Novel Therapeutics Regulation Quiz
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Questions and Answers

What is the first step in the prescription medicines registration process?

  • Submission of dossier
  • Manufacturing process (correct)
  • Pre-clinical data
  • Clinical data

    • Which schedule categorizes controlled drugs in the medicine scheduling system?

  • Schedule 6
  • Schedule 2
  • Schedule 8 (correct)
  • Schedule 9

    • Which type of data is required after the product development phase in the registration process for prescription medicines?

  • Import/export documentation
  • Clinical data (correct)
  • Cautionary data
  • Market analysis

    • What does ARTG stand for in the context of therapeutic goods?

    <p>Australian Register of Therapeutic Goods</p> Signup and view all the answers

    What type of submission guidelines must a sponsor follow for the registration process?

    <p>Australian and international guidelines</p> Signup and view all the answers

    Which component is NOT part of the dossier preparation for prescription medicines?

    <p>Market forecast</p> Signup and view all the answers

    What is a key requirement before therapeutic goods can be supplied or imported in Australia?

    <p>Entry on the ARTG</p> Signup and view all the answers

    Which of the following is TRUE regarding the regulatory submission process?

    <p>Only completed applications are accepted.</p> Signup and view all the answers

    What is typically required for the scheduling of prescription medicines?

    <p>Obtaining GMP certification</p> Signup and view all the answers

    What constitutes the final step in the submission basics for prescription medicines?

    <p>Submit dossier and fees</p> Signup and view all the answers

    What is the maximum allowed extension period for Provisional Approval under the Standard PM Pathway?

    <p>6 months</p> Signup and view all the answers

    Which of the following paths allows for expedited review under the TGA guidelines?

    <p>Provisional Approval Pathway</p> Signup and view all the answers

    What is the TGA's target timeframe for a Priority Review application?

    <p>150 working days</p> Signup and view all the answers

    How many phases does the Standard PM Pathway consist of?

    <p>8 phases</p> Signup and view all the answers

    Which option is NOT a facilitated pathway available under the TGA evaluation process?

    <p>Parallel Submission</p> Signup and view all the answers

    What triggers the TGA clock to stop during the submission evaluation process?

    <p>Sponsor's response to queries</p> Signup and view all the answers

    In the context of regulatory submissions, what does COR-A and COR-B refer to?

    <p>Consultation Outcomes Reports</p> Signup and view all the answers

    Which country is included under the Work-sharing arrangement for TGA submissions?

    <p>Brazil</p> Signup and view all the answers

    What milestone signifies the end of the planning phase in the submission evaluation process?

    <p>Milestone 1</p> Signup and view all the answers

    Which pathway has no provision for extension beyond its initial evaluation period?

    <p>Priority Review</p> Signup and view all the answers

    What is the estimated time frame to develop a new pharmaceutical product?

    <p>6-11 years</p> Signup and view all the answers

    Which step comes first in the traditional pharmaceutical development pathway?

    <p>Drug Discovery</p> Signup and view all the answers

    What legislative framework governs the regulation of therapeutic goods in Australia?

    <p>Therapeutic Goods Act, 1989</p> Signup and view all the answers

    What is the purpose of Good Manufacturing Practice (GMP)?

    <p>To establish licensing requirements for manufacturers</p> Signup and view all the answers

    During the Clinical Development phase, what is primarily assessed?

    <p>Drug efficacy and safety</p> Signup and view all the answers

    What is included in the Health Technology Assessment strategy?

    <p>Evaluating the cost-effectiveness of products</p> Signup and view all the answers

    Which of the following is NOT a function of the Therapeutic Goods Administration (TGA)?

    <p>Conducting clinical trials for new drugs</p> Signup and view all the answers

    What precedes the Regulatory Submission in the pharmaceutical development process?

    <p>Clinical Trials</p> Signup and view all the answers

    What type of product is classified as a therapeutic good?

    <p>Therapeutic devices</p> Signup and view all the answers

    What does the acronym GCP stand for in the context of pharmaceuticals?

    <p>Good Clinical Practice</p> Signup and view all the answers

    What is a key component of the Product Development Plan?

    <p>Competitor analysis</p> Signup and view all the answers

    What is the final step before a product launch in the medical device development pathway?

