[R] Clinical Trials, Type, Phase 1/2/3,

CCTR 630(총리뷰)

-Clinical trials: prospective study, 

                        comparing effect and value of intervention against control

                        in human being

-VCU: Clinical trial office(support - ask Dr. Juan Lu) 

-Types: 

 1)Treatment: new combination/new approach

 2)Prevention: medicine, vaccine, vitamin, mineral, lifestyle change

 3)Diagnostic: find better test/procedure for diagnosis

 4)Screening: detect certain disease earlier(ultimate goal)

 5)QoL trial: comfort for chronic illness

어떻게 하면 빨리, 제대로 진단을 해서, 치료로 나아갈 수 있는가?

치료는 어떤 게 잘 적용이 되려나?(Pathophysiology) 

모두 Pathophysiology, science, Preclinical experiment 에서 idea가 많이 나올 수 있겠다. 

-PD: effect of drug(mechanism of action)

 PK: half life(absorption, distribution, excretion)

-Phase1, Phase2: exploratory trial. small/narrow population

 Phase 3; therapeutic confirmation -> FDA approval. 

-Details

 Phase 1: pharmacology, toxicity

(drug tolerance, metabolism, and interactions, and PK, PD)

 Phase 2: effect(Small group)

                (effects of various doses and typically use biomarkers as the outcome)

 Phase 3: confirm effect(full scale evaluation) 

(therapeutic confirmatory studies= mortality/morbidity/adverse outcome)

 cf) life style, surgical approach: will not fit to this phase. 

  

 Phase 4: post-marketing(surveillance = 10yrs)

 

	Phase  I	Phase  II	Phase  III	Phase  IV
Objectives	Determine the metabolic and pharmacological actions and the maximally tolerated dose	Evaluate effectiveness, determine the short-term side effects and identify common risks for a specific population and disease	Obtain additional information about the effectiveness on clinical outcomes and evaluate the overall risk-benefit ratio in a demographically diverse sample	Monitor ongoing safety in large populations and identify additional uses of the agent that might be approved by the FDA
Factors to be identified	-Bioavailability -Bioequivalence -Dose proportionality -Metabolism -Pharmacodynamics -Pharmacokinetics	-Bioavailability -Drug-disease interactions -Drug-drug interactions -Efficacy at various doses -Pharmakodynamics -Pharmakokinetics -Patient safety	-Drug-disease interactions - Drug-drug interactions -Dosage intervals -Risk-benefit information -Efficacy and safety for subgroups	-Epidemiological data -Efficacy and safety within large, diverse populations -Pharmacoeconomics
Data Focus	-Vital signs -Plasma and serum levels -Adverse events	-Dose response and tolerance -Adverse events -Efficacy(Start)	-Laboratory data -Efficacy (Clinical outcome) -Adverse events	-Efficacy -Pharmacoeconomics -Epidemiology -Adverse events

	Phase  I	Phase  II	Phase  III	Phase  IV
Objectives	Determine the metabolic and pharmacological actions and the maximally tolerated dose	Evaluate effectiveness, determine the short-term side effects and identify common risks for a specific population and disease	Obtain additional information about the effectiveness on clinical outcomes and evaluate the overall risk-benefit ratio in a demographically diverse sample	Monitor ongoing safety in large populations and identify additional uses of the agent that might be approved by the FDA
Factors to be identified	-Bioavailability -Bioequivalence -Dose proportionality -Metabolism -Pharmacodynamics -Pharmacokinetics	-Bioavailability -Drug-disease interactions -Drug-drug interactions -Efficacy at various doses -Pharmakodynamics -Pharmakokinetics -Patient safety	-Drug-disease interactions - Drug-drug interactions -Dosage intervals -Risk-benefit information -Efficacy and safety for subgroups	-Epidemiological data -Efficacy and safety within large, diverse populations -Pharmacoeconomics
Data Focus	-Vital signs -Plasma and serum levels -Adverse events	-Dose response and tolerance -Adverse events -Efficacy(Start)	-Laboratory data -Efficacy (Clinical outcome) -Adverse events	-Efficacy -Pharmacoeconomics -Epidemiology -Adverse events

• Basic research: fundamental understanding of biology and disease processes.

• Translational research: moving basic discoveries from concept into clinical

     evaluation (specific disease entities or therapeutic concepts.)

• Critical path research: Clinical development.

내가 하고 싶은 스터디가 이것인데,

HFpEF 의 basic 결과를 통해서, 지금 여기서 할 수 있는게 무엇인지 알아보도록 하자! 현재까지의 진단과 치료에 대한 연구에 대해서 공부하고, 그 리뷰를 작성하는 것으로부터 시작하도록 한다. PPT를 만들어서, 발표하고, 거기에서 내가 뽑아낸 질문들에 대해서 더 깊이 연구해서 뽑고 또 뽑아낸다!!

- Starting dose of medication: traditionally 10% of lethal dose of rodent.