Capitalizing on the Promise of Precision Medicine

The Personalized Medicine Coalition (PMC) is pleased that the President has announced the launch of a new Precision Medicine Initiative during his State of the Union Address. Personalized medicine exemplifies the potential of emerging science to improve patient outcomes and support efficient health delivery. We hope that this new effort will renew focus on policies that can help accelerate advances in targeted treatment, all the way from discovery though delivery.

As President Obama said during Tuesday night’s address, personalized medicine has reversed diseases once thought to be unstoppable and as a nation, we need to do more to harness the benefits of such innovative treatment advances.  The President stated, “I want the country that eliminated polio and mapped the human genome to lead a new era of medicine — one that delivers the right treatment at the right time.”

In order to reap the rewards of our federal investment in the human genome, innovative reforms will be necessary throughout the development process – all the way from discovery through delivery and payment for health care. Focusing on any one aspect of this complicated continuum is not enough. We have worked with stakeholders to identify policies that will accelerate these advances such as increased coordination across FDA in the review of personalized medicine products, appropriate reimbursement for the diagnostic tests that enable the use of targeted therapies and incentives for their development.

PMC has been working to secure these reforms and more in Congress through the bi-partisan 21st Century Cures Initiative and with the broader personalized medicine community to educate key stakeholders about the advances in science that are leading us to this new era in medicine. We look forward to working with both the Administration and Congress to accelerate innovation in support of better patient care, and deliver on the promise of personalized medicine.

2014: The Year of the Patient

As we reach the end of another year, we once again look back at recent advancements and milestones in the field of personalized medicine. As we celebrated 10 years of progress, we also looked toward the future, identifying changes needed to ensure another decade of discovery. Reflecting upon the highlights of 2014, it is clear that this truly was the year of the patient. A renewed sense of urgency to shift towards a more patient-centered approach to care has been created across the healthcare system.

The following captures highlights from The Age of Personalized Medicine Blog for 2014.

Michael Kolodziej, M.D., National Medical Director for Oncology, Office of the Chief Medical Officer, Aetna, kicked off the year with reflections on the challenges facing the adoption of personalized medicine.

So what are the practical challenges? There are many. And many share the underlying theme that the old paradigms do not work so well. … Perhaps the biggest challenges lie in the area of clinical utility, which impacts providers, payers and regulatory agencies. Patients are impacted in a huge way. Most people have an idea of where we need to go, but we have a shortage of ideas about how to get there. Finally, all of this occurs in the setting of unsustainable growth in health care spending, and the near uniform agreement that we need to spend our money in a more intelligent, impactful way. … We have a lot of work to do together.

In March, we were honored to share the personal and candid story of Stephanie Dunn Haney, a lung cancer survivor, and her experience with molecular testing and targeted therapies. Stephanie’s story continues to remind us of the hope personalized medicine offers to so many.

Molecular testing and personalized medicine gave me my life back, and my sense of a future back. While I’m realistic enough to know that my daughters are fairly certain to lose their mother before they are grown, I also know I have tools to fight with, and a responsibility to share my story.

As we talk about the need to keep the patient at the center of all that we do, we at the Personalized Medicine Coalition (PMC) saw a need to establish a baseline of consumer awareness, knowledge and attitudes about personalized medicine. In July, PMC released a national survey, U.S. Public Opinion About Personalized Medicine, with key findings that will guide future outreach and education efforts.

We’re at the beginning of the golden age of personalized medicine. Armed now with a clear picture of the public’s opinion, we have an opportunity to raise awareness and increase understanding of what personalized medicine is, and how it can transform approaches to healthcare delivery.

Of course, in order to bring molecular tests and targeted therapies to patients, key regulatory and reimbursement areas must be addressed. PMC also published The Future of Coverage and Payment for Personalized Medicine Diagnostics in July, which took a critical look at CMS policies, highlighting specific challenges to the further implementation of personalized medicine diagnostics.  Later that month, the U.S. Food and Drug Administration’s (FDA) released its long-awaited final guidance on the regulation of companion diagnostic devices, as well as proposed framework for regulating laboratory developed tests (LDTs).

