2013: A Year of Innovation, Inspiration

As we look forward to 2014, we engage in the time-honored tradition of reflecting on the year of activities and progress as we bid 2013 farewell.

For personalized medicine, it was another year with big advances, and big questions. The following captures highlights from The Age of Personalized Medicine Blog for 2013.

We started the year with a call to action from Amy Miller, PMC vice president, public policy, about the power of personalized medicine, the need for “accurate and actionable” data, and for a shift in traditional healthcare models. As Amy noted:

…The power of personalized medicine lies not only in treatment, but prevention. The best scenario for personalized medicine in action will come when patients are able to see not only the implications of their genetic dispositions, but also are motivated and empowered to use that information in preventative care.

In May, personalized medicine took the international spotlight as Angelina Jolie shared with the world her genetic prognosis and personal decision to pursue a course of preventative care. The high-profile case inspired much discussion of the value of personalized medicine, as well as the need for access to diagnostic testing for all:

Ms. Jolie’s contribution demonstrates that without patients who are educated, empowered to get tested and to act, the progress and innovation could be for naught. We need to make sure that patients are knowledgeable enough to pursue personalized options for their own health but also so that they are motivated to support policies that foster continued progress in this area.

Kristin Ciriello Pothier, Diagnostics and Life Science Practice lead at Health Advances, shared key take-aways from the PMC/BIO Solutions Summit panel she moderated on “Evidentiary Standards and Data Requirements for Payer Coverage.”

Ms. Ciriello Pothier examined the challenges facing the personalized medicine diagnostics industry and summarized her panel’s conclusions:

First, panelists agreed that there must be more education for all stakeholders so that each stakeholder can actually evaluate novel products appropriately, a key finding echoed throughout the day. Second, the emphasis on outcomes must shift from only clinical outcomes to clinical outcomes and quality of life for patients. Finally, all panelists agreed the ideal situation is open, trusting lines of communication and split of the responsibility according to expertise.

William Chin, M.D., identified research, collaboration, and innovation as critical components to advancing personalized medicine, while Stephen Eck, M.D., Ph.D., cited the need for funding for basic research, diagnostics testing research, increased healthcare provider education, and reimbursement for diagnostic testing as key.

At the mid-point of 2013, pressures to reduce federal spending elevated the need for policy that will support biomedical research and innovation. PMC responded to these concerns through comments submitted to the Centers for Medicare and Medicaid Services (CMS) regarding the charge on regional Medicare Administrative Contractors (MACs) to set new prices for molecular diagnostic test reimbursement through a process called “gapfilling”:

The Personalized Medicine Coalition (PMC) submitted comments to CMS on the Gapfill Payment Amounts and CLFS, iterating the concerns of PMC members that insufficient payment amounts threaten the sustainability of the laboratory industry and continued investment in the developing field of personalized medicine. As a consequence, this policy has the potential to stifle innovation and progress in healthcare and possibly eliminate the potential for lowering overall costs through the elimination of unnecessary and or ineffective treatments.

In 2013, PMC also released a set of policy principles key to advancing personalized medicine in a deficit-reduction environment. Debuted at a Capitol Hill Briefing, these principles lay the groundwork for consensus-building around key issues to ensure that efforts to contain rising healthcare costs do not undermine continued progress in personalized medicine and protect innovation, the physician-patient relationship, patient values, and choice.

The reality of personalized continues to be realized with patient triumph stories bringing new advances in science, technology, and research to life:

The promise of personalized medicine is very real. Personalized medicine is not an abstract concept for the future of medicine. It is here, it is now, and the true promise has been realized in the lives of Kellie and Stephanie, and the precious days, weeks, months, and years they have taken back from their disease.

I encourage you to read more about Kellie and Stephanie, and to draw inspiration from their strength.

At the 9th Annual Personalized Medicine Conference in November, it was an honor to present Kathy Giusti, founder and CEO of the Multiple Myeloma Research Foundation (MMRF) with the 2013 Leadership Award. Ms. Giusti’s story of ingenuity in the face of adversity has led to a redefinition of the role of patient advocacy organizations in research:

Clinically, MMRF has funded research and paved the way for FDA approval for six multiple myeloma treatments in 10 years, and doubled the lifespan for many patients. Today in her remarks, Kathy shared that her daughter, who was just one year old when Kathy received her myeloma diagnosis, is now a 19-year-old college student with a younger brother. Incredible results.

