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Applied Clinical Trials November 2022

Ukraine invasion tests the resiliency and quick action of CROs and sponsors

Mobilizing and Maintaining Clinical Trials in Midst of War

ACT's biennial Salary Survey takes a look at the future of industry following increased adaptation of decentralized technology 

Salary Survey: The Age of COVID

Regulatory inspections are not something clinical research personnel enjoy; however, they often have to deal with them. Inspection readiness is an emerging concept that is rapidly gaining importance in clinical trials, and Marci Macpherson, executive director, quality assurance and compliance at Ionis Pharmaceuticals, will discuss her perspectives on inspection readiness and offer a few tips on preparing for inspections.

Moe Alsumidaie: What is clinical trial inspection readiness?

 Inspection readiness is a state of being in which sponsors and organizations are prepared for an audit at any time. This includes having procedures to enforce adherence to applicable regulatory guidelines and promoting a culture focused on quality—a solid commitment to proactive quality and risk management from every department involved in clinical trials at all times. Quality is not solely a QA responsibility; activities conducted by other teams are essential in this space, too, ensuring compliance with your procedures and having adequate policies and practices, such as real-time storyboarding, which helps with audit preparation efforts several years down the line.Inspection readiness is distinct from inspection preparation, which typically is owned by QA at sponsor companies and is a discrete activity focused on an upcoming planned inspection.

MA: How do you anticipate what regulatory authorities will look for during inspections?

 Following FDA warning letters and other published inspection metrics from health authorities, such as EMA and MHRA, are a good source, as surveilling them helps you keep tabs on what the agencies focus on. And then there's much outreach that the health authorities are doing; for example, FDA holds many webinars and other outreach efforts. I attended the first FDA MHRA joint conference in Washington, DC in 2018, and the next one in the UK in 2020, both of which focused on data integrity. The third was held earlier this year in London and also included Health Canada.Many of these sessions are downloadable, and they sometimes publish papers following those sessions. Another good place to start is the revised BIMO compliance program, especially if you're anticipating a potential FDA inspection. This document will tell you exactly what topic FDA inspectors will review.While many of the areas remained the same, there are new sections and expanded areas of focus included in the 2021 sponsor/CRO revision, including oversight documentation for outsourced services, remote monitoring, data safety monitoring board/data monitoring committee, just to name a few.

MA: What sorts of findings are regulators focusing on right now?

 Sponsor oversight and risk management are lighter in the literature, but emerging topics as health authorities look at sponsors' approaches particularly if issues have been noted.For example, in phase one trials, they’ll focus on dose escalation, and you'll see findings about those decisions. We continue to see findings with trial management; EMA and FDA are looking at the sponsors’ failure to monitor their trials and failure to select qualified monitors. Another area is the investigational product supply chain; are sponsors keeping up with their record-keeping requirements to show that chain of custody? Next is safety-related adverse event reporting. Are you meeting your 7–15 day reporting timelines? Data integrity and protocol compliance are also long-standing topics. Finally, the trial master file (TMF) is an area of focus (particularly for EMA/MHRA) and something you may want to storyboard if you had a transition in personnel or technology systems.

MA: Why is it essential to engage QA before and during an inspection?

 I can't stress enough how important it is toidentify subject matter experts as early as possible and engage them. Cross-functional involvement is critical, and QA is your partner and, hopefully, your best friend. The value is helping teams anticipate questions when issues arise. What questions will regulators ask, and how do we practice that? We've done these inspections before and can help. What went well, what didn't, and what needs to be followed up on.

MA: How can QA help personnel prepare for an inspection?

 Your QA department can sit down with the subject matter experts and conduct Q&A sessions, which are very useful. Knowing what to expect makes personnel more comfortable during an inspection and removes fear. Additionally, I recommend conducting a formal mock inspection, when feasible, to help with preparation, particularly for inspection-naïve teams.

Articles

Marci Macpherson, executive director, quality assurance and compliance at Ionis Pharmaceuticals, discusses her perspectives on inspection readiness.

