Industry Insight

With a region as large and diverse as Europe, Mike Ryan and Paolo Correale at EVERSANA explain how pharma companies can best engage with the myriad of expectations and regulations associated with launching in the EU

Bringing a new drug therapy to market is challenging anywhere. But it's even more complex in Europe, and especially across the EU with its 27 member states and nearly 450 million residents. This complexity is further compounded when crucial markets such as the UK and Switzerland are added to the mix.

Launching here often presents greater complexity compared to markets like the US due to many variables, especially the pricing and reimbursement processes. Yet pharmaceutical and life sciences brands have a responsibility to the millions of patients across the region and the world who could benefit from new therapies to overcome common hurdles.

Let’s explore a few common challenges along with what a constantly evolving geopolitical landscape could mean to the future of approvals in the regions.

The first thing that must be considered is how the reimbursement landscape looks in Europe. It’s vastly different when compared to the process in the US, and fragmented due to the diversity and complexity of multiple countries so close in proximity. This causes, in many instances, longer timelines for launches, which delays the time frame for how long it will take for a drug to reach patients – many of whom will have participated in clinical trials to prove the drug's safety and efficacy.

In most cases, each country conducts its own health technology assessments (HTAs) and price negotiations for each product. With all the countries in Europe, one can infer just how long that process could take. These HTAs control access to each country’s national health system, which treats the majority of patients in that country. This is much different from the process in the US.

Therapies typically reach the market faster due to this centralised approval process because, once a therapy earns US Food and Drug Administration (FDA) approval, commercial availability normally follows driven in large part by negotiations with private payer insurance companies. This streamlined process allows for faster patient access to treatments.

In Europe, it is different. After the European Medicines Agency (EMA) approves a new therapy, each country conducts individual HTAs based on its own laws and regulations. Price negotiation also occurs on an individual country basis.

Germany, as a general rule of thumb, has the fastest HTA pathways – normally between one to three months between EMA market access and launch. However, in other countries like Italy and Spain, this same approval process could take on average 20 to 26 months.1 As a result, a family living in Berlin could access a therapy more than a year in advance of family or friends living in Barcelona, simply because of the disparity in how each country manages approvals.

Another variable that can impact approval is how some countries may make additional investments to ensure new therapies come to market faster via paid early access programmes because they can, whereas smaller nations may not have the same resources. Wealthier nations like Switzerland and France often expedite the reimbursement process for innovative treatments, ensuring quicker access for their populations. In contrast, countries in Eastern and Southern Europe, such as Poland and Hungary, can face significant delays that can stretch to several years. These delays are primarily due to budget constraints and less robust healthcare infrastructures, which hinder their ability to swiftly integrate new therapies into their healthcare systems.

Understanding reimbursement considerations and potential hurdles across each country is critical to a product launch success, but health inequity due to country processes isn’t the only challenge companies face.

When it comes to treatment availability for rare and orphan diseases, Europe also faces significant challenges. While the US sees about 90% of rare disease drugs reaching the market, only around 64% become available in Europe.1 But why is this? Don’t patients with rare conditions deserve the same access to proven therapies like a colleague or friend, for example, in the US? European families and patients absolutely do deserve the same access, but this too is an economic challenge facing the region.

Because rare and orphan therapies cost more to develop and produce due to the limited number of patients they may serve, the cost is high. Many EU countries prioritise cost-effectiveness when buying and negotiating with pharma companies for product availability, delaying or even preventing the introduction of new treatments, as manufacturers struggle to justify the soaring prices needed to recoup their research and development investments. But it doesn’t have to be this way, nor should it.

The need for better access has spurred policymakers across the region to challenge the status quo and begin rethinking new ways of bringing hope to patients. Five newer concepts or programmes continue to emerge that are providing hope to patients and new options for brands to consider as they plan future launches.

The first concept that truly could be game-changing is the new HTA framework in the EU, which went live in January 2025. It represents a significant shift from the previous decentralised model. The proposed framework will introduce joint clinical assessments (JCAs), which will be conducted jointly for all EU member states, focusing on the clinical effectiveness and safety of new health technologies. This approach is designed to reduce duplication of efforts and ensure consistency in the evaluation process.

