MEDICINE DISCOVERY - 3 YEARS
What kind of medicines could do the job?
Compounds are a chemical mixture which may change the way the body works.
- Scientists look at existing compounds or make something from scratch.
- Finds which compound may act on the target to treat disease.
- A new medicine can also be a protein, such as antibody, or even a therapy that alters faulty genes.
PHASE 1 CLINICAL TRIAL - 6 ~ 12 MONTHS
How is it tolerated for people without the condition?
Phase 1 trials often rely on healthy people to volunteer to test a new medicine, to show that it is safe for further studies.
- This involves use of a medicine in low, single doses in a very small number of humans without the condition for the first time.
- The tests find out how the body reacts to the medicine.
PHASE 3 CLINICAL TRIAL - APPROX 3 YEARS
Does the medicine work in larger, and more diverse populations?
- Compares the new medicine to current treatments and / or placebo across different locations (several countries) for several years.
- Explores if the new medicine gives more benefit to patients compared to current treatments and further assesses safety.
NHS APPROVAL
Can the medicine be used in the NHS?
- It needs to show good health benefits and be cost effective.
- Reviewed by national body who gives advice to improve healthcare e.g. the National Institute for Health and Care Excellence (NICE) in England.
- Review allows drug to be prescribed by doctors in NHS or not.
PRE-DISCOVERY - 5 ~ 7 YEARS
What are the problems caused by the disease?
- Scientists work to understand what causes the disease and the mechanisms involved.
- From this work scientists are able to propose possible treatment targets (for example: proteins or genes).
PRE-CLINICAL TESTING - 2 ~ 3 YEARS
Does the new medicine work under different tests in the laboratory?
- Rigorous testing takes place in the laboratory. Scientists want to see if the new medicine works on the intended target, whether it works well and whether it passes safety checks.
- The medicine is tested to see if it works to show health benefit and has minimal harmful effects.
PHASE 2 CLINICAL TRIAL - MONTHS TO YEARS
How does it work for people with the condition?
Placebo is identical in appearance to the medicine, but has no active ingredient
- Sees if the medicine shows benefit in the relatively small clinical trial and if it is tolerated well by people with the disease.
- Compares the new medicine to a placebo or another existing medication used in the trial.
- This is more complex in certain groups e.g. children and young people.
LICENCING APPROVAL - UP TO 2 YEARS
How is it approved by national regulators?
In the United Kingdom, licensing approval is completed by the Medicines and Healthcare Products Regulatory Agency (MHRA).
- Regulators review the data from the trials to decide if the medicine can be approved to treat people in real life.
- A licence to sell the medicine is granted.
REAL WORLD EVIDENCE - TRIALS AND BEYOND
How is this medicine monitored over long periods of time?
- After a medicine is available to prescribe, its real-world results are continually monitored.
- This ensures the medicine continues to show the same benefits and safety profile as seen in the clinical trial.
DID YOU KNOW?
Any new medicines are developed through long, costly and challenging research before they can be used by patients. The main reasons a medicine is not used include; it does not work, the side effects are too significant, and it is too expensive to prescribe.
It can take up to 13 years from initial development of a medicine to it being available to patients in the clinical setting.
Out of every 10,000 new compounds that researchers start work on, only around 1 or 2 become a useful medicine. This explains why it can be difficult to get a drug to a person who may need it.
It costs £1.15 billion to produce a new medicine from scratch.
THE FUTURE
OF DRUG DEVELOPMENT
Artificial Intelligence may allow a faster and more efficient approach to drug discovery.
Licensing sometimes takes years, but now processes are becoming more streamlined whilst maintaining strict regulations.
Precision medicine aims to improve response rates by matching specific medicines to target cellular pathways that might drive disease subtypes in specific groups of patients.
Changes in trial design now allow a wider range of patients to be able to access new medicines through clinical trials than has previously been possible.
For Juvenile Idiopathic Arthritis (JIA), previously trials were limited to only particular types of JIA. Trial recruitment has now broadened to allow more subtypes of JIA to be involved.
Research collaborations such as CLUSTER can influence all stages of this process i.e. in identifying targets, modifying trial design, and influencing regulatory guidelines.
Produced by the CLUSTER Champions and the CLUSTER Industry Committee on behalf of the CLUSTER Consortium. Find out more: www.clusterconsortium.org.uk
References
| EFPIA. EFPIA The Pharmaceutical Industry in Figures Key Data 2021; Available from: https://www.efpia.eu/media/602709/the-pharmaceutical-industry-in-figures-2021.pdf.
| Richards, N. and I. Hudson, UK medicines regulation: responding to current challenges. Br J Clin Pharmacol, 2016. 82(6): p. 1471-1476.
| FDA. The Drug development process. 2018; Available from: https://www.fda.gov/patients/learn-about-drug-and-device-approvals/drug-development-process.
| Anderson, M., et al., Promoting innovation while controlling cost: The UK’s approach to health technology assessment. Health Policy, 2022. 126(3): p. 224-233.