Rare diseases often receive less funding for research compared to more common diseases due to their small patient population.

Rare Diseases International (RDI) is a global alliance of patient organizations dedicated to improving the lives of people with rare diseases.

Natural history studies aim to gather information about the natural course of a rare disease, including its symptoms, progression, and prognosis.

Patient advocacy groups play a crucial role in raising awareness, advocating for funding, providing support, and participating in research activities related to rare diseases.

Single-arm trials are often used in rare disease research due to the limited number of patients available for participation.

Orphan drug designation offers financial incentives, expedited regulatory approval, and exclusive marketing rights to encourage pharmaceutical companies to develop treatments for rare diseases.

Adaptive clinical trials allow researchers to modify the trial design during the study based on emerging data, such as safety and efficacy information.

All of the mentioned ethical considerations are important in rare disease research, given the vulnerable nature of patients and the potential risks associated with experimental treatments.

Rare disease registries serve as valuable resources for collecting patient data, supporting research, and providing information and support to affected individuals and their families.

The Food and Drug Administration (FDA) is responsible for approving orphan drugs in the United States.

Phase 1 clinical trials primarily aim to assess the safety and tolerability of a new drug in a small group of patients.

Genetic, protein, and imaging biomarkers can all be used in rare disease diagnosis and monitoring depending on the specific disease and its characteristics.

International collaboration in rare disease research enables the sharing of resources, expertise, patients, and harmonization of regulatory requirements, leading to more efficient and effective research efforts.

The National Institutes of Health (NIH) is the primary government agency in the United States that provides funding for rare disease research.

Patient-reported outcome measures (PROMs) are used in rare disease clinical trials to assess the impact of the disease on patients' quality of life, evaluate the effectiveness of treatments from the patient's perspective, and identify potential adverse effects of treatments.