The personalized-medicine bandwagon is on a roll, paralleling the death-knell for blockbuster-drug development. Strategies
for identifying patients who will respond to a certain therapy, as well as those most likely to suffer adverse events, promise
to improve healthcare quality while also eliminating waste and inappropriate spending. Interventions based on individual characteristics
may have limited sales, but they may justify higher prices and reduce the high cost of clinical development.
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.