Academic literature on the topic 'Clinical trials – Research'

The reported research was an investigation of attitudes and beliefs associated with participation in screening programmes and clinical trials, carried out by general practitioners. Particular focus was given to cardiovascular risk-reduction. The work comprised two main studies. The preliminary study was entirely exploratory, designed to guage public attitudes towards GP involvement with preventive screening programmes and clinical research; and to identify the range of variables associated with participation in such projects. The subsequent study utilised a more formal approach in which the Behavioural Intention Model was utilised to evaluate the power of influencing factors. Both studies employed self-completion questionnaires, developed from preliminary in-depth interview data. For the first study instrument distribution was effected by personal approach, for the second study postal distribution was employed. In all, 1,037 respondents contributed to the surveys - 442 to the preliminary exploration and 695 to the follow-on study. These represented response rates of approximately 65% and 36% respectively. The main findings were that attitudes towards screening were generally favourable, though there was less conformity in attitudes expressed towards clinical trials. These findings were reflected in reported participatory intentions. No evidence was found of any factors which might pose widespread barr i ers to screen ing part ic i pa t ion, though some potent ia 1 deterren ts were identified for older women. It was also noted that other potential deterrents may have been masked by the 'middle class' bias of the sample. Major deterrents to trial entry were identified as worries about: sideeffects, acquired resistance, discontinuation of current effective medications and lack of adequate information. These all interacted with the 'guinea pig' factor. Response rates and responses associated with medical and non-medical sampling sources were also discussed; and consideration was made of the general utility of the Behavioural Intention Model for research of this kind.

Meta-analysis is now commonly used in medical research. However there are statistical issues relating to the subject that require investigation and some are considered here, from both a methodological and a practical perspective. Each of the fixed effect and the random effects models for meta-analysis are based on certain assumptions and the validity of these is investigated. A formal test of the homogeneity assumption made in the fixed effect model may be performed. Since the test has low power, simulation was used to investigate the power under various conditions. The random effects model incorporates a between-study component of variance into the model. A likelihood based method was used to obtain a confidence interval for this variance and also to provide an interval for the overall treatment effect which takes into account the fact that the between-study variance is estimated, rather than assuming it to be known. In order to obtain confidence intervals for the treatment effect for both the fixed effect and the random effects models, distributional assumptions of normality are usually made. Such assumptions may be checked using q-q plots of the residuals obtained for each trial in the meta-analysis. In both meta-analysis models it is assumed that the weight allocated to each study is known, when in fact it must be estimated from the data. The effect of estimating the weights on the overall treatment effect estimate, its confidence intervals, the between-study variance estimate and the test statistic for homogeneity, is investigated by both analytic and simulation methods. It is shown how meta-analysis methods may be used to analyse multicentre trials of a paired cluster randomised design. Meta-analysis techniques are found to be preferable to previously published methods specifically developed for the analysis of such designs, which produce biased and potentially misleading results when a large treatment effect is present.

Mestrado em Medicina Farmacêutica<br>This report describes several activities and projects developed in the context of a curricular training in a clinical research unit, Centro de Investigação Clínica (CIC), led by Professor Joaquim Ferreira. The CIC is one of the research groups of Instituto de Medicina Molecular (IMM) and it is also a group of the Centro Académico de Medicina de Lisboa (CAML) consortium. The principal area of training was the coordination of clinical trials and observational studies. Additionally, other research activities were conducted during the training such as, pharmacovigilance, monitoring, data entry, medical writing and some language coordination activities in a European observational study about Huntington’s Disease founded by European Huntington’s Disease Network (EHDN). It is mention on the State of the Art the Research & Development Process of a new drug and it is characterised some issues about clinical research in Portugal, including advantages in the establishment and organisation of clinical networks. During the training, with the duration of 10 months (that started on 1st September 2013 and finished on 1st July 2014) I deepened my knowledge in clinical research area, understand the importance of the clinical research units, the importance and the role of the study coordinators and expand my areas of interest. The specific training focused in neurological clinical. I had opportunity to understand the practical and logistical difficulties that a research unit faces during the conduction of clinical studies I consider that this training was a valuable experience of introduction of the practice of clinical research. I finished this training with the motivation and interest in working in the area of coordination and monitoring of studies.<br>Este relatório descreve as actividades e projectos desenvolvidos no âmbito de estágio curricular numa unidade de investigação clínica, o Centro de Investigação Clínica (CIC), liderada pelo Professor Doutor Joaquim Ferreira. O CIC faz parte dos grupos de investigação do Instituto de Medicina Molecular (IMM) inserindo-se na iniciativa do consórcio Centro Académico de Medicina de Lisboa (CAML). A principal área de estágio foi a coordenação de ensaios clínicos e estudos observacionais. Adicionalmente foram abordadas outras actividades durante o estágio, tais como farmacovigilância, monitorização, preenchimento de bases de dados, escrita científica e algumas actividades de coordenação a nível nacional de estudo observacional europeu sobre a doença de Huntington financiado por European Huntington’s Disease Network (EHDN). Refere-se no estado da arte o Processo de Investigação e Desenvolvimento de novos medicamentos e caracteriza-se alguns aspectos da investigação clínica em Portugal incluindo vantagens na organização de redes clínicas de investigação. Ao longo do estágio, com 10 meses de duração (início a 1 Setembro de 2013 e fim a 1 de Julho de 2014) aprofundei o conhecimento na área de investigação clínica, percebi a importância de unidades de investigação clínica, a importância e papel de coordenadores clínicos e expandi as minhas áreas de interesse. O treino específico centrou-se em estudos clínicos na área da neurologia, nomeadamente ensaios de clínicos de fase II e III, e estudos observacionais. Tive ainda oportunidade de compreender a realidade prática e logística da condução de estudos clínicos num centro de investigação. Considero que este estágio foi uma experiência valiosa de introdução á prática de investigação clínica. Desta forma, termino o estágio com motivação e interesse em trabalhar na área de coordenação ou monitorização de estudos.

Thesis (S.M. in Operations Research)--Massachusetts Institute of Technology, Sloan School of Management, Operations Research Center, 2013.<br>This electronic version was submitted by the student author. The certified thesis is available in the Institute Archives and Special Collections.<br>Cataloged from student-submitted PDF version of thesis.<br>Includes bibliographical references (pages 67-71).<br>Since chemotherapy began as a treatment for cancer in the 1940s, cancer drug development has become a multi-billion dollar industry. Combination chemotherapy remains the leading treatment for advanced cancers, and cancer drug research and clinical trials are enormous expenses for pharmaceutical companies and the government. We propose an analytics approach for the analysis and design of clinical trials that can discover drug combinations with significant improvements in survival and toxicity. We first build a comprehensive database of clinical trials. We then use this database to develop statistical models from earlier trials that are capable of predicting the survival and toxicity of new combinations of drugs. Then, using these statistical models, we develop optimization models that select novel treatment regimens that could be tested in clinical trials, based on the totality of data available on existing combinations. We present evidence for advanced gastric and gastroesophageal cancers that the proposed analytics approach a) leads to accurate predictions of survival and toxicity outcomes of clinical trials as long as the drugs used have been seen before in different combinations, b) suggests novel treatment regimens that balance survival and toxicity and take into account the uncertainty in our predictions, and c) outperforms the trials run by the average oncologist to give survival improvements of several months. Ultimately, our analytics approach offers promise for improving life expectancy and quality of life for cancer patients at low cost.<br>by Stephen L. Relyea.<br>S.M.in Operations Research