Stratified (personalised) medicine

Site: EUPATI Open Classroom
Course: Role of Pharmacogenetics / Pharmacogenomics in the Development of Medicines
Book: Stratified (personalised) medicine
Printed by: Guest user
Date: Friday, 19 July 2024, 11:31 PM

1. Same Symptoms, same disease, same treatment?

(This section is organised in the form of a book, please follow the blue arrows to navigate through the book or by following the navigation panel on the right side of the page.)

Patients that are diagnosed with the same disease have generally been thought to have the same root cause. Doctors therefore often give these patients the same treatments. However, clinical experience tells us that patients do not all respond in the same way.

In reality, patients diagnosed with the same disease can have very different experiences with their disease. For example, the disease might develop slowly or quickly. It might be more or less severe or it might be chronic (long-term). In addition, different patients may respond very differently to the same treatment:

  • Some will respond well.
  • Some will respond, but less well.
  • Some will not respond.
  • Some may have serious side effects (adverse reactions).
  • Some may develop ‘resistance’ to the medicine (they stop responding) - this can happen even if they responded well at first.

Doctors try to take into account various clinical and lifestyle factors when they treat individual patients. However, often the only option is to try out a treatment, and monitor the patient’s response. If necessary, the dose, schedule or medicine can be changed.

Today, we know that these patients are probably affected by ‘different’ diseases. In other words, patients can show the same symptoms but the symptoms can be caused by different processes in their cells (they have a different underlying pathology). These differences can have a great effect on how a disease will progress, and which is the best treatment.

This is an important change in the way to diagnose and treat disease.

2. New Understanding of Diseases

This section gives an overview of the research areas that are driving forward personalised medicine. More detail on specific technologies (such as ‘omics’) is given elsewhere in this training unit.

3. Stratification versus Personalised

As mentioned in the introduction, the term personalised medicine includes both stratification and personalised medicine as it is used at the moment. The terms are often used as synonyms. The difference could be explained with the example presented in the Toolbox

4. Challenges

Personalised medicine requires that researchers develop more targeted medicines for a smaller patient group. These are likely to be used by fewer patients than ‘blockbuster’ medicines that treat more common diseases or conditions. The cost of developing a medicine is extremely high, so there has been concerns if the pharmaceutical industry can make targeted medicines profitable.

However, one advantage of targeted medicines is that they increase the efficiency of clinical trials. For example, if new medicines target a known cause of a disease, fewer medicines should fail at each stage of the development process. As explained earlier in this topic (section 3.3), the use of biomarkers is key to this work, and this is also explained in detail in the EUPATI biomarkers topic.

For personalised medicine to progress, new findings from molecular research, and new technologies (such as ‘omics’ technologies), must be translated (adapted) for use in clinical trials.

5. References

  • Armelle Corpet & Geneviève Almouzni. Science et Avenir. Dec 2006-Jan 2007