Personalized Medicine Education and Advocacy

Thought leadership in personalized medicine

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Informed Perspectives: A Preview of the Personalized Medicine & Diagnostics Track at the 2016 BIO International Convention

by Christopher Wells, PMC Communications Director


Christopher Wells

The Biotechnology Innovation Organization (BIO) has a long history of delivering impactful educational sessions about personalized medicine, and PMC is pleased to collaborate with BIO again this year to put together a Personalized Medicine & Diagnostics Track at the BIO International Convention.

Touching on a variety of topics that include clinical care, investment and public policy reform, the three-day program promises to deliver informed perspectives on the field’s current direction:

Tuesday, June 7

Titled “30 Years of Genomics — Bridging the Past With the Future,” the opening session details how the plummeting cost of genomic sequencing is making it possible for an increasing number of health care providers to begin delivering personalized care. We no longer ask whether we can afford to sequence human genomes at scale. Instead, we have begun to examine how we will analyze incredible quantities of genomic data and what that will mean for patients.

A panel of representatives from BioNano Genomics, Domain Associates and Edico Genome will join David Barker, Ph.D., former Chief Scientific Officer of Illumina, to examine these questions during the session, which takes place from 3:30 – 4:30 p.m.

Wednesday, June 8

Some believe widespread genomic sequencing will mean better preventive care for patients. Following introductory remarks from PMC President Edward Abrahams, Ph.D., the second day of the track will begin with an exploration of that idea during a session titled “Lifespan or Healthspan: Is it Time for a Paradigm Shift? An Interview with William N. Hait, Global Head, R & D, Janssen Pharmaceutical Companies of Johnson & Johnson.” Keith Yamamoto, Ph.D., who is the current Vice Chancellor for Science Policy and Strategy and the Vice Dean for Research in the School of Medicine at the University of California San Francisco (UCSF), will lead the discussion with Dr. Hait from 10:45 a.m. – 12:00 p.m.

But as PMC Executive Vice President Amy M. Miller, Ph.D., argues, the improved treatment and better preventive care to be discussed during that session can only be realized if public policies support personalized medicine. After lunch, she will lead a discussion with an industry expert and former Congressional staffers from both sides of the aisle regarding how recent legislative proposals may pave the way for the field. The session, called “Building Personalized Medicine Policy: The 21st Century Cures and Healthier Americans Legislative Proposals,” will take place from 1:00 – 2:00 p.m.

If successful, the kinds of public policies PMC champions will open the door to a future in which preventive care and improved treatment help lower systemic costs. Informed by that possibility, experts from JAWBONE, Johnson & Johnson, Simpatica Medicine and Sutter Health will then take another look at preventive care with a more concentrated focus on its systemic impact during a session titled “Personalized Health as a Potential Enabler of Personalized Medicine,” which will take place from 2:15 – 3:15 p.m.

Finally, informed by an understanding that industry stakeholders are invested in a personalized health care system, a panel of innovation experts from Oberland Capital, Roche, Helix, Renwick Capital, Parthenon-EY and GE Ventures will analyze which products and services in development may hold the greatest promise for advancing that kind of future during a session titled “Strategic Investment in Precision Medicine: Where to Put Your ‘Omics Dollars, NOW,” which will take place from 3:30 – 4:30 p.m.

The second day of the track will conclude with a cocktail reception co-organized with the leaders of the Digital Health Track.

Thursday, June 9

In recognition of the synergies between personalized medicine and digital health, the track’s final day will open with an important examination of the similarities between how evidence supporting both fields is evaluated. The session, titled “What Goes Around Comes Around: Applying Lessons Learned from Personalized Medicine on Evidence and Payer Coverage Requirements to Digital Health,” will take place from 9:00 – 10:15 a.m. It will draw on the perspectives of leaders from Evidation Health, GE Ventures, Quorum Consulting and UCSF to highlight the paths forward for both fields.

