Revealed preferences towards the appraisal of orphan drugs in Poland - multi criteria decision analysis

Kolasa, Katarzyna; Zwoliński, Krzysztof; Zah, V.; Kaló, Zoltán; Lewandowski, Tadeusz

doi:10.1186/s13023-018-0803-9

Cited by 20 publications

(18 citation statements)

References 26 publications

(26 reference statements)

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“…This might enrich discussions and potentially lead to a better understanding of the evidence. On the one hand, a multiple stakeholder approach combined with transparent reporting helps decision-makers to justify a final decision ( Iskrov et al, 2017 ; Baltussen et al, 2018 ; Kolasa et al, 2018 ). This is important, as the OMP appraisal process may often turn political, putting high pressure on decision-makers to avoid a negative outcome ( Simoens et al, 2013 ).…”

Section: Resultsmentioning

confidence: 99%

How to Value Orphan Drugs? A Review of European Value Assessment Frameworks

et al. 2021

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Background: Decision-makers have implemented a variety of value assessment frameworks (VAFs) for orphan drugs in European jurisdictions, which has contributed to variations in access for rare disease patients. This review provides an overview of the strengths and limitations of VAFs for the reimbursement of orphan drugs in Europe, and may serve as a guide for decision-makers.Methods: A narrative literature review was conducted using the databases Pubmed, Scopus and Web of Science. Only publications in English were included. Publications known to the authors were added, as well as conference or research papers, or information published on the website of reimbursement and health technology assessment (HTA) agencies. Additionally, publications were included through snowballing or focused searches.Results: Although a VAF that applies a standard economic evaluation treats both orphan drugs and non-orphan drugs equally, its focus on cost-effectiveness discards the impact of disease rarity on data uncertainty, which influences an accurate estimation of an orphan drug’s health benefit in terms of quality-adjusted life-years (QALYs). A VAF that weighs QALYs or applies a variable incremental cost-effectiveness (ICER) threshold, allows the inclusion of value factors beyond the QALY, although their methodologies are flawed. Multi-criteria decision analysis (MCDA) incorporates a flexible set of value factors and involves multiple stakeholders’ perspectives. Nevertheless, its successful implementation relies on decision-makers’ openness toward transparency and a pragmatic approach, while allowing the flexibility for continuous improvement.Conclusion: The frameworks listed above each have multiple strengths and weaknesses. We advocate that decision-makers apply the concept of accountability for reasonableness (A4R) to justify their choice for a specific VAF for orphan drugs and to strive for maximum transparency concerning the decision-making process. Also, in order to manage uncertainty and feasibility of funding, decision-makers may consider using managed-entry agreements rather than implementing a separate VAF for orphan drugs.

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Section: Resultsmentioning

confidence: 99%

How to Value Orphan Drugs? A Review of European Value Assessment Frameworks

et al. 2021

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“…Moreover, throughout the years the EU has created an operational framework and coordinates several areas of common health policy in the field of RDs, including the classification and codification of RDs and orphan medicinal products, an ICD-10 revision and the creation of European Reference Networks or a European Platform for Rare Diseases registration (Moliner, 2010;Aymé, 2014, 2015;Moliner and Waligora, 2017;Khosla and Valdez, 2018;Montserrat and Taruscio, 2019;Czech et al, 2020). Nevertheless, while a lot has been done in the field of recommendations, in funding, and the reimbursement of orphan drugs in Europe (Kawalec et al, 2016;Zelei et al, 2016;Kolasa et al, 2018;Szegedi et al, 2018), still one of the most urgent areas in both the European and national health policies in the field of RDs is the medical education of healthcare students and professionals (Miteva et al, 2011;Budych et al, 2012;Anderson et al, 2013;Engel et al, 2013;Krajnović et al, 2013;Zurynski et al, 2017). Meanwhile, according to the EU recommendation, social and medical education on RDs should be one of the key areas of each national plan or strategy (Council of the European Union, 2009).…”

Section: Introductionmentioning

confidence: 99%

Knowledge and Attitudes of Future Healthcare Professionals Toward Rare Diseases

Domaradzki

Walkowiak

2021

Front. Genet.

