Introduction to Drug Development & Regulation

Summary

The script delves into the intricate process of drug development with Novartis, emphasizing patient-centricity and the pursuit of innovative medicines. It outlines the stages from target discovery to clinical trials, highlighting the challenges, costs, and regulatory requirements. The script also touches on the ethical considerations in animal testing and the importance of safety, efficacy, and pharmacokinetics in bringing a drug to market. The discussion includes the specific case of COVID-19 vaccines, orphan drugs, and the classification of drug safety in pregnancy.

Takeaways

• 🛑 The primary mission of Novartis in drug development is to help patients overcome diseases and improve their quality of life.
• 🔍 The initial step in drug development is identifying the best targets for treating or preventing diseases, which are often proteins associated with diseases.
• 🌐 Novartis emphasizes understanding cellular networks and pathways to identify the most appropriate drug targets, allowing a better grasp of disease mechanisms.
• 🧪 Drug discovery involves methods like high throughput screening and computer-based design to find compounds that bind to identified targets and modulate them as expected.
• 💊 The process of discovering and bringing a new drug to market takes an average of 14 years and costs around 2 billion US dollars.
• 🐁 Animal studies are critical in drug discovery, despite efforts to minimize their use, due to the need to understand complex disease mechanisms and regulatory requirements.
• 📊 Clinical trials are conducted in several phases to evaluate drug safety, efficacy, and optimal dosage, with increasing numbers of subjects as trials progress.
• 📚 Proof of Concept (PoC) trials provide early indications of whether a drug candidate affects the target as intended and has a beneficial impact on the disease.
• 🌐 Post-approval, ongoing activities such as drug safety monitoring, post-authorization safety updates, and annual reports are mandatory to ensure ongoing compliance with health authority requirements.
• 🚫 Novartis is committed to refining, reducing, and replacing the use of animals in research while upholding high standards in animal welfare.
• 📈 The process from target identification to clinical trials is a high-leverage point, where getting details right early can prevent wasting resources later in the process.

Q & A

What is the primary mission of Novartis in drug research and development?

The primary mission of Novartis is to help patients overcome diseases and improve their quality of life by ensuring that innovative new medicines are effective, safe, and available to patients in the shortest possible time.

What is the first step in the drug development process according to the script?

The first step in the drug development process is to discover the best targets for treating or preventing a disease, which are usually proteins in the patient's body or microorganisms associated with the disease.

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

Novartis focuses on understanding cellular networks or pathways to identify how a single protein may transmit messages to several other proteins, affecting their function in multiple pathways, which helps to identify the most appropriate target for a drug.

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

In drug discovery, methods like high throughput screening and computer-based design are used to find chemical compounds or biologics that bind to the identified target.

How many hits out of 10,000 tested in early drug discovery may eventually lead to a drug that reaches the market?

Out of 10,000 or more hits tested in early drug discovery, only one may eventually lead to a drug that reaches the market.

What role do animals play in the drug discovery process?

Animals play a critical role in the drug discovery process, as complex disease mechanisms can often only be understood through the use of animal studies. They are also required by governments and regulatory authorities before medicines are tested in humans.

What is the purpose of phase one clinical trials?

The purpose of phase one clinical trials is to test the drug in healthy volunteers to determine its safety and pharmacokinetics.

What does Novartis aim to achieve with proof of concept (PoC) trials?

Novartis aims to provide an early indication of whether a drug candidate actually affects the target in the intended way or has a beneficial impact on the disease through PoC trials.

What is the purpose of phase three clinical trials?

The purpose of phase three clinical trials is to confirm the effectiveness of the new drug, monitor side effects, compare it to established treatments, and gather additional information to allow the drug to be used safely.

What is the significance of the registration dossier in bringing a new medicine to the market?

The registration dossier is significant as it collects and compiles data from all clinical trials into a document that includes efficacy and safety data to support the final intended use of the drug. It is then customized for different regions to meet health authority requirements, ensuring global access to the medicine.

What are orphan drugs and why are they often neglected in research and development?

Orphan drugs are drugs for rare diseases affecting fewer than 200,000 people. They are often neglected because the profits from sales may not cover the development costs, making it less attractive for investment from a business perspective.

What is the current phase of COVID-19 vaccine trials as mentioned in the script?

As per the script, it is suggested that the COVID-19 vaccine trials are likely in phase three, as they are targeting a larger number of people and are being tested in various countries.