- Full participation across all stakeholders — clinical trial sponsors, sites, investigators, patients and their healthcare providers
- Information is fully used to improve the overall quality, design and development process. We protect patients through shared, proactive safety science
- Improving the execution of research and development through greater harmonization with the potential to conduct collaborative trials
Newest Resources and Company Milestones
- TransCelerate debuted a new look for the website. It now boasts exciting new features, such as the Our Solutions Interactive Tool, blog, an updated News Center and an Engage with Us page.
- Dietmar Berger, Chief Medical Officer and Head of Global Development at Sanofi, has joined TransCelerate’s Board of Directors.
- Robert Baker, Vice President, Clinical Program Design and Exploratory Medicine/Pharmacology at Eli Lilly & Company, has been appointed as the new Corporate Secretary of TransCelerate.
- TransCelerate is a participant in the newly-launched Project Vulcan HL7® FHIR® Accelerator program and has played an active role throughout the setup process, including designing the Project Vulcan logo. For more on Project Vulcan, visit here or read this issue’s The Latest feature for in-depth details.
Pulse On Progress Updates
Improve the Patient & Site Experience
Improve the patient and site experience by decreasing burden, enabling a better-informed patient and improving clinical research awareness, participation & engagement.
- The Patient Experience team published two papers, both in DIA’s Therapeutic Innovation & Regulatory Science (TIRS). The first, entitled “Amplifying the Voice of the Patient in Clinical Research: Development of Toolkits for Use in Designing and Conducting Patient-Centered Clinical Studies,” discusses how the Patient Protocol Engagement Toolkit (P-PET) and the Study Participant Feedback Questionnaire Toolkit (SPFQ) were developed. The other, entitled “Evaluation of the Content Validity and Cross-Cultural Validity of the Study Participant Feedback Questionnaire (SPFQ),” evaluates the content and cross-cultural validity of the SPFQ in indications other than oncology and non-English-speaking countries. The paper discusses findings that provide evidence of its validity in these instances and supports the implementation of the SPFQ as a means of obtaining clinical trial participant feedback across global development programs.
- The Modernizing Clinical Trial Conduct Initiative team published a whitepaper, “Beyond COVID-19: Modernizing Clinical Trial Conduct.” The whitepaper proposes the ongoing utilization of innovative continuity solutions implemented during COVID-19 by specifically citing implementation challenges, lessons learned and opportunities for the continuity solutions moving forward. The team also developed five continuity solution case studies.
Information Sharing & Harmonization
Enable the industry to move toward greater harmonization of process and facilitate the sharing of information amongst industry stakeholders to enable the industry to capture, optimize and experience efficiencies.
- In July, TransCelerate participated in the webinar “Accelerating Science in the Age of COVID-19: Three Key Data Initiatives” with Vivli and the COVID Collaboration Platform (CP). The webinar examined the work that each organization is doing to support stakeholders to more quickly collaborate, share and analyze COVID-19 data to accelerate science. TransCelerate specifically discussed COVID-19 data sharing through DataCelerate®. For those that missed the webinar, you can watch a recording of the discussion or view the slides
- The Historical Trial Data (HTD) Sharing (Controls) Initiative team announced two new publications. One paper, entitled “Assessment of Placebo Response in Objective and Subjective Outcome Measures in Rheumatoid Arthritis Clinical Trials,” found that even patients randomized to placebo in a rheumatoid arthritis trial demonstrated improved outcomes, a finding that may influence future trial design. The other, entitled “Historical Controls in Randomized Clinical Trials: Opportunities and Challenges,” includes insights into the potential for large-scale historical placebo data to be used by drug developers and patients to guide trial design and analysis. Both papers provide helpful insights on the use of historical trial data, along with associated challenges and opportunities, to provide guideposts and use cases for the industry in drug development for both during and beyond COVID-19.
- The Clinical Content and Reuse Initiative led a webinar on clinical study report considerations for studies disrupted by COVID-19. Watch a recording of the webinar here.
