US can't afford to be left behind in precision medicine 'gold rush'
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Precision medicine is the next step in the medical profession’s 2,500-year quest to individualize medical treatments — and one of the most significant. Often called the next “space race” because of the immense technological advances required to make it a reality, precision medicine allows clinicians to advance beyond the relatively coarse diagnostic and therapeutic categories of the present day, towards a more targeted, fine-grained system, based in part on genetics and molecularly defined phenotypes.

With about 20,000 genes to understand and analyze in the context of phenotypic and clinical information, the next few years will see a land-grab for the intellectual property that these global efforts will produce.  It will be the 21st Century’s gold rush.  But, rather than being contested in mines, it will be contested in research labs, clinics, and patent offices. And without robust federal investment, the United States will be in danger of being left behind.

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To capitalize on existing momentum and advance the United States’ leadership in this sphere, the federal government must modernize several of its policies around data governance and continue federal funding for strategic health initiatives that are critical for progress.  

  

Precision medicine is “an emerging approach for disease treatment and prevention that takes into account individual variability in genes, lifestyle, and environment.”  It is the acme of evidence-based medicine, and as such, requires a massive amount of data. The data isn’t merely sequenced genes, it is the translation of these sequences into disease types and cures.

Our notions of “disease” are being fundamentally revised. What was previously considered one disease may now, with new genetic and molecular phenotyping tools, be split into two, 20, or 200 diseases that differ in ways both great and small.

Industry today has a difficult time recruiting individuals for a clinical trial that addresses a single disease afflicting millions of people. Can anyone imagine how hard it would be to recruit individuals for dozens of diseases that each affect only thousands of people? This is the crux of the challenge for the new era of precision medicine. Targeted therapies warrant targeted validation efforts.

To this end, no single institution can recruit enough patients to accommodate all of these new disease types. Clinical trials rely on having a large number of patients. A trial with 1000 people can answer many more questions with much more certainty than a trial with 10 people. The only way to amass enough patients in a new era of clinical trials is for institutions to share the data they collect from clinical records and research studies in better ways.

This is America’s competitive advantage. No other country has a broad, nationwide, biomedical research establishment like the one the American taxpayer has so generously invested in since the 1950s.  No other country has as much talent and energy in the superb pharmaceutical corporations that have grown up as a result of American capitalism. No other country (for now) has the digital technology to support the immense amounts of data and processing that are and will be required to tease out nature’s secrets from the work that academia and industry will perform.

So, although the United States enters the race today with large advantages that did not spring entirely from the marketplace, other nations see this new era of precision medicine as an opportunity to leapfrog into pre-eminence.

These other nations are working very hard to accomplish this (e.g. Beijing Genomics Institute; France’s $745 million investment to establish a population-scale genome sequencing operation; Estonia’s goal to collect the genetic information of its entire population). That is why federal funding of large-scale genomics programs, such as the National Institutes of Health’s “All of Us” initiative, is essential in maintaining the United States’ lead in healthcare innovation.

The recently passed 21st Century Cures Act included provisions that require data sharing as a part of federally funded projects at the National Institutes of Health. We need to ensure this important shift in “understanding the value of data sharing” is at the forefront of federal research programs.  

Data sharing and funding are critical in generating evidence that enables a consensus regarding the clinical impact of individual genes and associated disease. Centralized databases such as ClinGen are early examples of this approach.  More robust regulatory practices that expand this promising avenue of work, spearheaded by the NIH and FDA, should bring genomic medicines to the patients that need them faster.

The concept of data sharing isn’t confined to the research bench.  For example, the Report of the Cancer Moonshot Task Force specifically cited innovation in payment and care delivery models as a priority for study, with the aim of achieving “a multi-payor model focused on incentivizing high-quality, high-value, patient focused cancer care.”  New forms of analysis, fed by data sharing over longer time horizons, are required to determine the cost effectiveness of genomic based treatments and approaches, as well as their clinical effectiveness.

By some estimates, moving forward on the path towards precision medicine in everyday care will generate 100 million to two billion — if not more — human genome sequences. This requires two to 40 exabytes of data storage (one exabyte = one billion gigabytes).

Only through collaboration and investment can such massive data volumes be generated, managed, and optimally utilized. Policymakers and regulators should work with industry to establish realistic guidelines and investments that encourage innovation and risk-taking, while still emphasizing patient safety and privacy.

We are encouraged by the recent, bipartisan efforts of the last Congress and administration to establish precision medicine and data sharing as key tenets in the evolution of the United States’ healthcare system — a pattern that has continued with Congress’s wise decision to include an additional $2 billion in needed National Institutes of Health (NIH) funding in this week’s omnibus bill. We are still wary, however, that the new Administration’s budget proposal for the coming fiscal year, with its drastic cuts in biomedical research, will have a detrimental impact on progress.

We encourage the new Congress and administration to ensure our historical headway is not lost, and look forward to working together with them for the future of American medicine.

John Sotos, M.D., M.S., is the Chief Medical Officer at Intel.

Steve Rosenberg is the Senior Vice President and General Manager of the Health Sciences Global Business Unit at Oracle.

Morten Sogaard, Ph.D., is the Vice President and Head of Genome Sciences & Technologies at Pfizer.


The views expressed by contributors are their own and are not the views of The Hill.