A 12-point prescription for Europe’s healthcare biotech sector

Europe is recognised for its excellent medical science, but shortcomings in the framework for translating this to available treatments leaves it at risk of failing to capture the value – both in terms of health and of wealth – that biologic therapies provide.

The pharmaceutical and biotech industry is a big deal: It invests €30 billion a year in research and development, supports 170,000 jobs, and is part of the value chain from discovery to treatment that helps manage European health. But a Science|Business roundtable in the European Parliament on October 9th highlighted some of the hurdles still to be cleared in getting new medicines into use. The outcome: A list of 12 “to do’s” for the new European Commission and Parliament.  

The debate was held, with the support of industry association EuropaBio, as part of Biotech Week, a series of events across Europe to highlight the value that the bioeconomy is currently - and can in the future – contribute.

1.     Build awareness in the European Parliament and the European Commission

MEPs of all political colours are united in wanting to improve the climate for innovation. It is critical they understand the specifics of what is needed to drive innovation in healthcare biotech, said Kay Swinburne, MEP. “As MEPs, we should identify colleagues to make sure everyone with an understanding of science and engineering comes forward to help with putting out the message, and to look for opportunities going into the future,” Swinburne said.

2.    Make Member States aware that this is a pan-European issue

While much of the leverage and policy initiatives needed to support healthcare biotech reside at Member State level, action is needed at a European level. “We can develop strategies but if Member States are not engaged, things won’t change,” said Philippe de Backer, MEP. The Commission needs to make sure Member States follow through. One of the most important things the Commission can do is to provide independent evidence in support of the various options and policies.

3.    Prepare the path for future technologies

The value of much of the knowledge generated in Europe is never realised because the environment is not receptive. There should be a systematic effort at a European Commission level to develop policies to take advantage of future science and technology developments.

“Although there’s an awareness across the Commission that emerging science and technology will be important, there are no mechanisms in place to inform what should be done to react to, or use, the technology that will be on the table,” Anne Glover, Chief Scientific Adviser to the President of the European Commission, told the meeting.

One of the keys to translating knowledge into successful new products and services is to give citizens a say. This is particularly important in healthcare, where there is an expectation among the public that emerging science and technology will deliver new opportunities.

One potent example here is medical data, which is poised to have an unprecedented impact on health – if it can be analysed and re-used properly. However, there is understandable nervousness about letting this intimate personal information be used in research. “It’s a big decision for politicians; there must be a balance between data protection, custodianship and storage on one hand, and the use of data to benefit all society on the other,” Glover said.

4.    Involve all Directorates in a ‘health in all policies’ approach

Health is an issue that cuts across all of the Commission and which should be factored into all its policies. Dealing with antimicrobial resistance for example, requires a re-invigoration of basic research, policies to prevent overuse of current antibiotics in healthcare, better diagnosis, business models and incentives to develop new antibiotics and moves to limit the use of antibiotics in farming.

The new structure of the Commission, with links and oversight across the Directorates, will make it possible to work together and connect thinking. “We won’t have a healthy Europe, if we don’t have all the Directorates involved,” said Glover.

5.    Recognise that value is created at all stages of translation

The current sentiment, which holds that public sector financing of R&D and paying for biologic drugs is an overhead, must be reversed. “The one key message should be that value is created at every step along the healthcare biotech chain, from research to development, manufacturing and patient access,” said Emmanuel Chantelot, Chair of EuropaBio’s Healthcare Council and Head of International Government Relations at Shire.

The pharmaceutical and biotech industry tops the EU Innovation Scoreboard, investing €30 billion per annum in R&D. Individual companies invest a high proportion of revenues in research, with Shire as a $5 billion turnover company that invests $1 billion per annum in R&D, being a case in point (and incidentally, putting the EU’s healthcare research budget in context).

The return on this investment should not be viewed solely through the prism of medicines that make it to patients: healthcare biotech supports 170,000 jobs. Biologic drugs that have won approval have transformed treatment of diseases including rheumatoid arthritis and multiple sclerosis, relieving symptoms, modifying pathology and reducing morbidity. This is increasing Europe’s human capital by allowing people who previously would have been forced into early retirement to remain active and fit for work.  Healthcare biotech also underpins better diagnostics, supporting prevention.

6.    Update education and training, and organise data stores, to enable healthcare professionals to use knowledge

There is undoubtedly a great deal of new knowledge being generated within medical science, but much of it is hard to use. What would a doctor make of a patient’s DNA sequence at the point of care? Knowledge is needed to implement knowledge.

“We are gaining much knowledge that we don’t know how to use; this should be part of our education,” said Sandra Hocevar, Vice President of Education at the European Pharmaceutical Students’ Association. “Pharmacists advise patients on how to use biotech products but there is only one semester about biotech in our studies. There is a need to connect pharmacy education to the biotech sector,” Hocevar said.

Similarly, the rise of personalised medicine creates the need to update education and training of healthcare professionals. Instead of one pill for all, biologic products are coming to market that are only effective and suited for subsets of the patient population.

7.    Create an enabling framework to marry academic knowledge with industry expertise

Healthcare biotech recognises it requires access to knowledge held in Europe’s academic base, but the reverse does not hold true for academic researchers, who feel little need to know about industry skills or requirements.

