human research lecture Flashcards
1
Q
What are the two principles that underlie the doctrine of informed consent?
A
The principles of autonomy and beneficence underlie the doctrine of informed consent in healthcare.
2
Q
What is the duty of care for a treating practitioner in regards to informed consent?
A
A treating practitioner has the duty to give the patient enough information, in non-technical language, to allow the patient to understand and to make a meaningful choice among the available treatment options. This includes conveying the diagnosis, the nature and purpose of the proposed procedure or treatment, material risks and consequences of the proposed procedure or treatment, reasonable treatment alternatives/modalities, and the prognosis without treatment/consequences if the patient elects not to have a given treatment or procedure
3
Q
What is the definition of informed consent in pharmacy?
A
Informed consent in pharmacy occurs when a patient is given sufficient information to understand the need, reason, use, side effects, and adverse reactions of a medication and decide whether or not to use a prescribed medication. Pharmacists have the duty to educate and ensure that patients are well-informed partners in their own medical care.
4
Q
What is the percentage of patients who do not use medication as prescribed because they lack information?
A
Approximately 30% to 50% of patients do not use medication as prescribed because they lack information.
5
Q
What is the definition of research?
A
Research is a studious, rigorous inquiry or examination, especially investigation or experimentation aimed at the discovery and interpretation of facts and the revision of accepted theories or laws in the light of new facts, or the practical application of such new or revised theories or laws to better the human condition.
6
Q
What is one of the first principles of ethical clinical research?
A
One of the first principles of ethical clinical research is that ethical studies start with good science.
7
Q
What is biomedical research?
A
Biomedical research is an organized and systematic way to find answers to questions, involving the translation from basic science to human studies through the application of clinical science and knowledge.
8
Q
What is the ultimate goal of biomedical research?
A
The ultimate goal of biomedical research is to prevent, cure, or treat disease and related human conditions and to make the lives of patients better.
9
Q
What are the determinants of the ethical nature of a clinical trial?
A
The determinants of the ethical nature of a clinical trial include whether the research is of social or scientific value, whether there is scientific validity, fair subject selection, favorable risk-benefit ratio, subject to independent (peer) review, proper informed consent, and respect for potential and enrolled subjects (respect of person standard).
10
Q
What is the key question regarding the ethical concerns of clinical research?
A
The fundamental ethical concern raised by clinical research is whether and when it can be acceptable to expose some individuals to risk and burdens for the benefit of others.
11
Q
What are some of the ethical dilemmas posed by clinical research?
A
Clinical research poses many ethical dilemmas from the time of formulation of research hypothesis to the final implementation of the research and its conduct till completion including post research assessment.
12
Q
What is Eugenics?
A
Eugenics is a theory of “racial improvement” that aims to improve humans through selective “planned breeding” by eliminating undesirable characteristics and diseases from populations.
13
Q
What is the basis of Eugenics?
A
Eugenicists believed in the Mendelian “laws of heritance” which claimed that human qualities like intelligence, mental illness, criminal tendencies, poverty, drug use and other social behaviors were inherited in a simple fashion, and that complex diseases and disorders were solely the outcome of genetic inheritance.
14
Q
How did Eugenics justify their methods?
A
Eugenicists believed that methods such as involuntary sterilization, segregation, and social exclusion would rid society of individuals deemed unfit, and used the methods and legitimacy of science to argue for the superiority of white Europeans and the inferiority of non-white people.
15
Q
Who were the targets of Eugenics?
A
Eugenics disproportionately targeted Latinxs, Native Americans, African Americans, poor whites, and people with disabilities during the entirety of the 20th century.
16
Q
What was the American Breeder’s Association?
A
The American Breeder’s Association was created in 1903 to study eugenics and hosted national conferences on eugenics in 1914, 1915, and 1928. Led to the establishment of the Eugenics Record Offices, which tracked families and their genetic traits.
17
Q
How were minorities impacted by Eugenics in the US?
A
Around 20,000 sterilizations occurred in California state mental institutions between 1909 and 1979, with many being forced and performed on minorities. Thirty-three states eventually allowed involuntary sterilization in whomever lawmakers deemed unworthy to procreate.
18
Q
What was the impact of Eugenics on Native Americans?
A
According to a 1976 Government Accountability Office-GAO investigation, between 25 and 50 percent of Native Americans were sterilized between 1970 and 1976, with some sterilizations happening without consent during other surgical procedures such as an appendectomy. In some cases, health care for living children was denied unless their mothers agreed to sterilization.
