Table 2 Combined metrics for both patients and providers for rare diseases
Patients and their families | Health Systems |
|---|---|
Health quality and outcome Physical health and disease progression Mental health Survival Socio-economic burden Work and productivity Financial status Social integration Time availability | Economic costs and efficiency of delivery Integrate the quality of the health care service into economic analysis (cost effectiveness, pricing, reimbursement) Compare the rising cost of diagnosis and the benefit of treating patients early on or at a later stage, and how it affects treatment efficacy and disease progression Develop cost effectiveness criteria based on social preferences (quality of life, social value) for the conventional evaluation of medical components Evaluate the impact of access to diagnosis on the cost of clinical components such as appropriate decision making by the physicians, appropriate therapy delivery, appropriate study design and development of end points in clinical trials |