Therapy and Treatment 2

Drug Development steps

Discovery and development → preclinical research → clinical research → FDA review → FDA post market safety monitoring

Preclinical research

Testing drugs to make sure they’re safe for human use

Aspects of Discovery and Development

New insights into diseases

Testing molecular compounds

effects/side effects of existing drugs

New technologies

^^ New promising compound

Aspects of Preclinical Research

Test the drug in living organisms (in vivo) and in testing tube (in vitro)

Toxicity: make sure it doesn't cause any harm to humans

Purpose of a randomized clinical trial

safety, dosage, efficacy, side effects

protocol

a specific study plan

Phase 1 of Clinical Research

Study Participants: 20 to 100 healthy volunteers or people with the disease/condition

Length of Study: Several months

Purpose: Safety and dosage

Approximately 70% of drugs move to the next stage

Phase 2 of Clinical Research

Study Participants: Up to several hundred people with disease/condition

Length of study: several months to 2 years

Purpose: efficacy and side effects

Approximately 33% of drugs move to next stage

Phase 3 of Clinical Research

300 to 3000 volunteers who have disease or condition

Length: 1 to 4 years

Purpose: efficacy and monitoring of adverse reaction

Serendipity

unexpected discoveries that are made by accident or while searching for something else entirely

Initial purpose of Minoxidil

treatment for blood pressure and hypertension

New purpose for Minoxidil

hair regrowth

Original Purpose of Atomoxetine

treat depression

New purpose of Atomoxetine

ADHD

Aspects of FDA Drug Review

New Drug Application (NDA)

Medical officer and statistician review clinical data

A pharmacologist reviews the data from animal studies

FDA approves/rejects the medication

George Bush

Very concerned with infectious diseases

President’s Emergency Plan for AIDS relief

Scientific understanding of infectious diseases and a global network

Obama

Global Health Initiative (GHI)

Ebola response

mRNA Vaccine Technology

Trump

Operation Warp Speed (OWS)

Operation Warp Speed (OWS)

A public-private partnership initiated by the United States government to facilitate and accelerate the development, manufacturing, and distribution of COVID-19 vaccines, therapeutics, and diagnostics

Pharmacodynamics

The study of what the drug does to the body

Pharmacokinetics

The study of what the body does to the drug

Aspects of Pharmacokinetics

Absorption, Distribution, Metabolism, Elimination

Absorption

Test peak blood levels. Used to determine when medication should be taken

Distribution

how the drug is dispersed. Helps determine/understand side effects

Metabolism

Metabolic breakdown of the drugs

Elimination

How it is eliminated from the body

When the drug will stop interacting with other things like alcohol

Psychotropic Agents

Antipsychotic, Anti-depressants, Anxiolytics, Mood stabilizers, Stimulants

Classic Antipsychotic

Developed in 1950s

Treat symptoms of schizophrenia

Ex: Haloperidol

Neurotransmitter Dopamine

Very severe side effects → involuntary muscle movement

Atypical Antipsychotic

1980s

Ex: Clozapine, Risperidone

Treat symptoms of schizophrenia

Neurotransmitters: serotonin, dopamine

Less severe side effects than classical antipsychotic

Anti-depressants

Treatment of depression but also anxiety and OCD

Tricyclic antidepressants

Neurotransmitter: serotonin and norepinephrine

Ex Impramine, clomipromine

Side effects: serious especially when starting

Nausea

Vomiting

Etc

SSRI

Neurotransmitters: Serotonin

Ex: Prozac

Less severe side effects than tricyclics

Continued use reduced side effects

Anxiolytics

Ex: Xanax, Valium

Treatment of anxiety disorders

Neurotransmitter: GABA

Highly Addictive

Mood stabilizers

Ex: Lithium

Treatment of bipolar disorder

Neurotransmitter: decreasing norepinephrine release and increasing serotonin synthesis

Not serious side effects but can cause damage to liver or other organs

The patient should constantly be monitored for side effects

Stimulants

Ex: Adderall, Ritalin

Treatment of ADHD and sleep disorders (like narcolepsy)

Neurotransmitter: dopamine

Addictive for people who do not have ADHD

Transcranial Magnetic Stimulation (TMS)

treatment for medication-resistant depression.

How it works: The stimulator rapidly turns a magnetic field on and off, which induces weak electrical currents in the brain. This stimulates nerve cells and neural circuits

Effects don’t last very long

Treatment for Medication Resistant OCD

Deep Brain Stimulation

Putting Electrodes inside your brain