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Flashcards in Final Exam Deck (49):

What are the three main elements of a drug conjugate?

1. A targeting moiety or ligand that typically detects a targeted receptor on a cancer cell.
2. Payloads which are highly potent toxins intended for release inside the cell.
3. Specialized linkers which attach the payload to the targeted moiety and are intended to cleave inside the cell.


What separates the action of ADCs from TDCs?

An ADC links a payload first to an antibody, whereas a TDC delivers the payload directly to the targeting moiety of a tumor which offers a more precise and direct delivery system.


What is structure-based drug design?

The process of utilizing a molecule's chemical and stereochemical structure to mold it into a drug which can more effectively "hit" its target.


What is observed in ligand-fit analysis?

How well the target substance binds with its target, i.e. hydrogen bonding, hydrophobic interactions, electrostatic interactions, and bioactive conformity.


How is Gleevec used to treat cancer?

Gleevec binds to the Bcr-Abl protein and inhibits its ability to catalyze phosphorylated cancer proliferation.


What factors other than ligand binding are important in structure-based design?

Keeping the drug from binding to other irrelevant receptors and to maintain drug-like properties (i.e. lipinski rule of 5).


What are the 6 main elements of a clinical research project?

Research question, Background and significance, design, participating subjects, variables, and statistical analysis.


What is a clinical trial?

A scientific study of a drug which involves human subjects in which a drug is offered as an intervention to study its efficacy and safety.


What information is to be discovered from clinical trials?

-Effectiveness of treatement
-Drug Formulation
-Combined therapies
-Effect of therapies on quality of life


True or False? Bias can be quantified.

Usually False


What are the benefits of randomizing control groups?

Eliminates bias, and provides an accurate estimate of random error.


What is a medical device?

Any device, tool, or component part designed to diagnose, treat, or otherwise mitigate the effect of a disease, and which does not achieve effectiveness through chemical means and does not need to be metabolized.


What is the difference between a pilot study and a pivotal study in medical device trials?

A pilot study is the initial study of the device to ensure safety; it is only done with fewer than 100 patients. A pivotal study is done to determine effectiveness of the device.


What is the difference between a high and low risk medical device?

Low risk devices are not sterile, are non-invasive, and do not measure things. High risk devices are life-sustaining, invasive, and/or may produce radiation.


What are some examples of class II (moderate risk) medical devices?

Powered wheelchairs, infusion pumps, and surgical drapes.


What is a 510K?

An application to the FDA for a medical device to be cleared for use. Predicate devices can be cleared in as little as 90 days.


What is PMA?

Thorough review by CDRH or in concert with CBER, CDER for design, manufacturing – can take up to 2 years


What does De Novo refer to?

A new technology never before seen by the FDA.


What is an IDE?

FDA grants permission to a sponsor to study an investigational device clinically.


What is HUD/HDE

a designation given to a device by the FDA to treat or diagnose a disease or condition affecting fewer than 4,000 individuals in the US per year, and follow-up reporting required


What are considered acceptable primary objectives for a clinical study?

Improving survival of patient, results that are detectable to patient, and/or decreasing the chances of developing complications associated with a disease which are detectable and not preferred.


What is the difference between efficacy and pragmatic trials?

Efficacy trials have a narrow selection for subject population leading to a more homogenous sample.

Pragmatic trials have a less restrictive population and can measure drug effects across a number of variables.


What is the FINER guideline for good research questions?



True or False? A data monitoring plan is required in all clinical trials.



What are the main functions of a data and safety monitoring plan?

Ensures patient safety and data validity.


Define GCP.

Good clinical practices, and international, ethical, and scientific standard for Design, Conduct, Performance, Monitoring, Auditing, Recording, Analysis and Reporting of clinical trials or studies.


What are the 7 steps in the clinical trial timeline?

1. Study Start
2. Investigators Meeting
3. Site of Action
4. Patient Enrollment
5. Study Closeout
6. Database Lock
7. Clinical Study Report


What role does the sponsor play in clinical trials?

-Pays for all expenses
-Appoints competent investigators
-Ships all drugs for the trial
-Files all paperwork to legal and regulatory authorities.


What is the role of clinical research associated?

-Clinical Trials moniter
-Regular visits to the site
-Compliance with protocol and revision of case report forms.


What is the role of the clinical research coordinator?

-IRB submissions
-Informed consent
-Contracting with pharma companies
-Budget negotiations
-Subject recruitment
-Adverse effects and case reports
-Data capture.


What is a patent?

Grants property rights on inventions that are novel, useful, and non-obvious allowing the inventor to temporarily exclude other manufacturers from making and selling the item.


What is a utility patent?

A patent for the manufacture or use of a machine/apparatus/product.


What is the defensive patent strategy?

Patent only key technologies.


What are fences?

Patent only critical features that are difficult to design around.


What are "land mines"?

File blocking patents.


What is a trademark?

Protects the word, phrase, symbol, or design that distinguishes the source of the goods or services of one business from its competitors.


What is a copyright?

Protects the works of authorship, such as literature, music, artistic works, and computer software. “Work” must be reduced to a tangible medium of expression.


When is a copyright valid?

As soon as the work is reduced to a tangible medium.


What are trade secrets?

Trade secret" means information, including a formula, pattern, compilation, program, device, method, technique, or process, that the company is better off keeping secret rather than making public because another entity could reap economic benefit from it at the expense of the company.


What is Qualification?

Documented evidence that all equipment is found to be suitable for its intended use.


What is Validation?

Documented evidence that a processes achieves established criteria and adequately controls process risks.


What are the stages of validation?

Stage 1: Develop/finalize control strategy/plan.
Stage 2: Primarily concerned with making sure that the plan is working properly.
Stage 3: A quality unit will assure that systems and procedures are are established to measure and characterize normal variability and process behavior, as well as to detect undesirable process variability from target quality parameters and control limits.


What is the main purpose of stage 3

To provide assurance that the process will remain stable.


How do you calculate maximum allowable carryover?

Lowest dose (A)/1000 x Batch size/Max Daily Dose (B)


What are some of the major components of quality systems?

-Quality unit and quality policies
-Records and documentation
-Discrepancy and failure investigations
-Training programs
-Data Tracking
-Internal Audits


What are the main steps in a facility inspection?

-Inspector shows their credentials
-Open meeting
-Facility tour-warehouse, lab, production
-Documentation review
-Interviews by the inspectors


What defines a drug product's surrogates?

Its established quality attributes like identity, potency, purity, strength, bioavailability, etc.


How can you identify potential risks to pharmaceutical quality?

By assessing their probability and severity.


What are the three classes of product recalls?

Class I: Likely to cause extreme health problems or death.

Class II: Likely to cause moderate but not life threatening medical complications.

Class III: Not likely to cause adverse health effects.