New Drug Discovery and Development (Overview) - Part 1 | Dr. Shikha Parmar
Summary
TLDRThis video offers an insightful overview of the complex process of new drug discovery and development. It highlights the multi-phase journey from screening thousands of compounds to identifying a lead drug candidate. The process includes target identification, lead generation, preclinical studies in animals, and clinical trials in humans, which can take up to 15 years and cost around $1.5 billion. Post-market surveillance ensures long-term drug safety even after FDA approval. The video emphasizes the importance of monitoring adverse drug reactions, particularly in larger populations after a drug is released.
Takeaways
- 💊 Drug discovery and development is a complex, lengthy process that takes 10-15 years and costs around $1.5 billion.
- 🧪 The drug discovery phase involves screening 5,000 to 10,000 compounds to identify potential leads or drug candidates.
- 🔍 Drug discovery has two main steps: identifying a target in the disease and validating it, followed by screening active compounds to find hits and leads.
- 📊 Hits are the initial 100-200 compounds with potential, and from these, dozens of leads are optimized for effectiveness and selectivity.
- 🧬 Lead optimization involves modifying chemical structures to improve selectivity, efficacy, and the ADME (absorption, distribution, metabolism, and excretion) profile.
- 🐁 In drug development, 250 optimized compounds are tested in animals during preclinical trials, with only around five moving forward for human testing.
- 👩🔬 Clinical trials are conducted in three phases (Phase 1, 2, 3), and they typically last six to seven years.
- ✅ If a drug passes clinical trials, a New Drug Application (NDA) is submitted to the FDA for approval, which takes one to two years.
- 🛡️ After approval, the drug undergoes post-marketing surveillance (Phase 4) to monitor its safety in the general population.
- ⚠️ Drugs may be withdrawn from the market during Phase 4 if serious adverse effects are discovered, as was the case with the drug Sibutramine.
Q & A
What are the two main stages in the process of new drug discovery and development?
-The two main stages are the new drug discovery stage and the new drug development stage.
How many compounds are typically screened during the drug discovery process?
-During the drug discovery process, around 5,000 to 10,000 potentially active compounds are screened.
What are 'leads' in the drug discovery process?
-'Leads' are potential drug candidates identified during the drug discovery process after screening thousands of compounds.
What is the role of high-throughput screening (HTS) in drug discovery?
-High-throughput screening (HTS) is used to screen thousands of compounds to identify around 100 to 200 hits that interact with a specific target in drug discovery.
What is lead optimization, and why is it important?
-Lead optimization involves modifying lead compounds chemically to produce candidates with higher affinity, selectivity, and a good ADME (absorption, distribution, metabolism, and excretion) profile, which is crucial for developing effective drugs.
What happens during the pre-clinical studies in the drug development process?
-During pre-clinical studies, around 250 lead compounds are tested in animals for safety, efficacy, and toxicity. By the end of this phase, typically only 5 compounds remain for further testing.
What are the three phases of clinical trials, and how long do they take?
-The three phases of clinical trials are Phase 1, Phase 2, and Phase 3, and they take around 6 to 7 years to complete.
What is the role of the FDA in the drug development process?
-The FDA reviews the Investigational New Drug (IND) and New Drug Application (NDA) submissions, monitors safety and efficacy data, and grants approval for a drug to be marketed if it meets safety and efficacy requirements.
What is post-marketing surveillance, and why is it important?
-Post-marketing surveillance, also known as Phase 4 trials, involves monitoring the drug's safety once it is available to the public. It helps detect rare or long-term adverse effects that may not have been observed during clinical trials.
Why was the drug 'Sibutramine' withdrawn from the market?
-Sibutramine was withdrawn from the market because it was found to increase the risk of heart disease and stroke during post-marketing surveillance.
Outlines
💊 Introduction to New Drug Discovery and Development
The video introduces the process of new drug discovery and development. It highlights the significance of drugs in saving lives and improving quality of life. The process of introducing a new drug to the market is complex, involving over 1.5 billion USD in costs and taking 10 to 15 years of research. The drug discovery stage involves screening thousands of compounds to identify potential candidates (called leads), while the drug development stage focuses on testing and refining these leads for safety and efficacy.
