The personalized medicine bandwagon is on a roll, offering a new model for calculating reimbursement of high-cost biotech
therapies. 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 eliminating waste and inappropriate spending. Interventions based
on individual genetic characteristics may have limited sales, but support higher prices and less costly clinical research
On the down side for manufacturers, personalized medicine means developing research and marketing strategies for therapies
that work in only a small patient population. Diagnostics have to be incorporated into treatment regimens, which adds to development
and payer costs. It's not clear if personalized medicine represents the new frontier of biomedical development or if it will
only apply to niche products.
These issues are gaining attention in industry and health policy circles. At a September conference in Washington hosted by
the Personalized Medicine Coalition (PMC), Health and Human Services (HHS) 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, macular degeneration, diabetes, and others. Researchers can use this information to validate
new biomarkers and other tools to define 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.
TRANSLATION IS KEY
Successful implementation of personalized medicine requires physicians to tap 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 to decide appropriate treatment
for breast cancer patients. An HHS advisory committee is preparing a report on the ethical and medical issues in incorporating
genetic testing into clinical practice.
The campaign for evidence-based medicine encourages a shift to more personalized care. 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 round table 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 may
be years away. It notes that accumulating data on individual genetic markers for disease raises concerns regarding 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 offer exciting opportunities
for biotech manufacturers. The US Food and Drug Administration (FDA) has approved 20 therapies that use genomic information
to target use, including several innovative cancer treatments. A report from the National Institute of Mental Health (NIMH)
indicates that genetic variation in patients may relate to suicidal thoughts when taking antidepressants. Information on gene
variations is already providing more accurate chemotherapy dosing for children with leukemia and promises to refine treatment
for individuals with asthma.
FDA has championed the shift to personalized medicine as the 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 to determine
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
Sponsors are exploring protocols for incorporating genomic data into clinical and toxicology studies through FDA's voluntary
genomic data submission (VGDS) program. The agency has discussed approximately 50 research plans under the program, has developed
guidance for submitting such data, and recently expanded VGDS into VXDS to include additional "'omic" exploratory data.