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Kurt Zatloukal: New framework for global research collaboration
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Kurt Zatloukal: New framework for global research collaboration

23.04.2012 - The increase in lifestyle and ageing-associated diseases that has paralleled a rise in drug development costs – along with the financial constraints of the global economy – is putting enormous pressure on the sustainability of healthcare systems.

These challenges can only be addressed by innovations for improved prevention and treatment of diseases that are based on scientific evidence. Biobanks are a key resource for delivering such solutions, as they provide access to human biological samples linked with detailed information on diseases and lifestyle. These resources are essential for a more mechanism-based understanding of diseases as a prerequisite for the further development of personalised medicine. After all, access to high-quality human samples is vital when it comes to supporting the development of molecular diagnostics, particularly companion diagnostics. Furthermore, to aid in better understanding gene-lifestyle interactions, large population-based cohort studies that contain information on lifestyle will help deliver a scientific basis for evidence-based disease prevention.

In order to address these needs, several European countries have made major investments in favour of biobanks. However, the challenges of providing sustainable healthcare are global rather than national issues. The need for action coordinated at the global scale is therefore obvious for many reasons. First, addressing the same problem in multiple non-coordinated approaches is neither efficient nor financially justified. Second, non-coordinated actions result in unnecessary duplication and – even worse – in non-compatible solutions that would undermine competitiveness in global biotech and the pharmaceutical industry.

The planning team of the European research infrastructure for biobanking and biomolecular resources (BBMRI) has developed the concept of a global network of expert centres to achieve global harmonisation in biobanking and bio­sample analysis. One rationale for the expert centres was to improve access to biobanks for industry and to avoid scenarios of sample commercialisation, which is illegal in Europe. Expert centres could be established as public-­private-partnerships that perform the analysis of biological samples using the latest technologies, and according to internationally harmonised procedures. Pre-competitive data generated in expert centres can be used by industry for product development, and will be made available to the public following procedures developed for the Innovative Medicines Initiative. Expert centres could also be established as public entities outside Europe to transform biological samples into high-quality data and information. This would avoid the need for transnational sample shipment in international research collaboration, thereby providing a new cooperation solution with countries that have legal restrictions on sample export. In addition, sample analysis in the country of origin would exert a strong positive stimulus on generating local know-how and regional development. However, such a global network of expert centres can only work if pre-analytical and analytical procedures are thoroughly standardised, and if expert centres share common reference material and regularly participate in proficiency testing. The first voluntary activities for testing the feasibility of the concept, with a focus on genomics, metabolomics and molecular pathology technologies, are currently in development.

Kurt Zatloukal

Kurt Zatloukal is Professor of Pathology at the Medical University of Graz, Austria. From 2008-2011 he coordinated the preparatory phase of the European research infrastructure for biobanking and biomolecular resources (BBMRI). Currently he is involved in several FP7 projects that focus on standardisation of molecular biosample analysis and is Director of the Christian Doppler Laboratory for biospecimen research and biobanking technologies.

18.03.2014 In October 2012, the European Commission issued a communication on its new Industrial policy identifying biotechnology as one of six Key Enabling Technologies. Biotech will be an important tool in helping Europe deliver on its Europe 2020 strategy of creating an innovative, resource-efficient, smart, sustainable and inclusive economy.

Beat Spaeth, Director Green Biotechnology, EuropaBio, Brussels

Due to the many challenges facing Europe and the world today - among them the threat of climate change, a decreasing amount of arable land and a growing population worldwide - we will need to use every available tool to optimise the use of land and resources and enable those in developing countries to improve their quality of life. Innovation in plant breeding is essential to feed the world's growing numbers and help reduce poverty by improving food security. Global demand for food is expected to increase by 70% by 2050. To meet this demand, we will need to produce the same amount of food in the next 40 years as we did in the past 8,000.

However, land is the limiting factor in producing sufficient food, feed and fuel to meet global demands. Biomass should therefore always be produced with land and resource efficiency in mind, and should ultimately be used in a smart and sustainable way. To make this possible, farmers need the right to choose what they plant and grow. That will allow them to make the best use of the available resources and enable them to preserve other land for biodiversity purposes. In biobased industries, there should be a level playing field when it comes to biomass used for energy purposes and that used for high value chemicals and biobased plastics production. The production of the latter two provides greater economic benefits in terms of economic growth and jobs, and uses far smaller amounts of biomass. In addition, helping to create new markets for biobased products through incentives and public procurement would help increase the value of agricultural residues, thereby bringing greater benefits to farmers.

