lecture 1.1 (R&D) Flashcards
What is the BioPharmaceutical industry?
An industry which focuses on research, development, manufacturing, marketing and distribution of pharmaceutical products in the context of healthcare.
Both ethical and OTC products can be branded or generic.
true or False
True
Give a few examples of ethical and OTC products
OTC: panadol, Tylenol, paracetamol
Ethical: lipid-lowering drug -Lipitor
Atorvastatin (generic)
Give an example of a class of ethical drug which is generic
Atorvastatin
What are the 4 types of industry players in the pharmaceutical industry?
- Big pharmas (e.g Pfizer, Novartis, Merck, Roche)
- Branded OTC products
- Generics
- Biotechs
Name some third party vendors for research and development and manufacturing support in the pharmaceutical industry
Contract Research Organisations (CROs) –> provide support for early stage research and development
Contract Manufacturing Organisations (CMOs) –> manufacture drugs for pharmas and biotech companies
Name a CRO (contract research organisation),
IQVIA
Name a CMO (contract manufacturing organisation)
Thermo Fisher Scientific
What is the industry approach to doctors/hospitals?
Sales force and marketing with medical services support, Multichannel marketing / Omnichannel marketing.
What is the industry approach to pharmacies?
Trade Deals. Discounts/ rebates. On site promotion. Posters
What is the industry approach to government hospitals and clinics?
Multi-level approach: sales and marketing, medical services / clinical trials, logistic support, cost benefit analysis (pharmacoeconomics).
What is the industry approach to public?
Advertising, Internet, Social media, Use of celebrities etc, company patient clubs
What is the value of medicine?
- save lives and increase life expectancy
- improve quality of life
- new drugs and vaccines to reduce total health care costs
- contribute to economy
What are some cancers that have had a reduction in occurrences since innovations in Pharma over the past couple of decades?
Lung cancer
Breast cancer
Colorectal cancer
prostrate cancer
How has biopharma benefitted society over the past few decades?
- 35% increase in life expectancy from 1990-2015
- Covid 19 crisis: 1.6mln lives saved through vaccines and drugs
- heart failure and heart attack rates declining
- reduce health care costs
How do medicines and vaccines produced by pharmas contribute to reduction in health care costs?
Newer drugs shorten hospitalisation, improve remission rates and save total treatment costs
Give some examples of medicines or vaccines reducing health care costs.
- statins cutting heart attack surgeries
- new hepatitis C drugs reduce liver transplants
- newer antidepressants cutting cost on psychiatrists
What is the contribution of biopharma to world economy according to the IFPMA 2024 report on economic impact of the global pharma industry?
- contribution of USD 2,295 bln in 2022
- support 74.9mln jobs
What are some breakthroughs in biopharma industries in 21st century?
- Recombinant technology used for production of very high-quality Insulin (biologics)
- Monoclonal antibodies for cancer treatment
- Newer Cancer therapies like Immunotherapy (Keytruda)
- Antisense therapy for rare diseases which are untreatable in the past
- New vaccines and drugs for Covid 19 pandemic.
Rank the biggest pharmaceutical markets
- North America
- Europe
- Africa, Asia and Australia (excluding China and Japan)
- China
- Japan
- Latin America
What are the top 3 global therapeutic areas?
- oncologies
- anti-diabetics
- immunology
What were the top 10 global Pharma products in 2023?
- keytruda
- comirnaty
- Humira (for rheumatoid arthritis)
- ozempic
What are the sequence of events in the discovery process?
- pre-discovery
- target identification
- target validation
- drug discovery
- early stage –> pharmacokinetic and toxicology test in animal, living cells and computational models
- lead optimisation
- preclinical testing –> test safety of drugs on animals
What is the purpose of phase 1 clinical trial?
to discover if the drug is safe
e.g. pharmacokinetics, pharmacodynamics, side effects
How many test subjects are there in phase 1 clinical trials?
20-100 HEALTHY volunteers,
usually conducted in hospitals
What is the purpose of phase II clinical trials?
- To evaluate the effectiveness of the drug in treating an illness or medical condition
- To examine the safety, possible side-effects and potential risks.
- To analysis the optimal dose and schedule of using the drug
How many test subjects are there in phase II clinical trials?
100 to 500 patients with disease or condition under study.
What is the purpose of phase III clinical trials?
- To test in a large group of patients to show safety and efficacy of the drug.
- Comparing with existing treatments or placebo
- Provide primary basis for benefit-risk assessment and core information.
How many test subjects are there in phase III clinical trials?
Patient no. about 1000 to 5000 from multiple sites with many investigators.
What happens after completion of all 3 phases of trial proves that the drug is safe and effective?
submit to Health Authorities for marketing approval.
What is required for new drug application and approval requirement?
- registration dossiers: clinical and manufacturing data
- certificate from country of origin and referenced countries
- local clinical trial (if required)
- GMP requirements
5.Bioavailability/Bioequivalent studies (BABE) required for some generic product registration
What are Bioavailability/Bioequivalent studies (BABE) ?
Bioavailability – rate and extent to which the active ingredient of a drug is absorbed and becomes available at the site of action
Bioequivalence – compares the bioavailability of the active ingredient from two different drug formulations.
What are the ‘drug-approving’ agencies in USA, Europe, China and Hong Kong?
- In USA – US Food and Drug Administration (USFDA)
- In Europe – European Medicines Agency (EMA)
- In China – National Medical Products Administration (NMPA)
- In Hong Kong – The Department of Health (DoH)
What is the purpose of phase IV clinical trial?
Post marketing studies – to collect additional information of the drug about benefits, optimal dose or possible risk.
–> harmful side effect observed –> product withdrawn from market
Name a drug which was withdrawn from market after phase IV clinical trial
Vioxx
an anti- inflammatory and pain killer, was found to have serious cardiac side effects in phase 4 trials and was withdrawn from market.
What is the average cost of developing a drug?
USD 3.5 bilion
pharmaceutical companies spend 15-30% on R and D
What are the current global trends in R&D?
- More R&D investment in new technologies like biopharmaceuticals eg. biologics, gene and cell therapy.
- Collaborate with outside partners like research centers , universities and biotech companies, including small emerging biopharma companies for new drug discovery.
- Focus more on top product candidates with foreseeable high sales/profit potential and higher probability for commercialization like anti cancer drugs.
The WHO GMP for pharmaceutical regulators in developing countries is less strict.
True or False
True
What is GMP’s objective?
To reduce risks inherent in pharmaceutical production to ensure adequate safety and quality of drug such as
- contamination (contagious microbes)
- cross-contamination
- mix ups
- wrong labelling
What are some guiding principles in manufacturing of pharmaceuticals?
- Hygiene of the manufacturing facilities.
- Prevention of cross contamination of drugs.
- Manufacturing process clearly defined and controlled.
- Records well kept during manufacturing.
- Well trained workers.
- Records of distribution from batch to batch.
- Recall system in place.
