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.

Outlines

00:00

🛠️ Drug Development Focus and Target Discovery

The script emphasizes Novartis' commitment to patient-centric drug development, aiming to improve quality of life by overcoming diseases with effective and safe medications. The process begins with identifying disease-associated proteins or pathways as potential drug targets. Understanding these pathways is crucial for target discovery, as it helps in comprehending the disease mechanisms and addressing unmet medical needs. High-throughput screening and computer-based design are employed to find compounds that can modulate these targets, which are then refined for safety and effectiveness. The journey from discovery to market involves significant time and financial investment, with a low success rate, highlighting the complexity and challenges of drug development.

05:00

🔬 Clinical Trials and Drug Registration

The script outlines the clinical trial process, which includes multiple phases designed to evaluate drug safety, efficacy, and optimal dosing. Phase 1 focuses on healthy volunteers, while Phase 2 involves patients to assess efficacy and side effects. Proof of Concept trials provide early indications of a drug's impact on its target. Phase 3 trials involve a larger patient population to confirm effectiveness and monitor side effects. Post-approval, the drug must be registered with health authorities, involving the compilation of a registration dossier with efficacy and safety data. Ongoing safety monitoring and reporting are mandatory, and Phase 4 trials may be conducted for additional data collection. The ultimate goal is to ensure global access to innovative medicines that have successfully navigated the rigorous drug development process.

Keywords

💡Patient-Centric

Patient-centric refers to an approach where the patient's needs, preferences, and values are at the core of healthcare decisions and practices. In the context of the video, Novartis's mission is patient-centric, focusing on helping patients overcome diseases and improve their quality of life. This approach is evident throughout the drug development process, ensuring that innovative medicines are tailored to meet the needs of patients.

💡Drug Development

Drug development is the process of bringing a new pharmaceutical drug to the market. It includes a series of stages from target identification to clinical trials and regulatory approval. The video emphasizes the importance of this process in ensuring that new medicines are effective, safe, and accessible to patients in the shortest time possible.

💡Targets

In drug research, 'targets' are proteins in the body or microorganisms associated with a disease that are identified for treatment or prevention. The script mentions that Novartis focuses on understanding these targets and their roles in diseases, which is crucial for developing drugs that can effectively address these conditions.

💡Cellular Networks or Pathways

Cellular networks or pathways refer to the interconnected series of actions through which a cell communicates with itself and its environment. The script explains that understanding these pathways is key to identifying the most appropriate targets for drug development, as they can affect the function of multiple proteins and, consequently, the progression of a disease.

💡High Throughput Screening

High throughput screening (HTS) is a method used in drug discovery to test large numbers of chemical compounds for their potential biological activity. The script mentions HTS as one of the methods used by Novartis to find compounds that bind to identified targets and could potentially alter the disease.

💡Drug Candidate

A drug candidate is a chemical compound or biologic that has shown potential in early testing to become a drug. The script describes how a 'hit' from HTS is refined to improve its safety and effectiveness, eventually becoming a drug candidate that can proceed to clinical trials.

💡Pharmacokinetics

Pharmacokinetics is the study of how a drug moves through the body, including its absorption, distribution, metabolism, and excretion. The video script mentions that pharmacokinetic properties are essential to evaluate during preclinical and clinical stages to ensure the drug behaves as expected in the human body.

💡Animal Studies

Animal studies are research conducted on animals to understand complex disease mechanisms and test the safety of new medicines before human trials. The script notes that while animal studies are critical in drug discovery, Novartis is committed to minimizing their use and adhering to high standards of animal welfare.

💡Clinical Trials

Clinical trials are research studies conducted to determine the safety and effectiveness of medical interventions, such as drugs. The video script outlines the various phases of clinical trials, from Phase 1 focusing on safety and pharmacokinetics in healthy volunteers to Phase 3 involving thousands of patients to confirm a drug's effectiveness and monitor side effects.

💡Proof of Concept Trials

Proof of concept (POC) trials are early-stage clinical trials that aim to provide an early indication of whether a drug candidate has the desired effect on its target and impacts the disease as intended. The script mentions that Novartis conducts POC trials to rapidly assess the potential of drug candidates, ensuring resources are invested in effective and safe compounds.

💡Registration Dossier

A registration dossier is a comprehensive document that includes all the data from clinical trials to support the application for marketing authorization of a new drug. The script explains that once the dossier is approved, it allows the drug to be marketed, and it must be customized for different regions to meet local health authority requirements.

💡Post-Authorization Safety Updates

Post-authorization safety updates refer to the ongoing monitoring and reporting of a drug's safety after it has been approved and is on the market. The script mentions that these updates, along with annual reports and any additional information required by health authorities, are mandatory to ensure the continued safety of the drug for patients.

Highlights

Patient is the main focus in drug research and development at Novartis.

Mission is to help patients overcome disease and improve quality of life.

Drug development process ensures innovative medicines are effective, safe, and available quickly.

First step is to discover best targets for treating or preventing disease, usually proteins.

Challenge is identifying relevant proteins and confirming their role in disease.

Novartis focuses on understanding cellular networks and protein pathways.

Pathway approach helps identify most appropriate drug target.

Drug discovery uses high throughput screening and computer-based design.

Hit compounds are refined for improved safety and effectiveness.

On average, discovering and bringing a new drug to market takes 14 years and $2 billion.

Only one out of 10,000 hits tested may become a marketed drug.

Preclinical stage includes experiments to ensure drug safety and pharmacokinetics.

