Summary: Process that is gone through in order for a drug to receive approval by a government regulatory agency. This includes any required pre-clinical or clinical testing, review, submission, and evaluation of the applications and test results, and post-marketing surveillance of the drug.
Publications222 found, 100 shown here
- The price of innovation: new estimates of drug development costsJoseph A DiMasi
Tufts Center for the Study of Drug Development, Tufts University, 192 South Street, Suite 550, Boston, MA 02111, USA
J Health Econ 22:151-85. 2003..When compared to the results of an earlier study with a similar methodology, total capitalized costs were shown to have increased at an annual rate of 7.4% above general price inflation...
- Adaptive licensing: taking the next step in the evolution of drug approvalH G Eichler
European Medicines Agency, London, UK
Clin Pharmacol Ther 91:426-37. 2012....
- Worldwide experience with biosimilar developmentMark McCamish
Sandoz Biopharmaceuticals, Sandoz International GmbH, Holzkrichen, Germany
MAbs 3:209-17. 2011....
- FDA drug approval summary: erlotinib (Tarceva) tabletsMartin H Cohen
Division of Oncology Drug Products, Center for Drug Evaluation and Research, US Food and Drug Administration, HFD 150, 5600 Fishers Lane, Rockville, Maryland 20857, USA
Oncologist 10:461-6. 2005..In the first-line treatment of NSCLC, two large, controlled, randomized trials showed no benefit from adding erlotinib to doublet, platinum-based chemotherapy. Therefore, erlotinib is not indicated for use in this setting...
- FDA drug approval summary: bevacizumab (Avastin) plus Carboplatin and Paclitaxel as first-line treatment of advanced/metastatic recurrent nonsquamous non-small cell lung cancerMartin H Cohen
Division of Biological Oncology Products, Office of Oncology Drug Products, Center for Drug Evaluation and Research, US Food and Drug Administration, Silver Spring, Maryland 20993 0002, USA
Oncologist 12:713-8. 2007....
- Can the pharmaceutical industry reduce attrition rates?Ismail Kola
Basic Research at Merck Research Labs, 126 East Lincoln Avenue, Rahway, New Jersey 07075, USA
Nat Rev Drug Discov 3:711-5. 2004
- FDA drug approval summary: bevacizumab (Avastin) as treatment of recurrent glioblastoma multiformeMartin H Cohen
Division of Biological Oncology Products, Office of Oncology Drug Products, Center for Drug Evaluation and Research, U S Food and Drug Administration, Silver Spring, Maryland 20993 0002, USA
Oncologist 14:1131-8. 2009..g., CNS hemorrhage, wound-healing complications, and thromboembolic events) to either bevacizumab, underlying disease, or both could not be determined because of the single-arm, noncomparative study design...
- FDA drug approval summary: pegaspargase (oncaspar) for the first-line treatment of children with acute lymphoblastic leukemia (ALL)Patricia Anne Dinndorf
U S Food and Drug Administration, White Oak Campus, 10903 New Hampshire Avenue, Building 22, Room 2102, Silver Spring, Maryland 20993 0002, USA
Oncologist 12:991-8. 2007..Disclosure of potential conflicts of interest is found at the end of this article...
- From idea to market: the drug approval processM S Lipsky
Department of Family Medicine, Northwestern University Medical School, Chicago, IL, USA
J Am Board Fam Pract 14:362-7. 2001..Proponents claim that changes in the approval process help patients with debilitating diseases, such as acquired immunodeficiency syndrome, that were previously denied critical medication because of bureaucratic regulations...
- Analytical aspects of biosimilarity issues of protein drugsZsuzsanna Kálmán-Szekeres
Gedeon Richter Plc, 19 21 Gyömrői út, H 1103 Budapest, Hungary
J Pharm Biomed Anal 69:185-95. 2012....
- Bad bugs need drugs: an update on the development pipeline from the Antimicrobial Availability Task Force of the Infectious Diseases Society of AmericaGeorge H Talbot
Talbot Advisors, Wayne, Pennsylvania, USA
Clin Infect Dis 42:657-68. 2006....
- Trends in antimicrobial drug development: implications for the futureBrad Spellberg
Research and Education Institute and Department of Medicine, Harbor University of California, Los Angeles Medical Center, Torrance, California 90502, USA
Clin Infect Dis 38:1279-86. 2004..Despite the critical need for new antimicrobial agents, the development of these agents is declining. Solutions encouraging and facilitating the development of new antimicrobial agents are needed...
- Key factors in the rising cost of new drug discovery and developmentMichael Dickson
College of Pharmacy, University of South Carolina, Columbia, South Carolina 29208, USA
Nat Rev Drug Discov 3:417-29. 2004
- FDA drug approval summary: bevacizumab plus FOLFOX4 as second-line treatment of colorectal cancerMartin H Cohen
Division of Biological Oncology Products, Office of Oncology Drug Products, Center for Drug Evaluation and Research, US Food and Drug Administration, HFD 150, 5600 Fishers Lane, Rockville, Maryland 20857, USA
Oncologist 12:356-61. 2007....
- Economics of new oncology drug developmentJoseph A DiMasi
Tufts Center for the Study of Drug Development, Tufts University, Boston, MA 02111, USA
J Clin Oncol 25:209-16. 2007..Review existing studies and provide new results on the development, regulatory, and market aspects of new oncology drug development...
- The potential investment impact of improved access to accelerated approval on the development of treatments for low prevalence rare diseasesBrigitta E Miyamoto
Kakkis EveryLife Foundation For Rare Diseases, 77 Digital Drive, Suite 210, Novato, CA 94949, USA
Orphanet J Rare Dis 6:49. 2011..the Accelerated Approval (AA) regulations were implemented allowing the use of surrogate endpoints to achieve drug approval and accelerate development of life-saving therapies...
- Vandetanib for the treatment of symptomatic or progressive medullary thyroid cancer in patients with unresectable locally advanced or metastatic disease: U.S. Food and Drug Administration drug approval summaryKatherine Thornton
Office of Hematology and Oncology Products, Center for Drug Evaluation and Research, U S Food and Drug Administration, White Oak, MD, USA
Clin Cancer Res 18:3722-30. 2012..Treatment-related risks should be taken into account when considering the use of vandetanib in patients with indolent, asymptomatic, or slowly progressing disease...
