In order to leverage pre-approval and post-market data collected by FDA to advance comparative effectiveness research and personalized medicine,
the agency is considering establishing two centers to provide agency researchers with analytic CER strategies and suggestions for clinical trial designs, according to a notice released to industry earlier this month. Even though FDA does not have explicit statutory authority to mandate that new products are superior to legacy medical interventions, the new centers would provide the agency with strategies to potentially incorporate CER into its new life-cycle approach to regulating health care products (see related story).
The focus on comparative effectiveness at the agency comes as FDA Commissioner Margaret Hamburg and other high-ranking officials recently affirmed their views on the importance of personalized medicine and CER, with
Through the Partnership in Applied Comparative Effectiveness Science initiative, FDA would establish two centers that would use data compiled by the agency “to form an enhanced standardized database” and provide strategies for using CER at FDA. The agency recently began soliciting information from potential contractors to establish the CER centers that could potentially be erected by two different organizations.
“The PACES initiative will be conducted with centers residing outside of the FDA who have medical, biological, and clinically relevant computational expertise,” FDA says in its notice. “These centers will work with
FDA contends that a science-based approach to evaluating data and the regulatory process are critical to realizing the full potential of CER by leveraging data collected by the agency and other institutions.
While the details of the center and data access remain undetermined, the agency encountered significant resistance to the development of its Sentinel post-market data surveillance system because of privacy concerns.
“FDA houses the largest known repository of clinical study data — including data on drug biologics and devices safety, effectiveness and performance,” the agency says. “These data are an invaluable resource for CER. Both pre- and post-approval data collected and housed at the FDA can be combined with other datasets on long term health outcomes that reside in other agencies or the private sector.”
The centers will provide CER scientific training and bolster coordination between the private sector and FDA staff, including sharing studies and possibly collaborating on the publication of scientific manuscripts. “Using CER methodologies, these Centers will address potential uses of large FDA datasets as well as or in conjunction with health outcomes data from other sources,” according to FDA. “In addition, the Centers will help to develop innovative clinical trial design and data analysis strategies that can be used by the Government to facilitate prospective studies that incorporate CER and personalized medicine approaches.”
The PACES centers will also evaluate CER clinical trial designs and recommend to FDA methodologies to more fully incorporate personalized medicine into oversight of medical products.
“The Contractor shall develop innovative clinical trial design strategies for prospective CER clinical trials, including providing formal recommendations for best practices for submission of studies to the FDA when they involve product comparisons as well as CER related work targeted towards personalized medicine,” according to FDA. “These strategies and recommendations shall be documented in reports and manuscripts suitable for publication in scientific journals.
In its notice to industry, FDA heralds comparative effectiveness studies as improving knowledge of the effects of certain interventions on subpopulations by determining what groups would benefit from certain medical products or face severe adverse events. In order to realize the benefits of CER, the agency must identify the subpopulations associated with different medical conditions through a scientific framework bolstered by the work of the centers.
“CER can be instrumental in supporting and driving the development of personalized medicine, and improving not only the efficiency of care, but also outcomes for all individuals,” FDA says. “The science can be used to increase patient benefit, by identifying individuals who are most likely to benefit from an intervention, and to reduce risks, by identifying and protecting individuals who may be harmed.”
Each center is expected to house multiple staff members familiar with CER and personalized medicine issues, including two principal investigators, a senior statistician, a programmer, a post-doctoral fellow and a graduate student.
“The application must demonstrate the availability of personnel with the necessary expertise to study a wide range of topical areas in CER research and have experience in the areas of dissemination, implementation, and evaluation of such,” FDA says.
Through the contract, the centers would be established for up to five years that includes a three-year base period and two single-year options. FDA intends to issue either a fixed-cost contract or based payment on work hours, but the agency does not specify an anticipated funding parameter for the program.