“Within universities there is a striking lack of people with experience of what it takes to develop a new drug,” said Sylvie Bove, Head of Life Science at Medicon Valley. Knowledge of disease biology should be brought together with understanding of what will be required to translate these insights into clinical development programmes, she said.

Although the industry recognises the need to tap into academic knowledge, this can be difficult. The Commission could do more to help with enabling mechanisms that encourage researchers to “go and find a friend in business” and helping industry by signposting routes into the academic world.

8.    Promote collaboration on a European scale

Public private partnerships, as embodied in the European Institute of Innovation and Technology’s (EIT) Knowledge and Innovation Communities (KICs), are finding their feet and beginning to make inroads to the challenges they were set up to address.

This is starting to demonstrate the benefits of having such a framework for working at scale, said Johan Wallin, Project Leader, Innovation for Healthy Living and Active Ageing at the Karolinska Institutet. “The KIC is a catalyst for doing things together. No company can do all research in-house,” Wallin said.

In addition, scale clearly adds value in the context of healthcare, for example, when pooling and analysing patient data (with appropriate security) on a European scale.

The KIC is also providing a platform on which new business models are emerging, creating new opportunities, for example, in promoting patient involvement in the management of their own conditions through mobile health. “Pulling together academics, industry and citizens in this way, is attracting private capital and has a big contribution to make to the sustainability of healthcare systems,” Wallin said.

9.    Support biotech SMEs as risk mediators

The biggest risk of drug programmes failing comes in moving from animal research into humans and demonstrating an effect in patients in phase II clinical trials. This is the area of the discovery and development pathway where biotech SMEs tend to cluster, playing the crucial role of translating research from the academic environment and shaping it into something that can fit into the bottom end of a bigger biotech or pharmaceutical company’s pipeline.

“One of Europe’s relative advantages is our knowledge base, and SME biotechs are critical in adding value to knowledge generated in Europe,” said Thomas Saylor, CEO of Arecor and chairman of the EuropaBio SME platform.

Confronted with the problem of falling in house R&D productivity, the pharma industry has turned to SME biotechs to plug the gap. “There is a very natural synergy and that’s why it is important to have a vibrant SME environment and to support SMEs as risk mediators,” Saylor said.

This includes providing public funding for SMEs to foster development to the point where science is refined and validated, and a biotech can attract private investment.

10.    Make the medicines regulatory system reflect the science and the patient needs

The system for regulating drugs remains fixated on one-size-suits-all small molecules and is failing to get healthcare biotech products based on detailed understanding of disease biology and designed for small patient populations, from lab to clinic.

“We can understand the flaws in basic biology that are driving disease and increasingly have the tools to do something about it: this is a challenge we should rise to,” said Alastair Kent of Genetic Alliance UK, a patients’ body representing a range of rare diseases.

To get a sense of what a 21st century regulatory system should look like, reflect on how patients with rare diseases would regulate drugs. Obviously they are not interested in things that do not work, but they are interested in things that might work. However, the current model for carrying out clinical trials makes it extremely difficult to deliver statistical proof of efficacy in rare diseases.

In addition, regulators are focussed on hard clinical endpoints that do not take account of what is significant for patients. One notable example is the six minute walk test, used to assess biotech products now coming through the pipeline for treating the inherited muscle wasting disorder, Duchenne Muscular Dystrophy. The test is irrelevant to many boys with the condition who are unable to walk, and for whom an effective therapy would be one that maintained the muscle function they need to keep operating an electric wheelchair, or be able to use a computer.

“The regulatory system tends to be drawn up by lawyers, whereas it needs to reflect biology,” Kent said. “If you put patients at the top of the process, you will quickly realise what is important.” For example, there should be more use of adaptive licensing, in which products that are safe and have been shown to have some effect in a limited population are allowed on the market, so that real world data of how a product performs can be gathered and the license extended to other patient groups if appropriate.

“Classical clinical trials do not work in the context of specific understanding of disease biology and targeted products. We need the regulators to give ideas a chance to work and to put the patients’ perceptions of benefits/risks at the centre of their thinking,” said Kent.

11.    Encourage Member States to harmonise health technology assessments and simplify commissioning and market access

Getting regulatory approval is no longer the finishing line. Now products need to get a tick from national health technology assessment bodies too. These operate to different rules and for the most part their cost benefit analyses are based on existing, embedded – frequently generic – treatments. “Health economics is stygian. It knows the cost of everything and the value of nothing. Health Technology Assessment agencies should look at the value of innovation, as well as its cost,” Kent said.

If health technology assessment is anti-innovation, reimbursement, commissioning and the lack of receptiveness with which healthcare systems greet novelty, is worse. “These processes are based on the fear of doing the wrong thing, not on the impetus to do the innovative thing,” Kent observed. “How can you ever hope to get healthcare biotech applied in systems like that?”

12. Use unmet medical need to create a pull mechanism and reduce risk

Early stage research frequently consists of an idea looking for a problem. This increases the risk for banks and other financiers, noted John Davis, Head of Division, Life Sciences and Health at the European Investment Bank (EIB).

The EIB has been collaborating with the Gates Foundation and others to create a pull mechanism, in which a product that meets an unmet medical need is scoped in advance. “We have been working to come up with product specifications and then going to public health bodies and asking if they would buy such a vaccine and implement it, in effect getting market commitment in advance of investing in development,” Davis said.