scholarly journals Orphan Medicine Incentives: How to Address the Unmet Needs of Rare Disease Patients by Optimizing the European Orphan Medicinal Product Landscape Guiding Principles and Policy Proposals by the European Expert Group for Orphan Drug Incentives (OD Expert Group)

2021 ◽  
Vol 12 ◽  
Author(s):  
Annemieke Aartsma-Rus ◽  
Marc Dooms ◽  
Yann Le Cam
Keyword(s):  
Medicinal Product ◽  
Rare Disease ◽  
Orphan Drug ◽  
Unmet Needs ◽  
Policy Framework ◽  
Expert Group ◽  
Orphan Medicinal Product ◽  
Policy Makers ◽  
Guiding Principles ◽  
Policy Proposals

Today policy makers face the challenge to devise a policy framework that improves orphan medicinal product (OMP) development by creating incentives to deliver treatments where there are none and to authorize innovative and transformative treatments where treatments already exist. The European Expert Group on Orphan Drug Incentives (hereafter, OD Expert Group) came together in 2020 to develop policy proposals to facilitate EU policy makers to meet this challenge. The group brings together representatives of the broad rare disease community, including researchers, academia, patient representatives, members of the investor community, rare disease companies and trade associations. The group’s work builds on the recognition that only an ambitious policy agenda developed in a multi-stakeholder setting can bring about the quantum leap needed to address unmet needs of rare disease patients today. Along the OMP development path, the OD Expert Group has identified four main needs that a policy revision should address: 1) Need to improve the R&D ecosystem for basic research and company take-up of development. 2) Need to improve the system of financial incentives and rewards. 3) Need to improve the flexibility, predictability and speed of the regulatory pathway. 4) Need to improve the coherence and predictability of demand and pricing for OMPs. This article presents the results of the OD Expert Group work as a set of guiding principles that the revision of the policy framework should follow and a set of 14 policy proposals that address the main needs of OMP development in Europe today.

scholarly journals The urgent need to empower rare disease organizations in China: an interview-based study

2020 ◽  
Vol 15 (1) ◽  
Author(s):  
Xuefeng Li ◽  
Zijuan Lu ◽  
Jianyong Zhang ◽  
Xiangyu Zhang ◽  
Shu Zhang ◽  
...  
Keyword(s):  
Rare Disease ◽  
Orphan Drug ◽  
Rare Diseases ◽  
General Public ◽  
Unmet Needs ◽  
Financial Resources ◽  
Western Countries ◽  
Disease Awareness ◽  
Patient Organizations ◽  
The Government

Abstract Background Each rare disease only affects a small number of population. However, a total of 7000 rare diseases may affect 10% of the population. Due to the severity and lack of rare disease awareness, rare disease represents a huge challenge for the healthcare system. In Western countries, patient organizations have been playing an integral role in raising awareness, advocating legislation, and supporting drug development. This study aims to assess the unmet needs of rare disease patient organizations in China, and identify their unmet needs, providing essential information for the government and legislators. Results A total of 28 individuals representing 28 patient organizations in China were interviewed. Most organizations do not have official registration, employees, written standard operation protocol, or reliable financial resources. Misdiagnosis or delayed diagnosis is common, and treatment is often lacking. Due to the lack of financial resources, no organizations have been able to sponsor academic research, unlike their counterparts in Western countries. As to challenges, 71.4% of interviewees listed lack of rare disease awareness among the general public, while 67.9% selected lack of financial resources. Further, only 7.3% of these organizations received support from the government, and 28.6% received support from the general public. As to recommendations to the government, 82.1% of interviewees selected special insurance programs for rare diseases because rare diseases have been generally excluded from the national medical insurance programs. In addition, 78.6% of interviewees recommended to stimulate rare disease research, 75% recommended to import orphan drugs, and 71.4% recommended legislation of an orphan drug act, highlighting the urgent need of therapies. Conclusions Due to lack of support and rare disease awareness, patient organizations in China are still in the early phase. To empower these patient organizations, the interviewees’ recommendations, including legislating orphan drug act and releasing official definition of rare diseases, should be considered by the government and legislators.

