Novel Therapeutics Regulation Quiz

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

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

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

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

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

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

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

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

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

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

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

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

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

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

<p>Brazil (C)</p> Signup and view all the answers

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

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

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

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

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

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

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

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

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

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

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

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

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

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

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

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

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

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

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

<p>NGF (C)</p> Signup and view all the answers

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

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

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

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

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

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

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

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

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

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

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

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

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

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

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

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

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

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

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

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

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

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

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

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

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

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

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

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

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

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

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

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

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

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

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

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

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

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

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

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

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

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

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

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

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

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

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

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

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

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

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

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

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

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

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

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

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

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

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

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

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

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

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

<p>Must lack registered competitors (C), Must be life-threatening or seriously debilitating (D)</p> Signup and view all the answers

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

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

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

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

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

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

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

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

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

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

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

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

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

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

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

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

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

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

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

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

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

<p>Yescarta (C)</p> Signup and view all the answers

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

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

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

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

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

<p>NTF3 (C)</p> Signup and view all the answers

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

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

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

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

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

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

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

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

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

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

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

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

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

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

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

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

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

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

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

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

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

A standard process for drug approvals, with phases and milestones.

Drug approval with a 6-month validity period, extendable by 6 months.

Drug approval with a 255-day target, aiming at 150 days.

Timeframe tracking for drug evaluation.

Standard timeframe for evaluation in most cases.

Specifies how long an approval decision remains valid.

Processes to speed up the drug approval for specific conditions.

Sequential steps with specific checkpoints in drug approval.

The legally set time limit for an approval action

Target is 150 working days for drug approval.

Categorization of prescription drugs, encompassing new chemical entities, biologics, vaccines, recombinant products, plasma-derived products, and gene therapy.

Different types of medical products (medicines, devices, biologicals) categorized for regulatory oversight.

All therapeutic goods must be registered with the Australian Register of Therapeutic Goods (ARTG) before distribution in Australia.

Classifying medicines based on potential risk and necessary oversight by pharmacists.

Regulatory framework for therapeutic goods applying oversight based on identified risk levels.

The level of control and supervision exercised by the Therapeutic Goods Administration (TGA) on different classes of therapeutic goods.

The steps involved in registering prescription medicines, including manufacturing, pre-clinical, clinical data, and NRA review.

A meeting that happens before submitting a comprehensive dossier to the regulator on the proposed drug.

The act of submitting a complete application for pharmaceutical products to the Australian regulatory authority.

Electronic Common Technical Document format used to submit comprehensive information for drug approval processes.

The typical time required to bring a new pharmaceutical product to market, ranging from 6-11 years.

A systematic series of steps, including drug discovery, pre-clinical studies, clinical trials, registration, and marketing, in the creation of new medicines.

Good Laboratory Practice, Good Manufacturing Practice, Good Clinical Practice, and Good Vigilance Practice; standards to ensure quality and safety at different stages of pharmaceutical development.

A comprehensive action plan outlining the steps involved in bringing a new product through its development lifecycle, from planning to launch.

A structured approach to bringing medical devices to the market, including phases of initiation, formulation, design, verification, manufacturing, clinical testing, and regulatory approvals.

Australian legislation governing the regulation of therapeutic goods, ensuring quality, safety, efficacy, and timely availability.

Australian government agency responsible for regulating and overseeing the pre-market and post-market performance of therapeutic goods.

A product represented as, or likely to be taken as, having therapeutic use, including preventing, diagnosing, or treating diseases.

Scheduled periods for each stage in developing a pharmaceutical or medical device, including research, development, clinical trials, and approvals.

Guidelines to ensure quality and safety in the production and manufacturing of pharmaceutical products, from raw materials to final product.

Systematic testing of new medical products, treatments, or procedures on humans to evaluate their safety, efficacy, and optimal use.

The plan for obtaining regulatory approvals and compliance, including navigating the regulatory environment to gain market access.

The process of obtaining payment for healthcare products, procedures, or services

Laws, regulations, orders, guidances, and standards which collectively define the regulatory framework for therapeutic goods in Australia.

A review of the current market conditions, including competitors, to understand and make decisions about product development.

The 1962 thalidomide tragedy highlighted the need for stronger safety and efficacy regulations in the pharmaceutical industry.

Beecham (1859) was an early pioneer in medicine manufacturing.

1982. First registered recombinant human insulin in the US.

2017: Glybera was the first approved gene therapy product.

2017. Kymriah, a game-changing CAR-T cell therapy, was first approved.

International agreement regarding 20-year patent protection for new pharmaceutical products.

