Archive for June, 2012

Congress Recognizes Value of Personalized Medicine Approaches in FDA User Fee Authorization Legislation

June 27, 2012

Regulatory uncertainty has long been a concern for personalized medicine innovators and the Turning the Tide Against Cancer conference earlier this month reinforced the need for regulatory changes to facilitate personalized medicine.  While that uncertainty is not gone, the Food and Drug Administration (FDA) user fee reauthorization legislation, which passed its last major hurdle through Congress yesterday, contains personalized medicine-relevant policy changes that will enhance the regulatory process for new products.

We applaud Congress for passing this legislation and commend the measures it takes to smooth the way for more personalized medicine products.  Provisions directly related to personalized medicine include:

  • The development of FDA’s capacity to review submissions involving pharmacogenomics and biomarkers by integrating and training staff with pharmacogenomics and biomarker expertise into the product review process.
  • The creation of an expedited approval process for “breakthrough therapies” — drugs that are both developed using biomarkers and used to treat serious or life-threatening ailments.
  • A requirement that the FDA notify Congress two months in advance of the Agency’s planned release of any guidance or draft guidance document related to the regulation of laboratory-developed tests.

We look forward to continuing to work with FDA to improve the complex regulatory process for personalized medicine and accelerate adoption new personalized treatments.

From The Catalyst (blog) – Conversation with Margaret Foti, Ph.D., M.D. (h.c.), CEO, American Association for Cancer Research

June 11, 2012

This entry is reposted with permission from PhRMA’s blog, The Catalyst.

ASCO’s Annual Meeting this week highlighted some of the exciting advances that are emerging in the fight against cancer. PhRMA has joined with many other organizations in supporting a conference next week, “Turning the Tide Against Cancer Through Sustained Medical Innovation,” which is focused on the critical issue of how we sustain this progress in an era of increasing cost-cutting pressure. Conference participants will examine how we measure the value of new treatments and how we can promote high quality, patient-centered care.

The conference is being convened by the American Association of Cancer Research, the Personalized Medicine Coalition, and Feinstein Kean Healthcare. We sat down with each of the conference co-hosts about the event and progress in cancer care.

Conversation with Margaret Foti, Ph.D., M.D. (h.c.), CEO, American Association for Cancer Research


Q: In looking at where we want to go in progress against cancer, we sometimes lose sight of where we’ve come. Set the stage for us – how has scientific progress changed the outlook for patients against cancer in recent years? 

Marge Foti: We have made tremendous progress against cancer.  There are more than 12 million cancer survivors in America today and cancer mortality rates continue to decline. This progress is thanks to scientific discoveries that have revealed the underlying biology of a cancer cell and how the survive and grow in our bodies—knowledge that has given us a new understanding that cancer is not, in fact, a single disease but more than 200 diseases, each with unique causes and characteristics that require different treatments. New advanced technologies have improved our ability to turn this knowledge of how cancer works against cancer. We now are better able to prevent, detect and treat cancer.

Q: AACR put out a great report last year, the Cancer Progress Report, which describes many of the exciting advances being made in cancer research. What big trends do you expect we will hear about at the upcoming conference? What big challenges? 

Marge Foti: Personalized medicine is more than just a buzz word; it is offering great promise to transform the outlook for many forms of cancer. We are truly entering an era when every patient’s tumor can be characterized at the molecular level, which is helping us redefine how we prevent, diagnose, and treat cancer.  While this unprecedented progress is the direct result of fundamental discoveries from the past 40 years, further exploiting this knowledge to improve the outlook for cancer patients is going to require that our national policies keep pace with the continual advances that take place in science.  For example, the recent approval of two new cancer medicines along with companion diagnostics is marking a new day of progress, but also is one that poses new challenges to researchers, companies, and regulators.

In addition, cancer’s complexity is astounding and exists at all levels–from populations, to individuals, to specific cancers, to the genes that underlie cancers.  A major hurdle in the treatment of cancer is that that there is a high degree of variability in gene mutations—even within a single tumor in a single patient—and the variability increases with later stage disease.

Cancer research takes time and progress builds over time as new treatments are added on to one another in a step-wise fashion. Our growing scientific knowledge has the potential to change the face of drug development, to combine therapies in a rational way based on the mutations within an individual patient’s tumor. This is an enormous scientific and regulatory challenge, but the science is telling us this is the path forward.

Q: Why is it important to connect the science to policy?

Marge Foti: Continued progress is not a given. Advancing care for patients is an incredibly long, complex and expensive process. We need to provide continued support for basic research, and we need to preserve strong incentives for private sector investments in this process. Without a good understanding of the science, we risk taking policy approaches that will hamper the progress we so desperately need to make for current and future cancer patients.

From The Catalyst (blog) – A Conversation with Marcia Kean, Chairman, Feinstein Kean Healthcare

June 11, 2012

This entry is reposted with permission from PhRMA’s blog, The Catalyst.

