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Rare diseases and orphan drugs
1 references·9 citing·42 second hop
Rare diseases and orphan drugs (2006)Rare diseases and orphan drugsOrphan drugs and the NHS: should we value rarity? (2005)Orphan drugs and the NHS: sho…Neuropsychological assessment in rare pediatric neurogenetic disorders: considerations for cross-cultural clinical research (2023)Neuropsychological assessment…The Grand Challenge of Human Health: A Review and an Urgent Call for Business–Health Research (2022)The Grand Challenge of Human …9th European Conference on Rare Diseases & Orphan Products (ECRD Vienna 2018) (2018)9th European Conference on Ra…Crowdfunding for Personalized Medicine Research. (2015)Crowdfunding for Personalized…Orphan drugs: the regulatory environment (2012)Orphan drugs: the regulatory …Global Spending on Orphan Drugs in France, Germany, the UK, Italy and Spain during 2007 (2010)Global Spending on Orphan Dru…Orphan drug development is not taking off (2009)Orphan drug development is no…Exploring emerging technologies using metaphors – A study of orphan drugs and pharmacogenomics (2008)Exploring emerging technologi…Orphan drugs: legal aspects, current situation (2002)Orphan drugs: legal aspects, …Orphan drug development is progressing too slowly (2006)Why rare diseases are an important medical and social issue (2008)An Empirical Review of Major Legislation Affecting Drug Development: Past Experiences, Effects, and Unintended Consequences (2011)Rare lung disease and orphan drug development (2013)Why we should care about ultra-rare disease (2016)Cost-Effectiveness Analysis and the Consistency of Decision Making (2001)National Institute for Clinical Excellence and its value judgments (2004)Issues Surrounding Orphan Disease and Orphan Drug Policies in Europe (2010)Drugs for Rare Diseases: Influence of Orphan Designation Status on Price (2011)Unintended effects of orphan product designation for rare neurological diseases (2012)The social control of technology (1981)The Social Control of Technology. By David Collingridge. (New York: St. Martin's Press, 1980. Pp. i + 200. $22.50.) (1982)Metaphors We Live By (2003)Design process outputs as boundary objects in agricultural innovation projects: Functions and limitations (2012)Easing the assessment of emerging technologies in technology observatories. Findings about patterns of dissemination of emerging technologies on the internet (2017)How to Value Orphan Drugs? A Review of European Value Assessment Frameworks (2021)Emergence of orphan drugs in the United States: a quantitative assessment of the first 25 years (2010)Rare diseases, orphan drugs and their regulation: questions and misconceptions (2010)Rare diseases and orphan drugs (2012)Orphan drugs policies: a suitable case for treatment (2014)The availability and affordability of orphan drugs for rare diseases in China (2016)Orphan medicinal products in Europe and United States to cover needs of patients with rare diseases: an increased common effort is to be foreseen (2017)QALYfying the value of life. (1987)Explaining the Identifiable Victim Effect (1997)A rational framework for decision making by the National Institute For Clinical Excellence (NICE) (2002)Public healthcare resource allocation and the Rule of Rescue (2008)SOCIETAL VIEWS ON NICE, CANCER DRUGS FUND AND VALUE‐BASED PRICING CRITERIA FOR PRIORITISING MEDICINES: A CROSS‐SECTIONAL SURVEY OF 4118 ADULTS IN GREAT BRITAIN (2012)Multiple Criteria Decision Analysis (MCDA) for evaluating new medicines in Health Technology Assessment and beyond: The Advance Value Framework (2017)How to Design and Report Experiments (2003)Clinical and Translational Science (2010)A Coefficient of Agreement for Nominal Scales (1960)The Measurement of Observer Agreement for Categorical Data (1977)The Iron Cage Revisited: Institutional Isomorphism and Collective Rationality in Organizational Fields (1983)The SDGs : A change agenda shaping the future of business and humanity at large (2022)Tracing the connections between international business and communicable diseases (2022)Using review articles to address societal grand challenges (2023)The impact of culture and education on non-verbal neuropsychological measurements: A critical review (2003)Increasing Culturally Competent Neuropsychological Services for Ethnic Minority Populations: A Call to Action (2010)Vineland Adaptive Behavior Scales (2017)Cross‐Cultural Paediatric Neuropsychological Assessment: Key Considerations (2025)Why orphan drug coverage reimbursement decision-making needs patient and public involvement (2015)Is Crowdfunding Suitable for Financing German Public Research Organization (PRO) Projects? (2019)
this work references cited by second hop

References

  • 2005Orphan drugs and the NHS: should we value rarity?229 cited

Cited by

  • 2012Orphan drugs: the regulatory environment144 cited
  • 2002Orphan drugs: legal aspects, current situation79 cited
  • 2008Exploring emerging technologies using metaphors – A study of orphan drugs and pharmacogenomics74 cited
  • 2009Orphan drug development is not taking off63 cited
  • 2010Global Spending on Orphan Drugs in France, Germany, the UK, Italy and Spain during 200757 cited
  • 2022The Grand Challenge of Human Health: A Review and an Urgent Call for Business–Health Research43 cited
  • 2015Crowdfunding for Personalized Medicine Research.11 cited
  • 2023Neuropsychological assessment in rare pediatric neurogenetic disorders: considerations for cross-cultural clinical research7 cited
  • 20189th European Conference on Rare Diseases & Orphan Products (ECRD Vienna 2018)6 cited

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