- Database identifies FDA-approved drugs with potential to be repurposed for treatment of orphan diseasesKui Xu
Office of Orphan Products Development, US Food and Drug Administration, Silver Spring, MD 20993 0002, USA
Brief Bioinform 12:341-5. 2011....
- Number of patients studied prior to approval of new medicines: a database analysisRuben G Duijnhoven
Utrecht Institute for Pharmaceutical Sciences, Utrecht University, Utrecht, The Netherlands
PLoS Med 10:e1001407. 2013....
- European Medicines Agency review of post-authorisation studies with implications for the European Network of Centres for Pharmacoepidemiology and PharmacovigilanceKevin V Blake
Pharmacovigilance and Risk Management Sector, Patient Health Protection Unit, European Medicines Agency, London, UK
Pharmacoepidemiol Drug Saf 20:1021-9. 2011....
- Evolution of the physicochemical properties of marketed drugs: can history foretell the future?Bernard Faller
Novartis Institutes for BioMedical Research, Basel, Switzerland
Drug Discov Today 16:976-84. 2011..In this new territory, semi-empirical rules derived from knowledge accumulated from historic, older molecules are not necessarily valid and different liabilities become more prominent...
- Bridging the efficacy-effectiveness gap: a regulator's perspective on addressing variability of drug responseHans Georg Eichler
European Medicines Agency, London, UK
Nat Rev Drug Discov 10:495-506. 2011..Successful approaches will not be limited to the current notion of pharmacogenomics-based personalized medicines, but will also entail the wider use of electronic health-care tools to improve drug prescribing and patient adherence...
- Placebo response and antidepressant clinical trial outcomeArif Khan
Northwest Clinical Research Center, 1900 116th Avenue NE 112, Bellevue, Washington 98004, USA
J Nerv Ment Dis 191:211-8. 2003....
- Drug discovery: playing dirtySimon Frantz
Nature 437:942-3. 2005
- Prospects for productivityBruce Booth
McKinsey and Company, 55 East 52nd Street, New York, NY 10055, USA
Nat Rev Drug Discov 3:451-6. 2004
- European Medicines Agency workshop on biosimilar monoclonal antibodies: July 2, 2009, London, UKJanice M Reichert
Tufts Center for the Study of Drug Development, Boston, MA, USA
MAbs 1:394-416. 2009..Proceedings of the workshop are presented in Part 1 of this report, and discussed within the context of the legal, regulatory and business environments of the European Union, Asia and the United States in Parts 2, 3 and 4, respectively...
- Evaluation of drug-induced QT interval prolongation: implications for drug approval and labellingM Malik
Department of Cardiological Sciences, St George s Hospital Medical School, London, England
Drug Saf 24:323-51. 2001..The regulatory perspective includes careful adaptation of new research findings...
- Evaluating drug effects in the post-Vioxx world: there must be a better wayJerry Avorn
Circulation 113:2173-6. 2006
- Cancer pharmacogenomics and pharmacoepidemiology: setting a research agenda to accelerate translationAndrew N Freedman
National Cancer Institute, National Institutes of Health, Bethesda, MD 20892 7393, USA
J Natl Cancer Inst 102:1698-705. 2010....
- Effectiveness of antidepressants: an evidence myth constructed from a thousand randomized trials?John P A Ioannidis
Clinical Trials and Evidence Based Medicine Unit, Department of Hygiene and Epidemiology, University of Ioannina School of Medicine and the Biomedical Research Institute, Foundation for Research and Technology Hellas, Ioannina, Greece
Philos Ethics Humanit Med 3:14. 2008....
- Drug approval triggers debate on future direction for cancer treatmentsSimon Frantz
Nat Rev Drug Discov 5:91. 2006
- Health-related quality of life (HR-QOL) and regulatory issues. An assessment of the European Agency for the Evaluation of Medicinal Products (EMEA) recommendations on the use of HR-QOL measures in drug approvalG Apolone
Istituto di Ricerche Farmacologiche Mario Negri, Milano, Italy
Pharmacoeconomics 19:187-95. 2001..Experts from different disciplines should be involved in the preparation of such documents to assure the necessary technical expertise and the representativeness of the various counterparts...
- The continuum of comparability extends to biosimilarity: how much is enough and what clinical data are necessary?M McCamish
Global Biopharmaceutical Development, Sandoz International, Germany
Clin Pharmacol Ther 93:315-7. 2013..This article discusses biosimilarity and comparability as related scientific and regulatory concepts and the usefulness of clinical data for both...
- Transporter biology in drug approval: regulatory aspectsKazuya Maeda
Department of Molecular Pharmacokinetics, Graduate School of Pharmaceutical Sciences, The University of Tokyo, Japan
Mol Aspects Med 34:711-8. 2013..Target transporters include OATP1B1 (SLCO1B1) and OATP1B3 (SLCO1B3) in the liver, and OAT1 (SLC22A6), OAT3 (SLC22A8), OCT2 (SLC22A2), MATE1 (SLC47A1), and MATE2-K (SLC47A2) in the kidney, and MDR1 (ABCB1) in the intestine...
- Cetuximab (erbitux)W Bou-Assaly
Department of Radiologyand, University of Michigan Health System, Ann Arbor, USA
AJNR Am J Neuroradiol 31:626-7. 2010..We present a review of its mechanism of action, indications, side effects and economic issues, accompanied by a clinical example from our institution...
- Food and Drug Administration Drug approval summary: temozolomide plus radiation therapy for the treatment of newly diagnosed glioblastoma multiformeMartin H Cohen
Division of Oncology Drug Products, Center for Drug Evaluation and Research, US Food and Drug Administration, Rockville, Maryland 20857, USA
Clin Cancer Res 11:6767-71. 2005..1 months (radiotherapy alone). Adverse events during temozolomide treatment included thrombocytopenia, nausea, vomiting, anorexia, constipation, alopecia, headache, fatigue, and convulsions...
- FDA drug approval summary: nelarabine (Arranon) for the treatment of T-cell lymphoblastic leukemia/lymphomaMartin H Cohen
Division of Oncology Drug Products, Center for Drug Evaluation and Research, US Food and Drug Administration, Rockville, MD, USA
Oncologist 13:709-14. 2008....
