Citeline Elevate Spring 2025: Brainpower in Boston

While many in the industry were eagerly anticipating the evening’s annual Citeline Awards in Boston, a group of highly engaged pharma professionals attended Citeline Elevate Spring 2025, Pharma Futurology: Clinical and Technical Innovation.

Claire Riches, Citeline Vice President of Clinical Solutions, kicked off the event, emphasizing the futurology theme. Futurology — the scientific field that explores, imagines, and evaluates possible, probable, and desirable futures through systematic research methods — aptly describes the day’s topics, from artificial intelligence (AI) to the changing pharmaceutical landscape.

Riches acknowledged that “the industry is facing a whole lot of headwinds.” She compared today’s environment as similar to where the industry was five years ago with COVID, with researchers tending to be reactive rather than proactive. “I think everything that we’re facing now will cause us to pivot again.”

On a positive note, Riches added that the pharmaceutical industry has more options than ever before, such as real-world data (RWD), real-world evidence (RWE), and AI.

A rapid-fire recap of the Pharma R&D Review

Dan Chancellor, Vice President of Thought Leadership for Norstella (Citeline’s parent company), shared both statistics from and insights on Citeline’s Pharma R&D Annual Review 2025. With 24,000 drugs in active development today, he said this reflects consistent growth. In fact, the industry has shown 145% growth since 2010. Chancellor noted that “it’s not just the numbers of drugs, but the universe” as a whole that is evolving.

“What we’re seeing is more fragmentation, more democratization,” Chancellor said. “In a word, biotechs are getting bigger. Biotechs are more important than ever before.”

Chancellor also touched on research pipeline churn. “We see rare diseases as an area of outsized growth,” he pointed out. However, about 40% of new drug development is in oncology.

Attendees were privy to a first showing of data focusing on R&D engines. “It’s very clear to see that China has grown to be a powerhouse in R&D, rivaling the US,” Chancellor said. He predicted there may be an inflection point where China actually overtakes the US and that South Korea will likely be the next hot spot for research. Rounding out the top five countries with new-to-pipeline drugs are the UK and Switzerland.

In terms of leading drug discovery companies, Chancellor said China also is making its mark, with China-based Jiangsu Hengrui Pharmaceuticals topping the list. CSPC Pharmaceutical and Sino Biopharmaceuticals, also based in China, made the top 20 list.

While overall productivity is declining, Chancellor said a certain amount of churn is healthy. “An abundance of R&D is not necessarily a good thing,” he cautioned. “Perhaps we should be asking ourselves how we can do more with less.”

The industry must deal with long-standing threats to the R&D paradigm, including development costs, attrition rates, clinical timelines, and patient availability.

Data — the name of the game

Liz McCann

Liz McCann, Citeline VP for Strategic Initiatives, took a quick dive into “Infusing AI and RWD into Clinical Planning, Strategy, and Recruitment.” As she outlined the advantages of Citeline’s many clinical solutions, she noted: “Tools alone don’t create value, they enable value.”

For example, she said, “AI is a component of the larger picture.” Any solution or AI tool is only as good as the data input. “Data quality is nonnegotiable,” she stated. It’s not only about cleanliness, it’s about completeness. And it’s not just about volume; it’s about structure and relevance. McCann said that powerful algorithms, such as those used in Trialtrove⁺ and Sitetrove⁺, start with trusted and reliable data.

“You need that data that’s not only accurate but available in the moment and actionable,” she said, referring to Citeline PatientMatch and Citeline SmartSolutions. McCann said Citeline PatientMatch flips the patient recruitment paradigm. “We’re no longer looking for patients for the trial. The trial is finding the patients.”

McCann explained that precision is crucial in clinical planning. Sponsors need a sharper, faster, more targeted way to gather insights, particularly in light of more restrictive inclusion/exclusion (I/E) criteria.

What sets Citeline apart, she said, is that it integrates proprietary performance data with RWD from its parent company, Norstella. “Citeline serves the building blocks so you can deliver on your clinical and regulatory strategy.”

She then introduced attendees to Ella, Citeline’s intuitive chatbot tool. Drawing upon Citeline’s manually curated Trialtrove and Sitetrove datasets, this AI chat assistant does more than create searches; it guides search strategies.

Throughout the event, audience members peppered the presenters with thought-provoking questions. One attendee asked about the ROI of investing in such solutions for small biotechs lacking the funding of their large-pharma counterparts. To date, he said patient recruitment has amounted to “just rolling the dice.”

Riches jumped into the discussion, answering that Citeline offers a pay-per-performance recruitment model. She noted that purchasing the data outright can be quite costly. “We’re not giving you the data,” she said. “We’re giving you snapshots of the data based on an algorithm.”

