Spartan Daily, October 8, 2003

Volume 121, Issue 29https://scholarworks.sjsu.edu/spartandaily/9895/thumbnail.jp

The potential investment impact of improved access to accelerated approval on the development of treatments for low prevalence rare diseases

<p>Abstract</p> <p>Background</p> <p>Over 95% of rare diseases lack treatments despite many successful treatment studies in animal models. To improve access to treatments, the Accelerated Approval (AA) regulations were implemented allowing the use of surrogate endpoints to achieve drug approval and accelerate development of life-saving therapies. Many rare diseases have not utilized AA due to the difficulty in gaining acceptance of novel surrogate endpoints in untreated rare diseases.</p> <p>Methods</p> <p>To assess the potential impact of improved AA accessibility, we devised clinical development programs using proposed clinical or surrogate endpoints for fifteen rare disease treatments.</p> <p>Results</p> <p>We demonstrate that better AA access could reduce development costs by approximately 60%, increase investment value, and foster development of three times as many rare disease drugs for the same investment.</p> <p>Conclusion</p> <p>Our research brings attention to the need for well-defined and practical qualification criteria for the use of surrogate endpoints to allow more access to the AA approval pathway in clinical trials for rare diseases.</p

University of Malaya Research Bulletin, Volume 17, Number 1, 2017

Previously known as IPPP UM Research Bulleti

UniverCity news (2000-01-10)

VCU Today, the University’s first official administrative organ, began as a somewhat irregular monthly publication but moved to a bi-weekly newspaper format in the 1980s. The newspaper changed its name to VCU Voice in 1988 and ten years later it appeared under the title UniverCity News. As it neared the end of its run as a physical newspaper, the publication became simply VCU News. These four publications were essentially the same periodical published under different titles by the Office of University Relations. VCU News appeared online for the first time in 2002.https://scholarscompass.vcu.edu/ucn/1029/thumbnail.jp

Pharmacotherapies for COPD

This review article summarizes the main treatments for chronic obstructive pulmonary disease, their mechanisms, and the key evidence from trials supporting their use. Drug classes covered were short acting beta agonists (SABA), short acting muscarinic antagonists (SAMA), long acting beta agonists (LABA), long acting antimuscarinics (LAMA), inhaled corticosteroids (ICS), LABA/ ICS combinations, specific phosphodiesterase (PDE4) inhibitors, non-specific PDE inhibitors, mucolytics, and oxygen. Non-specific therapies, such as opiates for relief of dyspnoea and therapies for smoking cessation, are also covered briefly. For each class of drug, mechanisms of action are described, key clinical trial results are reported, and available agents compared. Finally, the place of each drug in therapy is compared between current worldwide guidelines

Crossover designs: issues in construction, use, and communication

PhDIn a trial with a crossover design, participants receive a sequence of treatments over two or more periods, with the outcome measured at the end of each period. In order to estimate contrasts between direct treatment effects and between carryover treatment effects, we model each observation as a linear combination of the effects of period, participant, the direct effect of the current treatment, and, for all except the first period observations, the carryover effect of the treatment in the preceding period. In this thesis, we will consider some aspects of the design and use of crossover experiments. Our focus will be on methods for construction and comparison of designs which improve performance and are accessible to those researchers who need to use crossover designs but who are not specialists in statistical methodology or the design of experiments. In Chapter 2 we discuss the construction of balanced crossover designs. In Chapter 3 we consider visual methods for determining the connectedness of block, row- column, and a restricted class of crossover designs. In Chapter 4 we discuss participant dropout in crossover designs, and introduce a new criterion for selecting a design that is less likely to result in non-estimable treatment contrasts in the event of some participants not completing the trial. In Chapter 5 we present a review of the use of crossover designs in the scientific literature during a one-year period. In Chapter 6 we discuss the relationship between the theory of crossover designs as described in the earlier chapters, and the reality of the use of crossover designs as described in Chapter 5. We conclude by discussing potential practical approaches for making some experimental design methods more widely known and used by researchers who implement trials with crossover designs

