Archive for November, 2010

For BioPharma Enterprise It’s Full Speed Ahead into the Age of Personalized Medicine

November 23, 2010

As more is understood about diseases and the why and how of their effects on people through advances in biomarkers and genomics, personalized medicine becomes a natural result for biomedical science and a natural trajectory for the innovation-based biopharma industry.   While the majority of those in attendance at last week’s Personalized Medicine: Impacting Healthcare  Conference are deeply immersed in driving this transformation, I was pleased to present the results of  a new Tufts CSDD Impact Report that provides the first look at a year-long effort by the Tufts Medical Schools Center for the Study of Drug Development (Tufts CSDD) to measure progress and prospects in the field by means of interviews and a survey of some 20 companies on the front lines of the movement towards personalized medicine. The company sample consisted of 9 top biotech companies with an average market value of $22B and 12 Big Pharma firms with an average market value of $93B.

While many recognized the challenges ahead, considering the current level of scientific knowledge, all believe that eventually a personalized medicine approach will help to streamline the R&D process, tip the benefit/risk ratio in favor of product approval, and provide options that help reduce overall costs and side effects. Confirming firms’ commitment to push ahead, companies increased their investment in personalized medicine by a median of 30% from 2006 to 2010 (even in a “down” economy), and plan to do so again from 2011 to 2015. Structural change follows the funding flow and all companies have experienced some level of “internal paradigm shifts” in order to integrate the concepts of personalized medicine into their development process, but not without disruptive change in the way decision-making occurs and at what levels.

Regulatory and reimbursement hurdles loom large among the most immediate challenges affecting the advancement of personalized medicines.  Thus, 100% of companies say their discovery strategy involves biomarkers and/or targeted therapies, but having a biomarker was not a requirement to move into clinical development. In fact, the percentage of the pipeline relying on biomarker data decreased as products move downstream, from 60% in preclinical to 50% in early clinical development, and finally 30% in late development. Oncology is much further along with genomic targeting of medicines, but  other therapeutic areas ahead of the curve include cardiovascular, central nervous system and immunologic therapies, while in metabolic, virology, and respiratory research,  personalized medicine is growing but incipient.

The dichotomy of where the industry is at present from where it needs to be is evident from the fact that while 90% of companies say they are investing in personalized medicine, only 10% of compounds in late clinical development actually have companion diagnostics. Nonetheless, the industry commitment in terms of funding, research collaborations, technology uptake, organizational change and unanimous resolve to push forward could signal that rapid advances are on the horizon.

 To read more or purchase the full Tufts Impact Report visit:  http://csdd.tufts.edu/reports/description/ir_summaries

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

November 17, 2010

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.

Personalized Medicine Coalition Award for Leadership Awarded Moffitt Cancer Center CEO

November 17, 2010

Today at the Personalized Medicine: Impacting Healthcare conference, the premier gathering of leaders in the personalized medicine field, the Personalized Medicine Coalition presented its annual award for Leadership in Personalized Medicine to Moffitt Cancer Center President, CEO and Center Director William Dalton, Ph.D., MD.

The award, which recognizes an individual whose contributions in science, business, and/or policy have helped advance the frontiers of personalized medicine, is presented annually at the personalized medicine conference sponsored by Partners HealthCare Center for Personalized Genetic Medicine, Harvard Medical School and Harvard Business School.

Giving this award to Dr. Dalton sends the message that personalized medicine is no longer a promise of the future. It currently has the capacity, especially in cancer, to ensure that the right patients receive the right treatments at the right time.  Under Dr. Dalton’s leadership, Moffitt is paving the way towards a new era of customizing treatments based upon individual characteristics.

Dr. Dalton is also receiving the award in recognition of his leadership in advancing public policies designed to support personalized medicine innovation and clinical application and for Moffitt’s pioneering work to implement personalized medicine concepts both to improve patient outcomes and lower costs.     

He is a leading advocate for health policies that support personalized medicine for the benefit of patients and currently serves as the President-Elect of the Association of American Cancer Institutes (AACI) and is Chair of the Science Policy & Legislative Affairs Committee of the American Association for Cancer Research. 

Dr. Dalton’s work is on the leading edge of personalized cancer care and patient-centered outcomes research. Moffitt’s Total Cancer CareTM is an approach to enhancing access to evidence-based, personalized cancer treatments and information/decision tools for patients and clinicians. Total Cancer CareTM is one of the largest cancer tumor bio-repositories and data warehouses in the United States dedicated for use in development of personalized medicine. 

As the recipient of the PMC award, Dr. Dalton joins a group of innovators well recognized for their leadership in personalized medicine. Previous recipients of the award include Dr. Janet Woodcock, Director of the Food and Drug Administration’s Center for Drug Evaluation and Research, Dr. Elizabeth G. Nabel, former Director of the National Heart, Lung and Blood Institute at the National Institutes of Health, Michael Leavitt, former Health and Human Services Secretary, Dr. Ralph Snyderman, Chancellor Emeritus of Duke University and founder of Proventys, Inc., and, most recently, Brook Byers of Kleiner Perkins Caufield & Byers.

Welcome to Personalized Medicine: Impacting Healthcare

November 17, 2010

At this morning’s opening session of the Sixth Annual Personalized Medicine Conference, I welcomed conference participants with a quote from Charles Dickens to describe the current environment in personalized medicine:

“It was the best of times, it was the worst of times, it was the age of wisdom, it was the age of foolishness, it was the epoch of belief, it was the epoch of incredulity, it was the season of Light, it was the season of Darkness….”

There are many that believe we are experiencing the “best of times” in personalized medicine as the field has experienced continued growth and is being implemented in clinical practice. There are others who believe personalized medicine has over-promised and under-delivered. Certainly the theme of this year’s event, Personalized Medicine: Impacting Healthcare, will explore the progress that has been made in the past six years since the conference first began. The representation at this year’s event from across stakeholders in the biopharmaceutical industry, commercial entities, research and academia, and policymakers are a reflection of the groundswell taking place to make personalized medicine a reality.

Nevertheless, to demonstrate the lively debate on the issue, I asked those attending the conference to share their opinion on whether the field of personalized medicine is achieveing its mission, and via an audience response system, received the following feedback:

  • 26.4% believe personalized medicine is being achieved at a satisfactory rate
  • 67% believe personalized medicine is being achieved too slowly
  • 6.6% had no opinion

But what do you think? Use the poll below to share your response.


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