A SHARED PERSPECTIVE OF PATIENT TECHNOLOGY...

A SHARED PERSPECTIVE OF PATIENT TECHNOLOGY IMPLEMENTATION IN CLINICAL TRIALS

C O - A U T H O R S : U D A Y D E O R E , K A T R I N O N G , J A N I N E R E I S , H A R R Y T U N N E L L , P A U L O C O V I Z Z I , F A R H A N ( C J ) H A M E E D , T Y L E R R E Y N O L D S

C O N T R I B U T O R S : M A T T H E W M O Y E R , A N T H O N Y B R O G N O , P E T E R S C H A E F F E R , M I C H E L L E C R O U T H A M E L

Patient Technology

Copyright © 2020 TransCelerate BioPharma Inc. All Rights Reserved. 2

Table of Contents

Introduction Background Aggregate Data Findings Deep Dive Implementation Experience Findings Conclusions References

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INTRODUCTION Collaboration, transparency, and the sharing of data and experience across the pharmaceutical industry has been recognized by many as a vital step in accelerating the use of new and innovative technologies in clinical trials. Now, more than ever, is the time for sponsor companies to fully embrace these principles in order to bring therapies to patients faster. The United States (US) Food and Drug Administration (FDA) and the European Medicines Agency (EMA) have positively encouraged industry collaboration as recently as 2019, and each agency has publicly committed to additional guidance, workshops, and task forces to stay in line with the innovation curve. Former FDA commissioner Scott Gottlieb is quoted as saying, “Industry and academia also need to invest in and leverage these approaches and develop new incentives that reward collaboration and data sharing across the clinical research enterprise.” (Gottlieb, 2019) Similarly, the EMA has demonstrated early action by establishing the Innovation Task Force, a multidisciplinary group whose objectives include “establishing a discussion platform for early dialogue with applicants, in particular micro, small, and medium-sized enterprises (SMEs), academics and researchers, to proactively identify scientific, legal, and regulatory issues of emerging therapies and technologies”. (European Medicines Agency, 2020) Supporting these principles, a variety of industry groups have formed to foster collaboration in order to help bring effective, efficient, and high-quality new medicines to patients. TransCelerate Biopharma Inc., a non-profit membership organization whose members are pharmaceutical companies, is one industry group perfectly positioned to help carry out the principles of collaboration, transparency, and sharing of data and experience. As part of this mission, TransCelerate founded the Patient Technology Initiative in 2017 with an objective to accelerate the use and implementation of patient technologies in clinical trials. Patient technologies can include, but are not limited to, technologies that collect clinical data (e.g., an electronic patient reported outcome [ePRO] or wearable device), non-clinical data (e.g., patient engagement app), or technologies that do not collect data but facilitate patient participation in a trial (e.g., a telehealth platform).

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BACKGROUND To encourage collaboration and to help facilitate patient-centric information sharing, the Patient Technology Initiative completed a first of its kind exercise in knowledge sharing. Leveraging TransCelerate’s membership, the initiative team developed a survey to collect member company experience implementing patient technology in clinical trials between 2013 and 2018. The goal of this exercise was to create an environment of cross collaboration and learning. This is important because the use of patient technology in clinical trials can benefit patients, as well as sites, pharmaceutical companies, contract research organizations (CROs), and potentially regulatory authorities by delivering better patient experience and real-world data. We strongly believe that sharing information will lead to increased awareness and preparedness regarding the use of patient technologies in clinical trials. The data collection exercise concluded near the end of 2018 and resulted in the gathering of information about 81 different cases of clinical trials in which patient technology was used. The issued survey contained over 50 questions which spanned topics such as trial setting, patient technology use (e.g., purpose, activities the technology impacted, intended use of data from the technology, etc.), and, most importantly, implementation lessons and clinical trial team experience1. Key findings, themes, and learnings from that collection of information are presented in this paper.