- New drug approval times and safety warnings in the United States and Canada, 1992-2011Nigel S B Rawson
Eastlake Research Group, Oakville, Ontario, Canada
J Popul Ther Clin Pharmacol 20:e67-81. 2013..New drug approvals in the US and Canada were reviewed in short-term studies in the 1990s. A database of drugs approved in both countries between 1992 and 2011 exists allowing for a longer time horizon to assess trends...
- The US orphan drug programme 1983-1995S R Shulman
Tufts Center for the Study of Drug Development, Tufts University, Boston, Massachusetts, USA
Pharmacoeconomics 12:312-26. 1997....
- Aflibercept: newly approved for the treatment of macular edema following central retinal vein occlusionKirk E Evoy
College of Pharmacy, Purdue University, Lafayette, IN, USA
Ann Pharmacother 47:819-27. 2013....
- Unknown risks of pharmacy-compounded drugsLarry D Sasich
J Am Osteopath Assoc 108:86. 2008
- Does market exclusivity hinder the development of Follow-on Orphan Medicinal Products in Europe?Anne E M Brabers
NIVEL, Netherlands Institute for Health Services Research, PO Box 1568, 3500 BN Utrecht, The Netherlands
Orphanet J Rare Dis 6:59. 2011..In the interest of rare disorder patients better understanding of the effect of the market exclusivity incentive on follow-on OMP development is warranted...
- Failing the public health--rofecoxib, Merck, and the FDAEric J Topol
Cleveland Clinic Foundation, Cleveland, USA
N Engl J Med 351:1707-9. 2004
- CNS drugs approved by the centralised European procedure: true innovation or dangerous stagnation?Corrado Barbui
Section of Psychiatry and Clinical Psychology, University of Verona, Policlinico GB Rossi, Piazzale Scuro 10, 37134, Verona, Italy
Psychopharmacology (Berl) 190:265-8. 2007
- FDA drug approval summary: gefitinib (ZD1839) (Iressa) tabletsMartin H Cohen
Division of Oncology Drug Products, Center for Drug Evaluation and Research, U S Food and Drug Administration, Rockville, Maryland 20857, USA
Oncologist 8:303-6. 2003..Accelerated approval regulations require the sponsor to conduct further studies to verify that gefitinib therapy produces such a benefit...
- What is the European Medicines Agency?I Bighelli
Department of Public Health and Community Medicine, Section of Psychiatry, University of Verona, Verona, Italy
Epidemiol Psychiatr Sci 21:245-7. 2012..The main scientific limitations of this process are highlighted, together with some suggestions for dealing with them...
- [A system of prescriptions without drug approval: example of baclofen]Benjamin Rolland
Service d Addictologie, Universite Lille Nord de France, CHRU, Lille, France
Therapie 65:511-8. 2010..appetence for alcohol (anti-craving effect), although this effect has not been certified by Authorities for drug approval in France (AMM)...
- Food and Drug Administration (FDA) drug approval end points for chronic cutaneous ulcer studiesWilliam H Eaglstein
Department of Dermatology and Cutaneous Surgery, University of Miami Miller School of Medicine, Miami, Florida, USA
Wound Repair Regen 20:793-6. 2012..and mortality rate worse than many cancers, it is reasonable to compare the FDA trial end points for cancer drug approval with those for CCUs. And the difference is quite striking...
- Old drugs--new usesJ K Aronson
Br J Clin Pharmacol 64:563-5. 2007
- The quandary of compounding for MCOs: administrative costs, risks, and wasteThomas Kaye
Blue Cross and Blue Shield, Oklahoma, USA
Manag Care 12:42-8. 2003....
- Iloperidone redux: a dissection of the Drug Approval Package for this newly commercialised second-generation antipsychoticL Citrome
Department of Psychiatry, New York University School of Medicine and the Nathan S Kline Institute for Psychiatric Research, Orangeburg, NY, USA
Int J Clin Pract 64:707-18. 2010To describe the contents of a Drug Approval Package and to describe the efficacy and safety of iloperidone for the treatment of schizophrenia.
- The ongoing regulation of generic drugsRichard G Frank
Harvard Medical School, Boston, USA
N Engl J Med 357:1993-6. 2007
- Evidence vs. access: can twenty-first-century drug regulation refine the tradeoffs?J Woodcock
Center for Drug Evaluation and Research, US Food and Drug Administration, Silver Spring, Maryland, USA
Clin Pharmacol Ther 91:378-80. 2012..At the same time, prescribers and payers desire more information about drugs at the time they are released to the market. Will new regulatory schemes be able to accommodate these disparate needs?..
- U.S. Food and Drug Administration drug approval summaries: imatinib mesylate, mesna tablets, and zoledronic acidMartin H Cohen
Division of Oncology Drug Products, Center for Drug Evaluation and Research, U S Food and Drug Administration, Rockville, Maryland 20857, USA
Oncologist 7:393-400. 2002..These three drugs represent three different types of drug approval: Gleevec is an accelerated approval and supplemental new drug application (NDA); Mesnex tablets represent an ..
- U.S. Food and Drug Administration drug approval: slow advances in obstetric care in the United StatesDeborah A Wing
Department of Obstetrics Gynecology, University of California, Irvine, Orange, California 92868, USA
Obstet Gynecol 115:825-33. 2010The process for drug approval in the United States is complex and time-consuming. There are comparatively few drugs with U.S...
- An empirical review of major legislation affecting drug development: past experiences, effects, and unintended consequencesAaron S Kesselheim
Brigham and Women s Hospital, Harvard Medical School, Boston, MA 02120, USA
Milbank Q 89:450-502. 2011..With the development of transformative drugs at a low point, numerous commentators have recommended new legislation that uses supplementary market exclusivity as an incentive to promote innovation in the pharmaceutical market...
- A regulatory Apologia--a review of placebo-controlled studies in regulatory submissions of new-generation antidepressantsHans Melander
Medical Products Agency, P O Box 26, SE 751 03 Uppsala, Sweden
Eur Neuropsychopharmacol 18:623-7. 2008..Differences in the percentage of patients experiencing a clinically relevant response should also be demonstrated. In this respect, the approved SSRIs and SNRIs were found superior to placebo, independent of severity of depression...
