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Unintended effects of orphan product designation for rare neurological diseases
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Unintended effects of orphan product designation for rare neurological diseases (2012)Unintended effects of orphan product desi…Rare Diseases and Orphan Products (2011)Rare Diseases and Orphan Prod…Using Market-Exclusivity Incentives to Promote Pharmaceutical Innovation (2010)Using Market-Exclusivity Ince…Orphan drug pricing may warrant a competition law investigation (2010)Orphan drug pricing may warra…Incentives for Drug Development — The Curious Case of Colchicine (2010)Incentives for Drug Developme…Global Spending on Orphan Drugs in France, Germany, the UK, Italy and Spain during 2007 (2010)Global Spending on Orphan Dru…Emergence of orphan drugs in the United States: a quantitative assessment of the first 25 years (2010)Emergence of orphan drugs in …A comparative study of European rare disease and orphan drug markets (2010)A comparative study of Europe…The US Orphan Drug Act: Rare disease research stimulator or commercial opportunity? (2009)The US Orphan Drug Act: Rare …Balancing Innovation, Access, and Profits — Market Exclusivity for Biologics (2009)Balancing Innovation, Access,…Tier 4 Drugs and the Fraying of the Social Compact (2008)Tier 4 Drugs and the Fraying …The use of cost-effectiveness by the National Institute for Health and Clinical Excellence (NICE): no(t yet an) exemplar of a deliberative process (2008)The use of cost-effectiveness…Incentives for orphan drug research and development in the United States (2008)Incentives for orphan drug re…Orphan drug development is progressing too slowly (2006)Orphan drug development is pr…A journey of hope: lessons learned from studies on rare diseases and orphan drugs (2006)A journey of hope: lessons le…Orphan drugs and the NHS: should we value rarity? (2005)Orphan drugs and the NHS: sho…Two decades of orphan product development (2002)Two decades of orphan product…Adopting Orphan Drugs — Two Dozen Years of Treating Rare Diseases (2006)Why rare diseases are an important medical and social issue (2008)Orphan drugs: the regulatory environment (2012)The Roles Of Academia, Rare Diseases, And Repurposing In The Development Of The Most Transformative Drugs (2015)Drug discovery effectiveness from the standpoint of therapeutic mechanisms and indications (2017)In the Netherlands (1961)National Institute for Clinical Excellence and its value judgments (2004)Commonalities and differences in HTA outcomes: A comparative analysis of five countries and implications for coverage decisions (2012)Rare diseases, orphan drugs, and their regulation in Asia: Current status and future perspectives (2012)Recommendations from the European Working Group for Value Assessment and Funding Processes in Rare Diseases (ORPH-VAL) (2017)Economics of New Oncology Drug Development (2007)Market incentives and pharmaceutical innovation (2008)Open-source approaches for the repurposing of existing or failed candidate drugs: learning from and applying the lessons across diseases (2013)Using four decades of FDA orphan drug designations to describe trends in rare disease drug development: substantial growth seen in development of drugs for rare oncologic, neurologic, and pediatric-onset diseases (2021)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)Drugs for exceptionally rare diseases: do they deserve special status for funding? (2005)Challenges in Research and Health Technology Assessment of Rare Disease Technologies: Report of the ISPOR Rare Disease Special Interest Group (2018)Decisions with Multiple Objectives: Preferences and Value Tradeoffs. (1994)Development as Freedom (1999)Development as Freedom (2009)A discrete choice experiment investigating preferences for funding drugs used to treat orphan diseases: an exploratory study (2010)Incremental cost per quality-adjusted life year gained? The need for alternative methods to evaluate medical interventions for ultra-rare disorders (2014)Public Reasoning and Health-Care Priority Setting: The Case of NICE (2017)What Is Wrong with Orphan Drug Policies? (2012)Ethical imperatives of timely access to orphan drugs: is possible to reconcile economic incentives and patients’ health needs? (2017)Orphan drug development: an economically viable strategy for biopharma R&D (2012)Strategic Corporate Social Responsibility and Orphan Drug Development: Insights from the US and the EU Biopharmaceutical Industry (2012)Orphan drugs policies: a suitable case for treatment (2014)Orphan Drugs and Their Impact on Pharmaceutical Development (2018)
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References

  • 2005Orphan drugs and the NHS: should we value rarity?229 cited
  • 2006A journey of hope: lessons learned from studies on rare diseases and orphan drugs185 cited
  • 2009The US Orphan Drug Act: Rare disease research stimulator or commercial opportunity?166 cited
  • 2010Emergence of orphan drugs in the United States: a quantitative assessment of the first 25 years148 cited
  • 2002Two decades of orphan product development138 cited
  • 2008Incentives for orphan drug research and development in the United States138 cited
  • 2011Rare Diseases and Orphan Products135 cited
  • 2010A comparative study of European rare disease and orphan drug markets121 cited
  • 2008The use of cost-effectiveness by the National Institute for Health and Clinical Excellence (NICE): no(t yet an) exemplar of a deliberative process78 cited
  • 2010Incentives for Drug Development — The Curious Case of Colchicine77 cited
  • 2010Using Market-Exclusivity Incentives to Promote Pharmaceutical Innovation70 cited
  • 2006Orphan drug development is progressing too slowly70 cited
  • 2009Balancing Innovation, Access, and Profits — Market Exclusivity for Biologics69 cited
  • 2008Tier 4 Drugs and the Fraying of the Social Compact67 cited
  • 2010

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Global Spending on Orphan Drugs in France, Germany, the UK, Italy and Spain during 2007
57 cited
  • 2010Orphan drug pricing may warrant a competition law investigation53 cited
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