This has been a pivotal year for UCB as we deliver on our commitment to bring transformational solutions to patients with the launch of three new medicines in the U.S. Making these solutions available for patients requires our team to draw on our robust scientific expertise as well as our understanding of disease biology and our legacy of deep understanding of patients to provide differentiated solutions. Our continued work embodies what UCB stands for – we are inspired by patients and driven by science. 

Part of our ability to deliver value for patients, the healthcare system, and society at large comes from the connection and understanding of how innovation can be delivered and transform the lives of people living with severe diseases. At UCB, we continue to invest around 25% of our revenue in the research and development of new drugs and treatments, reflecting our ongoing commitment to discovery and innovation. Recently, Mark Morgan, President and Head of U.S. Operations and Payer Value Strategy, joined Roger Palframan, Head of U.S. Research, to unpack UCB’s journey across the discovery-development-delivery continuum, and to discuss how UCB works to deliver on innovation, now, and how we’ll continue in the future. 

Harnessing Innovation with Patients at the Center

Palframan: UCB reached several incredible milestones this year. What stands out for you most in this pivotal year?

Morgan: Looking back at 2023, it’s been an unprecedented year for innovation at UCB. We had three U.S. FDA approvals in rare neurological diseases and immunological diseases, nearly doubling the solutions UCB has available in the U.S. We also have a number of active Phase 3 development programs across immunology, neurology, and rare disease, demonstrating our ongoing investment in R&D and our unwavering focus on making a positive impact for patients. 

Palframan: It’s also important to note our latest wave of innovation is not only built from our strong heritage in neurology and immunology, but also from capabilities and knowledge gained from our global collaborators who are outside of organizational, sector, and geographical boundaries. There is nothing better than seeing innovation, wherever it comes from, translate into real benefits for people with severe disease. One way we do this is by building on our expertise and deep understanding of human biology. Our experience in immunology opened the discovery of a novel pathway and mechanism of action to target psoriasis and psoriatic arthritis and a number of other immunologic-mediated diseases more effectively and rapidly. We’ve also expanded our potential impact and capabilities through acquisitions like Element Genomics and Ra Pharmaceuticals  bringing to life the promise of functional genomics and macrocycles, and ultimately reaching the approval of a new solution for people impacted by rare disease where unmet needs still exist.  There are countless other examples of how the innovative work we do each day is really making positive changes in the daily lives of patients. 

These advancements go beyond science and get to the heart of what we aspire to do: discover, develop, and deliver highly differentiated medicines treating, modifying and ultimately, curing diseases. 

We have been so inspired by the people we serve and how we’ve been able to improve their lives and the lives of their families through our medicines and support. This impact fuels our passion and determination to do even more.

Morgan: How does UCB approach research and development? What sets UCB’s R&D work apart from others?

Palframan: We are pushing scientific boundaries, seeking to solve tough challenges to create tangible improvements in care. We’re investing in our pipeline for the long term, and we are prepared and well positioned to make bold bets. 

Our Global Discovery Platform consists of “hubs” around the world to tap into the best expertise and capabilities and maximize the benefits of proximity. Specializing in neuromuscular biology, Boston-Cambridge is one example of a “hub” connecting us to a diverse deep talent pool and leading research hospitals, biotech, and universities. The research conducted in this location has deepened our ability to define patient populations and identify the best drug targets for disease states. 

Additionally, technology and digital innovation, such as artificial intelligence (AI), play a huge role in how we accelerate discoveries and improve the pain points in the patient journey. We have collaborated with Microsoft to enhance our drug discovery and development capabilities, and we are also working with Stanford University to leverage data science, which combines clinical, real-world, omics, and other data sets, to determine which patients will respond best to a new medicine. Delivering on our innovation is key to improving health and people’s lives. 

Morgan: Let’s talk more about how UCB applies patient-centricity to its approach to understanding diseases and therapy modalities. 

Palframan: At UCB, listening to and engaging with people impacted by severe diseases is a critical component of our R&D process enabling our team to develop medicines to specifically meet their needs. This is a testament to our deep scientific expertise, R&D, and our corporate commitment to delivering moments that matter via innovative science and comprehensive support to people impacted by severe diseases. We’ve been proactive about incorporating the patient perspective, and that even culminated in UCB developing its own value assessment framework and releasing UCB’s Principles for Value Assessment in the U.S. 

