Our Global Drug Development (GDD) organization oversees drug development activities for our Innovative Medicines Division and the biosimilars portfolio of our Sandoz Division. Development of products for the Surgical and Vision Care franchises within our Alcon Division and of small molecule generics for our Sandoz Division are not included in GDD. GDD works collaboratively with the Novartis Institutes for Biomedical Research (NIBR), Innovative Medicines and Sandoz to execute our overall pipeline strategy and takes an enterprise approach to pipeline portfolio management. The GDD organization includes centralized global functions such as Regulatory Affairs and Global Development Operations, as well as Global Development units aligned with our business franchises. GDD was created to increase Group-wide coordination of drug development and to improve resource allocation, technology implementation and process standardization with a goal of further increasing innovation. GDD includes approximately 11,000 full-time equivalent associates worldwide.
Under our Global Drug Development unit, the focus of our development program is to determine the safety and efficacy of a potential new medicine in humans.
The traditional model of development comprises three phases, which are defined as follows:
Phase I: These are the first clinical trials of a new compound, generally performed in a small number of healthy human volunteers, to assess the drug’s safety profile, including the safe dosage range. These trials also determine how a drug is absorbed, distributed, metabolized and excreted, and the duration of its action.
Phase II: Clinical studies performed with patients who have the target disease, with the aim of continuing the Phase I safety assessment in a larger group, assessing the efficacy of the drug in the patient population, and determining the appropriate doses for further evaluation.
Phase III: Large-scale clinical studies with several hundred to several thousand patients, which are conducted to establish the safety and efficacy of the drug in specific indications for regulatory approval. Phase III trials may also be used to compare a new drug against a current standard of care to evaluate the overall benefit-risk relationship of the new medicine.
In each of these phases, physicians monitor volunteer patients closely to assess the potential new drug’s safety and efficacy.
Though we use this traditional model as a platform, we have tailored the development process to be simpler, more flexible and efficient. We view the development process as generally consisting of Exploratory Development where ‘‘proof of concept’’ is established, and Confirmatory Development where this concept is confirmed in large numbers of patients.
Exploratory Development consists of clinical ‘‘proof of concept’’ (PoC) studies, which are small clinical trials (typically 5-15 patients) that combine elements of traditional Phase I/II testing. These customized trials are designed to give early insights into issues such as safety, efficacy and toxicity for a drug in a given indication and are conducted by NIBR. Once a positive proof of concept has been established, the drug moves to the Confirmatory Development stage and becomes the responsibility of GDD. Confirmatory Development has elements of traditional Phase II/III testing and includes trials aimed at confirming the safety and efficacy of the drug in the given indication leading up to submission of a dossier to health authorities for approval. Like traditional Phase III testing, this stage can also include trials which compare the drug to the current standard of care for the disease, in order to evaluate the drug’s overall risk/benefit profile.
The vast amount of data that must be collected and evaluated makes clinical testing the most time-consuming and expensive part of new drug development. The next stage in the drug development process is to seek registration for the new drug.
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.
At each phase of clinical development, our activities are managed by our Innovation Management Board (IMB). The IMB is responsible for oversight over all major aspects of our development portfolio and oversees our drug development budget. In particular, the IMB is responsible for the endorsement of proposals to commence the first clinical trials of a development compound, and of major project phase transitions and milestones following a positive proof of concept outcome, including transitions to full development and the decision to submit a regulatory application to the health authorities. The IMB is also responsible for project discontinuations, for the endorsement of overall development strategy and the endorsement of development project priorities. The IMB is chaired by our Chief Executive Officer and has representatives from Novartis senior management with expertise spanning multiple fields, among its core members and extended membership.
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