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Andrea Rappagliosi: Challenges and answers: HTA in the economic crisis
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Andrea Rappagliosi: Challenges and answers: HTA in the economic crisis

13.09.2012 - During the last thirty years, healthcare expenditure has been growing much more rapidly than GDP in OECD countries, causing increasing concerns about the long-term sustainability of current trends. As we have seen with the global impact of the financial crisis, economic realities are moving faster than political ones.

The standards of healthcare that European citizens are provided with today risk becoming economically unsustainable, if the efficiency of healthcare systems is not factored in when assessing the value of prevention and treatment of diseases. Against this background, we need to look in more detail at the determinants of healthcare expenditure in the countries of Europe, explicitly taking into account aspects like the role of income, the effects of an ageing population, life habits, technological progress, and institutional and budgetary variables. 

When developing products and solutions for European patients today, the research-based pharmaceutical industry is responding to evi-dence--based requirements put in place by regulatory agencies – which request proof of efficacy, quality and safety – and 30+ Health Technology Assessment (HTA) agencies across Europe, which want proof of relative effectiveness. In the area of prevention, the vaccine industry has to provide dedicated evidence to public authorities, which in turn make recommendations prior to any public prevention programme. There is a potential for unnecessary duplication between these different requirements, as they are actually sourced from the same sets of data.

The need to secure timely, equitable and sustainable patient/citizen access to healthcare services has led to a growth in collaboration between HTA agencies recently, and this has been strongly supported by the European Commission. Industry believes there is room for this initiative, whilst simultaneously keeping regulatory and HTA processes separated and keeping pricing and reimbursement decisions fully at the national level. 

Of particular note is the collaboration between HTA agencies, which is supported by the European Commission and will be formalised in a permanent network as of next year. The current network (EUnetHTA) is working on developing methodological guidance for relative effectiveness assessment. Industry is supporting this approach, and is contributing to this discussion in order to feed in experience and know-how. Moving forward, industry would certainly favour increased collaboration between the regulatory world and the European collaboration on HTA, as this would ensure that evidence-based requirements receive the best scientific attention and are aligned across the range of decisionmakers, allowing for an ongoing dialogue between the parties. What is crucial is that consultation with stakeholders, including those in industry, is warranted. 

Today, the key challenge for governments is designing pluralistic systems of healthcare delivery and financing. In this area, a well-balanced mix of public and private financing would put market forces to work promoting investment and innovation, without imposing unsustainable burdens on public budgets or denying prevention and care to the disadvantaged population.


Andrea Rappagliosi

Andrea Rappagliosi is Vice President Market Access, Vaccine Advocacy & Medical Affairs at Sanofi Pasteur MSD, the only company in Europe totally dedicated to vaccines. During his career in both the public and the private sectors, he has focused on access issues that impact patients and healthcare systems in Europe. Rappagliosi is also VP of the European Vaccine Manufacturers Group and co-chair of the EFPIA HTA Task Force.

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.

12.04.2013 The use of diagnostic testing is at the centre of Personalised Healthcare (PHC), not only for classifying a disease but also for determining which therapy will be most suitable for an individual patient or a stratified group of patients suffering from that disease. It is all about improving outcomes and tolerability.

Dr Hagen Pfundner, Chairman of the German Association of Research-Based Pharmaceutical Companies (vfa) and General Manager of German Roche Pharma AG

This concept keeps transforming oncology, and has made its debut in other therapeutic areas like neurology as well. PHC represents not only medical progress, but also highlights the potential to improve cost-efficiency of healthcare delivery by helping to avoid ineffective or even harmful therapies. To fulfill this potential, however, it must find its way into routine healthcare practice. Until now, this has all too often remained a long and winding road.

The European Medicines Agency (EMA) and the European Commission embraced the concept of PHC from the beginning, and continue to encourage manufacturers to include up-front testing (patient stratification) in clinical trials design and subsequently in labelling for new drugs, provided the scientific evidence is sound.