When we begin a new project, one of the first steps we take is to connect with people living with the conditions we’re researching right from the very beginning to inform the design of our drug discovery research and find innovative ways to address diseases. Connecting our scientists and researchers with patients helps us be more successful uncovering new targets in clinically meaningful ways and can positively impact their lives. We work with people with severe diseases and advocacy organizations to listen firsthand to their experiences and embed their perspectives in every action we take so we understand areas of greatest unmet need. 

A recent example of this is partnering with the myasthenia gravis (MG) community to listen and elevate the voices of those impacted. While we understand clinical endpoints as we develop medicines remain critical, hearing from patients and their experiences puts things in perspective for us as researchers. Perhaps the thing that matters most to an MG patient is the ability to climb the stairs or to stand to cook without needing to sit down, or step away to rest. 

The Continuum of Innovation to Human Impact 

Palframan: I know you and the team are particularly attuned to how access factors into the R&D process. How does this translate into our strategy for delivering medicines?

Morgan: Medical innovation only matters if people can access their needed treatment—bottom line. At a health ecosystem level, we remain dedicated to the continued evolution of a public policy environment not only recognizing and rewarding innovation and encouraging value-based care, but also one promoting affordable access to medicines for patients. At a UCB level, we have developed comprehensive and personalized patient support services to address specific needs in different patient populations. Last year, our patient support programs helped more than 95,000 patients to navigate and better manage the complexities of their disease, including through financial assistance support, tools, and resources.

Through innovation and partnership, we can continue advancing the science to make a real difference in the future and address those unmet needs. One area of increased focus is health equity and addressing the existing patient access and affordability disparities. We’re piloting new social business approaches that stand up to health disparities, leveraging pilots to incubate innovative approaches, and engage with traditionally underserved communities to offer solutions, especially those solutions positively impacting access. We are committed to understanding the lives of people living with severe diseases to ensure our medicines work well with their lifestyles and fit their needs.

Morgan: It’s important for stakeholders who can impact diagnosis, availability, treatment affordability, care plans—basically every aspect of the patient journey—to have a vivid understanding of that journey. Access depends on it. Every stakeholder within the value chain has a role to play in ensuring access. It is our responsibility as innovators to engage payers and decisionmakers early – where possible – to find ways to ensure that our innovative treatments, once discovered and developed, are delivered in a way that is accessible for the right patients and sustainable for people impacted by severe diseases, for UCB, and for society. 

To that point, innovation and breakthroughs take so much time and commitment. The work UCB is undertaking today will change lives both near-term and 5, 10, and 20 years from now. Maintaining investment in research and development, like our commitment to our research hubs in Boston, Raleigh and Seattle with nearly 2,500 UCB employees dedicated to our R&D work, now is critical to advancing medicines and solutions of the future. Policies, like some provisions within the Inflation Reduction Act, which may have the unintended consequence of stifling innovation, could put future innovation at risk throughout the industry. 

Palframan: That’s why it’s so important to involve people living with these diseases early in the R&D process so we can better address the gaps and opportunities through our research. 

Innovating for the Future

Morgan: How are we focused on building a sustainable healthcare system and what’s next for us?

Palframan: It doesn’t stop in 2023 – UCB is poised to bring more new options to people living with severe diseases in the coming year in the U.S. and across the globe. We’re progressing work in rheumatology, hidradenitis suppurativa, lupus, and ultra-rare areas such as mitochondrial disease, while conducting research in gene therapy, targeted protein degradation, and more. Through our deep understanding of disease biology, we can continue to develop differentiated medicines and solutions for specific patient populations. We are taking tangible steps to move from symptomatic treatment to disease modification, and possibly towards a curative approach for certain conditions. Using a gene therapy approach, our teams at UCB are working together — across wet labs and computer labs — to discover innovative ways to address genomic instability and better understand diseases such as Huntington’s, myotonic dystrophy, and ataxias.

Morgan: I’m amazed at what we’re working to accomplish, especially for people living with severe diseases. We make every decision with an eye to how it affects the people who put their trust in us, and we strive to earn this trust every day by honoring our commitment to deliver moments that matter. This means we continue to advocate for policies focusing on continued innovation and affordable access for patients. Our work across the continuum of drug discovery to development and delivery to have a positive impact on the lives of people living with severe diseases, now and into the future, continues.