Advice on Preparing for Regulatory Inspections

The debates that have flared over clinical trial adequacy and authorization delays for COVID-19 vaccines acquire a different perspective in light of discussions currently underway over Ebola vaccines in Uganda.

With the emergence of the coronavirus, sharp divisions emerged from 2020 as health authorities in the developing world met, in differing ways, the challenges of countering the impact. The creation, testing, and delivery of effective vaccines provoked criticisms as well as congratulations, criticisms that continue to be mirrored in persistent allegations of insufficient safety or bitterness over inequalities in access. Within Europe, relations between the European Union and the UK are still under a shadow of resentment over the disputed allocation of AstraZeneca's production, and hardly a month goes by without suggestions that adverse effects are being underplayed or ignored.

But in Uganda, where an outbreak of the Sudan Ebola virus is affecting rural and urban environments, the evaluation of candidate vaccines is presenting a very specific challenge: can clinical trials deliver results in time to have a beneficial impact? Unlike the Zaire Ebola virus which sparked most recent outbreaks, there are no approved vaccines or therapeutics for the Sudan Ebola virus, leaving care workers operating in a void. Since this variety of Ebola occurs only rarely there has until now been little possibility to test vaccines against it. Experts working for the World Health Organization (WHO) agreed in November that three candidate vaccines should be deployed to Uganda for a clinical trial. Trial protocols are being prepared, and WHO is working with the national ministry of health and the university of Makerere, as well as with developers and regulatory authorities. A plan to accelerate research to ensure access to investigational doses, and to help in scaling up and access to any subsequent licensed vaccine, is already in place, under the lead of WHO, the Coalition for Epidemic Preparedness Innovations (CEPI)—the public-private partnership on vaccine development—and Gavi, the Vaccine Alliance focused on vaccinating children.

At the same time, there is evidence of a slowing down of transmission in many of the affected districts as the outbreak is controlled without vaccines. As Dr. Matshidiso Moeti, WHO regional director for Africa, acknowledged in late November, "It will take time to get trial results"—and they may come through so late as to provide no assistance in this outbreak. However, WHO favours continuing the trial. “What I don’t want to be doing in six weeks or eight weeks’ time, is looking back if there’s a deterioration of the situation and saying ‘God, we should have. We could have. And if only we had,’” Mike Ryan, head of WHO’s Health Emergencies Program, told a press conference. His view was backed by his colleague, Dr. Ana Maria Henao-Restrepo, co-lead, research and development blueprint for epidemics. The unpredictability of outbreaks means "it’s better for us to work toward generating the evidence,” she said.

As always, there is a resources aspect to the discussions. A WHO appeal for funds to combat the Uganda outbreak is still massively under-subscribed. Right at the end of November the European Union has chipped in with $6 million to support clinical trials. The money is coming from a multiannual EU health program, EU4Health. Stella Kyriakides, the European health commissioner, said it was important to invest in R&D to find vaccines and treatments for the Sudan Ebola Virus as part of the EU's action beyond its borders. Most of the money is earmarked for the trial, and is expected to cover much of the costs. The clinical evidence generated for safety and efficacy will support marketing authorization applications, and to prevent and address future outbreaks. To improve the resilience of the supply chains, the EU's new Health Emergency Preparedness and Response Authority is exploring with EU member countries the possibilities to support production once these medicines are authorized.

Meanwhile, disturbing reports of resurgent COVID in China are demonstrating that even where vaccines are available, management of epidemics is as crucial as immunization. The reports also suggest that confident predictions of having mastered the pandemic could yet prove premature and over-optimistic. There is likely to be still plenty for clinical trials to do on COVID, too.

The Sudan Ebola virus outbreak in Uganda spotlights this dilemma in responding to public health crises.

What if Clinical Trials Deliver Results Too Late for an Emergency?