However, drug approval by the European and national health authorities is only one step on the “road to the patient”. Delays most often occur when it comes to including newly-defined tandems of a drug and a diagnostic test (companion diagnostic) in national reimbursement schemes. Procedures and processes for this step are different in every European country, but there appears to be a common theme: no healthcare system is prepared to handle the situation in a synchronised fashion or within a reasonable time frame. In the out-patient setting, a newly-approved drug is reimbursed following health authority approval, but a new companion diagnostic test – even if mandatory by label – is not. These reimbursement decisions are made by a different body made up of health insurance companies and associations of office-based physicians that organise physician fees. In the past, decisions have often taken several years.

In the US, the Food & Drug Administration (FDA) is moving towards encouraging regular testing as the preferred model for companion diagnostics in PHC. It has issued guidance focusing on the dependency of Rx and Dx co-approval, while the tests have been provided as a service by specific laboratories.

So while PHC is meant to shorten the time it takes for a patient to receive the most suitable treatment, in practice medical treatment is often delayed for many patients due to a misalignment of the two divergent reimbursement systems for drugs and companion diagnostics. This is unacceptable for modern European healthcare systems, and provides a disincentive for pharmaceutical and diagnostic companies. The irony is that while the scientific advances of PHC have opened the door to significant improvements in therapeutic and cost efficiency, the current healthcare systems are not equipped to benefit from them. Last but not least, the barriers also run counter to government efforts and support aimed at making Europe the leader in PHC research.

There is no “one size fits all” approach in Europe when it comes to including innovative PHC treatments in national reimbursement schemes. But national stakeholders should increase their efforts to resolve this in their countries – and not just in their own interest. Patients, physicians and innovators deserve it! 

Hagen Pfundner

joined Roche Pharma AG in Germany in 1992. Since 2006, he has held the post of General Manager at Roche Pharma AG in Germany. Since 2008, he has also been a member of the executive board of the German pharma association vfa. 

21.02.2013 In an era when Member States are doing their utmost to limit national exposure in the EU budget, making the right choices has become more crucial than ever before to shaping Europe’s future.

Derrick Williams, European Biotechnology Network, Brussels

The problem with the current tenuous economic situation is that research-driven sectors like biotechnology need reliable support. Without it, they will be unable to deliver jobs and growth in the global markets of the future – among them biological production, personalized medicine and health-enhancing nutrition. To remain at the forefront of these fields and employ funds effectively, Europe has to foster even more collaboration, while at the same time putting frameworks in place to prevent research projects from being being carried out more than once.  Both Big Pharma and global agribiotech players have to fully implement biotechnological platform technologies in product development. To improve their products, global industry leaders – like BASF in chemistry, for example, or Nestlé in nutrition – increasingly rely on biotechnologies invented by academic groups and perfected by life sciences SMEs. While this biologisation of industry is moving forward at a global scale, continuous support for collaboration is essential to ensure that innovations continue to flow steadily in both directions. In the past, Europe has supported R&D collaboration with significant amounts of funding (Framework Programmes) as well as through the creation of European research infrastructures provided by the the European Strategy Forum on Research Infrastructures (ESFRI). One example is the EU biobanking resource BBMRI, which is aimed at pooling data from local biobanks in Europe and harnessing it for drug target and biomarker discovery. This lays the foundation for personalised medicine and therapies that target diseases where they need to be battled – at their roots. Better collaboration and data standardization are key to squeezing the greatest possible gain from pooled resources. That rule of thumb is also true for other collaborative EU projects that generate huge datasets for the research community. To ensure that public funds are invested well, one fairly simple step would be to make collaboration and standardization of research outcomes – i.e. by means of ring tests – a prerequisite for EU funding. This would help improve comparability in research results, and prevent duplication in research projects. The only way to guarantee the high-quality results that future markets will demand is through collaboration and networking in the 27 Member States of the European Union, Switzerland and Norway. Only through collaboration among all the stakeholders in the value chain can we set the common standards necessary to take full advantage of vast projects, like searching cancer patient genomes for new targets and diagnostic biomarkers. Realizing European synergies can only happen, however, if potential partners are aware of one another. EU science, research and industry has yet to make really good use of the advantages provided by a common market, when these could be helping to drive even faster technology transfer and even more flexible labour. And at the end of the day, establishing a truly sustainable future through biologisation will never be achieved without even more political commitment.

