Clinical Trials

Question 1

What is a clinical trial and how is this different from innovative care?

Answer 1

• Definition of Research: Observations systematically collected (data) to obtain generalizable knowledge
• Differentiating Innovative Care from Research
  
  • Drugs not approved for a specific condition may be prescribed as part of patient care
    
    • This is not research
  • The use of drugs not yet approved for marketing and drugs prescribed with the intention of generating new knowledge is research

Question 2

What are the important features of clinical trials that make them valid?

Answer 2

• Randomized – to attempt to equalize the group characteristics
• Blinded – to reduce bias based on expectations
• Controlled – to provide a comparison group
  
  • Placebo, inert substance, “sugar pill”
• Best available therapy
• Usual care

Question 3

What are the four phases of clinical trials?

Answer 3

• Phase I:
  
  • First time in “man” following in vitro and animal testing, healthy adults or seriously ill patients or any age who have exhausted all other treatment options, dose escalation design to measure toxicity and pharmacokinetics and to develop information on dosage
• Phase II:
  
  • Randomized, controlled study of up to several hundred patients to determine disease response and short term side effects
  • Comparison to placebo is justified when:
    
    • There is no proven therapy
    • Condition being studied is minor and there is a low chance of serious harm
  • Best available therapy should be used when:
    
    • There is a proven therapy for this condition
    • Withholding therapy would raise ethical concerns
• Phase III:
  
  • Randomized, controlled study of up to thousands of subjects including populations not studied in Phase II and expanding to additional combinations of drugs to determine efficacy and side effects.
• Phase IV:
  
  • Post marketing study to determine long term and rare side effects

Question 4

What are therapeutic misconceptions regarding clinical trials?

Answer 4

• Subjects may not appreciate that a clinical trial is not ordinary treatment and they may expect that research procedures are being done for their benefit rather than their true purpose which is to gather data
• Patients may believe that a clinical trial is the miracle cure they have been waiting for

Question 5

What are some groups who are underrepresented in clinical trials?

Answer 5

• Elderly < adults <young>
  </young><li>Low Socioeconomic Status (SES) &lt; High SES</li><li>Rural &lt; Suburban</li><li>Women &lt; Men</li><li>Minority groups &lt; Non-Hispanic White</li>

</young>

Question 6

What are some barriers to access to clinical trials?

Answer 6

• Cost
• Mistrust
• Lack of awareness by patient and/or physician
• Language barriers
• Low literacy
• Transportation
• Eligibility criteria/ concomitant disease

Question 7

What are principles of human subject research including clinical trials?

Answer 7

• Respect for Persons
  
  • Autonomy, and protection of those unable to be autonomous
  • Privacy and Confidentiality
  • Freedom to stop if participation is unbearable
  • Informed Consent
• Beneficence
  
  • To the subject who assumes the risk
  • To others with the condition and to society
• Justice
  
  • Equal distribution of risks and benefits
  • No exploitation of the desperate
  • No preference to the privileged

Question 8

What are the important principles of informed consent involved in clinical trials?

Answer 8

• Information
  
  • Why, Who, What, when, for how long, risks, benefits, costs, who to call for more information or if something goes wrong, financial considerations, privacy and confidentiality
• Comprehension
  
  • Lay language should be used
  • Consent forms should be written at a sixth grade level
• Voluntariness
  
  • There must be no undue influence of subjects
  • A subject may withdraw at any time
• IRB approves a written document (informed consent form)
• Consent is a verb as well as noun
  
  • Investigators should provide information, assess understanding of the information and determine that the subject is making a decision free from coercion or undue influence