Medication Access Pathways in Australia
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Questions and Answers

What role does a governance body play in clinical trials?

  • They handle patient recruitment solely.
  • They manage the data collection process.
  • They provide financial support for the trials.
  • They review the legal aspects and ensure insurance coverage. (correct)

Which statement about unregistered drugs is accurate under the TG Act?

  • They can only be advertised with the agent's name mentioned.
  • They cannot be advertised at all. (correct)
  • They must have HREC approval before advertisement.
  • They can be freely advertised without restrictions.

Which aspect is NOT addressed by the National Statement on Ethical Conduct of Human Research?

  • Promotion strategies for clinical trials. (correct)
  • Requirements for ethical research design.
  • Common ethical issues that arise in research.
  • Operational procedures of Human Research Ethics Committees.

What is a key theme in research ethics according to the National Statement?

<p>Risk and benefit, and informed consent. (B)</p> Signup and view all the answers

In clinical trial design, which element is emphasized for effective oversight?

<p>Ethical standards evaluation. (D)</p> Signup and view all the answers

Which section of the National Statement discusses ethical considerations in research design?

<p>Section 3 - Ethical Considerations. (C)</p> Signup and view all the answers

What key aspect should researchers consider during trial design according to the National Statement?

<p>Potential risks and benefits to participants. (C)</p> Signup and view all the answers

What is the primary goal of HREC approval in clinical trials?

<p>To assess the ethical implications and participant safety. (D)</p> Signup and view all the answers

What is the primary ethical concern with using placebos in clinical trials?

<p>They may withhold effective treatment from participants. (A)</p> Signup and view all the answers

Which method helps protect the integrity of trial data while ensuring fairness?

<p>Randomisation. (A)</p> Signup and view all the answers

What does 'blinding' refer to in clinical trials?

<p>Neither the participants nor the researchers know the treatment assignments. (B)</p> Signup and view all the answers

What is meant by 'clinical equipoise' in the context of trials?

<p>Equal therapeutic merit among treatment options. (B)</p> Signup and view all the answers

Why is ongoing interim data analysis important in clinical trials?

<p>To monitor safety and efficacy of treatments. (B)</p> Signup and view all the answers

What is a potential drawback of randomisation in clinical trials?

<p>Participants might receive suboptimal treatment doses. (C)</p> Signup and view all the answers

What is an essential component of obtaining informed consent in clinical trials?

<p>Participants must be fully informed and voluntary. (B)</p> Signup and view all the answers

Which of the following strategies is primarily aimed at reducing bias in clinical trials?

<p>Randomisation of treatment assignments. (C)</p> Signup and view all the answers

What is the primary purpose of an ethics review in clinical trials?

<p>To advocate for the scientific merit and integrity of the research (C)</p> Signup and view all the answers

Which aspect is NOT a responsibility of the ethics committee in a clinical trial?

<p>Conducting the clinical trial themselves (B)</p> Signup and view all the answers

What must participants in a clinical trial be provided with to give informed consent?

<p>Detailed information about risks, benefits, and procedures (C)</p> Signup and view all the answers

What is the main aim of the Guideline for Good Clinical Practice?

<p>To enhance the scientific quality and ethical standards of clinical trials (B)</p> Signup and view all the answers

Which of the following is a key ethical consideration in clinical trials?

<p>Ensuring that participants are under no duress when giving consent (D)</p> Signup and view all the answers

Which statement accurately reflects the role of the ethics committee?

<p>To express an opinion on the trial protocol and methods used (C)</p> Signup and view all the answers

What international agency is responsible for setting standards for registration of pharmaceuticals for human use?

<p>International Conference on Harmonisation (ICH) (C)</p> Signup and view all the answers

What does the ethical framework in clinical trials primarily protect?

<p>The rights, safety, and wellbeing of participants (B)</p> Signup and view all the answers

Which program allows access to medications at a reduced cost for individuals who otherwise might not afford them?

<p>Cost Share Programs (C)</p> Signup and view all the answers

Which principle of ethics emphasizes the importance of doing no harm to patients during medical practices?

<p>Non-Maleficence (C)</p> Signup and view all the answers

What is the primary ethical concern related to patient participation in clinical trials?

<p>Obtaining informed consent (D)</p> Signup and view all the answers

In the context of ethical principles, which term refers to the patient's right to self-determination?

