Podcast
Questions and Answers
What role does a governance body play in clinical trials?
What role does a governance body play in clinical trials?
Which statement about unregistered drugs is accurate under the TG Act?
Which statement about unregistered drugs is accurate under the TG Act?
Which aspect is NOT addressed by the National Statement on Ethical Conduct of Human Research?
Which aspect is NOT addressed by the National Statement on Ethical Conduct of Human Research?
What is a key theme in research ethics according to the National Statement?
What is a key theme in research ethics according to the National Statement?
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In clinical trial design, which element is emphasized for effective oversight?
In clinical trial design, which element is emphasized for effective oversight?
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Which section of the National Statement discusses ethical considerations in research design?
Which section of the National Statement discusses ethical considerations in research design?
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What key aspect should researchers consider during trial design according to the National Statement?
What key aspect should researchers consider during trial design according to the National Statement?
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What is the primary goal of HREC approval in clinical trials?
What is the primary goal of HREC approval in clinical trials?
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What is the primary ethical concern with using placebos in clinical trials?
What is the primary ethical concern with using placebos in clinical trials?
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Which method helps protect the integrity of trial data while ensuring fairness?
Which method helps protect the integrity of trial data while ensuring fairness?
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What does 'blinding' refer to in clinical trials?
What does 'blinding' refer to in clinical trials?
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What is meant by 'clinical equipoise' in the context of trials?
What is meant by 'clinical equipoise' in the context of trials?
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Why is ongoing interim data analysis important in clinical trials?
Why is ongoing interim data analysis important in clinical trials?
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What is a potential drawback of randomisation in clinical trials?
What is a potential drawback of randomisation in clinical trials?
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What is an essential component of obtaining informed consent in clinical trials?
What is an essential component of obtaining informed consent in clinical trials?
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Which of the following strategies is primarily aimed at reducing bias in clinical trials?
Which of the following strategies is primarily aimed at reducing bias in clinical trials?
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What is the primary purpose of an ethics review in clinical trials?
What is the primary purpose of an ethics review in clinical trials?
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Which aspect is NOT a responsibility of the ethics committee in a clinical trial?
Which aspect is NOT a responsibility of the ethics committee in a clinical trial?
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What must participants in a clinical trial be provided with to give informed consent?
What must participants in a clinical trial be provided with to give informed consent?
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What is the main aim of the Guideline for Good Clinical Practice?
What is the main aim of the Guideline for Good Clinical Practice?
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Which of the following is a key ethical consideration in clinical trials?
Which of the following is a key ethical consideration in clinical trials?
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Which statement accurately reflects the role of the ethics committee?
Which statement accurately reflects the role of the ethics committee?
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What international agency is responsible for setting standards for registration of pharmaceuticals for human use?
What international agency is responsible for setting standards for registration of pharmaceuticals for human use?
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What does the ethical framework in clinical trials primarily protect?
What does the ethical framework in clinical trials primarily protect?
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Which program allows access to medications at a reduced cost for individuals who otherwise might not afford them?
Which program allows access to medications at a reduced cost for individuals who otherwise might not afford them?
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Which principle of ethics emphasizes the importance of doing no harm to patients during medical practices?
Which principle of ethics emphasizes the importance of doing no harm to patients during medical practices?
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What is the primary ethical concern related to patient participation in clinical trials?
What is the primary ethical concern related to patient participation in clinical trials?
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In the context of ethical principles, which term refers to the patient's right to self-determination?
In the context of ethical principles, which term refers to the patient's right to self-determination?
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Which MAP program primarily involves drugs that are not yet registered but are being provided to patients?
Which MAP program primarily involves drugs that are not yet registered but are being provided to patients?
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What ethical principle is primarily concerned with the equitable treatment of all patients?
What ethical principle is primarily concerned with the equitable treatment of all patients?
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Which of the following is NOT one of the four main pillars of ethics in medical practice?
Which of the following is NOT one of the four main pillars of ethics in medical practice?
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Which statement regarding compassionate use programs is accurate?
