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

From: Are supplemental appraisal/reimbursement processes needed for rare disease treatments? An international comparison of country approaches

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
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
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.
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
  1. 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