- The eSource Initiative helped support the development of the Clinical Research Sponsor Laboratory Semantics in FHIR Implementation Guide (IG). The guide, published by Health Level Seven International (HL7®) and adopted as a standard for trial use, provides direction for using HL7’s Fast Healthcare Interoperability Resources (FHIR®) as a data transmission format for laboratory data, establishing a common format that is intended to reduce both site and sponsor burdens. Additionally, the SCDM eSource Implementation Consortium has expressed interest in using IG as a basis for developing requirements and guidance for sites to set up their FHIR®-based EHR environments. Find more information, including a list of FHIR® resources, here.
Enhance Drug Safety
Facilitate solutions that address a common set of challenges in drug safety surveillance such as engaging the public, collaborating with regulators, incorporating data and analytics, developing model approaches, and assessing the impact of efforts.
- The Intelligent Automation Opportunities (IAO) in Pharmacovigilance Initiative launched a new version of the Interactive ICSR and Automation Technologies Tool (IATT). The new IATT serves as a platform to report technology implementation data within the same ICSR Process map structure as the original tool. Additionally, the team has added a Case for Adoption resource that will help companies better understand and utilize TransCelerate’s IAO solutions as they explore the use of intelligent automation for pharmacovigilance.
- The Advancing Safety Analytics (ASA) Initiative published an infographic, “Investigating Overlap in Signals from EVDAS, FAERS, and VigiBase.” This infographic illustrates the results of their Drug Safety manuscript that indicates, for the majority of drugs, that there is no significant difference between the number and types of safety signals that arise from the three main data sources. Instead, each database by itself may constitute a valuable tool for signal detection.
Introducing the Vulcan HL7® FHIR® Accelerator
We spoke with Amy (Nordo) Cramer, Vulcan co-chair and Pfizer Global Product Development Clinical Operations Center of Excellence Strategic Partnerships, and Rakesh Maniar, co-lead of TransCelerate’s eSource Initiative and Head of eClinical Technologies, Global Data Management and Standards under Global Clinical Trials Operations Department, Merck. They both play critical roles in TransCelerate’s involvement in the Vulcan HL7® FHIR® Accelerator.
As healthcare continues to grow more digitized, modernized standards are being rapidly adopted for the electronic exchange of healthcare data. In the United States, this adoption was introduced by the Office of the National Coordinator for Health Information Technology (ONC) with the 21st Century Cures Act interoperability rule. This rule is intended to put patients in charge of their health records as a “key piece of patient control in health care” by “promoting modern health IT for all stakeholders”.
In an effort to standardize healthcare interoperability, Health Level Seven (HL7®), a standards development organization, created and introduced the modernized standard, Fast Healthcare Interoperability Resources (FHIR®). One of the key benefits of FHIR® is that it was built to exchange data with an “internet-based approach,” which enables and facilitates data interoperability in a more structured, scalable and standardized manner. Moreover, FHIR® has buy-in from major cloud vendors like Amazon, Google and Microsoft who have built FHIR® into their application programming interfaces (APIs), and almost 90% of healthcare applications exchange data via FHIR®.
FHIR® will complement other standards that already exist globally, such as the Observational Medical OutComes Partnership (OMOP) and International Organization for Standardization (ISO). Additionally, regulators are making efforts to harmonize FHIR® through projects such as Common Data Model Harmonization (CDMH), a multiagency collaboration sponsored by the Food and Drug Administration (FDA).
The improved method of data sharing creates significant benefits for patients and has led to its rapidly growing adoption around the world. In fact, several industry stakeholders in the clinical care space have taken advantage of the data sharing capabilities that HL7® FHIR® brings to the healthcare industry. Members of the health insurer and healthcare provider communities have established the HL7® FHIR® accelerator programs, Da Vinci HL7® FHIR® Accelerator and Argonaut HL7® FHIR® Accelerator, respectively. With the launch of the Vulcan HL7® FHIR® Accelerator, HL7® FHIR® is helping the clinical research space make great strides toward harmonized interoperability.