 

Derrick Williams

Derrick Williams is the head of communications at the European Biotechnology Network in Brussels. A biologist, the US native is a science correspondent and media professional who has worked in Europe since 1996. Williams is a journalist and PR professional in a variety of different formats, including both broadcast media and print. His productions, TV reports and articles generally focus on helping the wider public better understand biotech and life sciences topics. 

21.12.2012 The revision of the EU Medical Devices Directives (MDD) could not have come at a more critical moment. The recent PIP breast implant incident has made it crystal clear that healthcare actors must pull together to ensure similar cases of fraud never happen again.

Serge Bernasconi, Chief Executive Officer, Eucomed, Brussels

We believe Europe needs a clear, predictable and effective regulatory system that deserves patient trust – a system that provides them with safe and timely access to the latest innovative technologies. The European Commission has tabled a proposal, and we welcome the majority of their recommended measures. They improve patient safety, do not unnecessarily delay patient access to life-saving medical technologies and do not hamper innovation. The proposal is on the right track in several key areas, and we applaud the Commission’s calls for improving notified bodies, increasing EU Member State coordination on vigilance and establishing a central registration database that can be accessed by governments and patients alike.

But as we work together towards an improved system, we must remember that the current decentralised notified body system has provided a high level of safety, and consistently granted people in Europe the very fastest access to life-saving medical technologies.

We are convinced that, with the support of a regulatory framework that successfully marries safety and the availability of new technologies, we can remain in this privileged situation in Europe. The Commission’s proposed “scrutiny procedure”, however, represents a step in the wrong direction. When considering changes to the EU regulatory framework, we must strive to answer three questions:

1. Will the changes improve safety for patients?

2. Will they allow life-saving medical technologies to be made available to people in Europe just as fast as they do today?

3. Will they encourage the innovation that European healthcare systems urgently need to become more efficient?

In the case of the scrutiny procedure, we believe the answer to all three of these questions is “no”, and therefore strongly oppose the proposed measure. The procedure will fundamentally change the current system, but will not lead to the desired outcome: increased safety for patients. It would address some calls to move towards a centralised, pharmaceuticals-like system, but will ultimately harm European patients and negatively impact European small and medium sized enterprises (SMEs). Recent independent research has shown that systems like these (similar to that at the US FDA) delay patient access to new technologies by 3 to 5 years without delivering additional safety.

Europe needs a decentralised system that is specific to medical devices if it is to maintain safe and timely patient access to technologies, and keep Europe’s medical technology “innovation engine” running.

We are committed to providing medical technologies that improve people’s lives and that are at the same time cost-effective. Europe has become a leader in healthcare innovation, and now more than ever it needs to cope with increased pressure on national healthcare budgets. The current regulatory framework has provided a high level of safety for patients in Europe without delaying access to life-saving medical technologies. Let’s not unnecessarily drive Europe’s strong innovation and research capabilities to other continents at a time when they are urgently needed here.  

Serge Bernasconi

Serge Bernasconi, currently the Chief Executive Officer of Eucomed – the European Medical Technology Industry Association – has more than 30 years of experience in the worlds of pharmaceuticals and medical devices at companies such as Johnson & Johnson, Schering Plough in the US and Europe, and more recently Medtronic. In his capacity as President & International Regional Vice President of Medtronic France, Bernasconi was elected President of APIDIM (The French Association for the Promotion of Innovation in Medical Devices), and Vice President and Treasurer of SNITEM (French Medical Technology Industry Association).

20.11.2012 Partnering and even open innovation is becoming increasingly important for our industry in a world where health systems are undergoing profound transformations.

Nigel Sheail, Head of Global Business Development & Licensing, Bayer HealthCare, Leverkusen

As the global population has grown above seven billion, emerging markets have become major markets. Mature markets around the world are aging. These factors and others are giving rise to greater medical need at a time when many health systems are reaching their financial limits. One key to addressing these challenges is innovation – both internal and external. 

