Research Methodology and Biostatistics syllabus: Medical Research: History, values in medical ethics, autonomy, beneficence, non-maleficence, double effect, conflicts between autonomy. Medical research has a long and varied history. It has evolved from rudimentary practices to sophisticated, evidence-based methodologies. Some key milestones include the development of the scientific method, the use of randomized controlled trials, the discovery of antibiotics, and the mapping of the human genome. Ethical concerns have also played a significant role in shaping the history of medical research, especially in response to various ethical violations, such as the Tuskegee Syphilis Study and the Nuremberg Trials. Resolving conflicts between these principles often requires careful consideration, ethical analysis, and, in some cases, consultation with ethics committees or boards. The specific course of action may vary based on the individual circumstances and ethical frameworks employed by healthcare professionals and researchers. Ethical guidelines and regulations also play a significant role in addressing and preventing these conflicts in medical research.

1. MEDICAL RESEARCH: “RESEARCH
METHODOLOGY & BIOSTATISTICS”
PRESENTED & WRITTEN BY: *PRACHI PANDEY, RAHUL PAL
M. PHARM (PHARMACEUTICS), IIIRD SEM.
DEPARTMENT OF PHARMACEUTICS, NIMS INSTITUTE OF PHARMACY, NIMS UNIVERSITY, JAIPUR,
RAJASTHAN, 303121, INDIA.

2. INTRODUCTION
 Medical research in clinical settings is the study of human health
and disease in people. It is the primary way that researchers
determine if a new form of treatment or prevention, such as a new
drug, diet, or medical device, is safe and effective in people.
 A clinical trial is designed to learn if a new treatment is more
effective or has less harmful side effects than existing treatments.
 Clinical trail is basically have 4 phases. Phase I, Phase II, Phase
III, Phase IV

3. PHASES OF CLINICAL TRAILS

4. HISTORY
 The most important figures in the history of medical research is Louis
Pasteur. Pasteur was is known as medical microbiologist. A French
chemist and microbiologist who made significant contributions to the
understanding of infectious diseases. In the 1860s, Pasteur showed that
germs caused disease and that they could be killed by heat.
 This discovery led to the development of pasteurization, a process of
heating food to kill harmful bacteria.

5. HISTORY
 The history of medical research is Robert Koch. Koch is also
known as Father of modern microbiologist. He was a German
physician and microbiologist who is credited with discovering
the bacteria that cause tuberculosis and cholera. Koch's work
led to the development of new treatments and preventive
measures for these diseases.
 He was awarded the Nobel Prize in Medicine in 1905. He is
one of the founders of microbiologist
Robert Koch
(1843- 1910)

6. HISTORY
 In the 20th century, medical research accelerated
rapidly. This was due to a number of factors, including
the development of new technologies, such as the
electron microscope and the DNA sequencing machine.
 These technologies allowed scientists to learn more
about the human body and disease than ever before.

7. VALUE IN MEDICAL ETHICS
 Medical ethics in medical research is a complex and evolving field. It is concerned with the ethical
implications of research involving human subjects. The goal of medical ethics is to ensure that the rights
and well-being of research participants are protected, while still allowing for valuable research to be
conducted.
 There are a number of ethical principles that guide medical research involving human subjects. These
principles include:
 Autonomy
 Beneficence
 Non-maleficence

9. AUTONOMY
 Autonomy in medical research is the principle that research
participants should have the right to make their own decisions
about whether or not to participate in research, and they should
be provided with all of the relevant information about the
research before they make a decision.
 This principle is important because it respects the right of
individuals to make their own choices about their bodies and
their health. It also helps to ensure that research participants are
fully informed about the risks and benefits of the research, and
that they are not coerced into participating. Inform Concent form

10. HOW TO APPLY AUTONOMY
 Informed consent: Research participants must be given all of the relevant information about the research
before they consent to participate. This information should include the purpose of the research, the methods that
will be used, the risks and benefits, and the alternatives to participation. For example, in a clinical trial for a new
drug, participants must be informed of the potential side effects of the drug, as well as the risks of taking a
placebo.
 Right to refuse: Research participants have the right to refuse to participate in research at any time, without
giving a reason. Researchers should not try to pressure or coerce participants into participating in research.
 Respect for participant preferences: Researchers should respect the preferences of research participants. For
example, participants may have preferences about the type of research they are willing to participate in, or the
procedures that they are willing to undergo. Researchers should also respect the participants' right to withdraw
from research at any time.

