patients as Partners Newsletter Summer 2021 Issue 9 Hearing from Patients to Develop a Portal for Individual Result Return Kristin Blouch, Director, Molecular Disease Characterization & Strategy at GSK, leads a novel Molecular Disease Characterization program that provides new opportunities to benefit cancer research and patient empowerment. Within the program, she and her team are leading the development of a participant hub (Gather Share Know) focused on patient education and result return. Patients are able to see a timeline. What some of us might have taken for granted is that the physician will What was the origin behind creating the patient make patients aware of each of the study visits, and hub, “Gather Share Know,” for this GSK study? what they can expect. Under the hub, what we’ve done is provide that information in a timeline of the study visits The thought was threefold. First, society, in and of itself, with a picture format to show what might be involved at is wanting more information. Technology is advancing to each study visit. make access to that information easier. Second, patients themselves are wanting to be more involved, not just We give back data in real time. It was really important on an educational perspective, but also having access for myself and for our team, to have the data going back to their data and knowledge of what is generated from in real time. It’s not the model of “at the end of a study,” their samples, so that they can actively participate in which unfortunately is a time point when some patients discussions as it relates to their disease, their treatment can’t use that data. and their journey. Gather Share Know also displays clinical trials that may The third piece is the clinical trial we have Gather be potential options for patients. For those studies, we’ve Share Know coupled with called the Molecular Disease developed a plain language summary that hopefully is Characterization Initiative (MDCI) Study. It is a clinical trial easier to understand about what a study might involve, that we’ve designed, which takes a different approach to with the specific ClinicalTrials.gov link, as well. the current clinical trial screening model where instead of screening a patient for a single trial at a time, we use the There are also questionnaires available, the purpose of data collected to return all potential options. which is to get direct patient feedback: “Are we on the right path with data return? Did you feel better informed Since the MDCI study is a non-therapeutic trial that about your disease and about your treatment options analyzes patient tumor tissue and blood samples to screen available to you?” etc. for potential clinical trials, we can’t guarantee a patient will receive treatment. So we wanted to find a meaningful way that we could give back to patients and that is how the How do you present the data to make sure that it’s idea came about in building a participant hub, which really understandable? addressed where society is today, patient inclusion, and the needs for the clinical study itself. Our team didn’t want to give back the scientific format for the results. So instead, our format is the biomarker name, an icon that the patient can hover over that gives What does a patient get out of the hub? a plain language summary about that biomarker – why it might be important in their disease or their treatment The patient has the option if they want to log on to the decision – and then “positive” or “negative,” for the Gather Share Know hub or not. Once they are logged in, biomarker results. For the vitals, we have numerical they are able to see links to credible sources. We’ve heard values, and then we have a scale so they can see a the feedback: we all Google everything. We didn’t want to visual related to that vital result as well. leave our patients to search on their own; we wanted to provide credible sources specific to their disease. They’ll see links to patient advocacy groups, as well. I do hope that by providing They will also have links and resources available for this platform to our patients, their caregivers or their family members. We completely appreciate that when someone is diagnosed with that it will help in not just the cancer, it’s not something that they fight alone; they have their family members and caretakers alongside retention, but also the patient them through their journey. education, awareness and transparency. Patients as Partners Newsletter Summer 2021 Issue 10 You’ve mentioned before working with an Oncology When we did decide on a partner, we asked them Patient Council. What was their insight and input? to think outside of the box. They have met those expectations and have custom-developed a number of We have had regular touchbases and meetings with aspects under Gather Share Know. our Oncology Patient Council, which is an important group of patients with cancer who are knowledgeable For others who may be planning, the first step would about research and skilled in supporting other patients. be clearly understanding and outlining what you want We partner with the council on various initiatives and to have available, and then seeing from that list, what it’s been a two year journey together for this effort. I will discussions you need to have, who you need to be never be able to articulate just how much the team has partnering with to develop. Do expect some pushback appreciated those open and honest conversations and and do expect for it to take some time in building. When their feedback. We have partnered on the study design, you get to a good point, test all of those capabilities out on the ICF (informed consent form), and on the portal. and get feedback every step of the way.

