Published 13th July 2023
The European Commission (EC) has proposed a draft Directive and Regulation on the 26th of April 2023 for the reform of the pharmaceutical legislation. A Regulatory Sandbox is an initiative to support innovative developments where there are challenges within the scope of regulations.
A specific objective of the proposed pharmaceutical legislation is to offer an attractive, innovation and competitiveness friendly environment for research, development, and production of medicines in Europe. To meet this objective, the EC is looking for the right balance to support innovation. Medicinal products with science and technology developments and digitalisation will need suitable approval processes to keep up with global competition. Medicinal products will also need to consider unmet medical needs, measures on access, affordability, and the impact to the environment, to better address timely patient access to medicinal products in all Member States.
The Regulatory Sandbox may be proposed by the EMA as a process to enable marketing authorisation of medicinal product that are innovative. The outcome of the Regulatory Sandbox process may support Adapted Frameworks for marketing authorisation.
The Regulatory Sandbox is one of many measures that will contribute to ensuring the proposed legislation will be able to deal with emerging developments in science. Other measures include existing procedures such as scientific advice, enhanced scientific and regulatory support for priority medicinal products (PRIME), adapted clinical trials, use of real-world evidence, secondary use of health data, and additional incentives.
A ‘Regulatory Sandbox’ is defined in the proposed Regulation, Chapter 1, Article 2(12), to mean a regulatory framework during which it is possible to develop, validate and test in a controlled environment innovative or adapted regulatory solutions that facilitate the development and authorisation of innovative products which are likely to fall in the scope of this Regulation, pursuant to a specific plan and for a limited time under regulatory supervision.
Based on the proposed Regulation, Chapter IX, Articles 113 to 115, the EMA will make a recommendation for a medicinal product to the European Commission (EC), that is based on a detailed plan. The EMA is acting as the health agency, working in collaboration with agencies in Member States. To qualify, the medicinal product must meet eligibility criteria and conditions.
a. A medicinal product (or category of products) in development cannot comply with the requirements for authorisation due to scientific or regulatory challenges arising from characteristics or methods related to the product, and
b. The characteristics or methods related to the product will positively and distinctively contribute to the quality, safety, or efficacy of the product, or provide a major advantage contributing to patient access to treatment, and
In addition, the medicinal product must be in early development program, and will not be recommended by the EMA if already in advanced phases of development.
The detailed plan will set out a regulatory framework for medicinal product development, including scientific requirements necessary clinical trials for obtaining authorisation. The plan will explain the regulations that cannot be complied with, and a proposal for alternative or mitigation measures, setting out a timeline.
The Regulatory Sandbox creates the controlled environment to validate and test the innovative or adapted regulatory solutions. The environment is to ensure a high level of protection and legal certainty for those involved, having a level playing field and fair competition. The EMA supervises the Regulatory Sandbox and will notify any risks to health and/or the environment to the EC. If the justification supporting a Regulatory Sandbox no longer applies and/or to protect public health, the EC may stop the program.
Medicinal products developed as part of the Regulatory Sandbox will be contingent with the proposed Regulation, and the SmPC will indicate that the medicinal product has been developed as part of a Regulatory Sandbox.
Following on from the proposed Regulation, additional information will be need provided by the EC, on the eligibility criteria and the procedure for the application, selection, participation and exiting from the Regulatory Sandbox, and the rights and obligations of the participants. The EC does not provide full details in the proposal for reform of the pharmaceutical legislation.
As stated above, when a medicinal product in development cannot comply with the requirements for authorisation, the dossier requirements needed for authorisation may be adapted, based on proposed Directive (Section 5, Article 28).
The eligibility and criteria for the adapted dosser requirements and the Regulatory Sandbox (described above) are the same. Medicinal products that qualify for adapted dossiers may submit a marketing application in accordance with proposed Directive, Article 6 (2), General requirements for marketing authorisation applications).
The proposed Directive, Annex VI, Areas for Adapted Frameworks referred to in Section 5, Article 28, is a list of medicinal products that have specific detailed rules leading to adapted dossiers and adapted regulatory frameworks for authorisation. Products are placed on this list by the EC, who will take into consideration the outcomes of the Regulatory Sandbox and any available information from the process.
At present, Annex VI lists one area of medicinal products. These are ‘Phage-containing medicinal products, in cases where the medicinal product has a variable composition depending on the specific clinical context.’
Your company may be developing an innovative medicinal product. The proposed pharmaceutical legislation objective is to make sure all patients across the EU have timely and equitable access to safe, effective, and affordable medicines. Also, to offer an attractive, innovation-and competitiveness friendly environment for research, development, and production of medicines in Europe.
As part of this, Regulatory Sandboxes provide the opportunity for advancing approval of innovative medicinal products by gaining information, at an early stage of development, to find solutions to challenges within the scope of regulations.
The Regulatory Sandbox is just one of many measures, when a medicinal product cannot comply with the requirements for authorisation. A forward-thinking consultancy, DLRC understands individual company’s unique requirements for medicinal products in development and finds creative and pragmatic solutions for all regulatory challenges.
To discuss your product development, how the new pharmaceutical legislation may affect you and your regulatory strategy, contact our experts via the link below.