A Patient-Centered Perspective on Cancer Research and Care

Shelley Fuld Nasso, M.P.P., CEO, National Coalition for Cancer Survivorship, addressing the role of payment reforms in supporting innovative cancer care at the Turning the Tide Against Cancer 2014 national conference (Photo: Liz Roll)

A key theme at the Turning the Tide Against Cancer national conference was the need for a patient-centered approach to cancer care. Shelley Fuld Nasso, M.P.P., CEO, National Coalition for Cancer Survivorship (NCCS), shared her thoughts on the importance of patient education, shared decision-making, and aligning a treatment plan with the goals and priorities of the patient.

Read more about Shelley’s thoughts on incentive structures that encourage innovation, as well her personal experience with cancer, and the urgent need to push discovery to help save lives on the NCCS blog.

Developing Policies to Advance Personalized Medicine

In my previous post, I highlighted a Hill briefing hosted by the Personalized Medicine Coalition (PMC) and the great examples it provided of the impact of personalized medicine on patients. I was reminded why we must advocate for policies that support continued progress against disease and fulfill the promise of personalized medicine.

PMC provided an excellent starting point at the briefing, releasing a set of policy principles that offer a road map for fostering personalized medicine in a deficit-reduction environment.

As the panelists at the briefing emphasized, policy solutions must start with the patient – putting the patient at the center of decision-making, and supporting a shift in care delivery towards patient-centeredness and patient engagement.

Video highlights from the briefing panelists underscore why policy solutions like those advanced by PMC are so important – many patients continue to face significant unmet medical needs that can be effectively, efficiently met through novel targeted therapies; policy must provide adequate incentives for continued progress, recognizing the ways individual advances build on each other and understanding of an intervention’s optimal role and value evolves over time; and research and evaluation must adapt to rapid changes in clinical practice, the science of personalized medicine, and an increasingly robust “learning healthcare system.”

Below are some key, related statements from PMC’s policy principles:

“PMC believes that a deliberate and comprehensive shift away from health care based on population averages and towards patient-centered care is central to improving health care outcomes and addressing a patient’s perception of value.”

“A health system that focuses on care that is predictive, preemptive, and preventive has the potential to revolutionize health care by allowing clinicians to individualize therapy for patients through the early diagnosis of disease and risk assessment in order to optimize clinical outcomes and to better manage patients before disease symptoms appear.”

“Reform assessment tools (in particular, comparative effectiveness research and health technology assessment) to better align with emerging science and patient-centered health care.”

Coverage or reimbursement policy based on “one-size-fits-all” definitions of comparative clinical or cost effectiveness fail to recognize differences in patient needs and preferences and is misaligned with the progress in personalized medicine. This type of policy has resulted in significant barriers to access to the treatment options that are best for the individual and chills medical progress.

New payment models, such as accountable care organizations (ACOs) and bundled payments, which promote coordination and integrated care, hold potential to shift incentives to high-quality, high-value care for patients. However, if improperly designed, such models will set payment based on current standards of care and discourage advances in medical technology and medical practice.

PMC believes that there must be a continual learning system to aggregate, analyze, and apply evidence-based knowledge to patient care. PMC believes that health informatics and electronic health record (EHR) systems must be used to promote continual learning systems that will help improve patient care, reduce costs, and accelerate the process of drug development.

A version of this blog originally appeared in The Catalyst posted at the PhRMA website.

A Response to Ezekiel J. Emanuel’s NYT Op-Ed about the Cost of Cancer Care

Ezekiel J. Emanuel’s “A Plan to Fix Cancer Care” (The New York Times, Op-Ed, March 23, 2013) highlights the need for collaboration and commitment across the oncology community to find solutions for the financial burden cancer poses to our healthcare system.

In order to sustain progress while containing cost, though, we must recognize the heterogeneity of the dreaded disease by eschewing a one-size-fits-all approach. We must, in short, devise strategies that make medical sense. These strategies necessarily will involve adaptive clinical trials, an intelligent regulatory policy that encourages linking therapy and diagnosis, and a willingness to pay for products that improve patient care.

