Medicine and Health Sciences Commons^™

Forum 5: Understanding Vaccine Efficacy And Effectiveness: A Statistician's Perspective/Open Access Stem Research, Jyotishka Datta, Angie Ohler

Vaccine Course

In the first part of this presentation, Jyotishka Datta discusses the concepts and calculations behind the efficacy and effectiveness for vaccines from a statistician’s perspective, as well as the different factors contributing to it. Prof. Datta also offers a brief tour through the history of vaccines in preventing infectious diseases and the development of statistical tools such as randomized controlled trials. Lastly, Prof. Datta discusses the direct and indirect effects, the inherent uncertainties involved with the efficacy and efficiency numbers and remark on their comparison. In the second part of this presentation, Angie Ohler discusses the vital role of open …

Project Yes! Youth Engaging For Success: A Randomized Controlled Trial Testing A Peer Mentoring Approach Among Hiv-Positive Adolescents And Young Adults In Ndola, Zambia, Julie Denison, Virginia M. Burke, Sam Miti, Bareng A.S. Nonyane, Christiana Frimpong, Katherine G. Merrill, Elizabeth A. Abrams, Jonathan Kaunda Mwansa

HIV and AIDS

This research addresses the gaps in knowledge about how to best support adolescents and young adults transitioning to HIV self-management in the context of both child-focused and adult-focused HIV care settings. Johns Hopkins University, in partnership with the Arthur Davison Children’s Hospital, implemented this study through the USAID-funded Project SOAR (led by the Population Council).

Evaluating The Efficacy Of Vertebral Axial Decompression Therapy In Treatment Of Patients With Chronic Lower Back Pain: A Systematic Review, James M. Mckivigan, Brittany Krisak, Brittni Vogel, Ashley Harrington, Van Nguyen

College of Health & Human Services (TUN) Publications and Research

The primary objective of the study was to explore the effectiveness of Vertebral Axial Decompression (VAX-D) in treating patients with chronic low back pain (CLBP) as a safe and competent therapeutic method. Also, to determine the quality of life in alleviating chronic lumbar pain using mechanical Lumbar traction force applied to the lumbar spine. A systematic review and meta-analysis involving detailed literature survey on Vertebral Axial Decompression (VAX-D) therapy for patients with chronic low back pain were conducted in three databases namely MEDLINE, EMBASE and Cochrane Library from (January 1994 to February 2019). Studies supporting the outcomes with qualitative statistical …

Using Marginal Structural Models To Control For Time-Dependent Confounding And Detect Effect Modification In A Randomized Control Trial With A Time-Varying Exposure, Non-Adherence And Missing Data, Elizabeth A. Lancet

Dissertations and Theses

Background: Unlike traditional regression used in the Intention to Treat (ITT) approach, Marginal Structural Models (MSM) can account for joint effects of baseline and subsequent treatments as well as the presence of time-dependent confounding influenced by prior treatment and selection bias due to censoring. In addition, MSMs have been theorized to be able to assist investigators in determining the overall benefit of a drug in the total population as they are able to provide a summary effect size across all strata of an effect modifier which cannot be done via tradition regression techniques. The overall goal of this dissertation is …

The Nia Project: Baseline Survey Instruments, Karen Austrian

Poverty, Gender, and Youth

This document includes all of the Nia Project baseline survey instruments used to collect data in Kenya, providing a multidimensional description of a sample of the participating girls and the schools they attend. The Project is one of the first randomized controlled trials to explore the role of sanitary pad distribution and reproductive health education—individually and in combination—to improve girls’ educational and sexual and reproductive health outcomes. The study collected data from 3,489 girls from 140 schools in three rural Kilifi subcounties: Magarini, Kaloleni, and Ganze.

The Nia Project—Brief, Eunice N. Muthengi, Emily Farris, Karen Austrian

Poverty, Gender, and Youth

This brief provides highlights from the Nia Project conducted in Kenya. The study found that while gaps exist in girls’ capabilities, particularly knowledge and attitudes related to menstruation and reproductive health, the Nia Project has the potential to bridge those gaps. The Project is one of the first randomized controlled trials to explore the role of sanitary pad distribution and reproductive health education—individually and in combination—to improve girls’ educational and sexual and reproductive health outcomes. The findings of this study will make a critical contribution to filling an evidence gap in the field of menstrual health and hygiene management, and …

The Nia Project: Baseline Report, Eunice N. Muthengi, Emily Farris, Karen Austrian

Poverty, Gender, and Youth

The onset of puberty and menarche is a particularly vulnerable time for girls, when external pressures are exacerbated by their lack of knowledge of their bodies, their rights, and the implications of their decisions, and by their inability to manage puberty and adolescence safely and comfortably with appropriate menstrual health and hygiene management (MHM) products. Although several programs have previously been developed to address girls’ MHM needs globally, few have been evaluated, and where evidence does exist the results have been mixed. In collaboration with ZanaAfrica, the Population Council is evaluating the Nia Project, a set of interventions for adolescent …