Europe

2020 ◽  
pp. 649-666
Author(s):  
Andrea Renda
Keyword(s):  
Artificial Intelligence ◽  
Digital Technology ◽  
Policy Framework ◽  
Expert Group ◽  
Ethical Guidelines ◽  
Ethical Implications ◽  
Strong Focus ◽  
Ethics Guidelines ◽  
Definition Of ◽  
High Level

This chapter assesses Europe’s efforts in developing a full-fledged strategy on the human and ethical implications of artificial intelligence (AI). The strong focus on ethics in the European Union’s AI strategy should be seen in the context of an overall strategy that aims at protecting citizens and civil society from abuses of digital technology but also as part of a competitiveness-oriented strategy aimed at raising the standards for access to Europe’s wealthy Single Market. In this context, one of the most peculiar steps in the European Union’s strategy was the creation of an independent High-Level Expert Group on AI (AI HLEG), accompanied by the launch of an AI Alliance, which quickly attracted several hundred participants. The AI HLEG, a multistakeholder group including fifty-two experts, was tasked with the definition of Ethics Guidelines as well as with the formulation of “Policy and Investment Recommendations.” With the advice of the AI HLEG, the European Commission put forward ethical guidelines for Trustworthy AI—which are now paving the way for a comprehensive, risk-based policy framework.

Socio economic and psychological dimensions of migration-induced exclusion of the elderly in Kerala, India: an empirical analysis

2016 ◽  
Vol 12 (1) ◽  
pp. 51-65
Author(s):  
Damodaran Rajasenan ◽  
M. S. Jayakumar ◽  
Bijith George Abraham
Keyword(s):  
The Elderly ◽  
Policy Framework ◽  
Policy Makers ◽  
Content Type ◽  
Left Behind ◽  
Emerging Culture ◽  
Principal Factors ◽  
Working Groups ◽  
The Government ◽  
Role Of The Government

Purpose – The purpose of this paper is to link the multifarious problems of the elderly in a socio-economic and psychological framework. Design/methodology/approach – The universe of the sample is elderly left behind in emigrant households in Kerala. In total, 600 samples were mustered using multistage stratified random sampling method. The paper, with the aid of factor analysis, χ2 and correspondence analysis, blemish the principal factors responsible for the migration-induced exclusion of the elderly. Findings – The empirical result derived from the study shows that migration-induced exclusion is all pervasive in Kerala. The elderly left behind yearn for the presence of their children rather than the emigration and concomitant remittances. Research limitations/implications – The findings of the study are helpful to the policy makers to understand the issues faced by the elderly and include all stakeholders concerned to find a solution to tackle these problems faced by the elderly due to emigration of their children. Practical implications – The study is practically relevant in developing appropriate policy framework in Kerala as it illumines the role of the government to overcome the exclusionary trend and other manifold problems of the elderly. Social implications – The study sheds light to a new social problem developing in the state in the form of elderly exclusion owing to emigration of the young working groups in regional dimensions, demographic levels, community angles and the emerging culture of old age home in the Kerala economy and society. Originality/value – The study is a unique one and tries to situate the principal factors responsible for the emigration-induced exclusion of the elderly in Kerala with empirical evidence.

Where now for equity and effectiveness policy?

2018 ◽  
pp. 205-214
Author(s):  
Stephen Gorard
Keyword(s):  
Education Policy ◽  
Education System ◽  
Child Poverty ◽  
Short Term ◽  
Policy Makers ◽  
Root Cause ◽  
Specific Policy ◽  
Policy Proposals ◽  
Effective Education

This chapter considers solutions for a more effective education policy. Education mostly appears to reflect society, which suggests that the root cause of inequality is at least partly not educational. Education policy cannot be expected to solve issues such as child poverty alone, in the short term, or even at all. This means that education policy has to be humbler, but it still has important roles to play — in ensuring that inequalities are not worsened by the education system, and by promoting structures and interventions that can ‘compensate for society’, to some extent. In this light, this chapter provides examples of specific policy proposals, considers where policy-makers have erred, and shows how policy can work with research.

