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The Evolving Drug Development Landscape: From Blockbusters to Niche Busters in the Orphan Drug Space
12 references·1 citing·38 second hop
The Evolving Drug Development Landscape: From Blockbusters to Niche Busters in the Orphan Drug Space (2014)The Evolving Drug Development Landscape: …What Is Wrong with Orphan Drug Policies? (2012)What Is Wrong with Orphan Dru…Orphan drug development: an economically viable strategy for biopharma R&D (2012)Orphan drug development: an e…Rare Diseases and Orphan Products (2011)Rare Diseases and Orphan Prod…Pricing and reimbursement of orphan drugs: the need for more transparency (2011)Pricing and reimbursement of …Accelerating access to treatments for rare diseases (2011)Accelerating access to treatm…Emergence of orphan drugs in the United States: a quantitative assessment of the first 25 years (2010)Emergence of orphan drugs in …Translation of rare disease research into orphan drug development: disease matters (2009)Translation of rare disease r…Incentives for orphan drug research and development in the United States (2008)Incentives for orphan drug re…Assessing the economic challenges posed by orphan drugs (2007)Assessing the economic challe…Are rare diseases still orphans or happily adopted? The challenges of developing and using orphan medicinal products (2006)Are rare diseases still orpha…Two decades of orphan product development (2002)Two decades of orphan product…Orphan Drug Act (1990)Orphan Drug ActBudgetary Impact and Cost Drivers of Drugs for Rare and Ultrarare Diseases (2017)Budgetary Impact and Cost Dri…Drugs for exceptionally rare diseases: do they deserve special status for funding? (2005)Orphan drugs and the NHS: should we value rarity? (2005)A discrete choice experiment investigating preferences for funding drugs used to treat orphan diseases: an exploratory study (2010)Access to orphan drugs despite poor quality of clinical evidence (2010)Availability of and Access to Orphan Drugs (2010)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)Stratified medicine: strategic and economic implications of combining drugs and clinical biomarkers (2007)The importance of new companies for drug discovery: origins of a decade of new drugs (2010)Estimating the clinical cost of drug development for orphan versus non-orphan drugs (2019)Special FDA designations for drug development: orphan, fast track, accelerated approval, priority review, and breakthrough therapy (2023)Official Journal of the European Communities (1984)The price of innovation: new estimates of drug development costs (2003)Estimating The Cost Of New Drug Development: Is It Really $802 Million? (2006)An Empirical Review of Major Legislation Affecting Drug Development: Past Experiences, Effects, and Unintended Consequences (2011)The potential investment impact of improved access to accelerated approval on the development of treatments for low prevalence rare diseases (2011)Incremental cost per quality-adjusted life year gained? The need for alternative methods to evaluate medical interventions for ultra-rare disorders (2014)Turning a Blind Eye (2013)Orphan drugs policies: a suitable case for treatment (2014)Patient Access to Medicines for Rare Diseases in European Countries (2018)Establishing a reasonable price for an orphan drug (2020)A journey of hope: lessons learned from studies on rare diseases and orphan drugs (2006)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)Cost-Effectiveness Analysis and the Consistency of Decision Making (2001)National Institute for Clinical Excellence and its value judgments (2004)QALY maximisation and people's preferences: a methodological review of the literature (2004)Paying for the Orphan Drug System: break or bend? Is it time for a new evaluation system for payers in Europe to take account of new rare disease treatments? (2012)Does NICE have a cost‐effectiveness threshold and what other factors influence its decisions? A binary choice analysis (2004)Access to Orphan Drugs: A Comprehensive Review of Legislations, Regulations and Policies in 35 Countries (2015)Ethical imperatives of timely access to orphan drugs: is possible to reconcile economic incentives and patients’ health needs? (2017)Strategic Corporate Social Responsibility and Orphan Drug Development: Insights from the US and the EU Biopharmaceutical Industry (2012)Orphan Drugs and Their Impact on Pharmaceutical Development (2018)
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References

  • 2007Assessing the economic challenges posed by orphan drugs279 cited
  • 2011Pricing and reimbursement of orphan drugs: the need for more transparency261 cited
  • 2012Orphan drug development: an economically viable strategy for biopharma R&D161 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
  • 2012What Is Wrong with Orphan Drug Policies?110 cited
  • 2006Are rare diseases still orphans or happily adopted? The challenges of developing and using orphan medicinal products88 cited
  • 2009Translation of rare disease research into orphan drug development: disease matters75 cited
  • 1990Orphan Drug Act63 cited
  • 2011Accelerating access to treatments for rare diseases62 cited

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  • 2017Budgetary Impact and Cost Drivers of Drugs for Rare and Ultrarare Diseases47 cited

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