The feedback on the study design was really, at first, that initial surprise that this hadn’t always been the Did the oncology patient council or other patient model, that their samples weren’t being used and advocacy groups you consulted verbalize what considered against multiple trials at a time. For the having their data would mean? ICF: when we followed the original model, our study was three visits and our ICF was 26 pages long. It was It presents a number of different opportunities. Just nothing but words. I myself could not get through those having the resources available; knowing that patient 26 pages and fully appreciate what was being relayed to advocacy groups exist and how to even connect with me. So I could not expect a patient to do it. them; the visuals and what to expect at each of the study visits. We worked with the council and with our team, as well as our legal team, and raised We’re in a time where technology has advanced and we the question, “Can we start to develop a more patient- have easy access to information and we want access friendly ICF?” Our teams have been really supportive in to information. The feedback that we’ve gotten from all aspects of this study. We got the greenlight to put the council and from others is, “I especially want that together a model. And in that, we provide an overview information if it’s able to help me in understanding my of what’s involved in the study and what a patient might disease and helping me understand why I’m receiving expect, in picture format on the first page. the treatment that I am.” I do hope that by providing this platform to our patients, that it will help in not just We treated the ICF itself as our first engagement with the retention, but also the patient education, awareness a patient. It’s our first opportunity to introduce some and transparency in providing the data back in real time of the educational background. For example, under when patients can use it. the biomarker section, because we give data back on the hub, this was the first opportunity we took to explain what is DNA, what is RNA, what is protein, There are a lot of assumed hurdles in data return. how are they related, in a picture format. For the hub, How can someone overcome that mentality? the patient council was giving feedback when it was just a screenshot, and then when it was in a document First and foremost, when you hear of a potential hurdle, format, and later an environment that they could log look at the actual data. One of the first push backs that into and test. They’ve provided feedback all throughout we heard was that our clinical trial patients would be too development. old to use a resource like this (a website). Our team then looked at the average age of our clinical trial patients As it relates to the data itself, we got an absolute across several of our oncology studies. And since we resounding “Yes,” that they would want to see what have shown the actual data, we’ve not heard that same their results were. It was interesting when we got to the pushback. So make sure you look at the data: is it vitals, and that data return, I would say it was 50/50 in actually an issue? By determining an actual hurdle vs. the council. Some members wanted data specific to assumed, you’ll be able to then focus on an effort that their disease only under the hub and others wanted to may need more attention than others. know what their vital data was. We took that feedback and then had the data in two different tabs and left the option open that if a patient wanted to see their vital What would be your final thoughts or advice for information, it’s there; if not, they didn’t need to. others who want to accomplish patient data return? I think we’re moving into an exciting space where, What would you recommend in terms of the when we consult with experts, have events such as an ad board meeting, and we’re talking with different planning and development timeline? physicians on study design, that we should absolutely Ours has taken two years, and the hub was a Day 1 be including patients themselves as experts. We’ve discussion. So it really has taken that amount of time. received invaluable feedback on study design, different We had a really good idea up front of what we wanted documents, such as the ICF, and the technologies that to provide; the next step was determining who we could we have available today. If you have the opportunity to best partner with to make sure that we checked all of receive feedback from patients themselves, you should the boxes and that what we had envisioned could be absolutely be taking, or creating, that opportunity. My made possible. hope is that Gather Share Know serves as an example that data return can be done.

Patients as Partners Newsletter Summer 2021 Issue 11 Lessons Learned from Creating a Patient Portal for Result Return

Kafayat Babajide is Associate Director, Patient Portals, at Janssen Research & Development. LLC (Janssen), managing tech platforms that leverage patient insights intended to transform the clinical trials process. Right now, she is working on a digital platform that serves as a companion to the clinical trial process, called My Trial CommunityTM (MTC). What preferences for returns were identified from patient feedback? What was the original genesis and drive for creating a patient portal? Through the insights that we developed ourselves, Janssen is deeply committed to “Better Trials. Made and some existing data, we knew that many patients Together,” which is all about leveraging insights to were very interested in getting their data back and optimize the clinical trial experience. We believe the best seeing summary results. The Center for Information clinical trials are the ones where, together, we generate and Study on Clinical Research Participation (CISCRP) robust and groundbreaking data and at the same time cites that, an overwhelming majority would like to prioritize patient safety, privacy, and convenience. We see this information, but less than a quarter do. As have mined available insights across the pharmaceutical mentioned previously, data return is quite valuable to the industry and conducted an extensive body of patient patient. So, we want to provide that information to the voice work ourselves. We learned, based on listening participant. We also know quite a few patients have very to the patient perspective, about the huge opportunity low awareness and understanding of what a clinical trial to address unmet participant needs by providing a is, if they were not previously trial-exposed. connected experience in clinical trials. So, there is a need for basic primers and education My Trial CommunityTM is designed to increase around clinical trials. Even for some trial-exposed engagement, transparency, understanding, awareness patients, things come as a surprise. They’re unclear on and education for the patient and participant, and what to expect in the trial process and want to be better demonstrates our deep commitment to clinical trial prepared and informed. We embed the patient voice participants and investing in cutting edge capabilities and into everything we do through MTC to help address services for patients participating in clinical trials. such needs.

