Regulatory Sandbox to Support Innovation in the Pharmaceutical Field

Developing groundbreaking treatments like Advanced Therapy Medicinal Products (ATMPs) is currently slow, expensive, and often fails to provide the real-world evidence needed for government approval and purchase. This paper explores regulatory sandboxes-controlled environments where innovators can test new therapies under regulator supervision with temporary exemptions from certain rules. The study concludes that sandboxes can bridge the gap between innovation and regulation, foster collaboration, and generate vital evidence early. The implications are significant: using sandboxes during Phase II or III of clinical trials could speed up patient access to life-saving drugs and reduce development costs while maintaining high safety standards.

The paper focuses on the European Union but includes comparative analysis of systems in the United States, Canada, Indonesia, and France. Its scientific discipline is law and regulation, specifically looking at how "smart regulation" can balance innovation with safety. The primary objects covered are innovative medicines, with a specific emphasis on ATMPs, biological drugs (like mRNA), and medical devices.

• Anticipatory Regulation: sandboxes are a form of "anticipatory regulation," allowing regulators to work with companies to develop rules at the same time as the products themselves.
• Addressing Trial Shortcomings: traditional clinical trials often lack real-world data and pharmacoeconomic evaluations. Sandboxes allow for the collection of this data under real-world conditions before a drug is fully marketed.
• The "Sandbox Umbrella": a potential model where a non-profit entity supervises multiple companies, encouraging knowledge sharing and reducing individual legal or financial risks.
• Strategic Timing: sandboxes are most effective when used late in the research process - specifically during Phase II or III - to ensure the project is advanced enough to justify regulatory exemptions.
• Global Harmonisation: sandboxes can help reconcile different international definitions of medicines, such as the differing classifications of mRNA therapies between the EU and the US.
• Legal Clarity: sandboxes are distinguished from other tools like sunset clauses (which focus on evaluating a law's effectiveness) and statutory experiments (which test new regulations themselves). The regulatory sandbox is a specific "niche" within statutory experiments focused on product innovation.

I. Introduction

II. Using regulatory sandboxes in the development of innovative therapies

1. Why should regulatory sandboxes be used?
2. How to use a regulatory sandbox?
3. When should the regulatory sandbox be used? Regulatory sandboxes and pilot studies

III. What are the differences between regulatory sandboxes and other legal enclaves?

     Statutory Experiments/Experimental Legislation

     Sunset clause

IV. Conclusion

Full reference of the paper: 

Blanka Bartos, “Regulatory Sandbox to Support Innovation in the Pharmaceutical Field”, European Health & Pharmaceutical Law Review (EHPL), Lexxion, Volume 8, Issue 1 (2025), pp. 28 – 42, DOI https://doi.org/10.21552/ehpl/2025/1/6

Open Access link to the paper:

EHPL - European Health & Pharmaceutical Law Review: Regulatory Sandbox to Support Innovation in the Pharmaceutical Field