The Application and Implications of Novel Deterministic Sensitivity Analysis Methods

Vreman, Rick A; Geenen, Joost W; Knies, Saskia; Mantel‐Teeuwisse, Aukje K.; Leufkens, Hubert G. M.; Goettsch, Wim

doi:10.1007/s40273-020-00979-3

Cited by 25 publications

(22 citation statements)

References 31 publications

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“…The literature about challenges with MEA in different jurisdictions is relatively large ( Stafinski et al, 2010 ; Puig-Peiró et al, 2011 ; Wonder et al, 2012 ; Ferrario and Kanavos, 2013 ; Ferrario and Kanavos, 2015 ; Ferrario et al, 2017 ; Kanavos et al, 2017 ; Breccia et al, 2020 ; Zampiroli Dias et al, 2020) but there is a lack of published information about the factors that contribute to successful MEA systems and the constructs of MEA for individual products. Confidentiality clauses often drive the absence of published data in many countries ( Stafinski et al, 2010 ; Wonder et al, 2012 ), however there is important information about the constructs of MEA that could be shared, including core data sets and aggregated analyses.…”

Section: Discussionmentioning

confidence: 99%

The Evolution of AIFA Registries to Support Managed Entry Agreements for Orphan Medicinal Products in Italy

2021

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Italy has a well-established prominent system of national registries to support managed entry agreements (MEAs), monitoring innovative medicinal products (MPs) with clinical as well as economic uncertainties to ensure appropriate use and best value for money. The technological architecture of the registries is funded by pharmaceutical companies, but fully governed by the national medicines agency (AIFA). A desktop analysis was undertaken of data over a 15-year timeframe of all AIFA indication-based registries and associated EMA information. The characteristics of registries were evaluated, comparing orphan MPs vs. all MPs exploring cancer and non-cancer indications. OMP (orphan medicinal product) registries’ type vs. AIFA innovation status and EMA approval was reviewed. Of the 283 registries, 182 are appropriateness registries (35.2% relate to OMPs, with an almost equal split of cancer vs. non-cancer for OMPs and MPs), 35 include financial-based agreements [20% OMPs (2 non-cancer, 5 cancer)], and 60 registries are payment by result agreements [23.3% OMPs (4 non-cancer, 10 cancer)]. Most OMPs (53/88) came through the normal regulatory route. With the strengthening of the system for evaluation of innovation, fewer outcomes-based registries have been instigated. AIFA has overcome many of the challenges experienced with MEA through developing an integrated national web-based data collection system: the challenge that remains for AIFA is to move from using the system for individual patient decisions about treatment to reviewing the wealth of data it now holds to optimize healthcare.

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Section: Discussionmentioning

confidence: 99%

The Evolution of AIFA Registries to Support Managed Entry Agreements for Orphan Medicinal Products in Italy

2021

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“…The ISPOR Modeling Good Research Practices Task Force Report 6 on uncertainty states that in those cases where there is very little information on a parameter, a conservative approach should be adopted such that the absence of evidence is reflected in a very broad range of possible estimates [2]. This is exactly what we advocate in our paper: a broad range of possible estimates is provided through the use of intermediate steps between the set minimum and maximum values [3].This is especially relevant in those cases where probability distributions are not known, given that in those cases, the extreme values are most likely not known either (or they are at least very uncertain). As we also indicate in the paper, minimum and maximum values are often chosen arbitrarily.…”

Section: Dear Editormentioning

confidence: 85%

“…We welcome the comment [1] on our article [3] on deterministic sensitivity analysis (DSA) as it provides some interesting points for discussions regarding performing DSA under ignorance. The comment indicates that under complete ignorance, it is not rational to apply a distributional or probabilistic DSA, because the underlying distributions are not known, and that 'all variables to which uniform probability distributions would be applied otherwise due to a lack of information on probabilities appear to be candidates for the classic tornado diagram.…”

Section: Dear Editormentioning

confidence: 99%

Comment on “Deterministic Sensitivity Analysis Under Ignorance”

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“…Many HTA organizations review DSA as well as PSA due to the advantage of being able to transparently check the effect of uncertainty of each variable on the results ( Australian Government: Department of Health, 2016 ; The Canadian Agency for Drugs, 2017 ; The National Institute for Health and Care Excellence (NICE), 2013 ), Yet DSA, especially one-way SA, has a problem of underestimating the overall uncertainty because it examines only the effects of variations that one variable can have while other variables are fixed ( Claxton, 2008 ). In addition, there is a limitation that the nonlinearity of the model is not reflected, and if there is a correlation between variables, it cannot be considered appropriately ( Briggs et al, 2012 ; McCabe et al, 2020 ; Vreman et al, 2021 ). Moreover, despite many guidelines stipulated that that “clinically and statistically feasible ranges” are recommended, the ranges of the DSA are often chosen arbitrarily ( Vreman et al, 2021 ).…”

Section: Introductionmentioning

confidence: 99%

How Sensitive is Sensitivity Analysis?: Evaluation of Pharmacoeconomic Submissions in Korea

2022

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Purpose: We aimed to describe the types of uncertainties examined in the economic evaluations submitted for reimbursement in Korea and their impact on the incremental cost-effectiveness ratio (ICER).Method: Fifty dossiers were submitted by pharmaceutical companies to the economic subcommittee of the Pharmaceutical Benefit Coverage Advisory Committee (PBCAC) from January 2014 to December 2018. The types of uncertainties were categorized as structural and parametric, and the frequencies of the sensitivity analysis per variables were analyzed. The impact of uncertainties was measured by the percent variance of the ICER relative to that of the base case analysis.Results: Of the 50 submissions, varying discount rate (44 submissions), followed by time horizon (38 submissions) and model assumptions (29 submissions), were most frequently used to examine structural uncertainty, while utility (42 submissions), resource use (41 submissions), and relative effectiveness (26 submissions) were used to examine parametric uncertainty. A total of 1,236 scenarios (a scenario corresponds to a case where a single variable is varied by a single range) were presented in the one-way sensitivity analyses, where parametric and structural sensitivity analyses comprised 679 and 557 scenarios, respectively. Varying drug prices had the highest impact on ICER (median variance 19.9%), followed by discount rate (12.2%), model assumptions (11.9%), extrapolation (11.8%), and time horizon (10.0%).Conclusions: Variables related to long-term assumptions, such as model assumptions, time horizon, extrapolation, and discounting rate, were related to a high level of uncertainty. Caution should be exercised when using immature data.

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The Application and Implications of Novel Deterministic Sensitivity Analysis Methods

Cited by 25 publications

References 31 publications

The Evolution of AIFA Registries to Support Managed Entry Agreements for Orphan Medicinal Products in Italy

The Evolution of AIFA Registries to Support Managed Entry Agreements for Orphan Medicinal Products in Italy

Comment on “Deterministic Sensitivity Analysis Under Ignorance”

How Sensitive is Sensitivity Analysis?: Evaluation of Pharmacoeconomic Submissions in Korea

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