Applications of Data Mining in Healthcare

Indiana University-Purdue University Indianapolis (IUPUI)With increases in the quantity and quality of healthcare related data, data mining tools have the potential to improve people’s standard of living through personalized and predictive medicine. In this thesis we improve the state-of-the-art in data mining for several problems in the healthcare domain. In problems such as drug-drug interaction prediction and Alzheimer’s Disease (AD) biomarkers discovery and prioritization, current methods either require tedious feature engineering or have unsatisfactory performance. New effective computational tools are needed that can tackle these complex problems. In this dissertation, we develop new algorithms for two healthcare problems: high-order drug-drug interaction prediction and amyloid imaging biomarker prioritization in Alzheimer’s Disease. Drug-drug interactions (DDIs) and their associated adverse drug reactions (ADRs) represent a significant detriment to the public h ealth. Existing research on DDIs primarily focuses on pairwise DDI detection and prediction. Effective computational methods for high-order DDI prediction are desired. In this dissertation, I present a deep learning based model D 3 I for cardinality-invariant and order-invariant high-order DDI pre- diction. The proposed models achieve 0.740 F1 value and 0.847 AUC value on high-order DDI prediction, and outperform classical methods on order-2 DDI prediction. These results demonstrate the strong potential of D 3 I and deep learning based models in tackling the prediction problems of high-order DDIs and their induced ADRs. The second problem I consider in this thesis is amyloid imaging biomarkers discovery, for which I propose an innovative machine learning paradigm enabling precision medicine in this domain. The paradigm tailors the imaging biomarker discovery process to individual characteristics of a given patient. I implement this paradigm using a newly developed learning-to-rank method PLTR. The PLTR model seamlessly integrates two objectives for joint optimization: pushing up relevant biomarkers and ranking among relevant biomarkers. The empirical study of PLTR conducted on the ADNI data yields promising results to identify and prioritize individual-specific amyloid imaging biomarkers based on the individual’s structural MRI data. The resulting top ranked imaging biomarkers have the potential to aid personalized diagnosis and disease subtyping

Patient Safety in Pediatrics: a Developing Discipline

__Abstract__ The publication of the breakthrough report “To Err is Human” by the Institute of Medicine was the launch of patient safety initiatives all over the world. In the intensive care unit (ICU) of the Erasmus MC-Sophia Children’s Hospital this resulted in the institution of a multimodal patient safety management system under the name Safety First in 2005. This system now includes nine major elements, representing monitoring and intervention activities. In this thesis we report on the results and the implementation of the patient safety management system called Safety First. __Outline of this thesis:__ In part I the concept of patient safety and the Safety First project are introduced. The rationale for selecting the elements of the patient safety management system is explained. As preventable mortality and morbidity are the public focus as outcome parameters for quality and safety of care, we have studied very long stay patients in our ICU (chapter 2). The goal of this study was to determine characteristics and mortality in these patients as well as modes of death. Chapter 3 presents an evaluation of potentially preventable deaths in our ICU. An important question was whether five years of patient safety efforts had resulted in fewer potentially preventable deaths. Part II reflects on the difficulties in monitoring adverse events. In chapter 4 we present numbers and types of adverse events identified with real time physicians’ registration during a 3-month period in general pediatric practice. The next chapter is a study into adverse events in the surgical pediatric ICU in a 2-year period. We combined the physicians’ registration with the Trigger Tool methodology as developed by the Institute for Healthcare, Boston, USA. The goals were to determine the rate and nature of the adverse events and to compare the two methods. In part III a number of elements of Safety First are described, as well as other studies into patient safety issues relevant to bedside ICU care. Chapter 6 brings the results of critical incident analysis with a focus on the factors contributing to the incident and the resultant recommendations. The next study evaluated the availability and reliability of drug formularies used in our ICU, which are crucial in safe drug prescription. In chapter 8 we discuss the safety of routine MRI scans in preterm infants at 30 weeks gestational age, as reflected by safety incidents and adverse events. In the next chapter, safety focused Mortality and Morbidity conference reports were scrutinized for numbers and types of recommendations stemming from these meetings. Chapter 10 is a study about nursing protocol violations established with the Critical Nursing Situation Index. Part IV describes a study of safety culture in the ICU, as it emerged from a safety attitude questionnaire administered to all staff. We aimed to compare findings to benchmark data and explore any deficiencies. In the general discussion in part V the results of the studies are commented on and future directions are given, including guidelines for optimal implementation of a patient safety management system and future benchmarking