19
Q
What was the impact of Eugenics on Nazi Germany?
A
Between 1933 and 1945, the Third Reich implemented a campaign of forced sterilization that claimed at least 400,000 victims. They also used medical experiments involving brutalities, tortures, disabling injury, and death in complete disregard of international conventions, the laws and customs of war, and the general principles of criminal law as derived from the criminal laws of all civilized nations.
20
Q
What were the consequences of the Nazi medical experiments?
A
Approximately 200 internees at Dachau were used in experiments involving vacuum chambers that could duplicate the low air pressure and lack of oxygen at altitudes as high as 65,000 feet. About 40% died as a result, some from extended anoxia and others from lungs rupturing from the low pressures in the chamber. These experiments were contrary to “the principles of the law of nations as they result from the usages established among civilized peoples, from the laws of humanity, and from the dictates of public conscience.”
21
Q
What were some of the unethical practices used in human subject research during WWII?
A
Some unethical practices used in human subject research during WWII included the infliction of tremendous pain and suffering on human subjects without attempts to relieve it, treatment of gunshot wounds, burns, traumatic amputations, and chemical and biological agent exposures without the use of anesthesia, forcing prisoners to drink poisoned water and breathe noxious gases, and some were even shot with cyanide-tipped bullets or given cyanide capsules.
22
Q
What was the typical mortality rate among research subjects during WWII?
A
The typical mortality rate among research subjects during WWII was 25% or more.
23
Q
What were some of the groups targeted for elimination during WWII?
A
Some of the groups targeted for elimination during WWII included the Jewish people, the Sinti and Roma, individuals with disabilities, and LGBTQ+ people.
24
Q
What was the Nuremberg Doctors Trial of 1946?
A
The Nuremberg Doctors Trial of 1946, also known as the “Trial of the Century” or the “Nazi Doctors Trial,” was an American military tribunal in the case of the USA vs. Karl Brandt et. al. The 23 defendants, including 20 physicians, were charged with murder, torture, and other atrocities committed in the name of medical science.
25
What was the outcome of the Nuremberg Doctors Trial?
Fifteen of the 23 defendants were found guilty, all but three of whom were physicians. Seven were sentenced to death, and some of the doctors eluded prosecution.
26
What was the Nuremberg Code?
The Nuremberg Code was a set of standards developed as a result of the Nuremberg Doctors Trial. It served as an ethical yardstick by which the defendants had been measured and guilt determined.
27
When was the Nuremberg Code developed, and what is its significance?
The Nuremberg Code was developed in August 1947, more than 50 years ago, by American judges sitting in judgment of Nazi doctors accused of conducting murderous and torturous human experiments. It is the most important document in the history of the ethics of medical research and served as a blueprint for today's principles that ensure the rights of subjects in medical research.
28
What was the Mississippi Prison Experiment?
A research experiment conducted in 1915 in which prisoners were forced to work in inhumane conditions and exposed to dangerous levels of heat, which led to multiple deaths.
29
What was the Monster Experiment?
A research experiment conducted in 1939 in which children were subjected to negative feedback and made to believe they had speech impediments, which caused psychological harm.
30
What were the Refrigeration Experiments on Mental Patients?
A series of experiments conducted in the 1940s in which mental patients were exposed to freezing temperatures to study the effects of hypothermia.
31
What was the Willowbrook Hepatitis Study?
A study conducted from 1955 to 1970 in which mentally disabled children were deliberately infected with hepatitis, causing harm and controversy.
32
What was the Brooklyn Jewish Chronic Disease Hospital Cancer Study?
A study conducted in 1962 in which patients with cancer were injected with live cancer cells without their knowledge or consent.
33
What was the Stanford Prison Experiment?
A study conducted in 1971 in which college students were assigned to play the roles of prisoners and guards in a simulated prison environment, leading to psychological harm and ethical concerns.
34
What were the U.S. Government Sponsored Radiation Experiments?
A series of experiments conducted between 1944 and 1972 in which human subjects, including children, prisoners, and minorities, were exposed to dangerous levels of radiation
35
What was the Henrietta Lacks case?
A case in which cells taken from a cancer patient without her knowledge or consent were used for scientific research and commercial gain, raising ethical questions about informed consent and compensation for research subjects.
