Interview with Andrea Rappagliosi, EuropaBio

EuropaBio welcomes the engagement of the Commission, especially Commissioners Geoghegan-Quinn, Dalli and Tajani who have set personalised medicine as a priority in their respective portfolios. Today, personalised medicine represents an opportunity to bring the right medicine to the right person at the right time and can actually increase the value of treatment by deliberately selecting only eligible patients. Though much has been said about a new era of personalised healthcare, there is an urgent need for a predictable and coherent EU regulatory framework, including both incentive and reward mechanisms, adapted to the current paradigm shift in healthcare biotech.

Another area in which healthcare biotechnology can continue to contribute significantly is rare diseases. There are more than 5,000 rare conditions in the world and many of them have a genetic origin. The approval of the Orphan Medicinal Products Regulation in 2000 saw the Commission take major steps in order to address the access issue.

Furthermore, I believe that both the EUCERD (European Union Committee of Experts on Rare Diseases) monitoring programme and the development of Member State National Plans have great value. Biotech can contribute by delivering greater understanding of the components of rare diseases and therefore cooperation is needed with European and national institutions in order to increase the success rate of orphan drug availability and to ensure that these drugs reach patients in both a timely and equitable manner.

Another area of importance for the biotech industry is the series of initiatives aimed at simplifying the framework programme for SMEs, under FP7. Unfortunately we’re not there yet, however I think that by FP8 we’ll get to a level where SMEs can better participate. The Commission and Europe are on the right track to achieve the 2020 targets.

I would like to stress that the Active and Healthy Ageing Innovation Partnership recently launched by the Commission and Member States, aimed at increasing the average number of healthy years by two by 2020, has implications for biotechnology. An important aspect of the proposal concerns strategies to boost research in major diseases that are faced by the elderly, such as Parkinson’s disease and Alzheimer’s disease. Here, biotechnology can play a significant role in unveiling the genetic components of these diseases and by acting as a scientific platform to improve R&D performance.

In the long term, I believe there should be more than one EU policy to support the development of innovative drugs. There should be a convergence of policies to ensure a stable, predictable environment for biotechnology development and innovation.

Biotech takes time; the timeframe from the lab to the market and finally to the patient can easily take over 10 years, and this requires long-term stability and predictability of the environment in which we operate. Investors need to know exactly what is, and what will be, the process in order to arrive at a successful outcome. Moreover, while the biotech industry is willing to take entrepreneurial risks, there must also be a fair system of rewarding these risks at the end of the development phase.

From a biotech perspective, the proposed single European patent would be more than welcome, as it would bring simplification to the system. EuropaBio represents around 1,800 SMEs throughout Europe so the chance for them to share knowledge and translate this into a means to attract further investment would be a step in the right direction.

The Transparency Directive was and still is a visionary piece of legislation. It created two fundamental principles necessary to facilitate patient access throughout Europe: a maximum time of 180 days for the definition of pricing and reimbursement levels as well as the need for a visible rationale behind access decisions.

Unfortunately, during its 21 years, the Transparency Directive has not always been implemented as foreseen and significant delays in patient access were generated by short-term cost-containment policies by national governments, often aimed at rationing access as opposed to reaching optimal use of resources. EuropaBio is keen to engage and contribute to a series of policy interventions taken by the European Commission regarding the access issue, namely ensuring that drugs can be offered in an equitable, accessible and affordable manner to patients throughout Europe.

Vice-president Antonio Tajani launched a social responsibility initiative in the pharmaceutical sector to improve patients’ access to medicines in Europe. To do this, it sets out to assess the tools for orphan drugs and biosimilars and then address the issues from a European perspective about managed entry schemes and contractual mechanisms to better acknowledge the value of medicines and improve equity in access. The latter is extremely interesting as it can help foster a more predictable environment for the biotech sector.

Pharmacogenomics is playing a key role in biotech research. It is likely that by 2015, healthcare biotech knowledge will be used in the development process for all new pharmaceuticals. The pharma industry is rethinking and retooling its R&D. We are moving from a phenotype to a genotype approach, which involves understanding DNA and the basics of how individuals respond to drugs.

Genetics and pharmacogenomics will widen the numbers of diseases that can be treated effectively as well as limit the number of adverse drug reactions on patients. Moreover, there will be an increase in both the safety and efficacy of treatments as there will be less trial and error to find the optimal dose and treatment. Pharmacogenomics is a tool that has proved itself to be a powerful springboard of knowledge in R&D.

There is a growing debate surrounding the role of diagnostic testing in personalised medicine. The use of diagnostic testing has steadily expanded, moving from simple diagnosis into screening, predisposition, prognosis, prevention and therapy monitoring.

