Developers looking to bring their products to the European Union are often unaware of the different pathways open to them to get regulatory support. The European Medicines Agency (EMA) offers several initiatives to support innovators. However, there is some nuance to understanding which of these might be right for a developer, and at what stage of the process.
The EMA’s PRIME scheme and orphan designation are both reasonably well known and sought-after initiatives. However, before applying for these schemes, EMA offers several pathways to advice. Knowing which to leverage, when and for what purpose will help innovators make the most of the agency’s initiatives.
The SME Office
The entry point for small innovators is the small and medium-sized enterprises (SMEs) status. In 2005, the European Parliament introduced provisions designed to promote development of innovative new medicinal products by SMEs. For any company that fits this designation, the SME Office is an invaluable early resource. Briefing meetings are tailored to provide regulatory and administrative support to SMEs.
The SME Office does not provide in-depth scientific and regulatory support, but is a good way for developers to understand regulatory expectations and to be pointed in the right direction as to which experts they should be consulting with and which pathway or procedure might be best suited to their development programme.
For example, the SME Office would be a good source to ask whether ITF might be a suitable avenue if a developer wasn’t quite sure whether they would be eligible or whether ITF would be useful for them.
The SME might be best thought of as a help desk to ask high-level regulatory questions, get insights on fee payments, and other issues that developers often struggle with. For example, the office can provide advice on which discounts a developer might be eligible for, such as translational or procedural discounts.
We have worked with the SME Office on behalf of clients to get advice on when to apply for programmes such as PRIME or the orphan designation and to build a general regulatory plan and overall strategy.
Again, the SME Office is a good port of call early on in the development and planning process.
The Innovation Task Force (ITF)
The ITF was introduced to provide developers of innovative therapies with an early engagement forum. The ITF offers briefing meetings to help developers determine the next steps in their development programmes.
Meetings can cover regulatory, technical and scientific concerns and are an informal exchange with the agency. The forum is open to any developer of innovative products and are an opportunity for companies to discuss issues related to emerging technologies where there is no or very limited regulatory guidance.
Regulators will set up a meeting with developers after having read their briefing document. They will bring in a panel of experts with knowledge of the developer’s emerging technology to discuss questions posed by the developer.
The ITF discussion is more conversational and less binding than scientific advice. It is also free and is a great way for developers to connect early on with the regulators to make them aware of their programme and to work through any ambiguity with the regulatory guidelines.
For example, one of our clients was developing a gene editing therapy for a rare genetic disorder and turned to the ITF to better understand the regulatory pathways as there was no clear precedent for their technology.
The ITF is similar to initiatives in other markets, including:
- The US Food and Drug Administration’s (FDA’s) INTERACT (INitial Targeted Engagement for Regulatory Advice) meetings
- The Innovative Licensing and Access Pathway (ILAP) pathway offered by the UK’s Medicines and Healthcare products Regulatory Agency (MHRA).
The Quality Innovation Group
Manufacturing processes often create headaches for developers of innovative products and the Quality Innovation Group was established to address these types of questions.
The group comprises operational experts in the field from within EMA. Developers can set up meetings with the group if they have questions or concerns specifically related to the design, manufacture and quality control of medicines.
Among the examples shared in the group’s webpage are:
- Decentralised manufacturing
- Process models
- Platform technologies
- Digitalisation and automation of manufacturing and control
The group offers informal early engagement and support for companies struggling with some of the manufacturing issues that often accompany innovative technologies, such as cell and gene therapies. This type of advice can help companies navigate very complex areas where often the guidelines are not clear due to the rapidly evolving nature of the technology.
The agency recognises that developers are the experts in their own product and process, particularly when they are developing an innovative method. Often what is required is a regulatory expert to provide advice on how those processes might fit into the regulations and guidance on how to be compliant with those regulations. That is the type of support that the Quality Innovation Group can provide.
Guiding industry early on
EMA has made clear that early engagement is a priority, especially with highly innovative therapies where the guidelines continue to evolve. These three initiatives – the ITF, the SME Office and the Quality Innovation Group – seek to support and advance that objective. The SME Office can help companies to streamline their regulatory planning before engaging with the ITF and Quality Innovation Group to further their development strategy. By leveraging these resources, developers can navigate the regulatory landscape more effectively and find the best path for their development programme in the EU.
The EMA offers several pathways to advice. Knowing which to leverage, when and for what purpose will help innovators make the most of the agency’s initiatives.