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.

Inclusion/Exclusion Criteria

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.

Sample Analysis

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.

Age-Targeted Design

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.

Remote/Telehealth Design

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.

Endpoint Development

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.

Patient-Centered Design

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.

At Uncommon Cures, our goal is to make the uncommon possible. Explore our unique approach to rare disease study design and discover how we can support your clinical research efforts. For more information or to discuss a potential collaboration, contact us today.

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