Now, we’re getting ready to officially launch our first full-fledged trial portal with a couple of trials. We’ve done beta testing with patients to get a sense of what things we might need to improve upon, and what things they Returning personal trial data consider to be most valuable. and study results is a key value driver for patients. What can patients expect from the portal, in terms People are decision-makers in of materials and results? their health journey. Patients can expect clinical trial education and support before they become participants. Once they make an informed decision to participate, they can register for What was that process of creating patient-literate MTC to conveniently access details about their trial, and patient-accessible content, from the plain information on trial progress and visit prep, and a subset of labs and vitals. And, where relevant, patients can language summaries to the types of data that you get back a plain language summary that summarizes were returning? the results of the trial. Based on our insights, returning personal trial data and study results is a key value driver We took a highly cross-functional approach to mapping for patients. People are decision-makers in their health this out. We worked closely with our Patient Insights journey, and Janssen leverages insights to co-create Team and we conducted stakeholder interviews with solutions that provide the clinical trial transparency and some of the therapeutic area leads. We also worked trust that patients deserve. very closely with Health Care Compliance to look at what was needed and to find compliant ways to make that happen.

Patients as Partners Newsletter Summer 2021 Issue 12 First, we leveraged the insights we had, and we came up In some cases, you have the capabilities in-house and with a hypothesis of what we thought would be the key other cases, you’ll need to outsource. features and pieces of content that patients would need. Creating a sound feedback loop and implementing From that hypothesis, we then tested it with throughout is critical. Especially with anything digital, it’s stakeholders: sites, patients, and colleagues who help going to be highly iterative. You want to make sure that with operations to determine how it married up to our in the design and development, you are consistently hypothesis. We then looked at that delta and developed getting feedback from the right stakeholders so that a new prototype for the content. you’re ultimately developing something truly valuable to the patient. And we share the results with stakeholders to see if there was a good level of satisfaction with the findings, Partner effectively to find a solution that is workable and and if the information met the mark for what they introduces minimal friction. There’s a way to brainstorm valued. We’re planning on iterating on this process on how to get to a better solution that drives a better to further evolve what type of content we include on experience for the patient. the site. It could be the case that some content gets replaced, that certain content will remain evergreen, but And lastly, work collaboratively to develop a clear and we’ll build from there. It really will hinge on maintaining effective change management plan. Anytime you’re this sound and strong feedback loop with the introducing something new to an organization, to a appropriate stakeholders. patient population or stakeholder population, you always have to lead with empathy around how that’s going to impact people and the way that they work What was the process of collaborating with the and think. It’s important to communicate what’s being sites on delineating who would troubleshoot developed and why, and the cross-functional impact any access issues, or provide follow-up and support being provided. conversations, etc? What we were trying to do was meet patients’ needs What lessons can you share? while being very mindful and empathetic around the The first, as we say at Janssen is, “Ask. Listen. Act.” existing workload a site is already doing. We were trying Listen to your stakeholder’s perspective first. to make their jobs easier. So, we did partner with site staff to understand their journey and key pain points. Another is balancing the short-term needs with the long-term vision. Knowing our resources and what To ease the burden, we created a help desk for MTC we envision, there’s only so much we can get done in that patients can use to troubleshoot technical and the short term. It takes prioritization, alignment, and operational site issues. This prevents unnecessary effective planning to strike this balance. Start with your outreach to the site. minimally viable product but prepare an infrastructure that facilitates long-term goals. Don’t lose sight of that. Where we ask the sites for support is around helping to introduce MTC as an optional feature that patients Another lesson is that this entire process – and can participate in. We conduct site training and innovation in general – requires a lot of trust building supply collateral materials and talking points to aid in and leading from a place of empathy. Remember to be the onboarding. Sites also ensure clinically relevant human-centered. laboratory and other test data is discussed with patients before a site approves any data to be transferred to the Finally, communicate clearly and often. Emphasize patient portal. purpose, plans, roadmaps, timelines, so that the involved parties can manage expectations and be the best champions. Can you provide any development advice? How far back should the planning begin? Who are the key stakeholders to involve? Anytime you’re introducing It’ll depend on the build, actual team structure and organizational structure. Every company will have their something new to an own requirements and processes to follow, but I would organization, to a patient generally start as early as you can, because the lead time to launch is lengthy. population or stakeholder