    <p>Post-market compliance</p> Signup and view all the answers

    What does pharmacovigilance (PV) involve?

    <p>Monitoring and evaluating drug safety</p> Signup and view all the answers

    In the context of regulatory submission, what does ARTG stand for?

    <p>Australian Register of Therapeutic Goods</p> Signup and view all the answers

    What is the primary purpose of an in vitro diagnostic (IVD) medical device?

    <p>To provide information about a specimen derived from the human body</p> Signup and view all the answers

    Which document is NOT required for the inclusion of a medical device?

    <p>Clinical trial report</p> Signup and view all the answers

    What is the first step in the TGA regulatory process for IVD devices?

    <p>Identify the class of IVD</p> Signup and view all the answers

    What ligand is associated with the TrkA receptor?

    <p>NGF</p> Signup and view all the answers

    Which of the following is a characteristic of an IVD medical device?

    <p>It provides patient information for therapeutic measures</p> Signup and view all the answers

    Which receptor binds to neurotropin 3?

    <p>TrkC</p> Signup and view all the answers

    Which of the following steps is involved in the submission process for IVD devices to TGA?

    <p>Prepare a Declaration of Conformity</p> Signup and view all the answers

    What is the role of LOXO-101 in cancer treatment?

    <p>Inhibits tumor growth</p> Signup and view all the answers

    What must a manufacturer provide to support compliance with Essential Principles for IVD devices?

    <p>Evidence of compliance documentation</p> Signup and view all the answers

    Which is NOT a intended use for in vitro diagnostic medical devices?

    <p>Providing clinical trial results</p> Signup and view all the answers

    Which factor is associated with TrkB receptor activity?

    <p>BDNF/NTF4</p> Signup and view all the answers

    What does the acronym ARTG stand for in the context of medical devices?

    <p>Australian Register of Therapeutic Goods</p> Signup and view all the answers

    In which context was the inhibition of cancer cells by LOXO-101 investigated?

    <p>Colorectal xenograft model</p> Signup and view all the answers

    What type of study design did the research on NTRK fusion focus on?

    <p>Case report</p> Signup and view all the answers

    What is the primary function of the TRK signaling pathway?

    <p>Cell proliferation and survival</p> Signup and view all the answers

    Which receptor is NOT associated with the TRK signaling pathways?

    <p>TrkE</p> Signup and view all the answers

    Which statement correctly describes a genetically modified organism (GMO)?

    <p>A GMO is an organism that has inherited modified traits from a modified organism.</p> Signup and view all the answers

    What type of dealings with GMOs does not require a license?

    <p>Knockout mice transactions</p> Signup and view all the answers

    Which of the following therapeutic goods requires a license from the OGTR?

    <p>GM crops</p> Signup and view all the answers

    Which gene therapy product is delivered ex vivo?

    <p>Kymriah (tisagenlecleucel)</p> Signup and view all the answers

    Which of the following is considered a notifiable low-risk dealing?

    <p>Lab-based experiments</p> Signup and view all the answers

    What is the classification for gene therapy that does not involve intentional release into the environment?

    <p>License - does not involve intentional release (DNIR)</p> Signup and view all the answers

    Which of the following is an emergency dealing determination?

    <p>Equine influenza vaccine</p> Signup and view all the answers

    Which combination of gene therapy products is administered in vivo?

    <p>Zolgensma and Hemgenix</p> Signup and view all the answers

    What is a requirement for adult patients to be eligible for larotrectinib (Vitrakvi) treatment?

    <p>They must have locally advanced or metastatic solid tumors with an NTRK gene fusion.</p> Signup and view all the answers

    Which of the following tumour types is NOT included in the Phase 2 NAVIGATE study?

    <p>Infantile fibrosarcoma</p> Signup and view all the answers

    Which of the following testing methods is used to determine the presence of NTRK gene fusions?

    <p>Fluorescence in-situ hybridisation (FISH)</p> Signup and view all the answers

    What is the minimum age requirement for patients eligible for the SCOUT study?

    <p>1 month</p> Signup and view all the answers

    What defines a biological product according to the provided information?

    <p>A product that is used to test a person's susceptibility to disease.</p> Signup and view all the answers

    Which dosing schedule is used in the Phase 1/2 SCOUT study?

