Eric M. Reiman and Jessica B. S. Langbaum
- Published in print:
- 2009
- Published Online:
- February 2010
- ISBN:
- 9780195328875
- eISBN:
- 9780199864836
- Item type:
- chapter
- Publisher:
- Oxford University Press
- DOI:
- 10.1093/acprof:oso/9780195328875.003.0020
- Subject:
- Neuroscience, Techniques, Development
This chapter describes the emerging roles of brain imaging and other biomarker measurements of Alzheimer's disease (AD) progression and pathology in the evaluation of putative AD-slowing, ...
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This chapter describes the emerging roles of brain imaging and other biomarker measurements of Alzheimer's disease (AD) progression and pathology in the evaluation of putative AD-slowing, risk—reducing, and prevention therapies. It discusses the advantages, disadvantages, and complementary roles of structural magnetic resonance imaging (MRI), fluorodeoxyglucose positron emission tomography (FDG PET), and fibrillar amyloid-β (Aβ) PET in clinical trials of AD patients, patients with mild cognitive impairment (MCI), and cognitively normal people at increased genetic risk for AD. It proposes strategies to optimize these methods’ statistical power, address potentially confounding treatment effects, and develop reasonably likely surrogate endpoints for the rapid and rigorous evaluation of promising pre-symptomatic treatments. Finally, it recommends scientific strategies and new public policies to accelerate the identification of demonstrably effective pre-symptomatic AD treatments without losing a generation.Less
This chapter describes the emerging roles of brain imaging and other biomarker measurements of Alzheimer's disease (AD) progression and pathology in the evaluation of putative AD-slowing, risk—reducing, and prevention therapies. It discusses the advantages, disadvantages, and complementary roles of structural magnetic resonance imaging (MRI), fluorodeoxyglucose positron emission tomography (FDG PET), and fibrillar amyloid-β (Aβ) PET in clinical trials of AD patients, patients with mild cognitive impairment (MCI), and cognitively normal people at increased genetic risk for AD. It proposes strategies to optimize these methods’ statistical power, address potentially confounding treatment effects, and develop reasonably likely surrogate endpoints for the rapid and rigorous evaluation of promising pre-symptomatic treatments. Finally, it recommends scientific strategies and new public policies to accelerate the identification of demonstrably effective pre-symptomatic AD treatments without losing a generation.
Fabrizio Benedetti
- Published in print:
- 2008
- Published Online:
- September 2009
- ISBN:
- 9780199559121
- eISBN:
- 9780191724022
- Item type:
- chapter
- Publisher:
- Oxford University Press
- DOI:
- 10.1093/acprof:oso/9780199559121.003.0010
- Subject:
- Neuroscience, Molecular and Cellular Systems
The recent neurobiological advances in placebo research can stimulate the development of new clinical trial designs for the validation of new treatments, and lead to an uncertainty principle, whereby ...
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The recent neurobiological advances in placebo research can stimulate the development of new clinical trial designs for the validation of new treatments, and lead to an uncertainty principle, whereby it is not possible to understand fully the action of a therapeutic agent. One of the main implications of the recent advances of placebo research is the possibility to induce drug-like effects without drugs, thus opening up the possibility to reduce drug intake. As social stimuli may activate the same biochemical and receptorial pathways onto which drugs act, several cognitive and affective factors can eventually modulate the action of drugs.Less
The recent neurobiological advances in placebo research can stimulate the development of new clinical trial designs for the validation of new treatments, and lead to an uncertainty principle, whereby it is not possible to understand fully the action of a therapeutic agent. One of the main implications of the recent advances of placebo research is the possibility to induce drug-like effects without drugs, thus opening up the possibility to reduce drug intake. As social stimuli may activate the same biochemical and receptorial pathways onto which drugs act, several cognitive and affective factors can eventually modulate the action of drugs.
Lorene M. Nelson, Caroline M. Tanner, Stephen K. Van Den Eeden, and Valerie M. McGuire
- Published in print:
- 2004
- Published Online:
- September 2009
- ISBN:
- 9780195133790
- eISBN:
- 9780199863730
- Item type:
- chapter
- Publisher:
- Oxford University Press
- DOI:
- 10.1093/acprof:oso/9780195133790.003.17
- Subject:
- Public Health and Epidemiology, Public Health, Epidemiology
This chapter provides extensive coverage of concepts related to controlled clinical trials in neurology. It describes the main issues in clinical trial design, conduct, and analysis, by examples from ...