<p>Autonomy (D)</p> Signup and view all the answers

Which MAP program primarily involves drugs that are not yet registered but are being provided to patients?

<p>Extended Access Programs (A)</p> Signup and view all the answers

What ethical principle is primarily concerned with the equitable treatment of all patients?

<p>Justice (B)</p> Signup and view all the answers

Which of the following is NOT one of the four main pillars of ethics in medical practice?

<p>Transparency (B)</p> Signup and view all the answers

Which statement regarding compassionate use programs is accurate?

<p>It allows access to unapproved drugs for terminally ill patients. (D)</p> Signup and view all the answers

What historical event was significant in shaping the development of the ICH GCP guidelines?

<p>The Nuremberg Trials (B)</p> Signup and view all the answers

Which of the following best describes the role of a principal investigator in clinical trials?

<p>To oversee the trial at a specific site (A)</p> Signup and view all the answers

What does the Therapeutic Goods Act 1989 primarily mandate concerning clinical trials?

<p>Review and monitoring by an HREC (A)</p> Signup and view all the answers

Which of the following is a major responsibility of pharmacies in clinical trials?

<p>Ensuring compliance with medication labeling laws (B)</p> Signup and view all the answers

In the context of clinical trials, what role does the Ethics Committee serve?

<p>To review and assess the ethical implications of the trial (B)</p> Signup and view all the answers

Which key principle was established by the Universal Declaration of Human Rights in response to inhumane trials?

<p>Informed consent of participants (B)</p> Signup and view all the answers

What important factor must be considered in the governance of clinical trials in Australia?

<p>Compliance with ARTG for advertising (B)</p> Signup and view all the answers

Which of the following incidents significantly impacted the establishment of regulatory frameworks for clinical trials?

<p>Thalidomide scandal (A)</p> Signup and view all the answers

What additional requirement is necessary for a medicine to be recommended for PBS listing beyond safety and efficacy?

<p>Cost effectiveness (D)</p> Signup and view all the answers

Which of the following conditions allows for the PBS listing of a new drug?

<p>It treats a significantly harmful medical condition that is not addressed by existing drugs (C)</p> Signup and view all the answers

What does the Special Access Scheme provide for patients?

<p>Access to unapproved therapeutic goods that are essential (A)</p> Signup and view all the answers

Which statement accurately describes the role of TGA in the National Medicines Policy?

<p>TGA ensures the quality and safety of drugs (B)</p> Signup and view all the answers

Under which circumstance can a drug not on the ARTG be imported according to section 19A?

<p>When there is no TGA registered alternative (D)</p> Signup and view all the answers

What type of medications does the Serious Scarcity Substitution Instrument allow to be substituted?

<p>Any type of Schedule 4 medications (B)</p> Signup and view all the answers

What is a requirement for importing unapproved therapeutic goods for personal use?

<p>The medication is for immediate family members only (A)</p> Signup and view all the answers

What is the primary objective of the National Medicines Policy?

<p>To promote the quality use of medicines (C)</p> Signup and view all the answers

What is a primary focus of Phase II studies in clinical trials?

<p>Establishing optimal dosing in specific indications (A)</p> Signup and view all the answers

In Phase III studies, what is primarily compared in randomized controlled trials?

<p>The investigational agent against a known gold standard or placebo (B)</p> Signup and view all the answers

Who typically participates in Phase II clinical trials?

<p>Refractory patients without other treatment options (A)</p> Signup and view all the answers

What is a significant design characteristic of Phase III studies?

<p>They use randomization to assign participants to treatment groups. (D)</p> Signup and view all the answers

What is the primary endpoint often evaluated in Phase II studies?

<p>Optimal dose for a specified indication in the target population (A)</p> Signup and view all the answers

Why are Phase III studies considered important in the clinical trial process?

<p>They generate the most substantial data for drug registration. (A)</p> Signup and view all the answers

What distinguishes Phase II studies from Phase I studies in clinical trials?

<p>Phase II studies concentrate on specific indications rather than general safety. (C)</p> Signup and view all the answers

What role do Phase III studies play in clinical trial design feedback?

<p>They help reduce complexities observed in earlier trials. (D)</p> Signup and view all the answers

What is a necessary condition for clinical equipoise to exist in a randomized clinical trial?

