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Questions and Answers
What is the first step in the prescription medicines registration process?
Which schedule categorizes controlled drugs in the medicine scheduling system?
Which type of data is required after the product development phase in the registration process for prescription medicines?
What does ARTG stand for in the context of therapeutic goods?
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What type of submission guidelines must a sponsor follow for the registration process?
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Which component is NOT part of the dossier preparation for prescription medicines?
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What is a key requirement before therapeutic goods can be supplied or imported in Australia?
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Which of the following is TRUE regarding the regulatory submission process?
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What is typically required for the scheduling of prescription medicines?
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What constitutes the final step in the submission basics for prescription medicines?
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What is the maximum allowed extension period for Provisional Approval under the Standard PM Pathway?
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Which of the following paths allows for expedited review under the TGA guidelines?
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What is the TGA's target timeframe for a Priority Review application?
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How many phases does the Standard PM Pathway consist of?
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Which option is NOT a facilitated pathway available under the TGA evaluation process?
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What triggers the TGA clock to stop during the submission evaluation process?
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In the context of regulatory submissions, what does COR-A and COR-B refer to?
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Which country is included under the Work-sharing arrangement for TGA submissions?
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What milestone signifies the end of the planning phase in the submission evaluation process?
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Which pathway has no provision for extension beyond its initial evaluation period?
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What is the estimated time frame to develop a new pharmaceutical product?
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Which step comes first in the traditional pharmaceutical development pathway?
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What legislative framework governs the regulation of therapeutic goods in Australia?
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What is the purpose of Good Manufacturing Practice (GMP)?
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During the Clinical Development phase, what is primarily assessed?
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What is included in the Health Technology Assessment strategy?
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Which of the following is NOT a function of the Therapeutic Goods Administration (TGA)?
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What precedes the Regulatory Submission in the pharmaceutical development process?
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What type of product is classified as a therapeutic good?
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What does the acronym GCP stand for in the context of pharmaceuticals?
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What is a key component of the Product Development Plan?
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What is the final step before a product launch in the medical device development pathway?
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What does pharmacovigilance (PV) involve?
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In the context of regulatory submission, what does ARTG stand for?
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What is the primary purpose of an in vitro diagnostic (IVD) medical device?
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Which document is NOT required for the inclusion of a medical device?
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What is the first step in the TGA regulatory process for IVD devices?
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What ligand is associated with the TrkA receptor?
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Which of the following is a characteristic of an IVD medical device?
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Which receptor binds to neurotropin 3?
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Which of the following steps is involved in the submission process for IVD devices to TGA?
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What is the role of LOXO-101 in cancer treatment?
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What must a manufacturer provide to support compliance with Essential Principles for IVD devices?
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Which is NOT a intended use for in vitro diagnostic medical devices?
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Which factor is associated with TrkB receptor activity?
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What does the acronym ARTG stand for in the context of medical devices?
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In which context was the inhibition of cancer cells by LOXO-101 investigated?
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What type of study design did the research on NTRK fusion focus on?
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What is the primary function of the TRK signaling pathway?
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Which receptor is NOT associated with the TRK signaling pathways?
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Which statement correctly describes a genetically modified organism (GMO)?
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What type of dealings with GMOs does not require a license?
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Which of the following therapeutic goods requires a license from the OGTR?
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Which gene therapy product is delivered ex vivo?
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Which of the following is considered a notifiable low-risk dealing?
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What is the classification for gene therapy that does not involve intentional release into the environment?
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Which of the following is an emergency dealing determination?
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Which combination of gene therapy products is administered in vivo?
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What is a requirement for adult patients to be eligible for larotrectinib (Vitrakvi) treatment?
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Which of the following tumour types is NOT included in the Phase 2 NAVIGATE study?
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Which of the following testing methods is used to determine the presence of NTRK gene fusions?
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What is the minimum age requirement for patients eligible for the SCOUT study?
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What defines a biological product according to the provided information?
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Which dosing schedule is used in the Phase 1/2 SCOUT study?
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What does MSAC stand for in the context of medical test recommendations?
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In which PBAC meeting was the recommendation for larotrectinib treatment made?
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Which region has granted provisional approval for Vitrakvi for adult and paediatric patients?
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What specific condition must be met for paediatric patients to qualify for treatment with a NTRK inhibitor?
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What is the primary focus of the studies mentioned in the content?
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Which tumour type has the highest number of patients in the Phase 2 NAVIGATE study?
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Which type of cancers are specifically referenced as eligible for NTRK fusion testing in adults?
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What is the role of next-generation sequencing (NGS) in companion diagnostic testing?
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What is the maximum dosage used in the Phase 1 NCT02122913 study?
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Which of the following combinations represents a common demographic in the studies listed?
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What is not a function of a biological product?
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Which of the following patients is excluded from the eligibility criteria for the studies discussed?
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What is the primary mechanism of action for Vitrakvi?
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What major event in 1962 led to strengthened regulatory requirements in the pharmaceutical industry?
