Rare and Emerging Diseases

Westat is ready to respond, conducting rigorous clinical trials that support treatments for some of healthcare’s biggest challenges.

While rare diseases affect only a small percentage of the population (fewer than 200,000 people in the U.S.), their impact can be devastating. More than 7,000 rare diseases impact 25-30 million Americans. In an effort to drive research and treatment, Congress created the Orphan Drug Act of 1983, which provides financial incentives to encourage pharmaceutical companies to develop new drugs for these elusive diseases. Westat has long been a driver in bringing the intent of the Act into reality.

Rapid Response

Emerging diseases can sometimes start as rare or new diseases, affecting specific populations in a certain region of the world. Most of them are infectious diseases in nature, but the incidence can change dramatically and give rise to epidemics, like Zika Virus disease, or even pandemics, like COVID-19. In these circumstances, it is critical to respond quickly to understand the causes of the disease and develop preventive measures and treatments to combat them.

Westat has been at the forefront working with national and international partners for years to address emerging disease. During the Zika epidemic, we worked with international partners and provided administrative, technical support, and training. Our clinical team immediately responded to the COVID-19 pandemic of 2020 by implementing a clinical trial in eleven sites in the U.S. to address some of the devastating consequences of the virus.

Full Services for a Broad Range of Health Challenges

Westat supports studies for a wide range of rare diseases like Leigh Syndrome, Alexander Disease, Hereditory Angioedema, and Leber Congenital Amourosis. Our team is poised to support entire programs of study or its components, from Phase I to license application submissions and FDA approval. Westat can serve as a full-service CRO or customize our services to the needs of the sponsor and the program.

Our team of scientists, regulatory staff, protocol specialists, site monitors, biostatisticians, and experts in clinical trial management are experienced and well positioned to bring rigor, creative problem solving tools, and effective strategies to respond to biomedical and public health needs. We draw from decades of domestic and international experience to implement credible and effective clinical studies, while providing customized attention and flexibility to our sponsors.

Getting Treatment to Multi-drug Resistant HIV-Resistant Patients Sooner

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Julie DiStefano-Pappas

Senior Clinical Data Manager

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Lauren Laimon

Project Director