Authored by:

Erin Finot, Vice President of Immuno-Oncology and Cell & Gene Therapy
Katie Shaw, Senior Director, Patient & Site Networks
Dawn Muscolino, Director, Strategic Site Solutions

Every rare disease journey tells a powerful story, often defined by the unique challenges and circumstances of each patient. To design clinical trials that truly make a difference, we must go beyond statistics and connect on a deeper level. Tools like empathy mapping and a patient-centered perspective allow us to develop studies that prioritize the people behind the data, ensuring their voices are heard and needs are met.

At IQVIA Biotech, we’re reimagining how clinical trials are approached, with strategies that deliver better outcomes for both patients and researchers. In this blog article, we’ll discuss how we’re helping to change the landscape of rare disease research.

Designing patient-centric protocols

Building patient-focused clinical trials starts with protocols that consider the practical realities of participants’ lives. For individuals with a rare disease, trials can be physically demanding and emotionally draining. Reducing this burden is crucial. Flexible scheduling, simplified processes and fewer invasive procedures can go a long way in easing participation.

Making trials accessible with decentralized options

Traditional clinical trials can be overwhelming for many patients, especially those with a rare disease. Traveling to distant trial sites often adds physical, emotional and financial stress, creating barriers that deter participation. Decentralized trials (DCTs) offer a solution by bringing the trial to the patient. Through virtual platforms and home-based options, this approach removes logistical barriers, making participation more convenient and less disruptive.

For rare disease populations, where access to trial sites is often limited, DCTs are a breakthrough. They enhance accessibility and patient retention while ensuring data quality remains uncompromised. By reducing these obstacles, patients can focus on their health and fully engage in the potential life-changing opportunities that groundbreaking research provides.

Faster, safer solutions with adaptive trial designs

In rare disease research, time is a critical factor. Patients can’t afford to wait years for treatments and every day counts when managing their progressive condition. Adaptive trial designs address this urgency by allowing researchers to make adjustments as a trial unfolds. These modifications enable a more dynamic approach to clinical research, prioritizing patient safety and accelerating access to promising therapies.

The role of partnerships

Patient organizations are also central to the rare disease community. Often founded by individuals with personal connections to these conditions, they bring unparalleled insights, empathy and motivation. Their close tie with key opinion leaders (KOLs) and deep understanding of patient needs make them invaluable partners in clinical trial design.

At IQVIA Biotech, we collaborate closely with these organizations to ensure that patient voices are at the heart of every trial. Their involvement shapes trials that are effective, compassionate, and responsive to patient needs.

Beyond design, these partnerships drive impact across the entire drug development lifecycle, from early research to real-world applications. Collaborating closely with patients and stakeholders, to incorporate their perspectives at every stage, helps to improve trial outcomes and foster trust within the communities we aim to serve.

Transforming trials through empathy

Mapping and understanding the patient experience allows us to design clinical trials that align with their unique needs. This empathetic approach addresses the challenges faced by rare disease populations while improving outcomes and enhancing satisfaction.

Incorporating the patient voice at every stage—from design to post-trial care—creates a more patient-centered process, fosters trust among stakeholders and accelerates the development of therapies that address unmet medical needs.

Focusing on both patient and site centricity is essential for advancing healthcare and driving meaningful progress. By adopting this approach, clinical trials can deliver treatments that have a real, lasting impact on patients' lives.

Download our Insight Brief on Patient-Centric Strategies to explore innovative strategies and see how we can support your journey toward better patient outcomes.