Targeted Therapies: From Promise to Reality

A recent Wall Street Journal article featured the impact that the genomics revolution is having on cancer patient care with a focus on lung cancer. The article shares a strong message of hope, detailing the scientific progress made thus far by noting that we have experienced the “most extraordinary decade of progress ever in the long scientific struggle against lung cancer,” while also pointing to the promise further personalized approaches to cancer care holds.

Lung cancer patient Kellie Carey poignantly shares her story of how she had to demand testing for her tumor to identify its mutation, opening the door for her to molecularly targeted drugs that extended her life.

This message echoes what we heard during the briefing Personalized Medicine: How Medical Progress Happens held by the Personalized Medicine Coalition on Capitol Hill just last month — in particular, the experiences of one of the speakers, lung cancer survivor Stephanie Haney (click here for a video interview with Stephanie). Stephanie and Kellie share a disease and a journey as they have both been “lucky” enough to have been diagnosed with lung cancer in the midst of a revolution in care.

For thirty years the available treatments extended life a few weeks, possibly months. Lung cancer diagnosis was an immediate and imminent death sentence. Now, Stephanie, Kellie, and others with genetically identified tumor mutations have access to targeted treatments that are extending their lives years longer than those lung cancer patients on chemotherapy only and without identified mutations.

These are just two examples of the value of innovative therapies, particularly molecularly targeted drugs. 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.

And now it is our promise to Kellie, Stephanie, and others to continue to push forward, toward further discovery, and to drive innovation.  There are nearly 1,000 new medicines in the pipeline for cancer alone.  We can all do our part by supporting policies that enable and encourage innovation and allow the science to guide us.

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.

Answering the Call: Personalized Medicines Making Progress in Fighting Cancer

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.