On June 9, 2011, the Personalized Medicine Coalition (PMC) hosted its Seventh Annual State of Personalized Medicine Luncheon in Washington, D.C.  As stated on its website, PMC’s mission “is to build the foundation that underpins the advancement of personalized medicine as a viable solution to the challenges of efficacy, safety and cost.”  This year’s luncheon featured John J. Castellani, President and CEO of Pharmaceutical Research and Manufacturers of America (PhRMA) as its keynote speaker.

PhRMA is a well-known trade group representing U.S pharmaceutical research and biotechnology companies, including big names such as Amgen, Biogen Idec, Eli Lilly, GlaxoSmithKline, Novartis and Pfizer.  PhRMA advocates for public policies that encourage the discovery of new medicines for patients by such companies.  Like the PMC, PhRMA strives to educate the public and policymakers, and promote new ways of thinking about health care.

Speaking on behalf of PhRMA, Mr. Castellani began by talking about the power and potential of personalized medicine.  He reminded the audience about Adriana Jenkins, a cancer patient who inspired many through her experience with a targeted cancer drug.  He referred to Ms. Jenkins’s dying wish, as described in Forbes magazine, that patients, physicians and insurers create groups to support the commercialization of personalized cancer drugs.

Mr. Castellani spoke about personalized medicine’s substantial capacity to improve patient care and health care efficiency, as well as its ability to provide quality jobs.  Unlike in the past, PhRMA companies are now fully engaged and committed to advancing personalized medicine.  He referred to a report from Tufts University (Tufts CSDD Impact Report, Nov/Dec 2010), indicating a shift toward this field by the biotech/pharma industry. For example, companies reported that 12% to 50% of their current clinical development pipelines involve personalized medicines.  Moreover, 100% of the companies surveyed indicated that their discovery strategy includes biomarker and/or targeted therapies.  Further, 30% of the companies require that all compounds in development have an associated biomarker.

Current Obstacles to the Success of Personalized Medicine

According to Mr. Castellani, however, the success of personalized medicine will depend on the biopharmaceutical sector and other partners in the field working together to overcome scientific, business, regulatory and policy barriers.  Cooperation and communication must flow between diagnostic companies, researchers, payers/insurers, patients, medical practitioners, as well as regulatory agencies.

Regarding scientific barriers, initial work in personalized medicine has not yet succeeded in transforming patient care.  For example, when researchers completed the Human Genome Project in 2003, many speculated about a new age of treatment. Today, however, some ask “Where are the miracles?”, suggesting that scientists have over-promised and under-delivered.  In fact, translating DNA sequence information into treatment for patients has been more difficult than expected.  The association between genetics and disease has not panned out in certain clinical trials.  Mr. Castellani responded by saying that science takes time and every great success is built on the failure of others.

As it pertains to regulatory barriers, right now the science is changing faster than U.S. regulatory structure.  Mr. Castellani noted that current federal regulation of diagnostics is unclear and uncertain to providers and investors.  The industry needs clear guidance from the FDA, especially as it pertains to diagnostics and avenues for obtaining approval of medicines and relevant companion diagnostics, including simultaneously, in a cost effective way.

Regarding business and costs related barriers, Mr. Castellani discussed the need for the medical community to shift from simply reacting to disease to preventing disease and avoiding medical complications and hospital stays.  Health care costs are less when intervention occurs earlier.  Thus, personalized medicine, and its ability to be proactive rather than reactive, is a solution to rising health care costs.

In addressing costs, reimbursement obviously comes to mind.  The current U.S. reimbursement system considers costs, but fails to reflect the full value of personalized medicine.  Moreover, the potential and benefit of personalized medicine may only be realized through preventive medicine, education and restructuring of existing systems.  Such a proactive approach involves costs that are not currently adequately reimbursed, in contrast to more traditional medical products (drugs) and services that are based on existing symptomatology.

On the policy side, it is worth considering that in addition to being good for patients and reducing health care costs, personalized medicine is good for the economy.  This industry creates high quality, high paying jobs.  In addition to the 650,000 jobs created directly, the personalized medicine industry also creates 2.4 million jobs indirectly, corresponding to a 3.7 multiplier.

Education and Communication Is Key

As explained by Mr. Castellani, education of politicians, federal agencies, payers/insurers, physicians/clinicians, patients and the public at large will be key — the success of personalized medicine requires education and communication more than any other field today.  For instance, personalized medicine success is not necessarily about the number of patients directly benefiting from a particular personalized medicine regime (e.g., helping thousands rather than millions of patients).  Instead, education should emphasis what such care means to the patients themselves, i.e., personalized medicine has a better chance of working on any given individual.  Likewise, education can explain how personalized medicine will ultimately reduce health care cost, benefiting everyone involved.