According to the latest global biotech crop acreage report published by the International Service for the Acquisition of Agri-biotech Applications (ISAAA), more than 18 million farmers are currently planting biotech crops on 175 million hectares. Of those farmers, more than 90% - or 16.5 million of them - are small-scale and resource-poor. The benefits of agricultural biotechnology are clear in countries that have adopted it. A recent study has shown that the adoption of Bt maize allowed Spain to reduce its imports of the crop by over 853,000 tonnes between 1998 and 2013, with consequent savings of €156m. It is time that we begin to view agricultural biotechnology as what it is: a technique that is an integrated part of the European transition to a lower-carbon, more resource-efficient bioeconomy.

Beat Spaeth

studied European affairs and languages in the UK, France and Belgium. He began
his career in 2001 at the European Parliament, where he worked as an assistant to a German MEP, before moving on to join the Brussels office of the German Retail Federation as an advisor with a focus on the environment, social affairs and corporate social responsibility. At EuropaBio, Späth currently manages the political aspects of agricultural biotechnology at the EU level. He interacts with representatives from member companies to facilitate industry positions on political developments, and communicates with EU decisionmakers on behalf of the association.

04.03.2014 When penicillin was discovered by Dr. Alexander Fleming in 1928, people around the world believed it was one of the greatest medical and scientific advances in the history of mankind – and they were right. Before penicillin, a bacterial infection in a minor cut or injury could easily become fatal. Diseases like scarlet fever, pneumonia, meningitis and diphtheria were essentially untreatable.

Janet Hammond, Roche Pharmaceutical Research and Early Development, Basel

In the following decades, more antibiotics appeared on the scene, among them tetracyclines, isoniazid, macrolides, glycopeptides and cephalosporins. 

Doctors began to routinely prescribe anti-biotics – even when they weren’t sure that patients actually had bacterial infections – and it seemed that this miracle of modern medicine had the potential to eradicate one of mankind’s tiniest but deadliest enemies. 

Ominous data began to appear, however, suggesting that the miracle wouldn’t last forever. In the early 1980s, Staphylococcus aureus infections appeared that were resistant to mul-tiple antibiotics. At the same time, mul-tiple drug resistant Streptococcus pneuomoniae infections also surfaced. 

Each passing year carried more troubling findings and an expanding list of bacteria that challenged even the most powerful antibiotics. Methicillin-resistant Staphylococcus aureus (MRSA) outbreaks in hospitals became headline news. More than 1.5 million people around the world are now dying each year from bacterial infections. 

What happened to change bacterial infections from a health problem once considered by many to be nearly “solved” into an implacable foe described by a leading scientist as a “catastrophe” equal to bioterrorism?

Although pharmaceutical research into anti-biotics continues, a “discovery void” has emerged. Since 1987, the number of new antibiotics reaching the market has stalled. Meanwhile, new resistance mechanisms in bacteria have emerged, making even the most advanced antibiotics virtually ineffective. 

Fearing that widespread resistance to anti-biotics could bring “the end of modern medicine as we know it,” the World Health Organization has made finding new, effective antibiotics one of its top three priorities, and regulatory authorities now offer incentives related to the research and development of new anti-bacterials.

In November 2013, Roche pRED (Pharma Research and Early Development) entered a partnering deal to develop novel anti-biotic POL7080, a Phase II compound targeting Pseudomonas aeruginosa (PA), a Gram--negative bacterial species causing severe infections. PA is responsible for a significant percentage of multi-drug resistant pneumonias in the hospital setting. 

Encouraged by changes in the regulatory landscape and a better understanding of the under-lying biology, Roche is one of just a few companies that have so far returned to the development of antibiotics, determined to take on this threat to global health. Roche was an industry leader with Bactrim and Rocephin, two groundbreaking antibiotics credited with saving billions of lives, and the new focus on pathogen-specific drugs for multi-drug resistant bacteria plays on our legacy and our strengths.

