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Shang W.,CAS Academy of Mathematics and Systems Science | Chen H.,University of Arizona | Livoti C.,Deerfield Institute
Online Information Review | Year: 2017

Purpose: The purpose of this paper is to propose a framework to detect adverse drug reactions (ADRs) using internet user search data, so that ADR events can be identified early. Empirical investigation of Avandia, a type II diabetes treatment, is conducted to illustrate how to implement the proposed framework. Design/methodology/approach: Typical ADR identification measures and time series processing techniques are used in the proposed framework. Google Trends Data are employed to represent user searches. The baseline model is a disproportionality analysis using official drug reaction reporting data from the US Food and Drug Administration's Adverse Event Reporting System. Findings: Results show that Google Trends series of Avandia side effects search reveal a significant early warning signal for the side effect emergence of Avandia. The proposed approach of using user search data to detect ADRs is proved to have a longer leading time than traditional drug reaction discovery methods. Three more drugs with known adverse reactions are investigated using the selected approach, and two are successfully identified. Research limitations/implications: Validation of Google Trends data's representativeness of user search is yet to be explored. In future research, user search in other search engines and in healthcare web forums can be incorporated to obtain a more comprehensive ADR early warning mechanism. Practical implications: Using internet data in drug safety management with a proper early warning mechanism may serve as an earlier signal than traditional drug adverse reaction. This has great potential in public health emergency management. Originality/value: The research work proposes a novel framework of using user search data in ADR identification. User search is a voluntary drug adverse reaction exploration behavior. Furthermore, user search data series are more concise and accurate than text mining in forums. The proposed methods as well as the empirical results will shed some light on incorporating user search data as a new source in pharmacovigilance. © Emerald Publishing Limited.


Audibert C.,Deerfield Institute | Perlaky A.,Deerfield Institute | Glass D.,Deerfield Institute
Contemporary Clinical Trials Communications | Year: 2017

Objectives To examine colorectal cancer screening practices among colonoscopy specialists from 5 countries and inform public health needs in improvement of the ongoing global crisis in colorectal cancer. Methods An online survey among colonoscopy specialists was conducted in France, Germany, the United Kingdom, Japan, and the United States. The survey covered topics on colonoscopy practices in the screening as well as in the treatment setting, as well as expected trends. Results Participating colonoscopy specialists included 114 physicians from the United States, 81 from France, 80 from Germany, 80 from the United Kingdom, and 156 from Japan. Survey results revealed that 59%–73% of colonoscopies were performed in patients aged 50–75 years old, with 15%–23% performed in patients <50 years old. The proportion of patients with age-based versus symptom-based first colorectal cancer screening varied by country and age. Sedation protocols varied by country; however, rate of incomplete colonoscopy was low in all countries. The proportion of negative first colonoscopies decreased with age in all countries. Conclusions This multi-country survey of real-world clinical practices suggests a need for improved participation in population age-based colorectal cancer screening and possibly younger age of screening initiation than currently recommended by guidelines. The variation among countries in the proportion of patients who received their first colonoscopy due to age-based colorectal cancer screening versus symptom-based initial colonoscopy indicates that population-based screening initiatives and improved health outcomes will benefit from public health awareness programs. © 2017 The Authors