Animal studies play a critical role in understanding complex disease mechanisms.

Novartis minimizes animal research and upholds high animal welfare standards.

Clinical trials have multiple phases focused on evaluating drug safety and efficacy.

Phase 1 tests drug safety and pharmacokinetics in healthy volunteers.

Phase 2 evaluates drug efficacy, optimal dose, safety, and side effects in patients.

Proof of concept trials provide early indication of drug's impact on target and disease.

Data from Phase 1 and 2 studies informs larger clinical trials.

Phase 3 trials involve thousands of patients to confirm drug effectiveness and safety.

New drugs must be registered with health authorities, including efficacy and safety data.

Registration dossier is customized for different regions to meet health authority requirements.

Post-authorization safety monitoring and updates are mandatory for marketed drugs.

Phase 4 trials gather additional data on efficacy, safety, benefits, and risks.

Mission is to provide safe and effective treatments that benefit patients and improve their quality of life.

Transcripts

in drug research and development the

patient is the main focus the mission

for novaris is to help the patient

overcome a disease and improve their

quality of life the drug development

process is designed to ensure that

Innovative new medicines are effective

safe and available for patients in the

shortest possible

time the first step in drug development

is to discover the best targets for

treating or preventing a disease targets

are usually proteins in the patient's

body which are associated with a disease

or proteins in microorganisms causing a

disease the challenge is to identify

which proteins are relevant and more

importantly confirm their role in a

disease increasingly novaris focuses on

understanding cellular networks of

proteins or Pathways a single protein

May transmit messages to several other

proteins sometimes times in multiple

Pathways affecting their function

knowing how these Pathways work and

interact helps to identify the most

appropriate Target for a

drug the pathway approach allows novaris

to better understand the mechanisms of a

disease this knowledge together with a

desire to address unmet medical needs

determines the priorities in Target

Discovery in drug discovery

several methods like high throughput

screening and computer-based design are

used to find chemical compounds or

biologics that bind to the identified

Target if a compound modulates the

Target in a way that is expected to

alter the disease this so-called hit

will be refined to improve its safety

and Effectiveness eventually becoming a

drug

candidate discovering and bringing one

new drug to the market typically takes

an average of 14 years of research and

clinical development efforts and cost

around $2 billion

US of 10,000 or more hits tested in

early drug Discovery only one may

eventually lead to a drug that reaches

the

market in the late preclinical stage

further experiments are conducted on the

drug candidate to ensure it is safe for

patients and has the required

pharmacokinetic properties like

appropriate absorption and metabolism by

the human body these experiments are

executed with extraordinary diligence to

minimize any risks to human test

subjects animals play a critical role in

the drug Discovery process as well

although much research and development

can be done using various experiments or

using computers complex disease

mechanisms can often only be understood

through the use of animal studies also

governments and Regulatory authorities

require that medicines be tested in

animals before they are tested in humans

novaris keeps This research as limited

as possible and always ensures that

animal research is scientifically

acceptable according to current

standards and regulations for these

reasons novaris continues to use animals

in its quest to find Innovative safe and

life-saving medicines for patients at

the same time novaris is committed to

refining reducing and replacing the use

of animals in research and upholding the

highest standards in Animal

Welfare clinical trial programs consist

of several phases Each of which is

focused on evaluating drug safety and

Effectiveness or efficacy information on

ongoing clinical trials can be found at

www.clinicaltrials.gov

in Phase One of clinical trials the drug

is usually tested in healthy volunteers

to determine its safety and

pharmacokinetics in Phase 2 the drug is

given to a group of about 100 to 250

patients with the disease to evaluate

its efficacy and to determine the

optimal dose in addition the safety of

the drug and its side effects are

evaluated as these may be different in

patients compared to the healthy

volunteers tested in Phase 1 no arst

also conducts trials called proof of

concept or POC trials which are often a

mix of phase 1 and Phase 2 studies a

group of well-defined patients is

treated with the drug candidate to

provide an early indication whether it

actually affects the Target in the

intended way or has a beneficial impact

on the disease these studies allow

Novartis to rapidly invest resources on

drug compounds which are effective and

safe

this way the appropriate medicines can

reach the patients

faster the data from Phase 1 and Phase 2

studies provide the scientific

confidence necessary to continue the

development of the compound in larger

clinical

trials in phase three of clinical trials

1,2 3,000 or even more patients are

recruited for research with the

investigational drug phase three

clinical trials are undertaken to

confirm the effectiveness of the new

drug monitor side effects compare it to

establish treatments and gather

additional information to allow the drug

to be used

[Music]

safely to bring a new medicine on the

market every new drug needs to be

registered by the health authority data

from all clinical trials are collected

and compiled into a document called

registration dossier the the

registration dossier will include

efficacy and safety data to support the

final intended use of the drug the

dossier is then customized for different

regions and countries around the world

to meet the requirements of Health

authorities this is a crucial step to

ensure that patients all over the globe

can access the Innovative medicines that

have made it through the complex drug

Discovery and development process once

the registration dossier is approved and

the product is launched several

activities will continue like drug

safety monitoring which is mandatory

postauthorization safety updates annual

reports and any additional information

required by the health authority must be

provided at defined intervals as long as

the drug remains on the market often

phase four clinical trials are initiated

to gather additional data not collected

in the phase 3 program this may include

efficacy safety additional benefit and

risk information as well as

pharmacoeconomic data our mission is to

provide safe and effective treatments

that benefit patients and improve the

quality of their

[Music]

lives