In this context, partnering plays an important role. At Bayer HealthCare, we are committed to research & development in areas with high medical needs like oncology, cardiology, gynecological therapies, hematology and ophthalmology. Our partnering activities are also addressing both regional and local business needs, and we are constantly looking for new collaborations with academia, pharma and companies in the biotechnology field across all phases of the value chain to combine the innovative science of our partners with our drug discovery activities, our development experience and our marketing expertise worldwide. 

Today, we are well-positioned, with a strong portfolio of innovative products and an outstanding pipeline of new developments. However, to add more value for physicians and patients, our company is seeking to think beyond the scope of individual products and increasingly develop healthcare solutions. In the cardiovascular area, for example, such solutions could start with prevention, encompass diagnosis and treatment where disease does occur, and also protect against recurrence – a truly comprehensive approach to heart health. Following the idea of developing innovative solutions, as well as individual products, our business deve-
lopment organisation brings together colleagues from Pharmaceuticals, Consumer Care, Medical Care, and Animal Health divisions. 

One of the unexpected benefits of the tight financing environment over the last few years is that companies are forced to collaborate early on in the drug-discovery and development process. This may allow the next generation of molecules to benefit from ‘collaborative advantages’ – bringing together the science and experience of organizations for mutual benefit, and ultimately increasing the chance of developing new therapies that will have an impact. These days, more than 80% of Bayer HealthCare meetings at partnering conferences deal with pre-clinical opportunities. 

Our company is open to all types of collaborations, but prefers structures that allow both partners a seat at the table and real input to ensure the best decisions are made for the programs. We want to work with strong partners that thrive independently, and ideally will bring multiple opportunities to the table. We also recognize the need to meet the needs of investors who will need successful exits if they are to have confidence to reinvest in healthcare innovation

The growing importance of collaborations in our industry is also nicely reflected by the demand for, and increasing number of, partnering events worldwide. Events like the upcoming Bio-Europe offer excellent opportunities, both to catch up with companies we already know and to meet new companies or academic institutions at an early stage of their development. 

Nigel Sheail

Nigel Sheail, has been a member of the Bayer HealthCare Executive Committee and Head of Global Business Development & Licensing since November of 2011. The biologist (BSc, University of Edinburgh) began his professional career in the pharmaceuticals industry in 1990 at GlaxoSmithKline, before transferring to F.Hoffmann-La Roche Ltd. in 1993. There he held a variety of positions with increasing responsibility in both the firm’s finance and research organisations. Prior to joining Bayer HealthCare, Sheail was Head of Corporate Mergers & Acquisitions and Head of Global Pharma Licensing at F.Hoffmann-La Roche Ltd. in Basel.

17.10.2012 Since the beginning of the year, there have been numerous government initiatives in the most advanced countries to implement and strengthen policies to support biotechnology.

Emilio Munoz, Chair of the ASEBIO Scientific Committee, and Regina Revilla, Chair of ASEBIO

The UK launched a strategy early this year to promote the life sciences through a framework agreement between the pharmaceutical and biomedical industries, universities and the NHS that is aimed at making research more efficient. Sweden also implemented a national strategy to produce an economy based on biotechnology and sustainable development. The US unveiled the ‘Fostering a Biobased Economy’ programme, while Europe is working on the ‘Innovating for Sustainable Growth: a Bioeconomy for Europe’, which focuses on applying innovation and biotechnology in such areas as agriculture and energy.

ASEBIO thinks that the Spanish Government should follow this lead. Biotechnology is a field of scientific endeavour in which Spain is already successful. That success is attributable to many factors: a long-established scientific community that has attained a very high standard in qualitative and quantitative terms; strategies and policies to foster biotechnology, promoting an appropriate linkage between science and business, which has made good – though insufficient – progress; appropriate cooperation between the public and private sectors (e.g. ASEBIO, SEBIOT, SEBBM); a successful move to internationalisation and competitiveness (e.g. BioSpain, Zeltia, Grifols, Oryzon, Progenika); and intelligent use of the diversity of niches that biotech offers.

