Applied Clinical Trials Editorial Staff

Announced at SCOPE 2023, Los Angeles based AI company Narrativa is partnering with the Leukemia and Lymphoma Society to improve regulatory documentation. In order to achieve, the tech company is applying artificial intelligence models and automated data analysis to enhance the Beat AML Master Clinical Trial.

The trial is the first precision medicine clinical trial for blood cancer. As is the case with most precision medicine models, the trial uses advanced genomic technology to determine the best targeted treatment for each patient. The trial launched in 2016.

In a press release sent to ACT, Narrativa’s technology will automate the production of patient narratives and clinical study reports, along with other elements of the regulatory process. This should speed up the process and reduce the time it takes for new treatments to get approved and released to market.

Jenifer Bittinger, president of Narrativa, said, “The Life Sciences industry is at a precipice. Utilizing the advancements of artificial intelligence to automate clinical trial regulatory documentation will bring drugs to market faster and ultimately save more lives. Our goal is to use AI to improve systems within enterprises, but to also use it for good. We want to improve humanity.”

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The two groups will implement AI technology with an already existing precision medicine trial model.

AI Company Narrativa Teams Up with the Leukemia and Lymphoma Society to Improve Regulatory Documentation Process

On January 31, 2023, the use of the Clinical Trials Information System (CTIS) will become mandatory for new clinical trials applications and will serve as the single-entry point for submission by sponsors and for regulatory assessment. The EMA reiterated this deadline and updated the community on the current status of the system, which has been in transition for one year. 

EMA reminds the clinical trials community that the Clinical Trials Information System will be mandatory at the last day of this month. 

One Week Left for CTIS Mandatory Use Date

Mural Health Technologies, Inc., a new global provider of clinical trials technology, announced the launch of its new participant-facing technology platform, Mural Link at the CNS Summit in November. Sam Whitaker, co-founder and co-CEO of Mural Health, is no stranger to patient-facing technology. He founded Greenphire over 15 years ago to address participant payment technology. In this Q&A with 

, Whitaker discusses his time in the industry and patient needs in the current clinical trials market.

ACT: What have you learned from being in the industry for several years?

 The industry has talked about patient centricity for a long time, but it still feels like we are not putting our money where our mouths are. This is a cautious industry and opinions take a long time to change. But patient centricity is a real barrier to retention—each patient lost can delay drug approvals so we think the investment case for pharma companies is clear. Tech innovation done thoughtfully with real industry knowledge. Innovation really happens when businesses are given a chance to try something new and fail. This is counterintuitive to the spirit of our industry, where some companies maintain the status quo rather than disrupt their existing businesses.

ACT: Where did you see a gap in the market?

 Participant payments have not innovated to keep pace with what the average US consumer uses on a day-to-day basis. While consumers are most comfortable with digital payments such as Venmo, PayPal, Apple Pay, etc., participants payments are still stuck utilizing prepaid debit cards, which carry high fees, and are problematic for many reasons. Why is it that when participants enroll in a clinical trial, it has to feel like they stepped back 20 years in terms of the technology used?

The real gap in the market though, is missing the opportunity to leverage participant payments as a strategic angle to engage with patients and help shepherd them throughout the trial, getting their feedback on trial design and addressing the things that cause them to dropout of trials

ACT: What is the biggest challenge in participant payment and how can this be addressed?

 Similar to other eClinical technologies, there is an issue with the number of suppliers of clinical payment technology. The industry has a single large provider of clinical payment technology. The few smaller competitors have adopted a “me too” product philosophy, building replicas of the dominant company’s product, with no incentive to innovate. This dynamic is not unique to payment tech. A similar trend can be found in other eClinical product segments. The solution relies on new cohorts of companies to emerge with next generation technologies, for example, IRT space, in order to force incumbents to either innovate or step aside. Sponsor and sites, and other buyers in the clinical trials ecosystem, should remain curious and seek out new entrants to learn about emerging strategies.

ACT: How do participant payment methods impact patient retention?

Participant payments is critical for a study to deploy an effective retention strategy. 

 have long documented that financial barriers are among the highest reasons for loss-to-follow-up. The clinical trials industry continues to grow (est. 5.7% CAGR through 2030), increasing the demand for participants, and the economic environment continues to spiral (e.g. - rising unemployment, rising interest rates, and historic inflation). With 57% of US households not able to afford or manage an unexpected expense of $1,000, how can we expect individuals to take time off of work, incur expense to travel without delivering back to them a way to at least remain financially whole, during the study.

Sam Whitaker, co-founder and co-CEO of Mural Health discusses his time in the industry and patient needs in the current clinical trials market.

Identifying Patient Needs

Clinical eCOA/ePRO platform technology provider uMotif has released topline study results demonstrating its proprietary data capture interface incorporated into its eCOA/ePRO app offers a validated alternative to current text-based smartphone-based instruments for clinical, post-marketing and real-world research.

The independent quantitative equivalence study was conducted by SAFIRA Clinical Research at Dublin City University. Fifty-five participants completed testing in two randomized groups using the Motif visual interface and a traditional 5-point Verbal Response Scale (VRS-5). The study results showed equivalence between the Motif interface and standard VRS (high ICC) for capturing VRS data. Study results are expected to be published in 2023.

Bruce Hellman, Co-Founder and Chief Patient Officer expanded on the history of eCOA/ePRO to the significance of this study to 

. “Traditionally, PROs were paper, with a question at the top of the sheet, and patients would check one of the boxes [answers].” This same format migrated onto PDAs, then smartphones, but in the paper/text/content format. “We knew that patients found our app more engaging…it’s quicker to use, and it’s easier to use for different populations and age groups,” said Hellman. However, eCOA scientists have always asked that PROs could be validated from paper to PDAs, then PDAs to smartphones, now the equivalence of text-form smartphones to graphical user interfaces--the uMotif interface uses a flower-petal visual, with a finger-swipe indicator for responses.

Study shows eCOA/ePRO interface is equivalent to responses from text-based smartphone formats.

uMotif Announces Validated eCOA/ePRO Interface

Between June and August 2022, Tufts CSDD collected and analyzed financial, personnel, and trend data from 18 top public and private contract research organizations (CROs) to inform the industry on the current status of the CRO industry. This information is contained in the November/December 2022 issue of the Tufts CSDD Impact Report, 

Topline insights into the data include the following:

Total spending in the drug development industry on contract clinical services is approaching $50 billion in 2022.

Between 2020 and 2021, the top 10 CROs saw an unprecedented single year growth rate of 17%.

In 2022, the top 10 CROs captured nearly 70% of spending on contract clinical services.

The Impact report also summarized the problem of terms and definitions regarding sourcing strategies by sponsor companies that use a wide variety of outsourcing models to support their portfolios. A collaboration between Tufts CSDD, ICON, and a consortium of pharmaceutical companies proposed a new framework to differentiate sourcing models. A chart of which is included in the Impact report, along with a fuller discussion in this article appearing on the 

Tufts CSDD report based on data collected and analyzed financial, personnel, and trend data from 18 top public and private CROs.

Latest Tufts CSDD Impact Report Focuses on CROs

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.