WCG launched a new Electronic Seizure Diary (eDiary), which is used to help patients with seizure disorders and their caregivers self-monitor and manage their seizures. WCG is one of two providers selected to develop an eDiary by The Epilepsy Study Consortium.

eDiary to help patients with seizure disorders and their caregivers self-monitor and manage their seizures.

WCG Launches New Electronic Seizure Diary

Jane Myles, Vice President, Clinical Trial Innovation at Curebase, discusses her current role, what led her there, and challenges their clients face with DCTs.

Podcasts

The Key To Innovation In Clinical Research? Listening.

Peter O'Donnell is a freelance journalist who specializes in European health affairs and is based in Brussels, Belgium.

Europe’s bid to make better use of data in drug research took a step forward late this month with the announcement that the first data partners had been signed up to the new data network of the European Medicines Agency (EMA), DARWIN-EU. Half-a-dozen hospitals, registries, and universities from Finland, France, Estonia, Spain, Netherlands, and the UK have been selected to collaborate in this emerging Data Analysis and Real World Interrogation Network.

The data that DARWIN will be collecting is to be used for studies to generate real-world evidence (RWE) to support scientific evaluations and regulatory decision-making. All the selected partners have access to real-world healthcare data, and will provide DARWIN with results of analyses of these data.

The move enables EMA to initiate the launch of the first three studies to be provided by DARWIN, on blood cancers, on valproate, and on antimicrobial resistance. These studies mark the start of a new phase designed to support regulatory decision-making. The aim is to have 150 such RWE studies per year by 2025. The number of data partners will increase, with a target of adding at least 10 new partners every year.

DARWIN is a federated network that will provide the European medicines regulatory network with access to results from analysis of data from real-world healthcare databases across the EU whenever needed, to support decision-making throughout the life-cycle of a medicine. The network is seen as a pathfinder for the proposed European regulation on health data.

Coincidentally but separately, a drug industry-backed coalition of European health technology assessment (HTA) bodies announced the same day a push for wider use of RWE to support the introduction of new products, particularly cancer treatments and advanced therapies.

At a symposium in Brussels last week, the RWE4Decisions network set out its priorities for expanding RWE use by HTA bodies and payers. Its aim is to promote the sharing of experience of using real-world data (RWD) in outcomes-based managed entry agreements and similar conditional reimbursement processes.

Inaugural group of members added to EMA’s new data network focused on RWE.

Real-World Evidence Makes Further Headway in Europe’s Healthcare Planning

VIDA has announced the availability of VIDA Intelligence Portal 2.0, a major upgrade of its cloud native, AI-enabled Intelligence platform. The newly updated platform offers staff training, image data collection and data quality, curation and management.

Represents major upgrade of cloud native, AI-enabled Intelligence platform.

VIDA Launches VIDA Intelligence Portal 2.0

The European Medicines Agency (EMA) is continuing to forge ahead with its aim of improving the environment for clinical trials in Europe, and is now turning its attention to platform trials. It intends over the next year or so to deliver further guidance on their planning and conduct, particularly so that they can be used as pivotal trials. A reflection paper will cover some of the methodological issues these trials raise to bring greater consistency in scientific advice and regulatory assessment. Clarifying terminology and design features will help to allow reliable estimates of effect and complement existing guidance on issues such as multiplicity and adaptive design, it says.

The move is an explicit recognition of the continual growth in the use of platform trials, particularly for scientific advice (even if more rarely so far as part of marketing authorization applications). The new guidance will zero in on specifics, such as the introduction of new arms when the study is ongoing, and what sort of framework and constraints can generate reliable results. It will cover type I error rate or uncertainties such as the use of non-concurrently randomized controls and changes in allocation ratio, or the use of non-concurrent controls. It will also take account of the impact on external validity and potential bias resulting from inconsistencies over time in study population, or sequential dissemination of results. Other issues will include design characteristics that may risk interpretability of the results, and how requirements for type I error control at the level of a platform trial can lead to different operating characteristics when compared with an individual comparison within the trial results.

