The Patient as Collaborator: How Personalized Medicine is Giving Back to the Patient

While the advancement of personalized medicine hinges on collaboration among all stakeholders from researchers to industry to clinicians to policymakers, the ultimate stakeholders in personalized medicine efforts are the patients themselves.  In Total Cancer Care™, patients are not only the ultimate beneficiary, but also the major contributor to the effort.  More than 90% of patients who are invited to participate in the Total Cancer Care™ Protocol accept this offer.  This high participation rate is primarily a reflection of the intrinsic altruistic nature of patients and their desire to contribute to the solution through research.    We formed a Patient Advocacy and Ethics Council to assist us in developing and implementing the Total Cancer Care™ Protocol.  We asked this group, “What can we give back to the patient, not just those patients who may develop recurrent disease, but also those who may be cured by initial therapy?” (Approximately 55% are long term survivors.)  Without hesitation, the council told us that all patients desire to have access to their own information in a usable and understandable format.

To that end, we developed a Patient Portal to the data warehouse which provides patients with their own medical histories, data and other important information.  Under the leadership of Mark Hulse (formerly of Partners Healthcare) and Dr. David Fenstermacher, we began this effort at Moffitt in October 2009, and we are gradually extending the portal access to all patients.   Our goal is to extend this service and resource not only for patients at Moffitt but for all patients at all consortium sites.  Much work needs to be done in this area and requires a much improved “real-time” information system.  We also are developing the system to not only be a repository of patient’s personal health records, but a portal where the patients can use the information to make informed decisions.  Again, working with the Institute of Human and  Machine  Cognition (IHMC) we are developing virtual learning technology and applying a process called C-map tools, originally developed at IHMC, to assist patients and physicians to navigate the Internet resources to ultimately meet patient’s needs.

In summary, these are very exciting times.  I truly believe we are at the threshold of translating and just as importantly, DELIVERING on the promise of personalized medicine.  We hope that our effort in developing Total Cancer Care™ will be a major part of the foundation of what some day will be considered common place—a healthcare system and technologies that are organized in such a way that every patient’s needs are identified and inform an individualized approach to meet their needs.  Primary stakeholders in developing personalized medicine, including researchers, clinicians, industry, policymakers, and patients themselves must come together to organize the framework and environment to promote personalized medicine.  It is unrealistic to expect any one stakeholder to collect the resources needed to create a rapid learning information system that will be required to capture data, leverage and enhance informatics needed for analysis, and communicate new knowledge to all the stakeholders involved in developing a better healthcare system built on the foundation of personalized medicine.  Teams comprised of broad expertise across the healthcare and research spectrums, and an unprecedented effort by all will be required to exploit the advantages of the necessary team science approach.  To complement the scientific infrastructure and technology that has already been developed, additional resources will be required including expanding information systems to community hospitals and physician practices, such as electronic medical records, biomedical informatics applications, and information technology professionals.  Ultimately, by developing evidence-based healthcare systems, we will improve the quality of healthcare by identifying best options for patients based on their personal traits and characteristics; such is the promise of personalized medicine.

The Role of Comparative Effectiveness Research in Total Cancer Care™

In my previous entry, I discussed how the launch of the Total Cancer Care™ initiative at Moffitt Cancer Center nearly eight years ago led to the development of one of the largest prospective observational studies in the world.  Through the enrollment of more than 60,000 patients and collection and genetic profiling of tens of thousands of tumors, Total Cancer Care™ collaborators have generated a vast information system to be leveraged as a clinical decision tool, and as a means of quality performance and comparative effectiveness research (CER).

One of the stated aims of Total Cancer Care™ is to raise the standard of care for all patients by integrating new technologies in an evidence-based approach to maximize benefits and reduce costs.  Although we developed this aim over seven years ago, I believe it is completely consistent with the current definition of comparative effectiveness being used by AHRQ and other policymakers. 

As I mentioned in my previous entry, strategic partnerships are an essential component to achieving the goals of Total Cancer Care™, and this is clearly demonstrated in our efforts in CER. Dr. David Fenstermacher and colleagues from Moffitt as well as the Institute of Human and  Machine  Cognition (IHMC), in Pensacola, Fla., are collaborating on a major NIH/NCI grant to enhance the Total Cancer Care™ infrastructure to support CER by expanding data management resources, integrating  automated data extraction methodologies (including natural language processing technology a particular area of expertise for IHMC), and creating user interfaces to data for researchers, clinicians and even patients. 

A major focus of our current efforts in CER is to determine the information and technology gaps in the CER infrastructure for data capture and data sharing.  Ultimately, it will be important to involve the community at large who are enrolling patients in the Total Cancer Care™ Protocol so that they can use the Total Cancer Care™ data warehouse as a decision tool based on evidence generated by the study itself.  The importance of the community network cannot be over emphasized both for populating the Total Cancer Care™ biorepository and database, and the ultimate utilization of the information and evidence generated for delivering the right treatment for the right patient.

