Nathalie Moll, Secretary General, EuropaBio




PATIENTS WHO ARE diagnosed with the same diseases may show similar symptoms but react very differently when given the same treatment. Today’s medical textbooks and diagnoses from doctors still define diseases on the basis that patients who have the same symptoms and whose course of disease follows similar patterns will therefore warrant the same medical treatment. However, our knowledge of disease mechanisms has not always resulted in the most efficient and beneficial treatment for the individual patient.

In the vast majority of cases, doctors have no other choice than to make decisions on the treatment for diseases based on symptoms and trial and error. However, groundbreaking developments in the field of healthcare biotechnology are at the cusp of changing how we define diseases and treat patients. These exciting new discoveries are leading to the replacement of traditional intuitive and empirically-based medicine by precision diagnosis and personalised medical treatment.


Personalised medicine is a concept that puts the patient at the heart of treatment and diagnostics. It individualises the doctor’s approach and ultimately involves matching patients’ unique profiles and needs to suitable therapies rather than simply addressing symptoms in isolation. The development of personalised medicine is based on an exact understanding of relevant mechanisms of diseases. Personalised medicine is providing the right treatment to the right patient at the right time, by using the latest scientific developments. In practise, this will lead to major improvements in patients’ healthcare outcomes and quality of life through increasingly tailored treatments. It will also improve safety and efficiency of medical care for patients – improving side effects and reducing costs.

There are already excellent examples of existing personalised medicine treatment, particularly in the area of oncology. In the treatment of rare diseases, which are often chronic and life-threatening genetic conditions, treatment is already based upon understanding the underlying and genetic mechanism of the disease and tailoring the treatment to target both that disease and that patient specifically. What is essential now is that this specialism is turned into the mainstream way of treating and diagnosing all illnesses. While oncology has been the traditional front runner in the field of personalised medicine, more recently a number of biomarker candidates have also been discovered that link their mode of action with disease pathophysiology for many chronic inflammatory conditions including arthritis and asthma.


For personalised medicine to work, it requires a very thorough understanding of the molecular mechanisms of the disease. The adoption of personalised medicine will also require a number of research tools including imaging, several types of ‘-omics’ technologies and much more. Therefore, a fundamental change in our approach to R&D&I will be necessary, as well as a shift in national economic evaluations of medicine. Our comprehension of diseases needs to change and traditional diagnoses and discoveries may have to be revised. In the future, healthcare practitioners should be able to identify underlying molecular disease mechanisms more precisely, and as a consequence, make more effective treatment decisions.

The concept of personalised medicine centres on the ability of biomarkers to select a patient population with a higher rate of favourable response to a specific type of medicine. If we are able to determine the biologically relevant dose, range and selection of the optimal target population by means of biomarker, we will increase R&D productivity by reducing development timelines and costs. Personalised medicine can also have a positive impact on the healthcare budgets of Member States if the pricing and reimbursement of personalised medicines is carefully considered and a balanced view on cost-effectiveness and incentives for innovation is taken.


For all of this to occur, a more supportive regulatory framework in Europe is essential. Positive steps can already be seen in Europe, particularly with the launch of the European Commission 8th Framework Programme for Research & Innovation funding, Horizon 2020, in November of last year. Under this Programme, one of the main focuses will be the grand challenge of health and ageing and ensuring important funding for R&D&I in healthcare biotechnology.


There are many challenges that lie ahead for the field of personalised medicine. In June 2011 EuropaBio produced a white paper on the topic of personalised medicine, outlining these challenges and proposing potential solutions. Currently, the worlds of drug manufacturing and diagnostics are in parallel universes in that they typically have very different development timelines, product lifecycles, return on investment and regulation. Drugs are valued and reimbursed as products, typically of high value. Diagnostics on the other hand are valued and paid for as services, typically at a much lower value. Currently relatively few, if any, models exist for valuing both a drug and diagnostic in combination.

The current EU regulatory pathway for development and approval of drugs and companion diagnostics is extremely different. Market access of medicines is highly dependent on the assessment process, in particular health technology assessment (HTA) and pricing and reimbursement decisions. In practical terms, policy makers need to introduce a long-term strategy for personalised medicine and increased funding for research and innovation at both European and national level.


Personalised medicine is about dealing with questions that relate to each individual’s health and wellbeing. It helps healthcare professionals to discover which diseases a patient is susceptible to, and how they are likely to respond to various treatments, based on a number of characteristics such as age, gender, blood type, immune system, genetics and metabolism. The latest developments in the field of biotechnology have reached a phase where healthcare professionals are now in a better position than ever before to answer these questions accurately. It is fundamental that this innovative field of healthcare is given the right level of support, with an appropriate regulatory framework both at national and EU level as well as ongoing communication between healthcare professionals, for this paradigm shift to occur. If personalised medicine is integrated into our healthcare systems, the patient’s world will change for the better. There is no doubt that with the right policy framework and the right shift in R&D&I within the healthcare biotechnology industry, personalised medicine can greatly improve the lives of patients and their families.