Personalised medicine: the future of healthcare and clinical research

Personalised medicine, also known as precision medicine, aims to change this paradigm by finding diagnostic markers (in terms of DNA, RNA, proteins or metabolites) that can predict therapy response and ensure that patients receive the right treatment from the start.

Why is personalised medicine an important step for healthcare?

The importance of personalised medicine as the next step in healthcare, has been captured very well by the former US president Barack Obama;

“(…) personalized medicine, gives us one of the greatest opportunities for new medical breakthroughs that we have ever seen.  Doctors have always recognized that every patient is unique, and doctors have always tried to tailor their treatments as best they can to individuals. You can match a blood transfusion to a blood type. That was an important discovery.  What if matching a cancer cure to our genetic code was just as easy, just as standard?  What if figuring out the right dose of medicine was as simple as taking our temperature? (…)” (source).

Personalised medicines puts the patient in focus, to deliver the right treatment, to the right person, at the right time, every time. The recent breakthroughs in this field are just the beginning, as Obama puts it;

“(…) the possibilities are boundless.  So, the time is right to unleash a new wave of advances in this area, in precision medicine, just like we did with genetics 25 years ago.” (source).

The transition from conventional medicine to personalised medicine

Patients that are being treated by current therapies can be put into two overall categories; those that benefit from therapy, i.e. responders, and those that do not benefit, i.e. non-responders. For certain drugs the patients that benefit from a therapy are just a small fraction of the patients that are being treated with the drug. The patients that do not benefit are instead treated with therapy after therapy until the right treatment is found. This method of treatment is not efficient from:

• A healthcare-cost perspective: inefficiency increases the duration and amount of treatments that patients must undergo and therefore also increase the costs;
• A patient perspective, as they not only use drugs that are not efficacious, but they might also suffer from unnecessary adverse events;
• A drug development perspective: this approach can lead to new drugs failing clinical tests while still showing a positive effect for a subpopulation of patients.

The cause for variation in response to therapy lies in the fact that the population is heterogeneous in terms of genetic make-up, which can determine the risk of developing a disease, and phenotype, which reflects the current state.

For example: the biochemical makeup of diseased patients with type 2 diabetes* is not the same and therefore certain therapies can only help a portion of patients to restore biochemical pathways to reach the desired outcome in terms of biomarkers: lowered fasting glucose and lower glycylated hemoglobin. In patients that do not respond, the biochemical makeup is different and there is an unmet biochemical need that is not addressed by the therapy.

*Note: certain genes might encode an increased risk for developing type 2 diabetes, but, amongst others, lifestyle factors, diet and degree of exercise determine whether type 2 diabetes manifests.

Benefits of personalised medicine

With current treatments, patients are treated with a one-size-fits-all approach and if the therapy is not effective another therapy is attempted. Personalised medicine or precision medicine aims to change this paradigm by finding diagnostic markers (in terms of DNA, RNA, proteins or metabolites) that can predict therapy response. By measuring this biochemical makeup, patients can be stratified based on predicted therapy response and the therapy that best addresses the unmet biochemical need can be selected. This combination of therapeutics selection and diagnostics is referred to as theranostics. Furthermore, stratifying patients based on their genetic makeup or phenotype enables the development of therapies that are specific to those strata of patients.

A personalised medicine approach tackles the issues associated with the traditional one-size-fits-all approach. Recently, EFPIA and EBE have categorized the benefits of personalized medicine as follows (Source) :

Better patient outcomes: 

• Improved efficacy i.e. patient more likely to receive a medicine delivering a clinical benefit;
• Improved in overall survival;
• Reduced adverse events.

Better drug development:

• More effective clinical trials;
• Efficient clinical trials and reduction in cost;
• More ethical clinical trials.

Benefits for the healthcare system and society:

• Prevention and prediction of diseases;
• Improvement in patient management of diseases;
• Prevention or delay of more expensive care costs and allowing scarce healthcare resources to be using most efficiently;
• Reduces hospitalisation.

Benefits of personalised medicine

Currently, personalized medicine is in its infant phase and we have only seen the first steps by stratification of patients into groups often based on single genes. If n = 1 clinical trials will be possible in the future, all genomic and phenotypic information can be utilized to inform a physician of all aspects of the disease. Enabling medical staff to select the right drug, at the right dose and right time to the right patient.

Here at PNO, our life science consultants and project managers are experienced in projects with a focus on personalising patient treatment, such as PIONEER, a project managed by PNO* that will harness the potential of big data and big data analytics as a means to ensure optimal personalised care for European prostate cancer patients. Most importantly, we believe in the vision of personalising patient treatment, and we are ready to support your project. Fill in the form below to discover how we can help your project to succeed.

* Formerly known as ttopstart, a separate Life Sciences & Health consultancy. The company has since fully integrated into PNO Innovation and no longer operates under the ttopstart name. For more information, see the press release “We continue as PNO Innovation.”