Overview of Drug Development
         and Clinical Trials




                Rajendra.Sadare
Senior Software Engineer-Tes...
The Drug Development Process


   Each country has a regulatory body which
    governs the approval process

   Drug mus...
Clinical Trial

   Give (new drug to a number of subjects and assess outcome
    (uncontrolled)




   Give new drug to ...
Clinical Trials: Major Questions


    What happens to the drug in the body?

    What happens to the body when given th...
Concepts In Clinical Development



   Pharmacokinetics (PK):             Evaluation or quantification of
    (absorption...
Concepts In Clinical Development



   Efficacy
          - A treatment is considered ineffective unless scientifically /...
Standards Of Clinical Development


                        Key concepts in clinical trial design


      •   Control:    ...
Patient Control Groups: Why?


• To provide a standard for comparison of new therapy
• To eliminate positive bias toward n...
The History of Clinical Trials


     First controlled clinical trial

         1753 – Lind’s study comparing the use of...
The History of Clinical Trials


      First randomised controlled clinical trial

          1948- First use of a random...
Development of New Drug -
A High-Risk Undertaking

   Time: 8 -12 years from discovery to market.

   Cost: average of $...
Major Stages of Drug
   Development

Preclinical Testing
   IND Application
      Clinical Testing – Phase I
             ...
Drug Development Process
 Preclinical    I
                                      Clinical           NDA Review       Post-...
Preclinical Testing




       Laboratory and Animal Testing is Done

       Is compound safe in living organisms ?

   ...
IND Application (Investigational New Drug)


    Report the results of preclinical testing

    Describes how the drug i...
Clinical Testing – Phase I



   Involves giving the drug to a small number of healthy volunteers

   Determines the saf...
Clinical Testing – Phase II



   Involves giving the drug to a large group (100-300) of patients who
    have the diseas...
Clinical Testing – Phase III

     Involves administering the drug to a large number of patients
      (1000-3000)

    ...
NDA – New Drug Application


   If the results of all the previous testing is positive, then the
    pharmaceutical compa...
Clinical Testing – Phase IV


    Once the NDA is approved and the drug is available, post-marketing
     studies are con...
Standards of Clinical Development


     US law requests “substantial evidence” to support claim of
                   eff...
Good Science + Good Study Logistics
=
Good Clinical Development




                Regulatory authority satisfaction
   ...
Thank U
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Drug Development Life Cycle

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Drug Development Life Cycle (DDLC)

