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Table 2 Key characteristics of quantitative approaches for benefits and harm assessment

From: A framework for organizing and selecting quantitative approaches for benefit-harm assessment

Approaches for B&H assessment

BLRA[22]

Boers[23]

Gail[3]

INHB[24, 25]

MCDA[4, 26]

MCE[6, 27]

NCB[28]

NNT& NNH[12, 13]

PSM[29]

QFRBA[6]

Q-TWiST[14, 15, 30]

RBC[31]

RBP[32]

RV-NNT[6, 27]

SPM & MAR[33–35]

TURBO[11]

Number of approaches in each category

Key characteristics

Types of data

                 

Require individual patient data

Yes

Yes

No

No

No

No

No

No

No

No

No

No

No

No

Yes

No

Indiv: 3

No: 13

Types of analyses

                 

Data driven (DD) versus simulation (S)

DD

NA

DD/S

DD

DD

DD

DD/S

DD

S

DD

DD

DD/S

DD

DD

DD

DD

DD: 14

S: 4

Types of B&H metrics

                 

Absolute versus relative measures versus QALY versus other (O)

Other

A

A

QALY

A / relative

A

A

A

A

A / relative

A /QALY

A

A

A

A

A / relative

Absolute: 14

Relative: 3

QALY: 2

Assumptions

                 

Put B&H outcomes on same scale

yes

no

yes

yes

yes

yes

yes

yes

yes

no

yes

no

yes

yes

no

yes

Yes: 12

No:4

Uncertainty estimates for B&H assessment

no

no

no

no

yes

no

yes

no*

yes

NA

no

yes

no

no

NA

no

Yes: 4

No: 12

Joint distribution of B&H outcomes considered for uncertainty estimates

no

NA.

P

NA

no

NA

no

no

P

NA

NA

P

P

no

NA

NA

Possible: 4

No: 4

n.a.: 8

Multiple endpoints versus composite outcomes for B&H

M

Comp

M

M

M

M

M

M/ Comp

M

M

M

M

Comp

M

M

Comp

Multiple:12

Comp: 5

Consideration of preferences

Explicitly considers preferences for B&H assessment:

yes

no

yes

no

yes

both

yes

no

both

no

yes

no

no

yes

yes

no

Yes: 9

No:9

Types of presenting benefit risk comparison

B&H difference versus B&H ratio versus Time gained/lost versus B&H graphic versus other

D

Graphic

D

D

D, ratio, other

D

D

Ratio

D

D, ratio, other

Time, D

Graphic

Graphic

Ratio

D

Graphic

Difference: 10

Ratio: 4

Time: 1

Graphic: 3

  1. # NNT and NNH are most commonly used separately but put on the same scale if their ratio is taken.
  2. NA, Not applicable; BLRA, Benefit-less-risk analysis; B&H, Benefit and harm assessment; INHB, Incremental net health benefit; MCDA, Multicriteria decision analysis; MCE, Minimum clinical efficacy; NCB, Net Clinical Benefit; NNT, Number needed to treat; NNH, Number needed to treat for harm; PSM, Probabilistic simulation methods; QFRBA, Quantitative Framework for Risk and Benefit Assessment; Q-Twist, (Quality-adjusted) Time without Symptoms and Toxicity; RBC, Risk–benefit contour; RBP, Risk–benefit plane; RV-NNT, Relative value adjusted number needed to treat; SPM, Stated preference method; MAR, Maximum acceptable risk; TURBO, Transparent Uniform Risk Benefit Overview; P, Possible; D, Difference; A, Absolute; M, Multiple.