A core drafting group will be responsible for delivering the first outline, which will then be discussed with the agency's methodology working party and associated specialized expert and stakeholders.Within the agency, the agency’s main scientific committee, the Committee for Medicinal Products for Human Use (CHMP), and the scientific advice working party will be most closely involved in the process. Other agency bodies that will be consulted prior to release of a draft to the public include its committee for advanced therapies, the pediatric committee, the pharmacovigilance risk assessment committee, and the committee for orphan medicines. Input will also be sought from regulatory agencies in the US, UK, and Japan.

As is its custom, the agency is setting out its planning early, to acquaint the clinical trials community with its intentions, to seek early reactions to its thinking, and to indicate when and how it will be seeking further input into the deliberations. The detailed timetable foresees setting up a drafting group before the end of 2022, discussion in the CHMP by the end of2023, the release of a draft in spring 2024 with a midsummer deadline for comment, re-discussion within the agency in the autumn, and adoption by CHMP in December 2024. Right now, comments are invited on the entire concept before Jan. 31 2023.

Meanwhile, closer scrutiny is now falling on the agency's poster-child, the mechanisms for the new European clinical trial framework that will be obligatory for all new trials in Europe from the end of January 2023. At the heart of the new system is the clinical trial information system that acts as a trial registry. This has been road-tested by one of the leading commentators on trial transparency, Nicholas DeVito, a key figure in the TranspariMED initiative that has recently been highlighting trial reporting failures across Europe and the US. In a late October assessment of the Clinical Trials Information System (CTIS) site, he found that the information on more than 10% of the trials listed is "extremely limited" and that it is difficult to identify the nature of the trials that appear. He says that more clarity is needed in the registry's presentation, so that casual users, or those with little European trial experience, are not going to be daunted by "the deep underlying regulatory mechanics that govern the registry." He found the options for exporting information about individual trials to be cumbersome and unfriendly, and concludes that "EMA hasn’t done anything yet to facilitate working with data from the new registry." And, in general, he finds that "there is no easy, simple way to have all the available information available in one scrollable screen."

His assessment is not all negative. He considers that a valuable area for researchers will open up with the trial protocol section—although for many trials this section will remain empty for up to five years after the end of the trial, owing to the way the legislation is framed. TranspariMED and other health campaigners have been calling for changes in this facility to oblige earlier publication of protocols and to end what it calls "protocol secrecy"—but that is a story for another occasion.

https://www.ema.europa.eu/en/documents/scientific-guideline/concept-paper-platform-trials_en.pdf

New guidance to drill down on more specifics in hopes of harnessing the growth of these types of studies. 


Bringing Further Certainty into Platform Trials in Europe

Mount Lebanon Hospital University Medical Center (MLHUMC) is partnering with Clinerion and has joined the global network of partner hospitals on Clinerion’s Patient Network Explorer. Patients at MLHUMC will gain access to therapies via participation in international clinical trials. MLHUMC will gain access to metrics to support patient care.

As part of the partnership, MLHUMC has joined the global network of partner hospitals on Clinerion’s Patient Network Explorer platform.

Mount Lebanon Hospital University Medical Center Partners With Clinerion

The Digital Medicine Society (DiMe) CEO Jennifer Goldsack and Takeda’s Head of Digital Health Sciences, Data Sciences Institute Shoibal Datta describe the current landscape of digitized clinical trials and discuss the business imperatives and strategic decisions needed to realize the full promise of digital in developing effective new therapies for patients.

ACT: As we emerge from the COVID-19 pandemic, is there still a case for digital in clinical trials?

 Yes, absolutely. Even before the pandemic we put one in five new drugs back on the shelf with no conclusive answer about their safety and efficacy because we were shutting down 20% of clinical trials due to recruitment challenges. Technical success rates continue to sit at around 5%. And even when we can bring a new molecule all the way through to market, it’s taking 10-15 years to make that new therapy available to patients. As the pandemic wanes, volunteer participants—the lifeblood of the clinical trial industry—are expecting hyper-personalized and low-friction interactions driven by their increasing exposure to digital in every aspect of their life. Today pharma also struggles with a huge unmet need for new medical products and pressure to generate new revenue streams as they face more patent cliffs. I don’t see another way to address these growing challenges without fully embracing digital.

