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Study design
Strategic guidance to establish the exact context of use and ensure your rare disease efficacy studies are strictly fit for purpose.
Expert input for rigorous study design
We provide direct advisory input early in your rare disease program to prevent costly downstream rework. Our senior scientists help you determine statistical power requirements, benchmark against historical data and select the exact bioassays required to support your clinical endpoints.
Strategic advisory to de-risk development
We leverage decades of rare disease research to help you design pilot studies and comprehensive evaluations that maximize your resources.
Context of use alignment
We help define the exact regulatory context early, so your bioassay selection and biomarker endpoints remain strictly fit for purpose.
Statistical powering
We apply vast historical data across established rare disease models to ensure your efficacy studies are accurately powered before human trials.
Pilot study planning
We advise on small proof-of-concept evaluations to identify operational challenges early and ensure readiness for larger clinical trials.
Discuss your study design needs
Engage our senior scientists to establish a rigorous testing plan for your rare disease program.
Specialized expertise in rare disease research
Our scientific team applies decades of focused research to the disciplined execution of your program. We understand the unique challenges of rare disease development and deliver guidance that translates into data regulators trust.
Historical benchmarking:
We leverage archival data from over 100 trials in mouse models of muscular dystrophy to accurately power your efficacy studies.
Early risk identification:
We proactively address scientific and operational challenges during early testing to ensure readiness for first-in-human trials.
Harmonized workflows:
We align our preclinical standard operating procedures directly with our clinical protocols to drastically reduce downstream technology transfer time.
Clear accountability:
We manage specialized workflows and provide strategic guidance without demanding total control of your rare disease program.
Predictable schedules and early intervention
Our lean model keeps scientific expertise close to the work, ensuring fast escalation paths and prompt advisory input during your planning phase.
Built to integrate
AGADA Biosciences acts as a collaborative partner that integrates smoothly into your existing vendor ecosystem. We coordinate directly with your clinical sites and central laboratories to standardize collection protocols and ensure seamless handoffs.
Direct access to senior scientific leadership
You work directly with the decision-makers, guiding your rare disease program so operational challenges are resolved in minutes rather than weeks.
Protocol oversight:
Our lean model ensures senior scientists actively guide your efficacy studies and interpret your biomarker data.
Accelerated approval pathways:
We provide targeted guidance on primary and secondary biomarker outcomes to support efficient regulatory reviews.
Consistent audit success:
We successfully pass an average of five sponsor audits annually to support clinical trials across multiple phases.
Fit-for-purpose alignment:
We align your bioassays with rigorous FDA expectations for primary trial outcomes.
Start a conversation
Reach out to our team to align your study design with rigorous regulatory standards.
Take a virtual tour of our Halifax facility
Explore our state-of-the-art laboratory and discover how our team manages samples from receipt through to analysis and release. In this virtual tour, you’ll see how we apply Good Clinical Practice and relevant bioanalytical regulatory expectations to protect sample integrity, carry out validated bioanalytical work and support dependable data.
Quality and compliance
We operate in strict compliance with GCP, GLP and GDPR regulations to ensure your data remains interpretable and defensible. Every team member must hold a CITI certification before supporting any sponsor trial.
Meet our scientific leadership
Our founders and senior scientists are globally recognized leaders in rare and neuromuscular disease research, bringing decades of specialized expertise to your program.
News and resources
AGADA Biosciences provides expert advisory and historical benchmarking to align your rare disease study design with strict agency expectations.