Wherever those paths may lead, there is widespread agreement that traversing them will require collaboration. In recognition of that opportunity, the track’s final session, titled “Companion Diagnostics: The Evolving Need for Progressive Partnerships Between Pharma and Diagnostics Companies,” will explore the value proposition for partnerships from both the pharmaceutical and diagnostic company perspectives. Senior leaders from Biodesix, Gritstone Oncology, Health Advances and Janssen Diagnostics will all take part in the discussion, which will run from 10:30 – 11:30 a.m.

As the personalized medicine community continues to navigate toward a paradigm shift in health care, PMC looks forward to discussing these and many other topics in San Francisco.

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Of Moonshots and Precision Medicine: Why the Time is Ripe for a National Cancer Moonshot

by Kenna R. Mills Shaw, Ph.D., Executive Director, Sheikh Khalifa Bin Zayed Al Nahyan Institute for Personalized Cancer Therapy, MD Anderson Cancer Center


Kenna R. Mills Shaw, Ph.D.

Vice President Joe Biden’s leadership of a national “cancer moonshot” is well-timed both to take advantage of and to enhance the hard-won capabilities of the nation’s cancer-fighting community.

Its proposed strategic investments in precision medicine, cancer immunotherapy, early detection, prevention, genomic analysis, data-sharing and collaboration can have a life-saving impact.

New approaches, based on decades of research, help patients now. The Wall Street Journal recently profiled the experience of a 23-year-old man struck with a particularly hard-to-treat form of acute myeloid leukemia (AML).

The story relates how genomic analysis of his leukemia led to targeted therapy that corralled his disease enough to permit a blood stem cell transplant. When his leukemia returned, his oncologists tried a series of treatments until another genomic analysis pointed to yet another targeted therapy. The result: remission, another stem cell transplant and now a year free of AML.

Such a relay race, moving from one therapy to the next to stay ahead of the disease, has been a hallmark of the treatment of advanced cancers for years. With the tools we have in hand now, we’re poised to ensure that more of these races than ever end with a victory.

How do we do better?

A national cancer moonshot can provide pivotal support and encouragement for focused efforts underway at comprehensive cancer centers and other institutions.

For precision medicine to flourish will take a coordinated research effort to truly understand the impact of genomic and molecular variations in cancer. About 120 genes out of the 21,000 in the human genome have variations that are actionable for cancer treatment. However, we find many more variations when we analyze tumors and most of the time we don’t know what they do.

One hindrance is the common assumption that all variations in a gene do the same thing — result in activation or inactivation of a molecular pathway. Not all mutations are created equally.

For example, a clinical trial underway at MD Anderson features a drug that targets a specific fusion alteration in a gene. Other variations in that same gene don’t necessarily cause malignancy or can render even therapy targeted at the fusion ineffective.

So it’s critical to combine genomic analysis with research in functional genomics, using patient-derived xenograft animal models and cell lines, and discovery genomics. We need to combine research data with clinical data, including treatment response, and to have this not just in a subset of patients, but in all patients.

It’s critical to use our combined research and clinical information in the context of tumor evolution during treatment. We need to change the paradigm of when we test patients’ tumors so it occurs closer to when they need to switch to a new therapy than is routinely the case now.

Multiple biopsies — be they liquid gathered from circulating biomarkers or through fine-needle or core needle approaches — will be needed to guide treatment, and are generally not covered by insurers now.

When MD Anderson established its Moon Shots Program in 2012, 10 platforms were established to support our effort to accelerate the pace of converting scientific discoveries into life-saving advances in treatment, prevention and early detection.

These platforms systematically and efficiently provide expertise, technological capacity and infrastructure to the “moon shots,” multidisciplinary teams of experts that focus on 12 cancer types.

Our areas of emphasis overlap with the priorities chosen for the national cancer moonshot. We’re connecting our clinicians, basic scientists, and professional drug discovery and development teams for innovative translational research and building a powerful infrastructure to combine, share and learn from clinical and research data.

All of this is coming together in innovative clinical trials and practice-changing advances.

The time is ripe for a national cancer moonshot to accelerate the progress that is underway now in institutions across the country.

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The Danger in a Demo: How CMS’ Medicare Part B Proposal Could Impede Patient Access to Personalized Medicine

by Amy M. Miller, Ph.D., PMC Executive Vice President


Amy M. Miller, Ph.D.