2013 was a year of progress and inspiration. We look forward to the new year ahead and personalized medicine advances on the horizon, bringing new treatments and improved outcomes to patients.

The Personalized Medicine Revolution Will Require Revolutionary Changes In How We Care For Cancer Patients

In the following post, Dr. Leonard Lichtenfeld, Deputy Chief Medical Officer for the American Cancer Society, shares his thoughts on how to improve the cancer patient care delivery process and reflects on conversations from a roundtable held by the Turning the Tide Against Cancer initiative.

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I recently attended a meeting in Washington that got me to thinking about the fact that as we revolutionize cancer research and treatment, we are also going to have to revolutionize cancer care. And that may prove to be an even more daunting task than finding new treatments for the disease itself.

The meeting was sponsored by a collaboration called “Turning The Tide Against Cancer“. The organizers brought together experts from a variety of disciplines ranging from insurance companies and economists to advocacy groups and highly regarded cancer specialists to discuss policy solutions to support innovation in cancer research and care. Walking in, I anticipated this was going to be another one of those sessions where we talked about funding for research, bringing research into clinical trials, and having patients get access to new drugs. But I was wrong. The discussions quickly steered into a different direction: what do we need to do to make the cancer care system work for patients?

Of course there were the continuing themes of “big data” and the impact of genomics on drug development and patient care, but a surprising amount of the discussion centered around new payment models, quality of care, and fundamental redesign of medical care to become more patient centric. And although we talked a lot about data gathering and analysis, what stuck with me was the redesign piece. I thought the discussion around redesign would focus on personalized medicine, but we spent a lot of time on changing the fundamental structure of cancer care and payment.

How are those two linked? Did we miss our focus?

The answer? If we don’t change the way the system is working, we won’t realize the promise of personalized medicine. Seems pretty simple and straight forward until you start thinking about the implications.

Our science is moving forward at a rapid pace, in significant ways. We are learning about the human/cancer genome, we have many new drugs in the pipeline that are targeted to the abnormal genetic signatures of cancer cells, we have research reports on a regular basis about a new finding that may predict who is at higher risk of developing certain cancers, or new tests to predict whether someone has an aggressive or indolent cancer. But if we don’t rethink the nuts and bolts of delivering the care, we won’t be able to get these remarkable tools and discoveries into the hands of patients and doctors. That in turn means we won’t be able to offer patients the benefits of the phenomenal advances we have made in cancer research and drug development. Heck, we won’t even have the tools to help develop the drugs or learn who the patients are who may benefit from the drugs or how patients respond to the drugs.

All of this means we have to figure out how transform the system. We need to be able to capture real data from real people. We need to have computer systems that talk to one another. We need to have a payment system that rewards quality-based and innovative patient care.

The list goes on, but you get the point. Being “patient centered”–which is a bit of a buzzword these days–is a call to action. The problem is that the barriers to change are substantial, and many more people and institutions pay lip service to the concept of patient centered cancer care than work to make their care truly patient centric.

Make no mistake: this is a gargantuan task. There are formidable obstacles to overcome which will favor keeping the status quo in place. Progress will be slow and difficult, but we have to keep remembering that we really haven’t tackled the issue of assuring every cancer patient and their loved ones that they got the right treatment for their disease at the appropriate cost and at the appropriate time. Fragmentation, duplication, and lack of information and access are no longer acceptable.

So it’s no longer just about the science and the research, as important as they may be. It’s not just surgery, chemotherapy, and radiation therapy. It’s about the care delivery process and the quality of that care. Reconstructing from the ground up. Thinking about new ways to help patients. It’s about nutrition, psychological and spiritual support, and financial guidance. It’s about making certain that people who live in rural areas can get care that is up to date and accessible through care collaborations designed to serve their needs and their expectations.

Is it too much to ask that patients, their families, and caregivers have genuine assurance that they are getting the best care available? Is it too much to ask that systems be in place to assure quality care? Is it too much to ask that we have computers that actually talk to each other? Is it too much to ask that in the most difficult moment of one’s life, they have an assurance that what was done for them was appropriate?

Don’t expect easy or quick answers to this situation. It took us decades of fundamental research to get us to the place we are today with the human genome. It is probably going to take a similar amount of time to change cancer practice. And I suspect changing practice is going to be even tougher to figure out than the gene thing.

But if we don’t do it, the power of what we will have to offer patients simply won’t be there for everyone. And that is something none of us should accept.

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A version of this blog originally appeared in Dr. Len’s Cancer Blog at the American Cancer Society.