Progress is driven by a dynamic process in which the full value of intervention evolves over time. We must be careful not to stifle innovation by overlooking the critical steps that can provide an answer to the correct question, which is: how can we maximize value from our growing, but not always wise, investments in health care?

National Bioeconomy Blueprint Showcases Personalized Medicine as Model for Strengthening U.S. Bioeconomy

Last week, the White House released its National Bioeconomy Blueprint.  It lays out some strategic objectives designed to help realize the full potential of the U.S. biotechnology sector to generate economic growth by creating jobs and addressing societal needs.

As an example of how the government’s efforts can facilitate the development of a more robust bioeconomy, the report discusses the impact of the Human Genome Project and the development of personalized medicine. The Blueprint cites the Case for Personalized Medicine, 3^rd Edition, noting “advances in recent technologies have increased the momentum of personalized medicine – customized healthcare based on specific genetic or other information of an individual patient.”

While we agree that policies are needed to help support research and development (R&D), improve translational and regulatory science, improve regulation in other areas, enhance workforce training, and develop new public-private partnerships and precompetitive collaborations, we are concerned they are not sufficient to allow us to realize Adriana Jenkins’ dying wish, that all patients have access to personalized treatments.

The White House is correct to shine a light on FDA and direct the agency to focus attention on application review times, coordinated parallel reviews of products, and continued improvement of regulatory science. In reality, we are already seeing the benefits of this increased coordination – with FDA’s accelerated review and approval of Kalydeco™ for cystic fibrosis and Xalkori^® for non-small cell lung cancer, each with a companion diagnostic approved together and in advance of FDA time lines.

Still, engaging on regulatory science and streamlining FDA processes will only go so far to improve the bioeconomy and bring personalized medicine to patients. For instance, current comparative effectiveness research (CER) and health technology assessment (HTA) models, which are not addressed in the Blueprint, are not well aligned with the science of personalized medicine and such misalignment causes additional barriers to market entry and patient access after FDA approval. The Blueprint highlights coverage with evidence development (CED) as a potential HTA model, but it should be noted that although CED can be a good tool, if done wrong, it can also chill innovation.

Likewise, unclear and unrealistic expectations for obtaining Medicare coverage and adequate payment for personalized medicine products and services are a substantial hurdle, preventing companies from seizing the full scientific potential and translating that into new treatments and the resulting high-paying jobs and economic contributions that follow.

We look forward to working with the Administration and with Congress to shape policies that will help support the ability of companies to continue to develop personalized medicines and bring them to patients, including research and development tax credits, delivery system reforms, and regulatory and reimbursement policies. Given the tremendous potential of personalized medicines, it’s key that we get the policies right to foster companies working on personalized medicine and thereby improve patients’ lives and our economy.

Our Health Policies Can’t Ignore Where Science Is Leading Us

Do we want continued progress in personalized medicine, or don’t we? Based on events last week, the answer might be ‘both.’ I attended a press conference here in Washington that celebrated the achievements made by the health sector in our efforts to turn cancer from a sure death sentence into a chronic disease. Simultaneous to this, the Medicare Payment Advisory Commission (MedPAC) proposed policies to offset the cost of a physician payment fix under Medicare. The disconnect between the two was striking. Unfortunately, the MedPAC proposals have the unintended effect of heavily impacting those on the leading edge of personalized medicine. Of the $233 billion in 10-year savings, $114 billion would come out of the biopharmaceutical and clinical laboratory sectors, thus possibly robbing seniors of access to the latest targeted therapies and the diagnostics used to guide them. The proposal includes savings from giving the Centers for Medicare and Medicaid Services (CMS) new authority to impose “least costly alternative” payment, which would result in locking us into our current one-size-fits-all medical paradigm and weakening physicians’ power to tailor care using diagnostics and targeted therapies.  Another troubling aspect of these suggestions is that they propose cuts by sector, ignoring the reality that personalized medicine brings efficiencies to the system of health care.

