[Induced Pluripotent Stem Cell] therapies – they said we want to be a leader. These are the environments we compete with,” said Bravo, suggesting that Europe needs to become a greater champion of advanced therapies to keep up with such counterparts.

Public funding, as well as private investment, is needed, panelists agreed, with Bravo pointing to newer initiatives like Horizon 2020 as showing promise.

An apparent lack of funding does not mean that Europe is at a lack for ideas. On the contrary: At Van Deventer’s Venture Capital firm, Forbion Capital Partners, “we see about 600 business plans per year – but we can only invest, at most, in six companies.” Offering the venture capital perspective, Van Deventer highlighted the difficulties that “big pharma” is facing and pointed to smaller biotechs – most of which are heavily VC-funded – as the being at the forefront of advanced therapies development. “There is no life science development without venture capital. To phrase it even more strictly: There will be no new drugs without venture capital,” he asserted. According to Van Deventer, currently 30-40% of all large pharma blockbusters have been sourced outside (i.e. Lipitor, Fosamax, cozaar, humira, remicade).This is expected to increase to 70% or more. Drugs are sourced from small to medium-sized biotech companies – and virtually all these companies are funded by VC.

Others in the room were more cautious in painting a fragmented picture that poses big pharma against smaller biotechs, noting that larger companies have also explored the potential of advanced therapies. Additionally, it was emphasised that such a polarisation does not help either side – or, in the end, patients.

A Need for Regulatory Stability and Streamlined Reimbursement

Drug development is a long and risky road. Adding complex European regulatory and reimbursement issues into the equation means additional hurdles for advanced therapies in Europe. The investments that are being made in advanced therapies today may not pay off for another ten years – it is thus key that investors can count on regulatory stability and reimbursement that is both consistent and cohesive. This presents a major gap in the current European environment, the panelists agreed.

“Are there better, cleverer, or shorter ways to get these new products approved?” – Eduardo Bravo, CEO TiGenix, on the lengthy and complex approval and reimbursement process faced by advanced therapies

“Are there better, cleverer, or shorter ways to get these new products approved?” asked Bravo. The TiGenix CEO pointed to his own experiences with TiGenix’s ChondroCelect, a product that was approved in 2009 – an approval that was technically allowing for launch in 27 European countries. However, approval does not equate to reimbursement – and approval for reimbursement was significantly varied between countries, Bravo noted.

The complexity doesn’t stop there. In Spain, Bravo’s homeland, there are 17 different reimbursement bodies alone – now multiply that for each European country. “What’s the point in having a drug approved if nobody is going to pay for it?” Bravo asked – a question that his fellow panelist Andrea Chiesi, Director R&D Portfolio Management, Chiesi Farmaceutici, echoed as he described his company’s own experiences in the advanced therapies realm.

Greater Cohesion and Clarity Across Europe

Speaking of his experience with Chiesi Farmaceutici’s Holoclar, Mr. Chiesi gave another compelling example of an additional unique obstacle advanced therapies face in Europe – hospital exemption. On one hand, Holoclar is a landmark for European progressiveness: The European Medicines Agency (EMA) was first to grant approval to this stem cell-based therapy Holoclar, an autologous treatment for Limbal Stem Cell Deficiency caused by chemical or physical burns to the eye.

“Unless we have more clarity and coherence in Europe, the next wave of innovation may not see the light.” – Andrea Chiesi, Director R&D Portfolio Management, Chiesi Farmaceutici

On the other hand, the case of Holoclar highlights the problematic hospital exemption issue in Europe. The Hospital Exemption Scheme was incorporated into advanced therapies regulation, under which cell and tissue products are prepared on a non-routine basis for treating individual patients. Ultimately, the scheme allows earlier access to experimental treatments.