Adolescent Girls Empowerment Program (Agep): Evaluation—Round 4 Update, Karen Austrian, Paul C. Hewett, Erica Soler-Hampejsek, Jean Digitale

Poverty, Gender, and Youth

To assess the impact of the Adolescent Girls Empowerment Program (AGEP) on mediating and longer-term demographic, reproductive, and health outcomes, Population Council researchers designed and implemented a longitudinal, cluster randomized controlled trial across all program areas. A baseline survey was conducted in 2013 prior to program implementation and data have been collected annually. A third round of data collection in 2015 produced the midline findings, measuring the program effect immediately at the end of AGEP. Details of the midterm results were published in a full technical report, executive summary, and brief. The focus of this brief is to provide an …

Adolescent Girls Empowerment Program (Agep): Adapting The Safe Spaces Model In Rural Settings, Population Council

Poverty, Gender, and Youth

The Adolescent Girls Empowerment Program (AGEP) was implemented with over 10,000 vulnerable adolescent girls aged 10–19 between 2013 and 2016 in five rural and five urban sites in Zambia. Safe Spaces was the core program element in AGEP, and was adapted based on prior programs implemented mainly in urban and peri-urban areas in East and Southern Africa. Given the key differences in the living environments and demographic data for adolescent girls in urban as compared to rural areas in Zambia, one key programmatic learning question for AGEP was if the Safe Spaces model could be adapted and successfully implemented in …

Neonatal Mortality Within 24 Hours Of Birth In Six Low- And Lower-Middle-Income Countries., Abdullah H. Baqui, Dipak K. Mitra, Muhammad Imran Nisar, Fyezah Jehan, Muhammad Ilyas, Murtaza Ali, Imran Ahmed, Shabina Arif, Sajid B. Soofi, Sachiyo Yoshida

Department of Paediatrics and Child Health

Objective: To estimate neonatal mortality, particularly within 24 hours of birth, in six low- and lower-middle-income countries.

Methods: We analysed epidemiological data on a total of 149 570 live births collected between 2007 and 2013 in six prospective randomized trials and a cohort study from predominantly rural areas of Bangladesh, Ghana, India, Pakistan, the United Republic of Tanzania and Zambia. The neonatal mortality rate and mortality within 24 hours of birth were estimated for all countries and mortality within 6 hours was estimated for four countries with available data. The findings were compared with published model-based estimates of neonatal …

Missing Data Frequency And Correlates In Two Randomized Surgical Trials For Urinary Incontinence In Women, Linda Brubaker, Heather J Litman, Hae-Young Kim, Philippe Zimmern, Keisha Dyer, John W Kusek, Holly E Richter, Anne Stoddard

NYMC Faculty Publications

INTRODUCTION AND HYPOTHESIS: Missing data is frequently observed in clinical trials; high rates of missing data may jeopardize trial outcome validity.

PURPOSE: We determined the rates of missing data over time, by type of data collected and compared demographic and clinical factors associated with missing data among women who participated in two large randomized clinical trials of surgery for stress urinary incontinence, the Stress Incontinence Surgical Treatment Efficacy Trial (SISTEr) and the Trial of Midurethral Sling (TOMUS).

METHODS: The proportions of subjects who attended and missed each follow-up visit were calculated. The chi-squared test, Fisher's exact test and t test …

Inadequate Reporting Of Research Ethics Review And Informed Consent In Cluster Randomised Trials: Review Of Random Sample Of Published Trials, Monica Taljaard, Andrew Mcrae, Charles Weijer, Carol Bennett, Stephanie Dixon, Julia Taleban, Zoe Skea, Martin P Eccles, Jamie Brehaut, Allan Donner, Raphael Saginur, Robert Boruch, Jeremy Grimshaw

Charles Weijer

OBJECTIVES: To investigate the extent to which authors of cluster randomised trials adhered to two basic requirements of the World Medical Association's Declaration of Helsinki and the International Committee of Medical Journal Editors' uniform requirements for manuscripts (namely, reporting of research ethics review and informed consent), to determine whether the adequacy of reporting has improved over time, and to identify characteristics of cluster randomised trials associated with reporting of ethics practices.

DESIGN: Review of a random sample of published cluster randomised trials from an electronic search in Medline.

