1
WGU IT C502 – Statistical Reasoning in Healthcare
Task 1
Clinical trials are the specific studies that allow for the evaluation of the efficacy of new
medications, procedures or devices. The goal of clinical trials is to determine if the new
treatments are safe and effective before they are released for use on the public. Clinical trials are
typically conducted in three stages with an optional fourth stage that evaluates alternative uses
and dosages. Stage I of clinical trials typically involved 80 to 100 healthy volunteers who are
given a small dose of the new medication. The goal of Phase I is to study the pharmacological
effects of the drug on human subjects. Phase I is considered the safest because it involves an
administering a single small dose. This phase also poses the most risk because it is the first time
a drug is given to humans. Dosages are titrated during Phase I to assist in determining the
therapeutic range. Side effects are also identified and monitored in this phase (LaTour, 2013, pg.
625). In Phase II of clinical trials, the number of participants is increased to between 100 and
200. Participants in Phase II clinical trials usually have the condition the new therapy is intended
to treat. The purpose of Phase II is to determine if the drug is effective while continuing to
evaluate it for safety. It is during this stage of study that new therapies usually fail because the
treatment did not do what it was intended to do. In recent queries, only 18% of research trials
successfully moved from Phase II to Phase III. Phase II studies typically involve control groups,
double-blind methods and close monitoring (LaTour, 2013, pg. 625). Phase III of clinical
research trials are only conducted after evidence of the drug’s safety and effectiveness has been
obtained. Phase III involves administering the medication or therapy to a large group of patients
, 2
in varying clinical settings. The number of participants in Phase III testing can range from
several hundred to several thousand. The purpose of this phase is to gather further evidence on
the drug’s safety, effectiveness, dosing range and side effects. In this phase the drugs overall risk
versus benefit is also evaluated and it is compared to alternative therapies. It is common practice
for new therapies to complete two successful Phase III trials before gaining approval from
regulatory agencies, such as the FDA (LaTour, 2013, pg. 625). Phase IV clinical trials are also
known as post marketing surveillance. This phase involves collecting data on specific patient
populations and optimal usage. Phase IV also looks at longer term usage than was originally
studied in phase II. Side effects of longer use are monitored as are alternative uses for the drug
(LaTour, 2013, pg. 625).
Phase Phase I Phase II Phase III Phase IV
Goal Testing of drug Testing drug to Testing drug to Studies done after
on healthy ensure efficacy measure the drug has been
volunteers to test and safety effectiveness and marketed to gather
for dose range identify side information on the
and safety effects drug's effect on
Final stage before various populations
FDA approval and any side effects
associated with
long-term use
Number of 80-100 100-200 300-3000 General Population
Participants
The four ethical principles in research are beneficence, autonomy, nonmaleficence and
justice. Ethical principles exist in research to protect vulnerable populations from being taken
advantage of or harmed. This includes persons in jail, mentally impaired people, children and
the elderly to name a few. In research, beneficence refers to the fact that researchers should have
the wellbeing of the patient as the primary goal of the trail. This means researchers should act in
, 3
a manner that maximizes benefit to the participate and/or society, while minimizing the risk of
harm to the participant. Beneficence also refers to promoting good. This means that the goal of
any research should be to promote new medications or treatments that can help sick people, and
refuse to promote any drugs that could be harmful (LaTour, 2013, pg. 625). Autonomy refers to
the right for a participant to make decisions for themselves. Even if a person has signed a
consent form, they must understand that they can withdraw the consent at any time, with or
without reason. The individual has the right to determine which activities they will or will not
participate in. Researchers must ensure that participants are free from coercive influence. The
most important factor for protecting autonomy is full disclosure and informed consent.
Participants must be made aware of all potential risks, benefits and alternatives as well as being
provided ample time to ask questions. Nonmaleficence means that no harm should come to
participants because of participating in a research trial (LaTour, 2013, pg. 625). Harm can be
physical, emotional, social or financial. As no research is without risk for harm, it is important
for researchers to properly weigh the potential risks against the benefits. If the risks outweigh
the benefits or the risk for significant harm to the patient is too high, the ethical principal of
nonmaleficence requires that researchers not conduct that study. The principal of justice in
research relates to the selection of participants. Participants should be selected fairly and not
based on vulnerability, privilege or other unrelated factors. The purpose of recruiting
participants should be the scientific goals of the study. Participants accept the risks of research
should be able to enjoy the benefits. Specific populations of people should not be excluded from
the study unless scientific evidence supports doing so (LaTour, 2013, pg. 625).
