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Table 1 Reimbursement criteria for orphan drugs

From: A multi-stakeholder multicriteria decision analysis for the reimbursement of orphan drugs (FinMHU-MCDA study)

Name of the criterion and definition Levels
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
3. Disease severity
Degree to which patient is affected
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
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
Not cost-effective
  1. aDirect medical costs associated with the diagnosis, treatment, and management of patients with the disease
  2. bNonmedical direct costs derived from the disease (generally borne by the patient, caregiver, or social services)
  3. cIndirect costs derived from the loss of productivity due to absenteeism/sick leave
  4. dCost per complete treatment in single-dose treatments