For patients who are under siege by a microscopic enemy that seems to ward off all weapons, there is hope on the horizon. 

Janet Hammond

is Head of Infectious Diseases at Roche Pharma Research and Early Development. She received her medical training at the University of Cape Town before specialising in internal medicine and pulmonary/critical care medicine. She then moved to the US, where she completed her fellowship in Infectious Disease at Duke University and Infectious Disease and Clinical Pharmacology at Johns Hopkins University. Prior to joining Roche in 2011, Dr. Hammond was Chief Medical Officer at Valeant Pharmaceuticals.  

03.01.2014 In the past year, the debate surrounding increased transparency of clinical trials has seen a great deal of progress. From an industry perspective, we have engaged more openly with a greater diversity of stake­holders on the topic - a positive development, as an intelligent exchange of ideas is needed to determine the best path forward.

Richard Bergstroem, Director General EFPIA, Brussels

Opinions on how to best increase openness around clinical trials data are varied, but as the conversation has progressed, one thing has become clear: It will be necessary to strike a certain balance if we are to develop data-sharing measures that will best serve public health interests. Greater transparency around clinical trials data is needed - but it must be a responsible transparency. This means protecting not only patients by ensuring their private data is appropriately protected, but also protecting the information contained in clinical trials data that is potentially commercially sensitive. This is a must if we are to safeguard innovation, the tool that the pharmaceutical industry relies on to develop treatments for improved patient outcomes. Publishing Clinical Study Reports (CSRs) in their entirety, as they are written now, does not strike this balance. Making patient-level data available to all does not strike this balance. Allowing indeterminate access to full data sets - again, does not strike this balance. A responsible transparency is one that safeguards patients and their privacy, respects regulatory systems at all levels and protects research incentives. These are the premises underlying the EFPIA-PhRMA Principles for Responsible Clinical Trial Data Sharing, which EFPIA and PhRMA member companies have agreed to implement from January 1, 2014. These commitments enhance patient and public access to clinical study information by providing synopses of clinical study reports, which give the public information it needs while protecting sensitive patient information. More detailed patient-level and study-level data can be made available on request to those who can do good with it, including qualified researchers who will further science.

In a recent NEJM article (doi: 10.1056/ NEJMp1310771), the EMA's Hans-Georg Eichler, Guido Rasi and other authors argue that access to patient-level trial data could help drug developers in how they conduct clinical trials - something that could potentially improve healthcare outcomes. Drugmakers have already recognised the value of sharing data with qualified researchers. We are increasingly seeing cases of open innovation and companies that were once competitors joining forces to tackle areas of unmet need. The Innovative Medicines Initiative, the public-private partnership between EFPIA and the European Commission, is fostering open innovation and encouraging collaboration, with companies sharing data and pooling resources. There are undeniable benefits to increasing openness around clinical trials data - but only if it is shared in the right way, with the right people.

Knowledge is power, and this is especially true when it comes to healthcare. The science of medicine has allowed for amazing progress in improving treatment for a variety of illnesses, from HIV to cancer. Medical and scientific innovation is one of the most powerful tools we have for improving patients' lives. Responsible data-sharing is about recognising this and striking the right balance - one that protects both patients and innovation.

Richard Bergstroem

was appointed Director General of the European Federation of Pharmaceutical Industries and Associations (EFPIA) in April 2011. Over the past 20 years, he has worked for Roche, Novartis and with the Swedish pharmaceutical industry association (LIF). A pharmacist by training, he received his MScPharm degree from the University of Uppsala (Sweden) in 1988. Since 2006, Bergström has been an advisor to the WHO on Good Governance in Medicine.

03.12.2013 Put simply, biotechnology is the use of living organisms to develop useful products. Its basic principles have been employed to alter plants and livestock for domestication for thousands of years.

Nathalie Moll, Secretary General EuropaBio, Brussels

In Europe today, however, biotech is either perceived at best as a dream, or at worse as a nightmare. Most consumers remain only vaguely aware of the biotech products that are helping Europeans live healthier, longer and greener lives. The short list alone includes vaccines, insulin, rare disease therapies, improved crops, detergents for washing at lower temperatures, bio-plastics, cosmetics and biofuels.