    <p>100 mg/m² twice daily</p> Signup and view all the answers

    What does MSAC stand for in the context of medical test recommendations?

    <p>Medical Services Advisory Committee</p> Signup and view all the answers

    In which PBAC meeting was the recommendation for larotrectinib treatment made?

    <p>March 2022</p> Signup and view all the answers

    Which region has granted provisional approval for Vitrakvi for adult and paediatric patients?

    <p>Australia</p> Signup and view all the answers

    What specific condition must be met for paediatric patients to qualify for treatment with a NTRK inhibitor?

    <p>Presence of a neurotrophic tropomyosin receptor kinase (NTRK) gene fusion.</p> Signup and view all the answers

    What is the primary focus of the studies mentioned in the content?

    <p>Tumours harbouring NTRK gene fusion</p> Signup and view all the answers

    Which tumour type has the highest number of patients in the Phase 2 NAVIGATE study?

    <p>Thyroid</p> Signup and view all the answers

    Which type of cancers are specifically referenced as eligible for NTRK fusion testing in adults?

    <p>Thyroid cancer and colorectal cancer.</p> Signup and view all the answers

    What is the role of next-generation sequencing (NGS) in companion diagnostic testing?

    <p>To determine eligibility for larotrectinib by detecting gene fusions.</p> Signup and view all the answers

    What is the maximum dosage used in the Phase 1 NCT02122913 study?

    <p>200 mg</p> Signup and view all the answers

    Which of the following combinations represents a common demographic in the studies listed?

    <p>Adult and paediatric patients with solid tumours</p> Signup and view all the answers

    What is not a function of a biological product?

    <p>To manufacture industrial chemicals.</p> Signup and view all the answers

    Which of the following patients is excluded from the eligibility criteria for the studies discussed?

    <p>Patients under 1 month old</p> Signup and view all the answers

    What is the primary mechanism of action for Vitrakvi?

    <p>Kinase inhibitor</p> Signup and view all the answers

    What major event in 1962 led to strengthened regulatory requirements in the pharmaceutical industry?

    <p>Thalidomide disaster</p> Signup and view all the answers

    Which of the following therapies was first approved in 2017?

    <p>CAR-T cell therapy</p> Signup and view all the answers

    What is a significant trend in current pharmaceutical industry practices?

    <p>Use of mRNA-based therapies</p> Signup and view all the answers

    Which drug was the top-selling medicine globally in 2022?

    <p>Comirnaty</p> Signup and view all the answers

    What does the TRIPS agreement relate to in the pharmaceutical context?

    <p>International trade and intellectual property</p> Signup and view all the answers

    Which drug had the highest cost to the Australian government during 2022-23?

    <p>Lagevrio</p> Signup and view all the answers

    How much does it typically cost to bring a new product to market?

    <p>$50-500 million</p> Signup and view all the answers

    What does 'precision medicine' aim to accomplish in healthcare?

    <p>Tailored therapies based on individual genetics</p> Signup and view all the answers

    Which is a key factor in current pharmaceutical corporate policies?

    <p>Increased transparency in corporate actions</p> Signup and view all the answers

    Which of the following therapies was first approved in 2015?

    <p>Spritam</p> Signup and view all the answers

    What is the focus of the pandemic treaty being discussed by the WHO?

    <p>Sovereign manufacturing capabilities</p> Signup and view all the answers

    Which of the following is an example of a biotechnology that impacts pharmaceutical health?

    <p>Genomic sequencing</p> Signup and view all the answers

    What was a pivotal regulatory change introduced in 1906?

    <p>Implementation of the Food, Drug, and Cosmetic Act</p> Signup and view all the answers

    Which therapeutic area is currently gaining importance in the pharmaceutical industry?

    <p>Cell and gene therapy</p> Signup and view all the answers

    What class of therapeutic goods includes drugs that require a prescription before they can be obtained?

    <p>Prescription medicines</p> Signup and view all the answers

    Which schedule in medicine scheduling indicates 'Caution' for the substances listed?

    <p>Schedule 5</p> Signup and view all the answers

    What is required before a therapeutic good can be supplied in or exported from Australia?