The proposal appears to reflect a lack of awareness of the impact that personalized medicine is making in health care and underscores the importance of the Personalized Medicine Coalition’s legislative work.  One of our policy suggestions, released over the summer, calls for inclusion of a personalized medicine representative on MedPAC and the creation of a new personalized medicine advisory committee to foster alignment of the policies across the entire Department of Health and Human Services with the science of personalized medicine. Personalized medicine challenges the status quo in medicine – the blockbuster business model, diagnostic coding and payment, or health care delivery systems. Maybe it’s time it challenged MedPAC, too.

Capitol Hill Briefing to Explain the Value of Personalized Medicine

Back in February this blog reported on the story of Adriana Jenkins, a breast cancer patient who, shortly before her death, called for policies to accelerate personalized medicine:

“A targeted cancer drug kept me alive for nine years. Congress must ensure that future patients get the same chance to live.”

PMC has been working hard to respond to Adriana’s call for action. As one step in this, we’re pleased to be taking part in a briefing on Capitol Hill describing the status of personalized medicine and potential steps to accelerate it.

Advancing personalized medicine involves the complex process of bringing together the many stakeholders who have a role in the health care ecosystem: pharmaceutical companies, diagnostics companies, clinical laboratories, health information technology companies, health care providers, payors, and researchers.  Each comes to personalized medicine with a different perspective but a common goal – improving treatment for patients like Adriana through personalized medicine.

As Adriana pointed out, policymakers play a key role in advancing personalized medicine.  Health care reform raised the profile of the term on Capitol Hill but several members of Congress and congressional staffers have told me that they and their colleagues want to better understand how personalized medicine can improve health outcomes, lower health care costs, and ultimately improve patient care. Today we will take an important step in raising awareness and understanding of personalized medicine on Capitol Hill.

Working with the House Medical Technology Caucus co-chairs Rep. Erik Paulsen (R-Minn.) and Rep. Anna Eshoo (D-Calif.), the Personalized Medicine Coalition is hosting a briefing on Capitol Hill to inform members of Congress and congressional staff about personalized medicine and how public policy can further its development through incentives, regulatory changes, and HIT-enabled opportunities.

At the briefing we will hear from researchers, providers and patient advocates.  Joining us will be Stephen Eck, M.D., Ph.D., Vice President and Global Head of Medical Oncology for Astellas Pharma Global Development, Amy Abernethy, M.D., Director of the Duke Cancer Care Research Program at Duke Cancer Institute, and Donna Cryer, J.D., CEO of CryerHealth and Chair of the American Liver Foundation’s Board of Directors.

The briefing will take place today in the Rayburn House Office Building Room B-338 on Capitol Hill from noon-1 pm.  Limited seating is available; please register here (http://events.constantcontact.com/register/event?llr=g8sac8dab&oeidk=a07e3mmnzg5cc593a37) or email Tswope@personalizedmedicinecoalition.org.

Letter to the Editor: The Proper Integration of Comparative Effectiveness Research

A recent editorial in The New York Times (Is Newer Better? Not Always, Sept. 11, 2010) suggests that the comparative effectiveness research (CER) provisions of health care reform are a solution to curtailing ever-increasing medical costs by providing patients, doctors, and the government better information about the efficacy of different drugs and treatment strategies. It also suggests that the real opportunity is in giving policymakers more power to enforce CER-informed practice guidelines. 

In response, I sent a Letter to the Editor, agreeing that CER can be part of the overall solution. However, I also cautioned that thinking simplistically about medical innovation as the “problem” and CER-based guidelines as the “solution” overlooks the value that medical advances like those in personalized medicine hold for patients, and for helping address rising health care costs.

