Regulatory affairs support for new drug approvals

We support your journey through global regulatory pathways — navigating efficiently from initial drug discovery through development and avoiding unnecessary delays to gain rapid approvals for clinical trials and marketing authorisations. Our aim, ultimately, is to leverage our experience and bring potentially life-saving treatments to patients as quickly as possible.

Many of our clients work with innovative drugs or in rare indications that fall outside typical regulatory guidelines. For drugs addressing rare conditions and diseases, the traditional stages of drug development often differ or don’t apply, meaning navigating the new drug approval pathway can be challenging.

At TMC, we will guide you, as your partner, throughout the drug development process — using our expertise in regulatory processes for rare diseases to help you navigate these challenges, design effective clinical studies and secure early-stage approvals.

At the heart of our regulatory support services is our mission — indeed our passion — to support drug development solutions that improve patients’ lives. By focusing on speeding up the clinical trial process and minimising the time to launch, our team ensures your novel therapies for rare disorders and diseases reach the patients who need them quickly and efficiently.

Types of regulatory submissions for rare diseases

As a specialist clinical research organisation for rare disorders and diseases, we have extensive experience with all types of regulatory submissions in this area — particularly across our key specialisms of oncology, neurological disorders, respiratory diseases, haematology, ophthalmological conditions, neonatology, hepatology and nephrology diseases.

For rare conditions and diseases, where clinical studies often involve smaller patient populations, our experience in securing orphan drug designations can provide significant benefits, including incentives such as fee reductions and market exclusivity. As the standard guidelines may not fully cover the development needs of drugs for rare diseases, our expert team can guide you through obtaining the appropriate scientific advice and agency meetings. Our expertise also extends to facilitating a smooth process for submitting paediatric investigation plans and common technical documentation.

As part of our commitment to building a trusted partnership, another key service we offer is acting as your marketing authorisation holder (MAH). We take on all the legal and regulatory accountability to ensure your product is manufactured, marketed and distributed according to the terms and conditions laid out in the marketing authorisation. We make sure, on your behalf, that the product always meets the quality, safety and efficacy standards as approved by the regulatory authorities, helping you get your product to market faster whilst you set up your local legal entity.

Discounts on regulatory submissions fees for SMEs

From 1 January 2025, the European Medicines Agency (EMA) is implementing the EU Regulation 2024/568 on fees for regulatory submissions. This change will result in a significant increase in some of the fees payable to the EMA for scientific assessment of a medicinal product for the application of a marketing authorisation, variation or authorised product.

As an established EU/EEA-based marketing authorisation holder with SME status, TMC can be your EU/EEA representative, ensuring compliance with all local regulations whilst securing substantial discounts on EMA fees.

The perfect FIT for regulatory support

We help you accelerate your progress through the regulatory pathway and bring treatments for rare disorders and diseases to patients swiftly through our flexible, integrated and tailored services.

Flexible model

Regulatory requirements differ significantly between regions. We’ve developed a close bond with our global network of trusted and highly skilled associates, enabling us to adapt our business model to ensure the swift and cost-effective deployment of resources. Whether you’re seeking approval in Europe, the US or globally, our team’s localised knowledge of various regulatory regions will ensure you remain compliant and meet the standards of each market you’re entering.

Integrated approach

From pre-clinical laboratory testing to clinical studies and final product approval, the regulatory pathway involves a series of intricate stages of drug development that require collaboration to ensure seamless progression. That’s why our people are your people. We support you every step of the way during the project journey, working in tandem with our clinical, pharmacovigilance and medical teams to achieve the best possible outcomes for you and your patients.

Tailored solutions

No two drug discovery and development programmes are the same — just as no two patients are the same. Our extensive experience in new drug approvals for rare diseases allows us to navigate complex regulatory processes and explore creative and effective solutions to gather the necessary data for approval. This approach helps us secure critical orphan drug designations and product licences, accelerating your journey to market.

Get In Touch

Together, we can ensure novel, life-saving therapies reach the patients who need them — faster and more effectively. Email our team today or contact us below to discuss how we can support your regulatory needs and help you navigate the complex landscape of new drug approvals for rare conditions and diseases.