In 1997, the Food and Drug Administration Modernization Act mandated that a database
for clinical trials be created so that physicians and patients could locate appropriate trials to
WGU IT C502 – Statistical Reasoning in Healthcare
Task 1
Clinical trials are the specific studies that allow for the evaluation of the efficacy of new
medications, procedures or devices. The goal of clinical trials is to determine if the new
treatments are safe and effective before they are released for use on the public. Clinical trials are
typically conducted in three stages with an optional fourth stage that evaluates alternative uses
and dosages. Stage I of clinical trials typically involved 80 to 100 healthy volunteers who are
given a small dose of the new medication. The goal of Phase I is to study the pharmacological
effects of the drug on human subjects. Phase I is considered the safest because it involves an
administering a single small dose. This phase also poses the most risk because it is the first time
a drug is given to humans. Dosages are titrated during Phase I to assist in determining the
therapeutic range. Side effects are also identified and monitored in this phase (LaTour, 2013, pg.
625). In Phase II of clinical trials, the number of participants is increased to between 100 and
200. Participants in Phase II clinical trials usually have the condition the new therapy is intended
to treat. The purpose of Phase II is to determine if the drug is effective while continuing to
evaluate it for safety. It is during this stage of study that new therapies usually fail because the
treatment did not do what it was intended to do. In recent queries, only 18% of research trials
successfully moved from Phase II to Phase III. Phase II studies typically involve control groups,
double-blind methods and close monitoring (LaTour, 2013, pg. 625). Phase III of clinical
research trials are only conducted after evidence of the drug’s safety and effectiveness has been
obtained. Phase III involves administering the medication or therapy to a large group of patients
, 2
in varying clinical settings. The number of participants in Phase III testing can range from
several hundred to several thousand. The purpose of this phase is to gather further evidence on
the drug’s safety, effectiveness, dosing range and side effects. In this phase the drugs overall risk
versus benefit is also evaluated and it is compared to alternative therapies. It is common practice
for new therapies to complete two successful Phase III trials before gaining approval from
regulatory agencies, such as the FDA (LaTour, 2013, pg. 625). Phase IV clinical trials are also
known as post marketing surveillance. This phase involves collecting data on specific patient
populations and optimal usage. Phase IV also looks at longer term usage than was originally
studied in phase II. Side effects of longer use are monitored as are alternative uses for the drug
(LaTour, 2013, pg. 625).
Phase Phase I Phase II Phase III Phase IV
Goal Testing of drug Testing drug to Testing drug to Studies done after
on healthy ensure efficacy measure the drug has been
volunteers to test and safety effectiveness and marketed to gather
for dose range identify side information on the
and safety effects drug's effect on
Final stage before various populations
FDA approval and any side effects
associated with
long-term use
Number of 80-100 100-200 300-3000 General Population
Participants
The four ethical principles in research are beneficence, autonomy, nonmaleficence and
justice. Ethical principles exist in research to protect vulnerable populations from being taken
advantage of or harmed. This includes persons in jail, mentally impaired people, children and
the elderly to name a few. In research, beneficence refers to the fact that researchers should have
the wellbeing of the patient as the primary goal of the trail. This means researchers should act in
, 3
a manner that maximizes benefit to the participate and/or society, while minimizing the risk of
harm to the participant. Beneficence also refers to promoting good. This means that the goal of
any research should be to promote new medications or treatments that can help sick people, and
refuse to promote any drugs that could be harmful (LaTour, 2013, pg. 625). Autonomy refers to
the right for a participant to make decisions for themselves. Even if a person has signed a
consent form, they must understand that they can withdraw the consent at any time, with or
without reason. The individual has the right to determine which activities they will or will not
participate in. Researchers must ensure that participants are free from coercive influence. The
most important factor for protecting autonomy is full disclosure and informed consent.
Participants must be made aware of all potential risks, benefits and alternatives as well as being
provided ample time to ask questions. Nonmaleficence means that no harm should come to
participants because of participating in a research trial (LaTour, 2013, pg. 625). Harm can be
physical, emotional, social or financial. As no research is without risk for harm, it is important
for researchers to properly weigh the potential risks against the benefits. If the risks outweigh
the benefits or the risk for significant harm to the patient is too high, the ethical principal of
nonmaleficence requires that researchers not conduct that study. The principal of justice in
research relates to the selection of participants. Participants should be selected fairly and not
based on vulnerability, privilege or other unrelated factors. The purpose of recruiting
participants should be the scientific goals of the study. Participants accept the risks of research
should be able to enjoy the benefits. Specific populations of people should not be excluded from
the study unless scientific evidence supports doing so (LaTour, 2013, pg. 625).
In 1997, the Food and Drug Administration Modernization Act mandated that a database
for clinical trials be created so that physicians and patients could locate appropriate trials to