1 A third-party consultant organized and distributed the survey, collected the responses, and blinded and, for certain information, aggregated the results before disseminating them to either the companies participating in the survey or TransCelerate’s broader membership. In addition, all results were reported in a vendor-agnostic and product-agnostic manner, meaning no results identified any brand of product or vendor used by any survey participant.

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AGGREGATE DATA FINDINGS The following figures show a visual representation of aggregated trial setting data across the 81 received cases. Specifically, the figures show geographic distribution of technology implementation (Figure 1), percentage of entries by technology category (Figure 2), percentage of entries by trial phase (Figure 3), percentage of entries by trial type (Figure 4) and percentage of entries by therapeutic area (Figure 5).

Figure 1: Heat Map of the Geographic Locations Where Patient Technology was Used in Clinical Trials

One of the more noticeable insights from the data is the global nature of patient technology use in clinical trials. Implementation of patient technology has occurred in at least 55 countries across the world and on all continents except Antarctica (Figure 1). Patient technology was most frequently used in clinical trials conducted in the United States (51% of entries), Germany (36%), Ireland (36%), and the United Kingdom (35%). We believe that the prevalence of global implementation of patient technology will continue to increase as more and more experience is gained and as regulators become more familiar with patient technologies and their use. The most commonly implemented category of patient technology was mobile applications with 53% of entries (Figure 2). This was not a surprising result for the team because mobile applications are a broad category that can include everything from electronic diaries to ePROs to companion applications. The second most common category of patient technology based on the survey results was wearable devices at 33%. Other collected data (on file) show that wearables

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were widely used across the spectrum of drug development from internal feasibility studies to Phase IV trials. Their primary purpose for use, as reported in the survey, was data collection and digital evidence generation, a trend we believe will continue. Bring Your Own Device (BYOD) was represented with 11% of entries. This is a category likely to increase in the future because of the global prevalence of smart phone and tablet usage.

Figure 2: Percentage of Entries by Technology Category2

With respect to clinical trial phase and type, the deployment of patient technology was most common in Phase III clinical trials as reported by 56% of collected cases (Figure 3). The most common trial type was interventional trials with 56% of the collected cases (Figure 4). When considered together, patient technology was used in Phase III interventional trials in 42% of the collected cases, making it the most common combination of trial type and trial phase among the collected data. Within this 42% of collected cases representing use of patient technology in Phase III interventional trials, approximately 29% utilized electronic clinical outcome assessments and the remaining 71% used technology in categories such as mobile applications, wearable devices, implantable technology, and ingestible technology. In addition, 82% of the cases that used patient technology Phase III in interventional trials reported using technology for the purpose of data collection/digital evidence generation. This supports the notion that industry has gained enough experience with certain technologies or technology types that it’s comfortable deploying them in late phase clinical trials. We see this as a building block for using more novel technologies in the future as they move from exploratory or proof of concept settings to use in advanced clinical trials.

2 Totals will add up to greater than 100%. Entries could select more than one patient technology type if the patient

technology fit more than one category.

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Figure 3: Percentage of Entries by Trial Phase in Which Patient Technology was Used

Figure 4: Percentage of Entries by Trial Type in Which Patient Technology was Used

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Regarding therapeutic areas, the collected data shows that patient technology use was predominately in the areas of metabolic and respiratory diseases (Figure 5). Patient technology in the categories of mobile application and wearable were the most commonly used in clinical trials in these 2 therapeutic areas. More specifically, patient technology was most commonly deployed in clinical trials for diabetes, asthma, or chronic obstructive pulmonary disease (COPD). Given the prevalence of more traditional medical devices to treat these conditions (e.g., asthmatic inhalers, continuous glucose monitors), it is not surprising to see use of patient technologies. Patients with these conditions may have an increased aptitude or acceptance of patient technology due to familiarity with using other devices. Thus, we anticipate these therapeutic areas will continue to lead other therapeutic areas in patient technology deployment in the near future. However, we are hopeful that therapeutic areas not yet utilizing the full potential of patient technologies will eventually get there. As an example, oncology, which represented 17% of cases using patient technology. Figure 5: Percentage of Entries by Therapeutic Area in Which Patient Technology was Used3

