During drug development pharmaceutical companies need to establish their regulatory strategy and define timelines for their clinical studies. To support small and medium-sized enterprises (SME, <250 employees and 50 million euros annual turnover), the European Medicines Agency (EMA) created the SME Office in 2005. This dedicated platform provides regulatory, financial and administrative assistance to SME. The overview of the incentives is available on the EMA website. In 2017, more than 1800 pharmaceutical companies were registered to the SME Office, confirming the success of this initiative.
The SME Office encourages pharmaceutical companies to initiate early dialogue with the EMA by promoting innovation task force (ITF) meetings. In early development (see Figure 1), all sponsors can proactively approach the ITF to have an informal dialogue on any issues in the development of their medicines (regulatory, technical and scientific issues). The objective of the ITF is to understand and discuss with the sponsors the challenges for innovative therapies and technologies (surgical implants, nanomedicines) or new scientific approaches (non-clinical and clinical methodology, new manufacturing techniques, etc.). In addition, the ITF can give advice for borderline products where there are some uncertainties about the definition of medicinal product or medical device. The ITF meeting is free of charge and can be considered as a complement or a preparation of the formal procedures (e.g., scientific advice and orphan drug designation). Participating in an ITF meeting could help to set a project on the right track and to already take strategic regulatory decisions in early development phase.
As one of the incentives, SME Office members benefit from a 90% fee reduction for EMA scientific advice. At any stage of development, a sponsor can request guidance to the national health authorities or to the EMA. This procedure is recommended when not enough relevant details are described in the scientific guidelines, the pharmacopoeia or when a developer wants to deviate from the guidelines. Questions during scientific advice are restricted to scientific issues about quality, non-clinical and clinical aspects or methodology issues. Definition of regulatory starting materials is a typical CMC aspect that can be discussed with the EMA. Failing to justify the regulatory starting materials is part of the top 10 deficiencies for new certificates of suitability to the monographs of the European Pharmacopeia (CEP). This topic can already be discussed after phase II to anticipate and reduce the risk at the time of Marketing Authorization Application. To request scientific advice, the sponsor should send a letter of intent and provide their questions in a briefing book. At Ardena, we have acquired a solid experience in providing appropriate details and data in the briefing book which is essential for a successful scientific advice procedure.
The Pediatric Investigation Plan (PIP) is a development plan ensuring the appropriate clinical evaluation of a drug in children. It also includes the adaptation of a medicine’s dosage and formulation to make its use more acceptable in children, such as use of a liquid formulation rather than large tablets. All applications for marketing authorization for new medicines have to include the results of studies as described in an agreed PIP, unless the medicine is exempt because of a deferral or waiver. To prepare the PIP, applicants can request scientific advice to EMA which is free of charge. The dialogue with the EMA can already start after phase I (see figure 1) but in practice the PIP can be submitted after phase II but before phase III to avoid delays at the stage of Marketing Authorization Application.
In response to the COVID-19, the EMA can provide early guidance to developers and have established accelerated scientific advice and PIP procedures for potential treatments and vaccines.
Please contact us to discuss your regulatory strategy. Ardena can provide full support for scientific advice meetings with the authorities including:
- Preparation of the briefing book
- Coordination of the procedure
- Preparation of a scientific advice meeting
This newsletter was written in collaboration with David Thomae, our Subject Matter Expert – Drug Substance Development. Want to know more about this topic, get directly in touch via email@example.com