We provide customized clinical trial solutions optimized through
systematic strategies that take into account the complex
pathophysiology of rare diseases and the dispersed patient
recruitment environment both domestically and internationally.
Rare Disease Therapeutics
Rare diseases are a complex and heterogeneous group of conditions that, while having low prevalence rates for individual diseases, collectively affect tens of millions of patients worldwide. Due to genetic factors, diverse mechanisms of onset, and heterogeneous symptoms, diagnosis and treatment are challenging, and standardized approaches to clinical trials are often limited. As a result, precise and systematic clinical development strategies are required.
Clinical development of rare disease therapies often employs methodologies such as single-arm designs, use of external control groups, and biomarker-based evaluations, rather than randomized controlled trials, due to the limited number of patients and considerations for patient ethics. In particular, the establishment of alternative evaluation variables and composite evaluation variables that reflect the natural course of the disease is important, and patient-reported outcomes (PROs) and imaging and biomarker analyses are also utilized as core tools.
In the early stages of clinical trials, it is important to establish inclusion/exclusion criteria based on the early identification of rare patient populations and genetic diagnostic technologies. Additionally, by linking with patient registries and real-world data (RWD) from around the world, a more feasible strategy for recruiting clinical trial participants is established.
Rare diseases require a high degree of scientific precision and strategic approaches in the drug development process due to their small patient populations and diverse pathophysiology. Based on our accumulated experience in developing treatments for various rare diseases, we provide customized clinical solutions that cover the entire cycle from initial strategy development to regulatory compliance. Considering the unique challenges of rare diseases, such as the difficulty of recruiting patients and the heterogeneous nature of disease progression, we design protocols that ensure both clinical significance and statistical validity.
We systematically plan clinical trials optimized for each disease characteristic, including sample size estimation, control group setting, utilization of alternative and composite endpoints, and efficient data collection strategies based on real-world clinical settings. Drawing on the expertise of medical doctors and professionals in pharmacy, nursing, biotechnology, and clinical statistics, we plan and execute clinical trials with disease-specific approaches.
We integrate and analyze various indicators, including genome- and proteome-based biomarkers, pharmacokinetics/pharmacodynamics (PK/PD), and patient-reported outcome measures (PROMs), and utilize them for treatment response prediction and subgroup analysis.
C&R leverages data-driven insights and disease-specific expertise to provide integrated, strategic services, from clinical trial design, statistical strategy, regulatory compliance, and decision support, professionally guiding every stage of rare disease therapeutic clinical trials.
clinical development with C&R Research.