Start with a clear, informed vision and strategy. population, you always have to “Informed” means stakeholder-informed, patient- lead with empathy around how informed. Consider cross-functional members that should be involved and partner early—especially for that’s going to impact people leadership alignment. For us, it was critical that we and the way that they work worked with our Janssen Clinical Innovation (JCI) team, compliance, clinical operations, IT and patient insights and think. in the beginning. Understand requirements and current business capabilities.

Patients as Partners Newsletter Summer 2021 Issue 13 Unraveling the Challenges of Genetic Data Return to Give Patients Agency Sandra Prucka is Assistant Professor of Clinical Medical & Molecular Genetics at Indiana University, leading the vision for providing genetic counseling services. Prior to that, Ms Prucka spent 8 years at Eli Lilly and Company working primarily in the area of Tailored Therapeutics. She is a current member of the MRCT Return of Individual Results Working Group focused on updating the 2017 guidance. For most data points, it must be clear what results can be returned versus not. But when dealing with Can you describe your involvement in the MRCT genetic information, how much thornier does that Return of Individual Data project and what that make the return of results? project hopes to accomplish? I was involved in the Industry Pharmacogenomics I have been involved in the return of research results Working Group, and we developed a publication for a very long time; it was fairly early in my career that because we felt like it was so essential to highlight some I took a role with Eli Lilly and Company, in their Tailor of the challenges in this area for that exact reason. Therapeutics group. Genetics and genomic data is probably the most complex case of data return. When I was there, I was really centered around tailoring medicines to an individual. And to do that, and to dig In a research context, you’re often talking about really into our genetics and other biomarkers, we’re looking large datasets that are being generated. And so a for differences in our body’s makeup, that may explain natural place for everyone’s mind to go is if you’re why one person responds differently than another to the generating that data, there has to be clinically relevant same medicine. information there. The complexity comes from the fact that those answers don’t pop out at us; we have to As I was starting to look at that data and work with seek them in the data. those researchers, I was bringing in my clinical mindset having just come out of genetic and genomic care. I Often when you’re doing this type of research, those thought, “What happens if in the course of doing this, are just not the questions you’re trying to answer. we uncover something that has clinical relevance? You’re trying to make new discoveries and find new Do we have a plan for that?” I found my way to our associations between genetic markers and response bioethics department at Lilly; I actually ended up to study interventions, as well as to understand the being the first person to graduate from their Bioethics genetics of the conditions that are the focus of the Leadership Academy (BELA). clinical trial. To do this we have to often analyze the data in aggregate to see if anything of significance for these The focus of the work I did in that training was to develop associations stands out. an internal policy on this topic. Eli Lilly and Company was part of the MRCT, and so, I got connected more For me to give you something that’s clinically relevant, it to that group and learned a little bit about what their might require that I take a different step, that I say “Even center is about. They had identified an interest in this though this has nothing to do with the research, let me area as well, mainly because there’s a real gap or a take the same data set and now dig into it to answer disconnect between what we were hearing from research the questions that you have.” This requires a different participants – which was that they had an interest in level of rigor and a different format appropriate for receiving this data – and the fact that there really wasn’t clinical interpretation. In addition, the research may have a lot of information out there for researchers to figure out to be performed in a different type of laboratory that is how to meet those needs. accredited for doing clinical genetic testing.

