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Paving the Way for Personalized Medicine

Facilitating Inter-Agency Coordination for the Integration of Personalized Medicine into Our Health Care System

medicine bottle that reads Rx Made for: You SOURCE: iStockphoto, SP There are promising developments heralding the arrival of personalized medicine, a new medical field where the results of genetic tests or other biomarker assessments are used to tailor drugs and treatments to individual patients.

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Much Promise and Many Questions

There are promising developments heralding the arrival of personalized medicine, a new medical field where the results of genetic tests or other biomarker assessments are used to tailor drugs and treatments to individual patients. A year ago, for example, the Food and Drug Administration approved maraviroc, the first drug designed specifically for HIV patients who have a particular genetic mutation of the virus. This was the first time a drug had been approved upon the condition that patients first have a genetic test.[1] Similarly, in July scientists at the Van Andel Research Institute published a paper reporting that high expression of the gene known as MET increases the aggressiveness of certain types of breast cancer. This means that the MET gene can be used as a target for new cancer therapies that may inhibit MET’s expression, thereby slowing down the most aggressive forms of breast cancer.[2]

In spite of this kind of progress on the scientific front, Americans today remain guinea pigs in a “one-size-fits-all” approach to medicine in which clinical trials to test the safety and efficacy of new drugs do not take into account the influence of individual genes on individual health and wellness. In contrast, a personalized medicine approach may well allow (perhaps in the not too distant future) every individual patient to receive the best in tailor-made, evidence-based pharmocogenomic medicine.

Similarly, research, development, and clinical care in our health care system merely ensure that medical treatments will work for most of the population most of the time. In fact, most drugs prescribed today only work in 60 percent of patients or less.[3] Personalized medicine promises that treatments will be tailored to individuals by researching the effects of specifically tailored treatments on genetic subpopulations. Since one size does not fit all, personalized medicine will represent a marked improvement over the current system where patients are left to travel down a winding path of physician-led trial and error.

Compounding the unwieldiness of today’s haphazard clinical approach is the disjointed health care informatics system that prevents scientists and physicians from making the most of our nation’s personalized genomics research data. Our impersonal and uncoordinated approach to care costs lives and squanders billions of dollars that could go towards insuring the 45 million Americans who are without coverage while also bringing down costs.[4]

In short, we are awash in evidence that not all individuals will respond similarly to the same medical treatment. But we have not taken the steps to integrate personalized medicine fully into our health care system in order to benefit individuals and society alike.

Granted, there is still a lot we don’t know, especially when it comes to genetics. Most of the genes that have been discovered only have small effects from a diagnostic perspective.[5] But, the bigger question is how can scientists who are eager to expedite the integration of personalized medicine into clinical practice efficiently gather and disseminate their discoveries? It is because of this question that we should look at personalized medicine as contributing to the ultimate goal of turning medical practice into a total learning environment. This means physicians would be able to apply the most recent findings about the efficacy of available treatments while also sharing the outcomes of their own treatment decisions with others so that all physicians can have better data the next time around. This information will also be available to academic scientists and industry researchers so that they can gear their research and product development in more patient-specific directions.

Of course, given the private interests of all the various stakeholders involved, it should be no surprise that bringing about the era of personalized medicine will be no easy task. Many lingering legal, political, and administrative questions remain about patient privacy and about the ownership, organization, and security of the data. And those are just the tip of the iceberg when one considers the vast technical difficulties that computer programmers and health technologists are trying to overcome.

In April, at the Bio-IT World Conference and Exposition in Boston, for example, Microsoft Corp. announced the coming release of Amalga Life Sciences, which promises to be a single platform for aggregating and modeling data from “basic research, clinical trials, health care delivery, and consumer health information needs.”[6] Amalga will also be linked to Microsoft HealthVault so that patients can import their medical data generated at the hospital into their own personal health file.[7] This is an ambitious project that will need to be watched closely in action to see if it enhances or limits the ability of physicians, researchers, and patients to access the information they need in a way that is useful, understandable, and comfortable for them.

For now, though, the bottom line is that there definitely needs to be a strong public debate about what health information is going to look like and what it is going to do. The most promising way to begin this debate is to not get bogged down in the technical questions just yet. Instead, this is a ripe time for taking stock in what values should guide our vision of personalized medicine; what tools we already have available to bring it about; and how responsibilities should be divided up or combined by public and private stakeholders.