11. EXAMPLES OF AUTONOMY IN MEDICAL
RESEARCH
 A patient is diagnosed with a terminal illness. The patient is offered the opportunity to participate in a clinical trial
of a new drug that is being developed to treat the illness. The patient is given all of the information they need about
the clinical trial, including the potential risks and benefits. The patient decides to participate in the clinical trial, but
later changes their mind and wants to withdraw. The researchers must respect the patient's decision and allow them
to withdraw from the clinical trial at any time.
 A pregnant woman is offered the opportunity to participate in a clinical trial of a new drug to prevent a rare genetic
disease. The drug has not been tested in pregnant women before, and there is a risk that it could harm the fetus. The
woman is given all of the information she needs about the clinical trial, including the potential risks and benefits.
The woman decides to participate in the clinical trial, but she also wants to have the option to terminate her
pregnancy if she decides that the risks are too high.

12. BENEFICENCE
 Beneficence in medical research is the ethical principle of promoting
the welfare of research participants and maximizing the potential
benefits of research while minimizing any potential harm. It is one of
the four core ethical principles of research ethics, along with respect for
persons, justice, and nonmaleficence.
 The principle of beneficence is based on the belief that we have a moral
obligation to help others and to do what is best for them. In the context
of medical research, this means that researchers have a responsibility to
design and conduct research studies in a way that minimizes the risks to
participants and maximizes the potential benefits.
Data safety monitoring
boards (DSMBs)

13. HOW TO APPLY BENEFICENCE
 Careful design of research studies: Researchers should carefully design their studies to minimize the risks to
participants and maximize the potential benefits. This includes carefully considering the study population, the
interventions being studied, and the methods being used.
 Informed consent: Research participants must be fully informed of the risks and benefits of participating in a study
before they can give their consent. Researchers have a responsibility to provide participants with all of the
information they need to make an informed decision about whether or not to participate.
 Continuous monitoring of participants: Researchers should continuously monitor participants during the study to
identify any potential harms. If any harms are identified, researchers should take steps to mitigate them.
 Data safety monitoring boards (DSMBs): DSMBs are independent groups of experts who review data from clinical
trials to assess the safety and efficacy of the interventions being studied. DSMBs can recommend that a trial be
stopped early if the risks to participants outweigh the potential benefits.

14. EXAMPLES OF BENEFICENCE IN MEDICAL
RESEARCH
 Developing new vaccines and treatments for diseases: This is one of the most important and direct ways
in which beneficence is promoted in medical research. By developing new vaccines and treatments,
researchers can help to prevent and cure diseases, thereby improving the health and well-being of
individuals and society as a whole.
 Conducting clinical trials to ensure the safety and efficacy of new medical interventions: Before a new
medical intervention can be widely used, it must be thoroughly tested to ensure that it is safe and effective.
Clinical trials are essential for this purpose. By participating in clinical trials, research participants help to
ensure that new medical interventions are safe and effective for everyone.

15. EXAMPLES OF BENEFICENCE IN MEDICAL
RESEARCH
 Studying the long-term effects of medical treatments. Many medical treatments have long-term effects, both positive
and negative. Researchers have a responsibility to study these long-term effects so that they can better understand the
risks and benefits of different treatments and make recommendations to patients and healthcare providers.
 Conducting research to improve the quality of life for patients with chronic diseases. Chronic diseases, such as cancer,
heart disease, and diabetes, have a significant impact on the quality of life of patients and their families. Researchers
are constantly working to develop new ways to improve the management of chronic diseases and improve the quality
of life for patients.
 Developing new methods for diagnosing and treating diseases. New diagnostic and treatment methods can help to
improve the accuracy and timeliness of diagnosis, as well as the effectiveness of treatment. Researchers are constantly
working to develop new and improved methods for diagnosing and treating a wide range of diseases.

16. NON- MALEFICENCE
 Non-maleficence in medical research is the ethical principle of
avoiding or minimizing harm to research participants. It is one of the
four core ethical principles of research ethics, along with beneficence,
autonomy, and justice.
 Non-maleficence is important in medical research because it ensures
that research participants are not subjected to unnecessary risks. It
also helps to ensure that research is conducted in a safe and
responsible manner.
Continuous Monitoring

17. HOW TO APPLY NON- MALEFICENCE
 Careful design of research studies: Researchers should carefully design their studies to minimize the risks to
participants. This includes carefully considering the study population, the interventions being studied, and the
methods being used.
 Informed consent: Research participants must be fully informed of the risks and benefits of participating in a study
before they can give their consent. Researchers have a responsibility to provide participants with all of the
information they need to make an informed decision about whether or not to participate.
 Continuous monitoring of participants: Researchers should continuously monitor participants during the study to
identify any potential harms. If any harms are identified, researchers should take steps to mitigate them.
 Data safety monitoring boards (DSMBs): DSMBs are independent groups of experts who review data from clinical
trials to assess the safety and efficacy of the interventions being studied. DSMBs can recommend that a trial be
stopped early if the risks to participants outweigh the potential benefits.

