Alexander Natz of the European Confederation of Pharmaceutical Entrepreneurs (EUCOPE) casts his eye over the potential effects of the upcoming EU Pharmaceutical Legislation for small- and medium-sized enterprises in Europe. Natz feels that Europe is at a crossroads in terms of its positioning as a driver of innovation and calls for inter-stakeholder collaboration to build a resilient EU pharmaceutical framework.


After numerous delays, the European Commission is expected to finally present its proposed revision of the EU Pharmaceutical Legislation on 26 April. The revision presents the opportunity to shape the biopharmaceutical ecosystem, building on the successes of the past two decades and ensure the EU is equipped to embrace innovative technologies. We continually advocate that this revised system must encourage innovation, attract further R&D and bring medicines to patients faster.

The past two decades have seen Europe lose ground to other regions – in clinical trials and the launch of new medicines – however, Europe still benefits from a high publication volume, patent activity, a growing industry pipeline, and an increase in medicine approvals. Our IP, regulatory expertise, and regulatory exclusivities are an example of where the EU maintains a competitive edge compared to other geographies.

Months back, I wrote on what’s needed to reinforce biopharmaceutical innovation in Europe and highlighted the importance of a competitive environment and the potential unintended consequences of innovation in small and mid-sized companies stemming from the proposals being explored by the European Commission.

The upcoming legislation should focus on unleashing the research potential of smaller companies and addressing underlying challenges in the system. Introducing concepts such as (High) Unmet Medical Need (HUMN) will be detrimental to the EU’s efforts to maintain and grow its global competitiveness. The EU Pharma Package places Europe at an enormous crossroads – either it chooses to fix its shortcomings and re-introduce itself as a viable choice on the global life science stage or takes a step back and follows other world regions that will drive the innovation train.


Unmet Medical Need

At the end of the day, the efforts to create a predictable and competitive EU biopharmaceutical environment are to develop medicines and therapies that will benefit patients. The concept of (High) Unmet Medical Need (HUMN) presents a pertinent issue, and a legislative definition must be avoided as it discourages innovation and hinders patient access. (H)UMN evolve with science and society, thus a definition in legislation would not stand the test of time.

Any definition of (H)UMN would be inherently narrow and would disincentivize developers from further research and innovation into disease areas. If the first direct-acting anti-viral in Hepatitis C had been considered to fully address an UMN, the development of pan-genotypic treatment regimens (acting against every genotype) would not have been developed.

In areas where therapeutic options might exist, there may still be unmet needs to be fulfilled. In the cardiovascular area, this is the case with oral factor Xa inhibitors and thrombin inhibitors anticoagulants or in asthma. As we know, no two patients are the same, and may not respond well to existing treatments.​

UMNs also evolve within the same disease. The onset and progression of a chronic disease make it challenging to define UMN, since these medical needs will change. No single, uniform, narrow definition of unmet medical needs is possible. While it is relatively easy to point to and recognize an example of (H)UMN, defining it in legislation is nearly impossible and would limit patient access and therapeutic innovation – contrary to the intentions of the revision.


The Value of Smaller Companies

Public discussions often revolve around academic developers and big pharma, but the role of small and mid-sized companies is regularly overlooked. These developers are key drivers of pharmaceutical innovation, upscaling innovative technologies, developing treatments for rare diseases, and translating concepts into breakthrough therapies. EUCOPE’s membership consists of European and global companies committed to ensuring that Europe remains an attractive location to undertake research and launch products. Smaller companies are at the forefront both of biopharmaceutical research and debates relevant to patient access, healthcare rationing, and reform.

The diversity in therapeutic technologies, disease epidemiology, and reach of marketing authorisation holders highlights that a one-size-fits-all solution would not be appropriate nor achieve the Commission’s goals of improving access. Measures within the revision that are not sufficiently tailored would be detrimental to small and mid-sized pharmaceutical companies, and make launching a product on the market extremely difficult based on resources, time-frames and 27 different P&R frameworks. A predictable framework is needed that incentivizes these dynamic developers of innovation to launch in Europe or else the continent risks falling further down the global life science pecking order.


Time to Make it Count

The European Commission now has its shot – tasked with unleashing the health, innovation and economic potential of the European biopharmaceutical industry to compete on a global scale. Reducing incentives, defining UMN and introducing conditionalities will undermine predictability – something extremely important to small and mid-sized companies to make investments – ultimately hindering the EU’s overall competitiveness and innovation within life sciences.

Let’s make it count and work together – with EU Institutions, Member States, clinicians, researchers and especially patients – to tackle the current issues and build a resilient EU pharmaceutical framework that rewards innovation and builds a healthier society.