<p>Lack of a compelling reason to prefer one intervention over another (C)</p> Signup and view all the answers

Why is monitoring interim data critical in long-term multi-centre trials?

<p>It allows for early detection of ineffectiveness or toxicity (A)</p> Signup and view all the answers

What ethical concern arises when a randomized trial includes a standard of care (SOC) treatment arm?

<p>There is a risk that participants may receive suboptimal treatment (D)</p> Signup and view all the answers

In the context of clinical trials, what outcome is associated with maintaining clinical equipoise throughout the study?

<p>Ethical justification for continued randomization of treatments (D)</p> Signup and view all the answers

What critical aspect should a researcher consider when designing a trial to adhere to clinical equipoise principles?

<p>Equal therapeutic merits of all trial arms at the time of design (A)</p> Signup and view all the answers

What is the ethical consideration regarding the use of placebos in clinical trials?

<p>Participants can be assigned to placebo even if effective treatments exist. (A)</p> Signup and view all the answers

What does the term 'clinical equipoise' imply in research ethics?

<p>A balanced uncertainty exists regarding the efficacy of treatment options. (D)</p> Signup and view all the answers

Why is it crucial to have informed consent translated by an official translator in trials?

<p>It ensures participants understand the treatment and associated risks. (C)</p> Signup and view all the answers

In the context of ethical trial design, randomisation and blinding serve to:

<p>Protect the integrity of data while potentially introducing unfairness. (C)</p> Signup and view all the answers

What essential aspect must be ensured when designing clinical trials involving multiple treatment arms?

<p>All treatment arms must have equal therapeutic merits. (A)</p> Signup and view all the answers

What is a significant ethical challenge when researchers randomize participants?

<p>To ensure participants remain unaware of their treatment. (D)</p> Signup and view all the answers

What is necessary for maintaining ethical standards throughout a clinical trial?

<p>Monitoring safety and regularly analyzing interim data. (B)</p> Signup and view all the answers

What should researchers consider when discussing the potential risks of therapies with participants?

<p>All risks must be communicated, regardless of severity. (C)</p> Signup and view all the answers

What is the purpose of the Therapeutic Goods Administration (TGA)?

<p>The TGA ensures that medications are safe, effective, and suitable for a specified indication before they can be marketed.</p> Signup and view all the answers

What criteria must be met for a drug to be subsidized under the Pharmaceutical Benefits Scheme (PBS)?

<p>Drugs must demonstrate safety, efficacy, and cost-effectiveness to be listed for subsidy under PBS.</p> Signup and view all the answers

Describe the concept of 'off label' use of medications.

<p>'Off label' use refers to prescribing medications for indications not approved by regulatory authorities.</p> Signup and view all the answers

What is the significance of the 'Bench to Bedside' concept in drug development?

<p>The 'Bench to Bedside' concept highlights the process of translating laboratory research into clinical practice.</p> Signup and view all the answers

What distinguishes classical pharmacology from reverse pharmacology?

<p>Classical pharmacology focuses on discovering drug effects through observation, while reverse pharmacology involves targeting specific biological pathways.</p> Signup and view all the answers

Explain the importance of post-marketing surveillance in medication safety.

<p>Post-marketing surveillance is crucial for monitoring the long-term safety and effectiveness of drugs after they are approved.</p> Signup and view all the answers

What is the role of Clinical Trials in the introduction of new drugs into practice?

<p>Clinical Trials assess the safety and efficacy of new drugs before they receive regulatory approval and enter the marketplace.</p> Signup and view all the answers

What purpose do placebo controls serve in clinical trials?

<p>Placebo controls help attribute effects solely to the new drug being tested, ruling out other factors.</p> Signup and view all the answers

Why is it considered unethical to use a placebo in certain clinical trials?

<p>It is unethical to withhold treatment from patients when doing so could lead to irreversible harm.</p> Signup and view all the answers

What is the placebo effect?

<p>The placebo effect is a psychosomatic response where participants experience real changes due to their belief in the treatment.</p> Signup and view all the answers

How does blinding enhance the integrity of clinical trials?

<p>Blinding prevents participants from knowing whether they receive the active treatment or a placebo, reducing bias.</p> Signup and view all the answers

What is the difference between an active control and a placebo control?

<p>An active control involves an established treatment, while a placebo control tests a treatment against a non-active substitute.</p> Signup and view all the answers

Why are unapproved drugs, like placebos, significant in clinical trials?

