New Regulatory Science Initiative Will See Focus on REMS, Safety Frameworks and Patients

Posted 12 March 2013 | By Alexander Gaffney, RAC 

The term "regulatory science" has been one of the hallmarks of US Food and Drug Administration (FDA) Commissioner Margaret Hamburg's tenure since she took office in 2009, and now the term is getting new institutional support in the form of a partnership with a leading healthcare organization that could make changes to the way FDA regulates many products.


The term, which FDA defines as the "science of developing new tools, standards and approaches to assess the safety, efficacy, quality and performance of all FDA-regulated products," has been central to many of FDA's efforts in the last three years.

In issue after issue and appearance after appearance, Hamburg has touted the approach, saying it's one way for regulators to better accomplish their jobs and avoid the scrutiny of skeptical politicians.

"Frankly, these days, at least in Washington, you are far more likely to hear the word "regulatory" linked to the phrase "job-killing" than to "science," quipped Hamburg in an April 2012 appearance before a New England Healthcare Institute (NEHI) conference . "But believe me, this is a short-sighted view."

And neither Hamburg nor her agency seem likely to be in it for anything but the long term. In November 2012, the agency started a campaign intended to promote regulatory science to the public by explaining the field in plain terms.

But public outreach is just one part of FDA's efforts. The other, larger effort, involves working with various stakeholders to advance the various fields of regulatory science.

In January 2013, for example, FDA issued a grant proposal calling for groups to help it address a number of regulatory science issues, including those related to the communication of risk. In December 2012, the agency announced it would partner with LifeScience Alley to ensure that the US maintains its status as "the leader in regulatory science."

A New Agreement

But both of those agreements pale in comparison to a new agreement FDA said it plans to accept between it and the Engelberg Center for Health Care Reform, part of the think tank Brookings Institute. Under the 2012 FDA Safety and Innovation Act (FDASIA), FDA was tasked with building new regulatory capabilities, and particularly ones related to regulatory science.

The initiatives include ones related to expediting drug development, advanced meta-analysis, the use of biomarkers and patient-reported outcomes, rare disease drug development, risk mitigation and post-market monitoring.

Patient-reported outcomes (PROs), in particular, are set to get a big boost under FDA's four-year, $3 million partnership with the Engelberg Center.

FDA said that while it's already started working toward the development of PRO tools by adding new staff, consulting with patients and promoting best practices, it has a lot more it wants to get done in the near future.

"FDA seeks to identify the challenges and opportunities within the current review and qualification of PROs, to address key issues with PRO evidentiary standards, develop new methods for communication between the multiple stakeholders involved in PROs, and identify best practices for evaluation and statistical analysis and design of PROs," FDA wrote in a 12 March 2013 Federal Register posting.

Benefit-Risk Assessments and REMS

Some of those patient-focused discussions will also work their way into a reassessment of the current benefit-risk structure used by FDA to review products. The agency recently launched a new framework for evaluating benefit and risk in which it rejected a wholly quantitative approach for one that takes into account flexible definitions of risk that depend on a variety of factors.

That approach is one that could stand to benefit from more input from stakeholders, FDA conceded. Under the agreement, the Engelberg Center will facilitate engagement with stakeholders in order to "develop a more structured approach to benefit-risk assessment," including premarket and postmarket safety reviews.

Finally, the partnership is set to put a heavy emphasis on the evaluation, standardization and integration of Risk Evaluation and Mitigation Strategies (REMS). FDA has recently started to issue class-wide and more standardized forms of REMS, which are used to control access to medicines and make sure they are taken safely.

The Engelberg Center is set to research approaches to standardize the methodology used to determine when REMS are necessary, as well as approaches for implementing and evaluating REMS plans.

The determination methodology would, if successful, "allow FDA and sponsors to proactively identify and address the underlying causes of patient harm, and evaluate and prioritize risk management interventions based on evidence of their effectiveness and burden on the health care delivery system," FDA wrote.

The partnership, which is being facilitated by FDA's Center for Drug Evaluation and Research (CDER), is set to begin on 1 June 2013.

Tags: REMS

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