“We realized that the only industry group who was missing from developing out these standards was pharma,” says Amy. “It only made sense for us to put together a group that would focus on the needs of clinical research, as a community, in HL7 FHIR standards.”
Thus began the HL7® FHIR® Accelerator Project Vulcan program. Stakeholders from across the pharmaceutical industry, including research academia, government agencies, patient advocates, technology vendors and more joined forces to kickstart this Accelerator program with a goal to help the research community leverage FHIR® effectively.
More specifically, the overarching goal of Project Vulcan can be broken into four parts.
First, it aims to bridge the existing gaps between clinical care and clinical research to improve patients’ lives. As Rakesh notes, “There are gaps in today’s standards that place burden on sites, patients and sponsors. Data needs to be collected twice, first for EHR [and] then for EDC. If we can create one single source of truth and collect data with a dual purpose, it will be a win-win situation for all.”
By using modernized standards to bridge these gaps, we can minimize study timelines, reduce the opportunity for error and enable faster access to data to respond to any safety signals quickly. Additionally, we can accelerate further development, refinement and use of the HL7® FHIR® standards to optimize the design, conduct and reporting of clinical studies.
Goals two and three are to make strategic connections and collaborations, maximizing the collective resources so that work can be shared and leveraged throughout the translational and clinical research community. “Vulcan is dedicated to both Translational and Clinical research to align all research on patients so that data can be reused and shared,” said Amy. “It’s important that we are bridging gaps in all research pertaining to patients.” These goals are about building in efficiencies and ensuring the group is representative of the research community, including all relevant stakeholders, to conduct research in a more improved and patient-centric way.
Last, the Accelerator aims to give sponsors an opportunity to provide tangible value to the clinical research space by delivering integrated tools and solutions. For example, connect-a-thons, in which hundreds of people from across the ecosystem come together to write code implementing FHIR®, help to take HL7® FHIR® from a straightforward standard to a demonstrated implementation. The Project Vulcan team plans to participate in its first connect-a-thon in 2021.
In addition to those four goals, immediate plans for Vulcan Accelerator include raising awareness of the need to close the significant interoperability gap between clinical care and clinical research. “The community is aware that a gap between clinical care data and clinical research data exists,” notes Amy. “FHIR allows for a clear path in bridging that gap.”
Over the next several years, the team hopes that healthcare data harmonization will be globally accessible to all stakeholders.
So, how can you support the Accelerator? “Have an open mind towards FHIR,” asks Rakesh. “These standards have a clear value story and benefit, and it is the right time for the industry to pay attention.”
“We have an opportunity as a research community to change the trajectory of clinical research,” says Amy. “I’d like to call on all research community members to join Vulcan and help us drive the industry forward.” For more information, read about HL7® and FHIR® standards on the organization’s website or email the team.
We’d like to extend a special thank you to the TransCelerate members who have played such an important role in the launch of Project Vulcan: Rob Goodwin (Pfizer), Mika Ogasawara (Pfizer, Japan) and Christi Denney (Eli Lilly and Company). We’d also like to thank project managers Madeleine Brennan and Stacey Tegan.
The Modernizing Clinical Trial Conduct Initiative
For the past 8 months the COVID-19 pandemic has disrupted the pharmaceutical industry’s traditional methods for conducting clinical trials and delivering care to patients. Despite the disruptions, regulators and industry stakeholders alike have been tasked with ensuring continuous, safe and reliable care to patients enrolled in clinical trials. With the help of innovative approaches, industry was able to successfully maintain continuity for many clinical trials, without compromising patient safety or data integrity.
Amidst the change and disruption, ample opportunity has emerged to understand and apply lessons learned—including both challenges and successes—to future ways of designing and conducting clinical trials. A recognition of this opportunity led to TransCelerate BioPharma’s creation of the Modernizing Clinical Trial Conduct (MCTC) Initiative, which aims to understand and share key lessons from the use of continuity solutions implemented during COVID-19.
We spoke with Rodrigo Garcia, Head of Clinical Innovation at EMD Serono/Merck KGaA, and Simon Trowell, VP Clinical Development Quality and Risk Management at GlaxoSmithKline, two individuals who played important roles in launching TransCelerate’s MCTC Initiative, to get more insight.
