In the EU, rare diseases that are also life-threatening or chronically debilitating with a low prevalence and a high level of complexity are defined as orphan diseases.
HTAs should be a flexible process used collaboratively. The use of HTA to make reimbursement dec
A disease is defined as rare in Europe when it affects less than 1 in 2,000 citizens (Orphan Drug
A ‘one size fits all’ approach to the timing and methodology of appraisals will fail to take acco
It is estimated that over 6,000 rare diseases exist, although some say the actual number is close