    <p>Inclusion on ARTG</p> Signup and view all the answers

    Which of the following is NOT a type of data required during the registration process for prescription medicines?

    <p>Market survey data</p> Signup and view all the answers

    What does the term 'dossier' refer to in the context of the registration process?

    <p>A compilation of documentation</p> Signup and view all the answers

    Which type of medicines can only be dispensed under the supervision of a pharmacist?

    <p>Schedule 3</p> Signup and view all the answers

    What is the primary focus of the risk-based approach to regulation of therapeutic goods?

    <p>To tailor oversight based on potential risk</p> Signup and view all the answers

    Which step comes immediately after writing AU-specific designations in the submission basics?

    <p>Publish dossier</p> Signup and view all the answers

    What classification encompasses substances deemed dangerous poisons?

    <p>Schedule 7</p> Signup and view all the answers

    Which category of therapeutic goods includes vaccines and gene therapy products?

    <p>Biologicals</p> Signup and view all the answers

    Which criteria must a medicine satisfy to qualify for the Priority Review designation?

    <p>Must lack registered competitors</p> Signup and view all the answers

    What is the maximum validity period for an orphan designation application?

    <p>6 months with one extension up to 6 months</p> Signup and view all the answers

    What distinguishes the Provisional Approval process from the Priority Review?

    <p>Requires submission of comprehensive clinical data</p> Signup and view all the answers

    Which of the following is a key criterion for a medicine to qualify for Provisional Approval?

    <p>Must demonstrate major therapeutic advance</p> Signup and view all the answers

    Which option correctly describes the pre-submission meeting recommendation for standard applications?

    <p>Optional only for complex applications</p> Signup and view all the answers

    What is the role of rolling data submission in the regulatory process?

    <p>Permits ongoing updates without a full resubmission</p> Signup and view all the answers

    Which of the following accurately represents the consolidated TGA request for information timeframe?

    <p>Sponsor commits to respond within 30 or 60 days</p> Signup and view all the answers

    What is the defining feature of a medical device according to the provided definition?

    <p>It can include diagnostic tools and software</p> Signup and view all the answers

    During which phase is the application lodged for a Priority Review designation?

    <p>Post-pre-submission meeting</p> Signup and view all the answers

    Which is NOT a valid reason for a medicine's eligibility under the orphan designation?

    <p>Significant financial viability concerns</p> Signup and view all the answers

    What classification does Kymriah (tisagenlecleucel) fall under?

    <p>Biological</p> Signup and view all the answers

    Which regulatory pathway is used for in vivo gene therapy?

    <p>Prescription medicine pathway</p> Signup and view all the answers

    When was Zolgensma (onasemnogene abeparvovec) first registered in the ARTG?

    <p>24 August 2020</p> Signup and view all the answers

    What type of therapy does Yescarta (axicabtagene ciloleucel) represent?

    <p>Ex vivo cell therapy</p> Signup and view all the answers

    Which of the following products requires an OGTR license for supply?

    <p>All of the above</p> Signup and view all the answers

    What is the therapeutic focus of Hemgenix (etranacogene dezaparvovec)?

    <p>Hematological diseases</p> Signup and view all the answers

    Which product is classified as a Class 4 biological?

    <p>Both A and B</p> Signup and view all the answers

    Which document indicates that a medical device complies with Essential Principles?

    <p>Declaration of Conformity</p> Signup and view all the answers

    What innovation is associated with future directions in therapeutic product development?

    <p>3D printing</p> Signup and view all the answers

    What is the primary intended use of an in vitro diagnostic (IVD) medical device?

    <p>Monitoring physiological states from human specimens</p> Signup and view all the answers

    Which gene therapy product is not classified under prescription medicines?

    <p>Luxturna</p> Signup and view all the answers

    What is the first step in the regulation process for in vitro diagnostic (IVD) medical devices by the TGA?

    <p>Identify the class of IVD</p> Signup and view all the answers

    Which gene therapy product did not receive commercial supply approval in 2020?

    <p>Yescarta</p> Signup and view all the answers

    Which of the following is NOT considered an in vitro diagnostic (IVD) medical device?

    <p>Device used solely in surgical procedures</p> Signup and view all the answers

    What step follows the preparation and submission of TGA conformity assessment?