3 Totals will add up to greater than 100%. Entries could select more than one therapeutic area as applicable.

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DEEP DIVE IMPLEMENTATION EXPERIENCE FINDINGS Our data collection effort sought to understand both contextual information and implementation experience of using patient technology in clinical trials. In order to maximize the understanding of experiential learnings, the survey included free-text questions with 10-point rating scales. The scales were specific to each free-text question and captured various categories of information. The goal of this dual question format was to build a more complete picture around the magnitude of any experience shared. The following sections contain observations made by the Transcelerate Patient Technology team based on information from the free-text implementation experience data. We focus primarily on the following areas:

• Experience with overall planning and implementation • Experience in different geographic locations • Experience with patient use • Experience with data management and statistical analysis • Experience with regulators, ethics committees and more • Experience with repeated use of patient technology

These findings are not meant to communicate correct or incorrect ways of operating. Instead, they are meant to provoke thought about how one might learn from another’s experiences and better implement patient technology in clinical trials.

Experience with Overall Planning and Implementation As can be expected with a survey distributed to numerous pharmaceutical companies, the experiences with planning, executing, implementing, and provisioning of patient technology in clinical trials varied. Half of the cases reported low to moderate difficulty with implementing patient technology in clinical trials, while the other half reported high levels of difficulty. Assessing the entries revealed some common pain points. The commonality of certain pain points supports the idea that sharing these challenges could help others avoid them in future patient technology implementations. Common pain points as well as corresponding considerations to take into account for mitigating those challenges are presented.

• Patient Population: The survey results indicate that clinical trial teams should take the patient population being studied into account when selecting patient technology. For example, some cases referenced a need to revise original patient recruitment requirements due to issues with recruiting elderly patients with sufficient technical literacy. Clinical trial teams should also anticipate the potential for patient population limitations depending on the patient technology in use (e.g., using complex mobile applications or BYOD with an elderly population may not result in the desired compliance).

• Vendor Management: Good planning, including working closely with the patient technology vendor during early stages of development, should improve implementation. Clinical trial teams should fully understand the technology, establish regular communications and meetings with the vendor, perform user acceptance testing far in advance of the anticipated live date to allow time for fixing problems, and

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consider customization of features and their potential downstream impact as well as anticipate translation needs ahead of time.

• Training: Developing a detailed training plan for all involved stakeholders (sites, patients, monitors, etc.) is critical for successful implementation. Training was most successful when it was offered via multiple mediums (e.g., in-person, online, paper, etc.). It was also helpful to be prepared to provide training on any back-up system to the patient technology (e.g., paper forms).

• Site Infrastructure: A comprehensive understanding of the specific patient technology and early engagement with site stakeholders is important for successful implementation. Site feasibility assessments should be robust and include questions on both site capability and the patient experience. Feasibility assessments may also include questions related to information technology (IT) infrastructure, staff experience with the patient technology, and internet connectivity (e.g., Bluetooth, Wi-Fi) in places where patients are expected to use the patient technology. In case the patient technology is to be used at the patient’s home, similar questions should be asked. For example, clinical trial teams should consider the likelihood of patients having adequate internet connectivity for transmitting the required data and create back-up plans in the event they do not. Teams should also have a plan to deal with security protocols or firewalls that could impact the patient technology use and data transfer. If the patient technology makes use of universal serial bus (USB) drives or secure digital (SD) cards, the team needs to confirm whether the site allows use of these in local systems.

• Risk Mitigation Planning: Creating a risk mitigation plan (RMP) is an important activity during implementation of any patient technology. This is especially the case when the patient technology is contributing data towards clinical trial endpoints. To protect against major issues, clinical trial teams should incorporate plans into the RMP for managing poor patient compliance, limited data availability, lost data, and other issues such as malfunctioning technology. It is important to ensure that the RMP and the clinical trial protocol include cross references to each other where appropriate. These issues often reveal themselves in the middle of a trial, making the need for back-up plans critical.