Some federal government entities have already started taking steps to answer these questions by moving ahead with initiatives that better streamline the data, technology, and research efforts that are already available. The National Institutes of Health, for example, announced in February that it is moving forward on a clinical trial that will test the effectiveness of integrating genetic data into the dosing protocol for the blood-thinning drug warfarin. This happened just weeks after then-acting Director of the Food and Drug Administration Frank Torti announced that the FDA created a new position in the Office of the Chief Scientist called Senior Genomics Advisor. This office has been filled by FDA veteran Dr. Liz Mansfield, whose job will be to provide “FDA physicians and scientists with tools and personnel capable of high-level analysis of complex genetic data.”[8]

Taking a broader view, the Personalized Healthcare Initiative in the Department of Health and Human Service’s Office of the Assistant Secretary for Planning and Evaluation has conducted reviews of current federal efforts in order to identify organizational challenges to achieving overarching goals. This PHC initiative highlights the need for connecting clinical records with genomic information, ensuring the integrity and privacy of genetic data, preventing discrimination, ensuring the accuracy and validity of genetic tests, and devising common access protocols for genomic databases.

The PHC initiative also highlights various tasks for many government agencies and programs to ensure that that they do their part to achieve these goals for the ethical and coordinated advancement of personalized healthcare. Some of these tasks include directing other agencies in Health and Human Services to devise ways for sharing their data so that the genomic, clinical, and public health aspects of personalized medicine can mutually reinforce one another rather than remain siloed and even redundant in their research and analyses. The PHC initiative also includes in its review the ethical analyses published by the HHS Secretary’s Advisory Committee for Genetics, Health, and Society, or SACGHS, on large-population genetic studies and the bureaucratic logistics of pharmacogenomic research.[9]

Other principled concerns about personalized medicine have also been addressed in general terms through SACGHS, a permanent group that advises the secretary on, among other things, personalized medicine and occasionally releases reports on the issues at hand.[10] Yet the fine practical details of these concerns still need to be hashed out by multiple collaborators on a case-by-case basis.

These concerns have to do with the inclusion of private entities in data-sharing about the validity, utility, and effectiveness of various technologies. What should private biotech companies, pharmaceutical companies, or diagnostic companies be required to share with the federal government? A recent SACGHS report recommends:

In situations where tests are essential to clinical drug use, HHS should require its
grantees and contractors to participate in FDA’s Voluntary Genomic Data Submission Program during the exploratory phase of drug development and/or the review process
for preinvestigational device exemption.[11]

This FDA program is overseen by a body known as the Interdisciplinary Pharmacogenomics Review Group, which was charged in 2005 with collecting phramacogenomic data about drugs in the developmental stage. This program has made regulators more cognizant of genomics, has influenced discussions on clinical trial design, and has even led to the development of a pilot process for qualification of biomarkers for use in regulatory decisions.[12]

From the standpoint of trying to better integrate pharmacogenomic data into the drug development process, this is a great idea. And personalized medicine would advance even more rapidly if pharmaceutical companies could cost-effectively collect information from large-cohort genetic studies and use that information to design better-targeted and more information-rich clinical trials. But companies are reluctant to invest more money in doing their own large-population-based genetic studies that may or may not help them to make a better product let alone recoup their investment.

So who pays for these large-population genetic studies? Usually, it is the NIH. But how can NIH orient its genetic research toward personalizing the drugs that the private sector is developing? SACGHS recommends that the recipients of NIH grants for research that “will be used to demonstrate safety and efficacy to support a [drug or device’s] premarket review application” to the FDA should consult with FDA “early in the study design phase.”[13] Again, this is a practical idea but there needs to be a concerted effort on the part of HHS to make this cooperation materialize on a case-by-case basis.

As pharmacogenomic research develops methodologically and as further evidence is gathered about the application of pharmacogenomic technologies in clinical practice, the policies and protocols for public/private collaboration will need to develop as well. For instance, the SACGHS report makes recommendations about stratifying subject populations based on their predispositions to adverse reactions as indicated by their biomarkers. These recommendations include having the FDA guide the collection of genetic and biological factors that are better predictors of drug responses than race, ethnicity, or gender; and having post-market follow up to find other biological, social, or environmental factors that influence drug response when there is a racial or ethnic disparity in drug response.[14]

Other examples include the plethora of recommendations that SACGHS makes concerning the increasingly controversial areas of insurance coverage and reimbursement, clinical practice guidelines, professional certification, and drug labeling.[15] What we know so far is that these are all relevant issues that can be dealt with by means of better coordination throughout the entire healthcare system.

Researchers need to be informed of all the relevant data collection initiatives. Regulators need to be better aware of the technologies that are coming down the pike. Corporations need to engage in partnerships with the public sector in order to share data for the public good and develop more personalized drugs. And, the FDA needs to encourage drug and device companies to do post-market follow up and coordinate it with the development of new products.