News Article | February 17, 2017
Site: www.biosciencetechnology.com

An expert panel convened in New York City on Tuesday at the BIO CEO and Investor Conference to discuss public health policy under the new Administration and what it might mean for the biotech industry. To start things off, moderator Susan Peschin, president and CEO of the Alliance for Aging Research, said that the discussion would take a look at what is likely to happen, and separate it from the hype that has grabbed news headlines. Two topics that panelists dove into were the Affordable Care Act, and thoughts on a new Food and Drug Administration (FDA) Commissioner. Overall the industry experts seemed to agree that a piece of legislation repealing the ACA will pass in 2017, but all were cautious with predictions as to what form it would take. Alex Azar II, Chairman of Seraphim Strategies LLC, former president of Lilly USA, and former Deputy Secretary of the U.S. Department of Health and Human Services from 2005 to 2007, said that he believes there’s been a broad mischaracterization as to what the debate is over healthcare. There is a responsibility to get healthcare to those who can’t afford it, he said, but the question is over how to go about that. “Some people in my industry disagree with the path Obama took, but it doesn’t mean we don’t believe people should have insurance,” Azar said. Straight repeal of the ACA won’t be acceptable without a replacement, in Azar’s eyes, a sentiment many of the panelists echoed. Panelist Jeanne Haggerty, senior vice president of Federal Government Relations for Biotechnology Innovation Organization (BIO) said that there’s currently a lot of disagreement about the path forward for ACA repeal and replace, and she thinks it’s likely we’ll see a number of hearings and potential mark ups on smaller bills.  Congress could potentially keep moving smaller pieces of legislation through one at a time, and then eventually package together. “Most republican leadership are feeling the heat of constituencies that don’t fully understand repealing and replacing won’t leave people without health insurance,” Haggerty said. Azar noted that it’s important to remember when thinking about repeal and replace that there is a “prime directive for politicians – to get re-elected.” On Thursday senior House Republicans revealed a preliminary outline of healthcare overhaul.  It involves a revamped Medicaid program for the poor, tax breaks to help people pay doctors’ bills and federally subsidized state pools to support those with costly medical conditions in buying insurance, the Associated Press reported. Panelist Johnathan Leff, partner at Deerfield Management and Chairman of the Deerfield Institute, gave some insight into what kind of implications repeal without a clear replacement would have on investments in the biotech industry.  While it’s fairly evident that there will be some kind of bill repealing ACA, Leff said, there is a right way and a wrong way to do it. Investors, especially in the biotech world where investments are made over a long period of time, don’t like uncertainty, according to Leff. Uncertainty can have a very negative impact on investments, Leff said and in his eyes “the right way to do it is replace when you repeal.” As long as legislation is rolled out in a way that people can see changes coming, it will be better from an investor point of view, he said. Leff is optimistic that “reason will prevail” and that people won’t dismantle what is in place without knowing what to replace it with.  However, he noted that in this environment with the current Administration there’s always a chance that won’t happen. He said he replace and repeal will hopefully be less disruptive than some other recent changes, such as Trump’s temporary immigration ban. Bryan Rye, a Senior Health Policy Analyst at Bloomberg Intelligence used an analogy about replacing a bridge. “You don’t just blow up the bridge, you provide temporary support,” he said. If the ACA was working as well as promised, Hilary Clinton might be president, Rye said, noting that there were not enough young healthy people in exchanges and insurers were pulling out. He expects that in new legislation there will be more emphasis on HSA’s, and no mandates, or at least penalties associated with them will go away. He does not think the process will be an easy one. “Don’t expect for it to be a kumbaya between republicans and democrats,” Rye said. FDA implications and thoughts on safety and efficacy policy issues The general consensus with the panel was that potential changes at the FDA could have a big impact on biotech, both in medical advances and investment climate. “In some ways it feels like an existential moment for the FDA,” Leff said. He noted that some of the people who have been rumored candidates for FDA commissioner have made statements that drugs should not be regulated for efficacy. “The FDA for the last 40 years has been about regulating efficacy and safety, if that were to change it would make it fundamentally different to how we think about developing and making drugs,” Leff said. A major change like that would have a “very very negative impact” on biotech investment, Leff explained. He said that in his own informal poll of talking to people in the industry gave him the sense that the current FDA environment works quite well, and that everyone has designed activity in line with the framework that’s been in place. Some people are unhappy with certain aspects of the FDA, Leff admitted, but said those were micro areas of concern.  It would become exceedingly difficult to invest in science-driven biotechnology if the need to test efficacy was eliminated, Leff said and would make most biotech executives nervous. “Throwing out the idea of drug regulation is a very dangerous idea,” Leff said, and emphasized that making policy makers understand that is very important. Good policy would work to continue to improve the process, such as improvement in clinical trial design. Leff pointed out that President Trump’s pick to lead the Environmental Protection Agency (EPA), Scott Pruitt, is someone who doesn’t fundamentally believe in the need for the agency.  Some people think that by appointing a person who questions the underlying mission of that agency that they are pursuing a pro-growth agenda, because there are unnecessary regulations that prohibit development – however the opposite is true in biotech, Leff said. He wants to see an FDA commissioner appointed that believes in the basic mission of the agency. Haggerty was also concerned over stopping the regulation of efficacy by the FDA, noting it is an important part of the drug approval process. She said it’s BIO’s job to educate people going into the FDA about changes that have happened in 21st century cures, such as increased biomarkers. Another goal of the organization is to make sure the Prescription Drug User Fee Act (PDUFA) is reauthorized when it is set to expire this September.  PDUFA was a law passed by Congress in 1992 which allows the FDA to collect fees from drug manufactures to fund the new drug approval process, and must be reauthorized every five years. Keeping the agreement intact is a primary objective for the organization, Haggerty said. Ultimately, it’s about making sure patients get access to therapies they need without disrupting the market, Haggerty said. In a time of uncertainty, the biotech industry will continue to watch any new developments closely.