• Data Collection: The cases described by TransCelerate member companies referenced challenges with various components of the data collection lifecycle including selection of algorithms, interpretability of data, accuracy of data, transfer of data, etc. Trial teams should work closely with both internal teams (data management, programmers, etc.) and external teams (e.g., vendor, site) to mitigate potential challenges with data collection.

• Patient Support: It is important to ensure that adequate support is available for patients when selecting patient technologies and determining the geographic locations in which the technologies will be used. Patients need to be made aware of the process for receiving both technical support or trial support. Issues with patient support were observed primarily due to time zone and language differences.

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• Post-Production Changes: The technical aspects of the technology and efforts and cost associated with any changes post-production should be discussed ahead of time. If not well understood, teams may be faced with unexpected, costly, and lengthy changes during clinical trial conduct.

• Patient Privacy: Teams need to understand the impact of the selected patient technology on patient privacy (e.g., videos and patient conversation recording) and be prepared to provide robust privacy guarantees that address concerns from patients, caregivers, sites, ethics committees, and regulatory authorities.

• Technology Complexity & Complexity of Use: Teams should consider how the patient technology will be used (e.g., sensors attached to a patient’s body for long time can be uncomfortable and reduce compliance or deter patients from participating) and whether it needs integration with other systems or technologies. For example, one case that scored planning, executing, implementing, and provisioning patient technology as highly difficult shared the following, “We implemented devices from different manufacturers across multiple measurement modalities, including audio, video (multiple camera angles), skeletal tracking, wearable inertial measurement units, biopotential recordings with the intention of synchronizing the data streams in time and with respect to protocol activities. This proved possible but very difficult.” In the cases collected from the TransCelerate member companies, it was observed that a high level of difficulty with implementing the patient technology correlated with challenges with training, getting patients to adopt the technology, and data management.

For additional considerations on how to implement patient technology effectively in clinical trials, teams should consider utilizing solutions within the TransCelerate Patient Technology Toolkit (TransCelerate BioPharma Inc, 2020). Specifically, the Patient Technology Implementation Framework is designed to address some of the challenges faced by clinical trial teams during implementation. The Patient Technology Toolkit is publicly available on the TransCelerate website.

Experience in Different Geographic Locations Implementation experience of patient technology in different countries or regions will vary across clinical trials. However, clinical trial teams should continually learn from their experiences. This includes frequently evaluating the feasibility of different countries in which to conduct trials with patient technology. In this scenario, similar questions to site selection apply such as assessing whether the country has appropriate infrastructure in place (e.g., internet services). Based on the experiences collected in our survey, it is clear that special attention should be given to understanding regulatory landscape, infrastructure (as mentioned earlier), and import license requirements. A select set of topics that should be assessed during that evaluation include the ones listed here.

• Regulatory Landscape: We propose that sponsors perform a regulatory assessment of a country before deciding to implement patient technology in trials within that country. A regulatory assessment should focus on understanding importation guidelines, registration requirements, privacy impacts, etc. related to use of the patient technology. Advanced assessment and subsequent planning should help teams to avoid delays with implementation. If possible, teams should consult with the local regulatory authority

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and ethics committees prior to selecting the country. With the recent release of General Data Protection Regulation (GDPR) in the European Union (EU), it is imperative to ensure compliance of the technology with the regulations before using the technology in EU countries. TransCelerate’s Patient Technology Regulatory Landscape Assessment Tool can be useful in evaluating a country’s regulations relevant to patient technology implementation. This tool is designed to help address ambiguity and represents the current state of regulations, guidance, and policy as of December 2019, pertaining to how various digital solutions are classified and used within a clinical trial. This tool presents an overview of US and EU regulatory requirements related to patient technology and their use in clinical trials as well as links to relevant regulations, guidance, and intelligence which address these topics (TransCelerate BioPharma Inc, 2020).

