10/13 Healthcare delivery

Drug 

substance that is introdcued that results in a physiological effect 

Biologics 

large molecules (entire proteins)

FDA

approves adversitement to public and providers prescribing them

Clnical trial approval process

Stages and phases

Stage 3

comprised of clinical testing

Current goal of FDA (Mission)

Safe and effective products

• it doesn’t hurt anyone and it works!!

• tested against a placebo

Off-label use

the practice of prescribing a medication for a purpose, patient population, or dosage not specifically approved by the Food and Drug Administration (FDA)

• Non indicated

FTC

responsible for printed materials and advertising once prescription product becomes OTC product (FDA no longer involved in advertisement)

Prescription only products are controlled by the

FDA

NIH

funds or has funded a lot of the arly stage conceptualization

Intramural

NIH investigators perform research

Extramural

independent investigators are awarded research grants to perform the research

• where most of the NIH research money goes !!!!!

Human testing is done in this stage

Sttage 3 

Stage 4 testing

done after the drug is approved

Pre-clinical testing

lab based

• animal based

IND (Investigational New Drug Application)

filed with the FDA starts the “testing clock” 

• starts patent protection “clock”

Stage 3 testing

human-based clinical testing 

• larger pop: done in thousnads of people

Stage 1

relates to the body (done in healthy patients)

-how does it metabkzied and excerted and did anything bad happen

Stage 2

first time used in disease we are trying to treat

Stages that deal with patients with a disease

Stages 2 and 3 

NDA

• occurs after Phase III trials

approved to allow the drug to manufactured on the market

• can take a while to occur

Patent protection

can take 20 years ( not long relative to NDA taking about 12 yrs)

Stage 3 Phase I 

Learn how the drug works in healthy humans 

• sees how metabolism works in the body

Stage 3 Phase II

demonstrate efficacy (proof of conepcet) and safety (toxicity) in patients 

Phase III

• very expensive

• provide strong evidence of efficacy and safety by studying candidate drug in a few thousand people with target disease in randomized, controlled

• controlled trials are done

Efficacy

-maximized by clinical trials

• ideal conditions

  • to demonstrate the drug does work under ideal conditions

extent to which a specific intervention, procedure, regimen, or service produces a beneficial result

Effectiveness

drugs in the real world

Phase IV 

post marketing surveillance 

MedWatch 

insutrcted by FDA to file report from al

ANDA 

submitted to FDA for a chemical entitiy already “approved” but is required when resquestiong 

• change from prescription to over the ocunter status 

• change in ingredients, dosage, manufacturing, formulation, or labelling 

Intellectual Property Rights (IPR)

Patent protection and reward for innovation 

Largest payer for Drugs in the US

The US government

Hatch-Waxman Act

result of negotation

• innovator data exclusivity for 5 years form FDA approval

• refers to les going from brand name to generic

Biologics

• large proteins 

• expensive bc complicated to make 

  • e.g: making an insulin is very complicated 

• bioactive, leading in drug development

• approval process is different 

• approved by BLA (Biologic Licencse Application)→ equivalent to NDA

BIosimilars

interchangeable 

• less clear by the FDA 

copies of generics (close but not similar to Biologics)

• does it work same way and not evoke immune system
  not tested against placebos but tested to see if they work better than innovative agents

Generics

identical to drug

• more clear by the FDA

• absorption ,s trucutre is mirror image

NDA is for _____, while BLA are for ______

small molecules, biologics