Full summary of the subject 'Preclinical drug research taught by prof. Van Cruchten & prof. Delputte. Both the general lessons as the guest lessons 'biopharmaceuticals' are included in this summary.
Nikita Cassimon 2e master geneesmiddelenontwikkeling: biofarmaceutische wetenschappen
Preclinical Drug Research
Introduction:
Molecule Drug
Up to 12 years
Costly
Depends on target population and market
Small molecules
• SMI = Small Molecular Entities Ex. aspirin, fluoxetine,
Chemicals
• Oral administration omeprazole
• For many diseases not a solution
Complex molecules Ex. artemether, paclitaxel,
Natural products
vinblastine
Large (complex) molecules
• Also: New Therapeutic Modalities =
Biologicals biopharmaceuticals + gene therapy Ex. insulin, interferon, EPO
included
• Parenteral administration
Drug R&D spending in different industry sectors & therapeutic areas:
• Biggest cost for • Spendings in • Smaller countries, smaller
biopharmaceuticals pharmaceutical companies expenses
because of small target increase • Cost of bringing a new SMI
population • Not many drugs are or biological to the market
• Ex. 200k euros for 1 approved → if approved it keeps increasing over the
administration in 1 patient needs to compensate for al years
the expenses and failures
• ‘Fail fast Fail cheap’
Major disease areas:
1. Cardiovascular diseases
2. Neoplasm (cancer)
• 3. Respiratory system diseases
• 4. Digestive system diseases
5. CNS diseases
,Nikita Cassimon 2e master geneesmiddelenontwikkeling: biofarmaceutische wetenschappen
R&D spending & therapeutic areas:
• Biggest investment: clinical trials → facilities in the world and amount of patients
• Need for a nonclinical package depends on the patients
• Phase I CT: healty young males people → no women: potential fertilty danger + abortion →
recovery spermatogenesis after a few months
• Conclusion: risk in men is lower + smaller clinical package (less data needed)
Other factors:
Annual population growth Healthcare spending in relation Pharmaceutical sales by
rates: to age: region:
• Ideal: lot of target patients • Diseases/treatments • Most sales: USA → a lot of
• China: boost of pharma increase with ages OTC in supermarkets
companies → competition • You don’t want: adverse • Emerging: China
• Latin America: less effects → • Established ROW: India,
investment subtherapeutic dose Brazil
• Africa: increasing trend • = RISK/BENEFIT-ratio
Total cost for one new marketed drug = about 0.8 – 1.5 billion USD (depends on therapeutic area)
Orphan diseases = diseases occuring in a low amount of patients → definition depends on the place
and amount of patients (< 1000)
• Pharmaceutical companies don’t make profit with the development or drugs → you can make
profit if you’re ‘first-in-class’
• Investment by the Bill Gates foundation
• Some bigger companies tactically buy smaller companies to make profit
Compensate
investment by
making profit after
Decrease of profit
launching the drug
after expiring
patent + generic
competition
Investment in clinial
development → no
profit is made here
, Nikita Cassimon 2e master geneesmiddelenontwikkeling: biofarmaceutische wetenschappen
Blockbuster drug = a drug with sales > 1 billion USD/year
• Ex. Humira: 20 USD/year
• Blockbuster drugs moslty come from bigger companies, because they can afford a big investment
• Detectives visit smaller companies and detect drugs with potential,bigger companies later buy
them or steal the idea and license it
Top launches 2019: Patents: Competition in pharmaceutical
• Increase of • Patent will expire after 20 market:
biopharmaceuticals years, but 12 years are • Most profit fort he ‘first-in-
• Nonclinical package needed to develop drug → class’ developper
specialized for monoclonal profit for 8 years • Shrinking period of market
AB • SPC = supplementary exclusivity
• Gene therapy → protection certificate → • Inceased competition
companies decide what max. +5 years extra between pharmaceutical
refund the patient will get patented companies
Ex. Zolgensma • Me-too drugs = drugs die
op elkaar lijken
• Generics: high volume, high level of
competition, low value product, low medical
differentiation
• Mega-blockbusters: everything medium
• Targeted therapeutics: low volume, low level
of competition, high product value, high
medical differentiation
Failure rates in drug development:
‘Fail Fast Fail Cheap’
Phase I: No adverse
effects in young healthy
men
Approved: post-marketting
Phase II: Lack of efficacy issues → impact on image
on bigger scale of company
, Nikita Cassimon 2e master geneesmiddelenontwikkeling: biofarmaceutische wetenschappen
Biotechnology-derived medicines:
• Oligonucleotides working on mRNA
• Small interfering RNAs Larger proportion of
• Micro RNAs ‘first-in-class’
• Vaccines therapeutics
• Monoclonal antibodies
Ex. In 1982: Recombinant human insulin or Humulin®
Major pharmaceutical companies (from biggest to smallest):
• Locations: 150 countries • Locations: > 140 countries and > 100,000
• Headquarter: Basel, Switzerland employees
• Diagnostics • Headquarter: Basel, Switzerland
• Sandoz for generics
• Locations: > 150 countries • Locations: 120 countries
• Headquarter: New York, USA • Headquarter: Whitehouse Station, New
• Belgian sites: Puurs (no R&D but Jersey, USA
manufacturing)
• Offers a broadspectrum of drugs
• Drugs not approved for humans used for
animals
• Locations: > 55 countries • Locations: > 100 countries and > 100,000
• Headquarter: New Brunswick, New Jersey, employees
USA • Headquarter: Paris, France
• Beerse: R&D, pharmaceutical production • Ablynx is bought by Sanofi in 2018
(tablets, ointments, e.a.)
• Geel: chemical production (API)
• Olen: pharmaceutical production
• Locations: 117 countries • Locations: mainly Japan, but also
• Headquarter: UK Switzerland, USA, UK and Singapore
• OTC and toothpaste focused • Headquarter: Osaka, Japan
The benefits of buying summaries with Stuvia:
Guaranteed quality through customer reviews
Stuvia customers have reviewed more than 700,000 summaries. This how you know that you are buying the best documents.
Quick and easy check-out
You can quickly pay through credit card or Stuvia-credit for the summaries. There is no membership needed.
Focus on what matters
Your fellow students write the study notes themselves, which is why the documents are always reliable and up-to-date. This ensures you quickly get to the core!
Frequently asked questions
What do I get when I buy this document?
You get a PDF, available immediately after your purchase. The purchased document is accessible anytime, anywhere and indefinitely through your profile.
Satisfaction guarantee: how does it work?
Our satisfaction guarantee ensures that you always find a study document that suits you well. You fill out a form, and our customer service team takes care of the rest.
Who am I buying these notes from?
Stuvia is a marketplace, so you are not buying this document from us, but from seller nikitacassimon. Stuvia facilitates payment to the seller.
Will I be stuck with a subscription?
No, you only buy these notes for $17.38. You're not tied to anything after your purchase.
