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Special FDA designations for drug development: orphan, fast track, accelerated approval, priority review, and breakthrough therapy
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Special FDA designations for drug development: orphan, fast track, accelerated approval, priority review, and breakthrough therapy (2023)Special FDA designations for drug develop…A comprehensive study of the rare diseases and conditions targeted by orphan drug designations and approvals over the forty years of the Orphan Drug Act (2023)A comprehensive study of the …2020 FDA drug approvals (2021)2020 FDA drug approvalsFDA Approval and Regulation of Pharmaceuticals, 1983-2018 (2020)FDA Approval and Regulation o…Design characteristics, risk of bias, and reporting of randomised controlled trials supporting approvals of cancer drugs by European Medicines Agency, 2014-16: cross sectional analysis (2019)Design characteristics, risk …Assessment of the Clinical Benefit of Cancer Drugs Receiving Accelerated Approval (2019)Assessment of the Clinical Be…Characteristics of Preapproval and Postapproval Studies for Drugs Granted Accelerated Approval by the US Food and Drug Administration (2017)Characteristics of Preapprova…Access to Orphan Drugs: A Comprehensive Review of Legislations, Regulations and Policies in 35 Countries (2015)Access to Orphan Drugs: A Com…Dealing with uncertainty and high prices of new medicines: A comparative analysis of the use of managed entry agreements in Belgium, England, the Netherlands and Sweden (2014)Dealing with uncertainty and …Clinical development success rates for investigational drugs (2014)Clinical development success …Managed entry agreements for pharmaceuticals: the European experience (2013)Managed entry agreements for …Influence of trial sample size on treatment effect estimates: meta-epidemiological study (2013)Influence of trial sample siz…Orphan drug development: an economically viable strategy for biopharma R&D (2012)Orphan drug development: an e…The Complications of Controlling Agency Time Discretion: FDA Review Deadlines and Postmarket Drug Safety (2011)The Complications of Controll…Pricing and reimbursement of orphan drugs: the need for more transparency (2011)Pricing and reimbursement of …Characteristics of Clinical Trials to Support Approval of Orphan vs Nonorphan Drugs for Cancer (2011)Characteristics of Clinical T…Accelerated Approval of Oncology Products: The Food and Drug Administration Experience (2011)Accelerated Approval of Oncol…Recommendations for examining and interpreting funnel plot asymmetry in meta-analyses of randomised controlled trials (2011)The Cochrane Collaboration's tool for assessing risk of bias in randomised trials (2011)Comparative efficacy and acceptability of psychotherapies for depression in children and adolescents: A systematic review and network meta-analysis (2015)Empirical Evidence of Study Design Biases in Randomized Trials: Systematic Review of Meta-Epidemiological Studies (2016)Evaluation of the Cochrane tool for assessing risk of bias in randomized clinical trials: overview of published comments and analysis of user practice in Cochrane and non-Cochrane reviews (2016)Cochrane Handbook for Systematic Reviews of Interventions (2020)The price of innovation: new estimates of drug development costs (2003)Can the pharmaceutical industry reduce attrition rates? 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(2019)Pricing of pharmaceuticals is becoming a major challenge for health systems (2020)CONSORT 2010 Explanation and Elaboration: updated guidelines for reporting parallel group randomised trials (2010)RoB 2: a revised tool for assessing risk of bias in randomised trials (2019)Prices and clinical benefit of cancer drugs in the USA and Europe: a cost–benefit analysis (2020)Reliability of the revised Cochrane risk-of-bias tool for randomised trials (RoB2) improved with the use of implementation instruction (2021)The Roles Of Academia, Rare Diseases, And Repurposing In The Development Of The Most Transformative Drugs (2015)Trends in utilization of FDA expedited drug development and approval programs, 1987-2014: cohort study (2015)Cardiovascular Drug Development (2015)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? 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References

  • 2014Clinical development success rates for investigational drugs2,531 cited
  • 2013Influence of trial sample size on treatment effect estimates: meta-epidemiological study543 cited
  • 2020FDA Approval and Regulation of Pharmaceuticals, 1983-2018377 cited
  • 2019Assessment of the Clinical Benefit of Cancer Drugs Receiving Accelerated Approval302 cited
  • 2015Access to Orphan Drugs: A Comprehensive Review of Legislations, Regulations and Policies in 35 Countries278 cited
  • 2011Pricing and reimbursement of orphan drugs: the need for more transparency261 cited
  • 2019Design characteristics, risk of bias, and reporting of randomised controlled trials supporting approvals of cancer drugs by European Medicines Agency, 2014-16: cross sectional analysis243 cited
  • 2011Characteristics of Clinical Trials to Support Approval of Orphan vs Nonorphan Drugs for Cancer230 cited
  • 2011Accelerated Approval of Oncology Products: The Food and Drug Administration Experience212 cited
  • 2017Characteristics of Preapproval and Postapproval Studies for Drugs Granted Accelerated Approval by the US Food and Drug Administration196 cited
  • 20212020 FDA drug approvals186 cited
  • 2013Managed entry agreements for pharmaceuticals: the European experience167 cited
  • 2012Orphan drug development: an economically viable strategy for biopharma R&D161 cited
  • 2014

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Dealing with uncertainty and high prices of new medicines: A comparative analysis of the use of managed entry agreements in Belgium, England, the Netherlands and Sweden
156 cited
  • 2023A comprehensive study of the rare diseases and conditions targeted by orphan drug designations and approvals over the forty years of the Orphan Drug Act134 cited
  • 2011The Complications of Controlling Agency Time Discretion: FDA Review Deadlines and Postmarket Drug Safety94 cited
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