Two Perspectives, One Approach: Developing Solutions to Support Patients with Rare Diseases

Jennifer Millian

Today marks Rare Disease Day. Recognized annually on the last day of February, it is an opportunity to raise awareness and push for change for the 300 million people living with a rare disease worldwide. At its heart, Rare Disease Day is about advocacy. So together, we join our colleagues at Insmed and the global community of patients, researchers, healthcare providers, and caregivers – all advocates in their own right – to bring attention to this day and the community behind it.

You might be wondering why the Executive Director of Patient Advocacy & Professional Society Engagement and the Vice President of Early-Stage Programs are writing a LinkedIn article together. On the surface, our two functions may appear distant, but in reality, our work is very much intertwined. That’s because at Insmed, engaging with and advocating for patients starts at the very beginning – during early-stage research and development.

Understanding the needs and desires of patients is integral to our ability to develop solutions that can potentially change their lives in a meaningful way. By aligning our two functions, we’re putting the lived experiences of patients and caregivers at the heart of drug development. Together, we believe this is the future of patient advocacy – infusing it into every stage of the continuum.

How do we achieve this? Our philosophy is simple. Start programs with science AND the patient at the forefront, do what’s right for the patient even when it isn’t easy, and collaborate with colleagues across departments to overcome challenges. In doing so, we ensure that patients are integrated into all aspects of our R&D process. Here’s how this works at Insmed:

Start programs with science AND the patient at the forefront.

Talking directly with patients and their families in an open and measurable way early in the R&D process is crucial to ensuring that our science is not only clinically meaningful, but also addresses needs as defined by the patient community. While understanding the science and underlying biology of a disease is important, there are certain details about a condition and how it presents that only a patient or caregiver can provide. Leveraging the science of patient engagement, we can incorporate patient reported outcomes (PROs) into our clinical programs that are substantive to those living with the diseases we are trying to treat. For example, in a pediatric movement disorder, a scientist may want to show clinical improvement by way of a validated scale; whereas, at home, a caregiver may notice that in parallel to this clinical improvement, their child went from being unable to brush their teeth independently to being able to do so. Only by speaking to both physicians and families to understand the clinical progression of a disease and what happens on a daily basis in a patient’s home can we fully understand the value of a measure like this is. Building in PROs based on real experiences versus those that rely solely on an outside researcher’s understanding of the disease or what measurements a clinician might track is the power of patient advocacy. It also allows us to develop long-term relationships with patients and give them a seat at the table at each step of the R&D process.

Do what’s right for the patient, even when it isn’t easy.

Patient advocacy in biopharma is two-fold: it is the external commitment to advocate for and support programs that provide help to the patient community, and it is the internal commitment an organization must make to always keep patients as the North Star. Our teams marry these internal and external commitments – ensuring that our advocacy and research go hand-in-hand to stand up for patient populations that have long been overlooked and underserved. For example, at the earliest stages of research, this means that our scientists must make difficult decisions to ensure that validation of a therapeutic candidate’s safety and efficacy outweighs the desire to move it into the clinic as quickly as possible. And outside of the lab, this means that when we believe we can make a difference for even one patient or one family, our leaders come together to find a path forward. We are in lock step in our belief that every patient deserves the utmost support – and we deliver that support through our team’s collaboration each and every day.

Collaborate with colleagues across teams and geographies – it’s the best way to overcome challenges.??

When it comes to working in rare disease, we are often exploring uncharted territory, and through that exploration, challenges can arise that seem impossible to overcome. The best approaches are those that prioritize collaboration and leverage the strengths and insights of our many global colleagues. At Insmed, by working early and often across departments – in our case, patient advocacy and early-stage programs – we create more opportunities to find sustainable solutions that work across the board. This collaborative approach also ensures that we’re bringing multiple perspectives, experiences, and possible solutions forward that can best support the patients we aim to serve.

What we’ve learned from collaborating so closely is that patient advocacy and drug development are inherently tied together. Today, on the rarest day of the year, we’re grateful to work for a company that encourages this connection for us to better serve the patients, families, and advocates who comprise the rare disease community. We encourage our peers to seek out counterparts in other functions and see where these connections take you. It is only by working together that we’ll be able to truly create change and improve patients’ lives.

How do you collaborate? What are your thoughts? We’d love to hear them – comment below!