Challenges

Personalised medicine, chronic diseases and ageing, as well as rare diseases, are the main global challenges for Eurobiomed that bring with them a major revolution in the treatment of patients, health care systems, and the strategies for public and private research and development. These cross-cutting elements provide a unique perspective on each area and the Eurobiomed excellence topics.

Personalised medicine is an on-going revolution from a scientific point of view as well as clinically and systemically. Beyond the financial issues, numerous challenges emerge for actors wishing to develop in this sector or contribute to its development, including: 

  • The required synchronisation of the development process between the diagnostic and pharmaceutical world: biomarkers and companion diagnostics. Another organisational challenge is also that of the interaction of businesses with the world of research, including academic and clinical, as well as biobanks by consideration of the arrival of new health objects. 
  • Regulations must take account of these innovations and the question of reimbursement arises. While the new drugs acceptance process will become more complex, that of medical devices, and in particular for diagnosis, is still under construction. The extensive use and support of biobanks, but also the collection of health data through the connected objects retrospectively raises the issue of patient consent: ethical issues accompanying the advent of biomarkers are essential. 
  • Finally, a new economic equation must be found: the pharmaceutical market is changing from a high volume / small profit blockbuster model to a niche model (small volume) with, to date, no guarantee of profits. Co-development of personalised medicine from pharmaceutical businesses, biotechs and academic researchers using biobank resources raises the question of revenue sharing.

The ageing population is partly responsible for the emergence of new diseases and reveals new health needs, such as prevention of frailty in the elderly, treatment of addiction and chronic diseases. Today, an estimated 11 million people in France, nearly 20 % of the population, are chronically ill.

The goal of "living longer" is transformed today into a goal of "ageing better". The INSEE analyses on life expectancy and life expectancy in good health conclude that if men and women live on average up to 78 and 82 years, respectively, their life expectancy in good health is only 60 to 65 years (with a recent shift since 2008). Life expectancy at birth continues to improve. The differences in life expectancy between men and women remain significant, but are reduced to life expectancy in "good health".

Chronic diseases such as cancer, diabetes, cardiovascular diseases and respiratory diseases or respiratory forms and neurodegenerative diseases are especially responsible.

Beyond conventional therapeutics, this new public health challenge opens the way for technological innovations, services or uses to respond in particular to patient monitoring needs for controlling health costs. This challenge mobilises numerous disciplines (monitoring biomarkers, ICT and communicating devices for remote monitoring, health economics assessment, epidemiology, etc.). This area involves, beyond technological approaches, economic, societal and human approaches.

Rare diseases are defined as diseases whose incidence is less than one person affected in 2,000; they now represent approximately 7,000 pathologies of which 80 % are genetic. Rare diseases affect 3 to 4 million people in France (1 in 20) and 35 million in Europe. The fight against rare diseases is one of the five public health priorities (public health law of 9 August 2004).

Fifty laboratories, including the main "big pharma", are positioned in this market, which has many synergies with extremely frequent pathologies (inflammatory diseases, for example), but also with the technological challenges of personalised medicine. The global market for rare diseases is estimated at about $ 100 billion in 2014 and should experience an annual sales growth of 10.5 % per year between 2014 and 2020 according to Evaluate Pharma.

 

The challenge is twofold: to provide an understanding of disease mechanisms and thus treat more patients who had previously little or no therapeutic solutions. Thus, market access is facilitated by regulatory authorities, which encourage businesses to address these pathologies. 

In France, this particularly coveted market can be estimated around € 1 billion. In 2012, the FDA approved 33 % of molecules dedicated to orphan diseases in 39 data approvals, and the EMEA approved the first gene therapy for application in an orphan disease (Glybera) (source Beyond Borders 2013, E&Y).