    <p>Submit application for inclusion</p> Signup and view all the answers

    Which document must a manufacturer obtain to guarantee compliance with the IVD regulations?

    <p>Declaration of Conformity</p> Signup and view all the answers

    Which of the following describes an essential characteristic of an IVD medical device?

    <p>It must provide information on pathological conditions</p> Signup and view all the answers

    What is one of the purposes of an in vitro diagnostic (IVD) medical device?

    <p>Examine samples for potential safety and compatibility</p> Signup and view all the answers

    What is the primary ligand associated with the TrkC receptor?

    <p>NTF3</p> Signup and view all the answers

    Which inhibitor is noted for its effect on cancer cells possessing oncogenic TRK?

    <p>LOXO-101</p> Signup and view all the answers

    In which type of cancer was an oncogenic NTRK fusion response to LOXO-101 observed?

    <p>Soft-tissue sarcoma</p> Signup and view all the answers

    Which signaling pathway is involved with the TrkA receptor?

    <p>TRK signaling pathway</p> Signup and view all the answers

    What is the role of BDNF in relation to TrkB?

    <p>It acts as a ligand for TrkB.</p> Signup and view all the answers

    What type of model was used to investigate LOXO-101's effects on tumor growth?

    <p>KM12 colorectal xenograft model</p> Signup and view all the answers

    Which receptor is linked to the neurotrophin known as NGF?

    <p>TrkA</p> Signup and view all the answers

    Which neurotrophic factor is linked to TrkB and recognized for its role in neural development?

    <p>BDNF</p> Signup and view all the answers

    What is the typical duration for drug discovery and development according to the traditional pharmaceutical development pathway?

    <p>6-11 years</p> Signup and view all the answers

    Which phase immediately follows clinical trials in the pharmaceutical development pathway?

    <p>Marketing Approval</p> Signup and view all the answers

    What does Good Laboratory Practice (GLP) pertain to within pharmaceutical development?

    <p>Standards for laboratory studies</p> Signup and view all the answers

    Which step is included in the medical devices development pathway after verification and validation?

    <p>Regulatory Submissions</p> Signup and view all the answers

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

    <p>Pre-market evaluation and approval</p> Signup and view all the answers

    Which of the following is NOT a component of the Product Development Plan?

    <p>Post-Market Surveillance</p> Signup and view all the answers

    Which practice focuses on monitoring the safety of medicines post-market?

    <p>Pharmacovigilance (PV)</p> Signup and view all the answers

    What type of goods falls under the regulation of the Therapeutic Goods Act?

    <p>Products intended for therapeutic use</p> Signup and view all the answers

    What is a principal aspect of the Health Technology Assessment strategy?

    <p>Evaluating drug efficacy and safety</p> Signup and view all the answers

    Which aspect does NOT fall under the regulatory requirements established by the Therapeutic Goods Act?

    <p>Setting drug pricing policies</p> Signup and view all the answers

    What is a key role of the TGA regarding unapproved medicines?

    <p>Establish assessment processes for access</p> Signup and view all the answers

    What stage occurs right before marketing approval in the pharmaceutical development pathway?

    <p>Clinical Trials</p> Signup and view all the answers

    Which of the following is described as a component of the regulatory submission process?

    <p>Dossier preparation</p> Signup and view all the answers

    Which regulation governs the licensing of manufacturers in Australia?

    <p>Therapeutic Goods Act</p> Signup and view all the answers

    Study Notes

    Course Information

    • Course code: PHAR4813
    • Course title: Novel Therapeutics - Regulation
    • Instructor: Dr Orin Chisholm
    • University: The University of Sydney

    Learning Outcomes

    • LO3: Demonstrate awareness of current biotechnologies and their impact on pharmaceutical health
    • LO4: Explain the principles involved in drug discovery and the decision-making process involved in developing drugs

    History of the Pharma Industry

    • 1859: First factory producing medicines (Beecham)
    • 1962: Thalidomide disaster - Safety and efficacy regulations strengthened
    • 1985: Recombinant human insulin registered in the US
    • 1995: TRIPS agreement (20-year patent term)
    • 1906: US Food, Drug and Cosmetic Act
    • 1976: Genentech founded
    • 1990: First HIV reverse transcriptase approved
    • 2000: Human genome sequenced
    • 2012: First gene therapy product (Glybera) approved
    • 2015: First 3D-printed tablets approved
    • 2017: First CAR-T cell therapy (Kymriah) approved
    • 2021: First mRNA vaccines (Comirnaty, Spikevax) approved