- EU law mandates drug testing in childrenGunjan Sinha
J Natl Cancer Inst 100:84-5. 2008
- Availability of comparative efficacy data at the time of drug approval in the United StatesNikolas H Goldberg
Division of Pharmacoepidemiology and Pharmacoeconomics, Department of Medicine, Brigham and Women s Hospital, Harvard Medical School, Boston, MA 02120, USA
JAMA 305:1786-9. 2011..Comparative effectiveness is taking on an increasingly important role in US health care, yet little is known about the availability of comparative efficacy data for drugs at the time of their approval in the United States...
- Development of and access to products for neglected diseasesJoshua Cohen
Tufts University Center for the Study of Drug Development, Boston, Massachusetts, United States of America
PLoS ONE 5:e10610. 2010....
- Reporting bias in drug trials submitted to the Food and Drug Administration: review of publication and presentationKristin Rising
School of Medicine, University of California San Francisco, San Francisco, California, USA
PLoS Med 5:e217; discussion e217. 2008....
- Disease registries and outcomes research in children: focus on lysosomal storage disordersSimon Jones
Willink Unit, Genetic Medicine, Manchester Academic Health Science Centre, Central Manchester University Hospitals NHS Foundation Trust, St Marys Hospital, Manchester, UK
Paediatr Drugs 13:33-47. 2011..Clinical trials are a pivotal part of the drug approval process; however, they are not always applicable to the diverse populations - including children - that ..
- Priority-setting decisions for new cancer drugs: a qualitative case studyD K Martin
Department of Health Policy, Management and Evaluation and Public Health Sciences, M5G 1L4, Ontario, Canada
Lancet 358:1676-81. 2001..INTERPRETATION: Observing priority-setting decisions and their rationales in actual practice reveals lessons not contained in theoretical accounts...
- Comparison of anticancer drug coverage decisions in the United States and United Kingdom: does the evidence support the rhetoric?Anne Mason
Centre for Health Economics, University of York, York, United Kingdom
J Clin Oncol 28:3234-8. 2010..Coverage decisions that consider cost effectiveness may lead to restrictions in access...
- Food and Drug Administration drug approval summary: Sunitinib malate for the treatment of gastrointestinal stromal tumor and advanced renal cell carcinomaEdwin P Rock
Food and Drug Administration, Division of Drug Oncology Products, 10903 New Hampshire Avenue, Bldg 22, Rm 2133, Silver Spring, Maryland 20903, USA
Oncologist 12:107-13. 2007..Caution should be exercised when administering sunitinib in combination with known CYP3A4 inducers or inhibitors...
- Economic issues with follow-on protein productsMichael Lanthier
US Food and Drug Administration, Rockville, Maryland 20857, USA
Nat Rev Drug Discov 7:733-7. 2008..For the years 2013-2015, we estimate that products representing US$20 billion in annual sales--approximately half of all sales in 2006--can be expected to lose patent protection...
- FDA is incapable of protecting US "against another Vioxx"Jeanne Lenzer
BMJ 329:1253. 2004
- The US Orphan Drug Act: rare disease research stimulator or commercial opportunity?Olivier Wellman-Labadie
Division of Dermatology, Department of Medicine, University of British Columbia, 835 West 10th Ave, Vancouver, BC, Canada
Health Policy 95:216-28. 2010..This study investigates issues associated with the United States Orphan Drug Act...
- United States Food and Drug Administration Drug Approval summary: Gefitinib (ZD1839; Iressa) tabletsMartin H Cohen
Division of Oncology Drug Products, Center for Drug Evaluation and Research, United States Food and Drug Administration, Rockville, Maryland 20857, USA
Clin Cancer Res 10:1212-8. 2004..Accelerated approval regulations require the sponsor to conduct additional studies to verify that gefitinib therapy produces such benefit...
- Emergence of orphan drugs in the United States: a quantitative assessment of the first 25 yearsM Miles Braun
Office of Orphan Products Development, US Food and Drug Administration, Silverspring, Maryland 20993, USA
Nat Rev Drug Discov 9:519-22. 2010..The implications of such findings for future development and marketing of therapies for rare diseases are discussed...
- The Food and Drug Administration's deliberations on antidepressant use in pediatric patientsLaurel K Leslie
Children s Hospital, Child and Adolescent Services Research Center, San Diego, California 92123, USA
Pediatrics 116:195-204. 2005..We also provide research, regulation, education, and practice implications for care for children and adolescents who may be eligible for treatment with these medications...
- Non-commercial clinical trials of a medicinal product: can they survive the current process of research approvals in the UK?L Sheard
Centre for Research in Primary Care, 71 75 Clarendon Road, Leeds LS2 9PL, England
J Med Ethics 32:430-4. 2006..The current approvals process could now be hindering non-commercial clinical trials, leading to a loss of important evidence-based medical information...
- Folotyn (pralatrexate injection) for the treatment of patients with relapsed or refractory peripheral T-cell lymphoma: U.S. Food and Drug Administration drug approval summaryShakun M Malik
Office of Oncology Drug Products, Center for Drug Evaluation and Research, US Food and Drug Administration, Silver Spring, Maryland, USA
Clin Cancer Res 16:4921-7. 2010..as a single agent for the treatment of patients with relapsed or refractory peripheral T-cell lymphoma (PTCL); it is the first drug approved for this indication...
- Predictors of orphan drug approval in the European UnionHarald E Heemstra
Division of Pharmacoepidemiology and Pharmacotherapy, Utrecht Institute for Pharmaceutical Sciences, Faculty of Science, Utrecht University, P O Box 80 082, 3508 TB, Utrecht, The Netherlands
Eur J Clin Pharmacol 64:545-52. 2008..Recent literature discusses factors that may influence the development of new orphan medicinal products in the EU. This study aims to identify predictors for successful marketing authorisation of potential orphan drugs in the EU...