<p>Placebos are crucial for exploring the placebo effect and understanding treatment responses independent of the active drug.</p> Signup and view all the answers

What is one major challenge that pharmacy staff face when managing clinical trials?

<p>Pharmacy staff often experience strain due to unblinded patients knowing their treatments, complicating trial management.</p> Signup and view all the answers

What masking techniques may be used to ensure blinding in clinical trials?

<p>Techniques like covering bags and lines in amber tape are used to obscure the treatment assignments.</p> Signup and view all the answers

What is the main advantage of a multiple dosing scheme in clinical trials?

<p>It allows the drug to reach steady state in the body and evaluate long-term effects.</p> Signup and view all the answers

In Phase II clinical trials, who are the usual participants and why are they chosen?

<p>Participants are typically refractory patients with no alternative treatment options.</p> Signup and view all the answers

What is the primary aim of Phase III studies in clinical trials?

<p>To compare the investigational agent with a gold standard or placebo to test a hypothesis.</p> Signup and view all the answers

How does Phase II differ from Phase I in clinical trials?

<p>Phase II focuses on dose finding for specific indications rather than establishing safety in healthy individuals.</p> Signup and view all the answers

What is a randomized controlled trial (RCT) and why is it important in Phase III studies?

<p>An RCT is a study design that randomizes participants into different treatment groups to ensure unbiased results.</p> Signup and view all the answers

What role does toxicity monitoring play in Phase II clinical trials?

<p>It helps assess the safety and adverse effects of the drug during the trial.</p> Signup and view all the answers

What is the significance of efficacy as an endpoint in Phase II studies?

<p>Efficacy as an endpoint helps resolve uncertainties and informs the design of subsequent trials.</p> Signup and view all the answers

During Phase III studies, what design strategies are commonly employed to minimize bias?

<p>Common strategies include blinding, parallel, or crossover design.</p> Signup and view all the answers

What historical events led to the establishment of the Nuremberg Code?

<p>The unethical medical experiments conducted by Nazi researchers during World War II were pivotal in establishing the Nuremberg Code.</p> Signup and view all the answers

How does the Therapeutic Goods Act 1989 interact with clinical trials in Australia?

<p>The Therapeutic Goods Act 1989 requires that all clinical trials of unregistered therapeutic goods be reviewed and monitored by a Human Research Ethics Committee (HREC).</p> Signup and view all the answers

What role does the sponsor play in a clinical trial?

<p>The sponsor, typically a manufacturer, is responsible for developing the protocol and financing the clinical trial.</p> Signup and view all the answers

Why is labeling of medications important in the context of clinical trials?

<p>Labeling is crucial for compliance with legislation and ensuring patients receive clear and accurate information about the medications being administered.</p> Signup and view all the answers

What significant impacts did the thalidomide incident have on clinical trials and regulations?

<p>The thalidomide tragedy highlighted the need for stricter regulations and ethical considerations in drug testing, leading to more rigorous oversight.</p> Signup and view all the answers

What is the primary function of an ethics committee in clinical trials?

<p>The ethics committee reviews the ethical implications of proposed clinical trials to ensure the rights and welfare of participants are protected.</p> Signup and view all the answers

What is the significance of the Universal Declaration of Human Rights in relation to clinical trials?

<p>The Universal Declaration of Human Rights established fundamental rights that protect individuals, influencing ethical standards in human research.</p> Signup and view all the answers

How does the HREC contribute to the governance of clinical trials?

<p>The Human Research Ethics Committee (HREC) ensures that clinical trials are ethically approved and monitored throughout their duration.</p> Signup and view all the answers

What is the main purpose of blinding in clinical trials?

<p>The main purpose of blinding is to prevent conscious or unconscious bias in the outcomes of the trial.</p> Signup and view all the answers

How does randomisation contribute to the validity of clinical trials?

<p>Randomisation ensures that each participant has an equal chance of being allocated to any treatment arm, which reduces bias in the trial outcomes.</p> Signup and view all the answers

What is the significance of using a crossover design in clinical trials?

<p>A crossover design allows participants to receive multiple treatments in different periods, acting as their own control, which helps in obtaining cleaner data.</p> Signup and view all the answers

Why are double-blind trials considered the gold standard in clinical research?

