Table 1 Country definitions for rare and ultra-rare disease treatments, and their use as eligibility criteria within supplemental processes for rare disease treatments

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

–