He P.,Genentech | Su Z.,Deerfield Institute
Contemporary Clinical Trials Communications | Year: 2015

The semi-parametric proportional hazards model is widely adopted in randomized clinical trials with time-to-event outcomes, and the log-rank test is frequently used to detect a potential treatment effect. Immuno-oncology therapies pose unique challenges to the design of a trial as the treatment effect may be delayed, which violates the proportional hazards assumption, and the log-rank test has been shown to markedly lose power under the non-proportional hazards setting. A novel design and analysis approach for immuno-oncology trials is proposed through a piecewise treatment effect function, which is capable of detecting a potentially delayed treatment effect. The number of events required for the trial will be determined to ensure sufficient power for both the overall log-rank test without a delayed effect and the test beyond the delayed period when such a delay exists. The existence of a treatment delay is determined by a likelihood ratio test with resampling. Numerical results show that the proposed design adequately controls the Type I error rate, has a minimal loss in power under the proportional hazards setting and is markedly more powerful than the log-rank test with a delayed treatment effect. © 2015 The Authors.


He P.,Genentech | Lai T.L.,Stanford University | Su Z.,Deerfield Institute
Contemporary Clinical Trials | Year: 2015

Time to event is the clinically definitive endpoint in Phase III trials of new treatments of cancer, cardiovascular and many other diseases. Because these trials involve relatively long follow-up, their protocols usually incorporate periodic interim analyses of the data by a Data and Safety Monitoring Board/Committee. This paper gives a review of the major developments in the design of these trials in the 21st century, spurred by the need for better clinical trial designs to cope with the remarkable advances in cancer biology, genomics and imaging that can help predict patients' sensitivity or resistance to certain treatments. In addition to this overview and discussion of related issues and challenges, we also introduce a new approach to address some of these issues. © 2015 Elsevier Inc. All rights reserved.


Lin Y.,Takeda Development Center Americas Inc. | Zhu M.,Abbvie Inc. | Su Z.,Deerfield Institute
Contemporary Clinical Trials | Year: 2015

Randomization is fundamental to the design and conduct of clinical trials. Simple randomization ensures independence among subject treatment assignments and prevents potential selection biases, yet it does not guarantee balance in covariate distributions across treatment groups. Ensuring balance in important prognostic covariates across treatment groups is desirable for many reasons. A broad class of randomization methods for achieving balance are reviewed in this paper; these include block randomization, stratified randomization, minimization, and dynamic hierarchical randomization. Practical considerations arising from experience with using the techniques are described. A review of randomization methods used in practice in recent randomized clinical trials is also provided. © 2015 Elsevier Inc. All rights reserved.


Daniel G.W.,Engelberg Center for Health Care Reform at Brookings | Caze A.,Deerfield Institute | Romine M.H.,Engelberg Center for Health Care Reform at Brookings | Audibert C.,Deerfield Institute | And 2 more authors.
Health Affairs | Year: 2015

New drugs and biologics have had a tremendous impact on the treatment of many diseases. However, available measures suggest that pharmaceutical innovation has remained relatively flat, despite substantial growth in research and development spending. We review recent literature on pharmaceutical innovation to identify limitations in measuring and assessing innovation, and we describe the framework and collaborative approach we are using to develop more comprehensive, publicly available metrics for innovation. Our research teams at the Brookings Institution and Deerfield Institute are collaborating with experts from multiple areas of drug development and regulatory review to identify and collect comprehensive data elements related to key development and regulatory characteristics for each new molecular entity approved over the past several decades in the United States and the European Union. Subsequent phases of our effort will add data on downstream product use and patient outcomes and will also include drugs that have failed or been abandoned in development. Such a database will enable researchers to better analyze the drivers of drug innovation, trends in the output of new medicines, and the effect of policy efforts designed to improve innovation. © 2015 Project HOPE-The People-to-People Health Foundation, Inc.


