Table 2 Regression model – explanatory 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) |