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This chapter provides extensive coverage of concepts related to controlled clinical trials in neurology. It describes the main issues in clinical trial design, conduct, and analysis, by examples from neurological research. It begins with a summary of the objectives of Phase I through Phase IV trials, followed by a discussion of the commonly used study designs for clinical trials (parallel group design, factorial design, crossover design, N-of-one design). It then describes important principles for the conduct of clinical trials, including methods for recruitment and retention of clinical trial subjects, approaches to minimizing bias due to unblinding, and techniques for monitoring compliance and adverse events. It discusses methods of randomization and choice of primary and secondary measures of treatment outcome and summarizes statistical approaches to clinical trial data, including intent-to-treat analyses, explanatory analyses, per protocol analyses, and on-treatment analyses. Methods for subgroup analyses and planning of interim analyses are also discussed. One section of the chapter is devoted to ethics of controlled clinical trials.Less
This chapter provides extensive coverage of concepts related to controlled clinical trials in neurology. It describes the main issues in clinical trial design, conduct, and analysis, by examples from neurological research. It begins with a summary of the objectives of Phase I through Phase IV trials, followed by a discussion of the commonly used study designs for clinical trials (parallel group design, factorial design, crossover design, N-of-one design). It then describes important principles for the conduct of clinical trials, including methods for recruitment and retention of clinical trial subjects, approaches to minimizing bias due to unblinding, and techniques for monitoring compliance and adverse events. It discusses methods of randomization and choice of primary and secondary measures of treatment outcome and summarizes statistical approaches to clinical trial data, including intent-to-treat analyses, explanatory analyses, per protocol analyses, and on-treatment analyses. Methods for subgroup analyses and planning of interim analyses are also discussed. One section of the chapter is devoted to ethics of controlled clinical trials.
Richard Brown and Terrance L Albrecht
- Published in print:
- 2010
- Published Online:
- November 2011
- ISBN:
- 9780199238361
- eISBN:
- 9780191730290
- Item type:
- chapter
- Publisher:
- Oxford University Press
- DOI:
- 10.1093/acprof:oso/9780199238361.003.0020
- Subject:
- Palliative Care, Patient Care and End-of-Life Decision Making, Palliative Medicine Research
Despite recent advances, the five-year survival rates for many cancers remains low, and there is a continued need for research to improve cancer outcomes. Clinical trials represent the gold standard ...
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Despite recent advances, the five-year survival rates for many cancers remains low, and there is a continued need for research to improve cancer outcomes. Clinical trials represent the gold standard approach to providing evidence for advances in treatment. Clinical trials are research studies designed to improve cancer prevention, diagnosis, treatment and survivorship. This research base necessarily involves enroling cancer patients and others (for example, family members for genetic linkage studies, healthy community volunteers to serve as matched controls) into clinical trials. As with any research enterprise, patients and others voluntarily enrol and maintain their status as participants in these trials. Unfortunately, many medical and surgical oncology trials have insufficient accrual rates, a problem that has plagued the implementation of clinical trials for many years. Low accrual severely hinders progress in cancer treatment and prevention. This chapter outlines issues involved in recruitment to clinical trials, describes the ethical principles underlying informed consent and suggests strategies to aid communication between healthcare providers and patients about clinical trials.Less
Despite recent advances, the five-year survival rates for many cancers remains low, and there is a continued need for research to improve cancer outcomes. Clinical trials represent the gold standard approach to providing evidence for advances in treatment. Clinical trials are research studies designed to improve cancer prevention, diagnosis, treatment and survivorship. This research base necessarily involves enroling cancer patients and others (for example, family members for genetic linkage studies, healthy community volunteers to serve as matched controls) into clinical trials. As with any research enterprise, patients and others voluntarily enrol and maintain their status as participants in these trials. Unfortunately, many medical and surgical oncology trials have insufficient accrual rates, a problem that has plagued the implementation of clinical trials for many years. Low accrual severely hinders progress in cancer treatment and prevention. This chapter outlines issues involved in recruitment to clinical trials, describes the ethical principles underlying informed consent and suggests strategies to aid communication between healthcare providers and patients about clinical trials.
