HTA's and access to rare diseases therapies: The view from the PID community

When managing their healthcare budgets, governments and national agencies responsible for the provision of healthcare are faced with a number of challenges. Assessing the clinical effectiveness of a medicinal product or device should be the main factor on which decisions to reimburse or not reimburse a specific health technology should be made. There are however many other factors that come into play. The rising costs of healthcare means that decision makers are increasingly implementing cost containment measuresto reduce expenditure. Lobbyingeffortsby stakeholders, the political environmentof a countryand the willingnessof healthcare managementbodiesto involvekeystakeholdersallareadditionalimportantconsiderationstotakeintoaccount in the outcomes of an assessment procedure.