- Safety-related regulatory actions for orphan drugs in the US and EU: a cohort studyHarald E Heemstra
Division of Pharmacoepidemiology and Pharmacotherapy, Utrecht Institute for Pharmaceutical Sciences, Faculty of Science, Utrecht University, 3508 TB Utrecht, The Netherlands
Drug Saf 33:127-37. 2010..Several studies have been conducted on safety-related regulatory actions for drugs, but none of these have specifically focused on orphan drugs...
- Biotechnology: identifying advances from the hypeRobert H Glassman
Division of Hematology and Medical Oncology, Weill Medical College of Cornell University, New York, New York 10021, USA
Nat Rev Drug Discov 3:177-83. 2004
- FDA drug approval summaries: oxaliplatinAmna Ibrahim
Division of Oncology Drug Products, Center for Drug Evaluation and Research, U S Food and Drug Administration, Rockville, Maryland 20857, USA
Oncologist 9:8-12. 2004..Approval was based on response rate and on an interim analysis of TTP. No results are available, at this time, that demonstrate a clinical benefit, such as improvement in disease-related symptoms or survival...
- FDA drug approval summaries: pemetrexed (Alimta)Maitreyee Hazarika
U S Food and Drug Administration, HFD 150, 5600 Fishers Lane, Rockville, Maryland 20857, USA
Oncologist 9:482-8. 2004..Patients should also receive corticosteroids with chemotherapy to reduce the risk of skin rashes. Approval was based on superior survival as a clinical benefit...
- Translation of rare disease research into orphan drug development: disease mattersHarald E Heemstra
Utrecht Institute for Pharmaceutical Sciences, Utrecht University, The Netherlands
Drug Discov Today 14:1166-73. 2009..Consequently, additional incentives should focus on stimulating the specific needs of rare disease research at disease class level...
- Drug approval summaries: arsenic trioxide, tamoxifen citrate, anastrazole, paclitaxel, bexaroteneM H Cohen
Division of Oncology Drug Products, Center for Drug Evaluation and Research, U S Food and Drug Administration, Rockville, Maryland 20852, USA
Oncologist 6:4-11. 2001..Information provided includes rationale for drug development, study design, efficacy and safety results, and pertinent literature references...
- Development of medicines for children in Europe: ethical implicationsAgnes Saint Raymond
European Medicines Agency, 7 Westferry Circus, Canary Wharf, London E144HB, UK
Paediatr Respir Rev 6:45-51. 2005..Future European paediatric regulations should encourage the development of medicines in high-quality ethical research and ensure availability of information to the public...
- Financial anatomy of neuroscience researchE Ray Dorsey
Department of Neurology, University of Rochester Medical Center, Rochester, NY, USA
Ann Neurol 60:652-9. 2006..To estimate the level of funding for neuroscience research from federal and industry sources and to examine the therapeutic advances in the neurosciences over the past decade...
- Should pharmacogenomic studies be required for new drug approval?M V Relling
Department of Pharmaceutical Sciences, St Jude Children s Research Hospital, Memphis, Tennessee, USA
Clin Pharmacol Ther 81:425-8. 2007..more examples being elucidated in the coming decades, dictates that pharmacogenetics be incorporated into the drug approval process...
- How can we regulate medicines better?Silvio Garattini
Mario Negri Institute for Pharmacological Research, Via Eritrea 62, 20157 Milano, Italy
BMJ 335:803-5. 2007
- End points in cancer clinical trials and the drug approval processRichard L Schilsky
Clin Cancer Res 8:935-8. 2002..This editorial will review commonly used clinical trial end points and describe their potential advantages and disadvantages to expedite the drug approval process required in the United States.
- BiDil for heart failure in black patients: The U.S. Food and Drug Administration perspectiveRobert Temple
U S Food and Drug Administration, Silver Spring, Maryland 20993 0002, USA
Ann Intern Med 146:57-62. 2007....
- Conditional approval: discussion points from the PSI conditional approval expert groupKevin Carroll
PSI Discussion Group on Conditional Approval, AstraZeneca, UK
Pharm Stat 7:263-9; discussion 270-1. 2008..The use of interim analyses in Phase III for supporting conditional approval raises some challenging issues regarding dissemination of information, maintenance of blinding, potential introduction of bias, ethics, switching, etc...
- A proposal for radical changes in the drug-approval processAlastair J J Wood
Vanderbilt University School of Medicine, Nashville, USA
N Engl J Med 355:618-23. 2006
- Impact of pharmacometric reviews on new drug approval and labeling decisions--a survey of 31 new drug applications submitted between 2005 and 2006V A Bhattaram
Office of Clinical Pharmacology, Office of Translational Sciences, Center for Drug Evaluation and Research, Food and Drug Administration, Silver Spring, Maryland, USA
Clin Pharmacol Ther 81:213-21. 2007..The objective of the current report is to assess the role of PM, at the Food and Drug Administration (FDA), in drug approval and labeling decisions...
- A comparison of new drug availability in Canada and the United States and potential therapeutic implications of differencesJoel Lexchin
School of Health Policy and Management, York University, 4700 Keele St, Toronto, Ont, and Emergency Department, University Health Network, Canada
Health Policy 79:214-20. 2006..This study uses a convenience sample of new drugs marketed in the United States and determines how many of these products are initially unavailable in Canada and their therapeutic value...
- Research design features and patient characteristics associated with the outcome of antidepressant clinical trialsArif Khan
Northwest Clinical Research Center, Number 112, 1900 116th Avenue NE, Bellevue, WA 98004, USA
Am J Psychiatry 161:2045-9. 2004..The authors examined which, if any, research design features and patient characteristics would significantly differ between successful and unsuccessful antidepressant trials...
- Learning the value of drugs--is rofecoxib a regulatory success story?Rebecca S Eisenberg
University of Michigan Law School, Ann Arbor, USA
N Engl J Med 352:1285-7. 2005
- FDA drug approval summary: azacitidine (5-azacytidine, Vidaza) for injectable suspensionEdvardas Kaminskas
U S Food and Drug Administration, 5600 Fishers Lane, HFD 150, Rockville, Maryland 20857, USA
Oncologist 10:176-82. 2005..S. FDA for MDS, has a favorable safety profile and provides a clinical benefit of eliminating transfusion dependence and complete or partial normalization of blood counts and bone marrow blast percentages in responding patients...