scholarly journals Assessment of electricity distribution business model and market design alternatives: Evidence for policy design

2018 ◽  
Vol 31 (1) ◽  
pp. 40-59 ◽  
Author(s):  
Guillermo Ivan Pereira ◽  
Patrícia Pereira da Silva ◽  
Deborah Soule
Keyword(s):  
Business Model ◽  
Delphi Study ◽  
Distribution System ◽  
Policy Design ◽  
Market Design ◽  
Electricity Sector ◽  
Policy Framework ◽  
Electricity Distribution ◽  
Policy Makers ◽  
Evidence For Policy

The transition toward smart electricity distribution grids is transforming the European electricity sector. This has contributed to increased attention by policy makers regarding the future role of electricity distribution system operators in a more flexible, digital, and renewables-based electricity system. A better understanding on how to support the adaptation of the electricity distribution industry can contribute to the introduction of an effective policy framework. Our research provides evidence for policy design by presenting the results of a Policy Delphi study focused on business model and market design adaptation alternatives. We highlight the importance of supporting innovation and a more proactive approach to adaptation from both distribution system operators and regulators. Our findings support the importance of electricity distribution for neutral market facilitation, contributing to market development and enabling new market players. The results obtained support policy makers working on electricity sector adaptation and can contribute to the ongoing market redesign efforts under the Energy Union.

Housing delivery problems in developing countries: a case study of Nigeria

2016 ◽  
Vol 14 (4) ◽  
pp. 315-329 ◽  
Author(s):  
Musa Mohammed Mukhtar ◽  
Roslan Amirudin ◽  
Ismail Mohamad
Keyword(s):  
Housing Policy ◽  
Building Materials ◽  
Vision 2020 ◽  
Policy Makers ◽  
Content Type ◽  
Depth Analysis ◽  
Guiding Principles ◽  
Efficiency And Productivity ◽  
National Housing

Purpose The purpose of this paper is to examine problems of housing delivery in Nigeria and propose some guiding principles that will lead to successful housing delivery in Nigeria. Design/methodology/approach The study was conducted through in-depth analysis of some documents related to housing sector in Nigeria. These include National Housing Policy of Nigeria, Report of the Vision 2020 National Technical Working Group on Housing, as well as publications from UN-Habitat. Moreover, literature on the subject matter have been also reviewed. Findings Major constraints to housing delivery in Nigeria includes lack of effective housing finance system, unstable macroeconomic environment, difficulty in accessing land with secure tenure, high cost of building materials, shortages of skilled labour and poor infrastructural facilities. Research limitations/implications The major limitation of this study is that no interview or field survey to collect data from stakeholders has been performed. Practical implications The study can assist housing policy makers to understands important elements that must be incorporated in the national housing policies. It can also assist construction industries to understand how to improve efficiency and productivity in their projects. Originality/value The findings of this paper was based on previous studies of housing delivery and analysis of data from some formal and informal documents The findings from this study have been used to suggest some guiding principles that can assist in solving the housing delivery problems in Nigeria.

Assessing Reserve Adequacy - Further Considerations

Policy Papers ◽  
2013 ◽  
Vol 2013 (89) ◽  
Author(s):  
Keyword(s):  
Policy Framework ◽  
Policy Makers ◽  
Credit Lines ◽  
Crisis Prevention ◽  
Transitory Shocks ◽  
Good Policy ◽  
Market Pressures

Reserves remain a critical liquidity buffer for most countries. They are generally associated with lower crisis risks (crisis prevention) as well as space for authorities to respond to shocks (crisis mitigation). While other instruments, such as official credit lines and bilateral swap lines, are also external buffers, for most countries they principally act as a complement to their official reserves. For countries with sound fundamentals and a good policy framework, reserves provide policy makers with considerable space to respond to transitory shocks. However, this space diminishes as fundamentals deteriorate and the existence of adequate reserves does not, by itself, eliminate the risk of market pressures.

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