<p>Double-blind trials are considered the gold standard because both participants and researchers are unaware of treatment assignments, minimizing biases and influencing factors.</p> Signup and view all the answers

Flashcards

Human Research Ethics

National standards for designing and reviewing human research, ensuring ethical conduct.

HREC

Human Research Ethics Committee, overseeing research projects' ethical compliance.

Ethical Clinical Trial

A clinical trial designed and conducted with respect for relevant ethical principles.

National Statement

Australia's national guideline for ethical conduct of human research, emphasizing principles and standards to consider during design review.

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Ethical Considerations (Trial)

Examines the ethical implications of research, including risk-benefit assessments and informed consent processes.

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Unregistered Drug Promotion

Promoting trial drugs not registered in Australia limits the promotion of the trial agent, and the advertisement must be approved.

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Elements of Research

Essential components of a research study, including ethical review, oversight, design and conducting process.

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Clinical Trial Review

Procedures and oversight systems required for a clinical trial designed for conducting review.

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Ethical Research

Research conducted considering the well-being of participants, including informed consent, voluntary participation, and adequate information.

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Informed Consent

Participants understand the study, risks, and benefits, and agree to participate voluntarily.

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Placebo

An inactive substance or procedure used in a trial as a control.

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Randomisation

Assigning participants to different groups (treatment or control) randomly to reduce bias.

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Blinding

Keeping participants and/or researchers unaware of group assignments to prevent bias.

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Clinical Equipoise

Ethical principle in clinical trials; no clear treatment superiority exists when study begins.

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Ethical Trial Design

A trial that considers participant safety and well-being whilst producing valuable data.

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Level of Evidence

Different types of research studies have varying levels of strength in proving drug/therapy effectiveness, with meta-analyses considered strongest.

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Ethics Review Committees (HRECs)

Groups that ensure research involving humans is ethical and protects participant rights.

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Informed Consent

Written agreement from research participants understanding the risks, benefits, and procedures of a clinical trial.

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Clinical Trial Protocol

A detailed plan for a clinical trial, outlining procedures, participant selection, data collection, and analysis.

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Participant Safety

Protecting the well-being, rights, and safety of individuals involved in clinical trials.

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Good Clinical Practice (GCP)

International standards for designing, conducting, documenting, and reporting clinical trials.

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Scientific Merit

The importance and quality of the research question and study design.

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Risk-Benefit Assessment

Weighing the potential risks and benefits of a clinical trial protocol to participants.

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International Harmonisation (ICH)

Global standard-setting for clinical trials, unifying regulations across countries

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PBS Listing Requirements

Medicines must demonstrate safety, efficacy, and cost-effectiveness to be listed on the Pharmaceutical Benefits Scheme.

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Cost-Effective Medicine

A medicine that provides sufficient health benefit relative to its cost.

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TGA Registration

The process by which the Therapeutic Goods Administration (TGA) approves the safety and efficacy of a medicine for use in Australia.

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Section 19A

A section in the Therapeutic Goods Act enabling temporary approval for unregistered medicines in specific situations, like emergency or compassionate access.

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SSSI (Serious Scarcity Substitution Instrument)

Allows substitution of Schedule 4 medications with lower/higher strength, IR/SR, or different salts when needed.

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Personal Importation

Importing unapproved medicines for personal use, or use by immediate family members, under specific criteria.

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Special Access Scheme

A pathway for accessing medicines not listed on the PBS for those with specific medical needs.

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National Medicines Policy

A framework promoting quality medicine use, involving multiple stakeholders (government, industry etc) focusing on quality and safety and access through the PBS.

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Compassionate Use

A program allowing non-Medicare card holders to access HIV medication, often unavailable or unaffordable otherwise.

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Extended Access Programs

Provide access to drugs exiting clinical trials, before official registration.

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Product Familiarisation Programs

Offer access to drugs awaiting PBS registration.

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Cost Share Programs

Allow access to medication at reduced cost.

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HIPPOCRATIC Oath

Physician's oath emphasizing "first do no harm."

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Autonomy

A patient's right to self-determination in treatment decisions.

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Beneficence

Duty to do good; ethical principle of helping others.

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Non-Maleficence

Duty to avoid harming others; ethical principle.

