Evidence and Values: Requirements for Public Reimbursement of Drugs for Rare Diseases - a Case Study in Oncology

Conclusion:

There should be a greater commitment by reimbursement agencies to a fair and transparent decisionmaking process with appropriate community input. Criteria should be developed to validate surrogate
markers for rare diseases. It should also be acknowledged that the traditional measures of benefit in
economic studies do not incorporate all elements of social value. The need should be recognized to balance equity with an efficient use of resources.