Riches jumped into the discussion, answering that Citeline offers a pay-per-performance recruitment model. She noted that purchasing RWD data outright can be quite costly. “We’re not giving you the data,” she said. “We’re giving you insights and snapshots of the data based on an algorithm to identify protocol-specific patients which is a much more budget-friendly option.”

McCann announced that a new patient cohort solution will launch this summer. Using I/E criteria, the tool — powered by natural language processing and more — will auto-identify the patient cohort, cohort count, and provide rich analytics. “And we didn’t stop there,” she said. The cohort output goes through a human validation process by content specialists. The result is a protocol-ready cohort. “This isn’t a static query engine,” she said with pride.

Kerry Culm, Dan Chancellor

Small biotech embraces technology

Chancellor took to the podium again, this time joined by Kerry Culm, Chief Development Officer for ModeX Therapeutics,  which, by the way, later won Most Successful Early Phase Research (preclinical & Phase I) – Oncology in the Citeline Awards.

As a small biotech, with fewer than 70 people, Culm said that accessing critical data — which she referred to as “currency” — is crucial to its survival. ModeX relies on Citeline’s Trialtrove and Sitetrove solutions for that data. She and her colleagues are well aware of the data that exist, they just have not mined it all yet.

When asked how ModeX uses RWD and AI, Culm said, “On the science side we most definitely use that in our lab space.” She cited personalized medicine as an area that greatly benefits from both.

“I am personally really excited in my role at ModeX,” she said, describing the “sweet spot” when it comes to data. “We get to peel away the layers” and see where the drug is effective.

Of the secret to ModeX’s success, Culm said, “I like to think it’s the innovation and the people, and we pulled in the right partners.” She credited her predecessors: “We are standing on the shoulders of those who came before us.”

She also attributes her company’s success to the fact that not only have they “cracked the code” to develop single molecules for combating complex diseases, they can manufacture them.

While being a small biotech sometimes has drawbacks, it also brings with it advantages such as agility. Culm said that for big pharma, it often can take nine months to get from study design to the clinic. “We did it in four.”

When asked by Chancellor, “Are you blazing a trail here that others can follow?” Culm responded: “I really hope we are. … The sky’s the limit.”

Ray Huml, Claire Riches

Rethinking rare disease

Riches also returned to the podium for a fireside chat with Raymond Huml, Vice President of Rare Disease Strategy for Sciensus, with whom she previously worked at Syneos. “We kind of need to treat every study as a rare disease study,” he said.

A veterinarian by training, Huml said what drives his passion around rare diseases is that his son and daughter both live with facioscapulohumeral muscular dystrophy (FSHD). Overall, most rare disease drug development has occurred in the past seven to eight years, he said.

Asked what his takeaways were from the World Orphan Drug Congress, Huml said, “I see the patient voice being permeated now in ways I’ve never seen before.” He also said a great deal of concern was expressed regarding funding cuts, but the jury is out on that for now in terms of impact. “It’s still unraveling. I don’t think we’ve had a chance to digest it. … It’s a bit of an unknown.” He also suggested an overhaul of the US Orphan Drug Act, which provides incentives for pharmaceutical companies to develop rare disease drugs.

Riches broached the subject of how difficult it is to recruit rare disease patients, comparing it to looking for a unicorn, that needle-in-a-haystack patient.

Huml said, “It’s one thing to ID a patient, it’s another thing to get them to participate.”

Of course, a patient must first be diagnosed in order to participate in a relevant clinical trial. This is an area where AI is picking up, providing more accurate and faster diagnoses. “To me,” Huml said, “not knowing what your disease is, is not a good place to be mentally.” A case in point is Citeline’s own Haley Quinn, who finally received her rare disease diagnosis after 26 years.

“The data and AI can almost give you a third-party perspective,” Huml said.

Both agreed that sponsors should start with patient voice, building the protocol with the patient in mind. “RWD gives us a lot more visibility into that patient profile that goes beyond the biomarker,” Riches said.

Riches asked about drawing conclusions from larger populations for rare disease predictions. For example, she raised the possibility of using social listening and epidemiology data, then matching  that dataset with other rare diseases with successful drug development. Could a synthetic dataset be created? She suggested that rare disease researchers be innovative and take more risks.

Huml cautioned that rare disease drug developers must diversify risk. “At the end of the day, it’s about managing risk,” he said.

Riches said that once a rare disease patient — or any patient, for that matter — is randomized, the key is to keep them in the trial. As an industry, she said, “We focus on enrolling and then step back a bit. What can we learn from the rare disease space?”

Huml applauded advocacy groups, whose members are incentivized to learn as much as they can about the specific disease. “Everything I’m hearing from patients is ‘Can we get some feedback?’” In Europe, he noted, plain language summaries (PLS) are required, but in the US they are merely recommended.