Audibert C.,Deerfield Institute | Glass D.,Deerfield Institute
Reproductive Biology and Endocrinology | Year: 2015

Background: Procedures that may optimize success in achieving live births from assisted reproductive technology (ART) continue to be examined. Not yet considered are the perspectives of fertility specialists regarding important developments in the fertility treatment field, current unmet needs, and anticipated future advances. In the current study, an 8-country survey of fertility specialists was conducted to provide a comprehensive, global depiction of fertility treatments across different regions. Methods: Fertility specialists from France, Germany, Italy, Spain, the United Kingdom (UK), the United States (US), China, and Japan were invited to participate in an online survey. Participants were eligible if they personally managed ≥25 patients/month who were experiencing difficulty conceiving, and if they had performed ART fertility treatment with ≥1 patient in the previous month. Quantitative questions addressed the number of patients seen, main infertility causes, number of cycles performed, ART procedure type, and ART outcomes. Qualitative questions covered diagnostic trends, unmet needs, important advances, and expected future developments. Results: The number of fertility specialists who completed the survey included 29 in France, 33 in Germany, 23 in Italy, 38 in Spain, 34 in the UK, 91 in the US, 50 in China, and 65 in Japan. Patient volume increased over the prior 2 years according to 67 % (242/363) of the fertility specialists. As expected, ART outcomes all declined with age in all countries. ART outcomes varied by country, with the highest implantation, pregnancy, and live birth rates reported by fertility specialists in the US and China and the lowest rates reported in France and Italy. The most frequently reported unmet needs in fertility treatment were financial coverage, improved implantation rate, and egg donation. Most frequently named future advancements expected to change the fertility treatment field included improved embryo selection through imaging and/or metabolomics, improved embryo implantation rate, and use of preimplantation genetic diagnosis. Conclusions: This study, which follows a rigorous survey methodology, elucidates the current state of fertility specialists' practices and perspectives on the global fertility treatment field, which highlights differences and similarities among countries. This research may inform further studies and procedural developments that might better improve and standardize ART. © 2015 Audibert and Glass.


Stuntz M.,Deerfield Institute | Des Vignes F.,Deerfield Institute
Contemporary Clinical Trials Communications | Year: 2015

Fecal microbiota transplantation (FMT) has emerged as a highly effective treatment for Clostridium difficile infection (CDI), the most frequent cause of hospital-acquired infectious diarrhea in developed countries and the cause of nearly 30,000 annual deaths in the US. FMT is proving to be more effective at treating CDI than traditional antibacterial therapy, and reduces the exposure of valuable antibiotics to potential resistance. A systematic review to assess the efficacy of FMT for CDI treatment showed that across all studies for recurrent CDI, symptom resolution was observed in 85% of patients. The United States Food and Drug Administration currently classifies FMT as an investigational drug, which imparts overly restrictive regulations that are impossible to apply to FMT in the same manner as conventional drugs. Reclassification of FMT to a human cell, tissue, and cellular and tissue-based product could potentially expand access to this important treatment while maintaining rigorous safety standards. © 2015 The Authors.


Stuntz M.,Deerfield Institute
Cardiology (Switzerland) | Year: 2016

Objectives: Abdominal aortic aneurysm (AAA) is a pathological condition characterized by an abnormal, localized dilatation of the lower part of the aorta. Due to a lack of data on the natural history of AAA and risk of death from other cardiovascular diseases attributable to AAA, the true number of AAA-attributable deaths may be higher than currently estimated. This study aims to produce more realistic estimates of the burden of AAA. Methods: A disease-modeling software, DisMod II, was used to assess the AAA burden via a multistate life table. Inputs included population, all-cause mortality, size- and sex-specific AAA prevalence, and relative risk of death estimates for persons with AAA compared with persons without AAA. Results: There were 2,347,339 prevalent cases of AAA in the USA in 2013 (95% CI: 2,131,964-2,524,116), resulting in 41,371 deaths attributable to AAA (95% CI: 34,090-49,234). Females constituted 21.1% of prevalent cases and 45.2% of deaths, compared with males constituting 78.9% of prevalent cases and 54.8% of deaths. Conclusions: This work shows that the burden of mortality attributable to AAA is more than twice the current estimates from the American Heart Association. Females account for a disproportionately high percentage of deaths despite constituting a low percentage of prevalent cases. © 2016 The Author(s) Published by S. Karger AG, Basel

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