Drug Approval

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  Subject Areas on Research

  • A Systematic Review of U.S. Biosimilar Approvals: What Evidence Does the FDA Require and How Are Manufacturers Responding? 
  • A comparison of moment-based and probability-based criteria for assessment of follow-on biologics. 
  • A note on special articles on adaptive clinical trial designs. 
  • A pre-marketing ALT signal predicts post-marketing liver safety. 
  • Accountability in Patenting of Federally Funded Research 
  • An application of the revised CONSORT standards to FDA summary reports of recently approved antidepressants and antipsychotics. 
  • Anti-VEGF therapies in the clinic. 
  • Application of the parallel line assay to assessment of biosimilar products based on binary endpoints. 
  • Assessing biosimilarity and interchangeability of biosimilar products. 
  • Assessment of Clinical Trials Supporting US Food and Drug Administration Approval of Novel Therapeutic Agents, 1995-2017. 
  • Association Between Food and Drug Administration Advisory Committee Recommendations and Agency Actions, 2008-2015. 
  • Balancing the benefits and risks of inhaled long-acting beta-agonists--the influence of values. 
  • Balancing the cyclooxygenase portfolio. 
  • Balancing the risks and benefits of long-term antiplatelet therapies for cardiovascular disease: clinical, research, and regulatory implications. 
  • Benefit the patient, manage the risk: a system goal. 
  • Bevacizumab biosimilars: scientific justification for extrapolation of indications. 
  • Blinatumomab: A New Treatment for Adults With Relapsed Acute Lymphocytic Leukemia. 
  • Building a drug development database: challenges in reliable data availability. 
  • Cancer pharmacogenomics and pharmacoepidemiology: setting a research agenda to accelerate translation. 
  • Cardiovascular drug development: is it dead or just hibernating? 
  • Ceftaroline applications for therapy in the United States. 
  • Ceftaroline: clinical and microbiology experience with focus on methicillin-resistant Staphylococcus aureus after regulatory approval in the USA. 
  • Certolizumab pegol. 
  • Characteristics of Clinical Studies Used for US Food and Drug Administration Supplemental Indication Approvals of Drugs and Biologics, 2017 to 2019. 
  • Characteristics of Preapproval and Postapproval Studies of Vaccines Granted Accelerated Approval by the US Food and Drug Administration. 
  • Clinical and Technical Considerations for Brain PET Imaging for Dementia. 
  • Clinical research in the lay press: irresponsible journalism raises a huge dose of doubt. 
  • Clinical strategies for selecting oral anticoagulants in patients with atrial fibrillation. 
  • Clinical trials bureaucracy: unintended consequences of well-intentioned policy. 
  • Clinical utility of the Food and Drug Administration Electrocardiogram Warehouse: a paradigm for the critical pathway initiative. 
  • Comments on the FDA draft guidance on biosimilar products. 
  • Congress, the FDA, and the fair development of new medications for children. 
  • Cryoprecipitate therapy. 
  • Current knowledge of the platelet glycoprotein IIb/IIIa receptor antagonists for the treatment of coronary artery disease. 
  • Demographic Characteristics of Participants in Trials Essential to US Food and Drug Administration Vaccine Approvals, 2010-2020. 
  • Demonstrating effectiveness or demonstrating not ineffectiveness - A potential solution for rare disease drug product development? 
  • Developing Breakthrough Drugs for Heart Failure: Lessons Learned From the Cystic Fibrosis Experience. 
  • Developing Treatment Guidelines During a Pandemic Health Crisis: Lessons Learned From COVID-19. 
  • Developing drugs for developing countries. 
  • Disagreements Within the US Food and Drug Administration Regarding Approval of Novel Therapeutic Agents, 2011-2015. 
  • Documenting the rationale and psychometric characteristics of patient reported outcomes for labeling and promotional claims: the PRO Evidence Dossier. 
  • Drug Development in Kidney Disease: Proceedings From a Multistakeholder Conference. 
  • Drug Development. Are trade secrets delaying biosimilars? 
  • Duration of Pediatric Clinical Trials Submitted to the US Food and Drug Administration. 
  • Early dissemination of bevacizumab for advanced colorectal cancer: a prospective cohort study. 
  • Economic return of clinical trials performed under the pediatric exclusivity program. 
  • Economics of new oncology drug development. 
  • Efficacy of the Priority Review Voucher Program. 
  • Emergency Use Authorization of Remdesivir: The Need for a Transparent Distribution Process. 
  • Endpoint surrogacy in oncology Phase 3 randomised controlled trials. 
  • Endpoints in Heart Failure Drug Development: History and Future. 
  • Evaluation of Flibanserin: Science and Advocacy at the FDA. 
  • Evaluation of Representation of Women as Authors in Pivotal Trials Supporting US Food and Drug Administration Approval of Novel Cardiovascular Drugs. 