Michael I. Bennett
- Published in print:
- 2007
- Published Online:
- November 2011
- ISBN:
- 9780198530251
- eISBN:
- 9780191729980
- Item type:
- chapter
- Publisher:
- Oxford University Press
- DOI:
- 10.1093/acprof:oso/9780198530251.003.0002
- Subject:
- Palliative Care, Palliative Medicine Research, Patient Care and End-of-Life Decision Making
Clinical practice in palliative care is focused primarily on improving the quality of life for people that are facing advanced and incurable disease. As such, clinical trials are used to test and ...
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Clinical practice in palliative care is focused primarily on improving the quality of life for people that are facing advanced and incurable disease. As such, clinical trials are used to test and quantify the benefits and harms of a particular clinical treatment. This chapter discusses the principles of designing clinical trials in palliative care and how to design experimental studies that seek to examine the effects of clinical interventions.Less
Clinical practice in palliative care is focused primarily on improving the quality of life for people that are facing advanced and incurable disease. As such, clinical trials are used to test and quantify the benefits and harms of a particular clinical treatment. This chapter discusses the principles of designing clinical trials in palliative care and how to design experimental studies that seek to examine the effects of clinical interventions.
Alla Digilova, Rebecca Li, Mark Barnes, and Barbara Bierer
- Published in print:
- 2015
- Published Online:
- May 2016
- ISBN:
- 9780231171182
- eISBN:
- 9780231540070
- Item type:
- chapter
- Publisher:
- Columbia University Press
- DOI:
- 10.7312/columbia/9780231171182.003.0009
- Subject:
- Law, Medical Law
In this chapter, the authors review recent regulatory and private efforts focused on broadening access to participant-level data collected as part of clinical trials. The authors consider the risks ...
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In this chapter, the authors review recent regulatory and private efforts focused on broadening access to participant-level data collected as part of clinical trials. The authors consider the risks and benefits of increased data transparency and outline the features of a well-designed data-sharing model that would protect the essential interests of research participants and clinical trial sponsors.Less
In this chapter, the authors review recent regulatory and private efforts focused on broadening access to participant-level data collected as part of clinical trials. The authors consider the risks and benefits of increased data transparency and outline the features of a well-designed data-sharing model that would protect the essential interests of research participants and clinical trial sponsors.
Katherine Wade
- Published in print:
- 2016
- Published Online:
- May 2016
- ISBN:
- 9780719099465
- eISBN:
- 9781526104410
- Item type:
- chapter
- Publisher:
- Manchester University Press
- DOI:
- 10.7228/manchester/9780719099465.003.0012
- Subject:
- Sociology, Culture
This chapter focuses on two key areas which require further clarity and elaboration in the context of research with children, namely, the requirements for research which does not offer the child the ...
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This chapter focuses on two key areas which require further clarity and elaboration in the context of research with children, namely, the requirements for research which does not offer the child the prospect of a direct benefit and the provisions around child assent and parental consent. The chapter examines legal and ethical guidance regarding these issues, setting out areas of uncertainty and ambiguity between the various governing instruments. It argues that the lack of clarity in these areas needs to be addressed in order to provide a robust system of protection for children, parents and those involved in clinical research with children. It makes proposals for reform drawing on the legal, ethical and academic guidance in the area and best practice in other countries.Less
This chapter focuses on two key areas which require further clarity and elaboration in the context of research with children, namely, the requirements for research which does not offer the child the prospect of a direct benefit and the provisions around child assent and parental consent. The chapter examines legal and ethical guidance regarding these issues, setting out areas of uncertainty and ambiguity between the various governing instruments. It argues that the lack of clarity in these areas needs to be addressed in order to provide a robust system of protection for children, parents and those involved in clinical research with children. It makes proposals for reform drawing on the legal, ethical and academic guidance in the area and best practice in other countries.