• Broadband Infrastructure: In our survey, clinical trial teams mentioned facing challenges with broadband connectivity at sites as well as in patient homes. Issues with connectivity can lead to incomplete data as well as issues with data transfer (loading data from the device to the trial database). Assessing overall broadband connectivity infrastructure may help in determining the ultimate feasibility of a country or region. Trial site location (major city versus rural setting) may also affect the internet connectivity (wireless connection) at a patient’s home and must be part of any evaluation.

• Importation of Technology: Additional steps may be required when trying to import new patient technologies into various countries. Different countries may have different requirements that must be met before new or novel technologies can be released within their borders. Study teams should take time to understand country-specific requirements (as applicable) and whether additional requirements (e.g., marketing approvals) are necessary. Allow for extra time to acquire appropriate documentation (e.g., for import licenses) and budget for additional costs (e.g., custom’s fees) in your planning. Additionally, prepare backup plans in the event the patient technology is damaged during the importation or shipping process.

Experience with Patient Use Patient technology can oftentimes feel new and exciting in the hands of sponsors as it poses new ways to interact with patients (Polhemus, et al., 2018). However, at the end of the day, the technology is designed to be in the hands of the patient. This makes understanding technology adoptability, user experience, and feedback critical. Strong patient adoption and effective use of the patient technology across a trial or program is a good predictor of positive trial outcomes (in terms of successful use of the patient technology). To understand this further, we explored patient technology adoptability as part of the collected cases. The responses reveal a nearly equal balance of sponsors experiencing slow, average, or fast patient adoption of patient technology. Several cases mentioned strong patient adoption in younger patient populations while older patient populations preferred not to use the technology when avoidable. This further illustrates our earlier finding about the importance of selecting technology based on your patient population. Specifically, patient age, habits, and technical literacy must be considered. Understanding this information in advance of the clinical trial kick

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off can help sponsors create tailored and understandable trainings and user-friendly device instructions. It can also provide useful information for future patient technology selections. Some shared challenges were observed in the cases with slow or weak patient adoption of the patient technology. These included non-user-friendly devices, trainings that were too long or unstructured, malfunctioning devices, and preferences for paper-based options. In a few cases, patients actually declined participation in a trial or dropped out of a trial because of the patient technology and its role. Losing trial participants can be detrimental and supports the idea of introducing alternative options (e.g., paper-based solution) in applicable situations. These challenges showed the importance of a well-planned strategy for the rollout of patient technologies. Some cases also shared a summary of feedback from patients on the patient technology. This feedback was received primarily in relation to areas that needed improvement. Examples of such feedback are shown here.

• Patients experienced frequent issues with patient technology connectivity, the device itself (battery), and transmission of data. The result was that not all data could be recorded as expected.

• In some cases, the device was disruptive to the patients’ lives (e.g., blood pressure taken every 2 hours).

• The technology was an extra burden for patients; however, it provided patients with extra healthcare support.

• Some patients did not want to return the device at the end of the trial. • Patients sometimes struggled to line up their face with the outlines on the application

because of slight tremors or poor coordination. • Adhesive stickers could not be worn in the shower during a required a 24-hour

recording. This, as well as a reluctance to wear the patient technology in hot temperatures, made compliance challenging.

• Patients found that the text characters in the application were too small to read in some instances.

• Electronic-based technology was too difficult for some patients, partly due to symptoms of the disease under study and partly due to the technology (touchscreen required significant force to register responses).