    Current Directions and Influences

    • Science: Oncology, rare diseases, cell & gene therapy, mRNA-based therapies, AI in drug development, precision medicine & data
    • Policies: WHO - universal health coverage, future pandemic preparedness, sovereign manufacturing capability, industrial & innovation, health funding, regulatory science, research and export/trade
    • Corporate: VALUE, ESG and climate change impacts, EDI, increasing patient voice, new ways of working, digital integration, codes of conduct, ethics, transparency, business & corporate policies

    Top Pharmaceutical Markets (2022)

    • United States: 42.6%
    • China: 7.6%
    • Japan: 4.5%
    • Germany: 4%
    • France: 2.8%
    • Italy: 2.4%
    • United Kingdom: 2.3%
    • Canada: 2%
    • Spain:1.9%
    • Brazil: 1.9%

    Top Therapeutic Classes by Sales (2023)

    • Anticancer, immunological: 4,492
    • Anticancer, other: 3,622
    • Gene therapy: 2,083
    • Other classes also listed in descending order by number of products

    Top MedTech Market Segments (2021)

    • Data shows distribution of global revenue of MedTech industry in 2021, by product type (e.g., Cardiovascular, Medical Imaging, Orthopedics). Specific segments and their percentage of revenue are detailed.

    FDA-CDER Annual Novel Drug Approvals (2023)

    • Data presented as a graph showing annual approvals of new drugs. Also includes a pie chart of the breakdown of First-in-Class versus other types of drug approvals

    Top 10 Drugs by Sales (2022)

    • Lists top 10 global drugs by revenue (in USD billion) with details of the drugs, indication, and companies responsible.

    Top PBS Drugs by Cost to Australian Government (2022-23)

    • A table listing the most expensive drugs on the Pharmaceutical Benefits Scheme (PBS) in Australia, with the cost to the government for each, sorted by cost from most to least.

    Expenses and Revenues Curve for a New Medicine

    • Graph depicting the typical expenses and revenues of a new medication over time. It illustrates stages of investment, product uptake and the return period, and how expenditures can outpace revenues initially.

    Cost to Bring a New Product to Market

    • Estimated costs range between $1-3 billion.

    Time to Bring a New Product to Market

    • Typical time frame is (6-11) years.

    Pharmaceutical Traditional Development Pathway

    • The correct order for development steps: Drug Discovery & Pre-clinical Studies -> Clinical Trials -> Marketing, PV, Further Clinical Studies -> Registration -> Reimbursement.

    Product Development Plan

    • Key components of a product development plan (e.g., situational analysis, competitor landscape, target product profile, regulatory strategy, health technology assessment strategy, timelines).

    Medical Devices Development Pathway

    • Steps involved in the development pathway of medical devices. Stages include: Initiation, Formulation, Design and Development, Verification and Validation, Manufacturing and Testing, Clinical Development, Regulatory Submissions, Launch, Post-Market Compliance, Reimbursement
    • A hierarchy of laws regulating medicines in Australia: Laws, Regulations, Orders, Guidances and Standards underpin and are supported by these.

    Australian Legislation

    • Therapeutic Goods Act (1989), Therapeutic Goods Regulations (1990) and Therapeutic Goods Orders (various).
    • Australian Regulatory Guidelines (Prescription Medicines, Medical Devices, Biologicals).
    • Adherence to International guidelines

    What the Therapeutic Goods Act Does

    • Establishes the TGA and advisory committees, regulates advertising, licenses and inspects manufacturers, establishes ARTG, post-market surveillance and testing of medical products, assessment of medicines for export and establishes processes for access to unapproved medicines.

    Therapeutic Goods Administration (TGA)

    • Responsibilities: Pre-market evaluation and approval of therapeutic goods, licensing of manufacturers, post-market surveillance, development/monitoring systems, and assessment for export

    Definition of a Therapeutic Good

    • Definition of therapeutic goods as per TGA

    Classes of Therapeutic Goods

    • Classes of Therapeutic goods include: prescription medicines, over-the-counter medicines, complementary medicines, sunscreens, medical devices, biologicals, and blood and blood components.

