Multi-criteria Decision Analysis for Assessing Orphan Drugs

The assessment of new medicinal treatments usually includes cost-effective analyses which estimate the efficacy of the drug, its safety, health-related quality of life benefits and the cost. This is a comparative process where a new drug is assessed in comparison to the existing treatments for that disease. In the event that there is no treatment, then the new drug is measured against the “best supportive care”. Orphan drugs are those that treat rare diseases. Frequently there are no existing treatments, and the comparison of a new, and often expensive drug with inexpensive best supportive care implies that the new treatment is not cost-effective, and might therefore not be reimbursed by payers, thereby denying patients access to much needed treatments.This research focused on developing and assessing the value of multi-criteria decision analysis (MCDA) frameworks as tools to provide better insights on the value of orphan drugs than is provided by standard cost-effectiveness analyses. In particular, the research reviewed the criteria that should be adopted when developing MCDA frameworks, how to give the criteria different importance through weighting exercises, and how to interpret the results. Several different MCDA frameworks were developed to compare drugs within the same therapy area as well as across different diseases. Furthermore, the MCDA models were considered in the context of additional support material in the submission of documents for health technology assessments with national payers