So what can be done to help make this invisible revolution more visible? We‘re living longer and healthier lives thanks to advances in medicine. More than 350 million patients globally are already benefiting from healthcare biotech that is helping to treat and prevent common ailments like heart disease and diabetes. And it is also developing therapies for rare diseases – often debilitating and life-threatening – that affect up to 30 million Europeans. 20% of all medications are made using its methods, and by 2015, 50% of our medicines will come from biotech.

In other aspects of our lives, industrial biotech is helping to minimise environmental impact while boosting manufacturing output and creating more jobs. Europe is a world leader in white biotech, producing about 75% of the world‘s enzymes. In production processes, industrial biotech reduces the need for crude oil by using renewable raw materials, leading to significant reductions in greenhouse gas emissions. In fact, the WWF estimates industrial biotech will help reduce up to 2.5 billion tonnes of CO2 equivalent per year by 2030.

With agricultural biotech, Europe’s farmers have been able to increase yields by up to 30% on the same amount of land, helping protect biodiversity and wildlife. These crops also reduce fuel consumption and CO2 emissions by requiring less tillage. In 2011, this was like removing 23.1 billion kg of CO2 from the atmosphere – the equivalent of taking over 10 million cars off the road for a year.

Great things of course often come in small packages, and the 2000+ biotech SMEs in Europe are crucial to delivering innovative solutions for our most pressing societal needs. Europe will suffer competitively if we don‘t give the right financial backing to these firms. We hope that the annual EuropaBio Most Innovative Biotech SME Award will go some way towards providing a voice for the sector, and highlight the importance of a supportive regulatory framework to help it flourish.

We cannot afford to let biotech‘s many positive aspects pass consumers by or – even worse – be misunderstood. It is up to our industry to better communicate the benefits of biotech to Europeans. This year we celebrate the first ever European Biotech Week – showcasing this innovative and vibrant sector. Over 60 events took place across the EU during the first week of October, underlining the importance its various fields now play in our lives.

The European Biotech Week will be an annual occurrence in October from now on to emphasise that biotechnology has grown more important than ever to job creation, improving health and creating a more sustainable environment. Anyone with a curious mind, an interest in how we can achieve better health as we grow older, and a cleaner environment is invited to celebrate and learn more about the incredible world of biotechnology and how it evolves each year. Let‘s make the invisible revolution visible!

Nathalie Moll

has spent almost a decade representing the European biotechnology industry through EuropaBio in the posts of External Relations Manager, Director for Strategic Policy, Director of the Healthcare Biotech sector, Director of the Agricultural Biotech sector and most recently as Secretary General - a post she took over in 2010. Prior to her career at EuropaBio, the trained biochemist worked in the biotechnology and food policy area for the European Crop Protection Association, as well as for the Italian National Biotech Association (Assobiotec) and Dompé Farmaceutici S.p. A dealing with the implementation of EU biotech legislation at the national level.

Photo: EuopaBio

04.11.2013 The start of Europe's new financial framework and the framework research programme Horizon 2020 seems a good moment to take a step back and look into some recent and not so recent - but even more substantial - changes in (bio-)technology policy paradigms.

Peter Schintlmeister, Austrian Federal Ministry of Economics, Family and Youth

From technology-orientation towards justification: In 2002, the European Commission (EC) published its first overarching strategy for Life Sciences and Biotechnology. The focus then was clearly on new technologies and their promise. Meanwhile policy mainstream has changed and all technological efforts are superseded by the need for justifying the possible contribution to society's 'Grand Challenges'. In practice, even technologies that have been politically identified as 'key technologies' fall under this regimen, which in turn narrows horizons for possibilities outside this scope. The good news for cutting-edge science and technology, however, is that new and more precise instruments (e.g. the ERC) are easily countermanding the loss of focus on technology.

