Breaking into Europe – challenges and strategies

The Sciensus team recently held a speaking session on Breaking into Europe at the World Orphan Drug Congress (WODC) in Boston

Speakers Mathieu Loiseau, Director, Rare Clinical Services; Andrew Cummins, Vice President, Business Development; Alastair MacDonald, Vice President, Medical Affairs; and Ray Huml, Vice President, Rare Disease Strategy; were joined by guests Kathy Senecal, a rare disease patient and the FSHD Society (facioscapulohumeral muscular dystrophy) New England Chapter director; and Grant Castor, Senior VP, Commercial Strategy & Operations at Sentynl Therapeutics.

Expanding into Europe comes with unique challenges – diverse regulations, cultural nuances, and high risks. The session was designed to look at the challenges of launching new rare disease drugs into Europe, possible strategies, and the importance of local collaboration and the patient voice.

Key takeaways

US biotechs launching into Europe face a diversity of languages, cultures and regulations that may vary by individual European Union member state, as well as differences in countries that are not members of the European Union. This leads to complexity in market access, the level of local expertise needed and tricky navigation around all the different healthcare requirements.

For example, these differences may mean at a practical level that companies need to develop differing packaging configurations, patient information and labelling as well as appropriate pricing. The time it takes to address these issues shouldn’t be underestimated, underlining the importance of calling on local expertise. Local partners can help with the challenge of managing the supply chain and distribution to make sure all the necessary quality, safety and regulatory processes are in place.

Companies looking to launch into Europe need to first consider, from a strategic point of view, what the ambition is. For example, a new company looking to launch its first product into Europe should consider the long-term future and whether it is planning more products to come. If so, it may make sense to develop its own European footprint. If there are no firm plans for additional products the cost of launch may be prohibitive. Every launch will come at a high level of time and money, particularly if you want to cover multiple countries in Europe.

Another option could be to out license, however this will mean giving away a high level of control. Out licensing would mean getting some money upfront and yearly royalties, but can you be sure how much you are trading away when you don’t yet understand what you can achieve in Europe? The third option is to partner with a service provider that regularly launches new products for biotechs in Europe and to benefit from their experience.

An important factor for success is to be very patient centric and find an organisation that can help you navigate the complexity of the European landscape. Ideally, biotechs should partner with an organisation that can engage with patient groups and advocates, which can be quite challenging in Europe. Often these are very small groups with members who don’t speak English.

US biotechs face several key challenges in Europe

US biotechs launching into Europe face a diversity of languages, cultures and regulations that may vary by individual European Union member state, as well as differences in countries that are not members of the European Union. This leads to complexity in market access, the level of local expertise needed and tricky navigation around all the different healthcare requirements.

For example, these differences may mean at a practical level that companies need to develop differing packaging configurations, patient information and labelling as well as appropriate pricing. The time it takes to address these issues shouldn’t be underestimated, underlining the importance of calling on local expertise. Local partners can help with the challenge of managing the supply chain and distribution to make sure all the necessary quality, safety and regulatory processes are in place.

Strategies to navigate European market complexities

Companies looking to launch into Europe need to first consider, from a strategic point of view, what the ambition is. For example, a new company looking to launch its first product into Europe should consider the long-term future and whether it is planning more products to come. If so, it may make sense to develop its own European footprint. If there are no firm plans for additional products the cost of launch may be prohibitive. Every launch will come at a high level of time and money, particularly if you want to cover multiple countries in Europe.

Another option could be to out license, however this will mean giving away a high level of control. Out licensing would mean getting some money upfront and yearly royalties, but can you be sure how much you are trading away when you don’t yet understand what you can achieve in Europe? The third option is to partner with a service provider that regularly launches new products for biotechs in Europe and to benefit from their experience.

How collaboration drives successful drug launches

An important factor for success is to be very patient centric and find an organisation that can help you navigate the complexity of the European landscape. Ideally, biotechs should partner with an organisation that can engage with patient groups and advocates, which can be quite challenging in Europe. Often these are very small groups with members who don’t speak English.

Companies can also develop patient support programmes (PSPs) where nurses visit patients at home or when digitally enabled solutions help people adhere to their treatment. These can be used to build communities and help people access new drugs. Local partners supporting these programmes should also have a good understanding of the local healthcare landscape and how to navigate it.

Local collaborations help biotechs to understand what’s possible, what’s not possible, how patients can get the best out of a product and ensure that the patient voice is integrated into drug launches.

PSPs are important not only in terms of the patients but to your company, as they have been demonstrated to increase adherence and persistency to the tune of 10-20%.
Alastair MacDonald Vice President, Medical Affairs

How biotech companies can help overcome the obstacles patients face in accessing trials

Kathy Senecal explained that one of the challenges in coming into a trial is simply understanding the design of that trial – for example, the physical location, the length of a trial, how long each visit to a clinic will take, and the issue of finances for individuals taking part.

“It’s very complex. We’re out here living our lives as best we can. We have a lot of hope that there will be a treatment out there for rare disease. We want to participate, but sometimes the general consensus is that it’s just difficult. We need to reach you, and you need to reach us.” Kathy Senecal – Rare disease patient and the FSHD Society (facioscapulohumeral muscular dystrophy) New England Chapter director.

Kathy added that medical professionals dealing with rare diseases and hospitals that probably could run a clinical trial should be trying to get in touch with patients and advocacy groups, for example by attending conferences or holding focus groups to discuss opportunities for participation.

“When you look at the pipeline design of drug development, you don’t really see advocacy groups in there very much, but we’re really important in that chain from going from nothing to a treatment programme.”

Importance of real-world data

In rare disease, there is often a low body of data because there are so few patients, which can affect the development of protocols and exclusion criteria, for example. Biotechs should start collecting real-world data (RWD) as early as possible to meet the demands of the stakeholders – the regulator, the payer, healthcare professionals and patients, for a successful launch.

For example, what evidence do you have about the actual burden of this disease? What are the key components around the quality of life of patients, the burden on the health systems and insurers for this disease?

It’s crucial to bridge those evidence gaps with real world evidence. The knowledge generated can also support future clinical trial design as biotechs move through the later phases. Collecting RWD also supports take-up by healthcare professionals and early patient adoption.

For a successful launch into Europe, biotechs need to engage early with the EU and national regulators and consider linking with a local partner

A European launch is a high-cost exercise, so economics are important – is a product economically feasible and will reimbursement come at a level that sustains the operational expense? By engaging early, you will have a better understanding of the options open to you.

In addition, consider working locally to develop patient support programmes. With the price point for rare disease drugs, a patient support programme is a small investment to get quite a large return. A 10 to 20% uplift in persistence and adherence makes a big difference on return on investment, making them important both clinically and when it comes to economics of your launch.

“Europe is a great opportunity. If you approach it in a non traditional way you can set up your company for success.” Grant Castor – Senior VP, Commercial Strategy & Operations at Sentynl Therapeutics

About Sciensus

Sciensus is a proven life sciences solutions partner with more than 30 years’ experience in navigating and unlocking the complex European healthcare ecosystem to maximise patient reach. We help accelerate the development journey, bringing medicines into the market and directly to patients to improve health outcomes. Through cutting-edge patient engagement programmes, offering an exclusive combination of skilled in-person care and digital support, we help patients make the most of their treatment and generate real-world evidence that can be used to improve treatment pathways.

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