Why orphan drug coverage reimbursement decision-making needs patient and public involvement

Douglas, Conor M.W.; Wilcox, Elizabeth; Burgess, Michael; Lynd, Larry D.

doi:10.1016/j.healthpol.2015.01.009

Cited by 35 publications

(49 citation statements)

References 25 publications

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“…Panelists’ individual value systems were reflected in the variation of weights, highlighting the critical impact of appraisal committee composition. Patient involvement in decisionmaking over a product’s life cycle is a much debated and researched topic, [47–54] particularly in the field of orphan diseases [49, 50, 53, 54]. Reflective MCDA approaches are well suited to capture the diversity of perspectives, enhance participation and communication, and improve understanding of the ethical trade-offs and dilemmas inherent in decisionmaking and resource allocation.…”

Section: Discussionmentioning

confidence: 99%

Appraising the holistic value of Lenvatinib for radio-iodine refractory differentiated thyroid cancer: A multi-country study applying pragmatic MCDA

Wagner¹,

Khoury²,

Bennetts³

et al. 2017

BMC Cancer

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BackgroundThe objective of the study was to reveal through pragmatic MCDA (EVIDEM) the contribution of a broad range of criteria to the value of the orphan drug lenvatinib for radioiodine refractory differentiated thyroid cancer (RR-DTC) in country-specific contexts.MethodsThe study was designed to enable comprehensive appraisal (12 quantitative, 7 qualitative criteria) in the current disease context (watchful waiting, sorafenib) of France, Italy and Spain. Data on the value of lenvatinib was collected from diverse stakeholders during country-specific panels and included: criteria weights (individual and social values); performance scores (judgments on evidence—collected through MCDA systematic review); qualitative impacts of contextual criteria; and verbal and written insights structured by criteria. The value contribution of each criterion was calculated and uncertainty explored.Results Comparative effectiveness, Quality of evidence (Spain and Italy) and Disease severity (France) received the greatest weights. Four criteria contributed most to the value of lenvatinib, reflecting its superior Comparative effectiveness (16–22% of value), the severity of RR-DTC (16–22%), significant unmet needs (14–21%) and robust evidence (14–20%). Contributions varied by comparator, country and individuals, highlighting the importance of context and consultation. Results were reproducible at the group level. Impacts of contextual criteria varied across countries reflecting different health systems and cultural backgrounds. The MCDA process promoted sharing stakeholders’ knowledge on lenvatinib and insights on context.ConclusionsThe value of lenvatinib was consistently positive across diverse therapeutic contexts. MCDA identified the aspects contributing most to value, revealed rich contextual insights, and helped participants express and explicitly tackle ethical trade-offs inherent to balanced appraisal and decisionmaking.Electronic supplementary materialThe online version of this article (doi:10.1186/s12885-017-3258-9) contains supplementary material, which is available to authorized users.

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Section: Discussionmentioning

confidence: 99%

Appraising the holistic value of Lenvatinib for radio-iodine refractory differentiated thyroid cancer: A multi-country study applying pragmatic MCDA

Wagner¹,

Khoury²,

Bennetts³

et al. 2017

BMC Cancer

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“…As underlined by Douglas et al [40] , reimbursement decision-making for ODs could benefit from public and patient involvement in the following 3 different types of procedures: HTA, priority setting to address public funding, and development of evaluation criteria. As recognised by Drummond et al [21] , patients could provide valuable information on the satisfaction and acceptability of health technologies and could inform decisions supported from their values, needs, and preferences.…”

Section: Comprehensive Value Assessmentmentioning

confidence: 99%

“…As recognised by Drummond et al [21] , patients could provide valuable information on the satisfaction and acceptability of health technologies and could inform decisions supported from their values, needs, and preferences. The interaction of increased transparency in public health policies, together with the recognition of the value of the patients' experiential expertise, could play a role in involving public/patient representatives in coverage decision-making, either directly or indirectly by means of setting and weighing specific criteria to support coverage decisionmaking [21,40] .…”