First mRNA vaccine to be approved (Comirnaty/Spikevax).

2015 was when 3D-printed tablets were first approved.

Information on the leading global markets for pharmaceuticals.

List of leading types of medicines.

Analysis of medical technology market divisions.

List of new medicines approved by the US Food and Drug Administration.

List of best-selling drugs worldwide in 2022.

Cost of top medicines to the government (Australian PBS).

Cost involved for bringing new drug to the market ranges between $1-2 to $50-500 million.

A genetic change where the NTRK gene is fused with another gene, often leading to uncontrolled cell growth.

Initial clinical trial to evaluate a new drug's safety and dosage.

Larger clinical trial to see if a drug is effective and further investigate safety.

Tumors that grow as masses in various parts of the body.

Solid tumors that have progressed to a more serious stage.

Increasing the dose of a drug in stages during a clinical trial.

Participants in clinical trials aged 18 years or older.

Non-small cell lung cancer; a common type of lung cancer

Focuses on treatment options to combat tumors that have mutated NTRK gene.

A drug that specifically targets the NTRK gene fusion in cancer cells to stop uncontrolled growth.

A medical device used in vitro (outside the body) to analyze human specimens. It's intended to provide information about the physiological or pathological state, monitor therapeutic measures, or determine safety/compatibility. It is not for general lab use.

Certificate from the Australian Register of Therapeutic Goods confirming a medical device is approved for use in Australia.

A document from the device manufacturer stating compliance with required standards and essential principles.

Proof that the medical device complies with the essential principles/standards for its category.

Identifying the appropriate class of in-vitro diagnostic device based on its intended use.

The process of preparing and submitting the necessary documents to the Therapeutic Goods Administration for an IVD device.

Submitting the necessary documents to the TGA for in-vitro diagnostic device inclusion.

Global Medical Device Nomenclature. The unique identifier for a specific medical device.

Signalling pathways that involve receptor tyrosine kinases (TRKs) and their ligands, crucial for cell growth and differentiation.

A receptor tyrosine kinase that binds to nerve growth factor (NGF).

Nerve growth factor, a neurotrophic factor that binds to TrkA in signalling pathways.

A receptor tyrosine kinase that binds to brain-derived neurotrophic factor (BDNF) and neurotrophin 4 (NTF-4).

A drug that inhibits TRK signalling pathways, potentially used to treat cancers with oncogenic TRK fusions.

An abnormal fusion of TRK genes that contribute to uncontrolled cell growth, often seen in some cancers.

A model system using cancer cells from human for testing drug efficacy and toxicity in a controlled setting.

A method to obtain tissue samples during procedures to aid cancer diagnosis.

A genetic change where the NTRK gene is fused with another gene, often leading to uncontrolled cell growth.

A drug that specifically targets the NTRK gene fusion in cancer cells to stop uncontrolled growth.

Tumors that grow as masses in various parts of the body.

Process for obtaining payment for healthcare products, procedures, or services in Australia

Test used to determine eligibility for a specific treatment like larotrectinib.

Pharmaceutical Benefits Advisory Committee (Australia)

Medicinal Substances Advisory Committee (Australia)

Testing for the presence of NTRK gene fusions in tumors.

A substance derived from human cells, tissues, or specified by the Secretary, used for treating, preventing, diagnosing, or influencing physiological processes in the human body.

A technique used to detect and visualize specific DNA sequences in cells

An organism whose genetic material has been modified by genetic technology (GT), excluding humans who have undergone somatic cell gene therapy, or any organism declared as not a GMO by regulations.

Activities involving genetically modified organisms (GMOs), including making, developing, producing, manufacturing, growing, raising, culturing, breeding, using in non-GMO manufacturing, and import.

Different levels of regulatory approvals for GMO dealings, categorized by risk (exempt, notifiable low risk, license with and without intentional release).

Techniques for administering gene therapy products, including in vivo and ex vivo methods.

Specific gene therapy products (e.g., Kymriah, Luxturna, Zolgensma, Yescarta, Hemgenix).

Regulation requiring a licence, issued by the Office of the Gene Technology Regulator, for certain therapeutic goods, including specifics relating to gene and cell therapy.

Deliberately introducing a GMO into the environment, requiring a specific license.

Activities involving GMOs that do not require regulatory approval.

Different types of medical products (medicines, devices, biologicals) categorized for regulatory oversight.

All therapeutic goods must be registered with the Australian Register of Therapeutic Goods (ARTG) before distribution in Australia.

Classifying medicines based on potential risk and necessary oversight by pharmacists.

Regulatory framework for therapeutic goods applying oversight based on identified risk levels.

