Table 1 Reimbursement criteria for orphan drugs
| Name of the criterion and definition | Levels | |
|---|---|---|
| Population | ||
|
1. Target population Number of patients affected by the disease who are candidates for treatment, according to prevalence and/or incidence | Prevalence < 0.2 per 10,000 inhabitants | |
| Prevalence between 0.2 and 1 per 10,000 inhabitants | ||
| Prevalence > 1 but < 5 per 10,000 inhabitants | ||
|
2. Age of target population Age at the beginning of treatment of the disease | Nonpediatric | |
| Pediatric | ||
| Disease | ||
|
3. Disease severity Degree to which patient is affected | Mild | |
| Moderate | ||
| Severe | ||
|
4. Economic burden of the disease Economic impact of the disease on the health system and society in general, considering the types of resources and costs involved a, b, c | Low economic impact | |
| Moderate economic impact | ||
| High economic impact | ||
| Treatment | ||
|
5. Safety Adverse events due to treatment 5.1 Seriousness 5.2 Frequency | Serious AE | Frequent AE |
| Nonserious AE | Infrequent AE | |
|
6. Availability of treatment alternatives Availability of different therapeutic options | No other therapeutic options | |
| There are other options, but the current treatment improves health more than the alternatives | ||
| There are therapeutic options with similar characteristics | ||
|
7. Efficacy Expected clinical benefit or actual clinical benefit in the framework of a clinical trial | High benefit: curative or significant increase in survival | |
| Moderate benefit: stabilization of the disease or improvement in quality of life | ||
| Low benefit: palliative or symptomatic | ||
|
8. Quality of evidence Credibility and robustness of evidence | Randomized controlled trial with comparator | |
| Other types of clinical trials or with inappropriate comparator | ||
| Nonrandomized study | ||
|
9. Health-related quality of life Change in patient’s health-related quality of life due to the treatment received, associated with impaired mobility, personal care, daily activities, pain/discomfort, or anxiety/depression | Treatment improves health-related quality of life | |
| Treatment does not modify health-related quality of life | ||
| Treatment decreases health-related quality of life | ||
| Economic evaluation | ||
|
10. Cost of treatment Cost per patient per yeard | < €100,000 per year | |
| €100,000 to €300,000 per year | ||
| > €300.000 per year | ||
|
11. Costs avoided by treatment Reduction in costs derived from application of treatment, including medical costsa, non-medical costs b, and indirect costsc | Avoids direct medical and nonmedical costs derived from the disease and indirect costs due to loss of productivity | |
| Avoids direct medical costs derived from the disease | ||
| Does not avoid direct/indirect costs of the disease, or there is not enough information on avoided costs | ||
|
12. Cost-effectiveness Efficiency of a treatment, according to the criterion and the payers’ willingness to pay, evaluated by the incremental cost-effectiveness ratio expressed as cost per quality-adjusted life year gained from the intervention against a comparator or standard treatment | Cost-effective | |
| Not cost-effective | ||