Society’s ties

Delivering true value for people living with rare diseases
comes from partnership and innovation

We need to get closer to patients’ needs to achieve the right solutions for them.
When we talk about patient value in rare disease what do we mean? The long-term question can’t be about how we get every single patient to try one particular medicine in a broad-sweeping approach.

That won’t deliver the right answers. As an industry, we need to ask how we can be more precise in the way we engage with these communities to meet their needs. What insights can we learn, what are the lived experiences patients face every day, what are the improvements that could tangibly improve their lives. It’s about understanding that each person living with the disease is an individual and our job is to help them get to the right medicine or solution, quickly.

Achieving true value for the community with our solutions is only possible with a strong foundation of partnership. Across the pharma and healthcare sector, it is common to talk about patient-centricity and partnership – indeed, this is a basis for all our interactions. To generate meaningful change, however, this needs to be more than just words on paper.

This is especially true in rare diseases where, in some cases, levels of support, funding and research lag behind better understood medical conditions.

Partnering with patients, advocacy groups and physicians over the long term enables us to get closer to patient needs, so that we can develop the right technologies, medicines and solutions.

It helps our scientists develop more precise, targeted therapies and it enables us to design clinical studies and generate evidence with end points that we know will be relevant to these communities.

For this reason, gaining knowledge and insight from genuine collaboration is one of the most important steps on our development journey.

There are many tools we, as an industry, can use to amplify insights – supporting patients and facilitating improved knowledge and understanding among clinicians, payers, policymakers and the public at large.

We’ve pioneered this approach across multiple projects, for example, a recent co-authored and peer-reviewed paper we worked on in partnership with Nancy Law – a former President of the Myasthenia Gravis Foundation (MGFA) and an expert patient – focused on the lived experience of myasthenia gravis (MG), where the gaps are and where we might be able to improve care for the MG community.

Everything we learned from Nancy and working with other groups that also have a great need for medicine and support, motivates us and should motivate the industry to do its best on every level for them. Our confidence in the importance of these types of initiatives is reinforced as we see the community citing this paper as being transformational in conveying the lived experience of MG.

Other groups that also have a great need for support and medicines are those living with rare genetic conditions – many of which manifest themselves in early childhood. Take, for example, thymidine kinase 2 deficiency (TK2d).

This is an ultra-rare, debilitating and life-threatening genetic mitochondrial disorder that causes progressive and severe muscle weakness, predominantly in a paediatric population. Here, the impact of success for patients can be measured in very tangible human terms – a reminder we keep at the forefront of our minds all the time to ensure that, in everything we do, we’re striving to help patients and their families.

By working with these communities, engaging with them from the beginning of research, through every stage from trials to outcomes, we can ensure we’re taking the right steps to help improve their diagnosis rates and education around the ultra-rare diseases they’re living with. In parallel we will be striving to deliver medicines that we hope will make a significant impact on their lives and those of their families.

Providing a space for an exchange of ideas and giving the medical community an avenue for conversations is important to change what the future looks like in rare disease. At UCB we’ve listened to the community and founded the Rare Disease Connect Network (RDCN), a forum for this very purpose, which will hopefully help lead to better care for myasthenia gravis and other rare neurological conditions. We are committed to being a partner in the conversation.

We are also committed to investing in digital technology and innovation in rare disease. We know there’s no magic moment to ‘turn on’ the future, rather it’s a trial-and-error evolution to meaningful results. There is great promise for digital as a more integrated part of care, taking the voice of the physician and the needs of the patient and connecting them outside of the clinic.

We’re investing in technologies that, in the future, could produce clinically meaningful algorithms to help detect and even predict the future health status of patients using a smartphone camera and microphone to observe their face and listen to their voice. The possible benefits of helping patients predict oncoming symptoms earlier and taking appropriate actions could be substantial and potentially lead to tangible improvements in care.

Until now, data to support these tools has eluded us. But with the rapid developments in smartphone and wearable technology seen in the last decade and combined with our dialogue with patients to understand the role technology plays in their lives, we can start to harness innovations to potentially transform outcomes and experiences for patients.

We often hear about ‘future-proofing the industry’ – perhaps the better discussion is about how we bring industry into line with the needs of society as a whole. It is vital that our goals and our approach are relevant to the community and the people we serve; hardwiring an innovation and collaboration mentality can yield a multitude of benefits.

We aspire to lead and be recognised as genuine partners in each of the communities we work with; as such, we attract individuals who are equally committed to this approach, and who are not afraid to embrace new and innovative activities.

It’s also imperative we illustrate the value of our innovations. The more articulately we can talk about the value we deliver into the marketplace, the more sustainable our business model will be. By demonstrating to our partners – patients, clinicians, researchers and payers – the value of our efforts, we help grow their understanding and confidence in what this partnership can potentially achieve.

Through our proximity to the communities, listening to their lived experience, participating in open learning, working on digital innovations and articulating clear translations of value, we can be best placed to deliver solutions to people living with rare diseases.