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Drug and device development

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406 papers 25 to 100 followers
By Jamie Jarmul Md / PhD student at UNC - Chapel Hill, PhD in Health Policy and Management
Ilke Akpinar, Philip Jacobs, Don Husereau
OBJECTIVES: Economic evaluations, although not formally used in purchasing decisions for medical devices in Canada, are still being conducted and published. The aim of this study was to examine the way that prices have been included in Canadian economic evaluations of medical devices. METHODS: We conducted a review of the economic concepts and implications of methods used for economic evaluations of the eleven most implanted medical devices from the Canadian perspective...
January 2015: International Journal of Technology Assessment in Health Care
Konstantin Mechler, William K Mountford, Georg F Hoffmann, Markus Ries
BACKGROUND: Lysosomal storage disorders are a heterogeneous group of approximately 50 monogenically inherited orphan conditions. A defect leads to the storage of complex molecules in the lysosome, and patients develop a complex multisystemic phenotype of high morbidity often associated with premature death. More than 30 years ago the Orphan Drug Act of 1983 passed the United States legislation intended to facilitate the development of drugs for rare disorders. We directed our efforts in assessing which lysosomal diseases had drug development pressure and what distinguished those with successful development and approvals from diseases not treated or without orphan drug designation...
2015: Orphanet Journal of Rare Diseases
Amit Agarwal, Dan Ressler, Glenn Snyder
Companion diagnostics are an indispensable part of personalized medicine and will likely continue to rapidly increase in number and application to disease areas. The first companion diagnostics were launched in the 1980s and in the face of significant initial skepticism from drug developers as to whether segmenting a drug's market through a diagnostic was advisable. The commercial success of drugs such as Herceptin® (trastuzumab) and Gleevec® (imatinib), which both require testing with companion diagnostics before they can be prescribed, has moved the entire companion diagnostic field forward...
2015: Pharmacogenomics and Personalized Medicine
Joseph S Ross, Kristina Dzara, Nicholas S Downing
The regulatory approval of new drugs by the Food and Drug Administration (FDA) is a long and complex process and often requires multiple cycles of review, potentially delaying patients' access to new and effective therapeutics. We used qualitative methods to characterize the safety and efficacy reasons why applications for novel therapeutics approved by the FDA between 2001 and 2011 required multiple review cycles prior to approval. Among ninety-six applications approved between 2001 and 2011 that required multiple review cycles, safety concerns contributed to seventy-four (77...
April 2015: Health Affairs
Rosalind J Walley, Claire L Smith, Jeremy D Gale, Phil Woodward
This paper illustrates how the design and statistical analysis of the primary endpoint of a proof-of-concept study can be formulated within a Bayesian framework and is motivated by and illustrated with a Pfizer case study in chronic kidney disease. It is shown how decision criteria for success can be formulated, and how the study design can be assessed in relation to these, both using the traditional approach of probability of success conditional on the true treatment difference and also using Bayesian assurance and pre-posterior probabilities...
May 2015: Pharmaceutical Statistics
S Amur, L LaVange, I Zineh, S Buckman-Garner, J Woodcock
The discovery, development, and use of biomarkers for a variety of drug development purposes are areas of tremendous interest and need. Biomarkers can become accepted for use through submission of biomarker data during the drug approval process. Another emerging pathway for acceptance of biomarkers is via the biomarker qualification program developed by the Center for Drug Evaluation and Research (CDER, US Food and Drug Administration). Evidentiary standards are needed to develop and evaluate various types of biomarkers for their intended use and multiple stakeholders, including academia, industry, government, and consortia must work together to help develop this evidence...
July 2015: Clinical Pharmacology and Therapeutics
Ruth Etzioni, Roman Gulati, Daniel W Lin
BACKGROUND: A variety of measures of survival benefit are available to an investigator comparing outcomes across the various arms of a drug development trial. In this article, we systematically review the most common measures of comparative survival used in published studies. MATERIALS: We distinguish between relative and absolute survival differences, and measures of instantaneous and cumulative risk. We consider settings in which the end point is overall survival as well as those in which disease-specific end points are of primary interest...
March 2015: Urologic Oncology
Jeremy D Caplin, Norma G Granados, Myra R James, Reza Montazami, Nastaran Hashemi
In recent years, the exploitation of phenomena surrounding microfluidics has seen an increase in popularity, as researchers have found a way to use their unique properties to create superior design alternatives. One such application is representing the properties and functions of different organs on a microscale chip for the purpose of drug testing or tissue engineering. With the introduction of "organ-on-a-chip" systems, researchers have proposed various methods on various organ-on-a-chip systems to mimic their in vivo counterparts...
July 15, 2015: Advanced Healthcare Materials
Alexander Jarasch, Hans Koll, Joerg T Regula, Martin Bader, Apollon Papadimitriou, Hubert Kettenberger
Therapeutic antibodies and antibody derivatives comprise the majority of today's biotherapeutics. Routine methods to generate novel antibodies, such as immunization and phage-display, often give rise to several candidates with desired functional properties. On the contrary, resource-intense steps such as the development of a cell line, a manufacturing process, or a formulation, are typically carried out for only one candidate. Therefore, "developability," that is, the likelihood for the successful development of a lead candidate into a stable, manufacturable, safe, and efficacious drug, may be used as an additional selection criterion...