There also needs to be coordination between the genetic test manufacturers, the drug manufacturers, and the health care providers who need to gather evidence for them as they implement tests and therapies in the clinic. The problem, however, is that we do not have sufficient knowledge—both in terms of biomedical data and real-world policy experience—to set in stone any policies for systemic coordination on personalized medicine just yet.[16] Therefore, the best course of action for the time being is for HHS to emphasize better coordination in general, and to guide various coordinated projects by holding them accountable to the broad goals and values put forth in the SACGHS reports and in the work done by the PHC initiative.

This might be a job for HHS’s Office of the Assistant Secretary for Planning and Evaluation, which could:

  • Consult with various agencies, programs, and private entities
  • Suggest opportunities for collaboration
  • Help to iron the terms on which these entities do collaborate

As various personalized medicine initiatives are implemented, HHS can then look at the protocols and policies that do and do not work in terms of data sharing, research coordination, or product development.

This would create an iterative self-correcting process that would allow us to gather more data on personal genomics and conduct more research into the implementation of personalized medicine. Thus, the United States will rapidly build a knowledge base for the future of personalized medicine while it still takes the time to learn how to develop the right policies for shaping that future.

Indeed, all of the initiatives described above are promising steps toward the development of personalized medicine as a new paradigm for medical practice. Nevertheless, the United States still has a long way to go before personalized genomics becomes a standard part of medical practice. Implementation and evaluation must proceed aggressively in tandem in order for us to not only achieve a personalized medicine revolution speedily, but also achieve it efficiently and ethically. This is the essence of progressive innovation and pragmatic policy making. For personalized medicine to fully come to fruition with the fewest number of bumps in the road, we must learn valuable lessons from the current piece-by-piece process as we ramp up our efforts to build upon it.

About the Authors

Michael Rugnetta is a Research Assistant with the Progressive Bioethics Initiative at the Center for American Progress and Whitney Kramer is an intern working on the Progressive Bioethics Initiative.


[1] “Virus-specific Drug Approved for HIV,” New Scientist, August 11, 2007, available at

[2] “Possible Drug Target Found For One Of The Most Aggressive Breast Cancers,” Science Daily, July 9, 2009, available at

[3] Federal Coordinating Council for Comparative Effectiveness Research, Report to the President and the Congress, (Health and Human Services, 2009) available at

[4] Paul Ginsberg, “Efficiency and Quality: The Role of Controlling Health Care Cost Growth in Health Care Reform,” (Washington: Center for American Progress, 2009) available at

[5] Alan M. Garber and Sean R. Tunis, “Does Comparative-Effectiveness Research Threaten Personalized Medicine?” New England Journal of Medicine 360 (19) (2009): 1925-1927.

[6] John Russell, “Microsoft Launches Amalga Life Sciences,” Bio-IT World, April 28, 2009, available at

[7] Microsoft, “Microsoft Introduces Next-Generation Amalga Unified Intelligence System,” Press release, April 6, 2009, available at

[8] U.S. Food and Drug Administration, “Viewpoint: FDA and Genomics,” February 2, 2009, available at

[9] Health and Human Services, “Personalized Health Care,” available at

[10] Office of Biotechnology Activities, “Secretary’s Advisory Committee on Genetics, Health, and Society,” available at

[11] Advisory Committee on Genetics, Health, and Society, “Realizing the Potential of Pharmacogenomics: Opportunities and Challenges; A Report of the Secretary’s Advisory Committee on Genetics, Health, and Society,” (Health and Human Services, 2008) p. 24, available at

[12] U.S. Food and Drug Administration, “Interdisciplinary Pharmacogenomics Review Group,” available at

[13] Advisory Committee on Genetics, Health, and Society, “Realizing the Potential of Pharmacogenomics: Opportunities and Challenges; A Report of the Secretary’s Advisory Committee on Genetics, Health, and Society,” (Health and Human Services, 2008) p. 24, available at

[14] Advisory Committee on Genetics, Health, and Society, “Realizing the Potential of Pharmacogenomics: Opportunities and Challenges; A Report of the Secretary’s Advisory Committee on Genetics, Health, and Society,” (Health and Human Services, 2008) p. 43, available at

[15] Advisory Committee on Genetics, Health, and Society, “Realizing the Potential of Pharmacogenomics: Opportunities and Challenges; A Report of the Secretary’s Advisory Committee on Genetics, Health, and Society,” (Health and Human Services, 2008) p. 4-8, available at

[16] Alan M. Garber and Sean R. Tunis “Does Comparative-Effectiveness Research Threaten Personalized Medicine?” New England Journal of Medicine 360 (19) (2009): 1925-1927.

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