Archive for June, 2011

How Personalized Medicine is Changing Cancer Care – A First Hand View

June 28, 2011

I care for people with melanoma. To give you an idea how personalized medicine is impacting care, take the example of a patient I treated at Duke Comprehensive Cancer Center 18 months ago – let’s call her Sarah.

A 37-year-old nurse, Sarah and her husband were trying to start a family. Like many melanoma patients she had fair skin and red hair. Her mother died of melanoma. She came to me after her surgeon had removed a small black-pigmented skin growth and lymph nodes under her arm. After complete review of her clinical story we diagnosed her cancer as Stage IIIB (“3B”) melanoma. Reviewing cancer statistics and matching them to Sarah’s disease tells us that Sarah had about a 50% chance of surviving 5 years.  In order to maximize her chances of long-term survival without melanoma, together with Sarah, I was trying to decide on a treatment and care plan that would reduce the chance of the melanoma returning; this is called adjuvant treatment. Sarah and I were considering whether an adjuvant treatment plan was right for her, and if so, what medicine, for how long, and with what personal impact on her life.

At this point we have one chance to select the right treatment plan, because melanoma that returns is more difficult to treat and rarely goes away for good. You go to the “cookbook,” look up the treatment guidelines and try to balance the standard-of-care with what you know about the individual needs and preferences of the patient.  In Sarah’s case, finding a balance between effective adjuvant treatment, preserving her ability to have children, and helping her maintain her job were important considerations.  The treatment guideline advised interferon, a clinical trial or no treatment, but we had few clues as to the best treatment path and certainly no information about fertility or the influence of Sarah’s genetic makeup on her ability to derive benefit from treatment.

In the year and a half since I treated Sarah, we now have new tests to predict risk and learn more about the specific tumor each patient has. Molecular descriptors about the cancer and striking radiological images provide important clues. The information available to us is getting better and better, which makes it more likely that we will hit the mark in the one shot we have to find the right treatment. In the future, I anticipate that other factors such as Sarah’s heritage, her symptom profile, environmental exposures, and personal values will also be incorporated into the decision-making process.  Sarah’s case reminds us that these are real people undertaking real and exceptionally critical decisions; we need to be able to harness all available information to maximize the chance that we make the right decision for Sarah and patients like her.

At the same time this growing wealth of information brings new challenges for physicians. The “cookbook” does not work well as we gather more detailed information about potential risks, likely side effects, and benefits of different treatment combinations. 

Physicians care about getting these decisions right for each patient. The current “cookbook”, though, is one-size-fits all. We need information science and technology (IT) to help match guidelines and predictive mathematical models informing patient care with new information available about individual patients and the type of tumor they have.  And it has to happen at the point of care, when we need it. The amount of information can be overwhelming. Evolving data systems and IT infrastructure that are reliable and trustworthy will be key to making the most of the information available. Along with this, we simply need more time with patients. We often have about 8 to15 minutes to make a decision with the patient that will affect their entire future. This is not what patient-centered care and personalized medicine are all about.

Someday I hope that when a patient like Sarah comes in I can look up her exact type of tumor, incorporate her personal family history and other medical information into the story, see what treatments she is likely to respond to, understand the potential risks, and balance her personal concerns like maintaining her fertility. Personalized medicine is advancing quickly, but we need information systems to support good decision-making for physicians and patients as the information available continues to expand exponentially.

The Ultimate Patient-Centric Solution

June 21, 2011

Personalized Medicine is the ultimate in patient-centric solutions.

Emerging personalized services can help guide treatment not only on efficacy, but also on likely side effect profiles, helping physicians select therapies to maximize the patient experience and shape that experience to patient preferences and tolerance levels.  My personal story exemplifies these possibilities.

I have experienced the promise of personalized medicine first hand. As a liver transplant recipient, I have to be constantly monitored to ensure that my body is not rejecting the transplanted organ nor leaving me open to life-threatening infections. Traditionally, this is done with painful and invasive liver biopsies as well as blood testing.

Recently, a new blood analysis-test was developed that can assess my immune system’s individualized response to the immunosuppressive medications I must take.  This test gives unique information about my immune system and the degree to which I am at risk for rejecting my organ or to vulnerable to infections. Using this information, my physician can determine the appropriate combination and dosing for my immunosuppressive medications.

 Although this test is FDA-cleared, current policy does not provide a level of reimbursement that accurately reflects the value of this new technology. Without proper funding and support, there is a strong risk that the test, and others like it, will be removed from the market.

If this happens, we will be taking a huge—and costly—step backward in patient care. Removing this test from the market would mean a return to invasive and expensive liver biopsies and a trial-and-error approach to managing my immunosuppressive medications. This would not only put me at significantly increased risk of hospitalization and organ rejection or retransplantation, but would also incur a much greater expense to the health care system. All these things are easily avoidable through the use of a personalized medicine test, but require its value-based reimbursement.

The patient experience is often marred by pain, fear, and confusion, particularly in the face of a life-threatening condition.  Personalized medicine can provide answers. As my experience shows, meaningful information to guide treatment and prevent adverse events can, indeed, be the best medicine.

