1. A journey through drug discovery The life cycle of a new medicine
It’s difficult to discuss improving pricing and affordability in healthcare without first understanding how a new medicine is developed.
© Prof. David Webb, Edinburgh (2016)
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2. Discovery Target selection to lead compound Biology of disease
The initial discovery of a potential new medicine relies on scientists having a good understanding of the biology of a disease and how to treat it, for example by selecting a key target in the body such as a receptor, enzyme, etc. Based on this expertise, scientists then take forward a selection of compounds that seem likely candidates for developing into a new medicine.
Created by Prof. David Webb, Edinburgh (2016)

3. Discovery Development Preclinical testing Phase I Phase II Phase III
Biology of disease
Target selection to lead compound
Preclinical testing
In vitro/in vivo models
Safety studies
Phase I
Phase II
Phase
III
This potential medicine is then taken forward into the lab for ‘pre-clinical testing’: part of the journey that tests how a compound works and whether it is safe – in cells and tissues, and if a compound passes successfully through these earlier stages, it is required by law to be tested in two species of animal before being testing in humans.
If that work goes well, the best performing compound starts the first of phases of drug development – each of which tries to understand more about how the compound works in humans:
Phase 1 often involves healthy volunteers who are monitored to see how well they tolerate the compound and how their bodies distribute and dispose it.
Phase 2 is the first chance to confirm whether the potential medicine works in real patients with the disease being targeted, and to get an idea of the the right dose.
Phase 3 is about proving the potential medicines works in large numbers of patients, with acceptable safety – although only the most common side effects will be detected at this stage.
After the first three phases are complete and everything is going well, a company will seek a license for the medicine to be used by patients.
Created by Prof. David Webb, Edinburgh (2016)

4. Discovery Development Marketing Preclinical testing Phase I Phase II
Biology of disease
Target selection to lead compound
Preclinical testing
In vitro/in vivo models
Safety studies
Phase I
Phase II
Phase
III
Phase IV
Phase 4 – after a medicine is licensed – is when a process called pharmacovigilance identifies rarer side effects and new patient groups that may benefit emerge.
These phases haven’t changed for many decades: the second half of the 20th century was a ‘golden era’ that improved – and in many cases saved – millions of lives, through the development of new antibiotics and treatments for cancer and heart disease. We’re now in an era when the diseases being targeted by scientists are more complex, and it is getting more challenging and costly to generate new medicines.
Created by Prof. David Webb, Edinburgh (2016)

5. Discovery Development Marketing 10,000 250 5 1 Compound attrition
Biology of disease
Target selection to lead compound
Preclinical testing
In vitro/in vivo models
Safety studies
Phase I
Phase II
Phase
III
Phase IV
To get to one new, licensed medicine, there’s an enormous amount of effort and investment: initial discovery work may generate 10,000 or so compounds but only around 250 are successfully taken into preclinical testing - and then of these, if around five reach Phase 1 of clinical development, on average only one new medicine will emerge successfully at the end of Phase 3.
Created by Prof. David Webb, Edinburgh (2016)

6. Discovery Development Marketing 10,000 250 5 1 Compound attrition
Biology of disease
Target selection to lead compound
Preclinical testing
In vitro/in vivo models
Safety studies
Phase I
Phase II
Phase
III
Phase IV
Generics
Clinical
trials
authorisation
Value
for
money?
Patent
expiry:
20 yrs
Patent
lodged
Marketing
authorisation
Patent protection is normally sought as late as possible, because development can take many years and the patent will expire after 20 years, at which point other companies can manufacture “generic” versions, usually at much lower cost.
In order to move from the pre-clinical testing to Phase 1 in the UK, a clinical trials authorisation has to be sought based on scrutiny of the available data. If Phases 1 to 3 are successful, then these findings will be submitted to gain a marketing authorization, which allows the medicine to be launched.
There have always been these “hurdles” for the pharma industry (safety, efficacy and quality), but now we have to add to that cost-effectiveness or value for money, which is assessed by NICE in England and Wales, and SMC in Scotland, and only if this hurdle is overcome is the company likely begin to recoup its investment.
Created by Prof. David Webb, Edinburgh (2016)

7. Discovery Development Marketing 3-6 years 6-8 years 6-10 years 10,000
Compound
attrition
10,000
250 5 1
Discovery
Development
Marketing
Biology of disease
Target selection to lead compound
Preclinical testing
In vitro/in vivo models
Safety studies
Phase I
Phase II
Phase
III
Phase IV
Generics
Clinical
trials
authorisation
Value
for
money?
Patent
expiry:
20 yrs
Patent
lodged
Marketing
authorisation
It can take some time to do complete the drug discovery and development phases, which means that a company often has only 6–10 years (after marketing authorisation and before patent expiry) to recoup the investment in this twenty or more year programme: this includes all the cost of development, plus a large opportunity cost associated with up-front investment in a medicine that doesn’t delivery any returns during this time (often up to 10–15 years).
3-6 years
6-8 years
6-10 years
Created by Prof. David Webb, Edinburgh (2016)

8. Discovery Development Marketing 3-6 years 6-8 years 6-10 years 10,000
Compound
attrition
10,000
250 5 1
Discovery
Development
Marketing
Biology of disease
Target selection to lead compound
Preclinical testing
In vitro/in vivo models
Safety studies
Phase I
Phase II
Phase
III
Phase IV
Generics
Clinical
trials
authorisation
Value
for
money?
Patent
expiry:
20 yrs
Patent
lodged
Marketing
authorisation
On this basis, an American university recently estimated the cost of developing a single successful medicine at more than 2.5 billion US dollars, or just under 2 billion pounds.
3-6 years
6-8 years
6-10 years
Cost for one marketing authorisation: Over $2.5 billion (Tufts report March 2016)
Created by Prof. David Webb, Edinburgh (2016)

9. Created by Prof. David Webb, Edinburgh (2016)
Thank you
The British Pharmacological Society is working with its over 3,500 members worldwide to improve the development of new medicines, so patients can benefit from quicker and affordable access to treatments: from improving open access through out publications to findings from all phases of drug discovery, to supporting education and training for the next generation pharmacologists, and engaging the public in discussions about the affordability and value of new medicines.
Visit for more information.
Created by Prof. David Webb, Edinburgh (2016)

10. A journey through drug discovery The life cycle of a new medicine
Created by Prof. David Webb, Edinburgh (2016)