Evidence, Coverage, and Incentives: A PMC/BIO Solutions Summit Conference

Personalized medicine is changing the way medicine is practiced, and how drugs and biologics are developed.  Systemic changes are needed to reflect this and support product development, but current economic, regulatory, and practice disincentives are impacting and slowing development. For example, medical advances often take years to be recognized in clinical care guidelines and cost containment policies often prevent new diagnostics from being priced at a level that supports innovation.

The Personalized Medicine Coalition (PMC) and the Biotechnology Industry Organization (BIO) have joined forces to address these challenges. A series of PMC/BIO Solutions Summits will convene stakeholders with the charge to identify and discuss potential solutions to these and other barriers.

The first of this series, Evidence, Coverage & Incentives, will be held Wednesday, April 17, in Washington, D.C. I encourage you to join this full-day conversation uniting stakeholders from across industry as we work to ensure that our healthcare system benefits from all personalized medicine promises to offer.

Specific discussions will focus on:

• Challenges of reimbursement;
• Evidentiary standards and data requirement for payer coverage;
• Clinical guidelines;
• Investing in molecular diagnostics;
• Issues impacting market access; and
• Incentivizing personalized medicine development.

The challenges are daunting, and it will take cooperation among all stakeholders to bring the benefits of our scientific advances to fruition. This summit will work toward solutions to foster innovation in our collective thinking and to identify pathways for improving the overall quality and efficiency of care. I encourage you to lend your voice to these efforts.

– This blog also appears on the BIOtechNOW website.

Putting the Pieces Together for Personalized Medicine

At the Personalized Medicine Coalition, our goal is to open the path to innovation in the science and practice of personalized medicine. “Personalized medicine” is the tailoring of medical treatment to the individual characteristics of each patient. For personalized medicine to work, many pieces must align, including detailed knowledge of the genomics underlying a given disease, effective targeted treatments, and accompanying diagnostic tests to identify the patients who are most likely to benefit.

Those pieces do not line up overnight but, instead, each aspect advances as knowledge builds cumulatively over time. For example, the technology to sequence genes has come a long way in the last 15 years. According to our latest edition of The Case for Personalized Medicine, the cost of sequencing the human genome dropped from $300 million in 2001 to $5,000 in late 2011. This dramatic price drop reflects the evolving science that built over time. Without the initial $1 billion investment to sequence the human genome by public and private partners, this progress would not have been possible.

Likewise, we see the same accumulation of knowledge related to personalized treatments over time. Even within one medicine, our understanding of its potential benefits and indications evolves.

Today, we can sub-classify certain diseases based on genetics: Melanoma can be BRAF positive or non-small cell lung cancer can be EGFR positive or ALK positive. Making these distinctions has led to targeting these gene mutations with specific treatments, leading to improvements in patient outcomes and quality of life. Tomorrow, our scientific knowledge base will continue to grow and is coupled with the technological advances that are enabling the analysis and interpretation of findings like no other time in history.

Increasingly, our expanding knowledge of the role of genetic variation in patient response to treatments will drive continuous learning about the optimal role and value of treatment regimens and diagnostic/treatment combinations. This emerging capability holds great potential to improve patient care and healthcare value, but it is at odds with our conventional approaches to assessing value at a point in time based on broad average study results.

To foster continued progress, the private and public sectors must work together to develop policies that align with scientific advances. We need adequate reimbursement for advanced diagnostics and targeted therapies, and flexible methods to access the value of personalized medicines, rather than one-size-fits-all determinations that don’t take the individual into account. We need policies that recognize the many factors that come into play to make personalized medicine possible, including the research, development, and performance of molecular testing.

At the Personalized Medicine Coalition, we firmly believe that we can and must bring personalized medicine to all patients. Collaboration among the many stakeholders involved will move us closer to this goal.

– A version of this blog originally appeared in The Catalyst series highlighting incremental innovation posted at the PhRMA website.

A Response to Ezekiel J. Emanuel’s NYT Op-Ed about the Cost of Cancer Care

Ezekiel J. Emanuel’s “A Plan to Fix Cancer Care” (The New York Times, Op-Ed, March 23, 2013) highlights the need for collaboration and commitment across the oncology community to find solutions for the financial burden cancer poses to our healthcare system.

In order to sustain progress while containing cost, though, we must recognize the heterogeneity of the dreaded disease by eschewing a one-size-fits-all approach. We must, in short, devise strategies that make medical sense. These strategies necessarily will involve adaptive clinical trials, an intelligent regulatory policy that encourages linking therapy and diagnosis, and a willingness to pay for products that improve patient care.

Progress is driven by a dynamic process in which the full value of intervention evolves over time. We must be careful not to stifle innovation by overlooking the critical steps that can provide an answer to the correct question, which is: how can we maximize value from our growing, but not always wise, investments in health care?