International experiences in multicriteria decision analysis (MCDA) for evaluating orphan drugs: a scoping review

Lasalvia, Pieralessandro; Prieto-Pinto, Laura; Moreno, Manuel; Castrillon, J.; Romano, Giulio; Garzón‐Orjuela, Nathaly; Rosselli, Diego

doi:10.1080/14737167.2019.1633918

Cited by 20 publications

(26 citation statements)

References 28 publications

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“…Difficulties stem from the fact that HTA evaluation results cannot be easily used for decision-making at the local level (Barasa et al 2015;Martelli et al 2015). Today, a consensus seems to form on the use of multicriteria decision analysis (MCDA) based on multiattribute value theory (Poulin et al 2013;Radaelli et al 2014;Angelis and Kanavos 2017;Antioch et al 2017;Lasalvia et al 2019) to help decision-making. This was initiated by the NICE during the 2010 s' (Antioch et al 2017).…”

Section: Hta's Limitationsmentioning

confidence: 99%

Automated hospital pharmacy supply chain and the evaluation of organisational impacts and costs

Mathy

Pascal

Fizesan

et al. 2020

Supply Chain Forum: An International Journal

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Supply chain digitalisation equipment's raise concerns for hospital decision-makers, especially regarding the cost-efficiency ratio, usually evaluated through health technologies assessments (HTA) at a national level or by return on investment (ROI) calculations by the hospital finance department. Unfortunately, none of the two methods identifies organisational impacts, nor hidden gains and costs, especially those that are related to the internal reallocation of resources as shows the example of automated dispensing systems (ADS) in the central pharmacy of a French hospital with a posteriori analysis from the hospital perspective. The introduction of the ADS caused some hidden costs or gains, which do not generate any financial flow and which are therefore not valued. When they are valued, as we do in this study by including them in the ROI calculation, the results change dramatically: the profitability of the investment goes from negative to positive. Given the importance of hidden gains and costs related to organisational impacts, they should be included in evaluations.

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Section: Hta's Limitationsmentioning

confidence: 99%

Automated hospital pharmacy supply chain and the evaluation of organisational impacts and costs

Mathy

Pascal

Fizesan

et al. 2020

Supply Chain Forum: An International Journal

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“…(2) Another feature justifying special status for the reimbursement of OMPs were unmet needs . Authors argued that in the case of unmet needs, it was unfair not to cover those treatments [ 24 , 36 , 57 , 69 , 75 , 77 , 79 , 87 , 89 , 99 , 102 , 114 , 116 , 122 , 129 , 130 , 136 , 178 , 184 , 189 , 202 , 203 , 205 , 208 , 220 – 224 ]. However, Sandman and Hofmann [ 184 ] found the concept of unmet needs unsustainable in this context and recommended instead considering disease severity.…”

Section: Resultsmentioning

confidence: 99%

“…However, Sandman and Hofmann [ 184 ] found the concept of unmet needs unsustainable in this context and recommended instead considering disease severity. (3) One extensively discussed reason (n = 68) related to unmet need was the lack of alternative treatment [ 8 , 24 , 37 , 44 – 46 , 51 , 54 , 56 , 60 , 62 , 64 , 70 , 75 , 77 – 79 , 83 , 84 , 87 , 96 , 111 , 112 , 114 – 116 , 122 , 123 , 128 – 130 , 135 , 139 , 141 , 142 , 147 , 150 – 152 , 161 – 163 , 167 , 174 , 177 , 178 , 184 , 193 , 199 – 203 , 206 , 208 , 210 – 215 , 217 , 219 , 223 , 225 – 228 ]: Since OMPs often represented the only hope for patients suffering from rare diseases, which justified their special status [ 70 ]. Relatedly, some studies also found a societal preference for covering drugs for diseases without treatment alternatives [ 45 , …”

Section: Resultsmentioning

confidence: 99%

“…For instance, it was suggested to use additional criteria for assessing the reimbursement of OMPs [ 29 , 45 , 114 ], such as the budget impact of a drug [ 54 , 87 , 114 , 120 , 120 , 141 , 150 , 174 , 200 , 202 , 208 , 224 , 244 ]. Treatment benefit Another line of argument was that a special status was justified if OMPs provided a high benefit [ 24 , 26 , 43 – 47 , 63 , 72 , 78 , 79 , 87 , 88 , 93 , 104 , 107 , 109 , 112 , 114 – 116 , 120 , 125 , 127 – 132 , 136 , 152 , 153 , 161 , 167 , 178 , 184 , 195 , 202 , 203 , 208 , 212 , 214 – 216 , 249 , 260 , 265 , 266 ]. The nature of this benefit, however, was defined differently [ 208 ]: some authors referred to characteristics such as life-saving [ 47 , …”

Section: Resultsmentioning

confidence: 99%

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A systematic review of moral reasons on orphan drug reimbursement

