Miriam Gargesi, Director of Healthcare, The European Association for Bioindustries


International Innovation – Research Media Ltd
For almost two decades, EuropaBio has been actively endorsing Europe’s biotech industry. Miriam Gargesi, Director of Healthcare, joins International Innovation to discuss personalised medicine, the regulation of clinical trials, and the forthcoming European Biotech Week


As an introduction, could you explain EuropaBio’s role within the biotech industry, outlining its top priorities for 2013 and 2014?

EuropaBio, the European Association for Bioindustries, was created in 1996. Our role is to promote an innovative and dynamic biotechnology-based industry in Europe. We represent biotechnology companies active in Europe as well as national biotechnology associations who in turn represent more than 1,800 biotech SMEs.

Members of EuropaBio are involved in research, development, testing, manufacturing and commercialisation of biotechnology products and processes. Our corporate members are active in a wide range of sectors: human and animal healthcare, diagnostics, bioinformatics, chemicals, crop protection, agriculture, food and environmental products and services. We are committed to an open, informed dialogue with all stakeholders about the ethical, social and economic aspects of biotechnology and its applications, and champion the responsible use of biotechnology to ensure that its potential is fully used to the benefit of the environment and humankind.

Our top priorities for healthcare biotechnology for the rest of 2013 and 2014 centre on:

• Approval of the proposed Regulation on Clinical Trials

• Being involved in the development of an EU methodology for Health Technology Assessment

• Presenting the many benefits of healthcare biotechnology to the public and the new stakeholders taking up posts in Brussels in 2014

With healthcare treatments advancing at a rapid rate, how close are we to seeing a personalised medicine revolution? What issues must be addressed and how might it change the face of healthcare forever?

Personalised medicine, which combines diagnosis and treatment, is a concept that puts patients at the centre of a given treatment, allowing medical practitioners to target medical care to suit the patient’s individual needs and disease pathology. While this novel concept could be a ground-breaking revolution for medical care, it still has a long way to go before it becomes the norm. Based on the realisation that many stakeholders do not fully understand personalised medicine, EuropaBio has pioneered a multi-stakeholder dialogue on the topic through workshops and position statements. In order for personalised medicine to become more central to the treatment of patients and disease, further investment in research, development and innovation is essential. Efforts undertaken through the Innovative Medicines Initiative (IMI) and the maintenance of a public-private partnership (PPP) for pioneering healthcare research under Horizon 2020 are steps in the right direction, but the inclusion of SMEs within these proposals will be essential.

Another challenge to the benefits offered by personalised medicine lies in regulation. The current EU regulatory pathway for the development and approval of drugs and companion diagnostics is extremely fragmented, with very different development timelines, product lifecycles and return on investment. Drugs are valued and reimbursed as products. Diagnostics, on the other hand, are valued and paid for as services – usually at a much lower price – meaning that, under the current regime, it is impossible for both to be marketed at the same time. Patients and medical practitioners are losing out due to this disconnect which must be addressed urgently by EU policy makers.

How has EuropaBio pushed the development of personalised medicine further up the policy agenda?

In order to engage with policy makers on the challenges and opportunities associated with personalised medicine, EuropaBio organised and has taken part in a number of public workshops both this year and last. In June 2012, we launched a white paper on personalised medicine in which we encourage discussion and thought over vital issues concerning this topic, as well as highlighting how personalised medicine is becoming a crucial element of our healthcare systems.

As a member of the recently formed European Alliance for Personalised Medicine (EAPM), how was EuropaBio involved with the recent Innovation & Patient Access to Personalised Medicine conference in Dublin?

EuropaBio is a member of EAPM and strongly supports its efforts to engage broadly with stakeholders and ensure that personalised medicine is a priority for policy makers in Europe. We, along with many of the other EAPM members, actively contributed to the success of the conference – which was held under the Irish President of the EU in Dublin on 21 March 2013 – by organising one of the Working Group sessions on the importance of healthcare technology assessment (HTA) in ensuring the growth and acceptance of personalised medicine in Europe. We believe there is a need for specific implementation proposals and for a more standardised approach to assessment. EuropaBio will therefore continue to be a leading voice in representing the interests of healthcare biotechnology in the HTA arena, as well as encouraging a more sustainable European HTA collaboration across Member States.

Biomedical science continues to develop at an exponential rate, affecting the advancement of medicinal research and its subsequent transition to praxis. With the proposed Clinical Trials Regulation planned to be implemented in 2016, how do you see the future of clinical trials progressing?

The proposed Clinical Trials Regulation, which is currently going through the EU’s legislative process, offers an improved, simplified and more efficient regulatory framework. It has the potential to achieve better harmonisation, transparency and consistency in the approval and conduct of clinical trials across the EU, while maintaining high standards of patient safety as well as robustness and reliability of clinical data. This could go a long way towards strengthening Europe’s position as a world leader in the R&D of innovative new medicines.

Provisions to speed up the process of extending a clinical trial to additional Member States once authorisation has been granted are welcome. However, assurances are needed that personal data, intellectual property, and regulatory and commercially confidential information relating to applicants contained in the EU database will be protected. In addition, we feel that there is no scientific justification for the longer assessment timelines proposed for advanced therapy medicinal products.

As you mentioned, privacy and confidentiality are of fundamental importance in research and clinical trials. How can this be assured?

Demands for greater transparency of regulatory decision making have led to an ongoing debate regarding public access to data held by regulatory authorities.

We believe that a number of factors should be taken into account when considering the level of information that should be disclosed: the stage of the product lifecycle, the type of information that could be released, the method used for its release, and the impact on the future development and value of products, as well as on patient access.

While we fully support the need for sensible regulation that promotes openness and transparency, it is also important to recognise the value of knowledge to companies actively involved in R&D. Protection of this knowledge underpins access to new and innovative treatments for patients, as well as collaboration between various R&D stakeholders such as academia, medical research charities, SMEs and multinational biopharmaceutical companies. EuropaBio strongly recommends that consideration be given to the issue of data protection and patient confidentiality during the development of the proposed Clinical Trial Regulation, to ensure that commercially confidential and sensitive information arising from various stages of R&D is not disclosed before commercialisation, unless such disclosure is justified on the grounds of patient safety or public health, following consultation with the data owner.

EuropaBio has expressed a desire for a PPP to be created under Horizon 2020 to build on the work of the IMI. How will this impact on the future of European healthcare?

Through its calls for proposals (the eighth being announced on 17 December 2012), IMI has contributed to innovation within the healthcare biotech sector. Both SMEs and larger companies should be involved in IMI as a means to jointly address the challenges and new opportunities within the field of healthcare research. We advocate that biotechnology should be at the heart of this process, due to the innovative biotech solutions being developed as a means to provide treatments and medicines for a wide variety of diseases and conditions.

Following a review of the successes and difficulties of the current IMI, a new PPP under Horizon 2020 should be well-funded with clear goals in place, and should particularly address the issue of SME involvement.