Which statement regarding compassionate use programs is accurate?
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What historical event was significant in shaping the development of the ICH GCP guidelines?
What historical event was significant in shaping the development of the ICH GCP guidelines?
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Which of the following best describes the role of a principal investigator in clinical trials?
Which of the following best describes the role of a principal investigator in clinical trials?
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What does the Therapeutic Goods Act 1989 primarily mandate concerning clinical trials?
What does the Therapeutic Goods Act 1989 primarily mandate concerning clinical trials?
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Which of the following is a major responsibility of pharmacies in clinical trials?
Which of the following is a major responsibility of pharmacies in clinical trials?
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In the context of clinical trials, what role does the Ethics Committee serve?
In the context of clinical trials, what role does the Ethics Committee serve?
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Which key principle was established by the Universal Declaration of Human Rights in response to inhumane trials?
Which key principle was established by the Universal Declaration of Human Rights in response to inhumane trials?
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What important factor must be considered in the governance of clinical trials in Australia?
What important factor must be considered in the governance of clinical trials in Australia?
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Which of the following incidents significantly impacted the establishment of regulatory frameworks for clinical trials?
Which of the following incidents significantly impacted the establishment of regulatory frameworks for clinical trials?
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What additional requirement is necessary for a medicine to be recommended for PBS listing beyond safety and efficacy?
What additional requirement is necessary for a medicine to be recommended for PBS listing beyond safety and efficacy?
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Which of the following conditions allows for the PBS listing of a new drug?
Which of the following conditions allows for the PBS listing of a new drug?
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What does the Special Access Scheme provide for patients?
What does the Special Access Scheme provide for patients?
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Which statement accurately describes the role of TGA in the National Medicines Policy?
Which statement accurately describes the role of TGA in the National Medicines Policy?
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Under which circumstance can a drug not on the ARTG be imported according to section 19A?
Under which circumstance can a drug not on the ARTG be imported according to section 19A?
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What type of medications does the Serious Scarcity Substitution Instrument allow to be substituted?
What type of medications does the Serious Scarcity Substitution Instrument allow to be substituted?
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What is a requirement for importing unapproved therapeutic goods for personal use?
What is a requirement for importing unapproved therapeutic goods for personal use?
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What is the primary objective of the National Medicines Policy?
What is the primary objective of the National Medicines Policy?
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What is a primary focus of Phase II studies in clinical trials?
What is a primary focus of Phase II studies in clinical trials?
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In Phase III studies, what is primarily compared in randomized controlled trials?
In Phase III studies, what is primarily compared in randomized controlled trials?
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Who typically participates in Phase II clinical trials?
Who typically participates in Phase II clinical trials?
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What is a significant design characteristic of Phase III studies?
What is a significant design characteristic of Phase III studies?
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What is the primary endpoint often evaluated in Phase II studies?
What is the primary endpoint often evaluated in Phase II studies?
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Why are Phase III studies considered important in the clinical trial process?
Why are Phase III studies considered important in the clinical trial process?
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What distinguishes Phase II studies from Phase I studies in clinical trials?
What distinguishes Phase II studies from Phase I studies in clinical trials?
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What role do Phase III studies play in clinical trial design feedback?
What role do Phase III studies play in clinical trial design feedback?
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What is a necessary condition for clinical equipoise to exist in a randomized clinical trial?
What is a necessary condition for clinical equipoise to exist in a randomized clinical trial?
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Why is monitoring interim data critical in long-term multi-centre trials?
Why is monitoring interim data critical in long-term multi-centre trials?
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What ethical concern arises when a randomized trial includes a standard of care (SOC) treatment arm?
What ethical concern arises when a randomized trial includes a standard of care (SOC) treatment arm?
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In the context of clinical trials, what outcome is associated with maintaining clinical equipoise throughout the study?
In the context of clinical trials, what outcome is associated with maintaining clinical equipoise throughout the study?
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What critical aspect should a researcher consider when designing a trial to adhere to clinical equipoise principles?
