Table 2.
Score assigned to the selected factors of the R&D cost estimation
| Article | Drug sample characteristics | Possible sources of variation in R&D costs | Monetary values | |||||||||||||
|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|
| Breadth of coverage | Replicability | Transition or success prob.—estimation | Risk of failure | Development time—estimation | Therapeutic class | NCE or NBE | Orphan drug | Self-originated or licensed-in | Firm size | Discovery/preclinical | Clinical phases | Opportunity costs | Tax-deductible costs | |||
| Considered | Source reliability | Specificity | Replicability | |||||||||||||
| Wouters et al. [19] (2020) | 5 | 6 | 4 | 6 | 6 | 6 | 3 | 3 | 5a | 4 | 6 | 6 | 5 | 6 | 6 | 4 |
| Jayasundara et al. [40] (2019) | 2 | 5 | 4 | 6 | 6 | 1 | 1 | 6 | 1 | 1 | 4 | 1 | 5 | 6 | 6 | 4 |
| Årdal et al. [47] (2018) | 2 | 2 | 1 | 1 | 6 | 4 | 4 | 1 | 3 | 3 | 6 | 3 | 2 | 3 | 2 | 1 |
| Prasad and Mailankody [37] (2017) | 2 | 6 | 1 | 1 | 6 | 4 | 6 | 6 | 6 | 3 | 6b | 5 | 4 | 6 | 6 | 3 |
|
DiMasi et al. [17] (2016) |
4 | 3 | 5 | 6 | 5 | 5 | 5 | 3 | 4 | 3 | 6 | 6 | 5 | 3 | 6 | 2 |
|
Chit et al. [45] (2014) |
5 | 4 | 6 | 6 | 6 | 4 | 4 | 1 | 1 | 3 | 6 | 6 | 3 | 4 | 6 | 5 |
|
Falconi et al. [50] (2014) |
5 | 6 | 6 | 5 | 5 | 4 | 3 | 1 | 1 | 1 | 1 | 1 | 3 | 2 | 5 | 1 |
|
Sertkaya et al. [49] (2014) |
3 | 2 | 4 | 6 | 4 | 4 | 4 | 3 | 1 | 3 | 6 | 6 | 3 | 2 | 6 | 3 |
| Mestre-Ferrandiz et al. [48] (2012) | 4 | 4 | 6 | 6 | 6 | 3 | 3 | 3 | 3 | 3 | 6 | 6 | 6 | 3 | 6 | 3 |
| Adams and Brantner [51] (2010) | 6 | 4 | 6 | 5 | 6 | 5 | 5 | 1 | 3 | 5 | 4 | 1 | 5 | 4 | 6 | 1 |
|
Paul et al. [52] (2010) |
4 | 3 | 5 | 6 | 6 | 3 | 3 | 1 | 3 | 3 | 6 | 6 | 6 | 3 | 5 | 1 |
| DiMasi and Grabowski [53] (2007) | 5 | 3 | 5 | 6 | 5 | 3 | 6 | 1 | 1 | 4 | 6 | 6 | 5 | 3 | 6 | 1 |
| Adams and Brantner [54] (2006) | 6 | 4 | 6 | 5 | 6 | 5 | 1 | 1 | 3 | 5 | 6 | 6 | 5 | 4 | 6 | 3 |
|
DiMasi et al. [55] (2004) |
5 | 3 | 5 | 6 | 5 | 6 | 3 | 1 | 4 | 1 | 1 | 1 | 5 | 3 | 6 | 1 |
|
DiMasi et al. [18] (2003) |
4 | 3 | 5 | 6 | 5 | 3 | 3 | 1 | 4 | 3 | 6 | 6 | 5 | 3 | 6 | 2 |
|
Global Alliance for TB Drug Development [56] (2001) |
3 | 2 | 4 | 5 | 6 | 4 | 1 | 3 | 3 | 1 | 6 | 5 | 5 | 2 | 6 | 1 |
| Young and Surrusco [24] (2001) | 6 | 5 | 1 | 4 | 2 | 1 | 1 | 1 | 3 | 1 | 1 | 1 | 4 | 5 | 2 | 6 |
|
DiMasi et al. [57] (1995) |
4 | 3 | 5 | 6 | 5 | 6 | 1 | 1 | 4 | 6 | 6 | 6 | 5 | 3 | 5 | 1 |
|
DiMasi et al. [58] (1995) |
4 | 3 | 5 | 6 | 5 | 6 | 1 | 1 | 4 | 1 | 4c | 1 | 5 | 3 | 6 | 1 |
|
DiMasi et al. [16] (1991) |
4 | 3 | 5 | 6 | 5 | 3 | 1 | 1 | 4 | 3 | 6 | 6 | 5 | 3 | 6 | 2 |
|
Wiggins [59] (1987) |
6 | 5 | 1 | 1 | 4 | 3 | 1 | 1 | 1 | 1 | 1 | 1 | 4 | 5 | 6 | 1 |
|
Hansen [60] (1979) |
2 | 3 | 4 | 5 | 5 | 1 | 1 | 1 | 4 | 1 | 6 | 6 | 5 | 3 | 6 | 1 |
A detailed explanation of the categories used are provided in electronic supplementary information 3.
Source: Authors’ elaboration
NBE new biological entity, NCE new chemical entity, prob. probabilities, R&D research and development
The score assesses how comprehensively the studies considered factors contributing to the R&D cost estimation and to what extent they addressed them. Higher numbers indicate that the estimations reflect the factor to a higher degree
aConsidered in the sensitivity analysis by changing the assumptions for preclinical costs
bGiven that the authors included the entire R&D investment of the company, we assumed that estimation should cover R&D costs from the time of discovery or initial acquisition of the compound to approval
cOnly for two of the estimations reported: (1) all drugs, and (2) anti-infectives (Abx)