Regulatory obstacles just one barrier to personalized medicine
By: Vivian Lee, M.D., Ph.D., M.B.A. | Sep 1, 2014 1:00 PM
If past performance is the best indicator of future outcomes, then concerted, national efforts to advance the science of personalized medicine are a good bet.
Recent advances in molecular biology have paved the way for personalized treatments tailored to a particular patient’s genetic makeup. Such treatments promise to improve health care and reduce costs by delivering the right care to the right patient at the right time, a central aim of federal health reform. But turning scientific discoveries into useful therapies is complicated, slow and expensive work.
The “21st Century Cures” initiative of House Energy and Commerce Committee Chairman Rep. Fred Upton, R-Mich. and Rep. Diana DeGette, D-Colo. Proposes to speed drug discovery by modernizing the United States’ outdated drug approval process, a relic of the 1960s. It’s a laudable goal and critical if America is to remain a world leader in the biopharmaceutical space. The current timeframe for bringing a drug to market – 10 years and a billion dollars per drug on average – is not sustainable.
Talk of accelerating innovation through smarter regulation must include support for a robust research pipeline to provide the basis for the development of new drugs.
Such a pipeline requires a well-funded infrastructure for discovery that will enable more therapies to get to the front lines. At a national level, prioritization of initiatives can really make a difference. Take for example the remarkable progress on HIV and AIDS, making what was once a death sentence to a manageable chronic disease, and all within the past three decades. Recently, the President has announced a prioritization on the neurosciences through the BRAIN initiative, which is aimed at revolutionizing our understanding of the human brain and stands to impact health care delivery in ways we can only begin to imagine. With the dramatic progress and insights made through scientific progress of the past decade, the timing of this initiative makes sense. Another top priority should be the field of genomics and personalized medicine.
Building on the Human Genome Project and the advances in genomic science and research that have already made personalized medicine a reality for many individuals and families. This national initiative has saved thousands of lives. At the University of Utah alone, we have used genomics advances to discover more than 30 important disease-causing genes that have prevented thousands from confronting the diseases that impacted their ancestors. Today, for example, those who test positive for mutations in disease-associated genes, including the BRCA1 and BRCA2 genes (breast and ovarian cancer), the APC gene (colon cancer), or for the KCNQ1 (sudden cardiac death), can take preventive measures.
Further discoveries and preventive measures like these also rely on the stewardship of unique American resources like the Utah Population Database. The database is the world’s largest repository of medical and public health records combined with family genealogies, enabling collaborative scientists to more easily pinpoint disease-causing genes. Advances in technology have made whole genome sequencing faster and less expensive, under $1000 in some cases. Add to that the nearby digital revolution that will both rapidly analyze and make portable genomic health data, and we are poised to open a massive pipeline of both diagnostic and therapeutic opportunity. As a reflection of this potential, AmGen acquired the rights to the next-largest database in the world called deCODE, in Iceland, a 500,000+ record resource for $450m.
Personalized therapies, accelerated in a regulatory vacuum, run the risk of churning out high-cost, low-impact benefits. When coupled with a concerted and robust research pipeline fed by strategic public/private partnerships and accessible, shared resources, we could find our way to justifiable returns and 21st century cures.comments powered by Disqus