Table 1 The core evaluation framework for value-added medicines
Cluster name | Domain name | Definition |
|---|---|---|
Unmet medical need | Extending treatment options in new indication with unmet medical need | Reduction of the unmet medical need for patients in a new indication due to additional therapeutical value |
Individual needs/special needs of patient (sub)population | Reduction of the unmet medical need in patients with special needs in the original indication (e.g., treatment resistant patients, vulnerable patients, etc.) | |
Health gain (measured by health care professionals) | Efficacy/Effectiveness | Improved clinical outcomes of a pharmaceutical treatment (e.g., extending survival, stabilizing disease, improving treatment response, etc.) in trial and real-world settings |
Patient safety and tolerability | Improved safety and/or tolerability of the pharmaceutical treatment | |
Patient Reported Outcomes | Patient experience related to the therapy | Improved patient's satisfaction, acceptance, convenience with the pharmaceutical therapy |
Adherence and persistence | Improved adherence and/or persistence of patients with the prescription guidelines (including duration, timing, dosage and frequency of medication use) | |
Quality of life | Improved health-related quality of life reported by patients | |
Burden on households | Patient's economic burden | Improved productivity of patients and/or reduced health and non-health care resource use (such as travel time) covered by patients |
Economic and health burden on informal caregiver | Improved quality of life of family caregivers and/or reduced financial or non-financial burden on households | |
Burden on health care system | Health care resource utilization, costs or efficiency | Reduced utilization of health care cost and/or resources |
Technological improvement with logistical considerations | Improved stability and/or shelf life of pharmaceuticals through technological improvement |