🧪 Stages of Drug Discovery: Target Identification and Lead Optimization
This section explains the drug discovery phase, starting with the identification and validation of a target involved in a disease. High-throughput screening (HTS) methods are used to screen 5,000 to 10,000 compounds, reducing the pool to about 100 to 200 'hit' compounds. These hits are further optimized into 'leads,' which are potential drug candidates. Lead compounds are refined through chemical modifications to improve their selectivity and ADME (Absorption, Distribution, Metabolism, Excretion) profiles. About 250 compounds enter the drug development phase.
🧬 Pre-Clinical Studies: Testing in Animals
In this paragraph, the pre-clinical phase of drug development is outlined. It involves testing the 250 optimized compounds on animals to assess their safety and efficacy. Pre-clinical studies take about 1 to 2 years and typically result in only five compounds advancing to the next stage. The data collected from these studies is compiled into an Investigational New Drug (IND) application, which is then submitted to the FDA for review before human trials can begin.
👩⚕️ Clinical Trials: Phases of Human Testing
Here, the video explains clinical trials, which follow the FDA's approval of the IND application. Clinical trials are conducted in three phases (Phase 1, 2, and 3) over a span of six to seven years. These trials evaluate the drug’s safety, efficacy, and ADME profile in human subjects. By the end of the trials, all but one drug candidate is typically eliminated. The data from these trials is compiled into a New Drug Application (NDA), submitted again to the FDA for final approval before the drug can be marketed.
✅ FDA Approval and Post-Marketing Surveillance
Once clinical trials are completed, the FDA reviews the NDA for one to two years. If satisfied with the drug's safety and efficacy, the FDA grants approval for large-scale production and marketing. However, even after approval, pharmaceutical companies are required to monitor the drug's safety through post-marketing surveillance, also known as Phase 4 studies. These studies track adverse drug reactions and long-term effects in a larger population, ensuring continued safety. In some cases, drugs are withdrawn from the market due to severe side effects.
🔬 Overview of the Entire Drug Development Process
The final section summarizes the entire process of drug discovery and development. It recaps the major steps, from target identification and screening of thousands of compounds to pre-clinical and clinical trials, FDA approval, and post-marketing surveillance. The process is long and expensive, taking up to 15 years to complete. The importance of Phase 4 studies is highlighted, especially in detecting rare adverse effects after the drug is widely available. The video concludes by emphasizing the need for ongoing monitoring to ensure drug safety.
Mindmap
Keywords
💡Drug Discovery
💡Lead Compounds
💡Preclinical Studies
💡Clinical Trials
💡FDA Approval
💡High Throughput Screening (HTS)
💡ADME Profile
💡Post-Marketing Surveillance
💡Target Identification and Validation
💡Investigational New Drug (IND) Application
Highlights
The entire process of introducing a new drug to the market takes around 10 to 15 years and costs approximately 1.5 billion US dollars.
Thousands of potentially active compounds (5,000 to 10,000) are screened during drug discovery, with unsuitable compounds rejected at each step.
The process of new drug discovery involves two main stages: new drug discovery and new drug development.
During drug discovery, the goal is to identify lead compounds from 5,000 to 10,000 compounds screened.
Lead compounds are the potential drug candidates that are further optimized during the discovery process.
New drug discovery takes around 3 to 5 years, focusing on identifying potential candidates and optimizing leads.
The next stage, drug development, involves further screening of lead compounds in animals and humans to ensure safety and efficacy.
Only 5 compounds from the initial 250 generated in pre-clinical studies proceed to clinical trials.
Clinical trials are conducted in three phases (Phase I, II, III), taking 6 to 7 years to complete.
Data from clinical trials is compiled into a New Drug Application (NDA) submitted to the FDA for approval.
FDA review and approval of a new drug can take around 1 to 2 years.
After FDA approval, the drug is manufactured on a large scale and launched for the treatment of specific diseases or conditions.
Post-marketing surveillance, or Phase IV studies, monitor the safety of the drug once it's available to the public.
