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Table 1 – Proposed criteria for evaluation of orphan drugs and corresponding potential parameters

From: Paying for the Orphan Drug System: break or bend? Is it time for a new evaluation system for payers in Europe to take account of new rare disease treatments?

Criteria Price Differential
Lower Medium Higher
Rarity 1:2,000 - 1:20,000 or COMP figures > 3 in 10,000 (11%) 1:20,000 - 1:200,000 or COMP figures 1–3 in 10,000 (51%) Less than 1:200,000 or COMP figures less than 1 in 10,000 (38%)
Level of research undertaken Literature review Building on previous existing knowledge “Blue-sky” – starting research & development programme in an unknown area
Level of uncertainty of effectiveness Immature, but promising data Appropriate surrogate end-points Robust clinical end-points
Manufacturing complexity Not complex – small molecule / classic galenic form Moderately complex Highly complex biological and galenic form
Follow up measures (additional benefits and associated costs) Moderate to none Designed to answer specific, defined, delineated question Safety and efficacy studies + size and duration of study
Characteristics without direct cost impact
Disease severity Morbidity Mortality / severe invalidity in adulthood Mortality / severe invalidity as infant
Available alternatives / unmet medical need Alternatives with similar characteristics Alternatives – but offering strong innovation to the disease treatment No alternative
Level of impact on condition / disease modification Low Medium Strong
Use in unique indication or not Existing orphan or non-orphan indications for the same molecule* Potential for multiple indications Unique indication – no other use possible
  1. *N.B. Another element could be the total revenues in the context of multiple indications for the same molecule owned by the same company.