Rare Disease Study Design
Patient-Centric, Efficient, and Impactful
Welcome to Uncommon Cures’ Rare Disease Study Design page. Here, we shed light on our unique, patient-centric approach to designing clinical trials for rare diseases. Our methodology is rooted in practicality, inclusivity, and relevance, ensuring each study is tailored to the unique needs of the rare disease community.
Types of Studies
Our expertise spans a range of study types, including drug trials, natural history studies, device trials, preclinical data studies, opinion surveys, post-marketing studies, and gene therapy trials.
We strive to develop the least restrictive criteria to maximize enrollment from a real-world relevant rare disease population. This inclusivity widens the scope of our trials and enhances their relevance and utility.
Clinical Form Development, Digital
We develop user-friendly clinical forms and provide secure, compliant digital housing and warehousing for efficient data collection and management.
We identify the best labs for sample analysis, considering both cost-effectiveness and capability to ensure accurate and reliable results.
Rare Disease Protocol Specialists
Our protocol designers bring extensive experience in rare disease clinical trials, ensuring each study is expertly crafted and executed.
Working closely with key opinion leaders (KOLs), the FDA, and patients, we identify the most relevant age groups for each study, ensuring the trials we design are reflective of the disease's impact across the lifespan.
To further reduce the burden on patients and families, we incorporate digital and remote visits whenever possible, making participation more accessible and convenient.
Minimal Volume Design
We are committed to reducing the burden on patients by determining minimal sample amounts, cutting down on blood draws, and streamlining sample processing.
Post-Marketing Study Design
We develop post-marketing study designs that align with the sponsor's needs and the patient's clinical journey, ensuring ongoing evaluation and improvement of therapies.
IRB Material Development
Our IRB materials are specially designed to be patient-approachable and emphasize remote consent and outreach to underserved groups, ensuring ethical and inclusive research practices.
Realistic Cost Projections
We provide a transparent, open pricing model with fixed margins and contingencies. Our service list includes well-defined costs for elements and pre-contracting for external services.
FDA Pre-Submission Meetings
Our team works hand-in-hand with the FDA to ensure our study designs meet regulatory standards and are optimized for success.
Rare Disease Relevant Statistics
We employ statistical analysis methods tailored to small population sizes, ensuring accurate sample size determination and success measures.
Our team meticulously develops endpoints that are achievable, relevant to the rare disease in question, and align with the patient community's views of utility. This approach ensures our studies are meaningful and impactful.
At the heart of our study design process is the patient. We believe that understanding and incorporating the patient's perspective is crucial to designing effective and low-burden clinical trials. We prioritize practicality and seek patient input at every stage of the study design.