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.

Reflections from the Turning the Tide Against Cancer Conference

Following the Turning the Tide Against Cancer national conference last Thursday, I’ve had time to reflect on what we must do next to improve how patients experience cancer care.

To make that change we must acknowledge key truths.

Science is driving us towards more targeted solutions and novel approaches to treatment. The clinical trials and regulations necessary to get new products to market must change with the science. They must be nimble to keep pace with the extraordinary advances we are seeing.

Coverage and payment decisions must also allow patients access to advanced treatments and not disincentivize innovations in health care. While new products sometimes seem expensive, we must remember that costs are saved by getting the right treatment to the right patient at the outset of care.

We must educate. Each of us has a responsibility and a role to play in educating all participants in health care about the changes we are seeing in cancer. We must educate patients and their families about the complexity of decision-making and share information with them so that each patient can make informed choices. We must provide resources so health care providers are able to stay current with the latest developments in care. We also must educate policy makers to ensure that negative unintended consequences of policies are avoided.

Everyone working in health care wants to provide patients with the absolute best treatments imaginable – and those we have not yet even begun to imagine. Sometimes that will require addressing very tough questions in order for personalized medicine to be fully integrated into the health system.

Finally, we should celebrate the improvements made in cancer care from innovations in clinical trial design, to imaginative diagnostic testing of solid tumors that allow for better targeting of treatments, and tests that help physicians and patients make the best treatment choices. Advances are being made in science that are extending and improving lives. This is no small feat, and while our goals are great, we must celebrate the achievements that have taken us this far, and acknowledge the policies and pathways that made those discoveries possible.

We have an opportunity and responsibility to change the future of cancer care through continued education and advocacy. I invite you to help be a part of this change by sharing your thoughts and joining us on this journey to turn the tide against cancer.

FDA Outlines Personalized Medicine Policy with Publication of LDT Draft Guidance Document, Final Guidance on Companion Diagnostics

On July 31, FDA announced drastic changes to regulation for personalized medicine products and services when it coupled the release of the long-awaited final guidance document on the regulation of companion diagnostic devices with a proposed framework for regulating laboratory developed tests (LDTs), which was also long-awaited or long-feared, depending on your perspective.

The final guidance on In Vitro Companion Diagnostic Devices was welcomed by the personalized medicine community because in the document, FDA clarified the path for co-developed drug-diagnostic products, and finalized their assertion that new targeted therapeutics will not be kept from the market if the diagnostic kit is not ready at the same time. This enables promising new drugs to come to market while also allowing the laboratory community to fill testing needs in cases where an FDA-approved kit is not available for therapeutic selection, dosing and avoidance decisions.

However, many issues remain to be addressed.

To address concerns that FDA regulation will pose obstacles to an already challenged laboratory industry, there is a rather long transition phase — nine years — and an initial focus on high-risk. FDA defines high-risk LDTs as those with the same intended use as cleared or approved companion diagnostics, LDTs with the same intended use as an FDA-approved Class III medical device, and certain LDTs for determining the safety and efficacy of blood or blood products. This focus and transition period will allow clinical laboratories and FDA time to adjust. By focusing initial regulation on high-risk LDTs, FDA makes a strong argument for the framework, and slices off a rather small segment of the LDT market.

Many have argued that FDA does not have the bandwidth to regulate LDTs. FDA responded to this claim by reiterating enforcement discretion for the vast majority of LDTs and outlining a process for LDT regulation, which might be less onerous than traditional regulatory pathways for medical devices.

Although FDA made great efforts to address concerns about the Agency’s new regulatory enforcement, it did not address perceived conflicts between laboratory regulation under Clinical Laboratory Improvement Amendments (CLIA) and this new framework. Furthermore, FDA intends to use an expert advisory panel to provide recommendations to the Agency on LDTs risks and classification on certain categories of LDTs, as appropriate. I suspect that defining those categories will be contentious and, at times, difficult.

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.

BREAKING NEWS: FDA Notifies Congress of its Intent to Publish Framework for Regulatory Oversight of Laboratory Developed Tests (LDTs)

Summers in D.C. are notoriously slow. FDA, however, has added excitement to this summer by informing Congress of its intent to publish a long-awaited framework for LDT regulation.

In its notice to Congress, FDA included what appears to be a draft of the document. After the mandatory 60-day Congressional review, the draft guidance document will be formally issued for public comment.

Within the draft framework, FDA proposes a risk-based, phased system of oversight. They recognize community concerns around access and do not intend to interrupt the marketing and sale of currently available tests. Furthermore, FDA expresses the intent to continue using enforcement discretion for forensic and organ transplantation uses, traditional LDTs, and LDTs for unmet needs.

The document outlines the history of LDT regulation, FDA’s policy of enforcement discretion, and how personalized medicine has caused FDA to reconsider that policy.

We will continue to provide updates on the development of framework for regulatory oversight of LDTs, with additional in-depth commentary next week on this issue and the related news of FDA’s final guidance on companion diagnostics.

For additional information on the current regulation of LDTs, please read PMC’s report “Pathways for Oversight of Diagnostics.”

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.

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.

Key SGR Fix Brings Relief to Diagnostics Industry

On April 1, the diagnostics industry let out a collective sigh of relief as President Obama signed into law HR 4302. Officially known as the Protecting Access to Medicare Act of 2014, many refer to the law as “Doc Fix” or “SGR fix”, focusing on the delay, yet again, of the 24 percent physician pay cut as a part of Medicare’s sustainable growth rate (SGR) formula.

However, my attention, along with that of the diagnostics industry, focused on the provisions in the law that protect clinical diagnostics from unpredictable repricing (subscription required) under the Centers for Medicare and Medicaid Services (CMS) Clinical Lab Fee Schedule (CLFS).

The passage of this law showcases the need and value of the industry to unite in highlighting policies that, though unintended, challenge innovators by creating an uncertain reimbursement environment. Predictable reimbursement policies encourage exploration and innovation within the diagnostics community, improving health care through advances in diagnosis and beyond.

This law acknowledges the transformative scientific advancements the industry has achieved in recent years, catching our policies up with our technologies.

Many questions remain as we look at the implementation of this law, and as such we remain committed to convening the personalized medicine community to work in collaboration with CMS.

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.

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.