Value-based decision-making for orphan drugs with multiple criteria decision analysis: burosumab for the treatment of X-linked hypophosphatemia

Figures & data

Table 1. Description of the domains and criteria used in the multiple criteria decision analysis framework.

Domains	Criteria	A priori level ranking^a
Disease burden
	B1 - Rarity of disease	No
	B2 - Population affected by the disease	No
	B3 - Disease severity	Yes
	B4 - Unmet medical need	Yes
Therapeutic value
	T1 - Treatment characteristics	Yes
	T2 - Clinical impact of treatment	Yes
	T3 - Treatment impact on PRO	Yes
	T4 - Treatment safety and tolerability	Yes
	T5 - Level of uncertainty regarding the available evidence for the treatment	Yes
	T6 - Level of treatment innovation	Yes
	T7 - Treatment impact on health equity	Yes
Economic burden
	E1 - Direct medical costs	Yes
	E2 - Direct non-medical costs	Yes
	E3 - Indirect costs	Yes

Abbreviation. PRO: Patient Reported Outcomes; ^aFor criteria with an a priori level ranking, levels within the criteria have a natural preference ordering, either best-to-worst or worst-to-best (e.g.: lower direct medical costs are preferred over higher direct medical costs (Supplemental File 1: Table S1–E1), whereas rare diseases affecting a predominantly infant population are not necessarily preferred over those affecting a predominantly elderly population (Supplemental File 1: Table S1–B2)).

Figure 1. Average importance estimates for the criteria considered in the decision-making context of OMP public financing. Legend: Error bars represent 95% confidence intervals; Criteria related to the Disease Burden, Therapeutic Value and Economic Value are painted in green, blue and red, respectively.

Figure 2. Overall socio-economic and therapeutic value for burosumab (B) and conventional therapy (CT). Legend: Superimposed are the portions of the overall value attributed to the Disease Burden (green), Therapeutic Value (blue) and Economic Burden (red).

Figure 3. Breakdown of the overall socio-economic and therapeutic value for burosumab and conventional therapy. Legend: Dark grey – Burosumab; Light grey – Conventional therapy; As a reference, the average importance estimates are presented for the criteria related to the Disease Burden (green), Therapeutic Value (blue) and Economic Burden (red).

Figure 4. Public financing likelihood for burosumab (B) and conventional therapy (CT).

Data availability statement

All data generated or analyzed during this study are included in this published article and its supplementary information files.