Advancing Personalized Medicine through Research, Collaboration, and Innovation

The healthcare community is at an important juncture; we must work together to ensure that policies and regulations align to help us meet the unique challenges and full potential of personalized medicine. Science and technology drive what we can achieve, but they also evolve over time. We, too, must evolve and adapt to this rapidly changing environment.

Indeed, as I told participants at the 9th Annual Harvard Personalized Medicine Conference , the continuing development of personalized medicines holds promise to define our age and change everything we do. These treatments will affect how we approach once seemingly intractable medical challenges, how we research and develop new medicines, and how patients interact with physicians and our healthcare delivery system.

The biopharmaceutical industry is strongly committed to researching and developing personalized medicines.  A Tuft’s University survey found that biopharmaceutical companies increased their investment in personalized medicines by 75 percent between 2005 and 2010. In fact, between 12 and 50 percent of all compounds currently being researched are potential personalized medicines, which are targeting a growing range of diseases that may be identifiable through a genetic test or biomarker – from depression to obesity to many forms of cancer. Through these advances, we are shifting from reactive to proactive medicine, possibly enabling us to identify a patient’s risk before symptoms appear, and to transform our approaches to diagnosis and treatment.

But with these hopes, there are hurdles.  We all know that we need forward-looking policies that foster innovation and a regulatory system that is in line with advances in science. But we also need strong collaboration among biopharmaceutical companies, academics, patients, hospitals, government labs and others to pave the way for the science and its application.

Achieving the full power and potential of personalized medicine is an urgent priority. But it is an attainable goal only if we work together. On behalf of our patients, I urge everyone in our healthcare community to continue working across sectors and disciplines to advance personalized medicine.

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A version of this blog originally appeared in The Catalyst posted at the PhRMA website.

Personalized Medicine Coalition Honors MMRF’s Kathy Giusti with 2013 Leadership Award

Edward Abrahams, Ph.D., President, PMC, (left) and William S Dalton, Ph.D., M.D., Director, Personalized Medicine Institute, Moffitt Cancer Center and CEO, M2Gen, recognize 2013 Leadership in Personalized Medicine Award recipient Kathy Giusti, Founder & CEO of the MMRF (Photo: Justin Knight)

The personalized medicine community has convened in Boston for the Ninth Annual Personalized Medicine Conference. Last night, the Personalized Medicine Coalition welcomed more than 250 attendees to the Boston Museum of Science and the new Hall of Human Life exhibit to kick off the two-day event. As colleagues gathered, the prevailing conversation focused on how far personalized medicine has come. It has been nine years since we first gathered researchers, academics, policymakers, clinicians, patient advocates and other stakeholders to identify the key questions facing – at the time – an emerging approach to healthcare.

Then, and over the years since we first gathered, we have often spoken of the “promise of personalized medicine.” Today, as the Personalized Medicine Conference opened, we focused on how that promise has come to fruition. The personal stories of panelists who carry a dual title of “healthcare expert and patient”, or “genomics expert and caregiver”, riveted the audience and provided a poignant reminder of the need to continue to encourage and enable innovation and access to personalized approaches to care.

One leader in the field, who embodies the promise of personalized medicine is Kathy Giusti, Multiple Myeloma Research Foundation (MMRF) Founder and CEO. This year, the Personalized Medicine Coalition honored Kathy with the 2013 Leadership in Personalized Medicine Award. The award recognizes an individual whose work and contributions are advancing personalized medicine research, product development, reimbursement and policies. Kathy Giusti has done all that and more through her passionate and driven work to empower patients and advance research in multiple myeloma.

The unique and creative business model Kathy employs at MMRF has yielded both clinical and personal results. Clinically, MMRF has funded research and paved the way for FDA approval for six multiple myeloma treatments in 10 years, and doubled the lifespan for many patients. Today in her remarks, Kathy shared that her daughter, who was just one year old when Kathy received her myeloma diagnosis, is now a 19-year-old college student with a younger brother. Incredible results.

The Personalized Medicine Conference continues to be an important gathering for information sharing, consensus development, and, just as critically, these inspirational moments that remind us all that the Age of Personalized Medicine is here as we continue to gain more evidence each day that the science is moving us toward more personalized approaches to research and care.

As Kathy shared at the podium, “I accept this award on behalf of all of you, because you are here as innovators in a field where patients desperately need you. You are here because you are innovators in the field of personalized medicine. You are going to change the lives of thousands and thousands.”

Strategies for Accelerating Progress in Personalized Medicine

Last year around this time, I wrote a blog aimed toward skeptics of personalized medicine, citing the progress we have made in lung cancer as just one example of how well this approach works for treating diseases. As I have reflected upon the advances we have made in areas like cancer and diabetes and how personalized medicine now seems to be the cornerstone for drug discovery and development for many pharmaceutical companies, it has become clear to me that there are still a number of areas for continued investment and focus.

What can we do to continue to grow the field of personalized medicine, find new and exciting therapies, and maintain the momentum we have seen over the past few years?

I believe that developing a personalized medicine strategy is the answer, but this is far from simple.

It begins by first appreciating that one needs to study the diversity inherent in diseases. When a new medicine is introduced in human clinical trials, for example, it is important to describe and understand the diversity of human responses to the medicine and to determine ways to identify those who will benefit the most from the medicine.

While we would ideally like to have such a clear scientific understanding of a disease that we can predict in advance who the likely benefiting patients are (which can be done in some instances), in most cases the personalization of a medicine comes from the observations made in clinical studies of diverse populations.

In contrast to basic laboratory research, where experimental designs seek to minimize variation, the “noise” in clinical trials can actually provide us with important clues to help form the basis of a personalized medicine. Well-designed studies, therefore, should study both the variation of the response as well as the scientific basis for that variation. When done well this can uncover biologic marker that become diagnostic agents for patient selection.

In addition to designing more clinical trials with these two aspects included, I would propose that those of us working in the field of personalized medicine should continue to support efforts that:

• Fund basic research on the biologic basis of disease. Personalized medicine is based on fundamental scientific discoveries. Without these, there will be no improvement in our understanding of disease.
• Fund research in diagnostic testing. Companion diagnostics can be useful tools to help healthcare providers and their patients make more informed treatment decisions.
• Educate physicians and other healthcare providers about personalized medicine. Patients in specialized populations that may respond to a good therapy for their subgroup need to have access to the appropriate tests. This means making sure that those who can order the tests and use them as a part of standard care are aware that these tests and potential therapies exist.
• Reimburse for diagnostic testing. Personalized medicine drugs will not have any benefit if they are not used and a healthcare provider won’t know to use a potentially life-saving drug unless the correct diagnostic tests have been performed.

Together, we can maintain and even accelerate the progress we have already made in delivering the right drug to the right patient at the right dose, every time.

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Join Dr. Stephen Eck on November 6, 2013 at the 9th Annual Personalized Medicine Conference hosted by Partners HealthCare Center for Personalized Genetic Medicine, Harvard Medical School, and Harvard Business School. 

View Dr. Eck’s video interview “Personalized Medicine: How Progress Happens” online at the Personalized Medicine Coalition.