The loss of the knowledge-base: the term 'knowledge-based bio-economy' first appeared on the radar of technology policy in 2005. At that time, it was totally in line with the (Lisbon) goals of making Europe competitive through enhancing its knowledge-base. When the 2012 strategy 'Innovation for sustainable growth: A Bio-economy for Europe' was published, the term 'knowledge-base' had interestingly disappeared from the title (although it still appears in places on the website). While this move widens the base of stakeholders significantly by embracing entire traditional industries like agriculture and forestry, it still has to be shown whether Europe can maintain a focus on innovation. This trend can also be seen in the composition of the new Bio- economy Panel, which is aimed at providing the European Commission with feedback from the community.

The shift from supply- to demand-side of technology policy: A couple of recent studies concur on the necessity of keeping current austerity measures in place in most EU Member States, and have concluded that investing taxpayer money in technology-based areas might only solve the technological side of the problem. Bringing innovation to the market - an area where Europe has a notoriously bad track record - is a wholly different affair. The Commission's Lead Market Initiative has sought to balance out issues by emphasising the importance of (low-cost) political measures, such as streamlining the regulatory framework, labeling and certificate schemes (both admittedly tasks worthy of Sisyphus) and enhancing the efficiency of standardisation. The technologypolicy community has recognised that the measures are bringing results, and they have acquired prominent positions in current high-level strategies like Europe 2020 and the Innovation Union. This will enable them to make a positive difference in the years to come.

European policies are about the larger and broader vision, but most of the impact they have still derives from the national implementation that takes place farther down the road. The Greek philosopher Heraclitus is attributed to have said that the only constant in life is change - and that aphorism applies to policies as well!

Peter Schintlmeister

joined Austria's Federal Ministry of Economics, Family and Youth in 2003 as an expert for life sciences and biotechnology. He has enjoyed chairmanships at a number of different entities, including the OECD Task Force on Industrial Biotechnology, the ERA-NET EuroTransBio and the European Commission's Advisory Group on bio-based products for the Lead Market Initiative. Schintlmeister is a member of the European Bioeconomy Panel and the Commission's expert group for bio-based products. He has spent the better part of 2013 in China, where he has been contributing to the establishment of Austria's Office of Science and Technology in Beijing.

26.09.2013 Biosimilar products are not generic medicines, nor are they identical to their reference product or each other. Instead, they are similar versions of well-established recombinant proteins with well-characterised structures and pharmacology. All biologics (biosimilars and reference products) have complicated safety and immunogenicity profiles.

So far only three classes of biosimilar have been approved for use in the EU (somatropins, epoetins and GCSFs), but a second generation of biosimilars is on the way that focuses on more complicated molecules – such as monoclonal antibodies and fusion proteins – to help patients fight diseases like RA and cancer. This has been made possible by the EU’s science-led regulatory framework, which has established confidence in the quality of existing biosimilars. Various biosimilars are currently in different stages of clinical development and regulatory approval in the EU, and biosimilar regulatory pathways are also in place in countries like Canada, the US and Australia. 

Biosimilars have been providing alternative therapeutic choices for European patients and physicians since 2006. The European Commission recently completed an exhaustive study on the introduction of biosimilars into European medical practice, and determined that their availability is helping increase market competition.

Although the available commercial data says otherwise, some claim biosimilars have had a disappointing introduction, and that regulators and politicians need to do ‘more’ to spur their uptake. As a representative for a company developing several biosimilar medicines, I tend to disagree. It’s important to let the market decide, and ensuring patient safety should be the foremost concern for both governments and the biosimilars industry. 

There have been significant advances in our ability to use analytics to better understand the biology of both reference product and biosimilar. However, we haven’t yet (and may never) reach a stage where biosimilars should not be absolutely required to exhibit fidelity to their reference products and be rigorously evaluated in analytical, non-clinical and clinical comparisons. 

The industry has an obligation to take proactive steps to be accountable to providers and patients for product quality. For example, all stakeholders, manufacturers, regulators and healthcare professionals should embrace pharmacovigilance and naming systems that allow all biologics to be properly identified and traced in cases of adverse events. The EU’s pharmacovigilance legislation addresses this, and will help instill further confidence in the field.

It’s evident that high-quality, reliably supplied biosimilars can offer additional choices to patients and other key stakeholders, although development and supply of these complex medicines is scientifically challenging and capital-intensive. We now know manufacturers need significant expertise, infrastructure and capital to successfully develop these molecules. 

