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Comparative Effectiveness Research to Advance Personalized Rx in FDA's Five-Year Plan

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Originally published April 25.

By Turna Ray

By integrating comparative effectiveness research in its review processes, the US Food and Drug Administration is hoping to gain more insight into the safety and efficacy of drugs in subpopulations of patients and advance personalized medicine.

In a report FDA released this week outlining its strategic priorities for the next five years, the agency highlighted an ongoing project that aims to use CER in order to improve personalized medicine.

A year ago, the FDA solicited proposals for the Partnership in Applied Comparative Effectiveness Science, or PACES, initiative, through which the agency was seeking to advance CER through data it already had and to develop a standardized database. One goal of the project, which was awarded last fall to a research team at Johns Hopkins University, is to use CER "to better understand what interventions work best for individuals and subgroups within populations," according to the proposal.

In the five-year priorities report released this week, the agency included the PACES effort among eight "signature" initiatives that exemplify its goals through 2015.

"FDA houses the largest known repository of clinical data — unique, high-quality data on the safety, effectiveness and performance of drugs, biologics and devices, both before and after approval," the agency said in the priorities report. "Despite the availability of these data, questions about subpopulation responses and underlying placebo effects remain unanswered."

The PACES initiative comes after the federal government funneled $1.1 billion into CER in the American Recovery and Reinvestment Act. Through PACES, the FDA is hoping to integrate comparative effectiveness research into its review process with more insight into the safety and efficacy of treatments in subpopulations of patients.

How this effort will translate into evidence requirements for investigational products reviewed by the agency is something industry players are undoubtedly wondering, but the FDA has disclosed little in this regard.

In its solicitation for PACES proposals, the FDA explained that it was looking to combine pre- and post-approval data collected internally at the agency with other datasets on long-term health outcomes at other agencies or at private companies.

"As personalized medicine develops, FDA expects an increase in the need for comparisons across products (and associated delivery mechanisms) to define how these products should be used in combination for individualized health care before and after FDA approval," the agency said in the proposal document. "Development and use of sound, consistent science-based approaches to the design, conduct, and analysis of such data and the assessment for labeling and regulatory purposes, will be critical."

Although the PACES effort grew out of FDA Commissioner Margaret Hamburg's efforts to increase the agency's commitment to advancing personalized medicine, the agency can't yet say for sure whether genomics data will feature prominently in the FDA and JHU's CER work.

When asked whether genomics data will be factored into the CER conducted under this project, an FDA spokesperson told PGx Reporter that the agency is currently evaluating the types of data that will be available for patient-centered outcomes research, or PCOR, initiatives with JHU. "In addition, the selection of data for this initiative will also depend on the types of PCOR questions we need to answer," the spokesperson said.

As the PACES initiative has been taking shape, the federal government has formed the Patient Centered Outcomes Research Institute, a private, non-profit entity charged with developing and funding CER. The institute's aim is to help patients, clinicians, purchasers, and policy-makers make "informed health decisions" by conducting research on the quality and effectiveness of medical treatments (PGx Reporter 09/29/2010).

Whether personalized medicine strategies will feature prominently in CER studies under PCORI also remains to be seen. While there are personalized medicine stakeholders represented in PCORI's board of governors, none of the industry representatives in the institute's methodology committee are personalized medicine experts. And although the integration of personalized medicine and CER has advocates in high places, such as National Institutes of Health Direct Francis Collins, some personalized medicine stakeholders fear that the CER mechanisms funded by the government will be mostly applied to advance and pay for treatments for the general population.

The Personalized Medicine Coalition — a non-profit advocacy organization that has been a strong proponent of conducting CER to advance safe and efficacious medicines in genomically defined subpopulations — had nominated 10 experts in the field for the PCORI methodology committee. None of them were picked for the committee.

"PMC was disappointed to see that none of the private sector members bring expertise in personalized medicine, particularly since the statute specifically identified experts in genomics as necessary to the methodology committee function," the organization wrote in the recently released Spring edition of its newsletter.

The coalition recommends that the PCORI board of governors create an ad hoc advisory panel focused on personalized medicine and innovation to ensure that genomic and other personalized medicine considerations are addressed in the development of research methods and priorities.

In its PACES solicitation, the FDA described the scope of work for contractors under the initiative. Contractors will work with the agency's experts to define the questions they seek to answer through CER; develop methodologies for conducting CER, formulate new scientific computing strategies; build innovative clinical trial designs for CER; conduct analyses on datasets, including those stored in the Janus data warehouse; and advance best practices for submission CER data to the FDA when products are being compared.

"The ultimate goal of having the ability to review multiple studies that can be compared and analyzed requires data harmonization and standardization such that comparisons between data can be made effectively," the agency stated in the strategic priorities report. "The data must then be organized in a common database so that it can be queried by topic and analyzed to address key questions."

To this end, the agency plans to invest in informatics hardware and software and develop standardized data models. Additionally, "FDA has sponsored the development of a clinical trials repository that will be capable of handling this task," the agency said.


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