National Bioeconomy Blueprint Showcases Personalized Medicine as Model for Strengthening U.S. Bioeconomy

Last week, the White House released its National Bioeconomy Blueprint.  It lays out some strategic objectives designed to help realize the full potential of the U.S. biotechnology sector to generate economic growth by creating jobs and addressing societal needs.

As an example of how the government’s efforts can facilitate the development of a more robust bioeconomy, the report discusses the impact of the Human Genome Project and the development of personalized medicine. The Blueprint cites the Case for Personalized Medicine, 3^rd Edition, noting “advances in recent technologies have increased the momentum of personalized medicine – customized healthcare based on specific genetic or other information of an individual patient.”

While we agree that policies are needed to help support research and development (R&D), improve translational and regulatory science, improve regulation in other areas, enhance workforce training, and develop new public-private partnerships and precompetitive collaborations, we are concerned they are not sufficient to allow us to realize Adriana Jenkins’ dying wish, that all patients have access to personalized treatments.

The White House is correct to shine a light on FDA and direct the agency to focus attention on application review times, coordinated parallel reviews of products, and continued improvement of regulatory science. In reality, we are already seeing the benefits of this increased coordination – with FDA’s accelerated review and approval of Kalydeco™ for cystic fibrosis and Xalkori^® for non-small cell lung cancer, each with a companion diagnostic approved together and in advance of FDA time lines.

Still, engaging on regulatory science and streamlining FDA processes will only go so far to improve the bioeconomy and bring personalized medicine to patients. For instance, current comparative effectiveness research (CER) and health technology assessment (HTA) models, which are not addressed in the Blueprint, are not well aligned with the science of personalized medicine and such misalignment causes additional barriers to market entry and patient access after FDA approval. The Blueprint highlights coverage with evidence development (CED) as a potential HTA model, but it should be noted that although CED can be a good tool, if done wrong, it can also chill innovation.

Likewise, unclear and unrealistic expectations for obtaining Medicare coverage and adequate payment for personalized medicine products and services are a substantial hurdle, preventing companies from seizing the full scientific potential and translating that into new treatments and the resulting high-paying jobs and economic contributions that follow.

We look forward to working with the Administration and with Congress to shape policies that will help support the ability of companies to continue to develop personalized medicines and bring them to patients, including research and development tax credits, delivery system reforms, and regulatory and reimbursement policies. Given the tremendous potential of personalized medicines, it’s key that we get the policies right to foster companies working on personalized medicine and thereby improve patients’ lives and our economy.

PCORI Research Priorities, Will They Support Personalized Medicine?

The Board of Directors of the Patient-Centered Outcomes Research Institute (PCORI) met last week, outlined research priorities, and asked for public feedback. What I found most interesting, and a little disheartening, is that the priorities are drafted in vague language, making it difficult to determine how they may (or may not) support personalized medicine.

I hope that as PCORI moves forward, it will give assurance to the Personalized Medicine Coalition (PMC) and other stakeholders that its research supports personalized medicine, as was the intent of Congress at the time of the Institute’s formation.

Specifically, research conducted in the category, “Comparative Assessment of Options for Prevention, Diagnosis, and Treatment,” will compare treatments, but at what level?  Researchers now know more about individual response to some drugs based on biomarker information. For example, when comparing a red pill to a blue pill, it is imperative that biomarker information be included in those examinations, especially when biomarker information is in the label for the drug. A list of drugs with biomarker information in the label can be found on pages 20-25 of The Case for Personalized Medicine, 3^rd edition. But, without clearly defining the details of PCORI’s research, we really can’t know whether or how the Institute will consider biomarker information in its priorities and research agenda.

The research priority “Improving Healthcare System” can support patient-centered care through innovations, but how? PMC suggests that the research be predictive, preventative, personalized and participatory. By emphasizing the “4Ps,” health system evaluation can support personalized medicine. However, without getting more specific about research priorities, we don’t know whether PCORI will include research that addresses these aspects of care delivery.

To support personalized medicine, the “Communication and Dissemination Research” priority should require researchers to answer one question: “Why does this treatment work and for whom?” (or, more frequently, “How likely is this treatment to work for me, and what are the potential trade-offs?”).  It is not enough, in the PMC’s opinion, to say that one therapy works for most people. PMC suggests that the research should explain why a therapy works (or is more likely to work) and for whom. For example, in research comparing red and blue pills, the communication and dissemination of the research results should stress that although the red pill works for most, the blue pill is particularly effective for people with a specific biomarker.

We are at a pivotal stage in the design of an entity that is tasked with assisting patients, clinicians, purchasers and policymakers in making informed, evidenced-based health decisions. PCORI could foster research that answers the important questions about what interventions work, and for whom. Or, PCORI could be another investigator-driven research institute that allows funded researchers to pursue questions of great scientific import, regardless of their practicality. Such a mission would be redundant with many others, most notably the National Institutes of Health, would be less likely to result in a cohesive national research program, and less likely to ensure personalized medicine is appropriately integrated.

It is my hope that PCORI supports the science that will drive personalized medicine forward, and will engage stakeholders in driving personalized medicine forward, by proposing specific research priorities and research questions to get answers to the questions that science, medicine and, ultimately, patients demand.

Personalized Medicine – Part of the Health Cost Solution

At the Research!America’s National Health Research Forum last week, personalized medicine was identified by several leaders as a solution to our health care cost challenges.  In reply to a question from the audience about how we can afford new cancer treatments,  NIH Director Francis Collins said “encouraging possibilities” exist with our “more precise understanding of the molecular basis of disease.” His comments underscore how medical innovation will be part of the solution not just for patients with unmet medical needs, but for our growing cost challenge as well. 

With the emergence of personalized medicine, we are in a better position to target treatments in cancer. Stratifying patients should make for smaller, more successful clinical trials. In addition, biomarkers are helping give more precise information earlier in the process. Collins said that if all goes well, the outcome “should be that we can actually get approvable compounds through the pipeline at a higher frequency of success and a lower cost than has often been the case in the past.” He stressed that partnerships with industry will be crucial.

PhRMA President and CEO, John Castellani added that drug development is getting dramatically more expensive, costing more than $1 billion and lasting more than 11-12 years per drug. “We have to get better. We have to get smarter. We have to get more targeted,” he said, “That’s where the science is going.” Biopharmaceutical companies are working on personalized medicine, he pointed out, and if we have a system that recognizes and delivers the “right care at the right time,” it will be “much more affordable than the trajectory that we’re on now.”

To listen to the full commentary from Collins, Castellani, and the other panelists on personalized medicine at the National Health Research Forum, visit http://www.researchamerica.org/forums, beginning at minute 137:00.

NIH Announces Genetic Testing Registry

On March 18, the National Institutes of Health (NIH) announced the development of a Genetic Testing Registry (GTR).   NIH established the GTR to serve as the single public resource to provide detailed information about the 1600+ genetic tests for patients and consumers.  The database, which will be populated voluntarily by genetic test providers, is expected to be available in 2011.
 
This is good news in that the database reflects impressive advances in the field of personalized medicine and an increased number personalized medicine products.

In announcing the GTR at the PhRMA annual meeting, NIH Director Dr. Francis Collins called for broad public and industry input on registry design.  The Personalized Medicine Coalition is encouraging its members and others interested in personalized medicine to share insights and ideas about the registry.  The database design can be reviewed and comments can be provided through the National Center for Biotechnology Information (NCBI) website at  http://www.ncbi.nlm.nih.gov/gtr/.

Opportunities to participate will be announced through the Federal Register soon.  Comments and questions can also be submitted anytime by emailing GTR@od.nih.gov.