  • Evaluation of Trials Comparing Single-Enantiomer Drugs to Their Racemic Precursors: A Systematic Review. 
  • Exploring options for improving healthcare. 
  • Exposure Matching of Pediatric Anti-infective Drugs: Review of Drugs Submitted to the Food and Drug Administration for Pediatric Approval. 
  • FDA approval of dapagliflozin for chronic kidney disease: a remarkable achievement? 
  • FDA review vouchers. 
  • Facial Filler Complications. 
  • Feasibility of Using Real-world Data to Emulate Postapproval Confirmatory Clinical Trials of Therapeutic Agents Granted US Food and Drug Administration Accelerated Approval. 
  • Food and Drug Administration: Cardiovascular and Renal Drugs Advisory Committee, 98th meeting, January 6th-7th, 2003. 
  • Gene Therapy 2017: Progress and Future Directions. 
  • Generating comparative evidence on new drugs and devices after approval. 
  • Generic clopidogrel: time to substitute? 
  • HIV pre-exposure prophylaxis. 
  • Haloperidol for postoperative nausea and vomiting: are we reinventing the wheel? 
  • Harmonising regulatory approval for antibiotics in children. 
  • Hurry up and slow down: lessons in drug development from the COX-2 inhibitors. 
  • Idarucizumab since FDA approval: Use in the real-world. 
  • Impact of pediatric exclusivity on drug labeling and demonstrations of efficacy. 
  • Improving Cardiovascular Drug and Device Development and Evidence Through Patient-Centered Research and Clinical Trials: A Call to Action From the Value in Healthcare Initiative's Partnering With Regulators Learning Collaborative. 
  • Improving pharmaceutical innovation by building a more comprehensive database on drug development and use. 
  • Improving the assessment of heart toxicity for all new drugs through translational regulatory science. 
  • Improving the tools of clinical pharmacology: Goals for 2017 and beyond. 
  • Individual bioequivalence testing under 2x3 designs. 
  • Industry perspectives on ICH guidelines. 
  • Information Transparency in the Drug Approval Process. 
  • Innovative design and analysis for rare disease drug development. 
  • Introducing CCR perspectives in drug approval. 
  • Introduction of European priority review vouchers to encourage development of new medicines for neglected diseases. 
  • Introduction: fusidic acid enters the United States. 
  • Is conflict of interest becoming a challenge for institution-based institutional review boards? 
  • Making genuine progress against metastatic breast cancer. 
  • Marketing drugs too early in testing. 
  • Medical Marijuana Laws Reduce Prescription Medication Use In Medicare Part D. 
  • Medicine. A portfolio model of drug development for tuberculosis. 
  • NCCN Biosimilars White Paper: regulatory, scientific, and patient safety perspectives. 
  • Neuropsychiatric adverse effects of oseltamivir in the FDA Adverse Event Reporting System, 1999-2012. 
  • New FDA breakthrough-drug category--implications for patients. 
  • New rules for clinical trials of patients with acute bacterial skin and skin-structure infections: do not let the perfect be the enemy of the good. 
  • Noncollagen Dermal Fillers: A Summary of the Clinical Trials Used for Their FDA Approval. 
  • Off-label applications for SSRIs. 
  • Off-label uses for selective serotonin reuptake inhibitors. 
  • On flexibility of adaptive designs and criteria for choosing a good one--a discussion of FDA draft guidance. 
  • On safety margin for drug interchangeability. 
  • On sample size requirement for analytical similarity assessment. 
  • Patients, physicians, and clinical trials: the other side of the coins. 
  • Peripheral T-cell lymphoma, NOS, and anaplastic large cell lymphoma. 
  • Pharmacokinetics and safety of recently approved drugs used to treat methicillin-resistant Staphylococcus aureus infections in infants, children and adults. 
  • Premature release of data from clinical trials of ezetimibe. 
  • Prescribing BiDil: is it black and white? 
  • Prescribing rates and characteristics of recipients of tenofovir-containing regimens before and after market entry of tenofovir alafenamide. 
  • Priorities for the Priority Review Voucher. 
  • Pro: The Food and Drug Administration Black box warning on droperidol is not justified. 
  • Probability monitoring procedures for sample size determination. 
  • Proceedings of the 99th meeting of the Food and Drug Administration Cardiovascular and Renal Drugs Advisory Committee. May 29th and 30th, 2003. 
  • Proposals of statistical consideration to evaluation of results for a specific region in multi-regional trials--Asian perspective. 
  • Proposed study designs for approval based on a surrogate endpoint and a post-marketing confirmatory study under FDA's accelerated approval regulations for disease modifying osteoarthritis drugs. 
  • Rationing in urologic oncology: lessons from sipuleucel-T for advanced prostate cancer. 
  • Recommendations for an update of 2003 European regulatory requirements for registration of drugs to be used in the treatment of RA. 