ASCO’s Annual Meeting this week highlighted some of the exciting advances that are emerging in the fight against cancer. PhRMA has joined with many other organizations in supporting a conference next week, “Turning the Tide Against Cancer Through Sustained Medical Innovation,” which is focused on the critical issue of how we sustain this progress in an era of increasing cost-cutting pressure. Conference participants will examine how we measure the value of new treatments and how we can promote high quality, patient-centered care.

The conference is being convened by the American Association of Cancer Research, the Personalized Medicine Coalition, and Feinstein Kean Healthcare. We sat down with each of the conference co-hosts about the event and progress in cancer care.

Conversation with Marcia Kean, Chairman, Feinstein Kean Healthcare


Q:  In the course of 30+ interviews of national thought leaders that you conducted to develop a Discussion Paper for the Turn the Tide conference, what themes emerged most strongly? 

Marcia Kean:  I was very surprised at how much consensus there was from such a disparate group of stakeholders from academe, patient advocacy, industry, providers and payers.  There was strong agreement, for example, that as daunting as cancer is, innovation is going to be the best way to overcome both the clinical and economic burden of cancer in coming years, and that above all, innovation has to be protected.

Five major themes recurred.  The first was around the need for an ongoing, community-wide commitment to overcome the issues we face as a society in oncology.  I don’t mean that everyone had the same perspective about those issues – remember, this was a very diverse set of individuals – but rather, there was agreement that we have the opportunity at this moment to do things differently to accelerate research and improve care, and that in order to reward innovation, it’s important for stakeholders to identify overlapping interests rather than remaining rigid in their safe and separate silos.  Creating a joint, defined vision of what constitutes value will help to outline a pathway for development.

Second, while everyone recognizes that we’ve made progress toward patient-centered cancer care, much more needs to be done. We need a better system for measuring value that puts patients’ needs and preferences first, and keeps up with the rapidly evolving scientific and clinical environment. As personalized medicine becomes more central, of course, this kind of dynamic measurement will only become more difficult.

Third, there’s huge excitement about the potentially positive impact of digital technologies to accomplish the dramatic productivity gains in cancer research and care that they’ve done for other fields.  Developing effective tools for shared decision-making and clinical decision support is seen as a major opportunity in that regard.

Fourth, there’s a desire to move to “next-generation” tools for assessing value through Health Technology Assessment, as well as a fervent desire to develop next-generation Comparative Effectiveness Research that aligns with personalized medicine and gives a fuller picture of real-world value. We heard that current approaches aren’t centered enough on patient value, and don’t do an adequate of job keeping pace with how the use and value of tests and treatments evolves over time – they are just too static in a highly dynamic world.

Finally, there’s a strong drumbeat about the shift to models of “continuous learning” that give a more dynamic, patient-centered picture of value, so that every clinical encounter informs our overall knowledge base and research advances are moved rapidly into clinical use.  That virtuous circle is seen as the light on the horizon for all of us.

Q:  Your firm has been very active in efforts to implement health information technology and develop a learning health care system. How will these concepts accelerate progress in cancer care?

Marcia Kean:  In some ways, the field of biomedicine has been the last to benefit from the digital revolution.  For many cultural – and technical – reasons, we just haven’t been able to move data rapidly from lab bench to doctor’s office to hospital to patient, and so on.  But there is a growing cadre of researchers and clinicians and patients who are demanding the kind of ‘data liquidity’ in science and health care that they see in every other sector of their lives, and they are gaining traction.  Once we have seamless data exchange, based on the use of standards in Health Information Technology and bioinformatics tools, we’ll have a solid foundation for a learning health care system. In that picture, everyone wins:  innovators can accelerate and improve research; patients and doctors can get the information they need to make wise decisions; and the efficiency of the process can drive down costs.

However, we need to remember that even a digitally-enabled system doesn’t happen in a vacuum — it has to unfold in a community where every sector is interconnected and working towards a common, patient-centered vision, because all these challenges and solutions are inter-related in a very complex and dynamic ecosystem.

From The Catalyst (blog) – A Conversation with Edward Abrahams Ph.D., President, Personalized Medicine Coalition

June 11, 2012

This entry is reposted with permission from PhRMA’s blog, The Catalyst.

ASCO’s Annual Meeting this week highlighted some of the exciting advances that are emerging in the fight against cancer. PhRMA has joined with many other organizations in supporting a conference next week, “Turning the Tide Against Cancer Through Sustained Medical Innovation,” which is focused on the critical issue of how we sustain this progress in an era of increasing cost-cutting pressure. Conference participants will examine how we measure the value of new treatments and how we can promote high quality, patient-centered care.

The conference is being convened by the American Association of Cancer Research, the Personalized Medicine Coalition, and Feinstein Kean Healthcare. We sat down with each of the conference co-hosts about the event and progress in cancer care.

