Rare Disease Moonshot contribution to the Call for Evidence on the EU Start-up and Scale-up Strategy

Start-ups and scale-ups play a crucial role in the development of innovative medicines in high unmet needs areas, such as rare disease, paediatric medicines or personalized medicines. To overcome regulatory burden, start-ups and scales-up require having access to high quality data fit for regulatory purposes, sustainable infrastructures that facilitate data collaboration but also access to expertise such as data science, regulatory science or regulatory affairs. There is an urgent need for additional work and funding to further stimulate and support the organisation of large-scale sustainable infrastructures gathering the knowledge and facilitating data collaboration both from the research and industry communities to foster the development and validation of project outcomes.

Public Private Partnerships (PPPs) can play a crucial role in strengthening and developing infrastructures that can advance data and regulatory science facilitating structured and continuous engagement with regulatory bodies. The impact should aim at streamlining approval processes and timely access to the patients benefits.

Thus, it is now essential to support initiatives focused on strengthening or developing infrastructure in Europe in the field of regulatory science, tools, and platforms. This will help foster innovation in an accessible and sustainable way from the early stages of drug discovery, while considering the prerequisites for market authorization or market access. Indeed, reducing the risks associated with investments in areas of high unmet medical need and optimizing the use of clinical trials ensures the long-term sustainability of research initiatives and sustainably drives the development of innovative products and solutions.

By implementing these recommendations, Europe can establish a resilient and forward-thinking regulatory science ecosystem that accelerates the development and approval of treatments in high unmet needs areas, strengthens public-private collaboration, and ensures sustained innovation at an affordable price for the European citizens.

As an example, a well-designed regulatory science platform must address these gaps by providing a centralized structure that:

Implements a neutral governance framework that guarantees the independence and transparency of research outcomes.
Supports high-impact public and PPP projects with clear regulatory objectives.
Establishes a robust infrastructure to manage large-scale research collaborations aligned with Health Authorities including national and European authorities, regulators and HTA.
Ensures access to skilled regulatory professionals to oversee compliance, approvals, and project execution.
Develops regulatory-grade databases and digital resources to support evidence-based decision-making.
Secures long-term funding mechanisms to sustain the platform’s operations and maintain research continuity.

Expected outcomes

Faster approvals through improved regulatory tools and better coordination between stakeholders.
Allow development of innovative product for unmet medical need (rare disease, long term evolutive disease, fragile populations)
Greater alignment between research and regulatory standards, leading to increased efficiency in drug development.
Optimized clinical trials, incorporating real-world data and predictive modeling to improve power and shorten approval timelines.
Increased accessibility of validated regulatory methodologies and tools, and frameworks for industry-wide adoption.
Enhanced investment and global leadership, positioning Europe at the forefront of regulatory science innovation and unsolved questions and research (personalized medicine, rare disease, fragile population, long term evolutive disease).

Recommendations on regulatory science, tools, and platform for supporting start-ups and scale-ups

Start-ups and scale-ups play a crucial role in the development of innovative medicines or solutions in high unmet needs areas, such as rare disease, paediatric or elderly medicines, or anti-microbial resistance. However, it appeared clear that, beside the difficulties in developing products for small populations and partially known pathologies in long-term diseases, these types of companies and their leadership are facing an obvious gap in regulatory knowledge and know-how, hampering health innovation from reaching the market. Additionally, these companies often struggle to capitalize on existing initiatives designed to support them, despite significant investments, including public funding. Many projects operate in silos, limiting access to sustainable infrastructure and key expertise necessary for medicinal products and innovations to secure marketing authorisation and reimbursement in Europe.

A key challenge within this landscape is bridging the innovation and industrial gap. While Europe excels in scientific research, this excellence does not consistently translate into innovative therapies. Technology and knowledge transfer remain critical bottlenecks, affecting the ability of start-ups and scale-ups to transition from discovery to application. This disconnect between research and industrial implementation exacerbates the challenges of regulatory complexity and the ability of companies to navigate the approval pathways effectively. Moreover, the EU’s fragmented regulatory landscape also poses a significant challenge. Innovators face an inconsistent and unevenly implemented regulatory framework, unlike in other regions that provide greater predictability.

To overcome regulatory burdens, start-ups and scale-ups require access to data and sustainable infrastructures that enable, for example, the sharing or pooling of patient samples, downstream analysis, or the integration of data from multiple sources. Additionally, specialized expertise, such as in data science, regulatory science, or regulatory affairs, is crucial to navigating the intricate regulatory landscape. Public-private partnerships (PPPs) can play a crucial role in advancing regulatory science by facilitating structured and continuous engagement with regulatory bodies. Improved access to relevant expertise and sustainable infrastructure would help streamline approval processes and facilitate timely access to the right regulatory pathways.

To enhance the impact of PPPs on the research and innovation ecosystem, as well as on patient outcomes, health systems, and Europe’s competitiveness, there is an urgent need for additional (institutional) public-private collaborative work. This collaboration should focus on developing large-scale, sustainable infrastructures that consolidate knowledge and foster data collaboration between research and industry communities. By strengthening or creating resources—both in terms of processes and human expertise—in regulatory science, tools, and platforms, Europe can foster innovation in an accessible and sustainable way. Ensuring that these efforts integrate drug discovery stages with marketing authorisation considerations in mind will help de-risk investments in high unmet needs areas, streamline clinical trial processes, and provide long-term sustainability for research initiatives.

Case study: Foreseeable impact of developing a regulatory science platform in Europe accessible to start-ups and scale-ups

The growing complexity of medical product development, preventive measures, and personalized therapies underscores the urgent need for a dedicated regulatory science platform. While scientific innovation continues to progress, the lack of structured mechanisms to integrate these advancements into regulatory frameworks results in inefficiencies, delayed approvals, and inconsistent regulatory guidance. Key challenges include insufficient applied research in regulatory methodologies, limited knowledge of validation frameworks for new tools and methodologies, limited harmonization of qualification procedures for new tools, and fragmented stakeholder priorities that slow decision-making. Moreover, public and charitable funders often lack the expertise and resources needed to assess the regulatory impact of the research they support, making it difficult to ensure that publicly funded projects align with regulatory expectations. A dedicated regulatory science platform would address these gaps by fostering collaboration among industry, academia, and regulators, ensuring that innovations are effectively translated into practice while maintaining high scientific rigor and regulatory compliance.