MRCT produced a guidance document and a toolkit There’s a paper that was put out by the American that was rolled out in 2017 that serves as a framework College of Medical Genetics that gave advice: if you’re for how to accomplish return of individual results to in a clinical setting, and you’re generating these large participants. And now, we’re actually in the midst of data sets from accredited laboratories, we know this reviewing everything. The group went back and said to information is there. There should be an opportunity to all of our stakeholders who use this guidance and toolkit, give people the option of receiving what we would call a “Where did we meet the mark? Where are things that are “secondary finding”. still gaps or challenges?”

Patients as Partners Newsletter Summer 2021 Issue 14 The things that they felt were most appropriate to Who are the key stakeholders when returning share were changes in genes we know are associated genetic information in the most impactful way? with conditions that there’s some action you can take, that would give a positive effect to the trajectory of I do think it’s really helpful to have a genetic counselor your medical care. If I know for example that you have or genetic professional involved. I also know that we are change in a BRCA gene that is associated with an not such a robust group that we can be in all places at increased risk for cancer, if you had that information, all times. Health literacy experts are also key. Even as a you can do things to try to decrease your chances to genetic counselor trained in communicating information develop cancer or to catch the earlier stages that were in a digestible way, when I looked at creating this kind more treatable, and the outcomes are better. of content for informed consent, I did work with a health literacy expert. That expert could say to me, “What if And so, they came up with a number of genes that we use this picture to explain that concept? Or what if fit that classification. Currently the ACMG does not we bulleted this information and made it a little bit less recommend returning these findings in a research dense? What if we gave somebody a glossary?” setting but leaves this up to the research team and to decide when and if that is feasible Instructional Design is a great example: to have a field and appropriate. that is really focused on “What is it you’re trying to convey? How can we convey it? And then how can we test whether or not we actually got this right?” How do you return genetic data in a way that is sensitive, accessible and impactful? Involving the research participants themselves as you’re developing these materials is absolutely essential. In our It is challenging, and in the clinical trial context, it’s even clinic last year, we had a focus group where we set out more challenging. When I worked in tailor therapeutics, different letter formats. And by simply gathering a group I often thought that someone who is coming to a clinical of people who are the recipients of that information and trial, it’s possible that the very last thing on their mind asking them those questions, we were able to use their would be to provide a sample for genetic research. feedback to keep refining. So when somebody is asking for a sample for genetic research, the response is, “Where is this coming from?” If I’m trying to explain something to you, and you say, And as a genetic counselor, we have been equipped with “Here’s what I don’t understand,” the next time I explain that skill set, to be able to take something that is very it, I’m going to incorporate that feedback. difficult to understand, and then break it into pieces that make it more digestible, and help people really understand the point in doing this: things that they need to consider With your experience in genetic data return and when they’re deciding whether or not they actually want this information. also your experience at a pharma company, where would you like to see patient data return in the next And then if people do decide to go forward, we work with couple of years? individuals to put this in the context of their health history and their family history. One of the biggest concerns I have Ultimately with pharmaceutical companies, I do feel like sometimes is not the information we’re sharing if we find they’re starting to recognize that this is something that’s something, but it’s more how people could interpret getting moving forward. Where we are now compared to when a result that says we didn’t find anything: that people will I first started working, the pharmaceutical industry has think that means they are not at risk for anything. made leaps in both aggregate and individual results return. The projects that are ongoing are just evidence That’s where, to answer the question, contextualizing of that. information for someone is key. And that’s why it’s important to approach this not in isolation, but in working With anything you’re learning to do, the hardest thing is with an investigator in your trial, who may need to connect taking the first step. What groups like MRCT are trying you with a genetic counselor. Or, to connect with your care to do is to provide enough examples and tools and providers who, again, may involve someone like a genetic context that enable that first step. The more companies counselor or geneticist, to break it down so it’s more that can share the output of that first step with others, understandable, but then also put it within a larger context. the better off we’re all going to be.