From the examples above, it is very easy to understand the importance of collecting feedback from patients on their experience using patient technology. Patient feedback can provide necessary data for decision making throughout the course of a trial. It might even be needed to define meaningful and validated questions at a specific timepoint during or at the end of a trial. It can also inform the design and execution of future trials using the same or similar patient technology. Despite this, the collection of feedback on the patient experience was not universal. Only 17% (4/23) of the cases with already completed studies collected such feedback from patients. Only 12% (7/58) of the cases with ongoing studies included the collection of such feedback. This was surprisingly low, considering the impact patient technology can have on a trial including participation and retention. Of the cases that involved trials that included patient feedback on the use of patient technology, feedback was collected via surveys, interviews, and satisfaction measurements. For these trials, the survey respondents noted that patient feedback was “very positive with lots of comments

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and ideas on what we can add for future versions”, “very well received”, and “very positive”. These results further support the concept that collecting experiential feedback from patients is valuable.

Experience with Data Management and Statistical Analysis One of the driving factors for utilizing patient technology in clinical trials is its ability to collect robust and meaningful patient-generated data including home-use settings. Patient technology can collect much greater quantities of data than traditional paper-based methods. As a result, it was not completely surprising to find that TransCelerate member companies’ ratings of their experience conducting data management and statistical analysis skewed towards some difficulty to high difficulty. This also aligned with the likely possibility that new or innovative technologies require new processes, systems, and methods for management and analysis of data. Selected lessons learned as well as challenges in conducting data management and statistical analysis are presented here. Trial teams should ensure that the selected patient technology can be supported by the organization’s systems and platforms for data collection. To avoid issues, the requirements of the patient technology should be reviewed carefully before selection of the technology. Data management teams, including programmers and developers, should be involved early in the discussions and should assist with defining the data collection specifications and transfer requirements (format, frequency, etc.). Additionally, mock runs can be valuable for testing the data mapping to the database or data transfer specifications before the start of the trial. Early and/or frequent data reconciliation as well as resolving reconciliation issues expeditiously will prevent unpleasant surprises prior to database lock. Furthermore, it is important to incorporate elements specific to patient technology in any data management plan with clear guidelines on aspects important to identification and handling of duplicate or incorrect data, missing data, data received outside the protocol window, data clean-up, etc. Teams should also provide clear instructions for a back-up process in case of malfunctioning technology. Possible challenges with data management and statistical analysis of data collected through the use of patient technology, as shared in the survey cases, is summarized below.

• Data Transmission Issues: Failures or issues with data transmissions from patients’ devices to vendor databases as a result of connectivity issues or malfunctioning technology was the most common challenge. To mitigate loss of information, it’s proposed that teams have a dedicated and well-trained helpdesk and backup options available for transferring data. One case in particular reported improvement over time with data collection but noted that success of data transfer often depended on the network condition at each site.

• Missing or Duplicate Data: Apart from data transmission issues, technology malfunction can result in lost or missing data. Some of the reasons for this include patients who lacked proper training, malfunctioning technology, and patient non-compliance with patient technology instructions. In one case, failure to use the patient technology per instructions led to loss of baseline measurements, impacting the ability to conduct data analysis. There were also cases that mentioned duplicate data or collection of more data

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points than expected. This might happen if a device is replaced mid-trial, causing an overlap of the allowed time window for the recording of data.

• Integration and Reconciliation with Other Datasets/Electronic Data Capture (EDC): In some cases, problems with integration and reconciliation with other datasets and/or EDC data were reported. Teams faced challenges with combining data from multiple data sources, including paper. Especially when discrepancies existed. Teams also faced challenges with the amount of time required to reconcile data and fix data issues. One collected case referenced difficulty reconciling the site visit date field between what was reported in eDiaries and what was reported by the site. In a related issue, the sheer amount and complexity of data collected from patient technologies made interpretation and display of integrated data difficult from a statistical standpoint. Concerns included a lack of clarity in scoring, missing data edit checks from the vendor side, and the need for monitors to review data and ask sites for clarification/correction via data correction forms.

• Handling of Data Correction Forms (DCFs): DCFs were utilized in situations in which patient technology data needed to be corrected (e.g., a wrong patient number was assigned). The process for creating, submitting, and approving DCFs may vary depending on the system/platform utilized. If this process is not well-defined and/or poorly communicated, teams are likely to face issues with handling and approving DCFs. This issue can be amplified in situations involving multiple languages or language barriers.