A Conversation with Edward Abrahams Ph.D., President, Personalized Medicine Coalition

Q:  What are the implications of personalized medicine’s growing role in research and patient care in oncology?

Ed Abrahams:  Technological improvements in genetic sequencing and molecular profiling of tumors have given researchers and clinicians a new ability to understand the molecular and genetic characteristics of cancer patients and their tumors.  By more accurately classifying disease, patients can benefit from targeted treatments that are more likely to work.

Simply put, personalized medicine means better patient care. Personalized treatment regimens allow clinicians to select treatments that are more likely to attack specific tumor types.  But personalized medicine also delivers better value to patients and the health system because it enables clinicians to spare patients the expense and side-effects of treatments that are not likely to provide tangible benefits and thereby to help address the challenge of rising health care costs.

We believe that the future of cancer therapeutics lies in molecular biology and genomics, supported by electronic health data systems and advanced health information technologies that support a rapid learning health care system.  Increased collaboration between academia, the biopharmaceutical industry, philanthropic organizations, and patient advocacy groups will maximize the development of new personalized medicine technologies and treatments.

Q:  What are some of the barriers to personalized medicine that need to be addressed to facilitate the adoption of personalized medicine?

Ed Abrahams:  Clearer regulatory and reimbursement pathways are needed to ensure that developers of new personalized medicine products – therapeutics, diagnostics, and others laboratory services – have a reasonable pathway to get their products approved by regulatory agencies and to ensure that products are reimbursed at rates reflective of the innovative value of the product and the investment necessary to develop it.

We must also aligning comparative effectiveness research (CER) with the science of personalized medicine and the fact that we are not always aware of all of the uses for a new therapy at the time of its approval. If we rush to judgment about value too early, we will cut short the process of continuous learning through which value emerges. If we take a traditional “one size fits all” approach to CER that judges “average” value at a point in time, we will stifle innovation.

Finally, our education system must evolve to ensure that our health care workforce is trained in genetics, genomics, and the technologies needed to integrate personalized medicine into cancer treatment decision-making.

Q:  What is your goal for the conference?

Ed Abrahams: This conference will provide stakeholders a forum to discuss major developments in oncology research and development and link the science of personalized medicine with public policy.

We intend the high-level discussion at the conference to illuminate policy solutions that will spur the development of new personalized medicine approaches to cancer.  By brining patients, payers, industry, and academe, we hope to identify ways to continue to deliver high value care in a cost constrained environment and to sustain innovation to improve cancer patient outcomes.

Progress in Cancer Highlighted by NEJM Retrospective; Turning the Tide Conference to Catalyze Comprehensive Dialogue on How to Sustain Cancer Innovation

June 8, 2012

In honor of the New England Journal of Medicine’s (NEJM’s) 200th Anniversary, the journal examined how medicine has evolved over the last two centuries, looking in particular at oncology diagnosis, prevention, and treatment. But while there has been tremendous progress in cancer, questions remain:  Where do we go from here?  And how do we get there in an era of immense fiscal discipline?  These are questions that we plan to address on Tuesday at our conference, Turning the Tide Against Cancer Through Sustained Medical Innovation.

In a similar vein to what Siddhartha Mukherjee, M.D., a special guest speaker at the conference, lays out in his book The Emperor of All Maladies: A Biography of Cancer, the authors of the NEJM article “Two Hundred Years of Cancer Research” provide a timeline of major discoveries and advances in cancer research and care.

They show how each milestone is built on the ones that came before it and trace the evolution of cancer progress from the early efforts to control the disease through surgery, advances in radiation, chemotherapy, and the targeted therapies that are redefining cancer treatment today.

The underlying science that made these treatment advances possible takes years to translate into clinical benefits for patients, but the original investments pay off. Our understanding of the genetic basis of cancer became possible only after decades of work on the basic biology of DNA beginning in the 1940s and 50s, but it was not until after the sequencing of the human genome that researchers were able to begin to translate genetics knowledge into new medicines.

Genetic understandings of cancer have led to breakthrough new medicines such as Xalkori® (for non-small cell lung cancer) and Zelboraf® (for melanoma) and more targeted therapies are on the way.  A new report issued by the Pharmaceutical Research and Manufacturers of America (PhRMA) found that there are 981 new medicines and vaccines for cancer in development today, many of which are likely to be personalized medicines.

Tomorrow’s progress in cancer therapies and treatment approaches depend on today’s policy makers recognizing the need for policies that holistically support cancer research and innovation.  In advance of next week’s conference, a Discussion Paper “Sustaining Progress Against Cancer in an Era of Cost Containment” coalesces the views of the conference advisory committee and other leading cancer experts about new models for cancer innovation, how to define value in cancer care, and how policy can support continued progress against cancer.


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