Table II.
Parameters evaluated for correlation of price and reimbursement outcome in repurposed drugs
| Attribute | Type of variable | Description of variable | Geographical specificity |
|---|---|---|---|
| Disease-related | |||
| ICD-10 chapter change | QUAL | Yes: Different ICD-10 chapter No: Same ICD-10 chapter |
Non-country/continent specific |
| Prevalence change | QUANT | Difference between prevalence (for the USA and for the EU) of target and source product, by 100,000 population | Continent specific |
| DALY change | QUANT | Difference between DALY (for the USA and for the EU) of target and source product | Continent specific |
| Demographic group change | QUAL | Adults: From adults plus pediatrics to adults only No change: No change in demographic group Pediatrics: From adults only to adults plus pediatrics |
Non-country/continent specific |
| Number of alternative treatments change | QUANT | Difference between the number of alternative treatments for the target and the source product | Non-country/continent specific |
| Drug-related | |||
| Frequency of use change | QUAL | Increase: Increase in frequency of use No change: No change in frequency of use Decrease: Decrease in frequency of use |
Non-country/continent specific |
| Change in administration setting | QUAL | Ambulatory: From hospital to ambulatory No change: No change in administration setting Hospital: From ambulatory to hospital |
Non-country/continent specific |
| Addressing unmet needs | QUAL | Yes: Addresses an unmet need No: No change |
Non-country/continent specific |
| Improved patient convenience | QUAL | Yes: More convenient use No: No change |
Non-country/continent specific |
| Repurposing-related | |||
| Repositioning | QUAL | Yes/No As per definition in Section I1 |
Non-country/continent specific |
| Reformulation | QUAL | Yes/No As per definition in Section I2 |
Non-country/continent specific |
| Repositioning target | QUAL | On-target: Target product acts via same pathway and/or protein interaction Off-target: Target product based on a newly discovered (or previously unexplored) pharmacological mechanism |
Non-country/continent specific |
| Repositioning approach | QUAL | Serendipity: Fortuitous discovery of target product indication Hypothesis driven: Discovery of the new indication relies on understanding of the disease physiopathology and/or drug mechanism |
Non-country/continent specific |
| Reformulation group | QUAL | Group 0: Chiral switch, other Group 1: Modified release formulations Group 2: New pharmaceutical form Group 3: New administration route |
Non-country/continent specific |
| Regulatory-related | |||
| Patent expiry | QUAL | Before: Repurposing occurred before patent expiry of source product After: Repurposing occurred after patent expiry of source product |
Non-country/continent specific |
| Brand name | QUAL | Same: Target product developed under the same brand name Different: Target product developed under different brand names |
Non-country/continent specific |
| Company | QUAL | Same: Target and source product developed by the same company Different: Target and source product developed by different companies |
Non-country/continent specific |
| Approval time | QUAL | Before 1999 1999–2008 After 2008 |
Country specific |
| Designation change | QUAL | Orphan to orphan: From orphan to orphan Non-orphan: From orphan to non-orphan Orphan: From non-orphan to orphan No change: From non-orphan to non-orphan |
Continent specific |
1
Repositioning was defined as a process of finding for a known product, a new indication in a different therapeutic area, and/or via a different pharmacological target.
2
Reformulation was defined as a process of making a change into the formulation of a known product excluding dose changes and modifications involving a change in the structure of the active pharmaceutical ingredient.