Investors have long argued that clarity is necessary in both regulation and reimbursement for continued advancement of personalized medicine. We now have clarity on FDA’s current thinking although many issues remain unresolved. The community has time to consider this framework and may soon have a chance to provide public comments. And finally, the pharmaceutical industry has the FDA’s assurance that targeted treatments will not be held up by co-development challenges.

In October 2014, the PMC, the American Association for Cancer Research (AACR), and Feinstein Kean Healthcare (FKH) convened the second national Turning the Tide Against Cancer national conference, which brought together leaders throughout the healthcare and policy communities for a passionate and engaging discussion on the importance of moving towards a more high-value, patient-centric system of cancer care.

Keynote speaker, cancer survivor, and The New York Times Emmy® Award winning columnist of “Life, Interrupted,” Suleika Jaouad, shared insights into communication challenges patients face during a Q&A session. Suleika’s words serve as a reminder that if we are to increase adoption of personalized healthcare, we must ensure patients are given the tools and education needed to properly understand their treatment options.

Communication is the golden ticket. We live in the WebMD age where patients often consult Google before they consult a doctor. This can be dangerous and can lead to misinformation and misunderstandings. Creating an environment where the patient feels comfortable asking questions and talking to their medical team is crucial.

Following the conference, FKH Chairman, Marcia A. Kean, M.B.A., proposed next steps:

I propose that every individual touched by cancer, and every organization concerned about the nation’s cancer burden, take responsibility for three actions.

1. Review the Issue Brief, and share your thoughts/ideas about the policy options and/or propose other options
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2. Involve your organization in the Turning the Tide Against Cancer Through Sustained Medical Innovation initiative, by participating in our activities and events.

3. Join our partners PMC and AACR and advocate for those options that you agree with, integrating them into your own policy platforms and your communications with policymakers in order to drive momentum and catalyze change.

We encourage you to learn more about the Turning the Tide Against Cancer initiative, and the important work we are doing to make a difference for cancer patients.

As an appropriate end to the year, we celebrated the 10th Anniversary Personalized Medicine Conference in November, and conference organizer Raju Kucherlapati, Ph.D., Paul C. Cabot Professor of Genetics, Harvard Medical School, reflected on a decade of developments in personalized medicine.

The past decade has witnessed many exciting new developments in personalized medicine: the significant reduction in the cost of DNA sequencing and related technologies; the use of these technologies for an unprecedented rate of discovery of human disease genes; a near universal acceptance of the importance of genetics and genomics in drug development, especially for cancer; the levels of investment in personalized medicine companies; recognition of the importance of personalized medicine by professional societies; and the deep involvement by the administrative and legislative bodies in the U.S. and throughout the world.

2014 was a milestone year. We look forward to the year ahead, and the continued opportunity to engage with leaders throughout the personalized medicine community, and across the healthcare system, to discuss the future of personalized healthcare and how we can provide the best value to patients.

Next Steps To Turn the Tide Against Cancer

Three weeks ago, 200+ health care leaders representing a broad spectrum of stakeholders convened in Washington, D.C., for the second conference of the Turning the Tide Against Cancer Through Sustained Medical Innovation initiative. There were intense discussions — among panelists and in the hallways between sessions — on the challenges facing cancer research and care.

Now back at our desks, the question arises, what do we want to do next — as individuals, as a community, and as a nation? And, critically, what must we do next to enable and encourage innovation in cancer research?

In 2011, Feinstein Kean Healthcare joined forces with the Personalized Medicine Coalition (PMC) and the American Association for Cancer Research (AACR) to create the Turning the Tide Against Cancer initiative, in response to disconnects within the health care system that threatened to stifle advances, ironically at an unprecedented moment of scientific progress.

We saw disconnects between patients and the health care system…disconnects between the accelerating pace of science and the slowness of change in our policy environment…and disconnects between the exigencies of driving innovation and the sober realities of a cost-contained era.

As we observed these disconnects, we also noted that as a nation we were attempting to solve complex problems while operating within traditional vertical silos (e.g., patients in patient advocacy organizations; physicians in physician professional societies; researchers in scientific societies) rather than cross-sector collaborations.

