RAPS recognizes that the current situation in Ukraine impacts our members and customers on many levels. If you are directly impacted by the current situation in the region and are challenged to meet your deadlines or obligations to RAPS, please reach out to raps@raps.org so that we can defer those challenges. Your health and safety are paramount to us.

Regulatory Focus™ > News Articles > FDA, EMA Officials: Regulators Must Adapt to Effectively Regulate Precision Medicine

FDA, EMA Officials: Regulators Must Adapt to Effectively Regulate Precision Medicine

Posted 14 October 2016 | By Michael Mezher 

FDA, EMA Officials: Regulators Must Adapt to Effectively Regulate Precision Medicine

In order to effectively regulate precision medicine, regulators must adapt to alternative methods of evidence generation, say top officials at the US Food and Drug Administration (FDA) and European Medicines Agency (EMA), as well as the former chair of the UK's Medicines and Healthcare Products Regulatory Agency (MHRA).

The call is made in a commentary published in Nature Reviews: Drug Discovery on Friday by former MHRA Chair Alasdair Breckenridge, EMA Senior Medical Officer Hans-Georg Eichler and FDA's Jonathan Jarow, who serves as senior medical advisor to the center director of the Center for Drug Evaluation and Research.

Specifically, the authors of the commentary say regulators must consider factors across five key areas—evidence generation, patient involvement, cost, access and risk tolerance—in order to help move precision medicine forward.

According to the authors, advances in precision medicine have enabled an approach whereby genetic heterogeneity in both patients and diseases can be used to optimize treatment by determining which patients may see the most benefit, or the greatest risks, from a particular treatment.

However, they argue that such advances also present a number of challenges that regulators must address in order to facilitate further progress.


The authors argue that the advent of precision medicine threatens to shake "the bedrock of regulatory decision-making for the past 50 years," the randomized controlled trial (RCT).

In traditional drug development, they say, RCTs are viewed as the gold standard to support regulatory approval. However, with precision medicine, the authors argue such trials "may not be feasible," especially in cases where a patient population is split into multiple, oftentimes miniscule, subgroups.

In light of this, the authors argue that regulators must look to other forms of evidence generation, such as adaptive clinical trials or observational studies from the vast array of digital data available via "electronic health records (EHRs), patient registries and, in the future, perhaps social media."

However, the authors say the use of these alternative methodologies should not replace RCTs entirely, "In our view, observational studies based on real-world evidence should complement rather than replace RCTs, and on occasions they may be the only source of information available," the authors write.

The authors also argue that regulators must continue to support broader patient involvement in regulatory decision-making.

"Rather than considering patients as a large heterogeneous group, precision medicine provides the possibility of working with smaller, more homogenous and better-informed patient populations with potentially unique urgencies and needs that are clearly expressed," the authors write.

For regulators, they say, this means keeping patient preferences in mind and looking to patient-reported outcomes in more disease areas, such as oncology, where the emphasis has traditionally been on clinician-reported outcomes.



© 2022 Regulatory Affairs Professionals Society.