36
Do individuals hold property rights to their donated tissue and cells
No, courts have held that individuals do not hold property rights to their donated tissue and cells.
37
What must subjects be informed of in the consent process for research using their biospecimens?
Subjects must be informed of the possibility that research using their biospecimens could lead to the development of commercial products, and whether the subjects will share in any commercial profits must be made clear in the consent process.
38
What was the Tuskegee Syphilis Study?
the Tuskegee Syphilis Study was a forty-year clinical study conducted by the U.S. Public Health Service to research the natural progression of untreated syphilis in rural, poor African-American men in Alabama.
39
When did the Tuskegee Syphilis Study begin and end?
The study began in 1932 and continued until it was terminated on November 16, 1972.
40
How many men were enrolled in the Tuskegee Syphilis Study?
A total of 600 African American sharecroppers from Macon County, Alabama, were enrolled in the study.
41
How many of the men enrolled in the study had syphilis?
Of the 600 men enrolled in the study, 399 had previously contracted syphilis before the study began, and 201 did not have the disease
42
What did the men receive for participating in the study?
The men received free medical care, meals, and free burial insurance for participating in the study.
43
What was the study's end-point after it was modified?
The study was modified into a "death as end-point study" after six months.
44
Why was the Tuskegee Syphilis Study controversial?
the study was controversial for ethical breaches because researchers knowingly failed to treat patients appropriately after validation of penicillin as an effective cure for the disease they were studying. Scientist also prevented participants from accessing syphilis treatment programs available to other residents in the area.
45
How were the men deceived into thinking they were receiving treatment?
The men were given only vitamins and aspirins to deceive them into thinking they were receiving treatments. They were told they were being treated for "bad blood," which was a local term for various illnesses that include syphilis, anemia, and fatigue.
46
Who leaked the revelations of the study failures to the press?
A whistleblower leaked the revelations of the study failures to the press.
47
What were the consequences of the Tuskegee Syphilis Study?
The victims of the study, all African American, included numerous men who died of syphilis, 40 wives who contracted the disease, and 19 children born with congenital syphilis. The study was terminated on November 16, 1972, after revelations of the study failures were leaked to the press.
48
What is the National Research Act and why was it established?
The National Research Act was passed by the U.S. Congress in 1974 in response to public concern about research abuses such as the Tuskegee Study. Its primary task was to establish ethical principles for all research involving humans.
49
What was the task of the National Commission for the Protection of Human Subjects of Biomedical and Behavioral Research?
The National Commission was established under the National Research Act and its primary task was to identify the ethical principles that would guide all research involving humans.
50
What is the Belmont Report and what is its significance?
The Belmont Report was promulgated by the National Commission in 1979 and it serves as the cornerstone of ethical principles for human subject research.
51
What was the Atonement and why did it occur?
The Atonement occurred on May 16, 1997, when President Bill Clinton issued a formal apology for the Tuskegee Study of Untreated Syphilis in the Negro Male, which was the "longest nontherapeutic experiment on human beings" in the history of medicine and public health.
52
What is the National Center for Bioethics in Research and Healthcare and who was its founding director?
The National Center for Bioethics in Research and Healthcare was inaugurated in 1999 at Tuskegee University and Dr. Marian Gray Secundy, one of the nation’s foremost bioethicists, was its founding director serving from 1999 - 2002.
53
What is the Nuremberg Code?
The Nuremberg Code is the first set of 10 principles outlining professional ethics for medical researchers.
54
What are some of the principles outlined in the Nuremberg Code?
Some of the principles outlined in the Nuremberg Code include the need for voluntary consent from research subjects, ensuring that the benefits of the research outweigh any risks, avoiding physical and mental harm, and conducting experiments only with qualified scientists.
55
What is required for a person to be able to give legal consent for human experimentation according to the Nuremberg Code?
According to the Nuremberg Code, a person involved in human experimentation should have legal capacity to give consent and be situated so as to be able to exercise free power of choice without any element of force, fraud, deceit, duress, overreaching, or coercion.
56
What is the duty and responsibility of individuals involved in human experimentation according to the Nuremberg Code?
The Nuremberg Code states that the duty and responsibility for ascertaining the quality of the consent rests upon each individual who initiates, directs or engages in the experiment. It is a personal duty and responsibility that cannot be delegated to another with impunity.
57
Why was the Nuremberg Code developed?
