PMCY 2020 EXAM 1 REVIEW

Manufactured through chemical synthesis

Small molecule drugs

Less expensive for patient

Small molecule drugs

Manufactured in microorganisms, plants, or animals

Biologics

Very large, complex molecules or mixtures of molecules

Biologics

Produced using recombinant DNA technology

Biologics

Cannot be characterized in a lab

Biologics

Time for market is 7-10 years

small molecule drugs

Better stability of dosage formulation

small molecule drugs

Unstable dosage formulation

Biologics

Mostly process independent

Small molecule drugs

Well defined chemical structure

Small molecule drugs

Completely characterized

small molecule drugs

Nonimmunogenic

small molecule drugs

Diseases that can be treated with biologics

Cancer, psoriasis, rheumatoid arthritis, inflammatory bowel disease, diabetes, muscular degeneration, hepatitis b, hemophilia, multiple sclerosis

Biosimilars

Highly similar biologic that doesn't have to be perfectly similar to the original biologic

Interchangeable biosimilars

Have met FDA requirements such as being expected to produce the same clinical effect as the biologic and have no additional safety risks

Market time is 10-15 years

Biologics

Immunogenic (able to produce an immune response to the drug)

Biologics

Easier to manufacture

Small molecule drugs

More expensive to manufacture

Biologics

Pancreatic insulin was created by

Frederick Banting, Charles Best, and James Collip

What year was insulin created?

1921

Insulin was the first protein to be

Sequenced and assembled by total peptide synthesis

The creation of insulin led to the beginning of

recombinant DNA

Higher affinity

biologics

More complex functionalities

biologics

Higher specificity brings less interference and off target effects

Biologics

Replacement therapies for missing or defective proteins

Biologics

Administered through injections or infusions in thigh or stomach

Biologics

Patients can develop immune responses to ____ drugs which results in less effective treatments over time

biologic

Similar to the brand name product and can be utilized after the patent has expired

Generics

Generics and brand name products contain the same

active ingredient

Biosimilars have different _____ as opposed to the original biologic

cell lines

Why are biosimilars more expensive than small molecule generics?

They require more testing

Biologics cannot be administered

by mouth

Why can't biologics typically be administered by mouth?

Because the digestive system would break down and destroy the drugs

What is the first biologic than can be taken orally?

Rybelsus

6 examples of small molecule drugs

Aspirin, penicillin, envoid, retrovir, adepas, and vitrakvi

What drug marked the beginning of the antibiotic era?

Penicillin

First anti-HIV drug and first effective treatment for AIDS patients

Retrovir (AZT)

who discovered penicillin?

Alexander Fleming

Which type of drugs dominate the market?

Small molecule drugs

_____% of drugs in the pipeline are small molecule drugs

70%

Has various routes of administration

small molecule drugs

can usually pass through cell membranes to reach intracellular targets

small molecule drugs

Which is cheaper? Brand name or generic

Generic

Which is more likely to be covered by insurance?

Generic

More risk of drug-drug interactions

small molecule drugs

bioavailability and solubility issues

small molecule drugs

lack high specificity so off target effects can occur

small molecule drugs

Ways to increase solubility and bioavailability

Particle size reduction (jet milling), salts/cocrystals, amorphous solid dispersions (ASD)

Small molecule drugs can treat

Neurological disorders, hepatitis C, cancer, diabetes, and infectious diseases

Most common dosage form for small molecule drugs

Tablets

Tablet are most commonly used because

long shelf life

How do vaccines work?

They teach the immune system how to recognize and fight off specific disease causing organisms

Key ingredient in vaccines

Antigens

How do antigens in vaccines work?

They are either a weakened non dangerous version or they are part of the disease causing organism

What are some of the ingredients in a vaccine?

Preservatives, stabilizers, surfactants, residuals, diluent, adjuvant

It is usually not recommended to take ____ before a vaccination because it might affect how well a vaccine works

Over the counter medication

Who developed the first polio vaccine?

Jonas Salk

Who developed the second polio vaccine?

Albert Sabin

When was polio considered eliminated in North and South America?

1994

How many cases of paralytic poliomyelitis were reported in the United States between 1980-1999?

162

The motto for drug development is

Fail early, fail often

What is the “valley of death” in the drug development process?

Where most drugs fail

Where is the best place for a drug to fail?

during early discovery; additional testing and adjustments can be made before it enters the market

What are common issues with drugs?

Price, quality, marketing, approval process, investment, counterfeits, drug abuse

what are statins?

drugs used to lower cholesterol in the bloodstream

Acceptance

Patient filled original prescriptions

Nonacceptance

Patient did not fill original prescription

Persistence

Patient remains on therapy by given date

Nonpersistence

Patient does not remain on therapy by given date

Adherence

The patient is taking the medicine appropriately

nonadherance

The patient is not taking the medicine appropriately

Types of therapeutics

Complementary/alternative therapies, standard dosage forms, biological agents, vaccines

True or false: Community and herd immunity are the same thing

True

Define community and herd immunity

When enough people in a population are immune to a disease, so the disease has a harder time spreading, which protects even those who can't get vaccinated

Risks of drug development

Safety, efficacy, and commercial viability

Drug discovery for academic and biotech are focused on…?

orphan and designer drugs

Step 1 of drug discovery

Idea

Step 2 of drug discovery

Discovery

Step 3 of drug discovery

Pre-clinical trials

Step 4 of drug discovery

IND studies

Step 5 of drug discovery

Phase 1

Step 6 of drug discovery

Phase 2

Step 7 of drug discovery

Phase 3

Step 8 of drug discovery

Sales/marketing

Big Pharma generally stays away from

Drugs with little return and orphan drugs

Big pharma became interested in _____ because there was a worthwhile market

antibiotics

Describe a pharmacologist’s role

Conduct research on potential drugs in lab settings to understand how compounds work and how they can be recommended for development

what is pharmacology?

the study of a drug’s action on the body + the relationship between living organisms and chemicals that affect their normal or abnormal functions

Pharmacists

Drug experts; dispense + educate patients on medication, advise healthcare providers on the proper drugs to administer, and monitor patient health

4 major classes of drug targets

Lipids, proteins, carbohydrates, and nucleic acids

Branches of pharmacology

Pharmacokinetics, pharmacodynamics, therapeutics, comparative, clinical, animal, and quantitative systems

Biomarker

Any indicator that doctors or researchers can use to evaluate the state of an individual's health or confirm disease onset/progression or gauge whether a treatment is working

ex: size of a tumor to measure if cancer treatment is effective

Types of biomarkers

Early detection, diagnostic, prognostic, predictive, translational, mechanism, disease, target, toxicity

Accuracy in biomarkers

How close a measured biomarker value is to the true or accepted value

Precision in biomarkers

How close repeated measurements of the same biomarker are to each other regardless of whether they are correct

Why are biomarkers useful?

They help to identify at-risk patients and predict disease progression

Pharmakos

Drug and logos (study)