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Working alongside the rare disease community to enhance access and elevate the patient voice

Donatello Crocetta, UCB Rare Disease Organization
Posted by
Donatello Crocetta, Global Rare Disease&Rare Medical

On Rare Disease Day - this year falling on the rarest of days, 29 February - we’re proud to show our support for the rare disease community. There are over 300 million people globally living with a rare disease, of which 95% still don’t have access to appropriate treatment options. With up to 90% of these rare conditions considered to be serious or life-threatening, increasing awareness and equitable access is ever more urgent.

Today, and every day, we’re committed to working alongside the rare disease community to make life better for patients and their families.

Striving for early and equitable access
At UCB, we’re committed to increasing equity for those living with a rare disease. Our rare disease philosophy is driven by our dedication to overcome the barriers that prevent early, sustainable and equitable access to health, social care, diagnosis and treatment. In tackling these obstacles, we can support patients in reducing the burdens of their condition, allowing them to live their best lives.

We’re constantly optimizing our approaches, including improving the speed of our clinical trial processes to get new treatments out into the world as soon as possible, as well as close collaboration with patients to learn from their expert insights.

Clinical development and clinical trial design is known to be particularly challenging within rare disease due to the smaller patient populations - it’s why we’re always engaging with networks of patients and advocacy organizations across the globe, helping to determine optimal trial design and support the participation and facilitation of a positive trial experience. Making these strides forward is only possible through partnering with patient and professional communities and Rare Disease Day plays a vital role in advancing these processes. Improving knowledge around rare diseases can help educate and empower patients to join us in driving research that is adapted to their needs and measure what truly matters to them.

A focus on the entire patient population
We’re conscious of the need to take into account Social Determinants of Health (SDoH) which can impact how patients respond to treatment. For example, a recent retrospective observational study using medical and pharmacy claims data, led by UCB and presented at the 2023 American Association of Neuromuscular and Electrodiagnostic Medicine (AANEM) annual meeting1, showed SDoH may be associated with poorer treatment among patients with myasthenia gravis. This type of research suggests there could be value in healthcare providers identifying at-risk communities who could benefit from additional support.

Elevating the patient voice for better outcomes
At UCB, we know from our experience working closely with the community that no two rare disease patients are the same. By listening to and learning from those living with a rare disease, we can better understand the lived experiences of patients and look for treatment options that best meet the individualized needs of patients. There are still significant gaps in care for people living with a rare condition; working in partnership is essential to identifying and addressing them.

Our proximity to the community was never stronger than during our recent Rare Disease Connect in Neurology meeting. We met with patients, patient organizations, pharmacists and other healthcare professionals to drive forward the scientific conversation and build momentum in the rare disease landscape, starting and continuing essential conversations.

This patient-centric approach, guided by lived experiences, is at the heart of our approach to raising awareness and improving outcomes in rare disease. We’re excited to continue fostering strong partnerships to help address unmet needs and make a positive difference to those living with a rare disease.

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