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Orphan Drugs and Their Impact on Pharmaceutical Development
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Orphan Drugs and Their Impact on Pharmaceutical Development (2018)Orphan Drugs and Their Impact on Pharmace…Trends in clinical success rates (2016)Trends in clinical success ra…The economics of biosimilars. (2013)The economics of biosimilars.Rare diseases and orphan drugs (2012)Rare diseases and orphan drugsPharmaceutical Research and Development: What Do We Get for All that Money? (2012)Pharmaceutical Research and D…Orphan drug development: an economically viable strategy for biopharma R&D (2012)Orphan drug development: an e…Diagnosing the decline in pharmaceutical R&D efficiency (2012)Diagnosing the decline in pha…The productivity crisis in pharmaceutical R&D (2011)The productivity crisis in ph…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 …How to improve R&D productivity: the pharmaceutical industry's grand challenge (2010)How to improve R&D productivi…Pharmaceutical R&D: the road to positive returns (2009)Pharmaceutical R&D: the road …The State of Innovation in Drug Development (2008)The State of Innovation in Dr…Rise and fall of modern medicine (1999)Can the pharmaceutical industry reduce attrition rates? (2004)Lessons learned from the fate of AstraZeneca's drug pipeline: a five-dimensional framework (2014)Are Ideas Getting Harder to Find? (2020)Generating Theory by Abduction (2021)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)Drug discovery effectiveness from the standpoint of therapeutic mechanisms and indications (2017)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)The price of innovation: new estimates of drug development costs (2003)Lessons from 60 years of pharmaceutical innovation (2009)Market spiral pricing of cancer drugs (2013)Are new models needed to optimize the utilization of new medicines to sustain healthcare systems? (2014)R&D Costs of New Medicines: A Landscape Analysis (2021)Protecting Their Intellectual Assets: Appropriability Conditions and Why U.S. Manufacturing Firms Patent (or Not) (2000)Changing R&D models in research-based pharmaceutical companies (2016)A Decade of FDA-Approved Drugs (2010–2019): Trends and Future Directions (2021)How Much Does It Cost to Research and Develop a New Drug? A Systematic Review and Assessment (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)A Proof for the Queuing Formula: L = λ W (1961)The Goal: A Process of Ongoing Improvement (1984)Innovation in the pharmaceutical industry: New estimates of R&D costs (2016)Estimated Research and Development Investment Needed to Bring a New Medicine to Market, 2009-2018 (2020)Orphan drugs: the regulatory environment (2012)Rare diseases, orphan drugs, and their regulation in Asia: Current status and future perspectives (2012)Why Are Biosimilars Not Living up to Their Promise in the US? (2019)Strategies to achieve fairer prices for generic and biosimilar medicines (2020)Expanding access to biotherapeutics in low-income and middle-income countries through public health non-exclusive voluntary intellectual property licensing: considerations, requirements, and opportunities (2022)Translation of rare disease research into orphan drug development: disease matters (2009)An Improved Approach To Measuring Drug Innovation Finds Steady Rates Of First-In-Class Pharmaceuticals, 1987–2011 (2013)Behavioral profiling as a translational approach in an animal model of posttraumatic stress disorder (2016)Measuring what matters to rare disease patients – reflections on the work by the IRDiRC taskforce on patient-centered outcome measures (2017)Approval success rates of drug candidates based on target, action, modality, application, and their combinations (2021)
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References

  • 2010How to improve R&D productivity: the pharmaceutical industry's grand challenge3,562 cited
  • 2012Diagnosing the decline in pharmaceutical R&D efficiency1,978 cited
  • 2011The productivity crisis in pharmaceutical R&D918 cited
  • 2016Trends in clinical success rates277 cited
  • 2008The State of Innovation in Drug Development276 cited
  • 2011Pricing and reimbursement of orphan drugs: the need for more transparency261 cited
  • 2012Pharmaceutical Research and Development: What Do We Get for All that Money?256 cited
  • 2013The economics of biosimilars.225 cited
  • 2012Rare diseases and orphan drugs214 cited
  • 2012Orphan drug development: an economically viable strategy for biopharma R&D161 cited
  • 2011Rare Diseases and Orphan Products135 cited
  • 2009Pharmaceutical R&D: the road to positive returns95 cited

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