SETTING: Cluster randomised trials in health research published in English language journals …

Inadequate Reporting Of Research Ethics Review And Informed Consent In Cluster Randomised Trials: Review Of Random Sample Of Published Trials, Monica Taljaard, Andrew Mcrae, Charles Weijer, Carol Bennett, Stephanie Dixon, Julia Taleban, Zoe Skea, Martin P Eccles, Jamie Brehaut, Allan Donner, Raphael Saginur, Robert Boruch, Jeremy Grimshaw

Stephanie Dixon

OBJECTIVES: To investigate the extent to which authors of cluster randomised trials adhered to two basic requirements of the World Medical Association's Declaration of Helsinki and the International Committee of Medical Journal Editors' uniform requirements for manuscripts (namely, reporting of research ethics review and informed consent), to determine whether the adequacy of reporting has improved over time, and to identify characteristics of cluster randomised trials associated with reporting of ethics practices. DESIGN: Review of a random sample of published cluster randomised trials from an electronic search in Medline. SETTING: Cluster randomised trials in health research published in English language journals …

Heads Or Tails: Randomized Placebo-Controlled Trials, Charles Weijer

Charles Weijer

No abstract provided.

The Quest For Legitimacy: Comment On Cox Macpherson's 'To Strengthen Consensus, Consult The Stakeholders', Charles Weijer

Charles Weijer

No abstract provided.

Will The Real Charles Fried Please Stand Up?, Paul Miller, Charles Weijer

Charles Weijer

In response to the preceding commentary by Jerry Menikoff in this issue of the Journal, the authors argue that Fried's central concern is not that randomized clinical trials (RCTs) are conducted without consent, but rather that various aspects of the design and conduct of RCTs are in tension with physicians' duties of personal care to their patients. Although Fried does argue that the existence of equipoise cannot justify failure to obtain consent from research subjects, informed consent by itself does not supplant ill subjects' rights to personalized judgment and care embodied in Fried's equipoise.

Rehabilitating Equipoise, Paul Miller, Charles Weijer

Charles Weijer

When may a physician legitimately offer enrollment in a randomized clinical trial (RCT) to her patient? Two answers to this question have had a profound impact on the research ethics literature. Equipoise, as originated by Charles Fried, which we term Fried's equipoise (FE), stipulates that a physician may offer trial enrollment to her patient only when the physician is genuinely uncertain as to the preferred treatment. Clinical equipoise (CE), originated by Benjamin Freedman, requires that there exist a state of honest, professional disagreement in the community of expert practitioners as to the preferred treatment. FE and CE are widely understood …

Placebo Trials And Tribulations, Charles Weijer

Charles Weijer

No abstract provided.

I Need A Placebo Like I Need A Hole In The Head, Charles Weijer

Charles Weijer

No abstract provided.

When Argument Fails, Charles Weijer

Charles Weijer

No abstract provided.

Informing Patients Of Uncertainty In Clinical Trials, S. Halpern, J. Karlawish, Charles Weijer

Charles Weijer

No abstract provided.

Clinical Equipoise And Not The Uncertainty Principle Is The Moral Underpinning Of The Randomised Controlled Trial, Charles Weijer, Stanley Shapiro, Kathleen Glass

Charles Weijer

No abstract provided.

The Future Of Research Into Rotavirus Vaccine, Charles Weijer

Charles Weijer

No abstract provided.

Clinical Equipoise And Rct Design, Charles Weijer

Charles Weijer

This presentation addresses these questions:
• “Upon what ethical grounds may the physician offer RCT enrollment to a patient?”
• Which is the preferred moral basis of the RCT?

Ethical Challenges Of The Randomized Controlled Trial, Charles Weijer

Charles Weijer

No abstract provided.

Why Should We Include Women And Minorities In Randomized Controlled Trials?, Charles Weijer, R. Crouch

Charles Weijer

No abstract provided.

Assessing The Interpretation Of Criteria For Clinical Trial Eligibility: A Survey Of Oncology Investigators, Charles Weijer, Benjamin Freedman, Stanley Shapiro, Abraham Fuks, Myriam Skrutkowska, Maria Sigurjonsdottir

Charles Weijer

OBJECTIVE: To investigate whether eligibility criteria that exclude the elderly, persons with psychiatric disease, and persons with substance abuse problems from participation in randomized controlled trials (RCTs) are subjective and hence a source of variability in enrolment decisions and investigator uncertainty.

DESIGN: Survey questionnaire.

PARTICIPANTS: Cancer investigators from the United States and Canada.

INTERVENTIONS: Investigators were presented with clinical vignettes from 3 patient categories--eligible, ineligible and uncertain--for each of 5 eligibility criteria--3 subjective and 2 objective--and were asked whether they would enrol the patient in a trial and how sure they were of this decision. Demographic characteristics of the investigators …

The Breast Cancer Research Scandal: Addressing The Issues, Charles Weijer

Charles Weijer

The three claims put forward by Dr. Roger Poisson to rationalize his enrollment of ineligible subjects in clinical trials do not justify research fraud. None the less, certain lessons for the conduct of clinical research can be learned from the affair: experimental therapies should be made available to technically ineligible subjects when no effective therapy exists for their disease; further research must investigate the possible benefits of clinical-trial participation; broadly based, pragmatic trials must be regarded as the ideal model; and each eligibility criterion in a clinical-trial protocol should be justified.