And so, we sought to catalyze the entire cancer and health policy community towards action by connecting all stakeholders through the Turning the Tide Against Cancer initiative. Uniquely, stakeholders from across the cancer ecosystem have been represented in all events and deliberations of the initiative.

Now, building upon several years of intellectual exploration, research, dialogue, debate and the deliberations of the broad range of experts that produced the Issue Brief, “A Pathway for Change: Supporting the Shift to Patient-Centered Cancer Research and Care and Addressing Value and Cost of Cancer Care” released at the conference, we ask again, what’s next?

I propose that every individual touched by cancer, and every organization concerned about the nation’s cancer burden, take responsibility for three actions.

1. Review the Issue Brief, and share your thoughts/ideas about the policy options and/or propose other options.
2. Involve your organization in the Turning the Tide Against Cancer Through Sustained Medical Innovation initiative, by participating in our activities and events.
3. Join our partners PMC and AACR and advocate for those options that you agree with, integrating them into your own policy platforms and your communications with policymakers in order to drive momentum and catalyze change.

In the past, individual champions such as the renowned Mary Lasker were a driving force to advance and change our approach to cancer research and care. As Dr. Len Lichtenfeld of the American Cancer Society asked following the first Turning the Tide Against Cancer conference, “Where is our Mary Lasker going to come from? …Who is going to guide this revolution?”

I believe that in this era, we are all destined and empowered to be leaders. And as I looked out at the attendees of the Turning the Tide Against Cancer conference, I saw Mary Lasker in the face of each participant. We are all Mary Lasker. We must all be Mary Lasker.

Please join us as we take the next step.

The History and Future of Personalized Medicine

Many of us in the genetics and genomics community think that this a golden age for our work. During the last thirty years or so, it has become apparent that genetics plays a very important role in virtually all aspects of human health and disease.

The completion of the human genome sequence at the beginning of this century promised that the use of genetic and genomic tools in understanding the basis for disease and in providing novel approaches to care would become available. It was anticipated that genetic and genomic testing would allow accurate diagnosis of disease, determine the prognosis for the patients with disease, and help physicians make the most optimal choices about how to treat their patients.

This promise launched the era of Personalized Medicine. Several academic institutions embraced this concept. In Boston, Harvard Medical School and Partners HealthCare (the parent organization for several major hospitals in Massachusetts including the Brigham and Women’s Hospital and the Massachusetts General Hospital) launched a new center designated the Harvard Partners Center for Genetics and Genomics (HPCGG), and I had the privilege of being its first Scientific Director.

HPCGG wished to promote personalized medicine and decided that one way to accomplish that goal was to provide a forum for review of the advances in personalized medicine, in all of its facets, and to discuss ways in which the field can be advanced and have an impact on patient care. This vision was shared by a few other organizations including Edward Abrahams of the Personalized Medicine Coalition and Marcia Kean of Feinstein Kean Healthcare. Together we launched the annual Personalized Medicine Conference.

We have always felt that to advance personalized medicine, business had to embrace the concept and find value in investing in this enterprise. To promote that goal, we were joined by Regi Herzlinger, Richard Hamermesh and their many colleagues at Harvard Business School.

In 2014, we are celebrating the tenth anniversary of the Conference as well as the anniversary of the birth of the Personalized Medicine Coalition. The past decade has witnessed many exciting new developments in personalized medicine: the significant reduction in the cost of DNA sequencing and related technologies; the use of these technologies for an unprecedented rate of discovery of human disease genes; a near universal acceptance of the importance of genetics and genomics in drug development, especially for cancer; the levels of investment in personalized medicine companies; recognition of the importance of personalized medicine by professional societies; and the deep involvement by the administrative and legislative bodies in the U.S. and throughout the world.

There have been exciting moments such as the passage of the Genetic Information Non-discrimination Act; the successful launch and execution of whole exome and whole genome sequencing to understand diseases such as cancer and several childhood disorders of unknown etiology; and the development of novel drugs and therapies based on the genetic constitution of individuals or tumors. There are frustrations around the lack of certainty about regulation and reimbursement — but such is progress!

