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Drug Approval (drug + approval)
Selected AbstractsA critical reappraisal of treatment response criteria in systemic mastocytosis and a proposal for revisionsEUROPEAN JOURNAL OF HAEMATOLOGY, Issue 5 2010A. Pardanani Abstract Mast cell disease (MCD) is a hematopoietic stem cell neoplasm that is associated with infiltration of one or more organs with cytologically abnormal mast cells (MC). MCD is frequently but not always associated with a KIT mutation and, in some cases, is associated with clonal expansion of non-MC lineage cells. In adults, there is almost always MC infiltration of the bone marrow, which is a cardinal feature of systemic mastocytosis (SM). While, as members of the wider community of physician scientists, we recognize the contribution of the current consensus treatment response criteria for SM, as individuals with more than average clinical experience in SM, we would like to point out their limitations and engage in a constructive discussion that will hopefully lead to a consideration for revisions. We present here an alternative proposal for treatment response assessments we believe is more objective, reproducible, and importantly, SM-subtype specific, given the recent progress in our understanding of the natural history of this disease. We believe this proposal is timely given the prospects for new clinical trials in SM, and the related regulatory aspects of new drug approval that are currently not adequately addressed. The intent of this exercise is not to undermine the complexity of the disease or previous work by other investigators, but to come up with ideas for response criteria that are more practical and consider meaningful patient outcome. [source] Exploration of developmental approaches to companion animal antimicrobials: providing for the unmet therapeutic needs of dogs and catsJOURNAL OF VETERINARY PHARMACOLOGY & THERAPEUTICS, Issue 2 2010AAVPT, White Paper Committee Committee members:, Workshop AAVPT Workshop White Paper Committee. Exploration of developmental approaches to companion animal antimicrobials: providing for the unmet therapeutic needs of dogs and cats. J. vet. Pharmacol. Therap.33, 196,201. The American Academy of Veterinary Pharmacology and Therapeutics (AAVPT) and the United States Pharmacopeia (USP) co-sponsored a workshop to explore approaches for developing companion animal antimicrobials. This workshop was developed in response to the shortage of antimicrobials labeled for dogs and cats, as there is a shortage of approved antimicrobials for the range of infectious diseases commonly treated in small animal practice. The objective of the workshop was to identify alternative approaches to data development to support new indications consistent with the unmet therapeutic needs of dogs and cats. The indications for currently approved antimicrobials do not reflect the broader range of infectious diseases that are commonly diagnosed and treated by the veterinarian. Therefore, the labels for these approved antimicrobials provide limited information to the veterinarian for appropriate therapeutic decision-making beyond the few indications listed. Industry, veterinary practice, and regulatory challenges to the development of new antimicrobial indications were discussed. The workshop resulted in short- and long-term recommendations. Short-term recommendations focus on the use of additional data considerations for product labeling. Long-term recommendations center on legislative or regulatory legal initiatives. The workshop recommendations will need collaboration from industry, academia, and regulatory authorities and a legal shift in the drug approval and availability processes. [source] Biomarkers in drug development: friend or foe?PHARMACEUTICAL STATISTICS: THE JOURNAL OF APPLIED STATISTICS IN THE PHARMACEUTICAL INDUSTRY, Issue 4 2007A personal reflection gained working within oncology Abstract Hopes and expectations for the use and utility of new, emerging biomarkers in drug development have probably never been higher, especially in oncology. Biomarkers are exalted as vital patient selection tools in an effort to target those most likely to benefit from a new drug, and so to reduce development costs, lessen risk and expedite developments times. It is further hoped that biomarkers can be used as surrogate endpoints for clinical outcomes, to demonstrate effectiveness and, ultimately, to support drug approval. However, I perceive that all is not straightforward, and, particularly in terms of the promise of accelerated drug development, biomarker strategies may not in all cases deliver the advances and advantages hoped for. Copyright © 2007 John Wiley & Sons, Ltd. [source] Limits of the applicability and generalizability of drug trials in maniaBIPOLAR DISORDERS, Issue 2002Rasmus W Licht Licht RW. Limits of the applicability and generalizability of drug trials in mania. Bipolar Disord 2002: 4(Suppl. 1): 66,68. ©Blackwell Munksgaard, 2002 During recent years, the majority of drug trials in mania have been conducted for the purpose of drug approval. On this background, this paper addresses to what extent these trials may actually provide the practising clinician with useful information. One major point is that selection prior to the point of randomization in RCTs in mania may limit the applicability of study results to patients seen in ordinary clinical practice. Limitations in study credibility and study design are also discussed. The need for large scale pragmatic studies using broad inclusion criteria, comparing the various treatments, alone or in combination, is emphasized. [source] Sample size considerations for Japanese patients in a multi-regional trial based on MHLW guidancePHARMACEUTICAL STATISTICS: THE JOURNAL OF APPLIED STATISTICS IN THE PHARMACEUTICAL INDUSTRY, Issue 2 2010Hui Quan Abstract Since the publication of the International Conference on Harmonization E5 guideline, new drug approvals in Japan based on the bridging strategy have been increasing. To further streamline and expedite new drug development in Japan, the Ministry of Health, Labour and Welfare, the Japanese regulatory authority, recently issued the ,Basic Principles on Global Clinical Trials' guidance to promote Japan's participation in multi-regional trials. The guidance, in a Q&A format, provides two methods as examples for recommending the number of Japanese patients in a multi-regional trial. Method 1 in the guidance is the focus of this paper. We derive formulas for the sample size calculations for normal, binary and survival endpoints. Computations and simulation results are provided to compare different approaches. Trial examples are used to illustrate the applications of the approaches. Copyright © 2009 John Wiley & Sons, Ltd. [source] | ||||||||||