The Nuremberg Code was developed in response to the unethical medical experimentation that occurred during World War II, particularly in Nazi concentration camps. The code was designed to provide ethical guidelines for human experimentation and ensure that such experimentation was conducted with respect for human rights and dignity.
58
What is the Declaration of Helsinki?
The Declaration of Helsinki is a set of ethical principles developed by the World Medical Association for use by the medical community to ensure that medical research is justified, conducted in the best interest of patients, and respects their autonomy and rights.
59
What are the three fundamental ethical principles outlined in the Belmont Report?
The three fundamental ethical principles outlined in the Belmont Report are respect for persons, beneficence, and justice. Respect for persons involves protecting the autonomy of all people and treating them with courtesy and respect and allowing for informed consent. Beneficence involves maximizing benefits for the research project and minimizing risks to the research subjects while following the philosophy of "Do no harm." Justice involves ensuring reasonable, non-exploitative, and well-considered procedures are administered fairly, with the fair and equal distribution of costs and benefits to potential research participants.
60
What is the duty of a physician according to the Declaration of Helsinki?
According to the Declaration of Helsinki, it is the duty of a physician to promote and safeguard the health, well-being, and rights of patients, including those who are involved in medical research. The physician's knowledge and conscience are dedicated to the fulfillment of this duty, and they should act in the patient's best interest when providing medical care.
61
What is the role of informed consent in ethical research according to the Declaration of Helsinki?
The Declaration of Helsinki makes informed consent a central requirement for ethical research. It requires that research subjects be provided with information about the nature, duration, and purpose of the research, as well as the potential risks and benefits. Informed consent ensures that participants understand the research and can make an informed decision about whether or not to participate.
62
What is the significance of the Belmont Report in human subjects research?
The Belmont Report outlines the three fundamental ethical principles that are essential for using any human subjects for research. It continues to serve as an essential reference for institutional review boards (IRBs) that review HHS-conducted or supported human subjects research proposals to ensure that the research meets the ethical standards of current Federal regulations.
63
What are the three ethical considerations of the Belmont Report?
The three ethical considerations of the Belmont Report are Autonomy - Informed Consent, Beneficence, and Justice.
64
What are the three components of informed consent?
The three components of informed consent are information, comprehension, and voluntariness.
65
What is the role of peer review in the determination of risk and benefits in research?
Peer review is used to justify the risks involved in research and ensure that the benefits outweigh the risks.
66
Who is responsible for study design in research?
The investigator is responsible for study design in research.
67
Who is responsible for determining whether to participate in research?
The subject is responsible for determining whether to participate in research.
68
What is the "Dual Hat" Problem in research ethics?
The "Dual Hat" Problem refers to situations in which a physician serves as both the primary caregiver and the principle investigator of a research study.
69
What is the role of selection of subjects in the Justice consideration of research ethics?
The selection of subjects is described by the researcher, reviewed during the peer review process, and determined to be equitable in the Justice consideration of research ethics.
70
What is the "Dual Hat" problem?
The "Dual Hat" problem refers to the situation where a physician who is the primary caregiver of a patient also acts as the principal investigator in a clinical trial. This creates a moral tension between the physician's obligation to the patient's well-being and their responsibility to conduct the clinical trial in accordance with institutional expectations, funding sources, and research protocols.
71
How can physicians fulfill their therapeutic obligation while conducting clinical trials?
Physicians can fulfill their therapeutic obligation by not confusing research with care and avoiding the therapeutic misconception. It is crucial to distinguish between the ethics of clinical research and medical practice. Clinical trials are designed to produce generalizable knowledge about treatment efficacy through controlled experimentation, while medical care utilizes current standards of care, drugs, and modalities to provide personalized care for the individual patient.
72
What is informed consent in clinical research?
Informed consent is the voluntary choice of an individual to participate in research based on an accurate understanding of its purposes, procedures, risks, benefits, alternatives, and any other factors that may affect a person's decision to participate. Informed consent is a process, and no investigator may involve a human being as a participant in research unless the investigator has obtained the legally effective informed consent of the participant or their legally authorized representative.
73
What are the eight specific elements required for informed consent in human subject research?
The eight specific elements required for informed consent in human subject research include a detailed description of the research and the subject's participation, identification of experimental procedures, description of reasonably foreseeable risks, description of expected benefits, potentially advantageous alternatives to participation, explanation of confidentiality protections, explanation of compensation for injuries policy, whom to contact with questions about the research and research participant's rights, and explanation that participation is voluntary.
