Rare Diseases

Because every single patient is precious

At Parexel, rare disease clinical research is personal – a mindset that is embedded into the fabric of our solutions. Parexel’s extensive experience in rare disease clinical research is exemplified by the hundreds of clinical studies we have completed across therapeutic areas that have contributed to 17 FDA drug approvals. Our customized and specialized solutions are developed to ensure that each customer’s unique study and program needs are met and exceeded.

At Parexel, field-leading experts understand the nuances of rare disease studies. We combine scientific rigor and patient-community engagement to ensure that every patient is identified; has the knowledge needed to decide if the trial is their best treatment option; and is retained throughout the duration of the trial. Given that the majority of rare diseases are genetic in origin, Parexel’s rare disease capability is led and underpinned by expertise in precision medicine and translated into study delivery.

Our integrated, cross-functional model is distinguished by:
  • Lean, nimble delivery team complemented by broader access to organizational expertise through customized innovation team (medical, scientific, regulatory).
  • Customized use of patient innovation tools and tactics to embrace and enhance the patient and caregiver journey.
  • Industry-leading medical, scientific, and regulatory expertise.
  • Operational excellence through deep understanding of the investigational product, the complexities of administering it and nuances of the patient journey.
  • Precision medicine expertise, culture, and mindset translated into a delivery model.
  • Data-driven insights and innovation applied from study design through implementation.
  • Delivery focused on protecting the clinical study endpoint.



Precision medicine

Clinical trials for precision medicines pose unfamiliar challenges: for example, qualifying patients for inclusion due to specialized biomarker or genetic testing required. Parexel has an expansive bench of experts with direct pharmaceutical and biotech experience, including early-stage biomarker strategy development and genomics. This is complemented by a multi-disciplinary approach regarding our genomic real-world evidence strategy. To ensure clinical study operational excellence, our Clinical Trial Supply & Logistics services team ensures that every sample is managed and processed in accordance with the rare disease mindset: that every sample is precious.

Scientific data organization

The Scientific Data Organization at Parexel is a team of scientists, data stewards, solution architects and informaticists with a quality and process overlay that empowers clinical research with real-world data, aiming to answer previously intractable research questions. This team supports research by exploring relevant topics of interest such as study feasibility, synthetic control arms, sensors/wearables, and machine learning.

Advanced therapies

Upwards of 75% of rare diseases are known to have a genetic cause. Parexel’s rare disease capability is built upon deep experience and expertise in the strategic application of genetics in drug discovery and development. Each clinical trial for a gene therapy or gene-editing therapy is meticulously strategized and operationalized to ensure care of the patient and caregiver, while enabling the delivery team to be a steward of the investigational product. 

Over the last 5 years, Parexel’s rare disease experience has included:

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