Post-marketing studies are essential for detecting adverse drug reactions, which may only emerge after a drug is prescribed to large populations.
An example of a drug withdrawn due to adverse effects is Sibutramine, which was removed from the market after it was found to increase the risk of heart disease and stroke.
Transcripts
[Music]
hello students
today's video is a very important video
in this video i will give a quick
overview or outline of the process of
new drug discovery and development
now this video is first in the series of
videos on new drug discovery and
development
now as we all know drugs are substances
that save lives and also improve quality
of life
introducing a new drug in market is a
highly complex process
it costs a pharmaceutical company around
1.5 billion us dollars and 10 to 15
years of research and development by
hundreds of research teams
now during discovery of a new drug
thousands of potentially active
compounds around 5000 to 10 000
compounds are screened unsuitable
compounds are rejected at each step
now the entire process of new drug
discovery and development has two main
stages or phases
the first stage is the new drug
discovery and the second stage is the
new drug development
now as we know five thousand to ten
thousand active compounds are screened
to get a new drug new drug discovery is
the process which aims at identifying
potential candidates from five thousand
to ten thousand compounds that are
screened
now these potential compounds that are
identified during drug discovery are
called as leads so leads or potential
drug candidates are identified during
drug discovery and the entire process of
new drug discovery takes around three to
five years
now next stage is the drug development
now drug development is the process of
further screening and evaluating lead
compounds which are generated during the
process of drug discovery now lead
compounds are further evaluated and
screened in animals and human beings so
that a new
safe and very effective drug could be
identified
its suitable formulation and dosage
forms are developed indications are
decided and if the new drug is approved
by fda it is manufactured on the large
scale and launched in the market for the
treatment of a specific disease or
condition
now the main steps during new drug
discovery are
the first step is the target
identification and validation now target
involved in a specific disease is
identified whose functioning could be
modified by a new drug to produce the
therapeutic effect
now once this target is identified it's
validated that is reconfirmation that
the target identified is correct
now next step is the screening of around
5 to ten thousand potentially active
compounds by high throughput screening
that is hds method hts screening methods
now from 5000 to 10 000 compounds around
100 to 200 compounds
specific for the target are selected and
these are called as hit these compounds
are called as hit so the next step in
the new drug discovery is the
identification of hit and these hits are
derived by the screening of 5000 to 10
000 active compounds
now from these 100 to 200 hits dozens of
lead compounds with affinity and higher
selectivity for the target are
identified now leads interact only with
the specific target excluding all the
other related targets so the next step
is the lead generation and optimization
now after the leads are generated they
are optimized
now
in the lead optimization lead compounds
are used as templates and from the lead
compounds more potential candidates are
designed by chemical modification
now these compounds which are produced
by chemical modification they possess
higher affinity higher selectivity for
the target they show good admi profile
that is absorption distribution
metabolism and excretion profile and
good structure activity relationship so
around 250 compounds
are designed from the lead compounds and
these 250 compounds that are designed
they enter the process of drug
development
now the process of new drug discovery
ends when the leads are generated and
optimized it is completed in around
three to five years and the next step is
the new drug development now around 250
compounds that were generated by the
chemical modification of leads are
further screened and are further
evaluated in animals and then in the
human beings now let's see what are the
different steps of drug development
now the first step here is the
pre-clinical studies now pre-clinical
studies refer to the testing of all
these 250 compounds in animals
now by the end of pre-clinical studies
only five compounds only around five
compounds are left to be further
screened all other compounds are
rejected and the process of pre-clinical
studies takes around one to two years
now findings of pre-clinical studies are
compiled
in a
investigational new drug application
that is ind application now this
application is submitted to fda that is
a food and drug administration for
review
now if application is approved by fda
all the five potential drug candidates
which were identified during the
pre-clinical studies are further
screened
and evaluated in human beings now these
studies in the human beings are called
as the clinical trials now clinical
trials are performed in three phases
phase one phase two and phase three
studies all three phases of clinical
trials are completed in around six to
seven years now data obtained from the
