General contact

Head Office
Novartis International AG
CH-4002 Basel

+41 61 324 11 11
+41 61 324 80 01
Monday - Friday,
8:30 - 17:00, GMT+1
(Central European Time)


Novartis International AG
Investor Relations
P.O. Box
CH-4002 Basel

+41 61 324 79 44


Global Oncology Media Relations
Denise Brashear
Novartis Oncology Global Public Relations

+1 862 778 7336

Novartis Media Relations
Eric Althoff
Basel, Switzerland

+41 61 324 7999

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How It Works

At Novartis, Research & Development work together seamlessly in an effort to bring new and better medicines to market in the shortest possible time.

This effort involves two phases: an “exploratory phase,” during which a candidate compound is discovered and a Proof of Concept (PoC) is established through studies in patients; and a “confirmatory phase,” during which the drug enters full development when studies in large numbers of patients are conducted.

The exploratory phase: drug discovery

All drug discovery efforts at Novartis focus on patients. Scientists determine which diseases will be the focus of research efforts based on two questions:

  • Do we have, or can we gain, significant understanding of the cause, or mechanism, underlying the disease?
  • Does this disease represent a significant unmet medical need?

If the answer to both questions is yes, then Novartis develops a research program aimed at better understanding the disease and finding an effective therapy. Early-discovery science determines how a disease is caused at the molecular level, using our own discoveries as well as those from external collaborators. We look for clues in both patients’ experience of the disease and the compendium of historical medical and scientific knowledge, integrated with the growing knowledge of human biology, chemistry and genetics.

Target discovery and drug design

Typically, making a drug begins with identifying a protein associated with human disease. These proteins are known as “targets”. When it is confirmed that a target plays a role in a disease, an experiment known as a high-throughput screen is conducted to find a chemical compound or antibody that binds or “hits” the target in a way that alters the disease. Once chemical compounds or antibodies are identified by their binding to a target, these hits are enhanced to improve their safety and effectiveness. The resulting chemical compound or antibody becomes a drug candidate.

Preclinical safety and efficacy

An initial profile of a drug candidate’s safety and effectiveness must be determined before it is tested in humans. In this phase, scientists use computer models and laboratory tests to assess the safety of a drug candidate. These tests determine how well a drug candidate is absorbed, where it goes within the body, how it is broken down or metabolized, and how quickly and in what manner it is eliminated from the system.

Proof of Concept and Phase I

In PoC trials, the drug candidate is given to a small group of patients (five to 15) to determine how the target functions in the human body, or its “mechanism of action.” It is also important to get an early understanding of how the drug candidate may enter Phase I trials (20-80 patients or healthy volunteers) to evaluate its safety, determine the safe dose and identify side effects. Sometimes drug candidates go directly from PoC to Phase II trials.

The confirmatory phase: drug development

Clinical development (Phases II and III)

In Phase II trials, the drug is given to a larger group of patients (100-300) to test its effectiveness, determine the appropriate dose, and to further evaluate its safety. In Phase III trials, the drug is given to large groups of patients (1,000-3,000) to confirm its effectiveness, monitor side effects, compare it to commonly used existing treatments and collect information that will allow the medicine to be used safely.


To register a new drug, the results of all preclinical and clinical studies, along with the description of the manufacturing process, are compiled and submitted to regulatory authorities. If regulators agree that the data establish the quality, efficacy and safety of the drug, a marketing authorization is granted. The new drug can then be made commercially available to patients. Once a drug is on the market, adverse effects need to be constantly monitored and reported to regulatory authorities. In addition, life-cycle programs—including Phase IV clinical trials—are often undertaken to explore and add new indications or improve existing formulations of the drug.

Learn about compounds Novartis Oncology is developing for a broad range of key therapeutic targets