Premier Research Releases Expert White Paper to Accelerate Pediatric Rare Disease and Oncology Research and Regulatory Submissions
For sponsors with limited experience in rare disease and pediatric drug development, transitioning smoothly from the formulation of rare disease and pediatric research plans to the successful implementation of clinical trials is a daunting challenge.
To help sponsors navigate challenges related to site selection, research center identification, and patient recruitment forecasting, a well-designed and rigorously executed feasibility study enables an objective assessment of clinical trial feasibility, thereby facilitating the selection of the optimal pathway for the successful conduct of global clinical trials.
To provide sponsors with guidance on rare disease and pediatric research, global experts in the fields of rare diseases and pediatrics have specially shared the white paper “The Science and Art of Conducting Clinical Trial Feasibility in Rare Disease and Pediatric Studies” (hereinafter referred to as the “White Paper”) to address professional expertise at the intersection of pediatrics and oncology.
This white paper delves into three critical aspects of clinical trial feasibility for rare diseases and pediatric research—country feasibility, site selection, and patient recruitment forecasting—and provides targeted insights and strategies. Sponsors can leverage these insights and strategies to conduct global studies successfully and efficiently, in accordance with applicable regulatory and product quality guidelines.
The white paper mainly includes:
Background Introduction
The Necessity and Positive Impact of Feasibility Studies for Clinical Trials
Country Selection
● Utility of National Feasibility Assessment
● Epidemiology and Eligible Population
● Nursing Standards and Patient Pathways
● Competitive Assay
● Research Initiation Timeline
● Patient Advocacy Organizations/Patient Networks
● Motivational Factors for Participation
Selection of Research Centers and Researchers
● Evaluation of Research Centers
● Researcher Assessment
● Useful Resources for Research Centers and Researchers
Patient Recruitment Forecast
Use a valid and accurate recruitment prediction model for forecasting.
Scan the QR code to download the white paper “The Science and Art of Conducting Clinical Trial Feasibility in Rare Disease and Pediatric Studies” to access more valuable insights on implementing rare disease and pediatric research.
Pharmaceutical and biotechnology companies lacking experience in rare disease and pediatric research may consider establishing industry collaborations or partnerships with CROs that possess expertise in these areas. For further information, please contact the Pumeier Pharma expert team involved in compiling this white paper to consult on strategies for enhancing R&D efficiency and improving product approval success rates in rare diseases and pediatrics.
Premier Research is a global clinical research organization (CRO) dedicated to helping biotechnology firms, pharmaceutical companies, and medical device innovators translate life-changing concepts and scientific breakthroughs into new diagnostic and therapeutic solutions. The company operates in 84 countries worldwide, employs 1,300 professionals, and maintains a robust network of international partnerships. Premier Research specializes in innovative technologies for smart study design and trial management, providing sponsors with comprehensive services that include developing product lifecycle strategies, accelerating clinical development timelines, ensuring the generation of clean and robust data, and navigating global regulatory affairs.