I agree. Digital technologies offered enormous promise to reduce participant burden and increase the reach of clinical trials to more diverse populations long before the pandemic. COVID-19 definitely spurred the industry into action, but the differentiator that digital innovation has on drug development is being determined now by those organizations prioritizing the intentional use of all digital tools to optimize our ability to develop new therapies for patients. Digital strategies in clinical trials are embedded into our ways of working at Takeda and we are committed to realizing their promise.

ACT: The use of digital tools in clinical trials has been steadily increasing. Are we getting close to achieving digital as the status quo in clinical trials?

 No, we are not. Although a tremendous amount of progress has been made in the last three years, we are still in the very early stages of adapting to and learning how to navigate this new digital world at scale. Digital success will not be achieved by individual point solutions. Incumbents across the clinical research ecosystem, as well as new entrants—for example the device manufacturers, the decentralized trial platform providers—must collaborate and learn from one another to build this future. Biopharma needs to learn how to integrate digital into its pipeline strategy, which will require support for an entirely new way of working across the drug development lifecycle. At Takeda, we are fortunate to enjoy broad leadership support and are convinced that digitizing our processes is going to optimize them for patients. We are collaborating with our partners across the ecosystem and collaborating with organizations such as DiMe to develop new ways of working, supporting our colleagues to successfully execute these new digital strategies, and creating new cross-functional teams to ensure that deep digital expertise is embedded into every one of our clinical program teams.

 Honestly? Not very close. We’ve seen the 

number of sponsors using digital endpoints increase by orders of magnitude

 over the last three years and sustained growth and interest in decentralized clinical trials. However, we’re only scratching the surface of how digital can improve the overall effectiveness, efficiency, equity, and patient centricity of clinical trials. This isn’t saying that no one is thinking this way yet. Those pharma companies embracing digital in a holistic way are already reaping the benefits of faster and more inclusive recruitment and better data powering better decisions, sooner. Takeda is a good example of this, but there’s a lot of work to do for the industry as a whole.

ACT: What will it take for digital to become a core competency in the pharma industry?

 It will require a two-pronged approach. First, strong, strategic leadership from the top. Second, meaningful investment in the skills of the current pharma workforce and technical infrastructure they depend on. And I cannot emphasize enough the importance of supporting the pharma workforce to best succeed in the digital era. When we dig into confusion and resistance, we see a misunderstanding of what digital is intended to do for clinical trials. Not only is digital not coming to replace the hardworking teams committed to developing new therapies, it’s here to make that job easier. We already see Takeda’s commitment to upskilling and the benefits that follow. They are providing resources like DiMe’s 

 course across the enterprise so each contributor understands how they can use and benefit from digital tools and technologies.

 We see education as critical to developing the digital clinical trials ecosystem to maturity. It is not enough to simply develop the tools and digital services ecosystem. We must also learn how and when to use these tools. While we may be digital natives in our personal lives, conducting rigorous clinical research harnessing the full power of today’s digital tools is not something that is well established in our field. The language is different, the approaches are different, and the risks are different, and we need to learn them and be comfortable with them before going to scale.

ACT: What are the business and patient imperatives to not fall behind in the race to digitize clinical trials?

 This is an exciting time of change and inevitable evolution. Even beyond clinical trials, the practice of healthcare is rapidly changing. In the past three years there has been a shift of primary care from the clinic to local pharmacies and people’s homes. The needs and expectations of patients and clinical research participants are changing. We pride ourselves in following a simple but profound value system based on patient, trust, reputation and business. In that order. Digital approaches to clinical research are simply the right thing to do for patients. They allow us to offer choice and meet every patient where they prefer to be met. This also makes clinical trials more resilient, diverse and opens the door to a deeper understanding of disease than what the legacy model affords us. Those who choose to be early in this space will be able to better navigate inherently disruptive change in an increasingly competitive landscape to bring life-changing therapies to patients.