June 2015: Journal of Pharmaceutical Sciences
Ross MacPherson
No abstract text is available yet for this article.
July 2015: Journal of the Royal Society of Medicine
Asher Mullard
No abstract text is available yet for this article.
April 2015: Nature Reviews. Drug Discovery
Charles Oo, Jui-Chen Tsai, H Danny Kao
No abstract text is available yet for this article.
June 2015: Drug Discovery Today
Adrián M Ramos, Cristian González-Guerrero, Ana Sanz, María Dolores Sanchez-Niño, Laura Rodríguez-Osorio, Catalina Martín-Cleary, Beatriz Fernández-Fernández, Marta Ruiz-Ortega, Alberto Ortiz
INTRODUCTION: Kidney disease remains one of the last worldwide frontiers in the field of non-communicable human disease. From 1990 to 2013, chronic kidney disease (CKD) was the top non-communicable cause of death with a greatest increase in global years of life lost while mortality of acute kidney injury (AKI) still hovers around 50%. This reflects the paucity (for CKD) or lack of (for AKI) therapeutic approaches beyond replacing renal function. Understanding what the barriers are and what potential pathways may facilitate the design of new drugs to combat kidney disease is a key public health priority...
May 2015: Expert Opinion on Drug Discovery
Yong Zang, J Jack Lee
Adaptive designs have become popular in clinical trial and drug development. Unlike traditional trial designs, adaptive designs use accumulating data to modify the ongoing trial without undermining the integrity and validity of the trial. As a result, adaptive designs provide a flexible and effective way to conduct clinical trials. The designs have potential advantages of improving the study power, reducing sample size and total cost, treating more patients with more effective treatments, identifying efficacious drugs for specific subgroups of patients based on their biomarker profiles, and shortening the time for drug development...
December 2014: Chinese Clinical Oncology
Mata Charokopou, Istvan M Majer, Johan de Raad, Stefan Broekhuizen, Maarten Postma, Bart Heeg
OBJECTIVES: To identify the factors that influence the Scottish Medicines Consortium (SMC) in deciding whether to accept pharmaceutical technologies for use within the Scottish health care system. METHODS: A database of SMC submissions between 2006 and 2013 was created, containing a range of clinical, economic, and other factors extracted from published health technology assessment reports. A binomial outcome variable was used, defined as the decision to "accept for use" or "not recommend" a technology...
March 2015: Value in Health: the Journal of the International Society for Pharmacoeconomics and Outcomes Research
(no author information available yet)
FDA approvals, drug indications, and updates.
March 2015: P & T: a Peer-reviewed Journal for Formulary Management
Katsura Tsukamoto
PURPOSE: The incidence of Alzheimer's disease (AD) has been steadily increasing worldwide. AD is a serious disease that has both societal and economic impacts. The greatest risk factor for AD is aging. Thus, because of the rapidly aging population in Japan, the development of new, effective drugs for AD is urgently needed. The goal of the present article was to analyze the status, clarify the problems, and discuss the scientific and political challenges of disease-modifying drug development for AD...
August 2015: Clinical Therapeutics
Corinna Sorenson, Michael Drummond
CONTEXT: Recent debates and events have brought into question the effectiveness of existing regulatory frameworks for medical devices in the United States and Europe to ensure their performance, safety, and quality. This article provides a comparative analysis of medical device regulation in the two jurisdictions, explores current reforms to improve the existing systems, and discusses additional actions that should be considered to fully meet this aim. Medical device regulation must be improved to safeguard public health and ensure that high-quality and effective technologies reach patients...
March 2014: Milbank Quarterly
Sai-Hong Ignatius Ou, Ross A Soo, Akihito Kubo, Tomoya Kawaguchi, Myung-Ju Ahn
The discovery of anaplastic lymphoma kinase (ALK) rearrangement in non-small cell lung cancer (NSCLC) in 2007 and the approval of crizotinib for the treatment of advanced ALK-rearranged NSCLC in 2011 represents a landmark in the development of targeted oncology therapy. The approval of crizotinib was accompanied simultaneously by the approval of the Vysis (Abbott Molecular) break-apart fluorescence in situ hybridization (FISH) test as the companion diagnostic (CDx) test to detect ALK rearrangement. Pfizer, the manufacturer of crizotinib, sponsored the screening of thousands of patients and the standardization of the ALK FISH test as part of the approval process for crizotinib, a first in class ALK inhibitor...
2014: Frontiers in Oncology
Faiez Zannad, Wendy Gattis Stough, Ileana L Piña, Roxana Mehran, William T Abraham, Stefan D Anker, Gaetano M De Ferrari, Andrew Farb, Nancy L Geller, Robert S Kieval, Cecilia Linde, Rita F Redberg, Kenneth Stein, Alphons Vincent, Holger Woehrle, Stuart J Pocock
Several features of cardiovascular devices raise considerations for clinical trial conduct. Prospective, randomized, controlled trials remain the highest quality evidence for safety and effectiveness assessments, but, for instance, blinding may be challenging. In order to avoid bias and not confound data interpretation, the use of objective endpoints and blinding patients, study staff, core labs, and clinical endpoint committees to treatment assignment are helpful approaches. Anticipation of potential bias should be considered and planned for prospectively in a cardiovascular device trial...
July 15, 2014: International Journal of Cardiology
2015-03-24 00:54:19
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