Launching a new orphan drug in Europe can be a daunting prospect. The 27 nations which make up the EU bring 24 different languages, and almost as many diverse cultures, healthcare systems, reimbursement approaches, and regulatory landscapes.

For any biotech company looking to gain a foothold in the European market, an understanding of these differences, particularly of the European rare disease medicine regulatory system, is essential.

First, gaining orphan designation for a new medication from the European Medicines Agency (EMA) is a necessary regulatory strategy. The EMA is a decentralized agency of the European Union (EU), responsible for the scientific evaluation, supervision, and safety monitoring of medicines in the EU and the countries of the European Economic Area (EEA). While the EMA supports this community of around 450 million people, it doesn’t serve as a single access route to the entire European market.

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European regulatory pathway: EMA approval

The EMA is responsible for approving medications for use in the region. A pan-European centralized procedure approval (the process used by the EMA) results in a centrally authorized product with a single marketing authorization.

Crucially, however, while the EMA can approve medication for use, each country is free to develop its own guidelines, regulations, and legislation in certain special instances. For example, 18 countries have developed national guidance around early access, or compassionate use programs (CUP). In effect, this means there are many individual markets in Europe, each with its own unique regulatory landscape to navigate.

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What happens after EMA approval?

Once approval from the EMA has been received, pricing and reimbursement negotiation with each member state can begin. With so many different regulations to navigate, early engagement with local experts to develop a solid regulatory strategy can be key. In some countries, engaging with a single partner may be possible, while in others, multiple smaller players could be needed to ensure nationwide coverage. Taking advice at an early stage from organizations that understand the nuances of local language, culture, licensing, and regulation can help ensure the success of each local regulatory submission.

In many cases,  expanded access programs or compassionate use programs can be an attractive option for gathering  real-world data and building relationships with clinicians, which can be extremely helpful in supporting a local regulatory submission.

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Our regulatory expertise

Sciensus has a 30-year proven track record in collaborating with US biotech companies to overcome regulatory hurdles and expand access to rare and orphan medications in Europe and beyond.

We help our partners to navigate the complex European regulatory landscape through tailored solutions and strategic guidance. Our team offers extensive regulatory, marketing, and commercial strategy expertise which we employ to develop the best regulatory pathway for our partners, reducing the risk of failure and getting their products to market faster.

We are also the only specialist provider in the industry who can carry out all activities in the rare and ultra-rare disease space. We can design and deliver expanded access and patient support programs ahead of a licensing application and guide manufacturers through to full commercialization and a bespoke supply chain throughout Europe.

Our drug development and regulatory services

Our expert team offers a wide range of services to support drug development, regulatory strategy, and submissions:

Product registration
  • Blue-box definition per country
  • Individual country registration – notification of intent to market
  • Submission of Marketing Authorization Application and deployment of MAH Responsibilities
Product delivery
  • SIBV to develop or plug into MAH’s existing systems for Med info, AE & PQC management
  • Support for overall product labelling
  • QP release
  • EU QP-PV
  • cal Contact Person for PV (LCPPV)
  • Exploitant requirement in France
  • MIA licence to import into EU
  • WDA for UK and NL for EU
Management of Promotion materials
  • Development of Copy with MAH
  • Pre-execution notification/approval with NCA
  • Interaction with NCA on behalf of MAH

Why choose Sciensus?

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Industry leading experience

We have a 30-year proven track record in global market expansion, and extensive experience in the rare and orphan disease market.

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Bespoken end-to-end solution

We offer a complete solution across the drug lifecycle, from early access all the way to full commercialization, including patient support programs.

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Plug and play model to fit your needs

We tailor our services to meet your specific requirements, ensuring a client-centric approach.

Request a meeting with our team!

To discuss your challenges and create a strategy for accessing Europe tailored to your business needs, please complete the form below. Our team will contact you to schedule a meeting and provide a complimentary consultation.

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