Table 2 Extrinsic value tool: component definitions and panelists' considerations on the value of growth hormone for Turner syndrome

Ethical framework*

   Goals of healthcare - utility*

Goal of healthcare is to maintain normal functioning. Such consideration is aligned with the principle of utility, which considers the act to produce the greatest good or "greatest benefits for the greatest number"

Considered: Panelists reported that the goal of healthcare (i.e., addressing medical issues rather than social issues) should be considered, and that height is not entirely a social issue, but also a medical issue for very short patients.

Impact: Therefore for very small patients, there is a medical utility in facilitating normal functioning (reaching car pedals, kitchen cabinets etc), which would impact positively on the intrinsic value of the intervention.

On the other hand, weak evidence linking improvement in minor short stature with personal gain would have a negative impact on value.

   Opportunity costs- efficiency*

Opportunity costs include resources or existing interventions that may be forgone if intervention under scrutiny is used/reimbursed. Such consideration is aligned with the principle of efficiency, which considers maximizing impact on health for a given level of resources (efficiency can be considered at the patient level and at the society level)

Considered: Panelists indicated that this should be considered to capture the opinions of stakeholders

Impact: would have a negative impact on value, more value might be derived from psychosocial support.

Comment: Resources are often allocated to measurable outcomes (e.g., height) rather than softer outcomes such as psychosocial benefits.

   Population priority & access - fairness*

Priorities for specific groups of patients are defined by societies/decisionmakers and reflect their moral values. Such considerations are aligned with the principle of fairness, which considers treating like cases alike and different cases differently and often gives priority to those who are worst-off (theory of justice)

Considered: Panelists indicated that this should be considered

Impact: mixed impact - negative impact related to the concept of treating like cases similarly (e.g., short stature due to other diseases) as it dilutes the importance of TS patients relative to other groups

Comment: should not discriminate against rare diseases; there should be public debate on priorities

Other components

   System capacity and appropriate use of intervention

The capacity of healthcare system to implement the intervention and to ensure its appropriate use depends on its infrastructure, organization, skills, legislation, barriers and risks of inappropriate use. Such considerations include mapping current systems and estimating whether the use of the intervention under scrutiny requires additional capacities (note: if available, economic estimate would be included in the economic component E3 of the MCDA Value Matrix)

Considered: some panelists indicated that it should be considered while others indicated there was no potential for inappropriate use

Impact: for those who indicated it should be considered, it would have a negative impact on value

Comment: although there is no risk of misdiagnosis (genetic testing), because guidelines are not clear on age initiation, there is a risk of having all toddlers initiated on treatment, which was considered as inappropriate use.

Misuse of growth hormone is possible (gaining height for no medical reason) and there are no mechanisms in place for surveillance of inappropriate use.

   Stakeholder pressures

Pressures from groups of stakeholders are often part of the context surrounding healthcare interventions. Such considerations include being aware of pressures and interests at stake and how they may affect values of decisionmakers

Considered: some panelists indicated that it should be considered while others reported that it should not be taken into account

Impact: for those who indicated it should be considered, it would have a negative impact on value.

Comment: Lobby groups are effective at reaching and impacting decisionmakers

   Political/historical context

Political/historical context may influence the value of an intervention in consideration of specific political situations and priorities as well as habits, traditions and precedence

Considered: Panelists indicated that this should be considered

Impact: none reported

Comment: This includes the political will to demonstrate fairness to rare disorders as well as universal access to care (guaranteed by the Canadian healthcare system) to satisfy entitlement felt by affected families.

Budgetary context (i.e., recession, balanced budget or surplus) affects decisions.

   Other components

Components that are not already captured in the standard set proposed