An initial approval of a treatment in a well-defined patient subgroup with a high medical need and subsequent widening of the indication to a larger patient population, or an early regulatory approval [..]

A group of people formed of international experts representing the major stakeholders involved in the clinical management of patients with rare diseases, in evidence-based medicine, and in developing [..]

AGREE II is a reliable and validated tool to assess the methodological rigour and transparency in which a guideline is developed. (http://www.agreetrust.org/)

The evidence which is judged to be strongest and the most reliable for any given topic.

A statement to direct the best achievable practice in specific areas of care emphasising the delivery of care that is patient centred, feasible and fair.

A person, paid or unpaid, who regularly provides care to a person because they are ill, frail or have a disability.

A study that compares people with a specific disease or outcome of interest (cases) to people from the same population without that disease or outcome (controls), and which seeks to find associations [..]

A study reporting observations on a series of individuals, usually all receiving the same intervention, with no control group.

Grouping of diseases with common characteristics.

Clinical recommendations provide guidance on how healthcare and other professionals should care for people with specific conditions.