Accelerating Hope: Clinical Trial Solutions

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The journey to bring a new therapy to patients is paved with unique and formidable challenges. From exceedingly small and geographically dispersed patient populations to limited natural history data and the imperative for innovative trial designs, navigating the clinical development pathway requires specialized expertise, unwavering dedication, and a deeply patient-centric approach.

As your partner, we are committed to transforming the landscape of rare disease and advanced therapy research. We offer a comprehensive suite of clinical trial services, meticulously designed to address these complexities. Our goal is to accelerate the development of safe and effective treatments, bringing hope and tangible solutions to patients and families affected by rare conditions.

We provide end-to-end support, acting as your dedicated partner or seamlessly integrating with your existing teams and preferred vendors.

1. Strategic Clinical Development & Regulatory Planning:

• Early-Phase Strategy & Integrated Development: We specialize in crafting agile and efficient clinical development plans, particularly for early-phase (Phase I/II) studies common in rare diseases, ensuring alignment with your overall product development strategy and target product profile (TPP).
• Regulatory Roadmapping: Our clinical strategies are interwoven with global regulatory insights, supporting Investigational New Drug (IND), Clinical Trial Application (CTA), and other crucial regulatory submissions from the outset.
• Feasibility & Risk Assessment: Comprehensive analysis to identify potential challenges early and develop mitigation strategies for study startup and execution.

2. Expert Clinical Trial Design & Biostatistics:

• Innovative Trial Methodologies: We champion the use of innovative and adaptive trial designs suited for small populations, including N-of-1 trials, basket/umbrella/platform trials, and Bayesian approaches, to maximize data generation and efficiency.
• Robust Biostatistical Expertise: Our in-house team of expert biostatisticians provides critical support from concept to close-out, including: 
  • Sophisticated sample size calculations and power analyses.
  • Development of robust Statistical Analysis Plans (SAPs).
  • Data modeling, simulation, and advanced statistical analysis.
• Endpoint Selection & Validation: Guidance on selecting and validating clinically meaningful primary and secondary endpoints, including the development and integration of Patient-Reported Outcomes (PROs), Clinician-Reported Outcomes (ClinROs), and Observer-Reported Outcomes (ObsROs), as well as leveraging novel biomarkers.

3. Full-Service Clinical Trial Management & Execution:

• Dedicated Project Management: Experienced project managers ensure meticulous oversight, adherence to timelines, budget management, and transparent communication.
• Site Identification, Qualification & Activation: Leveraging our networks and expertise to identify, qualify, select, and efficiently activate appropriate investigator sites globally, with a focus on centers of excellence for specific rare diseases.
• Patient Recruitment & Retention: Developing and implementing tailored strategies to effectively recruit and retain patients in rare disease trials, often involving close collaboration with patient advocacy groups (PAGs) and utilizing digital outreach.
• Clinical Monitoring: Comprehensive site monitoring (on-site, remote, and risk-based) to ensure patient safety, data integrity, and GCP compliance.
• Data Management & EDC Solutions: End-to-end data management services, including eCRF design, EDC system deployment (compliant with 21 CFR Part 11), data cleaning, query resolution, and adherence to CDISC standards (SDTM, ADaM).
• Pharmacovigilance & Safety Monitoring: Rigorous safety surveillance, adverse event reporting, and coordination with Data Safety Monitoring Boards (DSMBs).
• Compliance & Quality Assurance: Unwavering commitment to ICH-GCP guidelines, applicable global regulatory requirements, and internal quality standards.

4. CRO & Vendor Management/Partnership:

• Functioning as Your Dedicated CRO: We can take full responsibility for the operational execution of your clinical trial, offering a single point of accountability.
• Collaborative CRO & Vendor Oversight: If you have existing CRO or vendor relationships, or require specialized niche providers, we offer expert oversight services. This includes: 
  • Vendor selection and qualification.
  • Performance management and milestone tracking.
  • Ensuring alignment with your strategic objectives and quality standards.
  • Gap analysis and issue resolution.

5. CMO Coordination & Clinical Supply Chain Management:

• Seamless CMO Liaison: We act as a crucial link with your Contract Manufacturing Organizations (CMOs) to ensure timely production, quality control, packaging, labeling (including multi-language and country-specific requirements), and release of clinical trial materials (CTM).
• GMP-Compliant CTM: Verifying that all CTM is manufactured and handled according to Good Manufacturing Practices (GMP).
• Global Logistics & Distribution: Managing the complex logistics of CTM distribution to clinical sites worldwide, including cold-chain management for temperature-sensitive biologics, cell and gene therapies, and other advanced modalities.
• Import/Export & Depot Management: Navigating international trade compliance for clinical supplies and coordinating with global depots.

6. Medical Writing & Regulatory Submission Support:

• High-Quality Clinical Documentation: Preparation of key clinical documents, including: 
  • Clinical study protocols and amendments.
  • Investigator's Brochures (IBs).
  • Patient-centric Informed Consent Forms (ICFs).
  • Clinical Study Reports (CSRs) compliant with ICH E3.
• Regulatory Document Contribution: Providing expert input and authoring for the clinical sections of regulatory submissions (e.g., INDs, IDEs, NDAs, BLAs, MAAs).

7. Patient Centricity at the Core:

• Integrating the Patient Voice: We actively incorporate patient and caregiver perspectives into trial design and conduct to enhance relevance, reduce burden, and improve engagement.
• Collaboration with Patient Advocacy Groups (PAGs): Building strong partnerships with PAGs for insights, recruitment support, and dissemination of information.
• Decentralized Clinical Trial (DCT) Solutions: Implementing appropriate DCT elements (e.g., remote monitoring, telehealth, home health visits, direct-to-patient CTM shipment) to improve accessibility and convenience for rare disease patients.
• Ethical Considerations & Data Transparency: Upholding the highest ethical standards and supporting initiatives for the appropriate return of individual research results to participants.

• Unparalleled Rare Disease Focus: We live and breathe rare diseases and advanced therapies. Our team possesses a profound understanding of the unique scientific, operational, regulatory, and ethical considerations inherent in this space.
• Dedicated, Multidisciplinary Experts: Access a core team of seasoned clinical operations professionals, project managers, medical monitors, data managers, and biostatisticians with specific rare disease trial experience.
• Flexible, Scalable & Tailored Solutions: Our services are customized to your specific needs, asset type, phase of development, and organizational structure. We adapt to you.
• Unyielding Commitment to Quality & Compliance: Rigorous adherence to ICH-GCP, global regulatory standards, and robust internal quality management systems.
• Patient-First Philosophy: Patients and their families are at the heart of everything we do. We prioritize their safety, well-being, and the urgency of their unmet needs.
• True Collaborative Partnership: We function as a seamless extension of your team, fostering open communication, transparency, and shared goals.
• Mission Alignment: Our non-profit status allows us to prioritize impact and patient benefit, offering cost-effective solutions focused on advancing your mission.

If you are developing a therapy for a rare disease or pursuing advanced therapies, let's discuss how our specialized clinical trial expertise can support your journey from scientific discovery to patient impact.

Contact us today to learn more about our tailored clinical trial solutions.