Section: Comprehensive Value Assessmentmentioning

confidence: 99%

Reimbursed Price of Orphan Drugs: Current Strategies and Potential Improvements

Mincarone

Leo²,

Sabina³

et al. 2017

Public Health Genomics

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The pricing and reimbursement policies for pharmaceuticals are relevant to balance timely and equitable access for all patients, financial sustainability, and reward for valuable innovation. The proliferation of high-cost specialty medicines is particularly true in rare diseases (RDs) where the pricing mechanism is characterised by a lack of transparency. This work provides an overall picture of current strategies for the definition of the reimbursed prices of orphan drugs (ODs) and highlights some potential improvements. Current strategies and suggestions are presented along 4 dimensions: (1) comprehensive value assessment, (2) early dialogs among relevant stakeholders, (3) innovative reimbursement approaches, and (4) societal participation in producing ODs. Comprehensive value assessment could be achieved by clarifying the approach of distributive justice to adopt, ensuring a representative participation of stakeholders, and with a broad consideration of value-bearing factors. With respect to early dialogs, cross-border cooperation can be determinant to companies and agencies. The cost-benefit ratio of early dialogs needs to be demonstrated and the “regulatory capture” effect should be monitored. Innovative reimbursement approaches were developed to balance the need for evidence-based decisions with the timely access to innovative drugs. The societal participation in producing ODs needs to be recognised in a collaborating framework where adaptive agreements can be developed with mutual satisfaction. Such agreements could also impact on coverage and reimbursement decisions as additional elements for the determination of a comprehensive societal value of ODs. Further research is needed to investigate the highlighted open challenges so that RDs will not remain, in practical terms, orphan diseases.

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“…It should be explicit how these decisions are informed so as to ensure legitimacy and to provide clear guidance to OMP developers about the types of products that society wishes them to develop [48]. …”

Section: Principles On Omp Decision Criteriamentioning

confidence: 99%

Recommendations from the European Working Group for Value Assessment and Funding Processes in Rare Diseases (ORPH-VAL)

Annemans

Aymé

Cam

et al. 2017

Orphanet J Rare Dis

102

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Rare diseases are an important public health issue with high unmet need. The introduction of the EU Regulation on orphan medicinal products (OMP) has been successful in stimulating investment in the research and development of OMPs. Despite this advancement, patients do not have universal access to these new medicines. There are many factors that affect OMP uptake, but one of the most important is the difficulty of making pricing and reimbursement (P&R) decisions in rare diseases. Until now, there has been little consensus on the most appropriate assessment criteria, perspective or appraisal process. This paper proposes nine principles to help improve the consistency of OMP P&R assessment in Europe and ensure that value assessment, pricing and funding processes reflect the specificities of rare diseases and contribute to both the sustainability of healthcare systems and the sustainability of innovation in this field. These recommendations are the output of the European Working Group for Value Assessment and Funding Processes in Rare Diseases (ORPH-VAL), a collaboration between rare disease experts, patient representatives, academics, health technology assessment (HTA) practitioners, politicians and industry representatives. ORPH-VAL reached its recommendations through careful consideration of existing OMP P&R literature and through a wide consultation with expert stakeholders, including payers, regulators and patients. The principles cover four areas: OMP decision criteria, OMP decision process, OMP sustainable funding systems and European co-ordination. This paper also presents a guide to the core elements of value relevant to OMPs that should be consistently considered in all OMP appraisals. The principles outlined in this paper may be helpful in drawing together an emerging consensus on this topic and identifying areas where consistency in payer approach could be achievable and beneficial. All stakeholders have an obligation to work together to ensure that the promise of OMP’s is realised.Electronic supplementary materialThe online version of this article (doi:10.1186/s13023-017-0601-9) contains supplementary material, which is available to authorized users.

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Why orphan drug coverage reimbursement decision-making needs patient and public involvement

Cited by 35 publications

References 25 publications

Appraising the holistic value of Lenvatinib for radio-iodine refractory differentiated thyroid cancer: A multi-country study applying pragmatic MCDA

Appraising the holistic value of Lenvatinib for radio-iodine refractory differentiated thyroid cancer: A multi-country study applying pragmatic MCDA

Reimbursed Price of Orphan Drugs: Current Strategies and Potential Improvements

Recommendations from the European Working Group for Value Assessment and Funding Processes in Rare Diseases (ORPH-VAL)

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