The healthcare community, industry and regulators have a key opportunity – to work as partners to ensure appropriate standards for these products are maintained. As we at Amgen move forward with developing our own portfolio, we know our experience is critical in a field where patient safety and reliability of supply are paramount concerns.

Carsten Thiel

is the Vice President and Regional General Manager of Amgen Europe. He joined the European oncology franchise in 2002, and held the post of General Manager for Amgen’s Central and Eastern European operations from 2006-2007 before taking up duties as General Manager in Germany from 2007-2010. He moved to his current post in 2011.

In September of last year, the European Commission presented a proposal concerning a revision of the in vitro diagnostics medical devices (IVD) Directive. The European Parliament and Council will negotiate the concrete wording of the regulation in the coming months, which means it will probably enter into force within the next three to five years.

Many people consider the IVD medical devices regulation the "little sister" of the Medical Devices Regulation, which will be discussed in parallel in European institutions. In my opinion, however, IVD medical devices are less the "little sister" than they are the "parents" of medical devices. Indeed, in some ways they are the progenitors of all therapies, including pharmaceutical products and surgery. Without a proper diagnostic, there can be no proper treatment or prevention of diseases. Unfortunately, the current directive from 1998 has not stopped low-quality IVD medical devices from reaching the market. In the past, there have been cases where low-quality HIV tests were placed on the European market with a CE-label. In one particular instance, a scientific institute had determined even before the notified bodies approved the CE-label that a test delivered many more false negative results than other available HIV tests. In other words, it sometimes said there was no virus present when in fact a subject had contracted the disease. Patients in the EU were given this test for years. When it comes to patient safety, we have to strengthen our system. Similar cases have been reported with hepatitis C, which is still a life-threatening disease that cannot be treated properly.

Another report highlights a different facet of the diagnostics problem. Francis Collins - who headed the Human Genome Project - once sent samples of his own DNA to three different laboratories and received three different results predicting his risk for contracting certain genetic conditions. That's why I also believe it is indispensable to include some basic criteria at a European level for the application of genetic tests.

The Commission proposal focuses very much on the quality of a product. But experts and many international organisations - including the Council of Europe, the OECD and the European Society for Human Genetics - have articulated again and again that in many cases, the framework in which the product is applied is even more important than the quality of the product itself. When it comes to DNA testing in particular, it is very important to respect the principle of informed consent. The European Parliament has requested this several times. A legal opinion concludes that it is possible and appropriate to introduce respective wording in the proposal. Therefore, I have proposed respective amendments together with colleagues from all political groups and different countries on this issue. There is a consensus that it should not be the intention of the European Union to limit patient access to DNA tests, but that appro-priate genetic counselling should be offered in cases of predictive and prenatal diagnostics in order to inform patients properly about possible results before a test is performed. To respect the principle of subsidiary, it should be left to Member States to regulate details, and individual Member States need to have the option to go further than regulations require. One could even argue that it should be mandatory to include informed consent in the proposal, because it is a crucial element of the Charter of Fundamental Rights (Article 3) - a legally binding document for the European Union in those areas where it acts.

Peter Liese

was elected as a Member of the European Parliament in 1994. He is Chairman of the EPP Working Group on Bioethics in the European Parliament and a member of the Committee on Environment, Public Health and Food Safety (ENVI), as well as a substitute member of the Committee on Foreign Affairs and member of the delegation for relations with the countries of Central America. During this legislative term, Peter Liese became a co-coordinator for the EPP Group in the ENVI committee. Before going into politics, he worked as a doctor in a pediatrics hospital in Paderborn (Germany), and spent six months in Central America working in a state-owned hospital and on foreign aid projects. Liese received his medical degree from the University of Bonn's Institute of Human Genetics in 1992.

02.07.2013 As part of the outcomes of the European Commission’s Process on Corporate Responsibility in the Field of Pharmaceuticals from 17 April 2013, Member States formally endorsed recommendations on a potential coordinated and collaborative approach to address access to orphan medicinal products: the Mechanism of Coordinated Access to Orphan Medicinal Products (MOCA).

These recommendations – which have been developed collaboratively by a group of EU Member States, patient organisations, industry representatives and other stakeholder representatives – are aimed at exploring ways to increase patient access to Orphan Medicinal Products (OMPs) through cross-border cooperation.