With the new processes being streamlined, it is expected that the timeline for new treatments to reach patients will be expedited. The harmonisation of these processes is also expected to promote more equitable access to innovative health technologies throughout Europe, helping to overcome the pricing discrepancy by reducing the need for multiple, separate assessments, particularly for smaller countries. It will be a massive step towards a more equal playing field in the years ahead. Currently, this will not apply in the UK and Switzerland – although the possibility for mutual recognition may emerge in the future.

A second newer concept for the region is risk-sharing agreements (RSAs), which are arrangements between pharma companies and healthcare payers designed to manage the financial and clinical uncertainties associated with new drugs. This is particularly important for new and innovative treatments where long-term data may be limited. RSAs aim to balance the need for patient access to innovative treatments with the financial constraints of healthcare systems. By adopting performance-based contracts, manufacturers can make therapies more accessible to patients.

For example, if a drug fails to meet certain performance criteria, manufacturers could offer rebates or refunds to healthcare systems.

RSAs have already begun being implemented across the EU and have shown great benefits. Italy has been a pioneer behind the concept, particularly performance-based agreements, to manage the introduction of high-cost oncology drugs. The NHS in the UK also used RSAs to negotiate prices and manage the budget impact of new treatments, increasing patient access.

It's a growing model that has the potential to benefit everyone in the healthcare ecosystem – but applies mostly to cell and gene therapies in particular.

A third growing focus across EU countries is improving data integration in healthcare systems. This is difficult due to the unique systems held by each country, but if it can be figured out, it would involve several key advancements aimed at enhancing the efficiency, accessibility and quality of healthcare delivery. Collecting real-world evidence and harmonising data across EU nations could help provide a lot of answers to common questions on how to make healthcare better.

The European Health Data Space (EHDS) is a newer regulation designed to facilitate the exchange and access of health data across member states. It aims to create a unified framework for health data sharing, making it easier for patients, healthcare providers and researchers to access and utilise health information. It’s something desperately needed. Standardising data formats and protocols across the EU would be a huge step. Ensuring that electronic health records (EHRs), medical devices and other health IT systems can communicate seamlessly will help expedite this process. Yet to do so, the importance of security and privacy at every step of the road must be considered.

The EHDS framework includes robust measures to protect patient data, ensuring that it is shared securely and only with authorised parties. This, in turn, will hopefully build trust among patients and healthcare providers.

More needs to be done to bring more rare disease therapies to patients in the EU. Currently, the EU offers ten years of market exclusivity for orphan drugs, with the option to extend exclusivity by two years for paediatrics indications. If the union could expand this exclusivity period, it could be one more incentive for companies to bring their therapies to Europe faster.

In the US, a drug tax credit exists, providing tax benefits to drug companies conducting clinical trials for new therapies aimed at treating rare diseases. If a similar option became available across EU nations, it could help to reduce the financial burden on companies developing these treatments. While some already exist for small and medium enterprises and for certain services, expanding these reductions and waiving fees for all orphan drug applications could be life-changing for the industry, the region and patients. Currently, European governments are also trying to develop new early access programmes or improve existing ones, like those in Italy and Spain, to provide faster access to orphan drugs. These will be revenue-generating allowing early in-market income compared with standard pathways.

The final consideration for EU nations to reduce inequality across the region is to modify international reference pricing (IRP) rules. The current system is designed to control drug prices by comparing them to prices in other countries. Countries across Europe often use IRPs to set drug prices by referencing the prices of the same drug in a basket of other countries. But as noted earlier, not every country has the same financial means, creating natural inequalities.

By modifying IRP rules, a greater effort could be made to ensure that lower-income countries do not unduly depress prices elsewhere, encouraging manufacturers to launch in all EU nations. The opportunity here is further enhanced by countries offering confidential pricing agreements. This in turn will reduce the reference pricing risk thereby reducing downward pricing pressure because of reimbursement in those countries.

These five considerations are not easy. Some of them have been decades in the making and continue to take time to work towards. But if done right, the impact on the region, companies and patients, could be life-changing.

Finally, as the calendar has turned to 2025, a new administration could make significant shifts in the US pharma policy, especially related to pricing. It is likely too soon to know exactly what may happen. But any changes that happen in the US will likely have implications across the EU.

The pharma industry must stay connected and pool resources where possible to make access better and faster. The technology and resources are available, and the need to improve market access across Europe is massive. The 450 million patients across the EU are looking to industry leaders to make access better. They deserve the same treatment options as anyone else in the world, and it is the industry’s job to make it happen.

References
1. Visit: data.navlin.com