HTAs and rare diseases: How to assess their cost-effectiveness

摘要

The assessment of pharmaceuticals has, in recent years, expanded beyond efficacy and safety to cover economic implications and other consequences. The incorporation of an economic perspective and an assessment of cost-effectiveness into the decision-making process as to what therapies can be made available have aroused much debate and discussion, which at times has been quite heated. Newspaper headlines of people having to remortgage their houses to pay for 'life-saving' therapies and media frenzy when 'effective' treatments are denied to desperate patients are becoming all too common occurrences. The emotive nature of such 'decisions' are intensified when considering rare conditions 3, In rare diseases, the low number of patients makes therapies highly expensive, as they try to recapture research and development costs, and unlikely to meet the thresholds for cost-effectiveness of payment agencies 1, 2. The appropriateness of conventional metrics for assessing efficiency has been challenged 4,5 while issues relating to whether therapies for rare diseases warrant special status within health technology assessments and whether the 'conventional' approaches and criteria used to assess effectiveness and efficiency should be extended to incorporate equity and social values remain equivocal 1,2,5,