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Focus on Regulation

Personalised Medicines at the top of the European Agenda

In March 2017, the European Medicines Agency organised a workshop concerning personalised medicine. The results of the workshop have just been published.

The workshop discussed the views of patients, healthcare professionals and payers regarding personalised medicine. During the workshop future challenges and related actions, that should be taken regarding personalised medicine, were identified. The challenges for regulators and scientists were also addressed.


In the interests of clarity the definition of personalised medicine provided by the Council of Europe is used. Personalised medicine is considered the:

“Medical model using characterisation of individuals’ phenotypes and genotypes or tailoring the right person at the right time, and to determine the predisposition to disease and/or deliver timely and targeted prevention, and it relates to the broader concept of patient-centred care, which takes into account that, healthcare systems need to better respond to patient needs”

Sessions of the workshop

The workshop identified areas where major changes are needed to serve the needs of the development of personalised medicine. For example, the development of a patient-centred system would require significant changes concerning the testing and evaluation of medicinal products for human use.

In the first session of the workshop, the current situation concerning personalised medicine was discussed and related major challenges identified. These challenges included: the generation of big data, the translation of basic research into clinical research and implementation, bringing innovation to the market and shaping a sustainable healthcare. The requirement to ensure confidentiality of sensitive data and the necessity to obtain a truly informed consent from patients were also identified as current challenges.

The development of personalised medicine also raises significant challenges for regulators and researchers. In the second session of the workshop, it was concluded that the need for use of biomarkers in early drug development, as well as the necessity of conducting early stage clinical research, will change the traditional forms of clinical trials. Consequently, novel clinical trials designs should be developed. Such clinical trials will involve:

  • “Basket studies, which recruits patients on the basis of their molecular characteristics irrespective of the  diseased organ;
  • Umbrella studies, where patients with the same type of disease are screemed for a series of hypothesised predictive biomarkers. They are then allocated to appropriate therapies;
  • Platform trials, where multiple treatments are evaluated simultaneously”.

The potential need for novel clinical trials designs was discussed in tandem with the related raised regulatory issues concerning labelling, assessment and post-authorisation requirements.

The development of personalised medicine is challenging for researchers, developers and payers. Denis Lacombe of the European Organisation for Research and Treatment Cancer mentioned the need for innovative models of clinical research and improvement in the stages through the lifecycle of a medicinal product for human use.

During the third session of the workshop the focus was on the priorities of patients and healthcare professionals. The participants identified the ways in which patients’ and healthcare professionals’ input could be incorporated into personalised medicine related discussions. Furthermore, it was concluded that EMA could provide considerable help by continuing its collaboration with both healthcare professionals and patients.

The workshop identified actions that should be taken concerning personalised medicine. These actions involved among others, the organisation of a follow-up workshop involving industry stakeholders, collaboration among the EMA scientific committees and the Scientific Advice and Pharmacogenetics Working Parties, and help stakeholders to gather and discuss information concerning personalised medicines.