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New Regulatory Approaches Key to Future of Personalized Medicine

Posted 13 May 2015 | By Zachary Brousseau 

New Regulatory Approaches Key to Future of Personalized Medicine

Personalized medicine has been heralded as the future of medicine, a new way forward that will simultaneously trim healthcare costs and improve care by doing away with ineffective treatments. While these large-scale benefits are still to come, personalized medicine—also called or precision medicine—holds great potential, particularly if governments, researchers, regulators and industry can figure out how best to nurture it.

Efforts are underway to promote development of personalized medicine. Earlier this year, President Obama announced the launch of the Precision Medicine Initiative during his State of the Union address. Incidentally, it was one of very few of the president’s proposals that garnered some bipartisan support. In Europe, organizations like the European Alliance for Personalised Medicine, the European Association for Predictive, Preventive and Personalised Medicine and EuroBioForum are working to advance personalized medicine.

While full realization of the potential of personalized treatments may yet be years off, it already is beginning to pose new challenges for both regulators and those involved with developing and marketing products that rely on a personalized approach to patients and their treatments. Regulatory agencies must adapt and keep pace with the personalized medicine technology. Developing new regulatory frameworks to address the unique challenges of personalized treatments will be critical to unlocking their potential. Product sponsors must demonstrate safety and effectiveness in new ways.

Next week, 18–19 May, leading experts in the field will gather in Brussels to address some of these challenges during a free RAPS workshop on personalized medicine and companion diagnostics in the EU. The workshop will cover a range of issues associated with developing and licensing personalized medicines and matching diagnostics.

Personalized Medicine in the Next 10 Years

Two faculty members for the RAPS program recently spoke with Regulatory Focus and expressed cautious optimism about the potential for personalized medicine to make a significant impact within the next decade.

"I think the days of the big blockbuster 'one drug for everyone' are behind us," says David Kern, senior director, regulatory affairs for Illumina. Kern, whose work in HIV research has shown him how even a relatively simple genome can be very complex, says he expects the evolution of personalized medicine to be both challenging and perhaps more complicated than anyone would like, but he is nonetheless enthusiastic about its direction.

"I'm hoping that in the next 10–15 years as we really start to get these massive projects underway, sequencing hundreds of thousands of people, we will start to see some patterns and pathways," says Kern.

Geert Callaerts, director of regulatory affairs for Johnson & Johnson’s Janssen Diagnostics, also expressed optimism about the near future of personalized medicine and diagnostics, citing recent studies indicating that almost 50% of products currently in development that are expected to reach the market in 2020 will have a companion diagnostic. As far as when the promise of personalized medical technologies will be fully realized, that "will be a trade off with costs," says Callaerts.

Personalized Medicine in Practice

Of course, personalized medicine isn’t actually new. As then-US Food and Drug Administration (FDA) Commissioner Margaret Hamburg wrote in the introduction to FDA’s 2013 report, Paving the Way for Personalized Medicine, "clinicians have long observed that patients with similar symptoms may have different illnesses, with different causes; and similarly, that medical interventions may work well in some patients with a disease but not in others with apparently the same disease."

What has changed is technology. Recent advances in fields like genomics have sped the pace of development of treatments that can address individual differences like the ones Hamburg referenced. Many companies currently are developing and promoting genetic testing, but has the capability of healthcare providers to use genomic information kept up with the testing technology?

In areas that are fairly well established, such as mutations related to certain types of cancers, genomic data is already being used to determine treatment. However, in other less established areas, the testing is a bit ahead of the utility of the information it provides, but that could change quickly. According to Callaerts, "we have seen a fast uptake of molecular testing in infectious diseases and oncology; other areas will follow soon."

Regulatory and Reimbursement Challenges

Regulators are tracking these developments, and some may be surprised at how savvy agency officials actually are when it comes to personalized medicine, says Kern. But understanding the technology doesn’t alter its inherent challenges. Regulators at FDA, the European Medicines Agency and elsewhere around the world must figure out how to modify regulatory processes that have been used for simpler technologies and diagnostics. Regulatory standards and processes that work well for the old model of blockbuster drugs do not necessarily transfer to rapidly advancing technologies and new treatments that may apply to much smaller patient populations.

How to adapt is the question faced by government agencies, companies and other stakeholders around the world. "I think the challenges for the EU are similar to the challenges we're seeing in other parts of the world," says Kern. "I think when it comes to personalized medicine, in addition to the regulatory challenges, you have to consider reimbursement and demonstrating the clinical utility." Payers need to make sense of highly complex and vague data to determine whether a personalized treatment is likely to yield results. Developers of personalized medicine must prove to both payers and regulators that their products really do add value.

It isn't clear what the future of personalized medicine holds in store, but we're likely to begin to glimpse that future within the next decade or so. Regulators, regulatory professionals and other stakeholders need to examine the key issues now in order to be ready.

Find out more about, or register for, RAPS’ EU Personalised Medicines and Companion Diagnostics Workshop.

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