clinical trials is compiled in new drug
application and again submitted
to fda for review
now fda review takes around one to two
years
now if fda is satisfied with the safety
efficacy adme profile of the drug
official permission is granted by the
fda to launch the drug in the market
so drug is formulated indications are
decided it is manufactured on the large
scale and made available in the market
for the patients now the entire process
of
drug discovery to the launching of drug
in the market takes around 10 to 15
years
now following fda approval
fda once the drug is available in the
market fda requires pharmaceutical drug
companies to monitor the safety of drug
and the safety of drug is monitored
using the fda adverse event reporting
system
so this process is called as a post
marketing surveillance that is a very
close
monitoring of drug is dr is done to
ensure that the drug is safe
now these studies are also called as a
phase four studies which are conducted
when the drug is in market and available
to the prescribers and to the patients
now look at this figure
it gives a compiled schematic
representation of new drug discovery and
the development process now let's once
again quickly review the entire process
so uh the
drug discovery stage
the drug discovery involves
identification of target to be modified
by the drug to produce the therapeutic
effect then screening of around 5000 to
10 000 compounds active compounds and
identifying around 100 to 200 hits
further screening of hits to generate
the leads
and once the leads are generated these
leads are used as templates and around
250 molecules are designed by chemical
modification of the leads and the entire
process of drug discovery takes around
three to five years
now during uh drug development further
screening of
250 compounds derived
from the leads
are screened and evaluated first in the
animals and the studies are called as a
preclinical studies so during
pre-clinical studies pharma these are
the pharmacological studies that are
performed on the animals to determine
safety
and efficacy of all 250 compounds
then pharmacokinetic studies
evaluate uh administration distribution
metabolism and excretion that is adme
profile of all 250 compounds in the
animals while toxicity studies performed
during the pre-clinical studies
determine safe dose of the drug and the
dose range
so by the end of pre-clinical studies
almost all the drug candidates are
rejected and around five compounds are
left to be further screened so five
compounds are left at the end of
pre-clinical studies now the entire
process of pre-clinical studies takes
around one to two years now data that is
obtained from these preclinical studies
is compiled in investigational new drug
application that is ind application and
this application is submitted to fda for
review and approval
now further
if the fda finds investigational new
drug application satisfactory approval
is granted to conduct studies in the
human beings and thus clinical trials
are conducted
now clinical trials are conducted in
three phases namely phase one phase two
and phase three
and by the end of
clinical trials all the compounds are
rejected
and only one drug
candidate called as a new drug is
identified it is developed indications
are decided now data of these clinical
trials is compiled and new drug
application that is nda is submitted to
fda for again review
now all the three phases of clinical
trials are completed in around six to
seven years
now if fda
approves the new drug it's given
marketing approval grant of approval by
the fda is completed in about one to two
years
so now new drug is made available in the
market for the patients
now to further ascertain and confirm the
safety of new drug investigational
studies are continuously performed when
the drug is in the market
now the main name of these studies is to
report if any adverse drug reaction has
been observed with the use of new drug
now these studies are called as a
post-marketing surveillance or phase
four studies or the phase 4 4 trial
now when in market the drug is
prescribed to a large number of patient
population drug is studied in large
population and for a very long period of
time
and therefore it has been found that
several times some major adverse drug
reactions are detected only when the
product has been prescribed to
a large patient population
now many a times a drug is withdrawn
from the market because of serious is
adverse drug reactions for example
cybertron
and appetite suppressant now
cybertramine was withdrawn from the
market as it increase the risk of heart
disease and stroke so monitoring of
drugs safety is very essential when the
drug is in the market so phase 4 trials
are very important for the reporting of
adverse drug reactions especially uh
unpredictable adverse drug reactions for
example
idiosyncratic reactions so this is in
brief a quick overview of the entire
process of new drug discovery and
development if you find the video useful
kindly like subscribe and share this
video thanks for watching this video
[Music]
[Music]
you
Weitere ähnliche Videos ansehen
Drug discovery and development process
Introduction to Drug Development & Regulation
Introduction to pharmacology
From idea to medicine | Drug development at Roche
What Students Need To Know About History of Pharmacovigilance
How AI is accelerating drug discovery - Nature's Building Blocks | BBC StoryWorks
5.0 / 5 (0 votes)