He said sponsors should not hesitate to report results of a failed trial. “It’s OK if the trial wasn’t a success. You still advanced science. A failed trial is not a worthless trial. It raises a lot of awareness. It gives patients what they want, and that is hope.”

L-R: Sarah Karlin-Smith, Shalome Sine, Greg Manning, Francesca Barone

Panelists sound off on innovation, patient-centricity

The conversation continued with a lively panel discussion on “How Innovations Are Impacting Clinical Trials Today,” moderated by Sarah Karlin-Smith, senior writer for Pink Sheet. She was joined by Shalome Sine, Senior Manager and Quantitative Insights Specialist at the Center for Information and Study on Clinical Research (CISCRP); Greg Manning, Head of Centralized Network Operations at Atlas Clinical Research, a site network; and Francesca Barone, Chief Scientific Operator at Candel Therapeutics, which that night became a Citeline Awards winner for clinical trial result of the year, for its Phase III trial of CAN-2409.

Barone gave one example of Candel’s innovative approach, saying it believes in small trials, especially in early phases. “It’s quite controversial,” she said, citing the benefit of stopping programs that are not working and refining them.

Karlin-Smith asked the panelists how their organizations use AI. Manning responded, “Sites can use it in a lot of ways. They use it for recruitment.” He said sites have trialed using AI callers on large groups of patients for basic trials, comparable to human staff, adding, “so that’s very promising.”

Manning said AI also is used to streamline data entry and forms such as source documents and contracts in start-up operations. “I think there’s a lot more usage in the recruitment space.”

From the sponsor’s perspective, Barone said, “We use it in a very different way. We don’t use it to interface with patients. We’re very traditional in that way.” Candel does, however, use AI to develop biomarkers. “For us it’s been extremely successful … and extremely practical.”

As with any discussion of AI comes the caveat of mitigating risk. Manning said it is important to choose an AI solution that guards protected health information (PHI).

Providing the patient angle, Sine said participants’ main concerns are privacy and accuracy. She referred to a CISCRP survey on how familiar patients are with AI. “It’s a very mixed bag,” she said. When patients were asked if they thought AI use in clinical trials would increase or decrease errors, their responses were split down the middle. However, when patients were asked whether they would want to know if their medical data were being put through AI, about 90% responded yes. “I don’t think the trust is quite there yet,” she said, “because people don’t understand enough about it.”

Themes that surfaced in CISCRP’S Perceptions and Insights Study, conducted every two years, include barriers to participation such as logistics (location of clinical trials, school/work commitments), safety, and clinical risks/benefits. A whopping 83% of patients are concerned about treatment side effects.

Those concerns can be magnified by the paperwork required of participants. Barone, like Huml, is the parent of a child with a medical condition. Her daughter has a form of neuropathy, and she had to sign a 50-page consent form to be part of a research project. “It was very scary,” she said, and even scarier for her husband, who lacks her scientific background.

When it comes to technology commonly used in decentralized clinical trials (DCTs), Sine said patients “are generally comfortable with these technologies — computers, wearable devices, especially if it means they’ll have to go into the clinic less.” Manning added that patients prefer to use apps on their own devices as opposed to new technology.

With Pink Sheet’s focus on the regulatory side of pharma, Karlin-Smith asked the panelists for insights on how regulators are adapting to the use of AI. Coinciding with Citeline Evaluate, on May 8 the US Food and Drug Administration (FDA) announced that AI will be used in scientific reviews of medical products across the agency by the end of June.

“I think in general the regulators need to keep up with this,” Barone said. Manning agreed: “You have to have a defined approach. … There are certain lines we’re not crossing yet. And we’ll wait for guidance on that.”

Despite rollbacks in the US on diversity, equity, and inclusion (DEI), Karlin-Smith asked the panelists how their organizations are addressing clinical trial diversity. Interestingly, Manning said Atlas discovered that some patients choose white/Caucasian as their race of prevalence because it is easier to just check that box. That’s why sites should pay close attention to patients during the screening process, he advised.

Manning emphasized the importance of patients being an advocate for their community. One patient, whose relative died in the infamous US Public Health Service’s Tuskegee experiment, actually became a clinical trial participant herself. Manning said it was the stipend that helped her overcome her reluctance to participate. Not only did she participate, she remained in the two-year trial and returned for a second trial.

At the site level, Manning said that sites sometimes front-load diversity patients. If the sponsor does not provide diversity goals, Atlas will develop its own.

To encourage participation from diverse populations, Sine said sponsors must build trust. “Patients want to see themselves reflected in other patients that are participating. … Also consider who you hire at your sites.”

In addressing an audience question regarding mental health and trial participation, Sine cited a recurring comment from patients: “I like it when my doctor sees me as a whole person rather than as just the disease.”