Supporting Drug & Biologic Development

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Dedicated support accelerating the development, approval, and accessibility of drugs and biologics. Our expertise spans navigating unique clinical trial challenges (small populations, ICH-GCP compliance), preparing specialized regulatory submissions leveraging orphan and expedited pathways (e.g., ODD, PRIME; BLA, NDA, MAA), managing complex CMC for advanced therapies, providing tailored technical writing (CMC, CSRs), implementing robust pharmacovigilance crucial for novel treatments, and guiding programs through global regulatory agencies (FDA, EMA, NMPA, TGA, PMDA, Anvisa) to achieve patient access aligned with our mission.

Regal Intel is committed as a non-profit partner to supporting organizations developing innovative pharmaceuticals and biologics and also targeting rare diseases. We understand the distinct scientific, clinical, manufacturing, and regulatory hurdles inherent in this field. Our comprehensive support services leverage deep industry expertise gained since 2001, now focused entirely on navigating this unique landscape. We partner with you from preclinical considerations and clinical strategy through global regulatory approval and post-market stewardship, aiming to bring vital therapies to patients faster and more efficiently.

We provide expert guidance tailored to the complexities of clinical trials ensuring compliance with ICH-GCP and addressing unique challenges like small and heterogeneous patient populations, geographical dispersion, potential use of novel endpoints, and frequent pediatric focus. Our support includes input on protocol development to optimize design for rare conditions, site feasibility assessment, strategies for robust data management with potentially sparse data, and ensuring trial execution maintains integrity for successful regulatory submissions across all phases (Phase I-IV) relevant to your program.

Our consultants specialize in preparing and managing comprehensive regulatory dossiers for rare disease therapies, particularly Biologics License Applications (BLAs), New Drug Applications (NDAs), and Marketing Authorisation Applications (MAAs). We expertly integrate requirements associated with Orphan Drug Designation (ODD), Rare Pediatric Disease Designation (RPDD), Humanitarian Use status (where applicable), pediatric plans (PIP/PSP), and expedited pathway designations. We ensure meticulous compilation, review, and submission in eCTD format to FDA, EMA, NMPA, TGA, PMDA, Anvisa, and other global health authorities, streamlining the path to approval for critical treatments.

Our technical writing team excels in creating clear, concise, and compliant documentation specifically tailored to the nuances of rare disease therapies, which often involve novel mechanisms or limited precedent. We develop comprehensive CMC documentation (often complex for biologics, cell/gene therapies), Clinical Study Reports (CSRs adeptly addressing unique trial designs and analyses), regulatory submission modules, and operational SOPs, ensuring adherence to cGMP and ICH guidelines within the rare disease context. Accuracy, consistency, and audit-readiness facilitate smooth regulatory reviews.

We provide expert CMC guidance critical for developing, manufacturing, and controlling often complex rare disease therapies like biologics, cell therapies, and gene therapies. Our support includes developing robust and phase-appropriate manufacturing processes, validating sensitive analytical methods (e.g., potency assays), designing appropriate stability studies, defining starting material controls, and establishing comparability protocols. We ensure strategies align with global regulatory expectations (FDA, EMA, etc.) for product quality, consistency, and supply chain integrity, addressing challenges like small batch manufacturing.

Robust pharmacovigilance is paramount for novel therapies, especially those approved via expedited pathways or based on limited initial data. We help develop and implement tailored, global pharmacovigilance systems compliant with reporting requirements. Our services include efficient adverse event processing and reporting, signal detection strategies suitable for sparse datasets, development and management of Risk Management Plans (RMPs) or Risk Evaluation and Mitigation Strategies (REMS), and support for fulfilling post-marketing commitments, ensuring ongoing patient safety and sustained compliance.

Regal Intel offers comprehensive regulatory partnership across diverse global markets. We possess deep expertise in the specific requirements and pathways for rare disease therapies within key agencies including the FDA (US), EMA (Europe), NMPA (China), TGA (Australia), PMDA (Japan), Anvisa (Brazil), Health Canada, and more. We act as an extension of your team, providing tailored solutions focused on achieving and maintaining global patient access, staying abreast of evolving rare disease regulatory trends, and ensuring your strategies effectively support your mission worldwide.