The flip side is that personalized medicine means developing research and marketing strategies for therapies that work in maybe half the population. Diagnostics have to be incorporated into these programs, which adds to development costs and treatment expense. It's not clear whether personalized medicine represents the new frontier of biomedical development or is something that will never be more than niche products.
These issues are gaining attention in industry and health-policy circles. Last month, a conference at the Harvard Medical School explored strategies for accelerating personalized-medicine adoption. At a September conference in Washington hosted by the Personalized Medicine Coalition (PMC), Department of Health and Human Services Secretary Mike Leavitt unveiled a federal report on personalized healthcare that outlines numerous HHS projects for developing biomarkers, expanding health IT, and translating genomics discoveries into evidence-based medicine for clinicians.For example, the National Institutes of Health (NIH) is funding dozens of studies to identify genetic factors related to cancer, heart disease, obesity, AIDS, diabetes, and others. Researchers can use this information to validate new biomarkers and other tools for defining individual differences affecting disease. These results can lead to new diagnostics that identify patients more susceptible to disease and likely to benefit from preventive care and treatment.
Successful treatment requires physicians to use patient genetic information in deciding whether to conduct assays and revise prescribing. Medical societies are beginning to support such approaches, as seen in recent recommendations from the American Society of Clinical Oncology (ASCO) that back the use of certain genomic tests in deciding appropriate treatment for breast cancer patients. An HHS advisory committee is preparing a report on the ethical and medical issues associated with incorporating genetic testing into clinical practice.
Efforts to ensure that clinical decisions are based on accurate and timely evidence are expanding under the Agency for Healthcare Research and Quality and the Institute of Medicine's Roundtable on Evidence-Based Medicine. The ultimate goal is for the medical system to develop new evidence as a natural by-product of delivering appropriate care.
Such an approach requires an electronic medical-record system that can store and access information on patient genetic disposition, treatment, and response. The Leavitt report outlines multiple initiatives for establishing an e-health system, but that still may be years away. It notes that accumulating data on individual genetic markers for disease raises concerns about the need for privacy and security to ensure that personalized medical information is not misused.
Challenges for Industry
New discoveries on how variations in the human genome affect an individual's response to medications offers exciting opportunities for pharma. Researchers say that people with certain genetic factors may be less likely to resume smoking following smoking-cessation treatment. A report from the National Institute of Mental Health indicates that genetic variation in patients may relate to suicidal thoughts when taking antidepressants. Information on gene variations already is providing more-accurate chemotherapy dosing for children with leukemia and promises to refine treatment for those with asthma.
The Food and Drug Administration has championed the shift to personalized medicine as key to modernizing drug development and producing more high-value therapies. A prime goal of its Critical Path Initiative is to identify and validate biomarkers and other tools for determining the safety and efficacy of drugs in certain patients. Agency officials have found that dose-response differences can decrease adverse events, and categorizing patients according to response potential can increase treatment effect and lead to more informed labeling.