Rocket Pharmaceuticals

What is the Natural History of a Disease?

The Natural History of a Disease is the typical progression of the disease over a person’s lifetime starting from its onset

What is the purpose of a Natural History Study?

Natural History studies are observational studies that gather comprehensive data on the course of the disease to gain a better understanding of its progression and impact on patients. Data captured may include information on the physical signs and symptoms patients experience, test results (for example blood tests or imaging), genetic information, and health-related quality of life, over a period of time. This information helps describe the disease course and identify important factors (e.g. demographic, genetic, and/or lab test results), that may contribute to better or worse disease outcomes

What compensation is provided when participating in a Natural History Study?

Travel, accommodation, and food stipend would be provided. Study assessments are provided free of charge.

What are the benefits of participating?

Natural History Studies are particularly important for rare diseases. Given the diversity of symptoms and the small size of the patient population, these studies are often the only opportunity for researchers and physicians to address significant knowledge gaps and obtain a robust understanding of the disease and patient experience. Engaging in a Natural History Study offers numerous benefits to the community, such as: Raising awareness in medical, academic and pharmaceutical sectors Empowering rare disease patient communities with knowledge and support for advocating better healthcare policie

How do Natural History Studies help inform and design clinical trials?

These studies provide various avenues to help inform and design clinical trials, such as: Highlighting areas of unmet medical need Understanding characteristics of the patient population to define a treatment benefit from a patient’s perspective. Describing disease progression, enabling the identification of clinical outcome measures and biomarkers Provide patient-centric data for health authorities such as the FDA, and other public health institutions to use in their review processes of investigational treatments.