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Developing Policies to Advance Personalized Medicine

August 7, 2013

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.

Answering the Call: Personalized Medicines Making Progress in Fighting Cancer

August 2, 2013

Last week, the Personalized Medicine Coalition convened leaders from the field on Capitol Hill to discuss the promise of personalized medicine and the implications for policymakers. A number of important issues were raised (more on that in my next post), but the highlight was remarks by Stephanie Haney. Stephanie is a lung cancer patient and mother of two who said she is alive today thanks to a novel personalized medicine. She also said doctors believe that someday her cancer likely will come back despite the treatment. Check the Turning the Tide initiative website in the coming days for more information from the briefing including video interviews from Stephanie and other speakers.

Stephanie’s story reminded me of Adriana Jenkins. Adriana was a breast cancer patient who, before losing her battle against the disease in 2011, published an article in Forbes magazine called “A Dying Wish.” In it, she credited a personalized medicine with extending her life by nine years; and she called on the biopharmaceutical sector to do more to accelerate progress in this field.

We haven’t forgotten that call, and Stephanie’s life is a tangible sign of the progress we’re making against cancer, particularly through development and introduction of new personalized medicines that target cancer based on results from genetic tests. Her story is also a reminder of how important it is for us to make continued progress against cancer.

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

Sustaining Progress in Personalized Medicine

December 4, 2012

Last week, I had the opportunity to speak at the Harvard Personalized Medicine Conference in Boston, MA. No other conference on personalized medicine brings together the array of scientists, stakeholders, and experts that this event does. This year the conference drove home to me that the potential to improve patient care via personalized medicine is greater than ever – yet the scientific and clinical challenges remain daunting. It is more important than ever to sustain biomedical innovation, and to ensure that health policy is informed by the enormous opportunity, and complexity, of making continued progress in this field.

The event also underscored that biopharmaceutical research companies are deeply committed to advancing the science of personalized medicine and building it into their research and development strategies. It affirms findings of a report released by the Tufts Center for the Study of Drug Development in 2010 which found that 94% of biopharmaceutical companies surveyed are investing in personalized medicine and 100% are using biomarkers in the discovery stage to learn about compounds. This research has required large up-front investments in new research tools and training. But, as we have seen in the last year-and-a-half with FDA approval of new targeted therapies for lung cancer, melanoma, and cystic fibrosis, it is starting to bear fruit for patients.

I’m hopeful we’ll see more approvals in the months ahead. In the report from Tufts, companies reported that 12-50% of compounds being researched are personalized medicines and over the last five years, they have seen a roughly 75% increase in their investment in personalized medicines. The importance of personalized medicine was illustrated in the reauthorization of the Prescription Drug User Fee Act, which provides FDA with increased resources and staffing to advance the regulatory science in areas such as pharmacogenomics and biomarkers.

This progress, however, doesn’t happen in isolation. The Harvard Conference participants represented, and illustrated, the wide range of organizations and individuals from different sectors that make up the research ecosystem that drives progress in personalized medicine. As the science of personalized medicine advances, research partnerships and collaborations will be more important than ever. To sustain progress in personalized medicine, it is vitally important to ensure that policy and regulation do not erect barriers to these types of partnerships.

Biomedical innovations like personalized medicine will help address major unmet medical needs, and offer a solution to rising healthcare costs.  As we face continued pressure to contain healthcare costs, it is crucial to ensure that healthcare policy sustains the innovation ecosystem and incentivizes continued progress in personalized medicine.


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