Castellani Calls for Collaboration, Continued Hard Work at PMC Annual Luncheon

June 10, 2011

At yesterday’s Seventh Annual State of Personalized Medicine Luncheon, I couldn’t help but reflect on how far the field of personalized medicine has come. John Castellani, President and CEO of the Pharmaceutical Research and Manufacturers of America (PhRMA) delivered the keynote address, and Dr. Werner Verbiest, head of Johnson & Johnson’s Companion Diagnostics Center of Excellence, introduced him. Thinking through the varied and distinguished background of the other speakers at our Annual Luncheon over the years (including Dr. Margaret Hamburg, Commissioner of the Food and Drug Administration; Dr. Denis Cortese, President and CEO of the Mayo Clinic; and Dr. Francis Collins, then Director of the National Human Genome Research Institute, among others), I see strong representation of the collective commitment from the biopharmaceutical industry, government research institutes and agencies, and patient care providers to the advancement and provision of personalized medicine.

While much progress has been made in promoting an understanding of personalized medicine and widening the circle of those working to move it forward, as Mr. Castellani reminded us yesterday, there is still much work to be done. He emphasized that we cannot work alone in our silos, but must come together to “drive the science forward and create an environment that embraces change and opens the door to personalized medicine.”

Mr. Castellani mentioned several policy challenges that must be overcome, and this is an area of particular priority for the PMC. We have recently formed a workgroup that is developing legislative specifications for consideration by Congress in many of the areas Mr. Castellani highlighted, such as:

  • Ensuring that policy and regulation provide appropriate incentives for companies engaged in the research and development of personalized medicine;
  • Aligning comparative effectiveness research methodology to illuminate differences among patient subpopulations;
  • Improving patient access and sustaining innovation through reimbursement and payment policy reforms for treatments targeted to small patient groups;
  • Improving the regulatory environment for targeted therapies and companion diagnostics; and
  • Working with providers in areas such as physician education, health information technology and clinical decision-support so that caregivers have the information they need to apply evidence-based, personalized medicine at the bedside.

I’d like to thank Mr. Castellani and Dr. Verbiest for their participation in yesterday’s event. Calling to mind the 30th anniversary of the discovery HIV/AIDS, both speakers underscored how much can be accomplished when research and clinical care come together and are spurred on by the daunting challenges patients face, particularly where there is significant unmet need. In the same way, those of us committed to improving health care quality through personalized medicine and encouraged by our ever-increasing knowledge of the molecular basis of human disease are ultimately inspired by the patients who will benefit from a more targeted, effective approach to medical care.

The State of Personalized Medicine: Great Progress, Yet Looming Challenges

June 10, 2011

Yesterday I had the opportunity to speak at the Personalized Medicine Coalition’s Annual State of Personalized Medicine Luncheon. As I told that group, I am excited by the power and potential of personalized medicine, and humbled by the daunting challenges before us. 

I am excited by the potential to address unmet medical needs of patients with cancer, HIV/AIDS, and many other serious diseases. I also see the great promise of personalized medicine to help us meet the challenge of rising healthcare costs by avoiding treatment complications and making sure each patient gets the most effective care possible.

The biopharmaceutical research sector is strongly committed to advancing personalized medicine. A recent survey by theTufts Center for the Study of Drug Development found that 94% of our companies are investing in personalized medicine, and have increased that investment by 75% over the past five years.  As we saw with some of the exciting findings reported this month at the American Society of Clinical Oncology, this investment is beginning to yield results, promising better treatments for melanoma, lung cancer and other diseases.

Despite the promise and the deep commitment of biopharmaceutical companies to advancing personalized medicine, I am also truly humbled by the challenges we must overcome for the sake of patients who need these advances. The obstacles are broad and include scientific, business, regulatory and policy barriers.

Together we must break down the barriers and move this field forward. I urge the personalized medicine community to work together to accelerate the field for the benefit of our healthcare system and patients everywhere.

For Cancer Therapies, It’s Getting Increasingly Personal

June 6, 2011

Last November, the Tufts Center for the Study of Drug Development reported that more than 90% of biopharmaceutical companies are investing in personalized medicine, with 12-50% of compounds in the drug development pipeline qualifying as personalized medicines. According to the report, personalized medicine is having an impact across disease areas, but is most evident in new therapeutic approaches to oncology.

The latest research reported at this year’s annual meeting of the American Society of Clinical Oncologists taking place over the weekend in Chicago reflects the same momentum and enthusiasm for the potential of targeted therapies in advancing the treatment of cancer. A front-page article by Ron Winslow in today’s Wall Street Journal, “Major Shift in War on Cancer,” explores some of the exciting personalized medicine therapies under development for cancer, highlighting new data presented at ASCO.

Winslow notes, “New research is signaling a major shift in how cancer drugs are developed and patients are treated—offering the promise of personalized therapies that reach patients faster and are more effective than other medicines.” The article nicely illustrates the promise of the science as well as some of the challenges facing researchers, companies and regulators.

This Thursday, PhRMA President and CEO John Castellani will continue the discussion of promising advances on the horizon for personalized medicine at the Personalized Medicine Coalition’s Seventh Annual State of Personalized Medicine Luncheon at the National Press Club. We hope to see some of you there. To register for the luncheon, visit:

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