    Risk-based approach to regulation

    • Classifying medical products based on the risk level (low, medium, high)
    • Higher risk products require more rigorous regulatory oversight.

    Medical Device Classification

    • Medical equipment with their respective classifications (Class I, Class IIa, Class IIb, Class III), according to risk level

    Essential Principles

    • General principles, design and construction standards, labelling, standards and testing procedures.

    Conformity Assessment Procedures

    • Help demonstrate compliance with the Essential Principles by utilizing Quality Management System (ISO 13485) requirements.

    Supplying Medical Devices in Australia

    • Steps involved in supplying medical devices: Confirm your product is a medical device, determine the type and class, prepare documents for inclusion, Submit application for inclusion, print ARTG certificate, and finally supply the device.

    In-vitro Diagnostic (IVD) Medical Device Definition

    • Specific definition to describe in-vitro diagnostic medical devices (e.g., reagents, calibrators, control materials, equipment).

    IVD Regulation By TGA

    • TGA's classification of IVD’s based on factors such as the level and type of potential risk

    Case Study

    • Case study data detailed about a medication (larotrectinib). The study describes a timeline of the clinical development process for larotrectinib and entrectinib

    TRK signaling pathways

    • Illustrates how TRK receptors work to signal biological responses. (This is displayed with a diagram).

    Non-clinical studies

    • Data displayed as graphs about non-clinical studies

    Clinical Studies

    • Overview of various clinical studies including the study design, target patient populations, dosages, number of participants, and tumor types.

    Regulatory Milestones

    • A timeline of key regulatory events (e.g., US, EU, AU, Japan, China) for Larotrectinib and Entrectinib's approvals, and companion diagnostics.

    Co-dependent health technology

    • Describes co-dependent health technologies. Conditions and guidelines, e.g. PBAC guidelines

    Reimbursement - PBS/MBS

    • Criteria for reimbursement for new treatments, such as larotrectinib (Vitrakvi).

    Biologicals

    • Describes what a biological is, risk groupings, and the application process for these products in class 1, 2, or 4.

    Application process

    • Details the process for each class of biological

    Cell Therapies

    • An summary of cell therapies and their ongoing clinical trials by phase and region

    Genetically Modified Organisms (GMOs)

    • Information on regulatory integration of GMOs in Australia. Covers the involvement of agencies like Biosecurity Australia, TGA, APVMA, OGTR, and AICIS

    Gene Technology (GT) Act 2000

    • Details of the GT Act 2000, and its mirroring in State/Territory Acts; a national scheme to regulate GMOs, and the User’s guide

    What is a GMO?

    • Definition of GMO; and examples of items that are not considered GMO

    What are dealings with GMOs?

    • Definition to explain different dealings related to GMOs

    GMO Authorisations

    • Notifiable low risk and High risk GMO dealings defined; and example products for each category

    Cell and Gene Therapy Products

    • Details of the processes involved in delivering gene therapy: ex-vivo, and in-vivo

    Regulatory Pathways

    • Describes the regulatory pathways for gene therapy (ex vivo and in vivo), giving specific examples.

    Genetically modified human therapeutics

    • Summary on relevant documents showing the OGTR License, supply, dates, and ARTG start dates for specified therapeutic products.

    Class 4 Biological Cell Therapies - Examples

    • Lists examples of class 4 biological cell therapies (Kymriah, Yescarta, Tecartus, Carvykti), and their first ARTG entry dates.

    Future Directions

    • Outlines potential future trends in the development of therapeutic products including precision medicine, cell therapies, gene therapies, 3D printing, brain-device interfaces, nanotechnology, synthetic biology, and Al-driven devices.

    Questions?

    • Invitation to ask questions

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    PHAR4813 Novel Therapeutics - Regulation (2024) Lecture PDF

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    Test your knowledge on the history and regulation of novel therapeutics in the pharmaceutical industry. This quiz will cover key milestones in drug discovery, biotechnology innovations, and the evolution of safety regulations. Prepare to demonstrate your understanding of the principles that shape pharmaceutical health today.

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