74
What extra protections must be given to vulnerable populations in clinical research?
Vulnerable populations, such as prisoners, children, and pregnant women, must receive extra protections in clinical research. The legal rights of subjects may not be waived, and subjects may not be asked to release or appear to release the investigator, the sponsor, the institution, or its agents from liability for negligence.
75
When were US federal regulations governing the conduct of biomedical research involving human participants published?
The federal regulations governing the conduct of biomedical research involving human participants were published in 1981 by the federal Department of Health and Human Services (DHHS).
76
What is the Common Rule in human subject research?
The Common Rule is a set of regulations adopted by numerous federal agencies to protect human participants in any research protocol that those agencies sponsor.
77
What is the role of an institutional review board (IRB) in human subject research?
Research to which the Common Rule or FDA regulations, or both, apply must be reviewed and approved initially by an institutional review board (IRB) recognized by the federal Office of Human Research Protections (OHRP) within the Office of the Secretary, DHHS.
78
When were revisions to the Common Rule in human subject research published?
The revisions to the Common Rule were published on January 19, 2017, with a general compliance date of January 19, 2019.
79
What is the goal of the revisions to the Common Rule in human subject research?
The goal of the revisions to the Common Rule is to promote subjects’ autonomy so that they can make a more informed decision regarding participation in a research study.
80
What statement should be included in the consent form when research involves the collection of identifiable information or biospecimens?
A statement indicating whether identifiers may be removed and if the deidentified information or biospecimens may or may not be used or shared for future research.
81
What statement should be included in the consent form when research involves the use of biospecimens?
A statement indicating whether biospecimens may be used for commercial profit and if the subject will share in that profit.
82
What statement should be included in the consent form when research involves clinically relevant results?
A statement indicating whether clinical results, including individual research results, will be returned to the subject and if so, under what conditions.
83
What statement should be included in the consent form when research involves whole genome sequencing?
A statement indicating that the research will/will not/might involve whole genome sequencing.
84
What are the central tenets established by the Common Rule to protect the rights and welfare of human subjects involved in research?
Institutional assurances of compliance, Federal Wide Assurance (FWA), IRB registration, mandates role of the IRB and review of research by an IRB, requirement to obtain informed consent of subjects, and codifies special requirements for vulnerable (special) populations.
85
What is the role of the IRB in federally-mandated institutional protection of human subjects?
The IRB is a primary mechanism for federally-mandated institutional protection of human subjects.
86
What are the special populations that require extra protections as required by federal regulation and best practices?
Children, prisoners, pregnant women, mentally disabled persons, or economically or educationally disadvantaged persons are special populations that require extra protections as required by federal regulation and best practices.
87
What are the requirements for approving research involving children?
Research not involving greater than minimal risk may be approved with assent of the child and permission of parents. Research involving greater than minimal risk and the prospect of direct benefit to the child may be approved if the risk is justified by the anticipated benefit, the relation of the benefit to risk is at least as favorable to subjects as the alternative approaches, with assent of the child and permission of parents. Research involving greater than minimal risk and no prospect of direct benefit to the child may be approved if the risk represents a minor risk over minimal risk, the intervention presents experiences that are reasonably similar to those in actual medical, dental, social, psychological, or educational situations, the intervention is likely to yield generalizeable knowledge about the disorder or condition, with assent of the child and permission of parents.
88
What is an institutional review board (IRB)?
An institutional review board (IRB) is any board, committee, or other group formally designated by an institution to review, approve, monitor, and periodically review biomedical and behavioral research involving human subjects.
89
What are the “one-eleven criteria”?
The “one-eleven criteria” are federal regulations that outline the criteria used by IRBs to evaluate risk to research subjects (45 CFR 46.111).
90
What is the primary purpose of IRB review?
The primary purpose of IRB review is to ensure the protection of the rights and welfare of human subjects involved in research.
91
What are the responsibilities of an IRB?
The responsibilities of an IRB include conducting initial and continuing review of study protocols and related documents, reviewing reports of unanticipated problems (UAPs) and adverse events (AEs), and conducting periodic reviews of research to ensure continued protection of the welfare of human subjects and compliance with relevant regulations.
92
What are the two broad standards that IRBs uphold when reviewing research projects involving human subjects?