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Drug Development Life Cycle

  1. 1. Overview of Drug Development and Clinical Trials Rajendra.Sadare Senior Software Engineer-Testing and Validation Arisglobal Software Pvt.Ltd
  2. 2. The Drug Development Process  Each country has a regulatory body which governs the approval process  Drug must be proved to be safe and effective  Pre-clinical testing (laboratory and animals)  Clinical testing (clinical trials in humans)
  3. 3. Clinical Trial  Give (new drug to a number of subjects and assess outcome (uncontrolled)  Give new drug to one group and control to another group and assess outcome (controlled clinical trial)
  4. 4. Clinical Trials: Major Questions  What happens to the drug in the body?  What happens to the body when given the drug?  Is the drug clinically effective?  Is the drug clinically safe / tolerable?  How should the drug be taken?
  5. 5. Concepts In Clinical Development  Pharmacokinetics (PK): Evaluation or quantification of (absorption, distribution, what the body does to a drug metabolism, elimination - substance over time. A.D.M.E.)  Pharmacodynamics (PD): Evaluation or quantification of (effects on organs or systems) what a drug substance does to the body over time, and over drug concentrations.
  6. 6. Concepts In Clinical Development  Efficacy - A treatment is considered ineffective unless scientifically / clinically proven efficacious.  Safety - A treatment is considered unsafe unless scientifically / clinically proven safe / tolerated. Key is risk / benefit ratio!
  7. 7. Standards Of Clinical Development Key concepts in clinical trial design • Control: active (positive) placebo (negative) others • Randomization: treatment assignment left to chance • Blinding: double-blind, single blind, open • Parallel groups vs. cross-over vs. Others (simultaneous) (sequential in time) • Dose titration vs. fixed dose • Stratification: balancing relevant subset of patients
  8. 8. Patient Control Groups: Why? • To provide a standard for comparison of new therapy • To eliminate positive bias toward new therapy, including “placebo” effect. • To protect new therapy against negative bias concerning adverse experiences. • To increase scientific and regulatory acceptance of study results
  9. 9. The History of Clinical Trials  First controlled clinical trial  1753 – Lind’s study comparing the use of different treatments for scurvy  Treatment - lime & oranges, seawater etc.  2 patients in one group  Two patients on limes & oranges were cured
  10. 10. The History of Clinical Trials  First randomised controlled clinical trial  1948- First use of a randomised control group: streptomycin treatment of pulmonary tuberculosis  Treatments: streptomycin (antibiotic) versus bed rest  Patients received streptomycin OR just bed rest at random (randomised clinical trial)  Outcome: streptomycin was effective
  11. 11. Development of New Drug - A High-Risk Undertaking  Time: 8 -12 years from discovery to market.  Cost: average of $800-900 million.  Success: 1 in 4000 compounds synthesized or 1 in 5 tested in humans reaches the market.  Return: 1 in 3 drugs reaching the market recaptures development costs.
  12. 12. Major Stages of Drug Development Preclinical Testing IND Application Clinical Testing – Phase I Clinical Testing – Phase II Clinical Testing – Phase III New Drug Application Clinical Testing–Phase IV
  13. 13. Drug Development Process Preclinical I Clinical NDA Review Post-Marketing N Phase I D Adverse Initial Reaction Synthesis Phase II Reporting A P Phase IV P Animal L Phase III Surveys/ Testing I Sampling C Testing A T Treatment Use I O N Inspections Range 1-3 Range 2-10 Yrs. FDA Time Yrs. Avg: 5 Yrs. Range 2 Mon – 7 30 Day Yrs. Avg:18 Mos. Safety Review NDA NDA Avg:24 Mos. Submitted Approved Average of Approximately 100 Months From Initial Synthesis to Approval of NDA
  14. 14. Preclinical Testing  Laboratory and Animal Testing is Done  Is compound safe in living organisms ?  Is compound biologically active?  If YES, file an IND Application
  15. 15. IND Application (Investigational New Drug)  Report the results of preclinical testing  Describes how the drug is made  If the FDA does not disapprove of the IND application within 30 days, then testing in humans can begin
  16. 16. Clinical Testing – Phase I  Involves giving the drug to a small number of healthy volunteers  Determines the safety of the drug as well as the safe dosage range  Takes a year or less to complete
  17. 17. Clinical Testing – Phase II  Involves giving the drug to a large group (100-300) of patients who have the disease that the drug is expected to treat  Purpose is twofold…. - Does the drug work in the disease population? - At what dosage does the drug demonstrate efficacy?  Takes about 2 years to complete
  18. 18. Clinical Testing – Phase III  Involves administering the drug to a large number of patients (1000-3000)  Purpose is to…. - Confirm earlier efficacy results - Identify adverse events which occurs when the drug is given to a larger population over a longer period of time  Takes about 3 years to complete
  19. 19. NDA – New Drug Application  If the results of all the previous testing is positive, then the pharmaceutical company files an NDA  NDA contains all of the information gathered during preclinical to phase III  NDA can be thousands of pages long  Can take 2-3 years for FDA to review
  20. 20. Clinical Testing – Phase IV  Once the NDA is approved and the drug is available, post-marketing studies are conducted to further confirm safety and efficacy during long-term use  Can include mail-in questionnaires and personal interviews
  21. 21. Standards of Clinical Development US law requests “substantial evidence” to support claim of effectiveness for new drugs. Basis for determining the claim is to evaluate if a clinical investigation is “adequate” and “well controlled”.
  22. 22. Good Science + Good Study Logistics = Good Clinical Development  Regulatory authority satisfaction  Public health protection  Marketable product
  23. 23. Thank U

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