Table 1.
Country definitions for rare and ultra-rare disease treatments, and their use as eligibility criteria within supplemental processes for rare disease treatments
| PROCESS TYPE | COUNTRY | PROCESS DESCRIPTION | ELIGIBILITY | DEFINITION | |
|---|---|---|---|---|---|
| Rare disease | Ultra-rare disease | ||||
| Separate |
England Highly Specialised Technology Programme (HST) |
Main differences with standard process: willingness to pay threshold, specialised appraisal committee, more holistic perspective of value, managed access agreements possible | High cost technologies for ultra-rare conditions - see HST prioritisation criteria | – | No prevalence criteria, based on HST eligibility criteria |
|
Lithuania Ultra-OMP pathway |
Very rare disease committee: special appraisal committee decides on inclusion in special list. Main differences with standard process: therapeutic value not graded, no waiting list in case of positive decision, special pricing rules. Decision can be individual-case (yearly revised fixed budget) or generalised-case approach (general budget) | (1) ultra-rare, (2) life-threatening or significant disability, (3) subject to effective aetiology or pathogenic treatment, (4) effective treatment (increases survival or reduces disability) | – | < 1:200,000 | |
| Partially separate |
Scotland "Ultra-OMP pathway |
Assessment based on ultra-OMP decision-making criteria, routine use for 3 years after which re-assessment. Option for PACE process. Disease-specific experts describe treatment benefit not captured within original assessment |
URDT: (1) ultra-rare, (2) chronic and severely disabling condition, (3) highly specialised management PACE: OMPs (and end of life treatments) not considered cost-effective - after NDC decision (after 3-year monitoring) |
– | < 1:50,000 |
| Germany | Different reimbursement status: additional benefit guaranteed, strong negotiation power and reasonable reimbursement. Assessment by G-BA (instead of IQWIG), different evidence requirements | (1) OMP, (2) revenues from statutory health insurance < 50 million/last 12 months | OMP | – | |
| Adapted | Norway | Greater willingness to pay | (1) ultra-rare, (2) effective treatment (> 2 QALY gain), (3) severe condition (> 30 QALYs lost) | – | < 1:100,000 |
| Slovakia | Exempt from economic evaluation | Ultra-rare | – | < 1:50,000 | |
| Sweden | Greater willingness to pay | (1) ultra-rare, (2) good potential for effective drug, (3) very severe condition | – |
no fixed limit ~ < 1–2:100,000 |
|
|
Scotland Standard pathway with PACE and modifiers |
PACE: disease-specific experts describe treatment benefit not captured within original assessment. Modifiers: standard assessment for OMPs, but SMC recognises limitation in evidence generation and will accept greater uncertainty in the economic case |
PACE: OMPs (end of life treatments) not cost-effective, manufacturer can request a PACE to get additional insights Modifiers: OMPs, life-threatening, substantial increase in quality of life/life expectancy, can reverse the condition, bridges gap to a definitive therapy. |
OMP | – | |
| Expedited | Belgium | Earlier pricing: after positive CHMP opinion, before marketing authorisation. Exemption from economic model | OMP | OMP | – |
| Italy | Earlier pricing and reimbursement: after positive CHMP opinion, before marketing authorisation | OMP (and hospital or exceptionally therapeutic and social medicinal products) | OMP | – | |
| New Zealand | Earlier reimbursement: before marketing authorisation | Rare disease (as per country definition) | Cum prevalence < 1:50,000 | – | |
| Rarity weighted | Romania | Reimbursement status based on points cumulated (unconditional, conditional reimbursement etc.): OMPs get extra points | OMP | OMP | – |
| Slovakia | Willingness to pay threshold based on points system: OMPs get extra points | OMP | OMP | – | |
| Exempt from HTA | Bulgaria | All drugs to treat those rare diseases included in special list of rare diseases are 100% reimbursed | Drugs with indication included on special list of rare diseases | OMP | – |
| Latvia | Separate state-reimbursement budget for children with rare diseases | OMP for use in children | OMP | – | |
Legend: HTA Health technology assessment, OMP orphan medicinal product (refers to drugs with an orphan designation from the European Medicines Agency), RDT rare disease treatment, HST Highly Specialised Technology programme, SMC Scottish Medicines Consortium, PACE Patient and Clinical Engagement programme, G-BA Federal Joint Committee, IQWIG Institute for Quality and Efficiency in Health Care, CHMP Committee for Medicinal Products for Human Use of the European Medicines Agency, NDC New Drugs Committee, QALY Quality of Life Adjusted Life Years