Drug discovery and development process

Summary

The video discusses Novartis' mission in drug research and development, focusing on improving patient quality of life. It outlines the drug development process, from identifying targets to conducting clinical trials and ensuring safety and effectiveness. The process includes high throughput screening, animal testing, and various clinical trial phases. Novartis emphasizes the importance of understanding disease mechanisms and upholding animal welfare standards. Once a drug is proven effective, it undergoes regulatory approval and continuous safety monitoring to ensure global patient access to innovative medicines.

Takeaways

• 🛑 The primary focus of drug research and development is the patient, with the aim of overcoming diseases and improving quality of life.
• 🔍 Novartis is dedicated to discovering the best targets for treating or preventing diseases, often focusing on proteins associated with diseases.
• 🌐 The drug development process is designed to ensure that new medicines are effective, safe, and available to patients as quickly as possible.
• 🔬 Understanding cellular networks and protein pathways is crucial for identifying the most appropriate drug targets.
• 🧪 High-throughput screening and computer-based design are among the methods used in drug discovery to find chemical compounds or biologics that bind to identified targets.
• 💊 The process of refining a hit compound to become a drug candidate involves improving its safety and effectiveness.
• 📊 The journey from discovery to market for a new drug typically takes around 14 years and costs approximately $2 billion.
• 🐁 Animal studies play a critical role in understanding complex disease mechanisms and are required by regulatory authorities before human testing.
• 🔄 Novartis is committed to refining, reducing, and replacing animal use in research while maintaining high standards in animal welfare.
• 📝 Clinical trials consist of multiple phases focused on evaluating drug safety, efficacy, and optimal dosing.
• 🌐 Information on ongoing clinical trials is publicly available at www.clinicaltrials.gov.
• 📋 The registration dossier, which includes efficacy and safety data, is essential for drug approval by health authorities worldwide.
• 🔄 Post-approval, ongoing activities such as drug safety monitoring and annual reports are mandatory to ensure continued market access.

Q & A

What is the primary focus of drug research and development according to the script?

The primary focus of drug research and development is the patient, with the mission to help them overcome diseases and improve their quality of life.

What is the significance of identifying the right targets in drug development?

Identifying the right targets is crucial as they are usually proteins associated with a disease, and understanding their role helps in determining the most appropriate target for a drug.

Why does Novartis focus on understanding cellular networks or pathways in drug discovery?

Focusing on pathways helps Novartis better understand the mechanisms of a disease, as a single protein may transmit messages to several others in multiple pathways, affecting their function.

What methods are used in drug discovery to find chemical compounds or biologics that bind to the identified target?

Methods like high throughput screening and computer-based design are used to find chemical compounds or biologics that can potentially bind to the identified target.

How does the script describe the process of refining a 'hit' in drug discovery?

A 'hit' is refined to improve its safety and effectiveness, eventually becoming a drug candidate, which is a compound that modulates the target in a way expected to alter the disease.

What is the average time and cost involved in bringing a new drug to the market according to the script?

Discovering and bringing a new drug to the market typically takes an average of 14 years of research and clinical development efforts and costs around $2 billion.

Why are animals used in the drug discovery process and what is Novartis' stance on this?

Animals play a critical role in understanding complex disease mechanisms and are required by governments and regulatory authorities before testing in humans. Novartis keeps this research limited and ensures it is scientifically acceptable while being committed to refining, reducing, and replacing the use of animals in research.

What is the purpose of clinical trial programs and how many phases do they consist of?

Clinical trial programs are designed to evaluate drug safety and effectiveness or efficacy and consist of several phases, each with a specific focus.

What is the purpose of Phase One and Phase Two clinical trials as described in the script?

Phase One trials determine the drug's safety and pharmacokinetics in healthy volunteers, while Phase Two trials evaluate the drug's efficacy, optimal dose, safety, and side effects in patients with the disease.

What are proof of concept (POC) trials and how do they benefit Novartis' drug development process?

POC trials provide an early indication of whether a drug candidate affects the target as intended and has a beneficial impact on the disease, allowing Novartis to rapidly invest resources in effective and safe drug compounds.

What happens in Phase Three clinical trials and why are they important?

Phase Three trials involve a larger group of patients to confirm the drug's effectiveness, monitor side effects, and compare it to established treatments, gathering additional information for safe use.

What is a registration dossier and why is it important for bringing a new medicine to the market?

A registration dossier is a document that compiles all clinical trial data to support the drug's efficacy and safety for its intended use. It is crucial for meeting health authority requirements worldwide and ensuring global access to the medicine.

What post-launch activities are mentioned in the script and why are they necessary?

Post-launch activities include drug safety monitoring, post-authorization safety updates, annual reports, and providing additional information as required by health authorities. These activities are necessary to ensure ongoing safety and effectiveness of the drug while it remains on the market.

What is the purpose of Phase Four clinical trials and what type of data do they gather?

Phase Four clinical trials are initiated to gather additional data not collected in Phase Three, including efficacy, safety, additional benefit, risk information, and pharmacoeconomic data.