Those factors make Spain a country to be reckoned with in working towards a sustainable bioeconomy, but the industry here needs regular funding, continuous support for links between the public and private sectors, and clear strategies towards internationalisation and competitiveness. There are a number of success stories among Spanish companies, firms with a wholehearted commitment to R&D and innovation. They want biotechnology to play a more prominent role in the transformation of society and the economic model, with a tax policy that fosters the development of biotech, more uniformity in autonomous region policies, and determined support from public and private funding. This will  not only facilitate the creation of technology start-ups, but also finance proofs-of-concept to enable companies to bring products to market.

The BioSpain 2012 event showcased our potential. It saw over 1,800 attendees (+20%), 762 companies (+9%), 217 exhibitors (+29%), an international presence amounting to 28% of the total, and a partnering section that hosted nearly 3,000 business development meetings (+29%).

The time has come for us to help Spain rise from the ashes of its beleaguered economy and expand, buoyed up by innovation. We have the flame, and the proposals to achieve it: promotion of public procurement of innovative technology, enhancing Spain’s presence in European projects, establishment of a stable framework and favourable tax treatment, determined support for entrepreneurs and innovative products, and encouragement for large industrial groups to get involved in this sector. We have the potential here and now to catalyse a recovery from the crisis. To that end, we offer our full support at any time to politicians and businessmen who are willing to listen, and who are receptive to a common project that can effectively produce growth.


Regina Revilla Pedreira

Regina Revilla Pedreira is the President of Asebio since July 2011. During her career, the Director of Policy and Communication at Merck, Sharp & Dohme (España) has been Pharmacy General Director in the Ministry of Health.

Emilio Munoz

Emilio Munoz is a member of the National Research Council of Spain (CSIC) and the President of ASEBIO’s Scientific Committee. He also has been the General Director for Science Policy in Spain and the General Secretary of the Spanish National Plan on R&D. He has been elected to the French Legion of Honour for his achievements. 

26.06.2012 Twenty-eight percent of the products given marketing authorisation between November 2010 and October 2011 in the US and one-third of all the new drugs in the EU have biotechnological origins. And experts unanimously agree that the market share of biotechnology products will continue to grow in the future. While the small-molecule drug market is predicted to expand by 3.9% annually between now and 2015, the market for biopharma products is expected to grow by more than 10% a year.

Erik Bogsch, Managing Director of Gedeon Richter and Chairman of the Hungarian Pharmaceutical Manufacturers Association Board

The trend is further bolstered by the fact that approximately one-third of current clinical development projects are of biotechnological origin. According to expert estimates, seven of the world’s top ten drugs will be of biotechnological origin by 2016. Based on the above, it can be firmly stated that biotech medicines are making a substantial contribution to the world’s healthcare system, including that of Eastern Europe.

The most appropriate way to ensure affordable biological medicines is the use of biosimilar versions of new biological entities after patent protection has run out. These products have a very similar biological profile – including efficacy, safety and tolerability – and they are, of course, cheaper. The EU was the first to introduce this type of regulatory pathway, but now several other countries, including the US and Japan, apply a regulatory approach that differs in only minor ways.

These forecasts and data unequivocally support Richter’s strategic decision to set up a biotechnology portfolio. Our aim is to create a complex and competitive biopharma line that will help expand the company’s portfolio with high added-value products, and we are making great progress in establishing the infrastructure supporting the development of biosimilar products. In 2007, Richter and Helm AG established a joint venture for the development and manufacture of microbial proteins. A biotechnology pilot plant with the capability to develop biosimilar versions of monoclonal antibodies went operational at our Budapest site in 2009. Most importantly, Richter’s new biotechnology plant in the Hungarian city of Debrecen was completed and officially inaugurated in April. The state-of-the-art €84m  facility will initially produce samples for clinical tests with the use of mammalian cells, and will begin to manufacture drugs for treating human diseases in 2014. The plant will later produce proteins and antibodies for fighting cancer and chronic inflammatory diseases.

The governments of nearly all CEE countries have recognised the high priority and important role of biotechnology among innovative technologies,  and some of them (including Hungary) have made it a vital part of economic development plans. In this respect, Richter’s strategic move was definitely in harmony with the intention of CEE countries and, in a wider context, the world itself. 

