Due to their low occurrence and specific regulatory requirements, rare diseases present unique challenges in treatment development, evidence generation, and commercialization. We have a team with the knowledge and experience to support your decision making at all stages of product development.
Developing and launching new rare disease therapies can pose several unique challenges. We provide the required expertise to help you navigate these barriers.
Our experience in rare diseases spans all stages, from product development, market assessment, and asset prioritization through to real-world evidence generation and consulting support for commercialization. Our global, multidisciplinary team of experts takes a pragmatic, modular, and flexible approach to engagements. We’re proud to have conducted more 600 studies on rare diseases around the world, providing a strong knowledge base for determining study feasibility and optimizing design and execution.
We follow a data-first strategy, combining diverse data sources for deeper insights. Our first step is to determine what existing data is available, which can be very limited for rare diseases. We can also create custom primary studies, databases, or registries using direct-to-patient or site-based methods. Alternatively, we can adopt a hybrid approach, combining new and existing data. Ultimately, our goal is to access or generate the best achievable data for the situation.
To fully understand how decisions are made, you must understand the person behind the patient, caregiver, or healthcare provider. This is particularly true for rare disease research, where sufferers can be young, medical knowledge is often sparse, and the treatment journey is typically quite complex. Our team takes special care to ensure research is conducted sensitively, with empathy and without judgment.
Identifying the right stakeholders is key to understanding how a rare disease is diagnosed and treated. We have a proven track record of accessing patients, patient organizations and advocacy groups, caregivers, key opinion leaders, clinicians, and payers. We tailor our recruitment strategies to the needs of the indication to provide the insight and evidence you need for strategic decision-making.
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