Fabrizio Benedetti
- Published in print:
- 2008
- Published Online:
- September 2009
- ISBN:
- 9780199559121
- eISBN:
- 9780191724022
- Item type:
- chapter
- Publisher:
- Oxford University Press
- DOI:
- 10.1093/acprof:oso/9780199559121.003.0001
- Subject:
- Neuroscience, Molecular and Cellular Systems
The history of medicine is basically the history of placebos, as most early medical interventions were nothing but placebos, i.e., inert. Over the centuries, doctors started using sham treatments to ...
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The history of medicine is basically the history of placebos, as most early medical interventions were nothing but placebos, i.e., inert. Over the centuries, doctors started using sham treatments to see whether the clinical improvements were attributable to patients' imagination and/or spontaneous remissions. Today placebos are widely used in clinical research to validate the efficacy of a therapy as well as in clinical practice to please and to placate anxious patients. The placebo effect represents a good example of how a mental activity may affect several physiological functions, thus it is an excellent model to study the mind-body interaction. The nocebo effect, which is opposite to the placebo effect, is also a good model to understand the interaction between mind and body.Less
The history of medicine is basically the history of placebos, as most early medical interventions were nothing but placebos, i.e., inert. Over the centuries, doctors started using sham treatments to see whether the clinical improvements were attributable to patients' imagination and/or spontaneous remissions. Today placebos are widely used in clinical research to validate the efficacy of a therapy as well as in clinical practice to please and to placate anxious patients. The placebo effect represents a good example of how a mental activity may affect several physiological functions, thus it is an excellent model to study the mind-body interaction. The nocebo effect, which is opposite to the placebo effect, is also a good model to understand the interaction between mind and body.
Martin Niethammer and Andrew Feigin
- Published in print:
- 2011
- Published Online:
- September 2011
- ISBN:
- 9780195393484
- eISBN:
- 9780199914494
- Item type:
- chapter
- Publisher:
- Oxford University Press
- DOI:
- 10.1093/acprof:oso/9780195393484.003.0119
- Subject:
- Neuroscience, Disorders of the Nervous System, Techniques
This chapter reviews the role of neuroimaging in PD clinical trials to investigate both symptomatic and potential disease-modifying therapies. While clinical ratings are typically used as the primary ...
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This chapter reviews the role of neuroimaging in PD clinical trials to investigate both symptomatic and potential disease-modifying therapies. While clinical ratings are typically used as the primary measures of therapeutic response, these descriptors have inherent limitations. Imaging methods including FDOPA PET and beta-CIT SPECT have been used to provide objective and potentially less variable indicators of treatment outcome. The chapter describes the relevance of imaging biomarkers to trial design, including confirmation of diagnosis prior to randomization and as an adjunctive means for assessing efficacy. This chapter reviews the use of functional brain imaging in prior clinical trials of PD and discusses novel applications of these tools in the evaluation of new therapies for the disorder.Less
This chapter reviews the role of neuroimaging in PD clinical trials to investigate both symptomatic and potential disease-modifying therapies. While clinical ratings are typically used as the primary measures of therapeutic response, these descriptors have inherent limitations. Imaging methods including FDOPA PET and beta-CIT SPECT have been used to provide objective and potentially less variable indicators of treatment outcome. The chapter describes the relevance of imaging biomarkers to trial design, including confirmation of diagnosis prior to randomization and as an adjunctive means for assessing efficacy. This chapter reviews the use of functional brain imaging in prior clinical trials of PD and discusses novel applications of these tools in the evaluation of new therapies for the disorder.
Curtis L. Meinert
- Published in print:
- 2011
- Published Online:
- September 2011
- ISBN:
- 9780199742967
- eISBN:
- 9780199897278
- Item type:
- chapter
- Publisher:
- Oxford University Press
- DOI:
- 10.1093/acprof:oso/9780199742967.003.0020
- Subject:
- Public Health and Epidemiology, Epidemiology, Public Health
This chapter presents guidelines for persons in search of a clinical trial to enroll in. By and large, one should be shopping for randomized trials because two things are certain: firstly, that there ...