However, some hospitals use the exemption scheme routinely – a problem for companies like Chiesi Farmaceutici who must go down the formal approval routes for their therapies, especially for orphan diseases. It becomes difficult in such cases for a company to get to the market while a hospital is administering apparently similar treatments that are unproven, under the exemption scheme, Chiesi noted, admitting: “If we come back to the venture capital speech and the question What is the probability that we will develop additional products? I do have to ask myself – Why did you do it? But if you ever meet such a patient – i.e. a “butterfly child” you understand why you do it – it’s a disastrous disease,” he ended, referring to Chiesi Faramceutici’s work in the gene therapy treatment of epidermolysis bullosa or “butterfly children disease”.
“Unless we have more clarity and coherence in Europe, the next wave of innovation may not see the light,” said Chiesi. This was a view widely shared by panelists and the discussions on how to attain greater coherence and clarity made it clear that a collaborative effort across the EU would be needed if success is to be achieved.

Evolving Synergies, Collaboration and Attitudes

Panelists and audience members alike called for a need for greater synergy and collaboration – including diverse input from big pharma to smaller biotechs, to venture capitalists and regulators. Only with this type of behaviour of working together can we find true progress, it was agreed. In a fragmented Europe, however, this can be easier said than done. One audience member noted that fragmentation in Europe has been an issue discussed for many years – and one that remains to be solved.

Moderator Faraz Kermani of EFPIA, the European Federation of Pharmaceutical Industries and Associations, asked if the Innovative Medicines Initiative (IMI) might be viewed with optimism. Is this a model worth championing and mimicking further? The public-private partnership brings together diverse companies and research institutes to work on – projects under the auspices of the EU’s Horizion2020 programme.

While IMI and the second Innovative Medicines Initiative, IMI2, do represent an impressive model of collaboration (and collaborative funding), it was made clear that collaboration is about more than money. Behaviours and attitudes need to change if collaborative efforts are to succeed.

“One of the biggest challenges is cultural change,” noted Couto. Van Deventer agreed, also pointing out the fact that patients are becoming better informed and more vocal about the access to new medicines they may or may not have. Others in the room questioned whether EU culture is, as a whole, too risk-averse, especially in comparison to other markets, i.e. the US.

“What we need is smart money.” – Daniela Couto, Founding CEO, Cell2B, on the need for well-informed monetary investments in the advanced therapies field

Changes in financing and funding models, as highlighted by Van Deventer and others, would be a good step forward. But this isn’t enough – attitudes need to change too. “What we need is smart money,” Couto stated, making it clear that increased collaboration and funding in the EU will require an evolution of thought if advanced therapies are to achieve their full potential in Europe. Monetary investments need to be well-informed and “smart money” will take us further than uninformed investment.

Conclusion

The hope for pharma lies in what is new. New things drive the market forward and new medicines are what patients need. To secure potential benefits of advanced therapies, there must be support for clinical translation, and a regulatory and reimbursement framework that will allow these products to move on from the one-off, hospital-scale model, to become a commercial, European-scale sector.

The diversity of hurdles that advanced therapies face requires input from many different parties, as the panelists emphasised. The Report they were there to discuss was created in consultation with stakeholders from a broad spectrum, ranging from venture capitalists to regulators to biopharmaceutical experts.

Europe has the foundations in place to progress advanced therapy use. That much is clear. “The time for advanced therapies is now,” said Bravo. There was no disagreement among the panelists that this was true. The question, now, is how to ensure that Europe meets that potential. This is what the 15 April panel in Lyon sought to explore – and what the EBE – Vital Transformation Report examines in greater detail.

Download the full report here.

About the Report

The EBE – Vital Transformation report “Europe’s Emerging Science: Putting Advanced Therapies into Practice to Deliver Better Health” provides a guide to Advanced Therapies, seeks expert views on how to drive adoption, and considers obstacles in scaling-up manufacturing, meeting the requirements of regulators and securing reimbursement.

Prepared by the Advanced Therapies and Emerging Science Working Group of the European Biopharmaceutical Enterprises, in collaboration with Vital Transformation, the report aims to further the collaborative discussion on emerging sciences and advanced therapies – with the ultimate outcome of delivering better care for patients.

Download the full report here.

This report was produced in collaboration with the European Biopharmaceutical Enterprises (EBE).