The Novartis Development organization is responsible for taking innovative drug candidates from our Research organization (NIBR) and proving them to be safe, effective, and high-quality therapies with superior value to patients.

Early development
In the early development stages, the Development group works together with the colleagues from NIBR to analyze the drug candidates’ potential for full development into drugs for treating human diseases, both in preclinical research and in Proof-of-Concept (PoC) clinical trials.   To ensure the safety of administering a new drug candidate to healthy volunteers and patients, extensive toxicological and safety pharmacological profiles are done in both laboratory tests and animals. Once the initial safety profile is established, the drug candidate is used in a PoC trial (i.e., it is given to a small group of patients or healthy volunteers to verify the mechanism of action and to test if the new candidate is efficacious in human disease). Thanks to the use of innovative biomarker technologies for the assessment of the study endpoints, PoC trials can usually be completed in less than a year. Additional small clinical trials    in healthy volunteers − Phase I trials − are then undertaken to gain information about the safe and tolerable dose range and the metabolism of the drug candidate in the human body. In parallel with the Phase I trials, manufacturing processes are developed and a suitable drug product is formulated.

Full development
When a drug candidate is considered to be safe and promising, it enters full development with Phase II clinical trials involving up to a few hundred patients. The purpose of these trials is to establish the range of efficacious doses for further clinical testing. If these trials are successful, the drug candidate proceeds to Phase III. Phase III trials provide definitive information about the drug’s effectiveness and increase the knowledge on its safety profile in large groups of patients. They also provide information on how the drug compares to commonly used treatments. Some of these trials, referred to as mega-trials, involve more than 10,000 patients, and Novartis Development collaborates with physicians in more than 40 countries worldwide to complete them. Information from all trials is collected and stored electronically to allow most effective and rapid data handling and analysis.

Registration
For the registration of the new drug, the results from all preclinical and clinical studies are collected and analyzed together with the quality data and the description of the manufacturing process. All the material is compiled and subsequently submitted in an appropriate form to the regulatory authorities for review. If the regulators agree that the data proves the quality, efficacy and safety of the drug, a marketing authorization is granted. From then on, a new drug can be made commercially available to patients.

Life-cycle programs
During development and once a drug is on the market, we maintain a constant watch for adverse events and report these to the regulatory authorities. At times, additional life-cycle programs – or Phase IV studies – are undertaken after registration to add new indications or improve existing formulations of the drug.

Outlook
In average, only one out of   10,000 originally synthesized compounds will clear all the hurdles on the way to becoming a commercially available drug. The process of early and full development for a new drug can take six to eight years to complete, and cost approximately 800 million dollars. At Novartis, our challenge in drug development is to constantly improve current processes and introduce new cutting-edge technologies. Our goal is to reduce development time and control costs effectively without compromising safety and quality, in order to provide innovative and affordable therapies to patients as early as possible.