- Assessing tumor-related signs and symptoms to support cancer drug approvalGrant Williams
Division of Oncology Drug Products, CDER FDA, Rockville, Maryland 20852, USA
J Biopharm Stat 14:5-21. 2004..While prolongation of survival is an obvious end point for new cancer drug approval, the US Food and Drug Administration (FDA) has also utilized end points that evaluate patient symptoms...
- A new colonialism?--Conducting clinical trials in IndiaSamiran Nundy
Department of Surgical Gastroenterology, Sir Ganga Ram Hospital, New Delhi, India
N Engl J Med 352:1633-6. 2005
- Clinical trials for drug approval: a pilot study of the view of doctors at Tokushima University HospitalHiroaki Yanagawa
Clinical Trial Center for Developmental Therapeutics, Tokushima University Hospital
J Med Invest 53:292-6. 2006..as investigators, special attention requirements, and the expected role of CRC in clinical trials for drug approval. In addition, the appropriate use of the outpatient clinic was examined...
- Efficacy, safety, and cost of new anticancer drugsSilvio Garattini
Mario Negri Institute for Pharmacological Research, 20157 Milan, Italy
BMJ 325:269-71. 2002
- FDA drug approval summary: panitumumab (Vectibix)Ruthann M Giusti
Office of Oncology Drug Products, Center for Drug Evaluation and Research, U S Food and Drug Administration, Silver Spring, Maryland 20993, USA
Oncologist 12:577-83. 2007..The most serious adverse events were pulmonary fibrosis, severe dermatologic toxicity complicated by infectious sequelae and septic death, infusion reactions, abdominal pain, hypomagnesemia, nausea, vomiting, diarrhea, and constipation...
- Network analysis of FDA approved drugs and their targetsAVI MA'AYAN
Department of Pharmacology and Systems Therapeutics, Mount Sinai School of Medicine, New York, New York 10029, USA
Mt Sinai J Med 74:27-32. 2007..These initial observations allow for development of an integrated research methodology to identify general principles of the drug discovery process...
- BiDil for heart failure in black patients: implications of the U.S. Food and Drug Administration approvalKirsten Bibbins-Domingo
Division of General Internal Medicine, San Francisco General Hospital, University of California, San Francisco, San Francisco, California 94143 1364, USA
Ann Intern Med 146:52-6. 2007....
- Adopting orphan drugs--two dozen years of treating rare diseasesMarlene E Haffner
Office of Orphan Products Development at the Food and Drug Administration, Rockville, MD, USA
N Engl J Med 354:445-7. 2006
- Nikkomycin Z treatment of early coccidioidal pneumonia: Phase II clinical trialDAVID LARWOOD; Fiscal Year: 2010..2) using the drug produced by the new process in an initial efficacy trial in humans ("Phase II" in the FDA drug approval sequence)...
- Validation and Quantification of FFPE Antigen Retrieval by Proteome AnalysisXUPEING FANG; Fiscal Year: 2010....
- Bacterial-Induced Sepsis: A New Treatment StrategySharon L McCoy; Fiscal Year: 2013..support appropriate P13 dosing and exposure parameters for the IND-enabling studies required by the FDA for drug approval. In support of the IND filing to the FDA for drug approval of P13, an optimal clinical treatment protocol and ..
- A Chemical Biology Network for Personalized MedicineMaxim Totrov; Fiscal Year: 2010..Such a tool would have a profound impact on the rapidity of clinical trial completion and drug approval, as history has shown that clinical trials of drugs matched to biomarkers, such as her2-neu and Herceptin, ..
- Center for Research Translation of Systemic Exon-skipping in Muscular DystrophyEric P Hoffman; Fiscal Year: 2013..Given the high cost of drug development, there is an emerging consensus that exon skipping should achieve drug approval 'as a class'...
- Novel bisphosphonates for prostate cancer therapyAlexander Karpeisky; Fiscal Year: 2013..will guide the further development of this promising concept, greatly aid in obtaining investigational new drug approval, and lead to eventual clinical application...
- TAS::75 0849::TAS PURIFICATION, REFORMULATION AND PHARMACOKINETICS OF NITROSYLCOJoseph Bauer; Fiscal Year: 2010Oncology remains a major market of unmet need. BNOAT Oncologys long-term objective is New Drug Approval of its lead candidate nitrosylcobalamin, a tumor-specific, apoptosis-inducing, anti-cancer drug...
- Postmarketing Surveillance of Generic Drug Usage and Substitution PatternsIlene H Zuckerman; Fiscal Year: 2013..individual level use of a brand product and its generics;and 3) Determine if controversy around a generic drug approval impacted perceptions of the generic drug quality by conducting a national survey of patient and physician ..
- Electronic Image Trial Management SystemCOLIN EDWARD RHODES; Fiscal Year: 2010DESCRIPTION (provided by applicant): Delays in FDA drug approval are measured in lost lives (estimated to be hundreds of thousands over the last few decades) and increased costs to U.S. citizens for drugs that are eventually approved...
- The Co-Clinical Project: Informing clinical trials using preclinical mouse modelsPier Paolo Pandolfi; Fiscal Year: 2010..clinical trials that stratify patients on the basis of molecular and genetic criteria, in turn accelerating drug approval. In addition, to accelerate the optimization of combinatorial-targeted therapy on the basis of genetic ..
- Factors determining placebo response in drug-resistant focal epilepsyEmilia Bagiella; Fiscal Year: 2013..Clinical trials constitute the gold standard of research that informs treatment of epilepsy as well as the drug approval process...
- 2013 Computer-Aided Drug Design GRCAnthony Nicholls; Fiscal Year: 2013..No one is going to run, for example, double blind studies of two methods of drug discover through to drug approval, despite what might be learned...
- PARS inhibitor therapy for smoke inhalationAndrew Salzman; Fiscal Year: 2004..The proposed clinical trial is expected to provide the foundation for FDA orphan drug approval of INO-1001 for treatment of acute smoke inhalation injury.
- Improved methods to assess the comparative safety of new psychiatric medicationsKrista F Huybrechts; Fiscal Year: 2013..early in the life cycle of mental health drugs is of great public health importance as it may expedite drug approval and make prescribers more comfortable using new drugs by providing a mechanism for ongoing post-marketing ..