  • Recommendations for the registration of agents for prevention and treatment of glucocorticoid-induced osteoporosis: an update from the Group for the Respect of Ethics and Excellence in Science. 
  • Regulatory Review of New Therapeutic Agents - FDA versus EMA, 2011-2015. 
  • Regulatory Review of New Therapeutic Agents. 
  • Regulatory and cost barriers are likely to limit biosimilar development and expected savings in the near future. 
  • Repairing the broken market for antibiotic innovation. 
  • Report from the 94th Cardiovascular and Renal Drugs Advisory Committee Meeting, October 11, 2001. 
  • Reproducibility probability in clinical trials. 
  • Research design features and patient characteristics associated with the outcome of antidepressant clinical trials. 
  • Roundtable on postherpetic neuralgia--what, why, how long, and what's next? 
  • Scientific considerations for assessing biosimilar products. 
  • Scientific factors and current issues in biosimilar studies. 
  • Selection bias, phase II trials, and the FDA accelerated approval process. 
  • Sensible guidelines for the conduct of large randomized trials. 
  • Some thoughts on drug interchangeability. 
  • Some thoughts on the QR method for analytical similarity evaluation. 
  • Speed, Evidence, and Safety Characteristics of Vaccine Approvals by the US Food and Drug Administration. 
  • Spending on postapproval drug safety. 
  • Statistical and regulatory considerations in assessments of interchangeability of biological drug products. 
  • Statistical assessment of biosimilar products. 
  • Statistical considerations for rare diseases drug development. 
  • Strategic use of statistical thinking in drug development. 
  • Suicidality as a possible side effect of antidepressant treatment. 
  • Systematic review: reliability of compendia methods for off-label oncology indications. 
  • Systematic survey of therapeutic trials for metastatic colorectal cancer: room for improvement in the critical pathway. 
  • Telavancin. 
  • Ten-Year Trends in Enrollment of Women and Minorities in Pivotal Trials Supporting Recent US Food and Drug Administration Approval of Novel Cardiometabolic Drugs. 
  • The 21st Century Cures Act. 
  • The Commercial Market For Priority Review Vouchers. 
  • The FDA in the 21st Century: How Is the FDA Responding to the New World? A Focus on Cardiovascular Drug Development. 
  • The Society for Clinical Trials opposes US legislation to permit marketing of unproven medical therapies for seriously ill patients. 
  • The balance of benefit and safety of rosiglitazone: important lessons for our system of drug development and postmarketing assessment. 
  • The evaluation of biosimilarity index based on reproducibility probability for assessing follow-on biologics. 
  • The price of innovation: new estimates of drug development costs. 
  • The roles of patents and research and development incentives in biopharmaceutical innovation. 
  • Trends in hepatitis C treatment uptake in the United States. 
  • Trial Design and Objectives for Castration-Resistant Prostate Cancer: Updated Recommendations From the Prostate Cancer Clinical Trials Working Group 3. 
  • U.S. Food and Drug Administration-Approved Poly (ADP-Ribose) Polymerase Inhibitor Maintenance Therapy for Recurrent Ovarian Cancer: A Cost-Effectiveness Analysis. 
  • US Food and Drug Administration Facilitated Pediatric Approval Programs: Application to Pediatric Neurological Disorders. 
  • US Food and Drug Administration utilization of postmarketing requirements and postmarketing commitments, 2009-2018. 
  • Understanding the sleep-wake cycle: sleep, insomnia, and the orexin system. 
  • Updating the 2003 European regulatory requirements for registering disease-modifying drugs to be used in the treatment of rheumatoid arthritis. 
  • Use Patents Can Be Useful: The Case of Rescued Drugs 
  • Use of larger versus smaller drug-safety databases before regulatory approval: the trade-offs. 
  • Using Medicare administrative data to conduct postmarketing surveillance of follow-on biologics: issues and opportunities. 
  • Vaccine Development: Steps to Approval of an Investigational Vaccine. 
  • Value in Healthcare Initiative: Summary and Key Recommendations. 
  • Vorapaxar and diplopia: Possible off-target PAR-receptor mismodulation. 
  • What should work, may not. 
  • Who should go first in trials with scarce agents? The views of potential participants. 
  • Will Precision Medicine Move Us beyond Race? 
  • Will a new tenofovir prodrug for the treatment of HIV reduce the risk of nephrotoxicity? 
  • Will biosimilars gain momentum? 
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  Keywords of People

  • Cohen-Wolkowiez, Michael, Professor of Pediatrics, Pediatrics, Infectious Diseases
  • Grambow, Steven C., Associate Professor of Biostatistics & Bioinformatics, Biostatistics & Bioinformatics
  • Reed, Shelby Derene, Professor in Population Health Sciences, Duke Science & Society
  • Ridley, David Blaine, Professor of the Practice of Business Adminstration, Duke Science & Society
  • Zafar, Syed Yousuf, Adjunct Professor in the Department of Medicine, Duke Science & Society