BRCA is a great example: if we look at the BRCA 1 and A big driver for this change in data return has been the 2 genes they do not explain all hereditary breast cancers. persistent advocacy of the participants themselves. There are a number of other genes which, when altered, It’s been fantastic to see that voice continuously have been found to be associated with an increased risk represented and coming to the table, because none of for breast cancer and still more that we haven’t found this research would be accomplished if it weren’t for the yet. So if your testing comes back negative (no change in participants that make it. this gene has been found), but you tell me you’ve got a strong history in your family, and we’re seeing things that And so the least that we can do is figure out what type are atypical from general population risk, we may still want of information that we could provide to them to be able you to do different types of screening or consider other to help them have a more positive trajectory with their options. Making sure that people are getting that context is overall health. so essential in being sure that information is being reported in the right way.

Patients as Partners Newsletter Summer 2021 Issue 15 A Kaiser Permanente Researcher’s Perspective: The Importance of a Qualitative Design Approach in Patient Trial Experience Leah Tuzzio, MPH, is Senior Research Associate, at Kaiser Permanente Washington Health Research Institute. She is a qualitative researcher who uses human-centered design principles to improve people’s care experience. You were a part of a Rethinking Clinical Trials article about returning individual results. What was What is the research you’re conducting and the your involvement in that? work you’re leading in patient experience? The National Institutes of Health designed the NIH I am a collaborative scientist at Kaiser Permanente. And Collaboratory for Healthcare Systems Research. It’s the core of my work is really focused on stakeholder focused on embedded pragmatic clinical trials. I am one engagement: these stakeholders range from patients, of the members of the Healthcare Systems Interactions clinicians, administrators, you name it. My work really Core that works with all of the demonstration projects. focuses on applying human-centered design methods This initiative, this collaboratory, has been around since and qualitative research, and using participatory 2012. That’s how I became part of it. research approaches. It’s created something called the Living Textbook. So when you talk about rethinking clinical trials, that’s a living textbook that’s open to anybody who wants to What have you come across in your research, in learn about embedded pragmatic clinical trials. regards to ways of changing or evolving research and care that could impact patient experience? And what role does data return play in that work? A lot of researchers asked me that same question. We’re all trying to figure out how to do it better. And I consider patients as stakeholders in all of the research one thing that I’ve learned over my 20 years of doing we do. And so one of the aims of the NIH Collaboratory this kind of research, is that it’s really important to elicit is to engage patients who contribute data to these real people’s perspectives. world clinical trials. And so it’s their right to receive the data that they have contributed, and also to better The people that you’re trying to serve, you’re trying to understand what the potential outcomes are, whether help and you’re trying to resolve their problems. So you they’re positive or negative or unknown. must understand what their values are, what’s most important to them about their health and well-being and And in the NIH Collaboratory Living Textbook article what they need to be healthy, and what they prefer and that you asked me about we reference the National how they prefer to receive information and learn. Academy of Medicine’s consensus report on returning data to patients. This was written in about 2018 – I was Because if you don’t really do that, if you just do that on not part of it – but they really described how, why and your own, it’s not going to provide you that rich research how to return results and data to patients. and patient experience as much as you intended to do. And another thing that I recommend to researchers is being iterative and continually learning. Things How do you engage with patients as stakeholders change constantly, as we’ve just experienced with the to determine their value sets, preferences, etc? pandemic: life, circumstances, context, resources. You have to be open to continuing to learn. It’s so delightful that patients really want me to ask them about what they value and what they prefer. And so I don’t find it a difficult task. But since my research is mostly formative, or generative, it’s about designing a We used to do a lot of research research question or designing a healthcare experience to study. I get the chance to learn from all these people. about people. Now we’re doing But one of the techniques that we use in my research more research for people. is co-design research, where we are asking questions together and learning together.

