Dedicated Global Support for BioTech Innovators

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Rare diseases affect families worldwide, often requiring a global approach to development and access.

Our international expertise is dedicated to helping rare disease innovators navigate complex regulatory landscapes. 

We cover across key regions, facilitating the journey of vital treatments to patients, wherever they may live.

We streamline the pathway to US market access for rare disease therapies by expertly navigating FDA requirements. Our support covers submissions crucial for rare diseases, including Orphan Drug Designation (ODD), Rare Pediatric Disease Designation (RPDD), Humanitarian Device Exemption (HDE), and utilization of expedited programs like Accelerated Approval, Fast Track, Breakthrough Therapy Designation (BTD), and Regenerative Medicine Advanced Therapy (RMAT). We manage standard submissions (510(k), IDE/PMA, IND, NDA, BLA) for drugs, biologics, devices, and advanced therapies, ensuring meticulous preparation and eCTD compliance to accelerate review timelines.

Navigate the evolving European regulatory landscape with confidence. Regal Intel provides comprehensive support for EMA pathways critical for rare diseases, such as ODD, Priority Medicines (PRIME), Conditional Marketing Authorisation, and Authorisation under Exceptional Circumstances. We manage Clinical Trial Applications (CTA) and Investigational Medicinal Product Dossiers (IMPD), and ensure compliance with the Medical Device Regulation (MDR) and In Vitro Diagnostic Regulation (IVDR), including addressing the specific challenges and guidance related to orphan medical devices, facilitating seamless access for rare disease patients across the EU/EEA. 

Expand your reach to patients in the dynamic Asia-Pacific region. We provide tailored solutions for navigating the diverse regulatory frameworks specifically for rare disease products in key markets:

• China (NMPA): Leveraging recent technical guidelines, priority review channels, and potential market exclusivity provisions for rare disease drugs.
• Japan (PMDA): Including expertise with pathways like Sakigake designation for innovative therapies.
• South Korea (MFDS), Singapore (HSA), Australia (TGA - including its orphan drug program), and New Zealand. Our in-depth knowledge ensures strategies align with local requirements to effectively reach patient populations.

Unlock access for therapies including rare diseases in key Latin American markets. We offer strategic guidance for navigating the unique regulatory landscapes and varying definitions of rare diseases across the region (e.g., Brazil - ANVISA, Mexico - COFEPRIS, Argentina, Costa Rica, Chile). Our expertise facilitates compliant market entry, helping to address access challenges for rare disease patients in these growing regions.

Stay ahead in the complex and evolving rare disease environment. Regal Intel provides partners with timely, actionable intelligence on global regulatory changes specifically impacting rare and other diseases – such as new FDA guidances, evolving EMA policies (like those for orphan devices), NMPA initiatives in China, and more. This targeted intelligence informs strategic decision-making and enables effective navigation of regulatory complexities unique to this field. 

Wherever your mission leads, Regal Intel provides the dedicated expertise to help navigate the path forward.