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Table 2 Regression modelexplanatory variables

From: Shining a light in the black box of orphan drug pricing

Variable Value Description and source
ONCO 0 or 1 Value 1 renders all oncology orphan drugs. Oncology orphan drugs were defined as ATC-class L (Antineoplastic and immunomodulating agents).
YAUTH Continuous Number of years (as an integer number) since marketing authorization of the first indication by EMA.
AvailUS 0 or 1 Value 1 renders all orphan drugs with current authorization on the US market as listed by the FDA. AvailUS is a proxy for size of the potential treatment population. [37]
MultipleIND 0 or 1 Value 1 renders all drugs with multiple orphan indications according to EMA. MultipleIND is a proxy for size of the potential treatment population.
REPUR 0 or 1 Value 1 captures all repurposed drugs. [38]
QoLimp 0 or 1 Value 1 renders all drugs for which a statistically significant improvement in quality-of-life was described in the European Public Assessment Report.
SurvImp 0 or 1 Value 1 captures all drugs for which a statistically significant improvement in survival was described in the European Public Assessment Report.
dbRCT 0 or 1 Value 1 renders all drugs for which a double blind RCT was described in the European Public Assessment Report. dbRCT is a proxy for strength of clinical trial evidence.
AltTx 0 or 1 Value 1 renders all drugs for which an alternative drug treatment is available. Supportive care, diet, palliative care, transplantation and surgical procedures were not considered as an alternative.
ultrarare 0 or 1 Value 1 renders all drugs treating ultra-rare diseases. Ultra rare diseases have a prevalence of less than 2 per 100 000 as described in the European Public Assessment Report and/or Orphanet website.
chron 0 or 1 Value 1 renders all drugs for chronic (i.e.; longer than 6months) treatments.
oral 0 or 1 Value 1 captures all oral drugs.
Cosize 0 or 1 Cosize refers to the company size of the marketing authorization holder: respectively micro and small (0) and medium and large (1) pharmaceutical companies. Company size is based on the European definition described in EU recommendation 2003/361. Data on annual sales was derived from OneSource. [39]
Weightbased 0 or 1 Value 1 captures all drugs for which the annual treatment dose is dependent on the patient’s weight or body surface area (BSA)