Introduction to Drug Development Answer Key

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  1. How many clinical phases are there in therapeutics development?  Four. What does each phase test?

Phase 1:  Safety and tolerability, dose escalation

Phase 2:  Safety and Efficacy in the target population, proof of principle

Phase 3:  Pivotal, registrational studies that confirm efficacy and proven treatment effect, safety in large numbers

Phase 4:  Post-marketing studies, safety surveillance through long-term safety assessments for large numbers of patients, use drug and disease-specific patient registries 

  1. What types of studies are considered the “gold standard?” Why?

Randomized, placebo controlled studies because they provide the highest standard of evidence. 

  1. What is Epidemiology? A clinical research method that characterizes the rates, incidence, and prevalence of the disease.
  1. An Efficacy Trial compares what? Baseline characteristics to outcomes in a sample of patients.
  1. Dr. Adelman argues that the bar is getting higher for the development of therapeutics. Why does he believe that?

Drugs not only need to treat the disease, they need to be proven to be better than existing therapies already on the market. Some believe the bar is too high, while others believe it is too low. 

  1. List as many reasons as you can for why the drug process is slow. Which of these reasons protect the interests of patients?

Your thoughts

  1. If patients are fully engaged in a research project might they offer input that could make the research process less expensive and/or more efficient? Is this possible without jeopardizing patient safety?

Your thoughts