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Which of the following therapies was first approved in 2017?
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What is a significant trend in current pharmaceutical industry practices?
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Which drug was the top-selling medicine globally in 2022?
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What does the TRIPS agreement relate to in the pharmaceutical context?
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Which drug had the highest cost to the Australian government during 2022-23?
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How much does it typically cost to bring a new product to market?
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What does 'precision medicine' aim to accomplish in healthcare?
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Which is a key factor in current pharmaceutical corporate policies?
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Which of the following therapies was first approved in 2015?
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What is the focus of the pandemic treaty being discussed by the WHO?
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Which of the following is an example of a biotechnology that impacts pharmaceutical health?
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What was a pivotal regulatory change introduced in 1906?
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Which therapeutic area is currently gaining importance in the pharmaceutical industry?
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What class of therapeutic goods includes drugs that require a prescription before they can be obtained?
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Which schedule in medicine scheduling indicates 'Caution' for the substances listed?
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What is required before a therapeutic good can be supplied in or exported from Australia?
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Which of the following is NOT a type of data required during the registration process for prescription medicines?
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What does the term 'dossier' refer to in the context of the registration process?
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Which type of medicines can only be dispensed under the supervision of a pharmacist?
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What is the primary focus of the risk-based approach to regulation of therapeutic goods?
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Which step comes immediately after writing AU-specific designations in the submission basics?
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What classification encompasses substances deemed dangerous poisons?
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Which category of therapeutic goods includes vaccines and gene therapy products?
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Which criteria must a medicine satisfy to qualify for the Priority Review designation?
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What is the maximum validity period for an orphan designation application?
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What distinguishes the Provisional Approval process from the Priority Review?
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Which of the following is a key criterion for a medicine to qualify for Provisional Approval?
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Which option correctly describes the pre-submission meeting recommendation for standard applications?
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What is the role of rolling data submission in the regulatory process?
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Which of the following accurately represents the consolidated TGA request for information timeframe?
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What is the defining feature of a medical device according to the provided definition?
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During which phase is the application lodged for a Priority Review designation?
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Which is NOT a valid reason for a medicine's eligibility under the orphan designation?
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What classification does Kymriah (tisagenlecleucel) fall under?
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Which regulatory pathway is used for in vivo gene therapy?
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When was Zolgensma (onasemnogene abeparvovec) first registered in the ARTG?
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What type of therapy does Yescarta (axicabtagene ciloleucel) represent?
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Which of the following products requires an OGTR license for supply?
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What is the therapeutic focus of Hemgenix (etranacogene dezaparvovec)?
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Which product is classified as a Class 4 biological?
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Which document indicates that a medical device complies with Essential Principles?
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What innovation is associated with future directions in therapeutic product development?
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What is the primary intended use of an in vitro diagnostic (IVD) medical device?
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Which gene therapy product is not classified under prescription medicines?
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What is the first step in the regulation process for in vitro diagnostic (IVD) medical devices by the TGA?
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Which gene therapy product did not receive commercial supply approval in 2020?
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Which of the following is NOT considered an in vitro diagnostic (IVD) medical device?
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What step follows the preparation and submission of TGA conformity assessment?
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Which document must a manufacturer obtain to guarantee compliance with the IVD regulations?
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Which of the following describes an essential characteristic of an IVD medical device?
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What is one of the purposes of an in vitro diagnostic (IVD) medical device?
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What is the primary ligand associated with the TrkC receptor?
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Which inhibitor is noted for its effect on cancer cells possessing oncogenic TRK?
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In which type of cancer was an oncogenic NTRK fusion response to LOXO-101 observed?
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Which signaling pathway is involved with the TrkA receptor?
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What is the role of BDNF in relation to TrkB?
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What type of model was used to investigate LOXO-101's effects on tumor growth?
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Which receptor is linked to the neurotrophin known as NGF?
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Which neurotrophic factor is linked to TrkB and recognized for its role in neural development?
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What is the typical duration for drug discovery and development according to the traditional pharmaceutical development pathway?
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Which phase immediately follows clinical trials in the pharmaceutical development pathway?
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What does Good Laboratory Practice (GLP) pertain to within pharmaceutical development?
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Which step is included in the medical devices development pathway after verification and validation?
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What is the primary purpose of the Therapeutic Goods Administration (TGA)?
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Which of the following is NOT a component of the Product Development Plan?
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Which practice focuses on monitoring the safety of medicines post-market?
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What type of goods falls under the regulation of the Therapeutic Goods Act?
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What is a principal aspect of the Health Technology Assessment strategy?
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Which aspect does NOT fall under the regulatory requirements established by the Therapeutic Goods Act?
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What is a key role of the TGA regarding unapproved medicines?
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What stage occurs right before marketing approval in the pharmaceutical development pathway?
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Which of the following is described as a component of the regulatory submission process?
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Which regulation governs the licensing of manufacturers in Australia?
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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
Legal Basis of Medicines Regulation in Australia
- 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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Description
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.