Challenges in the health economics of familial hypercholesterolemia

The familial hypercholesterolemia field has limited evidence regarding cost-effectiveness, which limits optimum allocation of resources. Economic evaluations are necessary to appraise new agents and optimal timing of management approaches. Evaluations often have substantial data demands; consequentially, their applicability to medical decision-making or policy will be partly determined by the availability of data, particularly those providing information about the long-term trajectory of health benefit from familial hypercholesterolemia treatment.