What critical aspect should a researcher consider when designing a trial to adhere to clinical equipoise principles?
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What is the ethical consideration regarding the use of placebos in clinical trials?
What is the ethical consideration regarding the use of placebos in clinical trials?
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What does the term 'clinical equipoise' imply in research ethics?
What does the term 'clinical equipoise' imply in research ethics?
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Why is it crucial to have informed consent translated by an official translator in trials?
Why is it crucial to have informed consent translated by an official translator in trials?
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In the context of ethical trial design, randomisation and blinding serve to:
In the context of ethical trial design, randomisation and blinding serve to:
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What essential aspect must be ensured when designing clinical trials involving multiple treatment arms?
What essential aspect must be ensured when designing clinical trials involving multiple treatment arms?
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What is a significant ethical challenge when researchers randomize participants?
What is a significant ethical challenge when researchers randomize participants?
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What is necessary for maintaining ethical standards throughout a clinical trial?
What is necessary for maintaining ethical standards throughout a clinical trial?
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What should researchers consider when discussing the potential risks of therapies with participants?
What should researchers consider when discussing the potential risks of therapies with participants?
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What is the purpose of the Therapeutic Goods Administration (TGA)?
What is the purpose of the Therapeutic Goods Administration (TGA)?
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What criteria must be met for a drug to be subsidized under the Pharmaceutical Benefits Scheme (PBS)?
What criteria must be met for a drug to be subsidized under the Pharmaceutical Benefits Scheme (PBS)?
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Describe the concept of 'off label' use of medications.
Describe the concept of 'off label' use of medications.
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What is the significance of the 'Bench to Bedside' concept in drug development?
What is the significance of the 'Bench to Bedside' concept in drug development?
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What distinguishes classical pharmacology from reverse pharmacology?
What distinguishes classical pharmacology from reverse pharmacology?
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Explain the importance of post-marketing surveillance in medication safety.
Explain the importance of post-marketing surveillance in medication safety.
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What is the role of Clinical Trials in the introduction of new drugs into practice?
What is the role of Clinical Trials in the introduction of new drugs into practice?
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What purpose do placebo controls serve in clinical trials?
What purpose do placebo controls serve in clinical trials?
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Why is it considered unethical to use a placebo in certain clinical trials?
Why is it considered unethical to use a placebo in certain clinical trials?
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What is the placebo effect?
What is the placebo effect?
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How does blinding enhance the integrity of clinical trials?
How does blinding enhance the integrity of clinical trials?
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What is the difference between an active control and a placebo control?
What is the difference between an active control and a placebo control?
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Why are unapproved drugs, like placebos, significant in clinical trials?
Why are unapproved drugs, like placebos, significant in clinical trials?
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What is one major challenge that pharmacy staff face when managing clinical trials?
What is one major challenge that pharmacy staff face when managing clinical trials?
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What masking techniques may be used to ensure blinding in clinical trials?
What masking techniques may be used to ensure blinding in clinical trials?
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What is the main advantage of a multiple dosing scheme in clinical trials?
What is the main advantage of a multiple dosing scheme in clinical trials?
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In Phase II clinical trials, who are the usual participants and why are they chosen?
In Phase II clinical trials, who are the usual participants and why are they chosen?
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What is the primary aim of Phase III studies in clinical trials?
What is the primary aim of Phase III studies in clinical trials?
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How does Phase II differ from Phase I in clinical trials?
How does Phase II differ from Phase I in clinical trials?
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What is a randomized controlled trial (RCT) and why is it important in Phase III studies?
What is a randomized controlled trial (RCT) and why is it important in Phase III studies?
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What role does toxicity monitoring play in Phase II clinical trials?
What role does toxicity monitoring play in Phase II clinical trials?
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What is the significance of efficacy as an endpoint in Phase II studies?
What is the significance of efficacy as an endpoint in Phase II studies?
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During Phase III studies, what design strategies are commonly employed to minimize bias?
During Phase III studies, what design strategies are commonly employed to minimize bias?
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What historical events led to the establishment of the Nuremberg Code?
