Believing in biotech

03 October 2012

EuropaBio's Secretary General Nathalie Moll and Healthcare Director Miram Gargesi give an overview of the importance of biotechnology for Europe…

It is estimated by the Organisation for Economic Co-operation and Development (OECD) that by 2015 health­care biotechnology knowledge will be used in the development of all new pharmaceutical products. As it stands, more than 350 million patients¹ have already benefited from approved medicines manufactured through biotechnology to treat or prevent a multitude of infections and diseases, such as cancers, heart attacks, stroke, multiple sclerosis, leukaemia, diabetes and rare diseases.

The healthcare biotechnology industry in the EU is contributing to advancing patients' rights of access to the most innovative and modern medicines available. It has the potential to have a tremendous impact on meeting the needs of patients and their families. The industry not only encompasses medicines and diagnostics that are manufactured using a biotechnological process, but also novel biotech-based therapies, such as gene and cell therapies and tissue-engineered products. 

Currently, European firms account for 20.6% of biophar­maceuticals worldwide.² In order to maintain and grow this leading position, it is essential that Europe equips itself with the best possible policy framework to advance innovation, knowledge and growth in the healthcare biotech industry. Patients across Europe are relying on a coherent regulatory system, not only for research and innovation, but also for market access, so that the best possible treatments are available to them, wherever they reside. However, often legislation in Europe has a hard time keeping up with the extremely fast pace of the scientific discoveries and developments in healthcare biotechnology.

Personalised medicine
One emerging trend in the industry is the field of personalised medicine. Recent scientific developments have brought us closer to the concept of a more tailored healthcare system. This ultimately involves matching patients' unique profiles and needs to suitable therapies, rather than merely addressing symptoms in isolation. Personalised medicine provides the right treatments to the right patient at the right time by using biotechnology processes. Practically, it improves patients' healthcare outcomes and quality of life through increasingly tailored treatments. It also ensures better safety and efficiency of treatment; therefore, improving the costs involved
for patients.

For personalised medicine treatments to become reality for all patients, a fundamental change in our comprehension of diseases is required and traditional diagnoses may have to be revised. In addition to this, the regulatory framework in Europe needs to change.

Currently, the EU regulatory pathway for the development and approval of medicines and companion diagnostics is extremely fragmented. Market access of personalised medicines is highly dependent on the assessment process, in particular health technology assessment and pricing and reimbursement decisions. In practical terms, policymakers need a long-term strategy for personalised medicine, and significant funding for research and innovation – at both European and national level – is required.

Legislative considerations
A considerable amount of this required funding can come from Horizon 2020, the 8th European Framework Programme for research and innovation funding. Under this programme, funding will focus on Europe's grand challenges, one of which is health and ageing. Healthcare biotechnology is well placed to provide key solutions to this, and it is essential that the funding set aside for biotechnology remains in place after the dossier is discussed at European Parliament and Council level.

There is also a significant emphasis under Horizon 2020 on the role of industry-driven research, with a particular focus on SMEs. This is an important step forward for healthcare biotechnology in Europe, as many companies operating in this field are not only SMEs but micro-enterprises, which are leading the way in innovation and drug discovery across Europe.

Another prime piece of legislation currently being discussed in Europe is the revision of the Clinical Trials Directive. The recent publication of this revision by the European Commission has been eagerly anticipated, and is a very timely subject for the healthcare biotechnology industry, as many such medicines are currently in clinical development. Clinical trials are essential to the research and development of new and innovative medicines, and are conducted to ensure adequate benefit risk so that a medicine is safe and effective for patients. The dossier, which is pioneered by European Commissioner for Health and Consumer Policy John Dalli, sees for the first time efforts from the Commission to streamline and harmonise the clinical trials process.  The creation of a single EU portal for free-of-charge submission of an application, as well as the introduction of a common standardised format of the clinical trial application, are extremely positive developments that will be of benefit to all stakeholders. Such progress will translate into reduced administrative burdens and costs for public and private sector researchers, which will result in faster access to innovative treatments for patients.

The Commission has also proposed to introduce a Reporting Member State for multinational trials.  This member state will have overall responsibility for the assessment of an application, as well as for centralising requests for additional information. Many clinical trials take place in numerous locations throughout Europe; therefore it is essential that extending them to multiple member states does not trigger a costly and burdensome repetition of the scientific assessments by the different regulatory jurisdictions.

There are currently calls for a simplified and efficient regulatory framework for clinical trials in order for Europe to remain an attractive location for research. This will be of benefit to all stakeholders, allowing faster access to innovative treatments for patients, as well as reducing the administrative burden and costs for both public and private sector researchers and member states. It is also essential that the approval process for clinical trials is streamlined.

A common standardised format and harmonised content of the Clinical Trial Application is needed for all EU member states. There should be a single, pan-European assessment leading to a single, pan-European outcome. Of course, the directive should have patients' needs and safety as its focus point, and therefore all trial participants should be covered by insurance or indemnity.

Many of the benefits of a simplified and coherent Clinical Trials Framework were considered in December 2011 during a EuropaBio workshop held at the European Parliament. Pleasingly, many of the recommendations reached during these stakeholder discussions were reflected in the Commission Proposal when it was published  on 17th July. This is a key piece of legislation for the EU health community, and has the potential to improve patients' access to safe, effective and high-quality medicines, while simultaneously increasing Europe's competitiveness and reputation as a world-class centre for clinical research. It is important that the regulatory framework is robust enough to ensure the quality of the research and data that is collected, whilst at the same time being flexible enough to not hinder innovation.

Healthcare biotechnology is already playing a major role in meeting the objectives of the EU 2020 agenda. There can be no doubt that with the right policy and financial frameworks in place, this leading European sector can continue to improve the lives of patients and contribute to economic growth. While the industry has already achieved much and contributed greatly to improving quality of life for EU citizens, regulation adapted to the newest innovative developments is needed if we want to maintain an innovative and viable European healthcare biotechnology industry that can compete globally. We must ensure that the innovation and knowledge created in Europe, stays in Europe.

1 Biotechnology research continues to bolster arsenal against diseases with 633 medicines in developments, PhRMA report, 2008
2 Ernst & Young: Beyond Borders, Global Biotechnology report 2009