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Table 1 Initial judgement on the acceptability of evidence and complexity of evidence generation for each value domain in the core evaluation framework

From: Development of a core evaluation framework of value-added medicines: report 2 on pharmaceutical policy perspectives

Method of data collection Source Value domains
Unmet medical need Health gain (measured by health care professionals) Patient reported outcomes Burden on households Burden on health care systems
Extending treatment options in new indication with unmet medical need Individual needs/special needs of patient (sub)population Efficacy/ effectiveness Patient safety and
tolerability
Patient experience related to the therapy Adherence and Persistence Quality of life Patient’s economic burden Economic and health burden on informal caregiver Health care resource utilization, costs or efficiency Technological improvement with logistical considerations
Ex-ante Evidence from RCT  +  +  + / €€€  +  + / €€€  +  +  + / €€€  +  + / €€€  + / €€€ NA  +  +  + / €€€ NA  +  +  + / €€€ NA NA
Non-RCT evidence (e.g., observational study, patient registry, single-arm trial)  +  + / €  +  +  + / €  + / €  +  +  + / €  + / €€  +  + / €€  + / €€  + / €€  +  + / €€  + / €€  + / €
Ex-post Real-world evidence (e.g., coverage with evidence development, outcome guarantee) NA  +  + / €  +  + / €  +  + / €  +  + / €€  +  +  + / €  +  +  + / €€  +  +  + / €€  +  +  + / €€  +  +  + / €  +  + / €
  1. (+ / +  + / +  +  + : Perceived acceptability from the decision maker's perspective, €/€€/€€€: Perceived complexity and cost of data collection from the manufacturer’s perspective, RCT—randomized controlled trial)