Flashcards in Lecture 1: Intro to drug development and Pharmacoepidemiology Deck (18):
What are the Five healthcare system priorities
1) Data analytics and health information technology
2) Real world outcomes and evidence based medicine
3) Patient care management
4) New provider payment models and delivery systems
5) Strategic business growth and partnerships
What are some important healthcare market drivers?
Empowered consumers, real world analytics, and personalized medicine (all improving efficiency)
In the changing landscape, where does Pharmacopeia come into play?
Providing evidence-based information
What is the triple aim in healthcare system transformation?
Reducing costs, improving patient outcomes and enhancing the patient experience
What is the take home message in terms of the different FDA drug acts passed in 1906, 1938, 1962?
Drugs cannot be adulterated or misbranded, they must be shown to be safe prior to marketing, and they must be shown to be efficacious and the labeling must reflect the drug's characteristics.
What is a simple definition of Pharmacokinetics?
This term refers to the manner by which a drug moves into the body, throughout the body and eventually, out of the body. It is the time course of drug absorption, distribution, metabolism and excretion.
What is a simple definition of Pharmacodynamics?
This term refers to the relationship between a drug's concentration at its site of action and its resulting effect which includes time course of action, therapeutic and/or adverse effects.
What are some characteristics of a drug development program?
The primary goal is regulatory submission/approval, but also to identify a therapeutic niche, extensive safety experience and need to define risk/benefit clearly.
What is the process of obtaining approval for a new drug?
Preclinical research (In vitro and In vivo tests), Clinical studies (Phase 1-safety and toxic (20-80), 2 (100-300), 3 (1000-5000)), FDA review and post-marketing surveillance (Phase 4).
What does the FDA require in pivotal trials?
Randomized, well-controlled, blinded, must-centered, clinically valid endpoints, large number of patients to establish safety or efficacy if control group is active
Why does the FDA dislike surrogate endpoints?
They may not be predictive of a beneficial endpoint for the patient.
What are reasons why drug value appears to be waning?
Time to market entry for follow on products has decreased, so generics are in the market a lot more quickly than they used to be.
What is the pharmacoepi focus in Phase IV?
To manage risk for the customer, to generate publishable data for payers, regulators, etc. These studies are typically observational or interventional and the types are: comparative effectiveness, outcomes research, epidemiological, etc.
What is the conditional approval concept?
The current/traditional model is to do optional postmarking studies after drug is approved (following phase 3). Also using 3000 or more patients. The proposed model is to get conditional approval after Phase 3 and then do required postmarking surveillance on 30,000 to 300,000 people before the drug is fully approved. The post marketing studies after full drug approval will then be optional.
What are some risks and costs of drug development?
For every 10,000 chemical entities synthesized, 20 enter animal studies, 10 enter human trials and 1 gains FDA approval. The process takes approximately 12 years and costs >$1 billion.
What is Pharmacovigilance?
The science and activities related to the detection, assessment, understanding and prevention of adverse effects or any drug related problem.
What is the purpose of spontaneous reporting systems?
They identify rare and unusual events identified by practicing physicians, patients, literature. They started after thalidomide disaster and they can help to generate hypotheses.