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This chapter presents guidelines for persons in search of a clinical trial to enroll in. By and large, one should be shopping for randomized trials because two things are certain: firstly, that there was a series of trials prior to the one of interest showing the treatment to be reasonably safe, and secondly, that there is an underlying state of clinical equipoise. The first step when shopping for a trial is to go to www.clinicaltrials.gov. The site is a registry of 97,000 + trials (as of October 14, 2010) in different stages, ranging from still-being-planned ones to completed ones, and everything in between. Chances are that some relevant trials will be found and that they are open for enrollment. One can also surf the web for ongoing trials listed on the websites of major drug companies.Less
This chapter presents guidelines for persons in search of a clinical trial to enroll in. By and large, one should be shopping for randomized trials because two things are certain: firstly, that there was a series of trials prior to the one of interest showing the treatment to be reasonably safe, and secondly, that there is an underlying state of clinical equipoise. The first step when shopping for a trial is to go to www.clinicaltrials.gov. The site is a registry of 97,000 + trials (as of October 14, 2010) in different stages, ranging from still-being-planned ones to completed ones, and everything in between. Chances are that some relevant trials will be found and that they are open for enrollment. One can also surf the web for ongoing trials listed on the websites of major drug companies.
Claire Snowdon, Diana Elbourne, and Jo Garcia
- Published in print:
- 2009
- Published Online:
- September 2009
- ISBN:
- 9780199231461
- eISBN:
- 9780191723858
- Item type:
- chapter
- Publisher:
- Oxford University Press
- DOI:
- 10.1093/acprof:oso/9780199231461.003.0004
- Subject:
- Public Health and Epidemiology, Public Health, Epidemiology
This chapter examines the decisions of parents of critically ill babies who had not consented to take part in research. Their decisions were based on a misunderstanding of the information they had ...
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This chapter examines the decisions of parents of critically ill babies who had not consented to take part in research. Their decisions were based on a misunderstanding of the information they had been given. It is suggested that patients may be overburdened with information and perhaps in this kind of case better decisions could be facilitated by providing less information rather than more.Less
This chapter examines the decisions of parents of critically ill babies who had not consented to take part in research. Their decisions were based on a misunderstanding of the information they had been given. It is suggested that patients may be overburdened with information and perhaps in this kind of case better decisions could be facilitated by providing less information rather than more.
Yolanda Zuriarrain Reyna, Mchael I. Bennett, and Eduardo Bruera
- Published in print:
- 2007
- Published Online:
- November 2011
- ISBN:
- 9780198530251
- eISBN:
- 9780191729980
- Item type:
- chapter
- Publisher:
- Oxford University Press
- DOI:
- 10.1093/acprof:oso/9780198530251.003.0003
- Subject:
- Palliative Care, Palliative Medicine Research, Patient Care and End-of-Life Decision Making
This chapter discusses certain ethical and practical issues involved in designing and conducting clinical trials in palliative care. It also highlights several ethical, administrative, and ...
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This chapter discusses certain ethical and practical issues involved in designing and conducting clinical trials in palliative care. It also highlights several ethical, administrative, and methodological issues that need to be addressed, and their corresponding solutions. Some of the basic ethical principles that are included in this chapter are autonomy, beneficence, and justice.Less
This chapter discusses certain ethical and practical issues involved in designing and conducting clinical trials in palliative care. It also highlights several ethical, administrative, and methodological issues that need to be addressed, and their corresponding solutions. Some of the basic ethical principles that are included in this chapter are autonomy, beneficence, and justice.
William A. Silverman
- Published in print:
- 1999
- Published Online:
- September 2009
- ISBN:
- 9780192630889
- eISBN:
- 9780191723568
- Item type:
- chapter
- Publisher:
- Oxford University Press
- DOI:
- 10.1093/acprof:oso/9780192630889.003.0013
- Subject:
- Public Health and Epidemiology, Public Health, Epidemiology
This chapter presents a 1990 commentary on two clinical trials conducted in Kuala Lumpur and the US. A comparison of the trials provides supporting evidence for the claim that there has been ...
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This chapter presents a 1990 commentary on two clinical trials conducted in Kuala Lumpur and the US. A comparison of the trials provides supporting evidence for the claim that there has been considerable improvement in safeguards to protect the rights of patients enrolled in such therapeutic experiments. It further says that doctors have a ‘study design obligation’ in the conduct of trials.Less
This chapter presents a 1990 commentary on two clinical trials conducted in Kuala Lumpur and the US. A comparison of the trials provides supporting evidence for the claim that there has been considerable improvement in safeguards to protect the rights of patients enrolled in such therapeutic experiments. It further says that doctors have a ‘study design obligation’ in the conduct of trials.
