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Personalized Medicine 101:
The Challenges


As personalized medicine becomes more pervasive, a number of policy issues arise. A new healthcare paradigm with far-reaching implications, personalized medicine requires us to examine our current approaches to clinical trials, intellectual property rights, reimbursement policies and patient privacy and confidentiality. Given the array of issues, it is important that a broad spectrum of life science companies, healthcare providers, payers and policymakers participate in shaping the evolution of this new opportunity.  

Some of the issues raised by personalized medicine include:   

    • Intellectual property. A strong intellectual property system is necessary to stimulate investment in innovation. It is essential that government patent systems offer protection for innovations relating to personalized medicine, as well as high quality patent examination that allows patents of appropriate scope and quality.  
    • Regulatory oversight. The FDA is playing a key role in advancing personalized medicine, conducting discussions and convening debate about the implications for drug development and regulatory review. The FDA has been at the forefront of this issue, identifying emerging trends, and is in the process of developing guidelines for the submission of genetic data. Among the questions being considered: How narrowly should clinical trials be designed to include or exclude people based on the results of certain genetic screening tests? Should efficacy be defined in different ways for different genetic sub-groups? These are only a few of the questions that regulators — working hand-in-hand with academic and industrial scientists, professional associations and patient groups — will need to answer.  
    • Reimbursement. Personalized medicine will make it increasingly important for patients to have access to diagnostic and prognostic tests, as well as all appropriate medicines. As a result, public and private payers will need to grapple with new and complex questions, such as: Will therapies be reimbursed only for those patients who are identified, using whatever tests are available at the time, as likely to respond? Should all tests for all genetic traits that could pose serious safety issues be covered if they are available — or only if the trait is relatively common and the test relatively cost-effective? Should some demonstrated degree of efficacy in the general population be required to justify reimbursement for new therapies? Can insurance limit coverage to therapies that are effective only in populations of a certain size or prevalence? How should payment systems deal with testing and treatment for extremely rare conditions? How will the insurance concept of shared risk be affected by the increasing ability to individualize risk factors? Should improved medical education, new IT systems or patient education be covered?  
    • Privacy, confidentiality and patients’ rights. Patient protection is clearly a critical issue, and one that must be addressed to build public confidence — without which it will be impossible to collect the molecular and clinical data that is the foundation of personalized medicine capability. Among the issues that must be addressed: the implications of being identified as predisposed to a certain condition or non-responsive to available treatments; the rights of non-consenting family members of the tested individuals; the implications for existing ethnic groups or as-yet-undefined genetic subgroups; and the psychological and social effects of genetic testing for the individual tested.  

Pharmaceutical and biotech companies, diagnostics companies, researchers, medical educators, information technology managers, healthcare providers, laboratories, patient advocates, policymakers, payers and other stakeholders must all work together to carefully review the issues at hand and consider their interconnected implications. The common goal: an integrated policy framework that balances patient, industry and scientific interests without hindering advancement of this tremendously important sector. Through these efforts, we can help ensure that personalized medicine is able to fulfill its promise as rapidly as possible.    

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