The two broad standards that IRBs uphold are that subjects are not placed at undue risk and that they give uncoerced, informed consent to their participation (voluntariness).
93
What is the role of IRBs in ensuring research subject safety?
IRBs are responsible for reviewing reports of unanticipated problems (UAPs) and adverse events (AEs) which is an important mechanism for ensuring research subject safety.
94
What is the shared responsibility of principal investigators, other personnel involved in studies with human subjects, and the Institutional Review Board (IRB)?
Human subject protections are a shared responsibility of principal investigators, other personnel involved in studies with human subjects, and the Institutional Review Board (IRB).
95
What is Research Ethics?
Research Ethics is the ethics of the planning, conduct, and reporting of research, which includes the protection of human and animal subjects, collection, use, and interpretation of research data, methods for reporting and reviewing research plans or findings, relationships among researchers with one another, relationships between researchers and those that will be affected by their research, means for responding to misunderstandings, disputes, or misconduct, and options for promoting ethical conduct in research.
96
What are Research Ethics Committees (RECs)?
Research Ethics Committees (RECs) are independent bodies made up of a range of individuals including medical or scientific professionals and non-medical or non-scientific members (e.g. patients or lay members). RECs have a responsibility to ensure the protection of the rights, safety and wellbeing of research participants, as well as assuring the public of that protection. RECs engage in ethical deliberations and decision making, including careful consideration and discussion of research, taking into account the principles and values of research ethics from relevant local and international guidelines. RECs may also monitor studies once they have begun and once they are complete.
97
What is the role of Ethics Committees in clinical trials?
Ethics committees have a responsibility to ensure the protection of the rights, safety and wellbeing of research participants, as well as assuring the public of that protection. RECs may express an opinion on the clinical trial protocol, the suitability of the investigators involved in the trial, the adequacy of facilities, and on the methods and documents to be used to inform trial participants and obtain their informed consent. A trial should only begin when a favorable opinion by an REC has been given.
98
What is a Data and Safety Monitoring Board (DSMB)?
A Data and Safety Monitoring Board (DSMB) or Data Monitoring Committee (DMC) is a committee of clinical research experts, such as physicians and statisticians, and patient advocates who monitor the progress of a clinical trial and review safety and effectiveness data while the trial is ongoing. This committee is independent of the people, organizations, and institutions conducting the clinical trial. They periodically reviews clinical study data, incidental event reporting, and clinical study design to further ensure the ethical conduct of clinical trials and serve to protect the rights and safety of patients enrolled in the trial.
99
What is an Emergency Use Authorization (EUA)?
An Emergency Use Authorization (EUA) is one of several tools the FDA is using to help make certain medical products available quickly. In certain emergencies, the FDA can issue an EUA to provide access to medical products that may potentially be used when there are no adequate, approved, and available options. COVID-19 vaccines were initially rolled out under emergency use authorization, which lets the FDA speed the availability of medical products during the pandemic. The EUA process is different than an FDA approval or clearance. Under an EUA, in an emergency, the FDA makes a product available to the public based on the best available evidence, without waiting for all the medical & clinical evidence that would be needed for FDA approval or clearance.
100
What is the role of a DSMB in clinical trials?
A Data Safety Monitoring Board (DSMB) is usually required to determine safe and effective conduct and to recommend conclusion of the trial when significant benefits or risks have developed or the study is unlikely to be concluded successfully. Risk associated with participation in research must be minimized to the extent practical. A DSMB can recommend that a trial be stopped early because of concerns about participant safety or because the main research question has been answered.
101
What is Compassionate Use?
Compassionate Use, also called "Expanded Access," provides a pathway for patients to gain access to investigational drugs, biologics and medical devices for serious diseases or conditions.
102
What is the purpose of Compassionate Use?
The purpose of Compassionate Use is to allow patients to gain access to investigational drugs and devices that have not yet been approved by the FDA and have not been proven to be safe and effective.
103
What is the eligibility criteria for Compassionate Use?
The FDA regulations specify two groups of people eligible for expanded access: those with life-threatening diseases or conditions and those with serious diseases or conditions that have a substantial impact on day-to-day functioning.
104
What is the condition for patients who seek Compassionate Use?
In most cases, patients who seek Compassionate Use must have exhausted all approved therapies for their condition, and be unable to enroll in a clinical trial.
105
What is the future of medical treatments and devices?
The future of medical treatments and devices includes two approaches: human organs-on-chips and in silico trials.