Exploring new areas is always a risky proposition. Still, I am confident that Richter’s forward-looking innovative spirit will help us achieve our goal: a biosimilar-enforced product portfolio that can help to improve quality  of life for even more patients, whether they live in Eastern Europe or anywhere else on the planet.

Erik Bogsch

Erik Bogsch is the Managing Director of Gedeon Richter Plc., a leading European specialty pharma company, as well as Chairman of the Board at the Hungarian Pharmaceutical Manufacturers Association. He holds a degree in chemical engineering and economic engineering. Bogsch began his career at Richter in 1970 in R&D managerial positions, and became CEO in 1992. He was fundamental in turning the firm into a major multinational company. His achievements and efforts to promote Hungarian innovation have been recognised with a number of awards. 

30.05.2012 What is the Human Toxome Project? It’s a wide-reaching programme aimed at helping us to reconsider how hazard/risk assessment has been performed over the last 50 years on marketed substances like chemicals, cosmetic products, pharmaceuticals, pesticides, biocides and feedstuffs.

François Busquet, Johns Hopkins University, Center for Alternatives to Animal Testing, CAAT Europe Policy Coordinator, Brussels

It’s necessary because most of the scientific community now accepts that animal models for testing the safety of these products have more limitations than advantages. In basic terms, the loose genetic homology to humans is no match for modern cell-culture technology, which promises to be much more apt to correctly predict toxic effects in humans. These cell cultures can additionally be used in the high-throughput screening (HTS) necessary to test growing numbers of products. In the last 15 years, cell cultures have replaced acute/short-term in vivo tests. However, chronic/long-term tests are the real bottleneck for these models, which do not accurately represent the complexity of multiple mechanisms, and for which it is very difficult if not impossible to maintain integrity (e.g. for a month of exposure). More recently, new environmental and human health challenges have appeared along with the ubiquitous mix of chemicals in the environment, among them endocrine disruptors and nanoparticles, and no test can unequivocally determine the toxicity of – or absence of – these compounds.

In the US, the Toxic Substances Control Act Reauthorization – an equivalent of the REACH programme – might address more than 80,000 chemicals. In preparation, the ToxCast programme for predicting potential toxicity for pesticides or environmental pollutants has been started by the EPA based on HTS, and it has now been extended to nanomaterials, cosmetic ingredients and drugs that failed to make it to market. How is the US dealing with these issues? 2007 was a cornerstone year for Tox-21c and the beginning of a paradigm shift in hazard/risk assessment. The NRC’s hallmark publication “Toxicity Testing in the 21st Century: A Vision and a Strategy” prompted a new, upbeat atmosphere. Its key proposal is to base regulatory toxicology on mechanisms and modes of action (preferably human-based models). The term “pathway of toxicity” (PoT) was coined to describe this concept. Then in 2010, the EPA assessed the safety of the chemical dispersants being used to treat the massive oil spill in the Gulf of Mexico by performing HTS. Analyses were concluded in about two weeks, instead of the six months it would have taken using traditional animal models.

Assuming that the mechanisms of toxicity are finite in number, the next step would be to map and annotate all of these PoT in humans to extrapolate the toxicological effects at the individual scale. To this end, it makes sense to create a public database of PoT – the Human Toxome. This could tackle animal welfare in laboratory experiments, speed up access to market for safer and better products and aid in the development of new analytical tools based on the knowledge gained. Pilot projects steered by CAAT and funded by the NIH and the FDA already exist. At the moment, they are focussed on endocrine disruptors and neurotoxicity pathways respectively. Discussions about a European branch have been initiated for the project, which is likely to be the size of the Human Genome Project – a task not for a single group, but for the international community.

François Busquet

François Busquet studied Biotechnologies at the ENSTBB in France. He graduated from the TU Dresden, completing his PhD at the MerckSerono group in Germany on the development of a new screening assay to detect proteratogenic compounds using zebrafish embryos. After his studies, he worked in ECVAM at the Joint Research Centre (European Commission) in Italy on the coordination of the OECD validation study of the zebrafish embryo toxicity test. Since 2012, Dr Busquet has been responsible for the CAAT-EU Policy Programme in Brussels.

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No liability assumed, Date: 30.07.2014


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