The tenth anniversary of the Personalized Medicine Conference, to be held on the campus of Harvard Medical School November 12-13, 2014, will again bring together leaders from many different areas of personalized medicine and promises to provide a lively forum for exchange of ideas. I personally welcome the opportunity to again host this meeting in November and look forward to seeing you and greeting you there.

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The Personalized Medicine Conference is an annual two-day event co-hosted and presented by Partners HealthCare Personalized Medicine, Harvard Business School, and Harvard Medical School in association with the American Association for Cancer Research and Personalized Medicine Coalition. 

For more information and to register for the 10th Annual Personalized Medicine Conference, please visit http://www.personalizedmedicineconference.org.

Turning the Tide Against Cancer: Patient-Centered Care and Value

On October 9, the Personalized Medicine Coalition, the American Association for Cancer Research, and Feinstein Kean Healthcare will be convening the second Turning the Tide Against Cancer Through Sustained Medical Innovation national conference in Washington, D.C., to continue the ongoing dialogue which began at the initiative’s inaugural conference in June 2012.

This year’s conference will bring together leaders from across the cancer community to help identify specific policy solutions to the challenges of supporting the shift to patient-centered research and care and addressing the value and cost of cancer care — two key themes that have emerged through the initiative’s ongoing work.

The Age of Personalized Medicine editorial team sat down with each of the initiative co-conveners to talk about the current cancer research and care landscape,  the upcoming conference, and what progress has been made since the start of the Turning the Tide Against Cancer initiative in 2011.

Our conversation with Edward Abrahams Ph.D., president of the Personalized Medicine Coalition can be viewed below. Stay tuned for additional video interviews with Margaret Foti, Ph.D., M.D. (h.c.), chief executive officer of the American Association for Cancer Research and Marcia A. Kean, M.B.A., chairman of Feinstein Kean Healthcare.

Visit the Turning the Tide Against Cancer website to register for the conference and learn more about ways you or your organization can support the ongoing initiative. The Age of Personalized Medicine will also be tweeting live from the conference on October 9.

Survey Reveals Insights About Awareness, Understanding of Personalized Medicine, Part 2

Following the launch of the Personalized Medicine Coalition’s U.S. Public Opinion About Personalized Medicine survey results, the Pharmaceutical Research and Manufacturers of America’s (PhRMA) asked the panelists from our launch event at the National Press Club – What key benefits of personalized medicine do you think the public needs to know about in order to embrace this approach to health care?

The survey, conducted by KRC Research, tells us that most Americans do not know what personalized medicine is, but once the concept is explained to them they are very supportive of advancing the field. In these short video interviews, each of these experts weighs in on how we connect the dots from lack of knowledge to wide support for personalized medicine.

The full set of video responses can be viewed on PhRMA’s Conversations blog, with additional commentary from Raju Kucherlapati, M.D., Professor, Department of Genetics at Harvard Medical School and Mark Richards, Senior Vice President, Management Supervisor at KRC Research.

Amy M. Miller, Ph.D., Executive Vice President, Personalized Medicine Coalition, discusses how personalized medicine is changing the way we experience health care today, including the development of the first cystic fibrosis treatment in over 20 years and other medicines that are improving patients’ quality of life.

Donna R. Cryer, J.D., President and CEO, Global Liver Institute, talks about the importance of educating both patients and clinicians about personalized medicine and its potential benefit, as well as her personal experience as a patient who has benefited from targeted treatments.

Randy Burkholder, Vice President, Policy, PhRMA, highlights the important future of personalized medicine and the commitment of America’s biopharmaceutical research companies to advancing the field and the science of personalized medicine.

Learn more about U.S. Public Opinion About Personalized Medicine and review the survey findings by reading Part 1 of our series or visiting the PMC website.

Survey Reveals Insights About Awareness, Understanding of Personalized Medicine, Part 1

Earlier this year, the Personalized Medicine Coalition (PMC) commissioned U.S. Public Opinion About Personalized Medicine, a nationally representative survey of 1,024 adults gauging consumer awareness, knowledge and attitudes about personalized medicine.

