HMM101 Medical Biotechnology - Clinical Trials and Regulation

Flashcards on Clinical trials, animal welfare, ethics and drug regulation.

Acknowledgment of Country

Acknowledge the Wurundjeri and Wadawurrung people of the Kulin nations as the Traditional Owners of the land.

Universal Declaration on Animal Welfare (UDAW)

A proposed inter-governmental agreement to recognize that animals are sentient, to prevent cruelty and reduce suffering.

The Three Rs

Reduction, Refinement, and Replacement of animals in research.

The Five Freedoms

Freedom from thirst and hunger, discomfort, pain/injury/disease, ability to express normal behavior, and fear/distress.

Pathways to ‘Cure’ disease

Drug discovery & proof of concept -> Pre-clinical in vitro & animal studies -> Clinical trials Phases I-III -> Govt Review -> Large scale manufacture Phase IV -> Approval

Clinical Trial (WHO Definition)

Research study that prospectively assigns human participants to health-related interventions to evaluate the effects on health outcomes.

Purpose of Clinical Trials

Prevent, detect, treat, or manage various diseases or medical conditions.

Clinical Trial Interventions

Experimental drugs, cells, biological products, vaccines, medical devices, diagnostics, treatments, therapies, health service changes, preventive care, and educational interventions.

Phase I Clinical Trial

Small number of participants (e.g., 20-80) to evaluate safety and determine safe dosage range/side effects.

Phase II Clinical Trial

Larger group of people (hundreds) to determine efficacy and further evaluate safety.

Phase III Clinical Trial

Large groups of participants (hundreds to thousands) to study efficacy, compare to other interventions, monitor adverse effects, and collect safety information.

Phase IV Clinical Trial

Done after an intervention has been marketed to monitor effectiveness in the general population and collect information about adverse effects over longer periods.

Clinical Trial Protocol

A plan that describes what researchers will do in the study; ensures safety and accurate measurements.

Types of Trial Protocols: Comparing a new intervention to a standard intervention

Compares a new intervention with a standard or commonly used intervention.

Types of Trial Protocols: Comparing a new intervention with a placebo

Compares a new intervention with a dummy treatment (sugar pill) that has no known health effect.

The Placebo Effect

When you believe you are taking effective medicine, so your symptoms improve.

Randomisation

Participants are allocated to each group without decision by anyone involved in running the trial (usually done by a computer).

Blinding/Masking

Participants and/or researchers don’t know who is receiving the new intervention.

Single Blind Trial

Participants do not know which intervention they are receiving, but the researchers and medical staff do.

Double Blind Trial

Neither the researchers organizing the trial nor those taking part in the trial know who is receiving which intervention.

BEST Clinical Trials

Double Blind, Randomised, Placebo controlled

Orphan drugs

Rare conditions that affect a few people -Usually fast tracked when it comes to approval

Clinical Trial Team

Doctors, nurses, health care professionals, biostatisticians, trial coordinators and monitors.

Trial Guidelines

Guidelines about who can take part in a clinical trial (inclusion and exclusion criteria).

Indigenous health research

Focuses more on a collaborative partnership of researchers and local communities- getting consent from local leaders of the community

Epistemology

Is the study of knowledge acquisition. It involves an awareness of certain aspects of reality, and it seeks to discover what is known and how it is known

Why Regulate Drugs?

Effective drug regulation is required to ensure the safety, efficacy and quality of drugs, as well as the accuracy and appropriateness of the drug information available to the public.

USFDA (Food and Drug Administration)

An agency of the United States Department of Health and Human Services, responsible for protecting and promoting public health through the regulation and supervision of various products.

TGA (Therapeutic Goods Administration)

Part of the Australian Government Department of Health and Ageing, responsible for regulating therapeutic goods including medicines, medical devices, blood and blood products.

Is it a medicine or food?

A product's principal use is of primary consideration when determining whether it is a food or a medicine.

Is it a therapeutic good or a cosmetic?

The claims made about the product.