Zimmermann

Eichinger

Baumgartner

2021

Orphanet J Rare Dis

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Background The number of market approvals of orphan medicinal products (OMPs) has been increasing steadily in the last 3 decades. While OMPs can offer a unique chance for patients suffering from rare diseases, they are usually very expensive. The growing number of approved OMPs increases their budget impact despite their low prevalence, making it pressing to find solutions to ethical challenges on how to fairly allocate scarce healthcare resources under this context. One potential solution could be to grant OMPs special status when considering them for reimbursement, meaning that they are subject to different, and less stringent criteria than other drugs. This study aims to provide a systematic analysis of moral reasons for and against such a special status for the reimbursement of OMPs in publicly funded healthcare systems from a multidisciplinary perspective. Results With a systematic review of reasons, we identified 39 reasons represented in 243 articles (scientific and grey literature) for and against special status for the reimbursement of OMPs, then categorized them into nine topics. Taking a multidisciplinary perspective, we found that most articles came from health policy (n = 103) and health economics (n = 49). More articles took the position for a special status of OMPs (n = 97) than those against it (n = 31) and there was a larger number of reasons identified in favour (29 reasons) than against (10 reasons) this special status. Conclusion Results suggest that OMP reimbursement issues should be assessed and analysed from a multidisciplinary perspective. Despite the higher occurrence of reasons and articles in favour of a special status, there is no clear-cut solution for this ethical challenge. The binary perspective of whether or not OMPs should be granted special status oversimplifies the issue: both OMPs and rare diseases are too heterogeneous in their characteristics for such a binary perspective. Thus, the scientific debate should focus less on the question of disease prevalence but rather on how the important variability of different OMPs concerning e.g. target population, cost-effectiveness, level of evidence or mechanism of action could be meaningfully addressed and implemented in Health Technology Assessments.

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“…Furthermore, local adaptation allows for participation of local stakeholders, which is critical in incorporating societal values, preferences, or practices as reported for other decision problems such as evaluation of orphan drugs [ 28 ]. In addition, the multi-stakeholder creation process helps to create understanding, acceptance, and ownership among the local stakeholders (appropriation), and hence, it is an important step in the implementation process.…”

Section: Discussionmentioning

confidence: 99%

Case studies for implementing MCDA for tender and purchasing decisions in hospitals in Indonesia and Thailand

Holtorf

Kristin

Assamawakin

et al. 2021

J of Pharm Policy and Pract

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Background A multi-criteria decision analysis (MCDA) approach has been suggested for helping purchasers in low- and middle-income countries in an evidence-based assessment of multi-source pharmaceuticals to mitigate potential adverse consequences of price-based decisions on patient access to effective medicines. Six workshops for developing MCDA-instruments for purchasing were conducted in Indonesia, Kazakhstan, Thailand, and Kuwait in 2017–2020. In Indonesia and Thailand, two pilot-initiatives aimed to implement the instruments for hospital drug purchasing decisions. Objective By analysing and comparing the experiences and progress from the MCDA-workshops and the two case-examples for hospital implementation in Indonesia and Thailand, we aim to gain insights, which will support future implementation. Methods The selection of criteria and their average weight were compared quantitatively across the MCDA-instruments developed in all four countries and settings. Implementation experiences from two case-examples were studied, which included (1) testing the instrument across a variety of drugs in seven hospitals in Thailand and (2) implementation in one specialty hospital in Indonesia. Semi-structured interviews were conducted via web-conferences with four diverse stakeholders in the pilot implementation projects in Thailand and Indonesia. The open responses were evaluated through qualitative content analysis and synthesis using grounded theory coding. Results Drivers for implementation were making ‘better’ decisions, achieving transparency and a rational selection process, reducing drug shortages, and assuring consistent quality. Challenges were seen on the technical level (definition or of criteria, scoring methods, access to data) or change-related challenges (resistance, perception of increased workload, lack of competencies or capabilities, lack of resources). The comparison of the MCDA instruments revealed high similarity, but also clear need for local adaptations in each specific case. Conclusion A set a of measures targeting challenges related to utility, methodology, data requirements, capacity building and training as well as the broader societal impact can help to overcome challenges in the implementation. Careful planning of implementation and organizational change is recommended for ensuring commitment and fit to local context and culture. Designing a collaborative change program for each application of MCDA-based purchasing will enable healthcare stakeholders to maximally benefit in terms of quality and effectiveness of care and access for patients.

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International experiences in multicriteria decision analysis (MCDA) for evaluating orphan drugs: a scoping review

Cited by 20 publications

References 28 publications

Automated hospital pharmacy supply chain and the evaluation of organisational impacts and costs

Automated hospital pharmacy supply chain and the evaluation of organisational impacts and costs

A systematic review of moral reasons on orphan drug reimbursement

Case studies for implementing MCDA for tender and purchasing decisions in hospitals in Indonesia and Thailand

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