“If we lock ourselves into a one-size-fits-all paradigm, we will strangle the power of personalized medicine to prescribe the right drug for the right patient at the right time.” This approach could turn CER “into a blunt instrument that smothers the opportunity to improve care and reduce costs.”

Also worth noting is a separate letter from Tony Coelho, head of the Partnership to Improve Patient Care, who argued that the CER provisions of the health reform law “can be an important part of the solution to rising health care costs over the long term, but only because the health reform law gives patients and doctors the assurance that the program is centered on our health care needs.”

Read both of these published responses below and continue the discussion by contributing your own comment at The Age of Personalized Medicine Blog.

* * *

To the Editor:

Your editorial correctly states that new health care technologies often lead to medical progress, but also regrettably to increased and unaffordable costs, unlike in other fields where new technology usually causes costs to decline.

The danger is that the proposed solution, comparative effectiveness research (CER), could turn into a blunt instrument that smothers the opportunity to improve care and reduce costs.

When President Obama sought to explain how it would work, he said that with the new funds allocated for CER, researchers would be able to compare the red pill with the blue pill to determine which worked better and thereby, presumably, save money.

The key question to ask, as neither your editorial nor President Obama does, is which medicines work for whom? While the blue pill may work for me, there is a good chance it won’t work for you, especially if the diagnosis is cancer.

If we lock ourselves into a one-size-fits-all paradigm, we will strangle the power of personalized medicine to prescribe the right drug for the right patient at the right time. Personalized medicine enables us, over time, to save enormous sums because patients who should not receive particular medicines will not get them.

Edward Abrahams
President
Personalized Medicine Coalition
Washington, Sept. 13, 2010

* * *

To the Editor:

Creating a body with the power to enforce national guidelines for treatment based on clinical and cost effectiveness would be a serious distortion of the comparative effectiveness research (CER) provisions of the health reform law. It would confirm the fear of many that the real intent was to give government decision makers new power to cut costs by denying patients access to medically beneficial tests and treatments.

Fortunately, Congress took a different tack in the health reform law and got it right. It acted not in reaction to fear-mongering but because, as stated last year by one Democrat, the CER research center must be a partner to patients.

Yes, CER can be an important part of the solution to rising health care costs over the long term, but only because the health reform law gives patients and doctors the assurance that the program is centered on our health care needs. Newer isn’t always better, but in the case of the new patient-centered approach to CER in the health reform law, it is.

Tony Coelho
Washington, Sept. 14, 2010
The writer, a former member of Congress, is chairman of the Partnership to Improve Patient Care.

A version of these letters appeared in print on September 20, 2010, on page A30 of the New York edition.

Fall Forecast: Personalized Medicine Policy and Events

Labor Day has come and gone, Congress is preparing to go back in session, and shortening days remind us that Daylight savings will be here before we know it. Even as the long days of summer draw to a close, the fall promises to be a busy and productive time in advancing policies to support personalized medicine, as well as convening industry stakeholders to connect, share ideas, and collaborate toward advancing the science, business, and policy of personalized medicine.

This fall we expect to see a maturing of activities started over the summer.  FDA hosted a 2-day meeting in July on the regulation of laboratory-developed tests (LDTs).  The meeting docket (FDA-2010-N-0274) provides a good overview to any who missed the event.  After that meeting, the House of Representatives held a hearing on consumer genomics.  This hearing made it clear that Congress is not satisfied with how gene scans are regulated, and sometimes that dissatisfaction was aimed at LDT regulation in general. The Senate is also concerned; GenomeWeb reported that Senator Hatch (R-UT) is considering legislation on the subject. 

In other legislative news, Rep. Patrick Kennedy (D-1^st RI) partnered with Rep. Anna Eshoo (D-14^th CA) to introduce an updated version of the Genomics and Personalized Medicine Act (HR 5440). PMC will continue our work to strengthen this bill through inclusions of research  incentives for personalized medicine and other improvements in the Senate version as we look for it to be re-introduced  in the next Congress. 

