Sustaining Progress in Personalized Medicine

Last week, I had the opportunity to speak at the Harvard Personalized Medicine Conference in Boston, MA. No other conference on personalized medicine brings together the array of scientists, stakeholders, and experts that this event does. This year the conference drove home to me that the potential to improve patient care via personalized medicine is greater than ever – yet the scientific and clinical challenges remain daunting. It is more important than ever to sustain biomedical innovation, and to ensure that health policy is informed by the enormous opportunity, and complexity, of making continued progress in this field.

The event also underscored that biopharmaceutical research companies are deeply committed to advancing the science of personalized medicine and building it into their research and development strategies. It affirms findings of a report released by the Tufts Center for the Study of Drug Development in 2010 which found that 94% of biopharmaceutical companies surveyed are investing in personalized medicine and 100% are using biomarkers in the discovery stage to learn about compounds. This research has required large up-front investments in new research tools and training. But, as we have seen in the last year-and-a-half with FDA approval of new targeted therapies for lung cancer, melanoma, and cystic fibrosis, it is starting to bear fruit for patients.

I’m hopeful we’ll see more approvals in the months ahead. In the report from Tufts, companies reported that 12-50% of compounds being researched are personalized medicines and over the last five years, they have seen a roughly 75% increase in their investment in personalized medicines. The importance of personalized medicine was illustrated in the reauthorization of the Prescription Drug User Fee Act, which provides FDA with increased resources and staffing to advance the regulatory science in areas such as pharmacogenomics and biomarkers.

This progress, however, doesn’t happen in isolation. The Harvard Conference participants represented, and illustrated, the wide range of organizations and individuals from different sectors that make up the research ecosystem that drives progress in personalized medicine. As the science of personalized medicine advances, research partnerships and collaborations will be more important than ever. To sustain progress in personalized medicine, it is vitally important to ensure that policy and regulation do not erect barriers to these types of partnerships.

Biomedical innovations like personalized medicine will help address major unmet medical needs, and offer a solution to rising healthcare costs.  As we face continued pressure to contain healthcare costs, it is crucial to ensure that healthcare policy sustains the innovation ecosystem and incentivizes continued progress in personalized medicine.

Reinventing Business Models to Get Us Around the Bend in Personalized Medicine

In recent years the scientific community has taken great strides in reaching a new understanding of genomics and the molecular underpinnings of diseases.  As a result, we’ve reached a turning point in personalized medicine: for the first time, society is finally in a position to start bringing personalized medicine from the lab to the clinic on a large scale.

Johnson & Johnson is committed to helping “be the change” in bringing personalized medicine from the lab to the clinic, with the goal of helping guide delivery of our medicines to the right patients at the right doses at the right times.  Equipped with our new understanding of personalized medicine, we’re changing the way we approach all aspects of drug development programs.  Pharmacogenetic, genomic and imaging data are now guiding our actions related to nearly every therapeutic we develop, and we’ve modified our companion diagnostics programs to identify clinically relevant biomarkers and diagnostic tools earlier in drug development programs.

To bring personalized medicine forward, scientists, regulators, policymakers, physicians, the patient community, payors, and pharmaceutical and diagnostics companies must work in concert.  We must find ways to more successfully work together, adopt a new mindset, and pursue a more open, “networked” approach to innovation.  I’m thrilled to be leading a team focused on this effort at Johnson & Johnson.

But this effort—the striving for open, collaborative, multi-disciplinary innovation—by its very nature is bigger than just one company.  The challenge before us—before society, really—is to take the science, the efforts of industry and the complexity of our health delivery systems, and pave a clear path forward for fully realizing the promise of personalized medicine. Research and development of personalized medicines and companion diagnostics requires equal or sometimes larger investment than traditional drug development programs—even as target populations are, by definition, becoming narrower.  To ensure we’re able to fully tap into the promise and potential of personalized medicine, we must work together to reinvent current business models in ways that incentivize the further pursuit and use of personalized medicine.

Personalized medicine pushes us to look at disease from many different angles, to persevere through complexity, and to tailor healthcare solutions to the unique needs of individual patients or subsets of patients.  In doing so, we can improve health outcomes and enhance quality of life. Even with the challenges ahead of us, these are very exciting times.  Because along with the challenges – the horizon is full of possibilities.

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

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

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: http://www.personalizedmedicinecoalition.org/events/State-of-PM.

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

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