Pragmatic Clinical Trials Network to Focus on Genomic Medicine Interventions
The federal National Human Genome Research Institute (NHGRI) has announced a five-year effort to conduct pragmatic clinical trials to measure the clinical utility and cost-effectiveness of genomic medicine interventions and assess approaches for real-world application of genomic medicine in diverse clinical settings.
The IGNITE (Implementing Genomics in Practice) Network, funded in 2013 and 2014, worked to develop new methods for implementing genomic medicine in diverse clinical settings outside of specialized care and disseminated its findings to the genomic medicine community.
NHGRI’s genomic medicine research programs have explored and tested appropriate uses of genomic information in clinical care through collaborative networks evaluating electronic health record-driven phenotyping and return of genomic results, newborn and clinical genome sequencing, variant curation, and dissemination of genomic medicine approaches to diverse clinical settings outside specialized centers. Despite the positive impact of genomic medicine on improving health outcomes as demonstrated by these studies, it has become clear that a major barrier to large-scale genomic medicine implementation is the lack of compelling clinical evidence that would convince clinicians to adopt promising strategies and medical insurers to pay for them.
IGNITE II will support a network of multi-site clinical groups involving diverse settings and populations to conduct pragmatic clinical trials of genomic medicine interventions previously demonstrated to be feasible and of potential value in clinical care. Proposed trials will also include assessments of approaches for real-world applications and provide generalizable knowledge about when, how, and in what circumstances pragmatic clinical trials should be used in genomic medicine. Results of each trial will be compared to existing evidence from non-interventional studies to identify potential biases affecting the generalization of observational data to clinical practice in genomic medicine.
Research Examples
In its grant funding opportunity announcement, NHGRI said examples of research topics include, but are not limited to, the following:
• Pharmacogenomics-based drug selection and dosing
• Risk reduction in genetically defined high-risk individuals
• Early diagnosis in critically ill newborns or other groups at high risk of undetected genetic disease
A coordinating center will play a major research role in the development of the clinical protocols, analyses of data, protocol finalization and execution, development and statistical modeling, analyses of clinical and cost-effectiveness outcomes, and subgroup effect analyses.
In addition to the network-wide pragmatic clinical trials, IGNITE II may also include a network-wide study of ethical, legal and social implications research relevant to the diverse populations and healthcare settings represented in IGNITE II.
