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Natural History Studies

Overview

The natural history of disease is the natural course of the disease from the time immediately prior to disease initiation, through its pre-symptomatic phase and various clinical stages until a final outcome without treatment intervention. Natural history studies of ALS are designed to examine the course of the disease over time. They convey a specific purpose, are comprehensive and are intended to aid in describing the disease.

Types of natural history studies:

  • Retrospective study (i.e. looking back): Information has already been generated prior to the beginning of the study. An example is the review of old medical records.
  • Prospective study (i.e. looking forward): Information is gathered after study initiation. An example is a study that examines clinical manifestations of affected individuals over time. This study has flexibility to collect additional data as the study progresses. Other advantages include the ability to have consistent data collection and standard of care during the study.
  • Survey study (i.e. questionnaire): Information is collected from people living with ALS, caregivers or others through questionnaires that are analyzed. They typically do not involve doctor appointments and give a quick overview of a disease population.

What The ALS Association is Doing

The Association is supporting natural history studies to understand the natural course of the disease in people living with SOD1 and C9orf72 mutations. These studies are critical as they inform clinical testing of new treatment approaches, and in particular, the current clinical trial targeting SOD1 with antisense drugs and the C9orf72 antisense clinical trial planned for 2017.

Why It Matters

ALS is considered a rare disease by definition by the US Orphan Drug Act (ODA), since it has a prevalence of less than 200,000 people in the U.S. Based on its rare status; the existing knowledge on the natural history of ALS is limited. Natural history studies fill in this void.

The goals and uses of natural history studies vary depending on study type, although they all share the common goal to inform and support drug development and approval. Along that line, natural history studies can help in clinical trial design and identify study end points that lead to more rapid and efficient clinical trials. They aid in informing patient care, best practices and research priorities. Together, natural history studies are essential in building the scientific foundation of clinical development programs.

Source: U.S. Food and Drug Administration

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