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Multiple Ascending Dose Scheme

A clinical trial design where patients receive increasing drug doses, moving through cohorts (groups) until adverse effects appear or a maximum tolerated dose (MTD) is reached

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Phase II Study

A clinical trial phase focused on exploring a drug's effectiveness and optimal dose in a specific patient population.

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Phase III Study

Large-scale clinical trials comparing a new treatment to a standard treatment or placebo, testing hypothesis differences.

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Maximum Tolerated Dose (MTD)

The highest dose of a drug a patient can tolerate without experiencing unacceptable side effects.

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Randomized Controlled Trial (RCT)

A clinical trial design where participants are randomly assigned to different treatment groups (e.g., experimental drug or placebo).

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Gold Standard Comparator

An existing, well-established treatment used as a benchmark to compare the effectiveness of a new drug in a Phase III trial.

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Refractory Patients

Patients who have not responded to prior treatments and are considered for participation in a clinical trial (Phase II).

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Therapeutic Exploratory

Focus of Phase II studies where effectiveness and optimal dose is explored.

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Clinical Equipoise

The ethical principle in clinical trials stating that no clear treatment superiority exists at the trial's start.

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Clinical Trials Location

Clinical trials are conducted in various locations, often involving multiple centers, and typically span multiple years. Data and safety checks are regularly performed.

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RCT Ethical Considerations

Ethical dilemmas in RCTs can arise from treatment design choices, such as deciding whether to include standard-of-care options or new drugs, like in the New York Times example.

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Trial Design Influence

The design and conduct of clinical trials should respect ethical principles like equipoise and risk-benefit assessments.

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Equipoise Maintenance

Clinical equipoise needs to be maintained throughout a trial's duration, especially in large trials. Regular data and safety monitoring are crucial to stop ineffective or unsafe treatments early.

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ICH GCP

International Conference on Harmonisation - Good Clinical Practice guidelines, designed for global clinical trials, emphasizing ethical conduct.

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Therapeutic Goods Act 1989

Australian law requiring HREC review and monitoring of unregistered therapeutic goods clinical trials.

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HREC

Human Research Ethics Committee, reviewing clinical trial protocols for ethical compliance.

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Clinical Trial Sponsor

Individual or organization financing and overseeing clinical trial procedures, approaching clinicians for investigations.

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Clinical Trial Investigator

Clinicians who conduct clinical trials at a site, often with sub-investigators.

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Ethics Committee Review

Process where clinical trials are reviewed by an ethics committee for ethical soundness and participant safety.

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Unregistered Drug Promotion

Promoting trial drugs not registered in Australia, requiring HREC approval for advertisement.

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Pharmacy Role in Trials

Pharmacy oversees medication management in hospitals, ensuring adherence to regulations and labeling.

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Ethical Research

Research conducted with careful consideration for the well-being of participants, incorporating informed consent, voluntary participation, and provision of adequate information.

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Informed Consent

A process where participants in a study completely understand the research, including risks and benefits, and freely choose to participate.

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Placebo

An inactive substance or treatment used as a control group in a clinical trial, comparing it to the experimental treatment.

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Randomisation

Assigning participants to different treatment groups using a random process, minimizing pre-existing biases.

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Blinding

A process of keeping study participants and/or researchers unaware of participant group assignments to prevent bias.

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Clinical Equipoise

The ethical principle in clinical trials, ensuring no pre-existing belief about the superiority of one treatment over another at the initiation of the trial.

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Ethical Trial Design

A clinical trial design that balances producing valuable data with minimizing harm to participants.

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Level of Evidence

Different types of research studies vary in strength of proving effectiveness, with meta-analyses of randomized controlled trials as the strongest.

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Medication Access Pathways

The routes for obtaining medications, including registered options, special access schemes, and clinical trials in Australia.

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TGA Registered Medications

Medicines that are approved by the Therapeutic Goods Administration (TGA) for specific uses, considered safe and effective.

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Special Access Scheme

A program allowing access to unregistered medicines for patients with specific medical needs.

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PBS Listing

Process where a medicine is included in the Pharmaceutical Benefits Scheme, making it subsidised for patients under specific criteria.

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New Drug Entity Introduction

The steps involved in bringing a new medicine to clinical use, from initial research to clinical trials and eventual registration.

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Clinical Trials

Research studies testing new treatments and their effectiveness in patients.

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Authorised Prescriber

A type of Doctor or specialist who can prescribe medications under certain circumstances, outside of standard medical practice.