CVM news, 2013

2013 compilation of news and events articles published by the University of Missouri College of Veterinary Medicine.Previous Studies on Toxic Effects of BPA Couldn't... -- CVM's Cross Honored for Teaching Excellence -- CVM's Thorne, Buckaloo Honored by the MVMA -- Extension's Craig Payne is New MVMA President -- Change for Animals of Missouri Raises Funds for ReCHAI -- The Lyons' Den is Moving into the Tiger's Lair -- Drug Therapy May Bridge DM and ALS -- MU Raptor Project Come to Aid of Injured Illinois Eagle -- Minimally Invasive Orthopaedic Repairs Help Speed Recovery -- Certain Breast Cancers Could be Attacked by New Therapies -- CVM Alumnus Selected to Lead AAVC -- Foundation Awards $50,000 to study Therapeutic Riding -- MU Veterinary Hospital Saves a Horse Named Bunny -- MU Professor Awarded Kemper Fellowship -- Couch Potatoes May Be Genetically Predisposed -- Vaccine Developer to Address Class of 2013 -- Honor Society Taps CVM's Tappmeyer -- Booth Honored for Discussion-Provoking Reviews -- MU Veterinary Team Beats Odds to Save Horse's Vision -- Cook Honored for Enhancing Pets' Lives -- Student and Faculty Achievements Recognized -- Newest Veterinarians Learn the Value of Normal -- Former MU CVM Dean Kenneth Weide Has Passed Away -- Radioactive Nanoparticles That Target Cancer Cells -- Rubin Named Interim Dean of Graduate School -- CVM Faculty Part of Team Receiving Funds for Study -- MU Veterinarian Part of Team Establishing Trauma Network -- Hog Producers Should Take Steps to Avoid PEDV -- Associate Dean for Research, Graduate Studies to Retire -- MACC Welcomes New Director -- Researchers Find Condition in Dogs that May Help Further... -- Look Out For Ergot in Pastures -- Vet Med Students Traveled to South Africa -- Multidisciplinary Research Team Gives Voice to Airway Problems -- Cisco's Legacy : Research into Deadly Disease -- CVM Alumnus Named Iron Paw Recipient -- New VMDL Director Named -- The Vet Specialist : A visit with Professor Cohn -- Michael Lairmore is CVM 2013 Alumnus of the Year -- Accreditation Renewed for MU College of Veterinary Medicine -- Alumnus Jack Stephens is a Pioneer for Pets -- St. Louis comedian leaves$75,000 gift for Barkley House -- Guide Dogs : Comparing Prostate Cancer in Men & Dogs -- Veterinary Products Day Unites Companies with Future Clients -- Motorcycle Donation Revs Up Benefit -- Using Electrical Impedance Techniques to Assess... -- New Faculty Members Join CVM -- Meadows Earns Veterinary Dental Teaching Award -- Older Siblings' Cells Can Be Passed From Female Dogs... -- White Coat Ceremony Ushers in Clinical Training -- CVM Resident Wins Research Award -- Dr. Alex Bermudez Honored for Lasting Impact on CVM -- Pet Owners Should Be Aware but Not Panic about Pet Treats -- Dr. Henry Elected to National Health Care Advisory Group -- Low Levels of Blood Calcium in Dairy Cows May Affect Cow... -- MU SW Center Dairy Honored for Reproductive Efficiency -- New MU Program Offers Grief Counseling to People... -- Quality Beef Team Receives USDA Award -- Miracle Dog Fights to Recover After Brutal Beating -- Johnson Elected to Equine Veterinary Hall of Fame -- Alumna Wins Veterinary Pathology Award -- CVM Staff Member Named MU Service Champion -- Outgoing Texas A&M president becomes Mizzou's new... -- Devastating Fracture Heals Thanks to Expertise of... -- CVM Alumna Takes Raptor Project Under Her Wing -- ACVO honors MU's Elizabeth Giuliano -- Biological Patches May Treat Diseased Blood Vessels -- Ophthalmology Group Enjoys Success at Conference -- Research Association Names Rajiv Mohan a Silver Fellow -- CVM's Philip Johnson Helps Make Equine Research..