DPHARM Follow the Money: Investor Insights on Technology Investment in Clinical Trials

The DPHARM conference brought together a distinguished panel to discuss the critical role of technology investment in clinical trials. Moderated by Nick Slack, MBE, Executive in Residence at Warburg Pincus, the panel included Andrea Jackson from Northpond Ventures, Ryan Jones from Florence Healthcare, Andy Lee from Merck, Bari Kowal from Regeneron, and Bryan Spielman from Advarra. The discussion centered on aligning the interests of various stakeholders in the clinical trial innovation process to determine the best areas for investment, focusing on the industry’s growth, challenges, and potential for technological advancements.

Industry Overview: Growth Amidst Challenges

Nick Slack opened the discussion by highlighting the industry’s growth over the past two decades, noting a 71% increase in trial starts and a 4% annual growth in new R&D investments. Substantial investments in new platforms, molecules, and R&D initiatives largely drive this growth. However, Slack also pointed out significant challenges that accompany this growth. For instance, the complexity of clinical trials has increased, with a 20% rise in the number of endpoints being studied. This complexity often results in more amendments, which have risen by 60%, and timelines that are frequently missed by about 15%.

Slack also highlighted a concerning trend: a sharp decrease in investigator participation. Since 2018, there has been an 18% drop in the number of investigators involved in clinical trials, with only 37% conducting more than one trial. This decline is particularly alarming given that investigators are considered the lifeblood of the clinical trial process. Additionally, oncology’s largest therapeutic area has seen a 70% increase in trial starts since 2010, growing at a rate of 5% year on year. Despite this growth, the industry faces a crisis in oncology trials, exacerbated by nursing shortages and capacity constraints. Academic medical centers, which treat 20% of the cancer population, see 80% of the trials, creating a significant imbalance.

Navigating Headwinds with Strategic Planning

Andy Lee from Merck acknowledged the industry’s forward momentum but emphasized the need to address the headwinds that come with it. He noted that while trial starts and complexity are rising, investigator participation is declining, posing a significant challenge. Lee explained that the complexity of trials often stems from the need to refine patient populations for newer therapies, leading to higher response rates. For example, newer therapies require more inclusion and exclusion criteria to identify the right patients who will respond to the treatment, resulting in more complex trial designs.

Lee advocated for a more thoughtful approach to trial design, focusing on reducing unnecessary complexity and ensuring new therapies target the right patients in the right geographies. He mentioned that the industry often sees a proliferation of “me-too” drugs, where multiple companies develop similar molecules, leading to increased competition and higher standards of care. This situation necessitates running studies against the current standard of care, which has evolved and become more stringent. Lee suggested that limiting the number of new potential entities that are essentially “me-too” drugs could help reduce complexity and improve trial efficiency. He also highlighted the importance of careful planning and strategic thinking to mitigate the industry’s headwinds.

Investing in Durable Ideas

Andrea Jackson from Northpond Ventures discussed the role of investors as ecosystem builders, emphasizing the importance of finding durable ideas that can withstand market fluctuations and deliver tangible results. She explained that on the venture side, the goal is to invest in companies that can address the industry’s longstanding challenges. Jackson noted that while the current system is functional, there is significant room for improvement. According to Jackson, one of the biggest challenges is identifying ideas that can go from trial design to the completion of a trial. She mentioned that very few companies pitch ideas focused on helping large pharmaceutical companies like Merck complete their trials faster to bring drugs to market more quickly. Instead, many companies propose nuanced or esoteric solutions that may not be durable in the long run.

Jackson emphasized matching financial and human capital to the right ideas. She explained that investing is about providing financial resources and supporting the human capital behind these ideas. The goal is to find ideas that can be executed effectively and lead to meaningful trial efficiency and effectiveness advancements. Jackson acknowledged that this process involves a degree of imagination and hope, but it also requires careful execution to ensure that the ideas are durable and can make a real impact. She highlighted the need for investors to sift through numerous proposals to identify those that offer the most promise for long-term success.

Leveraging Technology and Data

Bari Kowal from Regeneron expressed optimism about the potential for technology and data to transform clinical trials. She pointed out that while the fundamentals of trial operations haven’t changed much over the past 30 years, the availability of new technologies and global reach offers unprecedented opportunities. Kowal emphasized that the industry now has access to data and technology that was unavailable a decade or two ago, which can significantly enhance trial efficiency. For example, data analytics and machine learning can improve trial design, patient recruitment, and data collection. However, she noted that no single company can be an expert in all areas, and it is crucial to have connectivity between different solutions to streamline the trial process.

Kowal also mentioned that the industry needs to capitalize on the changes in ways of working and global reach. She explained that the ability to conduct trials globally and access diverse patient populations can lead to more robust and generalizable results. However, this requires careful planning and coordination to ensure trials are conducted efficiently and ethically. Kowal stressed the importance of connectivity between the various components of the trial process. She explained that running a clinical trial involves numerous interconnected areas, each of which can benefit from technological innovations. The industry can achieve faster and more streamlined operations by focusing on these areas.

Building an Ecosystem

Bryan Spielman from Advarra highlighted the importance of building an ecosystem that includes sponsors and sites. He noted that while technology initiatives like TransCelerate have been useful, they often lack sufficient participation from academic medical centers and other key stakeholders. Spielman explained that TransCelerate’s initiatives, such as the Shared Investigator Platform (SIP), are developed from a sponsor perspective and may not fully address the needs of sites. He emphasized the need for a platform approach that facilitates connectivity and allows for integrating innovative solutions from various companies.

Spielman mentioned that Advarra has established a site-sponsor consortium to understand better the challenges faced by both sponsors and sites. This consortium aims to foster collaboration and develop solutions that address the needs of all stakeholders. He drew a parallel to Salesforce, a company that has successfully built an ecosystem in the enterprise space. Spielman explained that Salesforce’s ecosystem allows other companies to build their business models around its platform, creating a collaborative environment that drives innovation. He suggested that the clinical trial industry could benefit from a similar approach, where a central platform facilitates connectivity and allows for the integration of various solutions.

Enabling Trial Sites

Ryan Jones from Florence Healthcare emphasized enabling trial sites to do their best work. He pointed out that many clinical trial challenges are site-related, and improving site capabilities can significantly enhance trial efficiency. Jones explained that Florence Healthcare’s mission is to support trial sites, and this focus has helped align the company’s efforts with the needs of the industry. He mentioned that the preamble slides presented by Hassan showed that many of the issues in clinical trials are related to site operations rather than problems within sponsors or CROs. Jones argued that in 2023, trial site enablement is the most critical mission for the industry.

By providing sites with the tools and support they need, the industry can create the capacity to address the growing complexity of trials. Jones also shared advice from an early mentor who emphasized the importance of not doing anything unnatural. He explained that companies sometimes push for extreme measures that are not broadly accepted or fail to consider the necessary change management processes in a rush to innovate. Jones stressed the need for thoughtful and realistic approaches to innovation, considering the practical challenges trial sites face. He highlighted the importance of aligning the efforts of investors, customers, and the internal team to enable trial sites.

The panel agreed on collaboration among investors, sponsors, and sites to address the industry’s challenges. By focusing on durable ideas, leveraging technology and data, and building a connected ecosystem, the clinical trial industry can navigate its headwinds and continue its momentum. The insights from DPHARM 2023 underscore the importance of strategic investment and collaboration in driving innovation and efficiency in clinical trials. As the industry evolves, aligning stakeholder interests and the thoughtful application of technology will be crucial in overcoming persistent challenges and achieving better outcomes for patients worldwide.