Publication date: Jun 08, 2019
But precision medicine development can pose significant challenges as well: getting to a successful regulatory approval requires sponsor companies to embrace many new technologies and systems during the development process.
-Developing these medicines requires changes to traditional clinical trial designs, as well as the use of innovative testing procedures that result in new types of data,” she says.
-Precision guided medications are often able to show strong evidence of efficacy in early stage trials, where patients are selected based on discrete biomarkers and other identifying criteria” says Sugato De, vice president, Regulatory and Access at Parexel.
-The agency is committed to pursuing a regulatory framework that prioritizes novel clinical trials and real-world data solutions to provide robust evidence of safety and efficacy at early stages.
-Streamlining product development will require more agile approaches that leverage real-world data, data sharing, and the adoption of trial designs that emphasize demonstrating effectiveness at early stages” says De.
-In order to fully realize the promise of precision medicine” says De, -the clinical research ecosystem must transform to eliminate barriers between real-world data and traditional clinical trials, so that evidence can be shared efficiently to inform a learning construct for regulatory assessments and healthcare decisions. “
Precision medicine trials often generate new types of data, such as genome sequencing data, that doesn’t fit neatly into traditional clinical trial structures.
Precision medicine development may use data from a wide variety of sources, such as wearables, electronic health records, patient-reported outcomes, and complex genomic and biomarker results.
Biomarkers are a critical component of precision medicine development and, when used for patient selection in clinical development, are associated with 2-3 fold increase in gaining regulatory approval and often through accelerated pathways.
This early engagement and planning is critical; it will help ensure that a development program for a precision medicine includes a well-defined regulatory path, including what to file, when to file and what data are needed for the filing.
In some countries, it is very difficult to collect samples for genetic and biomarker research, and without a sufficient number of samples that represent the clinical trial population, it will be hard to demonstrate the safety and efficacy of a new therapy.
In addition, adherence to traditional clinical trial designs and resistance to leveraging real-world evidence and data sharing mechanisms may delay drug development.
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