The level of control and supervision exercised by the Therapeutic Goods Administration (TGA) on different classes of therapeutic goods.

Categorization of prescription drugs, encompassing new chemical entities, biologics, vaccines, recombinant products, plasma-derived products, and gene therapy.

The steps involved in registering prescription medicines, including manufacturing, pre-clinical, clinical data, and NRA review.

A meeting that happens before submitting a comprehensive dossier to the regulator on the proposed drug.

Electronic Common Technical Document format used to submit comprehensive information for drug approval processes.

The act of submitting a complete application for pharmaceutical products to the Australian regulatory authority.

Key stages in a process, not all are formal; only milestone 2 (submission acceptance) & 7 (decision) are formal.

A process of asking questions in a flexible, ongoing fashion, rather than in a fixed order.

Ongoing submission of data, not once.

Single request to the TGA for information.

Meeting with the TGA before submission of a complete application for new products.

Specific designation for medicines and treatments targeting rare diseases

A faster pathway for drug approvals, aiming for 150 working days.

Drug approval with 6 months validity, with one extension possibility.

Material used in healthcare, including instruments, apparatuses, implants etc. including software and in-vitro diagnostics.

Any material, instrument, apparatus, appliance, implant, component part, or accessory (including software), used in healthcare for in-vitro diagnostics.

The typical time required to bring a new pharmaceutical product to market, often 6-11 years.

A systematic series of stages (e.g., drug discovery, pre-clinical studies, clinical trials, registration, and marketing) in creating new medicines.

Good Laboratory Practice, Good Manufacturing Practice, Good Clinical Practice, and Good Vigilance Practice; standards for quality and safety in pharmaceutical development.

A comprehensive plan outlining the steps to bring a new product through its entire lifecycle, from initial planning to launch.

A structured approach to bringing medical devices to market, encompassing steps from initiation to launch and post-market compliance.

Australian law governing the regulation of therapeutic goods, ensuring their quality, safety, efficacy, and timely availability.

Australian government agency responsible for regulating therapeutic goods, including pre-market and post-market oversight.

A product represented as having therapeutic use, including prevention, diagnosis, or treatment of illness.

Scheduled periods for each stage of pharmaceutical or medical device development, including research, development, clinical trials, and approvals.

Guidelines for ensuring quality and safety in the manufacturing of pharmaceuticals.

Systematic testing of medical products on humans to assess their safety, efficacy, and optimal use.

The plan for obtaining regulatory approvals and compliance with relevant regulations.

A review of the current market conditions, to understand the competition, and support decisions about product development.

The process of obtaining payment for healthcare products or services.

Roadmap for clinical trials, details about intended population and trial design

A medical device used outside the body for analyzing human samples, intended to provide information on physiological or pathological states or to monitor therapeutic measures.

Document from manufacturer/supplier stating compliance with required standards for a medical device.

Proof that a medical device meets the essential principles/standards of its category/class.

Australian Register of Therapeutic Goods certificate approving medical device use in Australia.

Determining the appropriate class of in-vitro device based on how it's intended to be used.

The procedure to meet and demonstrate all requirements for approval of a medical device with the TGA.

Submitting required documents to the TGA to get an in-vitro diagnostic device approved.

Global Medical Device Nomenclature - a unique identifier for specific medical devices.

Signalling pathways involving receptor tyrosine kinases (TRKs) and their ligands, crucial for cell growth and differentiation.

A receptor tyrosine kinase that binds to nerve growth factor (NGF).

Nerve growth factor, a neurotrophic factor that binds to TrkA.

A receptor tyrosine kinase that binds to brain-derived neurotrophic factor (BDNF) and neurotrophin-4 (NTF-4).

A drug that inhibits TRK signalling pathways, potentially used to treat cancers with oncogenic TRK fusions.

An abnormal fusion of TRK genes that contributes to uncontrolled cell growth, often seen in some cancers.

A model system using cancer cells from humans for testing drug efficacy and toxicity in a controlled setting.

A method to obtain tissue samples during procedures to aid cancer diagnosis.

A CAR-T cell therapy regulated as a Class 4 biological by the TGA.

A gene therapy product regulated as prescription medicine by the TGA.

A gene therapy product regulated as a prescription medicine by the TGA.

A CAR-T cell therapy regulated as a Class 4 biological by the TGA.

Gene therapy, regulated as a prescription medicine by the TGA.

Gene therapy where cells are modified outside the body then reintroduced.

Gene therapy where genes are transferred directly into the body.

The Australian regulatory body for therapeutic goods.

A substance derived from living organisms used for medical purposes.

Treatment requiring a doctor's order for use.