Scientific discoveries hold the potential to address unmet medical needs and improve patient outcomes. However, the transformation of a novel scientific finding into a marketable drug is a complex and lengthy process that involves rigorous testing, regulatory approvals, and significant investments. While the timeline for drug development varies, the ultimate goal is to bring safe and effective treatments to patients. In this blogpost, we take you on the journey of a scientific discovery from the laboratory bench to the patient's bedside and explain the difference between a druggable target and a biomarker.
What is the aim of drug development?
A drug can be developed for different outcomes. Examples include:
- curing a disease or symptom (e.g. cure Long COVID or fully heal fatigue)
- making a disease easier to bear by reducing number, intensity or frequency of symptoms (e.g., less intense or less frequent headaches)
- masking one or several symptoms to enhance quality of life with the disease (e.g. pain killers don’t fix a broken arm but can make it a lot easier to endure)
All phases follow the main question: is the drug effective and is it safe?
It is somewhat intuitive what a safe drug is: it should have minimal side effects, or at least not be more harmful than the disease or symptom it was developed to treat. Efficacy simply means that the drug has to “work”. It has to be better than no treatment or other treatments that are already available or cause less side effects than the other options.
Phase 0: Discovery and Preclinical Research
The journey often begins with a scientific breakthrough. Researchers identify a potential drug target – a molecule, protein, or biological process associated with a particular disease. This discovery can stem from years of basic research in academic institutions, government laboratories, or private companies.
Once a target is identified, researchers embark on preclinical research to assess its feasibility as a drug candidate. This phase involves laboratory experiments, animal studies, and extensive testing to understand the compound's safety, efficacy, and potential side effects. If the results are promising, the drug candidate moves on to the next phase. Typically, this Phase 0 can last several years, depending on the complexity of the research and the success of early experiments.
Phase I: Initial Human Trials
With promising preclinical results, the drug candidate advances to Phase I clinical trials. These trials involve a small group of healthy volunteers and aim to evaluate the safety, dosage, and potential side effects of the drug in humans. Phase I trials generally last a few months, but the timeline may vary based on the nature of the drug and the results obtained.
Phase II: Expanded Clinical Trials
If the drug successfully passes Phase I, it progresses to Phase II clinical trials, where it is tested on patients with the target disease. These trials aim to assess the drug's efficacy, optimal dosage, and potential risks and benefits in the intended patient population. Phase II trials typically take a few years, depending on the complexity of the disease and the recruitment of suitable participants. It is one of the phases where ongoing clinical Long COVID trials are currently recruiting patients.
Read Part 2 of this Blogpost for Phases III and IV and the differences between a druggable target and a biomarker.
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