 It’s pretty simple; the current status quo in clinical trials is not sustainable. The digitization of clinical trials is certainly not going to solve all of the issues facing the clinical trials enterprise, but digital approaches offer real pathways to a transformed approach to drug development. And it’s becoming clear that digital strategies are necessary for any pharmaceutical company looking to increase their rates of technical success, speed the development of new drugs to market, and take a more inclusive approach to ensure our industry delivers value to all of the patients our industry exists to serve. Organizations who prioritize fit-for-purpose digital strategies and appropriately prepare their teams to deliver on these strategies will pull away from the competition as benefits are compounded rapidly over time.

The Digital Medicine Society (DiMe) CEO Jennifer Goldsack and Takeda’s Head of Digital Health Sciences, Data Sciences Institute Shoibal Datta describe the current landscape of digitized clinical trials and the decisions needed to fully utilize their potential.

How Clinical Trials Can Thrive in the Digital Era

Across the life sciences industry, there has been much talk about the cost and value of decentralized clinical trials (DCT) and the potential return on investment (ROI) when the expedited time to commercialization is considered. When faced with an 80% delay rate due to a myriad of reasons, the industry is potentially facing more than 51,000 delayed trials globally—not including new trials beginning in upcoming months and years.

 These delays are significant due to exorbitant associated costs and detrimental implications to breakthrough medicine.

A key component of the decentralized clinical trial model—a virtual site—allows participation in a clinical trial regardless of geographical location. Study care is delivered via telemedicine investigators, home healthcare staff, self/caregiver-reported data, community providers, and connected devices. In the Science 37 virtual site model, clinical trial execution is orchestrated by a unified technology platform and standardized processes with support from remote coordinators. Sponsors can expand the geographic footprint of a traditional trial, making participation possible for the 70% of patients who currently live outside a two-hour driving distance of a traditional site.

 This delivery model allows for participant access from anywhere, improving recruitment velocity, compliance, and retention.

As virtual sites are becoming a key part of protocols, sponsors face several questions. What is the ideal mix of traditional and virtual sites? What is the model that can help accelerate timelines? Can virtual sites help avoid delays while enabling diversity in clinical trials? Science 37 has examined the ROI of different scenarios of brick-and-mortar sites deployed in combination with a virtual site. Recognizing that most studies will likely contain this combination, we explore models that pharmaceutical sponsors can evaluate to find the best mix of virtual and traditional sites to meet objectives.

Science 37 released a new ROI model quantifying the value of virtual sites in multiple scenarios, based on seven drivers of value—and for each driver, an estimate of the impact on study costs and timeline. Scenarios are evaluated based on traditional and virtual site combinations to meet objectives of time, cost and risk mitigation, and diversity/inclusion.

The model output includes the cost and time associated with scenarios that correspond with key sponsor objectives, including:

The model is optimized based on sponsor objectives for the following scenarios:

Supplemental Virtual Site (for increased diversity and decreased risk)

To illustrate specific costs and benefits associated with virtual sites, we reference the following case study—a Phase II Hypertension study with 50 sites and 250 patients. The study forecasted a 6-month start-up (time to FPI) for traditional sites, a traditional site enrollment rate of 0.5 patients per site per month, and a virtual site enrollment rate of 18 patients per month.

By leveraging the ROI model, the sponsor evaluates each scenario selecting the best fit for the specific trial:

Scenario 1 - Add a supplemental virtual site

Many sponsors add a virtual site with a low-volume patient recruitment target to the existing site model as an introduction to DCT. Given the fixed start-up costs of the virtual site, this scenario can increase the R&D budget but has a more modest impact on the recruitment timeline because of the low patient volume assigned to the virtual site. Sponsors may utilize this approach to expedite time to first patient enrolled or as a risk mitigation alternative to planning for backup sites if needed. However, as the virtual site demonstrates greater recruitment velocity and diversity, Science 37 has observed that sponsors often increase the recruitment target, offering an even greater timeline benefit.

