Table 1 Definition and classification of the variables relevant for the price and reimbursement process in Spain
Variable | Definition | Classification |
|---|---|---|
Clinical variables | ||
(i) Therapeutic area | Therapeutic area was defined according to the ATC code [35] | Oncologic ODs Other |
(ii) Outcomes classification | Each ODs clinical trials were analysed and classified depending on the outcomes used in the pivotal study: survival-related outcomes (i.e. overall survival) vs other outcomes (PROs, biomarkers, etc.) [36] | Survival Other |
(iii) Therapeutic alternatives | ODs with therapeutic alternatives were defined as those intended to treat the same indication | Yes No |
(iv) Rarity of disease | Rarity was defined according to the prevalence of the disease for which ODs were indicated. Rare diseases affect < 5/10000 inhabitants and ultra-rare diseases affect < 1/50000 [3] | Rare Ultra-rare |
(v) Safety profile | Safety was defined according to the obligation by the EMA to carry out a PASS to obtain further information on a medicine’s safety [37] | PASS No PASS |
(vi) Type of population | Type of population refers to whether ODs are intended to treat paediatric patients or not, defined as those patients under 18 years of age | Paediatric Other |
Regulatory variables | ||
(i) TPR conclusion | The TPR conclusion was extracted from a positive opinion in the TPR (new drug recommended to a group of patients or equivalent to approved alternatives) or a questionable opinion with respect to the EMA [19] | Positive Questionable |
(ii) Conditional approval | ODs with conditional approval granted by the EMA were defined as those with a positive benefit-risk balance but requiring clinical studies that have not yet been completed [38] | Yes No |