Peter Keating and Alberto Cambrosio
- Published in print:
- 2012
- Published Online:
- September 2013
- ISBN:
- 9780226428918
- eISBN:
- 9780226428932
- Item type:
- book
- Publisher:
- University of Chicago Press
- DOI:
- 10.7208/chicago/9780226428932.001.0001
- Subject:
- History, History of Science, Technology, and Medicine
Until the early 1960s, cancer treatment consisted primarily of surgery and radiation therapy. Most practitioners then viewed the treatment of terminally ill cancer patients with heroic courses of ...
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Until the early 1960s, cancer treatment consisted primarily of surgery and radiation therapy. Most practitioners then viewed the treatment of terminally ill cancer patients with heroic courses of chemotherapy as highly questionable. The randomized clinical trials that today sustain modern oncology were relatively rare and prompted stiff opposition from physicians loath to assign patients randomly to competing treatments. And yet today these trials form the basis of medical oncology. How did such a spectacular change occur? How did medical oncology pivot from a nonentity and, in some regards, a reviled practice to the central position it now occupies in modern medicine? This book explores how practitioners established a new style of practice, at the center of which lies the cancer clinical trial. Far from mere testing devices, these trials have become full-fledged experiments that have redefined the practices of clinicians, statisticians, and biologists. The authors investigate these trials and how they have changed since the 1960s, all the while demonstrating their significant impact on the progression of oncology.Less
Until the early 1960s, cancer treatment consisted primarily of surgery and radiation therapy. Most practitioners then viewed the treatment of terminally ill cancer patients with heroic courses of chemotherapy as highly questionable. The randomized clinical trials that today sustain modern oncology were relatively rare and prompted stiff opposition from physicians loath to assign patients randomly to competing treatments. And yet today these trials form the basis of medical oncology. How did such a spectacular change occur? How did medical oncology pivot from a nonentity and, in some regards, a reviled practice to the central position it now occupies in modern medicine? This book explores how practitioners established a new style of practice, at the center of which lies the cancer clinical trial. Far from mere testing devices, these trials have become full-fledged experiments that have redefined the practices of clinicians, statisticians, and biologists. The authors investigate these trials and how they have changed since the 1960s, all the while demonstrating their significant impact on the progression of oncology.
Annette Rid and Annick Antierens
- Published in print:
- 2017
- Published Online:
- February 2017
- ISBN:
- 9780190624477
- eISBN:
- 9780190624507
- Item type:
- chapter
- Publisher:
- Oxford University Press
- DOI:
- 10.1093/acprof:oso/9780190624477.003.0008
- Subject:
- Public Health and Epidemiology, Public Health, Epidemiology
In 2014, the world began to witness an unprecedented Ebola epidemic in West Africa that is now smoldering. Ebola had a fatality rate of 40% to 60% in this epidemic due to a lack of adequate health ...
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In 2014, the world began to witness an unprecedented Ebola epidemic in West Africa that is now smoldering. Ebola had a fatality rate of 40% to 60% in this epidemic due to a lack of adequate health infrastructure and overwhelmed aid organizations at the height of the outbreak. These grim prospects quickly prompted proposals to use experimental treatments and vaccines for Ebola that were in the earliest phases of development at the time. Doctors Without Borders/Médecins sans Frontières (MSF) played a key role in the rapid launch of clinical trials of the available experimental interventions. This chapter traces how MSF negotiated the trials under challenging circumstances, focusing on trials of experimental treatments. It provides a detailed account of the difficult tradeoffs MSF faced and shows how the organization’s values and beliefs shaped key decisions about research priority setting and clinical trial design.Less
In 2014, the world began to witness an unprecedented Ebola epidemic in West Africa that is now smoldering. Ebola had a fatality rate of 40% to 60% in this epidemic due to a lack of adequate health infrastructure and overwhelmed aid organizations at the height of the outbreak. These grim prospects quickly prompted proposals to use experimental treatments and vaccines for Ebola that were in the earliest phases of development at the time. Doctors Without Borders/Médecins sans Frontières (MSF) played a key role in the rapid launch of clinical trials of the available experimental interventions. This chapter traces how MSF negotiated the trials under challenging circumstances, focusing on trials of experimental treatments. It provides a detailed account of the difficult tradeoffs MSF faced and shows how the organization’s values and beliefs shaped key decisions about research priority setting and clinical trial design.