- Late 20th-Century Consumer Advocacy and the Shaping of Public Health Policy and PAva Alkon; Fiscal Year: 2009....
- PRECLIN EVAL OF THERAPIES FOR CRYPTOSPORIDIUM PARVUM INFSaul Tzipori; Fiscal Year: 2000..providing contract resources for efficacy evaluations in culture and in animals, a critical component in new drug approval. Cryptosporidiosis can be life threatening in people with AIDS, and also causes a mold to severe, although ..
- SUSTAINED RELEASE CYCLOSPORINE FOR TREATMENT OF UVEITISPaul Ashton; Fiscal Year: 2000..Control Delivery Systems (CDS) has experience in all aspects of the drug approval process. Our first product the VitrasertTM was licensed to Chiron Vision...
- Novel Bisphosphonates for Multiple Myeloma TherapyAlexander Karpeisky; Fiscal Year: 2009..will guide the further development of this promising concept, greatly aid in obtaining investigational new drug approval, and lead to eventual clinical application...
- AMIA 2002 Spring Congress: A Drug By Any Other NameDaniel Sands; Fiscal Year: 2002..Point-of-Care Infrastructure; Behind the Scenes; The Role of Regulatory Informatics in Enhancing Patient Safety (including post-marketing surveillance and the drug approval process); Vocabulary Issues and Technologies.
- PNA FOR STROKECARLETON HSIA; Fiscal Year: 2007..the requirements of the FDA for an Investigational New Drug (IND) application, an essential step to new drug approval. There are three major components for the completion: chemistry manufacturing and control (CMC), GLP toxicity ..
- IMPROVED BIOCATALYST FOR EPOXIDE PRODUCTIONRobert Steffan; Fiscal Year: 2000..using enantio-pure building blocks to produce pharmaceuticals that are enantiomerically pure, the cost of the drug approval process can be significantly reduced because only one compound needs to be tested for efficacy and safety...
- EVAL OF THERAPIES FOR MYCOBACTERIUM AVIUM INFECOTIONLOWELL YOUNG; Fiscal Year: 2000..infections by providing contract resources for efficacy evaluations in animals, a critical component in new drug approval. The availability of animal model testing systems will provide the NIAID with testing capabilities to evaluate ..
- Pharmacotherapy for Minor DepressionRobert Howland; Fiscal Year: 2004..The results of this study will have profound public health implications by improving our understanding of the efficacy of SJW and standard antidepressants for the treatment of MinorD. ..
- Hormone Replacement Therapy and Prothrombotic VariantsBruce Psaty; Fiscal Year: 2005..Power for primary and secondary aims is excellent. Information from this project may help physicians counsel women about HRT to maximize either effectiveness, or safety, or both. ..
- Genome-wide case-only study of antihypertensive drug-gene interactionsBruce M Psaty; Fiscal Year: 2010....
- ANTIHYPERTENSIVE DRUG/GENE INTERACTIONS AND CV EVENTSBruce Psaty; Fiscal Year: 2004..58 (the difference between an odds ratio of 0.46 and 0.79 in subjects with and without the variant, respectively). Power for other primary aims is excellent; power for secondary aims is good to excellent. ..
- Trends in and Outcomes of Medication Use in Older AdultsBruce Psaty; Fiscal Year: 2004..abstract_text> ..
- Pharmacogenetics and Cardiovascular EventsBruce Psaty; Fiscal Year: 2006..The primary defense is replication: we would therefore welcome collaboration from others in the Pharmacogenetics Network in validation efforts. ..
- MUTATIONS, HORMONE THERAPY, AND VENOUS THROMBOEMBOLISMBruce Psaty; Fiscal Year: 2001....
- WGA Study to Identify Genetic Variants Associated with CV Events in CHSBruce Psaty; Fiscal Year: 2009..The proposed three-part study is efficient, has excellent power to detect small to modest-sized hazard ratios, provides a large sample of WG scans for aim 2, and includes an external replication. (End of Abstract) ..
- Depression in Alzheimer's Disease Study 2 (DIADS-2)Lon Schneider; Fiscal Year: 2007..unreadable] [unreadable]..
- DIETARY OBESITYGeorge Bray; Fiscal Year: 2007..We now propose these well founded, important and exciting studies which will provide critical new insights into anatomical, physiological and molecular mechanisms by which high levels of dietary fat induce obesity. ..
- Adrenergic Antagonism During Murine CandidiasisBrad Spellberg; Fiscal Year: 2008..For these reasons, the potential for adrenergic antagonism to act as an effective immunotherapy is highly meritorious of investigation in this training application. ..
- GERIATRIC CLINICAL EPIDEMIOLOGY TRAINING GRANTSTEVEN HALBERT; Fiscal Year: 2007..g., clinical trials, case control, cohort research, etc.), and the faculties' commitment to collaborative research. [unreadable] [unreadable]..
- Transporter-Enzyme Interplay Evaluation via Microfluidiic HTS Cell Culture DeviceLeslie Benet; Fiscal Year: 2008..unreadable] [unreadable] [unreadable]..
- STATISTICAL ISSUES IN AIDS RESEARCHThomas Fleming; Fiscal Year: 2008..Research into optimal designs of Phase HE "Intermediate Trials" of candidate HIV vaccines will also be continued. ..
- HIV PREVENTION TRIALS NETWORK LEADERSHIP GROUPThomas Fleming; Fiscal Year: 2005..abstract_text> ..
- ESCAPE Mechanistic SubstudiesRobert Califf; Fiscal Year: 2003..Catecholamines are also being collected at baseline and 3 months. In addition, the ESCAPE investigators are all ready capturing detailed demographic, clinical, and physiological information as part of the protocol for the primary study. ..
- CLINICAL RESEARCH CURRICULUMBRIAN STROM; Fiscal Year: 2004..I. and co-PI s in the three distinct aspects of Clinical Research targeted by this program represent particular features of this proposal. ..
- Psychobiology of IV Naloxone & Lactate in NormalsDonald Klein; Fiscal Year: 2005..This would further validate this model. It may also suggest new approaches to anti-panic agents, such as mixed opiate agonist-antagonist. ..
- ACYLCOA FORMATION--COVALENT BINDING, PHARMACOKINETICSLeslie Benet; Fiscal Year: 2002....
