Value and specificity of rare diseases business model-is the pursuit of this societal priority sustainable?

Dunoyer, Marc; Rollet, Pierrick

doi:10.1186/1750-1172-7-s2-a23

Cited by 2 publications

(4 citation statements)

References 1 publication

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“…Changes to ICER thresholds should adjust for value criteria that are not routinely captured within a CEA framework, such as the disease severity, unmet need (availability of alternatives) and disease prevalence. Weighting should be explicit and consider established public policy choices and societal preferences to encourage OMP development [ 7 , 43 , 63 , 64 ].…”

Section: Discussionmentioning

confidence: 99%

“…Value assessment frameworks should be flexible enough to reflect the specificities of rare diseases, such as the disease burden and lack of therapeutic alternatives [ 6 , 16 , 38 ]. There is a growing consensus that multi-criteria frameworks are particularly well-suited to capturing these specificities in rare diseases [ 42 , 43 , 64 ]. Patient access to OMPs can be optimised by using economic analysis adjunctively, rather than relying on incremental cost-effectiveness as the main driver of decisions.…”

Section: Discussionmentioning

confidence: 99%

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Principles for consistent value assessment and sustainable funding of orphan drugs in Europe

Gutiérrez

Patris²,

Hutchings

et al. 2015

Orphanet J Rare Dis

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The European Orphan Medicinal Products (OMP) Regulation has successfully encouraged research to develop treatments for rare diseases resulting in the authorisation of new OMPs in Europe. While decisions on OMP designation and marketing authorisation are made at the European Union level, reimbursement decisions are made at the national level. OMP value and affordability are high priority issues for policymakers and decisions regarding their pricing and funding are highly complex. There is currently no European consensus on how OMP value should be assessed and inequalities of access to OMPs have previously been observed. Against this background, policy makers in many countries are considering reforms to improve access to OMPs. This paper proposes ten principles to be considered when undertaking such reforms, from the perspective of an OMP manufacturer. We recommend the continued prioritisation of rare diseases by policymakers, an increased alignment between payer and regulatory frameworks, pricing centred on OMP value, and mechanisms to ensure long-term financial sustainability allowing a continuous and virtuous development of OMPs. Our recommendations support the development of more consistent frameworks and encourage collaboration between all stakeholders, including research-based industry, payers, clinicians, and patients.

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Section: Discussionmentioning

confidence: 99%

Section: Discussionmentioning

confidence: 99%

Principles for consistent value assessment and sustainable funding of orphan drugs in Europe

Gutiérrez

Patris²,

Hutchings

et al. 2015

Orphanet J Rare Dis

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“…The regulatory system for approval can affect the stream of DR significantly. A progressive approval system, in which the drug can be approved following proof of safety ( Loike and Miller, 2017 ), may accelerate DR greatly, as suggested for rare diseases ( Dunoyer, 2012 ) and regenerative medicine ( Caplan and West, 2014 ). However, the risk of eliminating phase-II and -III studies should also be considered carefully ( Loike and Miller, 2017 ).…”

Section: Future Directionsmentioning

confidence: 99%

“…However, the risk of eliminating phase-II and -III studies should also be considered carefully ( Loike and Miller, 2017 ). Conditional approval in Europe for orphan drugs ( Dunoyer, 2012 ) and an approval system for off-label use of drugs validated in publicly funded research in Japan ( Shimazawa and Ikeda, 2012 ) might serve as templates to consider the progressive approval system for DR. Lifecycle management, which includes patent protection and contributes to maximizing the return of investment for drug discovery, is another important aspect of DR. Analyses of lifecycle management in the Japanese market are undertaken actively ( Hashitera et al, 2013 ; Yamanaka and Kano, 2016 ).…”

Section: Future Directionsmentioning

confidence: 99%

Overcoming Obstacles to Drug Repositioning in Japan

et al. 2017

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Drug repositioning (DR) is the process of identifying new indications for existing drugs. DR usually focuses on drugs that have cleared phase-I safety trials but has yet to show efficacy for the intended indication. Therefore, DR can probably skip the preclinical and phase-I study, which can reduce the cost throughout drug development. However, the expensive phase-II/III trials are required to establish efficacy. The obstacles to DR include identification of new indications with a high success rate in clinical studies, obtaining funding for clinical studies, patent protection, and approval systems. To tackle these obstacles, various approaches have been applied to DR worldwide. In this perspective, we provide representative examples of DR and discuss the ongoing efforts to overcome obstacles to DR in Japan.

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Value and specificity of rare diseases business model-is the pursuit of this societal priority sustainable?

Cited by 2 publications

References 1 publication

Principles for consistent value assessment and sustainable funding of orphan drugs in Europe

Principles for consistent value assessment and sustainable funding of orphan drugs in Europe

Overcoming Obstacles to Drug Repositioning in Japan

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