What historical events led to the establishment of the Nuremberg Code?
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How does the Therapeutic Goods Act 1989 interact with clinical trials in Australia?
How does the Therapeutic Goods Act 1989 interact with clinical trials in Australia?
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What role does the sponsor play in a clinical trial?
What role does the sponsor play in a clinical trial?
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Why is labeling of medications important in the context of clinical trials?
Why is labeling of medications important in the context of clinical trials?
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What significant impacts did the thalidomide incident have on clinical trials and regulations?
What significant impacts did the thalidomide incident have on clinical trials and regulations?
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What is the primary function of an ethics committee in clinical trials?
What is the primary function of an ethics committee in clinical trials?
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What is the significance of the Universal Declaration of Human Rights in relation to clinical trials?
What is the significance of the Universal Declaration of Human Rights in relation to clinical trials?
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How does the HREC contribute to the governance of clinical trials?
How does the HREC contribute to the governance of clinical trials?
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What is the main purpose of blinding in clinical trials?
What is the main purpose of blinding in clinical trials?
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How does randomisation contribute to the validity of clinical trials?
How does randomisation contribute to the validity of clinical trials?
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What is the significance of using a crossover design in clinical trials?
What is the significance of using a crossover design in clinical trials?
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Why are double-blind trials considered the gold standard in clinical research?
Why are double-blind trials considered the gold standard in clinical research?
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Study Notes
Medication Access Pathways
- Romana Cecchele is the author and has extensive experience in Pharmacy
- The lecture is about the ability to engage in research and scientifically examine evidence
- Topics include understanding medication supply pathways in Australia, clinical evaluation of new and existing drugs, post-marketing surveillance, and prospects of new therapeutic approaches
- Key aspect of the process for introducing new drugs is bench to bedside
- Several aspects of a new drug entity includes serendipity, classical (forward) pharmacology, and reverse pharmacology (targeted)
- The process for introducing new drugs to practice includes clinical trials (pre- and post registration), early clinical experience, registration, and costs (who pays)
- Methods of accessing drugs in Australia are TGA Registered, Special Access Scheme, Authorised Prescriber, Clinical Trials, and Personal Importation
- Registered medications are those compliant with the Therapeutic Goods Administration (TGA) and are listed on the Australian Register of Therapeutic Goods (ARTG) as safe and effective for specified indications.
Registered Medications
- Legally marketed, prescribed, and dispensed
- Classified for availability according to schedule (S2, S3, S4, S8)
- Unregistered drugs cannot be advertised.
Status of Drugs in Clinical Practice
- The Pharmaceutical Benefits Scheme (PBS) is discussed
- Drug origins, purpose, section 85 (General Schedule), and section 100 (National Health Act 1953) are part of the PBS process.
Pharmaceutical Benefits Scheme
- TGA registration needs evidence of safety and efficacy
- PBS listing also needs cost-effectiveness
- An acceptably cost-effective medicine can be recommended for listing if the following conditions are met; it treats medical conditions not covered by currently listed drugs, it is more effective and less harmful than a currently listed drug, or it is as effective as an existing listed drug
- Section 19A of the Therapeutic Goods Act 1989 authorizes importing medications that are not registered on the ARTG for temporary use, as necessary
Special Access Scheme
- Provides import and supply of an unapproved therapeutic good to a single patient
- Conditions are met when the unapproved good is not substantially similar or available in the ARTG.
- Categories include A (notification for a seriously ill patient), B (application pathway), and C (notification of use of specified therapeutic goods)
Authorised Prescriber
- Used for multiple patients with the same condition
- Applies for individual patient
- Requires endorsement from the local human research committee (HREC)
- Reports on adverse events, product defects, and number of patients treated
Clinical Trials
- Approvals required: animal or human ethics research approval (HREC), TGA clinical trial notification (CTN) scheme (allows for importation or CTA), governance approval, drug committee approvals, equivalent health department approval, and IBC approvals.
- The Australian Clinical Trials Handbook provides guidance for conducting trials in Australia.
