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Tamsyn Frost

Managing Director of IDEA Regulatory

Should the USA always be your first regulatory target? The myths surrounding EMA vs. FDA for orphan drug designation applications 


There are approximately 7,000 different rare diseases, and while individually their prevalence is low, collectively they affect between 6-8 % of the EU and US population. This has made the development of orphan medicinal products (OMPs) of vital importance to public health. Many incentives have been introduced by the European Medicines Agency (EMA) and the US Food and Drug Administration (FDA) to incentivise the biopharma industry to meet these unmet needs, under the guise of Orphan Drug Designation.

Securing Orphan Drug status for a product is a key hurdle in any regulatory strategy. For companies based in the USA, it may seem natural to first seek FDA approval before sequentially targeting other markets such as the European Union (EU). However, this assumption could be a missed opportunity for your product.

In this article, we challenge some of the preconceptions commonly surrounding this “USA-first” approach for Orphan Drug Designation. 

Myth 1: the USA requires less information than the EU to decide on Orphan Designation

The vast majority of required information is similar to both the FDA and EMA, forming the basis for the parallel application process to both bodies. However, there are some key differences – particularly relating to definitions – that can jeopardise submissions if not clearly understood. 

 The key is to prepare the dossier using definitions and language broad enough to satisfy both boards.

For example, the definition of “unmet need”, and the methods of calculating and establishing disease rarity (prevalence vs. incidence), are different between the FDA and the EMA. This means it is possible to submit the same information to both bodies, but have one reject the product due to it not meeting geography specific benchmarks. The key here is to prepare the dossier using definitions and language broad enough to satisfy both boards. For both processes, it is important to ask questions and seek advice from regulatory committee members early on, and throughout the process, to minimise wasted time and effort.

Even if a decision is made to not enter the EU early in development (perhaps to avoid the complications of EU clinical trials procedures), it is possible to minimise the submission of information to the EMA by using the joint application process.  For example, it is possible to have the drug evaluated simultaneously by both boards at the pre-human trial stage, but then continue trials solely in the USA, submitting only an annual update report to the EMA (and FDA) as the product lifecycle progresses.  Such an approach ensures that the EMA are kept abreast of the progress of development and have agreed a clinical development plan, making it much easier to enter the EU market when you are ready to do so.

Myth 2: the USA is more open to discussion, advice and question answering than the EU

Both FDA and EMA processes pivot on building relationships with advisors and rapporteurs. Such relationships mean advisors will be more amenable to ad-hoc communication if time has been invested in building a solid reputation of collaboration, adherence and understanding. And the most effective way to establish such a reputation is to become known for writing good submissions, and for open and frequent communication with the agencies. 

A good way to foster these relationships in the EU is to engage first with a country level scientific board who, will have members who sit on the COMP at the EMA. Country level boards are often more accessible than approaching the centralised EMA board in the first instance. These boards encourage informal communication, questions and advice seeking, with the added benefit of ensuring early familiarity with your product before it reaches the formal EMA process as previously discussed.

In conclusion, the key to a successful and efficient regulatory strategy is avoiding inaccurate preconceptions. Ideally submissions would be written by (or under the advice of) someone with good market specific knowledge of processes and requirements; an ability to use language that is appropriate and understood correctly by the board in question and proactive fostering of relationships with regulatory board members for advice and mutual understanding. These factors, plus an understanding of the nuances of regulatory strategy design for each member state, will maximise the use of available incentives, and ensure a fit-for-purpose submission without unnecessary wasted time, effort, or cost.

The key to a successful and efficient regulatory strategy is avoiding inaccurate preconceptions. Ideally submissions would be written by (or under the advice of) someone with good market specific knowledge of processes and requirements; an ability to use language that is appropriate and understood correctly by the board in question and proactive fostering of relationships with regulatory board members for advice and mutual understanding. These factors, plus an understanding of the nuances of regulatory strategy design for each member state, will maximise the use of available incentives, and ensure a fit-for-purpose submission without unnecessary wasted time, effort, or cost.

Interested in reading about more myths that we dispel? Learn more here.

References: http://www.ema.europa.eu   | https://www.fda.gov

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