It was evident throughout the collected cases that using patient technology required extra effort and time as described in some of the topics above. Teams should plan accordingly and anticipate the need for dedicating more time and effort to the activities of data management and statistical analysis. While challenges may arise early, improvement over time is likely with repeated use, increasing familiarity, and knowledge as well as development of corresponding standards and procedures.

Experience with Regulators, Ethics Committees, and More Regulatory authorities play a critical role in the successful application of patient technology in clinical trials. Without regulatory acceptance of technology-generated data, the full value of a patient technology deployment is unrealized. We attempted to assess how frequently sponsors were engaging regulatory authorities about their patient technologies and the feedback they were receiving from these regulatory authorities. The responses we received in our survey indicated that in most cases, routine processes or internal experts were consulted when implementing patient technology. As part of these processes, 30% of cases mentioned consulting a regulatory authority. In the majority of these cases, the details of the patient technology were included in the protocol submission and no additional questions or concerns were raised by the regulatory authority. Unfortunately, our cases did not always provide an assessment of the value of such a regulatory authority consultation, and two respondents indicated that they did not receive feedback or recommendations from the consulted authority. Internal engagement within a sponsor company for purposes of obtaining legal, ethical and privacy-related approval to use the patient technology is also important. In the majority of the

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cases in our survey, clinical teams indicated either no difficulty or some difficulty obtaining internal approval for the use of patient technology. Even though most respondents did not indicate a high level of difficulty, some common themes about barriers emerged from the responses.

• Review Cycles: Additional reviews were sometimes required for use of patient technologies. For example, one case referenced additional reviews being needed for a mobile application that recorded videos of the face. Ultimately, the technology was made optional in order to obtain internal approval.

• Data Collection: The collection of certain types of data delayed decision making due to internal committee concerns. For example, the availability of electronic screen shots for submission caused delays, and the first time use of a device in a clinical trial encountered hurdles from legal review.

• Privacy: In a handful of instances, the terms and conditions had to be amended to include GDPR language. Additionally, privacy impact assessments were required in some scenarios for utilization of patient technology in GDPR impacted countries. In addition, some European countries raised concerns with collection of patients’ phone numbers, and one case mentioned providing devices to patients as an alternative solution.

• Location: Utilization of patient technologies in different geographic locations with local-language translations of the technology and associated documents can cause delay with obtaining approval from the necessary committees (both internal and external). Some devices were not approved for use in some countries or were difficult to import. Local laws, particularly on privacy, need to be well understood.

We concluded that introducing patient technology into clinical trials is not overly disruptive to the existing processes for obtaining internal legal, ethical, and privacy approval. Despite this, clinical trial teams should work proactively to consider the barriers mentioned above and plan to mitigate these potential issues in future patient technology implementations.

Experience with Repeated Use of Patient Technology To conclude our patient technology data collection, we sought to understand sponsors’ experiences with the deployment of patient technology and whether it was ultimately successful or not. We decided to measure this by asking whether one would consider using the patient technology again. The majority of cases (54%) would use the patient technology again in the same use case. Broken down further, 73% of cases who would use the patient technology again deployed it in late-stage trials (e.g., Phase III or IV) while the remaining 27% deployed it in early-stage trials (e.g., feasibility, Phase I, Phase II). This led us to believe that sponsors are generally making good technology selections as indicated by the feeling or belief that the patient technology can be used again. When asked why, respondents said that their patient technologies improved patient retention and compliance, reduced site and patient burden, collected more reliable data, reduced the need for paper, and optimized the number of site visits due to remote monitoring capabilities. In contrast, only 6% of cases definitively said they would not use the technology again. This was a much lower result than expected. This may point to optimism from sponsors on

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the benefits that patient technology provides, even in the face of potential or experienced implementation difficulties. The reasons for why the patient technology would not be used again ranged from a lack of user friendliness to insufficient features necessary for use in clinical trials. It should also be noted that 35% of cases were unsure about using the patient technology again or said they would maybe use it again. In almost half of these cases, the patient technology was still part of an ongoing trial and so a determination as to future use could not yet be made. In other cases, sponsors indicated a need for various types of both technical and/or operational improvements before the technology would be used again (e.g., improved data backup, greater emphasis on privacy).