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Caring for patients suffering from a rare disease (RD) requires the special and combined efforts of different healthcare professionals, including nurses, physiotherapists and physicians. Nevertheless, Poland still lacks a national plan for RDs and the undergraduate and postgraduate education of future healthcare professionals on RDs is also inadequate. Thus, the aim of this study was to assess the awareness of RDs among nursing, physiotherapy and medical students in Poland. It shows that although 98% of respondents had heard of the term “rare disease,” most students had problems in defining the most common causes of RDs and their prevalence. Students also lacked basic knowledge about the healthcare system for RD patients in the country. While over 95% of future nurses, physiotherapists and physicians assessed their knowledge about RDs as insufficient or very poor, almost 92% of medical students, and 84% of physiotherapy and nursing students, did not feel prepared for caring for RD patients. Furthermore, although the vast majority of respondents declared eagerness to broaden their knowledge on RDs, only 45% of medical students, 76% of nursing students and 88% of physiotherapy students believed that RDs should be included into the medical curricula. Simultaneously, for most students the Internet was the prime source of information on RDs. It is concluded that as caring for RD patients requires a multidisciplinary approach, by identifying the gap in the education of future nurses, physiotherapists and physicians this study shows that there is an urgent need of better education about RDs among future healthcare professionals.

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“…There is a growing interest in applying specific extended evaluation frameworks and multi-criteria decision analysis (MCDA) to support the priority setting, pricing, reimbursement and procurement decisions of special types of health technologies, including orphan medicines, offpatent medicines, vaccines, medical devices or intensive care for patients with severe infections [11][12][13][14][15][16][17][18][19]. MCDA is a methodology for explicit appraisal of different alternatives by aggregating individual and often conflicting criteria into a single overall score, allowing to transform ad hoc decisions into transparent and replicable processes [20,21].…”

Section: Multi-criteria Decision Analysismentioning

confidence: 99%

“…Out of the three potential decision contexts, the standardization of rules for exemption from internal price referencing or acknowledgment of a fair price premium for a reasonable differential value is the easiest approach to how the proposed value framework can be utilized in the majority of countries. As the utilization of MCDA in health policy decisions has constantly been increasing [11][12][13][14][15][16][17][18][19], the core value framework may be converted into an MCDA tool with some additional work, especially in countries with political and economic interest to strengthen the R&D activities of local generic and biosimilar manufacturers [20]. Discussions about augmented cost-effectiveness frameworks have only started recently at international conferences.…”

Section: Future Perspectivesmentioning

confidence: 99%

Development of a core evaluation framework of value-added medicines: report 2 on pharmaceutical policy perspectives

Kaló

Petykó

Fricke

et al. 2021

Cost Eff Resour Alloc

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Background A core evaluation framework that captures the health care and societal benefits of value added medicines (VAMs, also often called repurposed medicines) was proposed in Report 1, aiming to reduce the heterogeneity in value assessment processes across countries and to create incentives for manufacturers to invest into incremental innovation. However, this can be impactful only if the framework can be adapted to heterogeneous health care financing systems in different jurisdictions, and the cost of evidence generation necessitated by the framework takes into account the anticipated benefits for the health care system and rewards for the developers. Areas covered The framework could potentially improve the pricing and reimbursement decisions of VAMs by adapting it to different country specific decision-contexts such as deliberative processes, augmented cost-effectiveness frameworks or formal multi-criteria decision analysis (MCDA); alternatively, some of its domains may be added to current general evaluation frameworks of medicines. The proposed evaluation framework may provide a starting point for practices based on which VAMs can be exempted from generic pricing mechanisms or can be integrated into the reimbursement and procurement system, allowing for price differentiation according to their added value. Besides evidence from RCTs, pricing and reimbursement decision processes of VAMs should allow for ex-ante non-RCT evidence for certain domains. Alternatively, relying on ex-post evidence agreements—such as outcome guarantee or coverage with evidence development—can also reduce decision uncertainty. Conclusions The core evaluation framework for VAMs could trigger changes in the existing pricing, reimbursement and procurement practices by improving the appraisal of the added value created by incremental innovation.

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Revealed preferences towards the appraisal of orphan drugs in Poland - multi criteria decision analysis

Cited by 20 publications

References 26 publications

How to Value Orphan Drugs? A Review of European Value Assessment Frameworks

How to Value Orphan Drugs? A Review of European Value Assessment Frameworks

Knowledge and Attitudes of Future Healthcare Professionals Toward Rare Diseases

Development of a core evaluation framework of value-added medicines: report 2 on pharmaceutical policy perspectives

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