Drug Development
Years |
Process |
# of Compounds |
Stage |
|
1-4
|
Discovery
|
100,000 – 1,000,000
|
Screening
|
|
500 – 5,000
|
Hit
|
||
|
100 - 500
|
Lead
|
||
|
4-8
|
Development
|
10
|
INDE
|
|
5
|
Phase 1
|
||
|
2 – 3
|
Phase 2
|
||
|
2
|
Phase 3
|
||
|
Commercial
|
1
|
Drug development involves chemists and pharmacologists discovering or designing new chemical compounds that interact with either a defective molecule in the human body, or a molecule from a disease-causing microorganism.
A chemical compound could originate from a natural source, such as a random discovery in a lab, or a targeted effort to develop a compound - which addresses a particular issue. Once this compound has been acknowledged, tests can be utilized to determine its exact chemical makeup, and to document how effective it is, and what it could be used for.
The following research begins the process that results in the development of new medicines:
Target IdentificationTarget Identification - Determines what is causing a disease. Discovery often begins with target identification. Drugs usually act on either cellular or genetic chemicals in the body, known as targets, which are believed to be associated with disease. Scientists use a variety of techniques to identify and isolate individual targets to learn more about their functions and how they influence disease. Compounds are then identified that have various interactions with the drug targets that might be helpful in treatment of a specific disease. More about Target Identification > |
Hit Identification / Target ValidationHit Identification / Target Validation – Target needs a cavity/region to bind to a small molecule (for small molecule discovery). To select targets most likely to be useful in the development of new treatments for disease, researchers analyze and compare each drug target to others based on their association with a specific disease and their ability to regulate biological and chemical compounds in the body. Tests are conducted to confirm that interactions with the drug target are associated with a desired change in the behavior of diseased cells. This involves intensive in vitro, as well as in vivo studies that provide information on the effects of the pharmacological intervention. The result of these efforts is to establish sufficient knowledge so that physiologically relevant model systems can be developed into assays for downstream screening. Research scientists can then identify compounds that have an effect on the target selected. More about Hit Identificaiton / Target Validation > |
Lead Identification (Hit to Lead)Lead Identification (Hit to Lead) - A lead compound or substance is one that is believed to have potential to treat disease. Laboratory scientists can compare known substances with new compounds to determine their likelihood of success. Leads are sometimes developed as collections, or libraries, of individual molecules that possess properties needed in a new drug. Testing is then done on each of these molecules to confirm its effect on the drug target. (Use High Throughput Screens) More about Lead Identification (Hit to Lead) > |
Lead OptimizationLead Optimization – Determines whether the compounds that were created can work within an animal. Lead optimization compares the properties of various lead compounds and provides information to help biopharmaceutical companies select the compound or compounds with the greatest potential to be developed into safe and effective medicines. Often during this same stage of development, lead prioritization studies are conducted in living organisms (in vivo) and in cells in the test tube (in vitro) to compare various lead compounds and how they are metabolized and affect the body. This process leads to a good clinical candidate for IND enabling. (Where chemistry effort is initiated. Expensive $40-$50m) More about Lead Optimization > |
Investigational New Drug (IND) Enabling / Preclinical DevelopmentInvestigational New Drug (IND) Enabling / Preclinical Development - Before a new drug candidate can be administered to humans, a series of preclinical studies must be conducted to characterize the compound. IND enabling studies are integral to the IND package and must be included in the submission. Key components of the data package include pharmacology, toxicology and safety pharmacology, as well as absorption, distribution, metabolism, and excretion (ADME) and chemistry manufacturing & controls (CMC) sections of the submission. These components provide useful feedback for formulation scientists. Later on, this data is compared to data from the early stage clinical trials to check the predictive power of animal models. More about IND Enabling / Preclinical Development > |
Phase 1Phase 1 – Determines human safety. Determines the circulatory lifetime and safety of increased dosages of the drug. Usually tested in healthy patients to determine safety & pharmacokinetics. More about Phase 1 > |
Phase 2Phase 2 – Efficacy of drug in disease setting. Usually tested on 100-250 sick patients to evaluate its efficacy, and to determine the optimal dose. Safety & side effects are evaluated, as these may be different in sick vs healthy patients in Phase I. More about Phase 2 > |
Phase 3Phase 3– Fixes the formulation of the drug. Tests on roughly 1000-3000 healthy and sick patients - undertaken to confirm the effectiveness of the new drug, monitor side effects, and compare it to establish treatments. Additional information is gathered to allow the drug to be used safely. More about Phase 3 > |