106
What are human organs-on-chips?
Human organs-on-chips are micro devices lined by living human cells that can mimic the microarchitecture, organ level physiology and functions of human organs, making them ideal for replacing in vivo subjects for some phases of clinical testing.
107
What are in silico trials?
In silico trials are computer simulations that could be used in the development or regulatory evaluation of a medicinal product, device, or intervention.
108
What are the potential benefits of in silico trials?
In silico trials may potentially protect public health, advance personalized treatment, and be executed quickly and for a fraction of the cost of a full-scale animal or human trial.
109
What is the FDA's position on in silico trials?
The FDA has advocated the use of innovative research tools such as in silico trials and is already planning for a future in which more than half of all clinical trial data could come from computer simulations.
110
Who was Ellen Roche and what happened to her during a research study?
Ellen Roche was an employee at the Johns Hopkins Asthma and Allergy Center who enrolled in a research study to analyze the reflexes that protect the lungs during a mild asthma attack. She inhaled a chemical called hexamethonium as part of the study, which made her ill, leading to the failure of her lungs and kidneys, and eventually her death.
111
What was the objective of the research study that Ellen Roche enrolled in?
The research study that Ellen Roche enrolled in aimed to analyze the reflexes that protect the lungs when a mild asthma attack occurs.
112
What were the findings of the U.S. Office for Human Research Protections (OHRP) regarding the Johns Hopkins University asthma study?
The OHRP identified that Johns Hopkins failed to ensure adequate protections for research participants, and found that the investigator and Institutional Review Board (IRB) failed to conduct a thorough scientific review to analyze the effects of inhaling hexamethonium. The study documentation poorly communicated the risks of study participation, and the informed consent form did not properly document the use of hexamethonium, nor did it mention that it was experimental.
113
What was the role of the Institutional Review Board (IRB) in the Johns Hopkins University asthma study?
The IRB is responsible for reviewing research studies involving human subjects to ensure that they comply with ethical and regulatory guidelines. In the Johns Hopkins University asthma study, the IRB failed to properly review the study and communicate the risks of study participation.
114
What is RCR, and what topics does it cover?
RCR stands for Responsible Conduct in Research. It covers topics such as adherence to research protocols, guidelines, and regulations, inclusion of underrepresented groups in research, adherence to Institutional Review Board (IRB) requirements, mentoring, peer review, proper treatment of human and animal subjects, conflict of interest and commitments, dynamics and leadership of research groups, data acquisition, sharing, and ownership, publication practices and responsible authorship, intellectual property rights, patents, and trademarks, data, project, lab management, and communication, civility, harassment, and inappropriate conduct, implicit and explicit bias in the research setting, research reproducibility, authorship and collaborative science, science and social responsibility, differentiating between honest discourse and scientific misconduct, disposition of data that resulted from non-compliance or unethical conduct, whistleblowing, reporting misconduct, and scientific misconduct.
115
What is scientific misconduct?
Scientific misconduct refers to the violation of standard codes of scholarly conduct and ethical behavior in professional scientific research. It includes acts such as falsification, obfuscation, fabrication, suppression, plagiarism, ghost writing, improper authorship, misappropriation, bibliometric inflation, and violation of ethical standards regarding human and animal experimentation. It does not include honest error or honest differences of opinion. Acts of scientific misconduct are intentional and often motivated by financial gain, notoriety, power, or position, coercion, or manipulation. It also includes the destruction of, absence of, or accused person's failure to provide research records accurately documenting the research in question.
116
What is the role of an Institutional Review Board (IRB)?
The role of an Institutional Review Board (IRB) is to ensure the protection of the rights and welfare of human research participants. It reviews research proposals to ensure that they comply with ethical and regulatory requirements and that the risks to participants are minimized while the benefits of the research are maximized. The IRB ensures that the informed consent process is adequate and that the confidentiality of research participants is protected. It also monitors ongoing research to ensure that the conduct of the study remains consistent with the approved protocol and that any adverse events are reported in a timely manner.
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What is whistleblowing, and why is it important in research?
Whistleblowing refers to the act of reporting misconduct or unethical behavior in research to authorities or higher-ups. It is important in research because it can help to prevent further harm to research participants, protect the integrity of research, and promote responsible conduct. Whistleblowing can also help to hold individuals and institutions accountable for their actions and promote a culture of transparency and accountability in research.