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Registered Medications

Medicines recognized as safe and effective by the TGA.

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Multiple Ascending Dose Scheme

A clinical trial design where patients receive increasing doses of a drug, moving through cohorts until an adverse effect or maximum tolerated dose (MTD) is reached.

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Phase II Study

A clinical trial phase focused on exploring a drug's effectiveness and optimal dose in a specific patient population.

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Phase III Study

Large-scale clinical trials comparing a new treatment to a standard treatment or placebo, testing hypothesis differences.

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Maximum Tolerated Dose (MTD)

The highest dose of a drug a patient can tolerate without experiencing unacceptable side effects.

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Randomized Controlled Trial (RCT)

A clinical trial design where participants are randomly assigned to different treatment groups (e.g., experimental drug or placebo).

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Gold Standard Comparator

An existing, well-established treatment used as a benchmark to compare the effectiveness of a new drug in a Phase III trial.

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Refractory Patients

Patients who have not responded to prior treatments and are considered for participation in a clinical trial (Phase II).

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Therapeutic Exploratory

Focus of Phase II studies where effectiveness and optimal dose is explored.

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Randomisation

Assigning participants to different groups (treatment or control) randomly to reduce bias.

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Blinding

Keeping participants and/or researchers unaware of group assignments to prevent bias.

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Double-blind

Both participants and researchers are unaware of the treatment assignment.

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Gold standard (RCT)

A randomized controlled trial (RCT) that is considered the strongest evidence in clinical trials.

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Crossover design

A trial design where participants receive different treatments in a specific order, with washout periods in between.

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Parallel design

A clinical trial design in which participants are randomly assigned to different groups and remain in those groups throughout the study.

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RCT

Randomized controlled trial: A clinical trial type that uses randomization and control groups to compare different treatments or interventions.

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Trial design

The overall structure of a clinical trial, including factors like randomization, blinding, and the specific treatments and comparison groups involved.

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Safe Drug?

No drug is inherently safe; safety depends on how it's used and monitored.

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Ethics Committees

Independent groups reviewing research proposals, ensuring participant safety.

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HREC Role

Ethics committees responsible for approving human research protocols.

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Clinical Trial Oversight

Clinical trials require review and approval by an ethics committee.

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Multi-Site Trials

Trials conducted at multiple locations, often involving multiple ethics committees.

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Comparator

A treatment (either placebo or active) used to compare against a new treatment in a clinical trial.

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Placebo

An inactive substance or treatment used as a control in a clinical trial. It looks like the active treatment in every way.

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Active Control

An established treatment with proven efficacy used as a comparison in a clinical trial when a placebo is unethical.

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Placebo Effect

A psychological or physical response to a fake treatment, often due to hope or belief.

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Unblinded Trial

A clinical trial where knowledge of treatment assignments is known to some or all parties involved.

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Unblinded Pharmacy Staff

Pharmacy staff are often unblinded in a clinical trial, meaning they know the treatment type for the patient.

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Ethical Considerations (HIV)

It's unethically to withhold treatment for HIV, so an active comparator is used, making sure the new treatment is proven better than the existing one.

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Unethical Treatment

Withholding treatment from a patient when it could cause irreversible harm is unethical in a clinical trial, often resorting to an active control group treatment.

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ICH GCP

International standards for designing, conducting, documenting, and reporting clinical trials, ensuring ethical conduct worldwide.

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Therapeutic Goods Act 1989

Australian law requiring Human Research Ethics Committees (HRECs) to review and monitor all clinical trials of unregistered therapeutic goods.

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HREC

Human Research Ethics Committees that review clinical trial protocols to ensure ethical compliance in Australia.

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Clinical Trial Sponsor

The individual or organization funding and overseeing a clinical trial, often approaching clinicians for the study.

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Clinical Trial Investigator

Clinicians who conduct a clinical trial, usually at a specific location.

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Ethics Committee Review

The process of reviewing clinical trial protocols for ethical soundness and participant safety, ensuring compliance with ethical guidelines.

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Unregistered Drug Promotion

Promoting trial drugs not officially registered in Australia. Promotion requires HREC approval.

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Pharmacy Role in Trials

Pharmacy manages medications in hospitals, ensuring compliance with regulations during clinical trials.

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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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PHARM4813 Lecture Notes - Clinical Trials PDF

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.

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