The key conclusions and deliverables include a proposal for a “Transparent Value Framework” (TVF), which would form the basis for a structured discussion between all stakeholders around the value of an individual OMP. This would create a shared understanding between stakeholders, laying the foundations for different pricing and reimbursement discussions in individual countries.

A variety of aspects should be taken into account during this process, including the severity of the disease, the availability of other treatments as well as the impact of the new treatment on disease progression. Rarity is also a key aspect, because it increases complexity throughout the process. In many cases, an OMP might be the only available treatment for the rare disease in question. Therefore, the ability to assess its value and to make it available within a national healthcare system in a timely way is vital. The TVF attempts to capture these points and to provide the beginning of a framework for discussions around the value of an individual OMP in national healthcare systems.

The multi-stakeholder dialogue surrounding the shared challenges in ensuring that OMPs get to patients in a timely and sustainable way is also a key deliverable of the two-year MOCA process. Improved cooperation between stakeholders and across geographical boundaries is vital to giving European patients with rare diseases access to innovative and effective therapies.

The MOCA recommendations will, however, only create meaningful change for patients if there is a concrete strategy and action plan to develop the next steps that must be taken to have the necessary multi-criteria and multi-stakeholder discussions involving OMPs. Many issues still have to be resolved, and a number of steps still need to be taken to improve access to OMPs in Europe – including the need to ensure that Health Technology Assessments are able to effectively capture the value of an OMP for patients.

This sector is affected by a multitude of different factors, systems and actors. Each stake-holder within the process has a part to play. But only through a collaborative and connected approach involving all stakeholders - industry, patients, national HTA agencies, clinicians, payers and national healthcare authorities - will we be able to ensure that patients have access to the treatments they need. The joint work on the shared challenges over the past two years should now lead to the next steps as we move to increase access to rare disease treatments across Europe. Industry is looking forward to being part of that ongoing process.

Wills Hughes-Wilson

is Vice President External Relations & Chief Patient Access Officer at Swedish Orphan Biovitrum (Sobi). She is also Chair of European Industry Task Force on Orphan Drugs & Rare Diseases – a joint industry trade association group between EBE-EFPIA and EuropaBio, and is also an Industry Member of the European Commission’s EU Committee of Experts on Rare Diseases (EUCERD).

28.05.2013 In a post-petroleum society, biorefineries - along with the farmers and foresters who source raw materials - are at the heart of the economy. No matter how you define it, the concept remains the same: converting raw materials into useful products for society. Instead of fossil fuels, the biobased economy employs renewable resources and wastes to produce a series of products useful to society: biofuels, bioenergy, biochemicals, bioplastics and other biomaterials.

The potential social, economic and environmental benefits of this model are substantial across Europe. Farmers and foresters would play a pivotal role in enabling the biobased economy's goals of delivering locally-sourced and produced materials, chemicals, fuels, food and feed. With sustainability at the heart of the biobased economy, growth could be decoupled from resource depletion and environmental degradation. That would in turn boost the EU's ability to transition more rapidly to a low-carbon and resource­efficient society, and it would further enable the EU to lead and compete in a global biobased economy market that is expected to reach the 200bn mark by 2020. The biobased economy is just the project to propel Europeans onto the path of reindustrial­isation and sustainable growth, and to reverse the current investment trend toward other regions of the world. It offers a road back to prosperity across all of the regions in the EU, and creates new jobs that will not simply disappear in the mid to long term. It will build on existing EU strengths and resources, embracing technological and scientific excellence, and creating new and novel partnerships between industries that have thus far remained unconnected. Supplementing food production, the conversion of bio­mass into bioproducts will likewise present a chance for the EU27 agricultural and forestry sectors to diversify revenues and revitalise rural areas.

For about a year now, industries across sectors have come together to discuss partnership opportunities with the EU. The resulting effort is called BRIDGE - a proposed "Biobased Industries Public-Private Partnership" in the form of a Joint Technology Initiative (JTI) known as the "Biobased and Renewables Industries for Development and Growth in Europe."

