The process of getting a new drug all the way from the laboratory to the pharmacy shelf is lengthy (often 10-15 years) and expensive, with typical costs of well over £1 billion.
Before any initial compounds are even prepared, the research journey may have begun in a university laboratory, where researchers may have undertaken basic research to understand the processes behind a disease at a cellular or molecular level. Through better understanding of disease processes and pathways, targets for new treatments can be identified. Once a potential target has been identified, researchers can then try to devise a substance that acts on this target in a selective, efficacious and non-toxic manner.
Historically, researchers have sometimes looked to natural compounds from plants, fungi or marine animals to provide the basis for candidate drugs. Even today, some interesting new drugs are based on compounds from plants such as cannabis.
Increasingly however, scientists are using knowledge gained from the study of genetics and proteins to create new molecules. In recent years, the use of artificial intelligence to assist in the earliest phase of drug discovery has started to receive attention.
Once a promising new compound or biologic treatment is identified, the next stage involves carefully controlled clinical trials.
Many drugs do not make it through the clinical phase. The US medicines regulator, the FDA, estimates that whilst 70% of candidate drugs make it through phase 1 trials, only around a third of these pass through phase 2 successfully. Then, only 25-30% of these get through phase 3 trials and continue towards a marketing approval.
Given that drug development requires many years of careful research and clinical testing, while at the same time carrying a high degree of risk and cost, law-makers in many countries have recognised the need for extra IP protections to incentivise innovator pharma companies and to help recoup the enormous costs that are involved. These IP protections include the acceptability of ‘second medical use claims’ in patents, as well as different forms of regulatory exclusivity and patent term extensions.
Of course, efforts to innovate do not stop when a drug wins an initial approved for a given disease condition. Frequently, there is scope for further improvement: An improved formulation might improve an aspect of tolerability or patient compliance. With further trials a drug might be found to be useful against a broader segment of disease than was initially envisaged. Innovative improvements may also be found by combining two drugs – perhaps with a specific dosage and sequencing of administration that maximises the efficacy and/or tolerability against a given type of cancer.
The innovative pharma and life-sciences sector is making great strides forward to meet society’s medical challenges – but certainly, there is so much still to do.
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