- Innate Effectors of rAls3p-N Anti-Candida VaccineBrad J Spellberg; Fiscal Year: 2010..PNS 398/2590 (Rev. 11/07) Page - Continuation Format Page ..
- Effects of Mental Parity on High-Cost and Severely-Ill IndividualsHaiden A Huskamp; Fiscal Year: 2011..We will estimate a combination of two-part random-effects models for longitudinal data, latent class random-effects models, logistic regression models, Poisson regression models, and hazard models to address these aims. ..
- Economics of Formulary Design and Mental Health PolicyHaiden Huskamp; Fiscal Year: 2006..Huskamp the training, mentoring, time and resources to develop the skills that will put her in a position to lead independent research on the economics of pharmaceutical treatment for mental illnesses. ..
- Phase ii intraperitoneal rhIL 12Ralph Freedman; Fiscal Year: 2002..4) Determine whether expression of proangiogenic factors VEGF, FGF2 and IL-8 are decreased following IF rhIL- 12. ..
- DEFERIPRONE THERAPY FOR SICKLE CELL DISEASENancy Olivieri; Fiscal Year: 2001....
- The vital role of BAFF in the development of SLEWilliam Stohl; Fiscal Year: 2010..abstract_text> ..
- EARLY VISUAL PROCESSING IN SCHIZOPHRENIAMichael F Green; Fiscal Year: 2010....
- HRQL Impact of Chronic Conditions and Comorbidity BurdenPatrick Sullivan; Fiscal Year: 2006..S. [unreadable] [unreadable]..
- INTRACELLULAR T CELL REGULATORS OF HIV-1Garry Nolan; Fiscal Year: 2004..The roles of these evolutionarily disparate Rel molecules in T cells will be dissected with regards to their activity upon the HIV-1 enhancer and promoter region. ..
- 26th Princeton Conference on Cerebrovascular DiseaseJames Grotta; Fiscal Year: 2008..All presentations and discussion will be recorded and published. [unreadable] [unreadable] [unreadable]..
- Mixed-Effects ZIP Models--Mental Health Service ResearchRobert Gibbons; Fiscal Year: 2004..abstract_text> ..
- Outcomes of Sleep Disorders in Older Men-Palo AltoMarcia Stefanick; Fiscal Year: 2007..We will also supplement the bank of MrOS specimens to allow for testing of future hypotheses concerning the role of sleep in the development of age-related diseases and conditions. ..
- FURANOCOUMARINS AND DRUGS EFFECT ON CYP3A4Paul Watkins; Fiscal Year: 2007..abstract_text> ..
- Pilot Study of Huperzine A in Alzheimer's DiseasePaul Aisen; Fiscal Year: 2003....
- DAYTIME SLEEPINESS--PREVALENCE, CONSEQUENCES, AND RISKSThomas Roth; Fiscal Year: 2003..The important contribution of the present proposal is that it will be the first population-based study to use laboratory assessments, its consequences and risks to measure sleepiness. ..
- Immunity in Early Syphillis: Pathway to HIV CoinfectionJuan Salazar; Fiscal Year: 2007..In Aim 2 we will study the effect of secondary syphilis on viral load (VL) in HIV co-infected individuals. In this aim we will compare VL between HIV patients with and without syphilis and determine the effect of penicillin on VL. ..
- CARP, A Novel Cell Death RegulatorWafik El Deiry; Fiscal Year: 2006..Specific Aim 3: To study the functional consequences of perturbing or blocking CARP expression. These studies should provide novel insights into the cellular control of the extrinsic pathway of apoptosis. ..
- Digoxin Chiral Isolates as Improved PharmaceuticalsJohn Somberg; Fiscal Year: 2005..The advantage would be a treatment for AF that did not cause cardiac augmentation and vasoconstriction or a treatment for CHF that does not cause heart rate slowing or conduction disturbances. ..
- Potency of cryopreserved, irradiated sporozoite vaccineStephen Hoffman; Fiscal Year: 2005..The project will also provide a method for cryopreserving unirradiated sporozoites that could be used to challenge volunteers by injection with P. falciparum in vaccine studies worldwide. ..
- Microglia, Complement, and PainClifford J Woolf; Fiscal Year: 2010..We will use this information to devise and test novel treatment strategies for somatic and facial neuropathic pain based either on blocking complement gene induction in microglia or complement cascade activation in the nervous system. ..
- Lyophilization of Plasmodium falciparum sporozoite vaccineStephen Hoffman; Fiscal Year: 2007..Additionally, a dried vaccine may accelerate a concerted world-wide effort to eradicate this devastating disease. [unreadable] [unreadable] [unreadable]..
- MARROW REGULATION IN CYCLIC HEMATOPOIESISDavid Dale; Fiscal Year: 2003....
- Experimental Intracerebral HemorrhageJustin Zivin; Fiscal Year: 2004..These studies may provide insights about the causes of bleeding and neurological tissue damage that should be helpful in designing even more effective stroke therapies. ..
- Exploratory Trial of Curcumin in Pancreatic CancerRazelle Kurzrock; Fiscal Year: 2005..These studies should provide the foundation for the development of curcumin as an anticancer agent and may lead to a novel approach to the management of pancreatic cancer. ..
- Phase I Study of ALT-801 in Metastatic Melanoma/Kidney Cancer PatientsMayer Fishman; Fiscal Year: 2009....
- Investigation of influenza virulence mediated by the NS1A proteinDIANA NOAH; Fiscal Year: 2007..unreadable] [unreadable] [unreadable]..
- Nuclear translocation of urokinase/nucleolin complexesDouglas Cines; Fiscal Year: 2008..unreadable] [unreadable] [unreadable]..
- DEFENSIN AND THE PATHOGENESIS OF ATHEROSCLEROSISDouglas Cines; Fiscal Year: 2002..Finally, the interaction between endogenous defensin and endogenous Lp(a) will be examined in a novel transgenic mouse than overexpresses both human defensin and human Lp(a). ..
- Antidepressant Use and SuicideSusan Busch; Fiscal Year: 2009..The significance of this project lies in its potential to help clinicians and policymakers assess and manage competing risk and benefit claims. ..