BRIDGE is a €3.8bn commitment (EU: €1bn, Private sector: €2.8bn) over the 2014-2020 period, with a clear strategic research agenda that includes defined focus areas for demonstration projects and a set of flagship initiatives. Research institutes, academia and SMEs have been playing and will continue to play a crucial role in the PPP. The same goes for Member States, which will be instrumental in the process - particularly during the project deployment phase. The PPP might have been conceived in Brussels, but it will be implemented at national, regional and local levels across Europe. A Biobased Industries PPP in the form of a JTI is sending the right signals to invest in Europe, as well as to translate the EU's R&D potential into new, innovative and sustainable biobased products and markets.

By the end of June 2013, the European Commission is scheduled to propose a (recovery) package of JTIs aimed at stimulating growth and jobs and improving the quality of life in Europe. BRIDGE will be among these initiatives. The proposals are to be passed on to the European Parliament and the Council of the European Union for approval. The Biobased Industries Consortium (BIC) is calling on EU legislators to support the Commission JTI proposal for BRIDGE in order to unlock the biobased economy potential and trigger more sustainable growth in Europe.

Dirk Carrez

is the Coordinator for BIC, an industry consortium that includes more than 40 European companies and organisations from the fields of technology, industry, agriculture and forestry that are preparing the Biobased Industries PPP. Carrez is also the Managing Director of Clever Consult, a consulting firm dedicated to different aspects of the bioeconomy. He is currently the Vice-Chair of the Biotechnology Committee at BIAC (Business and Industry Advisory Committee to the OECD) and the Vice-Chair of the OECD’s Task Force for Industrial Biotechnology.

30.04.2013 The proposal for an in vitro diagnostics (IVD) regulation currently under discussion in the European Parliament and Council of the European Union will chart the future of the regulatory system for IVDs in Europe.

Dubbed the "father of healthcare" by rapporteur of IVD Regulation Peter Liese, the various rounds of discussions are beginning to underscore the importance that IVDs have in healthcare as a pathway for guiding patient management decisions. And with this recognition, an important and more detailed look at the propo-sal is also starting to take place.

It's clear that IVD manufacturers will be controlled more strictly under the new re-gulations, and that notified bodies will also be more deeply involved. With the new classification system, they will have levels of oversight in about 90% of all IVD applications. However, the involvement of reference labs - which will provide a pool of scientific expertise to authorities and notified bodies, and test samples collected through unannounced visits - will mean dealing with new operators in the future.

Specific device types are currently being highlighted, and special care is being taken to ensure that they are safe and effective. This is the case for companion diagnostics, where there is a need to ensure that both IVD authorities and medicinal products authorities are satisfied that personalised medicine is delivering on the promise it holds. The same holds true for near-patient testing systems, which are growing in importance as a means for ensuring that testing results actually reach patients and have an impact in managing their health. Finally, it is important to underline that the specific requirements for IVD software will enable a better integration of the information that is transferred from IVDs to information systems.

The regulation takes a balanced approach between pre-market and post-market controls. In the pre-market setting, the use of Common Technical Specifications (CTS) for the highest risk IVDs continues to provide a very stringent requirement that has to be met before a diagnostic reaches the market. In addition, all IVDs will now have to be supported by clinical evidence. This is to include not just their analytical performance, but also clinical performance and scientific validity - in other words, knowing how the information that is generated by the IVD benefits the patient.

A lot of the questions that have been raised by the regulation will be addressed through the way in which it is implemented. In this, it is essential that implementation be driven by the need to ensure the safety and effectiveness of IVDs, while at the same time retaining relatively rapid access to the market for them. Only this combination will result in direct benefits to both the patients and the users of these devices.

Industry will have time to adapt, but that time must be used wisely. Collecting clinical evidence must begin as soon as the details are finalised. Those companies that begin an early programme of implementation will be in the best position to benefit from the advantages of the new system, which will include greater international convergence, as well as an increased confidence in a more stringent - but possibly still supple - system for regulating IVDs.

Jesus Rueda Rodriguez

heads the regulatory team of EDMA and ensures the association's active participation in the regulatory debates that affect IVDs in the EU. He is also involved in work at the international level, acting as the EDMA representative to the WHO and ISO, as well as a liaison to other associations on regulatory matters.

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