The key findings of the survey were positive. Although less than 4 in 10 Americans had heard of it prior to being surveyed, respondents are interested in learning more about personalized medicine and are supportive of the concept. For those who had heard of personalized medicine, their knowledge was shallow and did not associate the term with diagnostic testing or targeted treatments. This highlights the need for education to a willing public.

When the surveyed individuals were given a definition of personalized medicine, approximately two-thirds were receptive and expressed positive opinions about its prospects.

Respondents easily pointed out the major benefits of personalized medicine, noting that it could give them more knowledge to prevent or treat their illness, help them choose the most effective treatments with their doctors, and lead to a decline in unnecessary treatments, side effects, invasive procedures and trial and error medicine.

Many of the individuals who were surveyed even raised questions, specifically regarding the efficacy, potential risks, cost, access and affordability. A majority agreed that insurance should cover personalized medicine if it is recommended by a doctor.

We’re at the beginning of the golden age of personalized medicine. Armed now with a clear picture of the public’s opinion, we have an opportunity to raise awareness and increase understanding of what personalized medicine is, and how it can transform approaches to healthcare delivery.

Stay tuned for part two of this blog post series as we hear from leading experts, and the researcher who conducted the survey, on what key benefits of personalized medicine they think the public needs to know about in order to embrace this approach to healthcare.

In the meantime, learn more about U.S. Public Opinion About Personalized Medicine and review the survey findings by viewing the slideshow below or visiting the PMC website.

Making the Case for Personalized Medicine

As lawmakers, industry, consumer groups and the general public debate the future of health care, personalized medicine continues to focus attention-with good reason. In a time of unprecedented scientific breakthroughs and technological advancements, personalized health care has the capacity to detect the onset of disease at its earliest stages, preempt the progression of disease, and, at the same time, increase the efficiency of the health care system by improving quality, accessibility, and affordability.

With that in mind, I’m excited to announce the release of the fourth edition of Personalized Medicine Coalition’s signature document, The Case for Personalized Medicine, which notes the steady development of the field. Most notably, in 2006, there were 13 prominent examples of personalized drugs, treatments and diagnostics on the market. In 2011, there were 72, and today there are 113-a 57 percent increase in the last three years.

Along with this steady growth, we also examine opportunities for the continued development and adoption of personalized medicine as several factors come into play: the cost of genetic sequencing declines, the pharmaceutical industry increases its commitment to personalized treatment, and the public policy landscape evolves. While momentum is building, much remains to be done to keep up with the evolving developments in science and technology, according to the report. With all this progress comes a greater responsibility to actually help patients by advancing personalized medicine to make it standard practice in the near future.

According to The Case, personalized medicine can accomplish the following:

• Shift the emphasis in medicine from reaction to prevention
• Direct the selection of optimal therapy and reduce trial-and-error prescribing
• Help avoid adverse drug reactions
• Increase patient adherence to treatment
• Improve quality of life
• Reveal additional or alternative uses for medicines and drug candidates
• Help control the overall cost of health care

But to advance personalized medicine, we need intelligent coverage and payment policies, clearer regulatory guidelines, and modernized professional education to prepare the next generation of doctors and other health care professionals for personalized medicine.

PMC is pleased to partner with BIO and release this report at the Personalized Medicine & Diagnostics Forum at this year’s meeting. The Forum takes place next Wednesday, June 25 at 9 a.m. PT in room 7AB and will explore many of the salient issues facing personalized medicine, as discussed in The Case. If you’re onsite at BIO this year, come check out the forum. We’d love to hear your thoughts on the current personalized medicine landscape and what’s to come.

The new edition of The Case for Personalized Medicine will be available online June 25 at 9 a.m. ET, but in the meantime, check out PMC’s other publications here.

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This blog post originally appeared in BIOtechNow, the blog of the Biotechnology Industry Organization (BIO).

 

Examining the Relationship Between Payment Policies and Personalized Medicine

In an era of increased focus on containing rising healthcare costs, personalized medicine represents an important solution in addressing these concerns, while improving the quality of patient care. In order to ensure that these important advances continue, it is essential to ensure payment policy supports personalized medicine.