Michael Kinch
- Published in print:
- 2016
- Published Online:
- May 2017
- ISBN:
- 9781469630625
- eISBN:
- 9781469630649
- Item type:
- chapter
- Publisher:
- University of North Carolina Press
- DOI:
- 10.5149/northcarolina/9781469630625.003.0003
- Subject:
- Business and Management, Innovation
The testing of new medicines is a complex, labor- and cost-intensive practice that has evolved over the past 200 years. This chapter explains some of the jargon used by medical practioners and ...
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The testing of new medicines is a complex, labor- and cost-intensive practice that has evolved over the past 200 years. This chapter explains some of the jargon used by medical practioners and industry scientists to explain how the cure for scurvy by a promising young British Navy captain gave rise to the naming of Royal Navy sailors as “limeys.” More than a century later, the Nazi atrocities of the Second World War likewise contributed to the rise of modern ethical practices and examples of how these have been implemented include the approval of breakthrough cancer medicines. In doing so, we discuss the different “phases” of clinical trials and relate the lessons learned from a tragic 2006 clinical trial in London that caused one volunteer's head to swell so large that he was referred to as “the Elephant Man.” By understanding how the size and intensity of clinical trials has grown over time, one begins to appreciate the parallel escalation of drug costs. Finally, we discuss the thalidomide crisis and how the bravery and persistance of a new female FDA employee overrode a multi-national conglomerate to prevent America from falling victim to the severe birth defects experienced by many other countries.Less
The testing of new medicines is a complex, labor- and cost-intensive practice that has evolved over the past 200 years. This chapter explains some of the jargon used by medical practioners and industry scientists to explain how the cure for scurvy by a promising young British Navy captain gave rise to the naming of Royal Navy sailors as “limeys.” More than a century later, the Nazi atrocities of the Second World War likewise contributed to the rise of modern ethical practices and examples of how these have been implemented include the approval of breakthrough cancer medicines. In doing so, we discuss the different “phases” of clinical trials and relate the lessons learned from a tragic 2006 clinical trial in London that caused one volunteer's head to swell so large that he was referred to as “the Elephant Man.” By understanding how the size and intensity of clinical trials has grown over time, one begins to appreciate the parallel escalation of drug costs. Finally, we discuss the thalidomide crisis and how the bravery and persistance of a new female FDA employee overrode a multi-national conglomerate to prevent America from falling victim to the severe birth defects experienced by many other countries.
Curtis L. Meinert and Susan Tonascia
- Published in print:
- 1986
- Published Online:
- September 2009
- ISBN:
- 9780195035681
- eISBN:
- 9780199864478
- Item type:
- chapter
- Publisher:
- Oxford University Press
- DOI:
- 10.1093/acprof:oso/9780195035681.003.0001
- Subject:
- Public Health and Epidemiology, Public Health, Epidemiology
This introduction chapter begins with a brief discussion of the definition of a clinical trial. It then presents a historical sketch of clinical trials followed by a terminology of conventions. The ...
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This introduction chapter begins with a brief discussion of the definition of a clinical trial. It then presents a historical sketch of clinical trials followed by a terminology of conventions. The chapter then defines the class of trials considered in this book.Less
This introduction chapter begins with a brief discussion of the definition of a clinical trial. It then presents a historical sketch of clinical trials followed by a terminology of conventions. The chapter then defines the class of trials considered in this book.
Barrie M. Margetts and Ian L. Rouse
- Published in print:
- 1997
- Published Online:
- September 2009
- ISBN:
- 9780192627391
- eISBN:
- 9780191723704
- Item type:
- chapter
- Publisher:
- Oxford University Press
- DOI:
- 10.1093/acprof:oso/9780192627391.003.0016
- Subject:
- Public Health and Epidemiology, Public Health, Epidemiology
Experimental studies set out to alter, rather than just observe, relevant exposures of interest, and to assess over time their impact on health status or disease outcomes. This chapter begins with an ...