Adding a virtual site to the study delivery model while maintaining the planned number of traditional sites increases the R&D budget for the study but also delivers a projected six-month acceleration in the timeline.The net value to the company suggests a greater than 5x return on investment compared to the traditional site model, especially when the increase in diversity/inclusivity and decrease in non-performing sites are considered. While diversity was not measured in this ROI model, Science 37 has seen a 3x more diverse patient population when executing with a virtual site, in comparison to traditional clinical trials.

In this scenario, a virtual site replaces brick-and-mortar sites.The additional cost is completely offset by a decrease in sites, resulting in savings in the R&D budget.While the timeline remains similar, Science 37 has observed an increase in diversity and inclusivity, due to the removal of geography as a barrier to enrollment.Site performance risk is also decreased through a multi-channel approach to participant recruitment and retention. While there are scenarios where a full DCT approach is not possible, a fully decentralized model is often the optimal approach in rare disease studies where it is difficult to identify sufficient patients that reside near sites, observational studies, and long-term follow-up studies.

Scenario 4 - Co-optimize timeline and budget

Looking to optimize for both budget and timeline, suggests replacing 20 sites with a virtual site and a shift of ~66% of patients recruited to the virtual site. Similar to the above scenarios, this delivery approach increases study access and decreases site performance risk but results in a 4.7-month timeline acceleration with a small decrease in the R&D budget.

In this case study, the ROI Model shows that Scenario 2 offers the shortest timeline, with a savings of six months, while Scenario 4 optimizes both time and cost savings.

The scenarios highlighted above demonstrate the cost and value impact of adding a virtual site to a traditional study. One challenge in introducing any new approach into clinical research is the lack of industry experience with delivery. As such, many sponsors will choose to “dip their toe in the water” by adding a virtual site with a limited enrollment target to the study. Consequently, while this delivery design will reduce risk, the maximum return on investment will only be realized once a virtual site is implemented at a scale that meaningfully impacts the timeline or R&D Budget.

Beyond risk reduction, bringing the study to the patient is the right thing to do. Adding a virtual site to the clinical trial model opens up the possibility for any patient, anywhere, to be enrolled in the study—regardless of geography. This is a necessity for rare diseases and underserved populations and a choice for others, such as those who: don’t have a strong relationship with a clinical trial physician/site, work during the day when the clinic is open, or provide childcare in the home.For the growing majority of clinical researchers who are committed to accommodating patient choice and optionality in study delivery, the virtual site is an important delivery component that should be planned for in the protocol design at the outset to avoid the costly and time-consuming need for a protocol amendment later, to add it into the study.

Considering the strong return on investment demonstrated in the model scenarios, the added benefit of diversity and site performance risk mitigation, with the ability to deliver on the promise of broad patient access to clinical research and participant optionality, the recommendation is that sponsors plan for a mix of traditional sites and a virtual site in the initial study design. Industry leaders who understand and believe in the broad benefits of decentralization can accelerate adoption by directing their organizations to build the virtual site option into their standard operating model.

Science 37’s ROI model is already being used by numerous sponsors globally. The White Paper 

Decentralized Clinical Trials—Is There a Positive Return on Investment on a Virtual Site?

further expands on the model and can be accessed at 

https://www.science37.com/Resources/dct-roi

ClinicalTrials.gov (n.d.). Retrieved October 19, 2022, from 

https://clinicaltrials.gov/ct2/resources/trends

Sanofi launches new virtual trials offering with science 37

. Fierce Biotech. Retrieved October 19, 2022, from 

https://www.fiercebiotech.com/cro/sanofi-launches-new-virtual-trials-offering-science-37

Anticipating the need for both virtual and traditional sites during the planning process can provide high ROI for sponsors.

Featured Content

Applied Clinical Trials November 2022

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