Much of PMC’s policy work in the coming year will focus on both the challenges and opportunities in Medicare payment policy. For several years, ongoing efforts to reduce reimbursement for advanced diagnostic tests have been at the leading edge of this work. Recent proposals to cut reimbursement for diagnostics and drugs in Medicare Part B underscore the importance of our continued focus in this area.

Building on the success of our reimbursement issues brief from 2010, we will soon publish an updated brief outlining the difficulties facing personalized medicine in coverage and reimbursement decisions. This paper will cover coding changes as well as the challenges that CMS has placed on innovators. It will also outline evidence issues and how the field might consider tackling them.

In addition, PMC will address how emerging, alternative payment models (such as medical homes, accountable care organizations, and pathway- or episode-based payment) also will have a significant impact on development and adoption of personalized medicine. While some of these approaches appear to hold promise, we must pay attention to the details to ensure that they are designed in ways that allow physicians to tailor care based on an individual’s genetics and other factors, and that support adoption of novel targeted therapies.

PMC already has begun engaging on this important set of issues. Based on our policy principles for deficit reduction, we are developing additional messaging and conducting further analysis to engage Centers for Medicare and Medicaid Services (CMS) throughout the year. Our next opportunity will be on April 10 when we will respond to a request for comments on Specialty Practitioner Payment Models. In our response, we will encourage CMS to see targeting drugs with diagnostic tools as a way to increase the quality of patient care and to ensure that policies encourage this direction.

As we engage on a growing range of important policy issues, it is timely that Dr. Patrick Conway, the acting director of the Center for Medicare and Medicaid Innovation (CMMI), will be joining us this week at our Tenth Annual State of Personalized Medicine Luncheon. CMMI has responsibility for testing and implementing alternative payment models at CMS. We look forward to hearing from Dr. Conway as he shares his vision of personalized medicine, CMS’ perspective of innovation and quality health care reform, and answers questions about developing payment policies.

We recognize that personalized medicine depends on our ability to pay for both diagnostic and therapeutic products, and to that end, the PMC is focused on advocating for public policies that support personalized medicine’s development through scientific, regulatory, and reimbursement-related activities. I invite you to join PMC at the Tenth Annual State of Personalized Medicine Luncheon Address at the National Press Club in Washington, D.C., on March 13.

A Look at the Regulation of Diagnostics

Participants at the upcoming Personalized Medicine World Conference 2014 (PMWC) will be engaging in thoughtful debate and discussion on some of the biggest topics in the field of personalized medicine. I anticipate a lively discussion with Andrew Fish, Executive Director of AdvaMedDx on the topic of regulatory issues in molecular diagnostics.

The regulation of diagnostic products is one of the most contentious issues within the personalized medicine community today. Regulatory issues have led to confusion and uncertainty in the industry due to the involvement of multiple agencies with varying standards. Consensus on solutions among kit manufactures and laboratory developed test companies has been hard to come by.

At the Personalized Medicine Coalition (PMC), we have heard from some who would prefer that the status quo is maintained; however, PMC contends that the status quo is not an option. While our members may not agree upon the exact course of action, it is time to acknowledge that action is needed to build a consensus around the development of an efficient, cost-effective process for bringing safe, high quality diagnostic tests to market in which patients, physicians, and payers can have confidence.

To initiate this process, PMC published Personalized Medicine Regulation: Pathways for Oversight of Diagnostics. The paper established baseline knowledge of the status of diagnostic regulation, and set the groundwork for future collaboration among industry, government, and other organizations.

To learn more about this important issue, with a look at the complex and diverse perspectives from key stakeholders, as well as to explore potential solutions, join me on January 28 at the PMWC 2014. Our discussion will look at areas of agreement and disagreement regarding the regulation of molecular diagnostics and likely scenarios for the future.

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 Look for additional posts from speakers and participants prior to the 6th Annual Personalized Medicine World Conference on January 27-28, 2014. For more information and the full agenda, visit: 2014sv.pmwcintl.com.