Patients Are at Core of Personalized Medicine Era

Patients Are at Core of Personalized Medicine Era

This week the Patient and Health Advocacy Summit concluded its sixth consecutive year, bringing hundreds of patient advocates and policymakers together to engage on important issues facing the patient community.  This year’s Summit featured a new Personalized Medicine Workshop that offered a unique opportunity for patient groups to engage with other stakeholders to learn more about this emerging field of medicine.  The workshop began with a session on “The Personalized Medicine Landscape,” and included subject matter experts Dr. Daryl Pritchard, Dr. Stephanie Devaney and Dr. Sherri Millis.

Dr. Daryl Pritchard, Vice President for Science Policy at the Personalized Medicine Coalition, provided attendees with an overview of the scientific foundation of personalized medicine.  Dr. Pritchard outlined the regulatory framework for diagnostics as well as the various challenges that lie ahead.  He showed how the uptake of personalized medicines has increased in recent years, as there were only about 14 personalized medicine products in 2005 compared with about 160 in 2015.

We are also seeing medical advancements come to fruition, including the first approval of a gene therapy biologic this year, and the drug development pipeline is currently littered with personalized medicines, representing 42% of all US drug approvals in 2016.  Dr. Pritchard also discussed how the value of personalized medicines are being demonstrated through examples like lymphoma treatments, where the 5-year survival rate has increased 70% over the last 60 years.

Dr. Stephanie Devaney, Deputy Director of the National Institutes of Health’s All of Us Research Program, provided the audience with background on the All of Us program, formerly called the Precision Medicine Initiative, at NIH.  Dr. Devaney explained that the program is part of an overarching effort to “change the way we treat people and match that with the policies we need, and [that] we must acknowledge personalized medicine as a new way of doing healthcare.”  The program now has the backing of Congress with the enactment of the 21st Century Cures Act, which funds the program over the next 10 years with $1.455 billion.

Dr. Devaney emphasized that the All of Us program must overcome many challenges with implementation, such as managing privacy protections for the 1 million individuals donating their health data information.  The program will build digital infrastructure to eventually make the data accessible to the research community, aiming to “catalyze an ecosystem for knowledge generation”.   The program is currently in beta testing with 3,200 participants, but sights are set on a national launch in spring of 2018 where program enrollment will be open to the general public.

Dr. Sherri Millis, Senior Medical Science Liaison for Medical Affairs at Foundation Medicine, spoke about the practical and transformative application of personalized medicine in a business environment.  Foundation Medicine has been a trailblazer in the industry that now offers over 65,000 laboratory test products, most of which are panels, including next generation sequencing (NGS).  These tests provide information that is utilized to profile genes related to certain diseases so biotechnology companies can develop medicines to target those genetic drivers, known as targeted therapies.

Dr. Millis also spoke about a coming paradigm shift in the era of personalized medicine—the parallel review of diagnostic tests—where sponsors will simultaneously seek analytical and clinical validation for tests.  However, reimbursement challenges remain as payers have been slow on the uptake to cover efficacious diagnostics.  And Dr. Millis importantly notes that this is where it “comes down to patients on the front line…telling this story [of how the lack of coverage] has an impact on patients”.  While the biotechnology industry navigates advances in personalized treatments and genomics as well as coverage challenges, there is a palpable appreciation that patients are at the core of getting drugs to market more quickly in this new era of personalized medicine.

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