Next week, I will be joining Carolyn Clancy, Francis Collins, and  many other leading experts at the 2nd National Comparative Effectiveness Summit which will feature presentations and discussion on the legislative history and future of comparative effectiveness research (CER), how federal stimulus dollars for CER are being put to use, as well as the intersection between CER and personalized medicine. The focus of this meeting is particularly timely as the expected announcement of the federally-appointed Board of Governors for the Patient-Centered Outcomes Research Institute (PCORI) is expected later this month. The discussion on balancing CER and personalized medicine objectives will continue in October at the Comparative Effectiveness and Personalized Medicine:  An Essential Interface meeting hosted by the ECRI Institute, Agency for Healthcare Research and Quality, and the National Institutes of Health.

There are a number of other meetings taking place throughout the fall focused on a variety of topics and issues relevant to personalized medicine, beginning with the 6th Annual Burrill Personalized Medicine Meeting on September 27 & 28. Hosted by Burrill & Co and the Duke Institute for Genome Sciences & Policy, the meeting will focus on the impact of healthcare reform on personalized medicine. On October 14 & 15, the 3rd Annual Personalized Health Care National Conference is taking place at Ohio State University Medical Center’s Center for Personalized Health Care. This conference will include discussion of the challenges of making predictive, preventive, personalized, and participatory medicine a reality.

The conference season will culminate on November 17 & 18 with the 6th Annual Personalized Medicine Conference. Each year, the Partners HealthCare Center for Personalized Genetic Medicine, Harvard Medical School, and Harvard Business School bring together representatives from academia, diagnostic and pharmaceutical companies, healthcare providers, policymakers, and other industry experts making contributions to advancing personalized medicine. This year we’ll explore the ways personalized medicine impacts clinical care, and how lessons from these experiences can inform policies and plans to implement personalized medicine to improve patient care.

For more information on these and other conferences of importance to personalized medicine, please visit the Events page at The Age of Personalized Medicine website. And throughout the fall, stay tuned to The Age of Personalized Medicine Blog as an opportunity to engage in discussions stemming from these events and new policy developments.

Charting the Course for Personalized Health Care Amidst the Turbulent Winds of Change

Can you recall a time in modern history marked by such dynamic forces of change in medical research, health, and health care? Today, during this period of fierce economic hardship, the U.S. is experiencing a dramatic up-tick in investment, incentives, and innovation aimed at developing better knowledge for health and applying it in new ways.  Much of this change can be attributed to massive public investment in health information technology and a new bolus of support for biomedical research – however, private R&D sector and global competitiveness forces are also in play. Health reform proposes massive shifts in health care policy, including larger roles for government in providing access to health care and the regulation of private insurance markets.

Recently, we published two papers that address these tectonic plates of change in health care and the plausible ways they may affect the course of personalized health care. In the first (1), we outline the policy changes in health care that are underway – these represent waves of opportunity for personalized medicine. There is no doubt we will be a much more connected environment – biologically, technically, and socially. The future of applying precise knowledge about the patient’s own biology to medical practice is highly dependent on the tools of the information age. We also presented some new ideas and concepts about the use of health care data to serve discovery, translational, and outcomes research.  Liberating clinical care data for research is a key element of opening doors for innovation to prosper. (2) One thing is certain – research and health care will increasingly be operating in data rich environments. Will this translate into relevant clinical knowledge and represent opportunities for personalized medicine?

Seizing these transformative changes in the health care setting should be viewed as the opening to interject new scientific approaches in answering questions that previously couldn’t be tested.

I encourage you to examine these papers and offer your comments. What do you think these incredible forces of change will mean for personalized health care?

1. Downing GJ.  Policy perspectives on the emerging pathways of personalized medicine. Dialogues Clin Neurosci. 2009;11:421-428.
2. Nadler JJ, Downing GJ. Liberating health data for clinical research applications. Sci Transl Med. 10 February 2010 2:18cm6.