Today, PMC sent a letter to the Centers for Medicare and Medicaid Services (CMS) about their proposal to test new prices for drugs administered under Medicare Part B — a proposal that was developed without input from those who are most directly impacted: patients and physicians.

PMC has a long history of pointing out the unintended consequences of policies under consideration. This one is no different. The plan’s structure fails to account for the fact that one drug is not always equal to another when we are talking about targeted therapy and personalized medicines, which often represent extraordinary medical breakthroughs. These therapies can have an inordinate impact on patient outcomes. Furthermore, because they are only prescribed to the patient populations known to benefit from them, they may help us make better use of our health care dollars in the long term. As such, we should encourage the use of targeted therapies.

In contrast, CMS’ proposal would systematically disadvantage personalized medicine by burdening the most innovative new treatments with the deepest payment cuts. Hospitals and physicians’ offices that cannot adequately offset the cuts may be forced to stop prescribing these therapies to patients. That is a step backward not only for the field, but for medicine itself.

It should be noted that for the past 20 years, personalized medicines were novel — but that is no longer true. Over the past two years and into the foreseeable future, personalized medicines account for about 25% of the new drugs that come to market. This statistic does not account for label changes to existing drugs that indicate better use of them through innovative diagnostics. For patients to benefit from these significant and revolutionary advances in science, we must ensure that our coverage and payment policies support the use of these products and that researchers continue to investigate them.

In other words, for scientific advancement to best serve patient care, we must get policies like this one right.

We urge CMS to carefully design a program that supports innovative, targeted therapies. Personalized medicines treat diseases more effectively. They can improve both quality of life and survival rates. We welcome the opportunity to work with policymakers on this question and suggest that other stakeholders in personalized medicine engage in this conversation as well.

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Challenges and Opportunities in an Era of Personalized Medicine

by Stephen J. Ubl, president & CEO, PhRMA


Stephen J. Ubl

We are at a pivotal time in health care in America. Biomedical discovery and innovation have the potential to revolutionize the way we diagnose and treat patients. At the same time, rapidly changing regulatory and market dynamics are shifting the ways we develop and pay for new technologies. Personalized medicine stands right at the center of this revolution, with the science enabling greater precision that not only can improve the lives of patients, but can also create efficiencies within the health care system by delivering the right treatment to the right patient at the right time.

We are at a unique juncture in this drive toward personalized medicine. We applaud Congressional focus on biomedical innovation to accelerate new treatments and cures for patients, as seen in the 21st Century Cures Act and in the Senate Health, Education, Labor and Pensions Committee’s ongoing effort to spur medical innovation and drive new research paradigms. We are energized by both the Precision Medicine Initiative and the National Cancer Moonshot, where we see stakeholders from across the health care community coming together to hasten progress for patients.

The biopharmaceutical industry has long been committed to advancing personalized medicine. Last year, more than a quarter of novel new drugs approved by the U.S. Food and Drug Administration were personalized medicines, offering patients important new treatment options across a number of diseases. In cystic fibrosis, a new era of personalized treatment is enabling patients to target the underlying genetic cause of their disease, where previously they were only able to treat symptoms. Oncology is on the leading edge of personalized medicine, with cancer patients seeing a four-fold increase in their targeted treatment options in just 10 years. Five-year survival rates have tripled for patients with chronic myelogenous leukemia since the introduction of the first targeted therapy for this devastating blood cancer, transforming a deadly disease into one in which patients live close to normal life spans.

The great news for patients is that biopharmaceutical companies are just getting started. Today, a full 42 percent of medicines in development are personalized medicines and 73 percent of oncology medicines in development are potential personalized medicines.

Private and public sector collaborations and the ability to maintain an ecosystem that supports and encourages innovation are key to driving continued progress in personalized medicine. As the health care market continues to evolve, we need to make sure we have policies that deliver innovative treatments to patients.

I look forward to discussing both the challenges and opportunities we are facing in this new era of personalized medicine on May 25 at the Personalized Medicine Coalition’s 12th Annual State of Personalized Medicine Luncheon. I hope you’ll join me.

Register for the luncheon here.