Even if two people are diagnosed with the same disease, it does not mean that they should be treated in the same way. This concept is called personalized medicine. Personalized medicine, also known as precision medicine, is an emerging approach that uses a person's biological profile (e.g. genetic, protein) to guide decisions regarding treatment, diagnosis and prevention of disease. Personalized medicine will transform the healthcare system by adapting treatment to each individual, rather than following the current "one medicine fits all" conventional therapy. Many patients may have the same diagnosis, but the nature of their disease may be different. By exploring the unique disease characteristics specific to each individual, personalized medicine aims to design tailored treatments that ensure more successful outcomes with fewer side effects.
The field of personalized medicine is expanding rapidly, with an increasing number of targeted therapies reaching the market. However, the development of more advanced technologies is required to meet the time and cost challenges of implementing more targeted therapies as part of clinical practice. Nevertheless, rapid progress in this field points towards a time when precision medicine will be part of routine medical care to target the right treatment to the right patient at the right time.
A biomarker, also known as a biological marker, is a measurable indicator of a biological condition. Biomarkers can have molecular, histological, radiographic or physiological properties. Examples of biomarkers can include everything from blood pressure and heart rate to MRI findings and complex histological and genetic tests in blood, body fluids or tissues. These act as indicators of specific physiological conditions. The discovery of a biomarker can be a sign of a normal or abnormal process, or a condition or disease.
There are different types of biomarkers, including diagnostic biomarkers that identify the presence and type of disease, prognostic biomarkers that provide information about the patient's overall disease course with or without standard treatment, and predictive biomarkers that help identify which treatment the patient is likely to respond to or benefit from. Biomarkers are becoming increasingly important in medicine, especially in personalized medicine. They play a crucial role in predicting prognosis and treatment response for therapies patients may receive in the future. This means that the biomarker can influence the outcome of the treatment.
Therefore, biomarkers are crucial for selecting appropriate treatment for patients with specific drugs, such as targeted therapies. This enables personalized medicine, where patients receive the right treatment tailored to their individual profile, with the right dose and at the right time. Biomarkers play an essential role in ensuring successful treatment outcomes with fewer side effects.
Emerging therapies represent innovative therapeutic approaches that show promising results based on previous research findings or clinical trials, but have not yet been established as standard treatment for patients.
A standard of care is a guideline that specifies the appropriate treatment for a patient with a particular condition and is widely accepted and recognized among healthcare professionals. Appropriate treatment options can range from the administration of drugs to surgical procedures or the use of medical devices, depending on the type of disease.
The latest technological advances and scientific discoveries are driving research into new diagnostic and treatment options. Sequencing technologies enable rapid characterization of a patient's complete DNA, allowing researchers and healthcare professionals to obtain a specific disease profile for each individual patient. This specific disease profile provides deeper insight into both the mechanisms behind the disease and enables customized treatment approaches that are most likely to benefit the individual patient (targeted therapy).
Personalized medicine aims to deliver a specific treatment to a patient with a specific profile, at the right dose and at the right time. New treatment options use these sequencing technologies to explore the application of innovative therapeutic approaches in a personalized way compared to existing conventional treatments.
Clinical trials differ from 'preclinical' trials in that they involve human subjects. These studies often test new drugs, combinations of drugs, medical devices or surgical methods. The aim may also be to find new ways to use existing treatments or to change behavior to improve health and quality of life.
Clinical trials help discover whether new treatments, preventive measures and behavioral changes are safe and effective in human volunteers. The first patients to receive a new and promising treatment are the participants in these trials. Clinical trials provide hope for many people and an opportunity to help researchers find better treatments for others in the future.
In the US, clinical trials are a legally required pathway for new drugs, diagnostic tests or medical devices. This means that only products that have been licensed can be legally sold to treat human diseases. Similar laws exist in most countries around the world. Agencies such as the FDA in the US or the EMA in the European Union decide whether a medical product should be licensed for use.
For a product (e.g. drug, test, device, etc.) to be approved by the FDA or EMA, it must undergo three phases of clinical trials (phases 1 to 3). Usually, several separate studies of the product are conducted in each phase. Each study has its own predefined experimental design. The institutions supporting the studies will make decisions along the way whether to continue with the product or withdraw it from the process. If all three study phases are completed to the sponsor's satisfaction, the results from each study (along with all supporting preclinical data) are submitted to the agency for evaluation.
Phase 1: These are usually studies of a product (usually a drug candidate) that has not previously been tested in humans. Only a couple of studies are usually planned per new product, and a limited number of participants are recruited for each study (there are no absolute rules, but usually around 20 to 30). The purpose is to document side effects (establish safety), find optimal dosing and may also measure drug excretion and metabolism.
Phase 1/2: These trials can be designed to take patients through a process depending on outcomes, and can effectively tie together phases 1 and 2.
Phase 2: Efficacy is the key objective in Phase 2 trials. At this stage, the tested drug is usually compared with other therapeutic approaches. Phase 2 trials typically recruit over 100 participants.
Phase 3: Once the phase 1 and 2 trials have established satisfactory safety and effectiveness for the study's sponsors, they may decide to progress the new drug to phase 3. The goal at this stage is to see if what was learned in phases 1 and 2 also applies to a larger group (often more than 1,000 participants).
Phase 4: Yes, there are "aftermarket" studies of drugs and other medical products that have already been licensed for sale by the FDA or relevant agencies. The aim here is to continue to collect efficacy and safety data from the regular use of the drug (and in some cases other products). These studies may provide new insights into how the therapy can be used in the best possible way. In some rare cases, Phase 4 may reveal safety or efficacy concerns that could lead to withdrawal of approval.
Phase not applicable: This includes studies without phases defined by the FDA, including studies of medical devices or behavioral interventions that are not seeking marketing approval. There are many studies that fall into this category. Some of these studies test different aspects of treatments, such as how instructions are conveyed or how a particular procedure works under different circumstances, etc.
Participation in trials: Clinical trial sponsors certainly want participants, but that doesn't mean that every single case fits their needs. Study descriptions state specific criteria for inclusion or exclusion of participants. The overarching principle of these criteria is that a participant should have at least some possibility of successful treatment with the tested therapy.
