Drug discovery and development process
Summary
TLDRThe 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
- đ The primary focus of drug research and development is the patient, with the aim of overcoming diseases and improving quality of life.
- đ Novartis is dedicated to discovering the best targets for treating or preventing diseases, often focusing on proteins associated with diseases.
- đ The drug development process is designed to ensure that new medicines are effective, safe, and available to patients as quickly as possible.
- đŹ Understanding cellular networks and protein pathways is crucial for identifying the most appropriate drug targets.
- đ§Ș 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.
- đ The process of refining a hit compound to become a drug candidate involves improving its safety and effectiveness.
- đ The journey from discovery to market for a new drug typically takes around 14 years and costs approximately $2 billion.
- đ Animal studies play a critical role in understanding complex disease mechanisms and are required by regulatory authorities before human testing.
- đ Novartis is committed to refining, reducing, and replacing animal use in research while maintaining high standards in animal welfare.
- đ Clinical trials consist of multiple phases focused on evaluating drug safety, efficacy, and optimal dosing.
- đ Information on ongoing clinical trials is publicly available at www.clinicaltrials.gov.
- đ The registration dossier, which includes efficacy and safety data, is essential for drug approval by health authorities worldwide.
- đ 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
đ ïž 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.
đŹ 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.
Mindmap
Keywords
đĄPatient-Centric
đĄDrug Development
đĄTargets
đĄCellular Networks or Pathways
đĄHigh Throughput Screening
đĄDrug Candidate
đĄPharmacokinetics
đĄAnimal Studies
đĄClinical Trials
đĄProof of Concept Trials
đĄRegistration Dossier
đĄPost-Authorization Safety Updates
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
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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
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lives
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