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.

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.

Ensuring a Personalized Future through Diagnostics Policies

In a time of rapidly rising healthcare costs, personalized medicine offers a more efficient approach to prevention, diagnosis and treatment.

However, special considerations and thoughtful policies that acknowledge the emerging and varied technology (and challenging science) at play are needed in order to ensure access to personalized diagnostics and treatments, while also encouraging further investment and development of technologies. Just as personalized medicine is moving us away from a one-size-fits-all approach to healthcare, we must too move away from one-size-fits-all approaches to reimbursement.

The Personalized Medicine Coalition recently released “The Future of Coverage and Payment for Personalized Medicine Diagnostics.” The white paper takes a critical look at the policies of the Centers for Medicare and Medicaid Services, which in turn inform the decision making process for payers. Three specific challenges to the further implementation of personalized medicine diagnostics are identified:

• Reimbursement levels that ensure access while also encouraging development
• Inconsistent standards for evaluating genomic tests
• A lack of incentives for genomic medicine

As we continue our journey in the post-ACA healthcare landscape, the need for objective, reliable and clearly defined standards for evaluation and reimbursement continue to grow. Uncertainty – especially in the form of payment – does not inspire investment or fuel growth through R&D.

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).

 

Prescribe by Price, But at What Cost to Patients?

In recent months, there has been increased discussion about the cost and value of cancer care. These conversations are important, but it’s crucial to make sure the patient’s unique perspective on value isn’t lost in the mix. We must also be sure we are considering each patient’s unique diagnosis, as well as the heterogeneity of their disease.

It is clear that targeted therapies play an important role in delivering personalized care to patients, and in particular, those with many forms of cancer. A recent article in Newsweek profiled a patient who, through a new tumor identification method, was able to receive a highly-targeted treatment for his sarcoma. As noted in the article, “the slow and steady march of innovation in our understanding of cancer and the emergence of companies providing personalized treatments have not only saved Rose’s life but allowed him to turn his cancer from a death sentence into a chronic illness that simply needs regular managing.”

These stories are becoming more common, yet as new targeted treatments work through the regulatory pathways, we are faced with ongoing questions: How do we ensure patients have access to these life-saving drugs and the diagnostics used to select them? And how do we ensure the system incentivizes research and development broadly, so more people benefit from personalized medicine?

Recently, the American Society of Clinical Oncology (ASCO) announced that it will be developing a scorecard, slated to come out this fall, to evaluate cancer drugs based on their cost and value, as well as their efficacy and side effects. This signifies a potential shift in the way that physicians make decisions about their patients’ health, encouraging them to consider the financial implications of interventions alongside patients’ needs. As this framework is developed, it will be important to ensure that the full value of innovative interventions is recognized.

Scientific advances have the potential to provide earlier and better diagnoses, more effective treatments and even possible cures for patients. Personalized approaches to care could provide a patient with more or better quality time to spend with their family or friends, and overall greater long-term value.

As Edward Abrahams, president of the Personalized Medicine Coalition told The Washington Post, these targeted treatments also bring a greater long-term value to the healthcare system as a whole by cutting down the frequency of unnecessary treatments and procedures, and avoiding unnecessary side effects.

Still our continued focus must be on providing high-value care and creating a system that improves patient outcomes and does not place the focus on prescribing drugs based on cost or financial incentives. The Personalized Medicine Coalition, along with the American Association of Cancer Research and Feinstein Kean Healthcare, continues to engage the cancer community to address these questions of cost and value through the Turning the Tide Against Cancer initiative.

Through ongoing dialogue and discussion as part of this initiative, two key themes continually resurface as components that should be considered when defining value: advancing approaches that are patient-centered, and developing new approaches to value assessment that align with and incentivize scientific progress and innovation.

On October 9, 2014, the Personalized Medicine Coalition, the American Association of Cancer Research and Feinstein Kean Healthcare will re-convene the cancer community in Washington, D.C., for a second Turning the Tide Against Cancer national conference to advance the conversation on the value and cost, while supporting a shift to patient-centered cancer research and care.

Collaboration Key to Improved Reimbursement Policies for Personalized Medicine

Significant reimbursement challenges to personalized medicine began in 2012 when the Centers for Medicare and Medicaid Services (CMS) changed payments for diagnostics from a stack of different types of codes describing different parts of lab testing to a unique code for a type of test.

The Personalized Medicine Coalition (PMC) has engaged with CMS on reimbursement issues for personalized therapies and companion diagnostics in a variety of ways, most recently when Patrick Conway, M.D., Deputy Administrator for Innovation and Quality and Chief Medical Officer, CMS, delivered the keynote address at the Tenth Annual State of Personalized Medicine Luncheon.

Dr. Conway outlined his perspective on the state of personalized medicine and where it is headed, noting that we are in an era in which the power of genetics is driving innovation, informing treatments and improving patient outcomes.

As Dr. Conway noted, diagnostics are a challenge as a new market, and CMS is working with FDA to streamline the process further. We want and need diagnostics that enable physicians’ to tailor therapies for individual patients and influence decision making, all resulting in better health outcomes and improved patient care.

Dr. Conway emphasized innovation, noting that we need a system that values innovation and pays for it appropriately. We should cover and pay for technology that may have greater costs in the short-term, but which will result in long-term savings and improved outcomes.

Yet, innovators are facing significant challenges stemming from CMS policies, through the unintended consequences related to coding, in particular. PMC is focused on working with CMS to ease the pressure on innovators, and identifying opportunities to make sure this does not happen again.

A key theme of Dr. Conway’s talk was that of collaboration. PMC will continue to facilitate engagement with CMS, taking up Dr. Conway on his open door policy to ensure that future policies encourage innovation and support continued developments in personalized medicine.

Dr. Conway’s presentation is available at the PMC website.