18. HOW TO APPLY NON- MALEFICENCE
 Use validated research methods and instruments: This will help to ensure that the research is conducted in a safe
and reliable manner.
 Pilot test new methods and procedures before using them with participants: This will help to identify any
potential risks and make necessary adjustments.
 Use the least invasive methods possible: This means using methods that are less likely to cause discomfort or harm
to participants.
 Provide participants with appropriate support and resources: This may include providing participants with access
to medical care, financial assistance, or counseling.
 Respect the confidentiality and privacy of participants: This means taking steps to protect participants' personal
information and ensuring that they are not identified in any published reports or presentations.

19. EXAMPLES OF NON- MALEFICENCE IN
MEDICAL RESEARCH
 A researcher is developing a new drug for treating cancer. The researcher knows that cancer is a serious and
deadly disease, and that there is a need for new and effective treatments. However, the researcher also knows
that developing new drugs is a complex and challenging process, and that there is always the risk of side
effects, even serious ones.
 The researcher carefully considers the potential risks and benefits of the new drug before starting a clinical
trial. The researcher also takes steps to minimize the risks to participants. For example, the researcher starts
by testing the drug in a small group of healthy adults to assess its safety. Once the drug is shown to be safe in
this group, the researcher expands the trial to include more participants, including people with cancer.
 The researcher also continuously monitors participants for any potential harms. If any harms are identified,
the researcher takes steps to mitigate them. For example, if a participant experiences a serious side effect, the
researcher will remove the participant from the trial and provide them with appropriate medical care.

20. DOUBLE EFFECT
 The doctrine of double effect (DDE) is a moral principle that can be applied to medical research to justify actions that
have both intended and unintended effects, where the intended effects are good and the unintended effects are
unavoidable. The DDE is often used in medical research to justify the use of animal models and the conduct of
clinical trials, which both have the potential to harm participants but also have the potential to benefit society as a
whole.
 The DDE has four main criteria:
 The intended effect must be good.
 The unintended effect must be foreseen but not intended.
 The unintended effect must not be a means to achieving the intended effect.
 The good effect must outweigh the bad effect.

21. EXAMPLES OF DOUBLE EFFECTS IN MEDICAL
RESEARCH
 Researchers develop a new drug to treat cancer, but the drug has the potential to cause serious side
effects, including death. The researchers conduct a clinical trial of the drug, and some of the participants
experience serious side effects, but the drug is also effective in treating cancer. The ethical justification for
conducting the clinical trial is that the good effect (developing a new and effective cancer treatment) outweighs the
bad effect (the potential to harm participants).
 Researchers study a new experimental treatment for Alzheimer's disease in mice. The treatment is effective in
reducing the symptoms of Alzheimer's disease in mice, but it also causes some of the mice to experience pain and
distress. The ethical justification for using mice in this research is that the good effect (developing a new and
effective treatment for Alzheimer's disease) outweighs the bad effect (the harm caused to the mice).

22. Medical Ethics Definition Examples
Autonomy
The right of patients to make their own decisions
about their healthcare, even if those decisions are
not in their best interests.
A patient has the right to refuse a life-saving
blood transfusion, even if it means that they
will die.
Beneficence
The duty of healthcare professionals to act in the
best interests of their patients.
A doctor prescribes a medication to a patient
to relieve their pain, even though the
medication has some side effects.
Non-maleficence
The duty of healthcare professionals to avoid
harming their patients.
A surgeon refuses to perform a risky surgery
on a patient, even though the surgery could
potentially save their life.
Double effect
A principle that allows healthcare professionals to
perform an action that has both good and bad
effects, as long as the good effect is the primary
intention and the bad effect is an unavoidable side
effect.
A doctor gives a patient a chemotherapy drug
that kills cancer cells, but also damages
healthy cells.
Conflicts between autonomy
and beneficence/non-
maleficence
Situations where a patient's autonomous decision is
not in their best interests, or where acting in the
patient's best interests would violate their autonomy.
A Jehovah's Witness patient refuses a blood
transfusion, even though they need it to
survive. A doctor wants to force-feed a patient
who is refusing to eat, even though the patient
is competent and understands the
consequences of their decision.

23. REFERENCES
1) Guraya, S. Y., London, N. J. M., & Guraya, S. S. (2014). Ethics in medical
research. Journal of Microscopy and Ultrastructure, 2(3), 121-126.
2) Hoeyer, K., & Hogle, L. F. (2014). Informed consent: The politics of intent and practice
in medical research ethics. Annual Review of Anthropology, 43, 347-362.