However there is a vicious circle in the access systems of diagnostics whereby poor reimbursement leads to less funding for public or private research, little incentive to use properly designed clinical studies, a lower level of evidence and few recommendations for clinical use, and therefore poor valuing of diagnostics. The value of diagnostics has not, so far, been appreciated. However this is beginning to change, in part due to the fact that pharmaceutical, biotech and diagnostic companies are increasingly collaborating in the development of companion diagnostics for medicinal products, thus breaking silos between disciplines.

Diagnostics plays a big role in biotech. Medical device regulation is separate from the pharma regulation and from a licensing or registration point of view they are different environments. What is important is to clarify which tools can be used to value companion diagnostics most appropriately and to make sure that this value is reflected in rewards to the manufacturers. Health Technology Assessment could be extremely helpful in framing and understanding the value of this novel approach to disease management.

Since the discovery of the human genome a decade ago, we have increasingly improved our understanding of the causes of diseases. Biotech has enabled us to move from treatment to cure and this represents a paradigm shift in the way we approach disease management. Several diseases that were once almost untreatable, such as certain types and subsets of cancer and autoimmune diseases like multiple sclerosis can now be treated and, importantly, better understood.

I think the greatest paradigm change is that biotech started in specific areas, including orphan drugs and autoimmune disease, but now more and more biotechnology is being used in the research and development of medicines for common diseases. As we speak, more than 350 million patients have already benefited from approved medicines manufactured through biotechnology to treat or prevent cancers, heart attacks, stroke, multiple sclerosis, leukaemia, diabetes, rare conditions and other diseases.

It is always difficult to compare the European and US environments and biotechnology is no exception. In the early 1980s, the US healthcare biotech environment was significantly boosted by two strategic pieces of legislation: the Hatch-Waxman Act of 1984 on intellectual property and the Orphan Drug Act of 1983.

The other main difference is the single stock exchange in the US, including the role played by Nasdaq in investing in small and medium life science enterprises. In Europe many roadblocks still need to be addressed; the fragmentation of the financial environment and of funding for research, dispersed venture capitalists, plus the fragmentation of patient access at the end of the R&D cycle.

As a result the situation in Europe is far more complex. A more holistic way must be found to avoid such disjointedness of the market, which hampers the development of therapies. The innovation race is not a 100-metre dash with one winner, but more like a marathon where everybody wins: patients, governments, regulators, payers, academia, researchers and industry.

Looking at examples of European countries that are supportive of the biotech industry, I think the UK has led the field historically, always paying attention to investment in R&D and maintaining and retaining excellent R&D. Initiatives like the Office for Life Science’s Blueprint and the Patent Box support the life science business environment and have kept the UK at the forefront of Europe.

Nevertheless, the intrinsic collaborative nature of the biotech industry, linking academia, patients and industry, has also found fertile ground in other areas of Europe, resulting in the creation of bioregions or bioclusters.

Examples include the Medicon Valley Academy (regional Swedish/Danish network organisation), Stockholm BioScience, Lyon Rhone Alps Life Science Network, ScanBalt Bioregion (a metaregion comprising Denmark, Estonia, Finland, Iceland, Latvia, Lithuania, Norway, Poland, Sweden, northern Germany and north-western Russia), Biotech Region Munich, as well as the BioAlps cluster in western Switzerland, with important collaboration in France and in Italy.

Since it was created in 1996, EuropaBio has grown substantially and evolved from representing just the science to representing and supporting society as a whole. It has adopted a set of Core Ethical Values relating to the way the biotech industry conducts R&D and it is very important to have these in place in order to meet the demands of society.

We are committed to the socially responsible use of biotechnology, including genetic engineering, to improve quality of life by the prevention, diagnosis, treatment and cure of diseases. Our members are committed to increasing understanding of biotechnology among all stakeholders including the public.

EuropaBio also promotes open, informed dialogue with all stakeholders about the ethical, social, environmental and economic aspects of biotechnology as well as its applications. It has a broad remit, representing the healthcare sector, industrial biotech, agri-food biotech, upstream biotech, and more than 1,800 SMEs.

Many of these organisations have central links with academic boards and universities in Europe, which is a great attribute. EuropaBio is increasingly the voice of this reality and of healthy research in Europe.

I think EuropaBio’s future lies in continuing to be the voice supporting this dynamic environment in Europe, assisting SMEs or spinoffs from universities, providing links with venture capitalists and focusing attention on the potential of R&D in healthcare. It will assist smaller companies with knowledge that they do not have in-house and ensure that they are engaged with pioneering European projects.

Further, it must continue to push for a system of rewarding R&D and ways to facilitate access to patients through value assessment of new drugs. There is certainly room for EuropaBio to grow in this debate.