Q: Tell us more about the goal of the MCTC program. What near- and long-term benefits does the MCTC program provide for industry stakeholders?
A: The goal of the MCTC program is to take lessons learned from the response to the COVID-19 pandemic and explore ways to increase patient centricity, all while maintaining and improving patient safety and the reliability of trial results. By modernizing and simplifying clinical trial conduct, we can create efficiencies in the clinical development process which can deliver innovative medicines to patients faster.
The near-term benefits include more efficient clinical trial conduct while still ensuring patient safety and the integrity of clinical trial data. The longer-term benefits would result from normalization of modern approaches (e.g., direct to patient shipping) and solutions (e.g., telemedicine) in a post-pandemic world to accomplish goals such as: increased trial participation, improved diversity of the trial participants and acceleration of clinical development to enable new medicines to reach patients faster.
Q: What are clinical trial continuity solutions and why are they important in our response to both the pandemic-stricken and post-pandemic worlds?
A: Clinical trial continuity solutions are the solutions and approaches utilized by industry that allow for the safe continuation of the clinical trial process during the COVID-19 pandemic. Broadly speaking, these solutions are intended to ensure trial product availability, protect participants, reduce burdens of clinical trial participation and the number of essential personnel needed at study sites, streamline or automate the collection of data while maintaining data quality, and further advance the concept of risk-based approaches.
Some of these solutions existed pre-pandemic and have since been accelerated or utilized in greater capacity, such as telemedicine for communicating with patients virtually. Other solutions were more novel, such as direct to patient shipping of investigational medicinal product (IMP) from either a site or drug depot.
These solutions have allowed for trial continuity during the pandemic, and their use, as the MCTC program outlines in the Beyond COVID-19 whitepaper, should be considered for extension beyond the pandemic to further harness the benefits for participants and modernize clinical trials.
Q: From your perspective, what were the top lessons learned from these continuity solutions?
A: Before applying any continuity solution, you need to clearly understand what the challenge or gap is that the solution will “solve”. For example, if digital data collection tools and the use of technology platforms are being used to facilitate remote participation, one must think about the technical challenges for both patients and sites and plan for their use of technology-based solutions. Planning could involve building in time to train sites, participants and other key stakeholders using this new technology or by including an in-person training to help set up the device or technology.
Applying continuity solutions is not a one-size-fits-all approach and sponsors must apply an individualized mentality when implementing these solutions. Implementing these solutions is an iterative process and often involves flexibility and adaptability. Reflecting on these lessons learned is important as it provides an opportunity to proactively handle future challenges.
Q: Looking ahead, what will be the main opportunities for implementing continuity solutions more permanently to improve clinical trial conduct?
A: Based on the progress already achieved during the COVID-19 pandemic, certain continuity solutions could remain as the “new standard” of improved clinical trial conduct. Some immediate examples that come to mind include telemedicine, home health nursing, direct-to-patient shipment of IMP, electronic informed consent and electronic patient-reported outcomes. These solutions can produce numerous efficiencies towards modernizing clinical trials by improving various aspects of the drug development process such as increasing patient engagement, creating efficiencies and minimizing burdens on both sites and trial participants.
Combining multiple solutions can provide additional benefits versus a single application. For example, combining the solutions of telemedicine, home health visits and local community-based laboratories to facilitate remote visits for patients, may alleviate travel burden, saving patients’ time, money and effort. By reducing major barriers to participation, we may also increase the willingness of individuals to participate in clinical studies.
While the normalized establishment of these solutions won’t occur overnight, we can positively influence industry’s speed of change by working collaboratively with all stakeholders (e.g., sponsors, sites, patients, regulators, vendors, etc.) It’s our hope that implementing these continuity solutions will not only improve clinical trial conduct, but also accelerate patient-centric approaches to clinical development, provide greater understanding of innovative clinical trial methodologies to support effective patient engagement, and create efficiencies when developing innovative clinical development methods.