Abstract: Patients' organisations call for an assessment of the clinical added
value of orphan drugs at the time of the marketing authorisation evaluation.
The European Commission has commissioned Ernst & Young to explore the
feasibility of a new Committee at the European Medicines Agency. A new drug candidate faces a number of hurdles on its journey to reach the patient.
Regulators are the first, and right after are the Health Technology Agencies
and payers. Things can turn sour, from a political point of view, when that
second scenario happens, when the regulators say "yes this drug is safe and
effective" and the payers say "Oh well but we won't reimburse it". Regulators and health technology assessment experts have recently started to
harmonise their respective methodologies to avoid some degree of redundancy and
contradictions in their respective opinions. Patients are participating in the
decision making at the European Medicines Agency, and work closely with some
national regulatory agencies. More systematic involvement with health
technology assessment agencies is only beginning. This paper illustrates some
of the issues patients are discussing with health technology assessment
Keywords: Health technology assessment, patients' representatives, Quality-Adjusted Life Years, Health Services Accessibility