A natural history study is a type of study/registry that follows a group of people over time who have, or are at risk of developing, a specific medical condition or disease.
The Desmoid Tumor Research Foundation has been selected as one of 20 rare disease patient groups who will develop a natural history study with the assistance of the National Organization for Rare Disorders (NORD), supported in part by a cooperative agreement with the U.S. Food and Drug Administration (FDA). Read NORD’s press release. DTRF was selected in a competitive application process reviewed by a NORD committee. This study in desmoid tumors will fill an important gap in existing data about the disease.
NORD’s announcement explains: “Natural history studies are… studies that aim to fill research gaps to help medical researchers better understand how diseases progress over time… Rare diseases have posed unique challenges to researchers and drug developers due to small patient populations, lack of data, clinical endpoints that are often unclear, and enrollment and retention challenges for clinical trials. …NORD’s Natural History Studies project empowers patients and families to help eliminate some of the ‘I don’t know’ in rare disease research, making way for progress.”