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Experimental studies set out to alter, rather than just observe, relevant exposures of interest, and to assess over time their impact on health status or disease outcomes. This chapter begins with an historical perspective, and goes on to define experimental studies with tables illustrating the differences between the types of study. This is followed by an outline of the practical approaches to study design, including selection of study populations, allocation of treatment regimes, length of follow up, observer and participant effects, compliance, ascertainment of exposure and outcome, and sample size and power. The chapter concludes with a consideration of analysis and interpretation, some useful variations in design (e.g., factorial designs), and the role of systematic review and meta-analysis.Less
Experimental studies set out to alter, rather than just observe, relevant exposures of interest, and to assess over time their impact on health status or disease outcomes. This chapter begins with an historical perspective, and goes on to define experimental studies with tables illustrating the differences between the types of study. This is followed by an outline of the practical approaches to study design, including selection of study populations, allocation of treatment regimes, length of follow up, observer and participant effects, compliance, ascertainment of exposure and outcome, and sample size and power. The chapter concludes with a consideration of analysis and interpretation, some useful variations in design (e.g., factorial designs), and the role of systematic review and meta-analysis.
- Published in print:
- 2009
- Published Online:
- March 2013
- ISBN:
- 9780226465319
- eISBN:
- 9780226465333
- Item type:
- chapter
- Publisher:
- University of Chicago Press
- DOI:
- 10.7208/chicago/9780226465333.003.0002
- Subject:
- History, History of Science, Technology, and Medicine
This chapter investigates work in the mid-1970s of a distinguished oncology researcher, Bernard Fisher, in order to bring out a number of salient characteristics of clinical trial culture. It ...
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This chapter investigates work in the mid-1970s of a distinguished oncology researcher, Bernard Fisher, in order to bring out a number of salient characteristics of clinical trial culture. It specifically addresses Fisher's multicenter trials that were governed by a tightly regimented system of practices that were capable of producing highly reliable results. It follows the growth of cancer clinical trials from the end of the Second World War until the early 1970s and President Richard Nixon's War on Cancer. The principle that adding prolonged, and aggressive, chemotherapy to conventional local treatments with surgery and radiotherapy had improved cancer cures was proved. With the growing postwar concerns with clinical conduct, medical investigators also had to show that their research was produced ethically.Less
This chapter investigates work in the mid-1970s of a distinguished oncology researcher, Bernard Fisher, in order to bring out a number of salient characteristics of clinical trial culture. It specifically addresses Fisher's multicenter trials that were governed by a tightly regimented system of practices that were capable of producing highly reliable results. It follows the growth of cancer clinical trials from the end of the Second World War until the early 1970s and President Richard Nixon's War on Cancer. The principle that adding prolonged, and aggressive, chemotherapy to conventional local treatments with surgery and radiotherapy had improved cancer cures was proved. With the growing postwar concerns with clinical conduct, medical investigators also had to show that their research was produced ethically.
William A. Silverman
- Published in print:
- 1999
- Published Online:
- September 2009
- ISBN:
- 9780192630889
- eISBN:
- 9780191723568
- Item type:
- chapter
- Publisher:
- Oxford University Press
- DOI:
- 10.1093/acprof:oso/9780192630889.003.0009
- Subject:
- Public Health and Epidemiology, Public Health, Epidemiology
This chapter presents a 1989 commentary on randomized clinical trials. It argues that given the uncertainties about the wide extrapolation of results after any and all formal clinical studies, it ...
More
This chapter presents a 1989 commentary on randomized clinical trials. It argues that given the uncertainties about the wide extrapolation of results after any and all formal clinical studies, it makes sense to consider a graded approach to the release of a promising approach for everyday use. Instead of country-wide release, in a step-wise plan new treatment would be made available in only one city or other specific region in order to closely monitor the initial field experience with unselected patients.Less
This chapter presents a 1989 commentary on randomized clinical trials. It argues that given the uncertainties about the wide extrapolation of results after any and all formal clinical studies, it makes sense to consider a graded approach to the release of a promising approach for everyday use. Instead of country-wide release, in a step-wise plan new treatment would be made available in only one city or other specific region in order to closely monitor the initial field experience with unselected patients.