CONCLUSIONS We believe that our efforts to carry out the principles of collaboration, transparency, and sharing of data and experience were a success. Our first of its kind data sharing activity helped organizations (TransCelerate member companies) shed light on the type(s) of information they view as shareable. Through this exercise, learnings across clinical trial sponsors have now been documented and distributed for the benefit of others. Notably, common challenges emerged in the areas of patient technology support, data collection and transfer, and technology adoptability. In a few instances, TransCelerate member companies shared that our data collection and sharing exercise forced their organizations to think harder about how to effectively collect and disseminate patient technology learnings internally. Many recognized that the ability to gather the type of information we were looking to collect was challenging and formal infrastructure to house this data did not exist. While there is still a lot of work to be done, we are excited to have initiated a mentality of learning from earlier experiences and sharing those learnings. In addition to helping organizations reconsider how they collect and share information, our data sharing exercise also resulted in actual change within TransCelerate member companies. In one example, a sponsor company noticed that patient technology was being used in clinical trials in a therapeutic area in which they had not considered deployment of patient technology. Reviewing the lessons learned and experience from the available cases in that therapeutic area helped this company to reconsider its approach for future trials. In a different example, an individual was able to use information shared as part of our exercise to formulate a better plan and set of topics to discuss with a patient technology vendor and the potential application of their technology. Using our exercise as an example, we encourage industry to identify those circumstances in which sharing experiences and lessons learned benefit all stakeholders and to consider sharing this information, as appropriate, in these circumstances. Without these shared experiences and lessons learned, we may not be able to anticipate the large study impacts from seemingly small decisions (e.g., asking patients to use a wearable technology during hot summer months resulting in poor compliance or requiring use of a touch screen technology in a patient population whose symptoms prevent them from pushing hard enough to register responses on the device).

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REFERENCES European Medicines Agency. (2020). Innovation in medicines. Retrieved from European Medicines Agency: https://www.ema.europa.eu/en/human-regulatory/research-development/innovation-medicines#eu-innovation-network-section Gottlieb, S. (2019, March 14th ). FDA Statement. Retrieved from U.S. Food & Drug Administration: https://www.fda.gov/news-events/press-announcements/statement-fda-commissioner-scott-gottlieb-md-new-strategies-modernize-clinical-trials-advance Polhemus, A., Kadhim, H., Barnes, S., Zebrowski, S., Simmonds, A., Masand, S., . . . Dupont, M. (2018). Accelerating Adoption of Patient-Facing Technologies in Clinical Trials: A Pharmaceutical Industry Perspective on Opportunities and Challenges. Therapeutic Innovation & Regulatory Science. TransCelerate BioPharma Inc. (2020). Patient Technology Assets. Retrieved from TransCelerate Biopharma Inc: https://transceleratebiopharmainc.com/patient-technology-implementation-framework/patient-technology-assets/

https://www.ema.europa.eu/en/human-regulatory/research-development/innovation-medicines#eu-innovation-network-section

https://www.ema.europa.eu/en/human-regulatory/research-development/innovation-medicines#eu-innovation-network-section

https://www.fda.gov/news-events/press-announcements/statement-fda-commissioner-scott-gottlieb-md-new-strategies-modernize-clinical-trials-advance

https://www.fda.gov/news-events/press-announcements/statement-fda-commissioner-scott-gottlieb-md-new-strategies-modernize-clinical-trials-advance

https://transceleratebiopharmainc.com/patient-technology-implementation-framework/patient-technology-assets/

https://transceleratebiopharmainc.com/patient-technology-implementation-framework/patient-technology-assets/

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