Evaluation of New Drugs
- Pre-evaluation involves early pharmacological studies
- Clinical evaluation involves pre- and post-registration human studies
Phases of Clinical Trials
- Preclinical studies are lab-based studies with several years needed to complete, focusing on dosing and toxicity levels.
- Phase 1 studies involve early human safety evaluation, including toxicity, maximum tolerated dose (MTD), and pharmacokinetics.
- Phase 2 studies involve further safety evaluation, dose finding, and efficacy testing.
- Phase 3 studies are comparative, randomized controlled trials (RCTs) to evaluate efficacy against a gold standard comparator, using large patient numbers.
- Phase 4 studies are post-marketing surveillance evaluations of the drug in different populations and long-term effects
Preclinical Evaluation
- Involves a new chemical entity, identification code or generic name, and application for a patent.
- Preclinical research includes toxicology screenings, in-vitro cell lines, pharmacogenomic probes, and in-vivo animal studies
Preclinical Studies
- Animal studies require ethics approval.
- Important for predicting human effectiveness and toxicity
Phase I Studies
- These are the first studies in humans, with small numbers of participants (10-100) highly monitored, specialist units
- Investigates safety, toxicity, maximum tolerated dose (MTD), pharmacokinetics and pharmacodynamics.
Phase II Studies
- Early phase and exploratory
- Involves 100-300 participants to assess therapeutic efficacy at the optimal dose
- Important for assessing optimal dose in a certain indication, often for refractory patients with no other treatment options
Phase III Studies
- Comparative, therapeutic studies involve 300-3000 patients
- Randomised controlled trials (RCTs) test hypotheses, comparing the experimental drug to a gold standard comparator.
Comparator
- A comparator may be a placebo or an active control treatment
- Active control is an established treatment that has efficacy
- Placebo control is a sham treatment without any active elements
Placebo Effect
- Placebo effect is a psychosomatic effect from anxiety relief or stress
- Placebos are considered unapproved therapeutic goods.
Trial Design, Manufacturing and Labelling
- Therapeutic Goods Act 1989 regulates investigational agents
- Pharmacists manufacturing for public use or in a hospital or other institution
Personal Importation
- Importing unapproved therapeutic goods for personal use or use by immediate family
- Meets conditions such as the goods are for individual use, in original packaging and not a controlled substance
National Statement on the Ethical Conduct of Human Research
- The National Statement sets out the requirements for an HREC
Ethics Committees
- Committees consist of university or hospital staff, researchers, practitioners, science training personnel, and lay persons, including a minister
- This is an independent body to assess ethical issues
- Multi-HRECs in multiple states can cause problems with applying state-specific laws
Clinical Equipoise
- Ethical basis for clinical research with patient allocation to different treatment arms
- Exists when there isn't a clear "better" option
- Requires regular interim data analysis and safety monitoring is required
- Ensures treatment arms have equal merits
Where are Clinical Trials Conducted?
- Conducted in hospital settings (specialist units, emergency settings)
Key Players in Clinical Trials
- Stakeholders involved in clinical trials include trial sponsors, principal investigators, clinical research associates, study coordinators, patients, and pharmacists
What is the Role of Pharmacists in Clinical Trials?
- Pharmacists can be involved in various roles, including design, monitoring, coordination of the trials, drug supply, auditing procedures, and patient follow-up
New Drugs - Impact on the Profession
- Pharmacists need to learn about new therapies through UpToDate.
- Understand research steps and critically evaluate literature.
Workshop
- Groups will evaluate Phase III clinical trials.
- Ethical considerations and features of specific diseases/cohorts.
Issues to Consider
- Relevant questions about participants, recruitment, ethical issues (refer to Section 3 of the National Statement), consent, data collected to demonstrate superiority, and trial completion.
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Description
Explore the essential pathways for medication access in Australia through this quiz. Delve into topics such as clinical evaluation, drug supply pathways, and the introduction process for new therapeutics. Understand the roles of TGA registration, clinical trials, and the economics of drug access.