Patients as Partners Newsletter Summer 2021 Issue 16 And other ways that we do it in the health services Have there been any surprises in how people research that I’m part of, is that we often have lived interpret that information? experience panels: people who are really living in the community with that condition, or those conditions I don’t think it’s a surprise, necessarily. I think it’s an that we’re trying to treat, and study. And it’s quite expectation that people who work in academia, and eye opening to have patient advisory boards, or lived researchers speak a different language because we experience panels, to help inform research. were schooled in a different way. But if we think about talking to our grandmother, for example, about what we do and what we studied, then everybody can How have you seen the role of patients as understand it. There are a lot of misunderstandings stakeholders evolve? and confusion. And some people just don’t feel comfortable even asking questions. It’s like going to We used to do a lot of research about people. Now your doctor if they were using only medical terms or we’re doing more research for people. It’s helped that talking a different language to you. You’re not going to a lot of our funding agencies, at least in my world, like get what you need from that relationship if you’re not the Patient-Centered Outcomes Research Institute, the speaking the same language. National Institutes of Health, are requiring researchers to actually genuinely and authentically engage patients Sometimes the researcher is unsure; they don’t know in the research as co-investigators, as partners, how to speak to a lay person. And so I wouldn’t throughout the trial, or throughout the study. It’s necessarily put it on the patient and say they are definitely changed. confused. So we researchers need to learn how to speak to everybody. We also have so much to learn still; how do you do that? It takes a lot more time than you might have budgeted for. But building relationships, as anyone From your perspective of improving patient knows, takes a lot of investment, and time to make experience, what would be your advice or that relationship strong. recommendations for getting started in data return to benefit all stakeholders? The Rethinking Clinical Trials article mentions It’s really important to start early, not just when you data return in both traditional RCTs and pragmatic have the trial results, and to build those relationships trials. Can you expand on that? and understanding from the beginning, so that you’re coming from the same playing field. And at the end I participate in both types: explanatory trials and of the process, member checking: going back to the pragmatic clinical trials. There are benefits to studying people that you have just studied, and working with efficacy and effectiveness in different contexts. I have them to create clear takeaway messages. What is that not personally explored returning data in each type, but elevator speech that you would understand: a very understanding contexts of different groups of people, simple 1-2 sentence message, in plain language, of and also preferences and values, has been important. what the outcome of this was? What was the learning We haven’t really done that for a very long time. And from this study? so we’re still constantly learning how to return results, and navigate the trickiness of “What if the result is not That way, that person can go and share it with their positive and not helpful to that person?” community and their people. It engenders more people who are interested to potentially be part of clinical trials. With recruitment, especially with diverse populations, What would be your thoughts on that trickiness? we’re really bad at that. We’ve historically not been When a result may not be positive? inviting, or we don’t have really strong trust with diverse populations. And so, it’s critical to take the time getting It’s explaining what those results mean, in context, and to know people and building that trust. also explaining upfront expectations of what different scenarios can occur. It’s also using plain language. It’s just how you do it, and how you speak to your different audiences. I work in academia: a lot of our academic Does patient data return help build that trust? journals are now starting to create lay person, everyday I only have anecdotes, but working with people language abstracts. engenders trust. It makes you more humble; you have to be honest and transparent. If we stop being transparent And that does require you to think about, as a researcher: or telling the truth to people, they’re going to see right “How can you explain the outcomes and the findings of through that; and they’re not going to want to work with or the data that was collected?” you. They’re not going to want to participate, and they’re going to tell all their friends not to participate. But if we I don’t work in health literacy exactly. I do work with a create a relationship, it can only improve our recruitment. lot of patients. We’re constantly collaborating on how Going back to those lived experience panels or advisory patients, as partners in our research, interpret the data. groups of people, they can really help across the whole What is it that they are seeing versus the researcher? And continuum of research to figure out, “What is that then figuring out how to disseminate the data, so it is the research question that’s meaningful to them?” Because most meaningful and impactful to the actual person who reaching a clinical endpoint doesn’t necessarily mean that is experiencing the study or the intervention. they’re going to be able to play with their grandchildren. And that might be the thing that’s important to them.

Patients as Partners Newsletter Summer 2021 Issue 17 Watch the Patient Data Return Webinar Series This four-part webinar series, organized by the Patient Data Access Initiative (PDAI) and the Patients as Partners in Clinical Trials conference team, shared best practices and guidelines for providing patients with appropriate access to their clinical trial data.

The series gave learnings from the members’ experiences in piloting data return, in order to support other sponsors who are interested in these topics and to further demonstrate a commitment to patients.

Introduction to Patient Data Return Hassan Kadhim Bristol Myers Squibb

Optimizing the Patient Experience in Data Return Processes David Leventhal Pfizer, Inc.,

Patient Data Return Pilots: Lessons Learned Jennifer Ribeiro Bristol Myers Squibb

How to Get Started in Patient Data Return Megan McBride Janssen

Click Here to Watch! Patients as Partners Newsletter Summer 2021 Issue 18