To enhance Moffit’ts ability to establish this large research initiative the cancer center formed a wholly owned for-profit company, M2Gen, in 2006.  Merck and Co., Inc., through a Merck affiliate, signed on as our ”Founding Collaborator”. This experience has taught us how to service a global healthcare client and produce measurable scientific insights to accelerate drug candidates through translational medicine advances.

Using Cancer(s) as a Model for Advancing Personalized Medicine

I am pleased to be attending this year’s Personalized Medicine Conference taking place this week and appreciate the opportunity to discuss why I, and many others, believe that cancer, or to be more accurate “cancers”, are an ideal model for the discovery, translation and delivery of personalized medicine.  Sheer numbers alone are reason enough to highlight the need for developing a personalized approach to cancer care: 1 out of 2 men and 1 out of 3 women will develop cancer in their lifetime in the United States.

The sequencing of the human genome has unraveled many mysteries as to how a normal cell can go awry and become cancerous. Further understanding of not only the genetics of cancer, but also the biology and metabolism of cancer, have increased our knowledge of biologic systems that support cancer progression, and this new knowledge has been translated into novel strategies for early detection, prevention and treatment.  And yet, these new discoveries that have heightened expectations of success have, in large part, fallen short in delivering cures anticipated by society.  The reality is that we have learned that cancer is actually an array of many diseases masquerading under the single name of “cancer.”   We need to learn to embrace the complexity of this disease we call cancer and stop the attitude of tunnel vision to cure cancer, and instead focus more on caring for the patient, the individual.  National policy must promote the search for solutions, not just cures.  Ultimately by providing solutions, we will reduce, and in many cases, eliminate death and suffering due to cancer.

In order to accelerate what is a continuum of discovery, translation and delivery of personalized medicine, or in this case personalized cancer care, multiple stakeholders must come together to pursue and deliver this common goal.  These stakeholders include researchers, clinicians, administrators (including policymakers and regulators), and of course, patients themselves.  This week’s gathering of the personalized medicine community in Boston is an example of the value of physically bringing together these stakeholders, and is an important forum for the discussion, exchange of ideas, and aligning of objectives that are crucial to the advancement of personalized medicine.

Total Cancer Care: An Approach to Creating Solutions for the Advancement of Cancer Research and Improved Care

Building on the themes of this year’s event – Personalized Medicine: Impacting Healthcare – I would like to describe to you one approach to personalized cancer care that is having a significant impact on the lives of patients. Nearly eight years ago, the Moffitt Cancer Center in Tampa, Fla., launched the Total Cancer Care™ initiative with the goal of identifying all the needs of a patient and developing a means to meet those needs.  By focusing on solutions to meet individual needs, we believed we could reduce death and suffering due to cancer, and that in order to do so we needed to develop strategic partnerships to perform the five following aims:

1. Create a system to identify the needs of individual patients
2. Identify markers that would predict needs and risks so that interventions could become preemptive
3. Identify molecular signatures for patients who are not likely to respond to standard of care
4. Utilize clinical characteristics and molecular profiling techniques to match the right patient to the right treatment at the right time and the right place
5. Raise the standard of care for all patients by integrating new technologies in an evidenced based approach to maximize benefits and reduce costs

Critical to the pursuit of these solutions was the development of a large regional cancer biorepository in parallel with the development of a relational data warehouse and an information system containing patient’s clinical data and molecular data. 

We soon recognized that although Moffitt had a large patient population to study and engage in this endeavor, to accomplish our goal, we ultimately needed hundreds of thousands (if not millions) of patients to study, and we sought the advice and support of our Florida network of hospitals and physicians.  To our pleasure, there was universal enthusiasm from our statewide colleagues to participate in what became the Total Cancer Care™ Protocol.  Dr. Tim Yeatman at Moffitt was the original Principal Investigator of this IRB- approved protocol which enrolled its first patient in 2006 and basically asked for patients’ consent to do three things:

1. Can we follow you throughout your lifetime by collecting and storing your clinical data and information?
2. May we study any excess tumor or normal tissue using molecular profiling techniques?
3. May we re-contact you?

What began at Moffitt within a year had been extended to eight different communities in Florida.  Within two years, this effort expanded to nine more communities in 10 different states, for a total of 18 participating sites.  Together, the 18 different sites form the Total Cancer Care™ Consortium with the aim of informing and consenting patients to the Total Cancer Care™ Protocol.  As of November 1, 63,754 patients have consented to the Total Cancer Care™ Protocol; 21,331 tumors have been collected and stored in a high technology biorepository located at Moffitt; and 15,093 tumors have been profiled using gene expression profiling technology.  To my knowledge, this effort makes Total Cancer Care™ one of the largest, if not the largest, prospective observational studies with tumor collection in the world. 

Ultimately, our community colleagues are not only contributors to establishing the foundation of personalized cancer care, but also the beneficiaries by being able to use the information system as a clinical decision tool, and as a means of quality performance and comparative effectiveness research.

In coming weeks, I am looking forward to further discussing the role of comparative effectiveness research in Total Cancer Care ™, and how the patient is not only a participant, but also the ultimate beneficiary of everything we do.