Our Health Policies Can’t Ignore Where Science Is Leading Us

Do we want continued progress in personalized medicine, or don’t we? Based on events last week, the answer might be ‘both.’ I attended a press conference here in Washington that celebrated the achievements made by the health sector in our efforts to turn cancer from a sure death sentence into a chronic disease. Simultaneous to this, the Medicare Payment Advisory Commission (MedPAC) proposed policies to offset the cost of a physician payment fix under Medicare. The disconnect between the two was striking. Unfortunately, the MedPAC proposals have the unintended effect of heavily impacting those on the leading edge of personalized medicine. Of the $233 billion in 10-year savings, $114 billion would come out of the biopharmaceutical and clinical laboratory sectors, thus possibly robbing seniors of access to the latest targeted therapies and the diagnostics used to guide them. The proposal includes savings from giving the Centers for Medicare and Medicaid Services (CMS) new authority to impose “least costly alternative” payment, which would result in locking us into our current one-size-fits-all medical paradigm and weakening physicians’ power to tailor care using diagnostics and targeted therapies.  Another troubling aspect of these suggestions is that they propose cuts by sector, ignoring the reality that personalized medicine brings efficiencies to the system of health care.

The proposal appears to reflect a lack of awareness of the impact that personalized medicine is making in health care and underscores the importance of the Personalized Medicine Coalition’s legislative work.  One of our policy suggestions, released over the summer, calls for inclusion of a personalized medicine representative on MedPAC and the creation of a new personalized medicine advisory committee to foster alignment of the policies across the entire Department of Health and Human Services with the science of personalized medicine. Personalized medicine challenges the status quo in medicine – the blockbuster business model, diagnostic coding and payment, or health care delivery systems. Maybe it’s time it challenged MedPAC, too.

Personalized Medicine: Celebrating Progress and Looking Ahead

The release of the American Association for Cancer Research (AACR) Cancer Progress Report last week, reminded me of the progress that has been made in cancer care since the passage of the National Cancer Act in 1971.  When President Nixon signed the law committing significant U.S. funds to cancer research, little was known about the disease and it was thought that a one-size-fits-all approach might eradicate cancer–what we know today is more than 200 different diseases; and that number will likely grow.  The personalized approach necessary to combat cancer was then not yet imagined, let alone understood.

But the progress in personalized cancer care, and in personalized medicine more broadly, has been recent.

For my September column in Personalized Medicine, I interviewed Harvard Medical School Professor of Genetics and Medicine Raju Kucherlapati, Ph.D., to discuss how personalized medicine has evolved since he launched Harvard’s annual personalized medicine meeting in 2005.  He explained that at first, business leaders and policymakers were skeptical that personalized medicine would have any impact in the near term, but that the many examples of personalized medicine products and new tools to speed innovation have changed minds and forever improved medicine.

No one better exemplifies progress in this field than President and Co-founder of the Institute for Systems Biology, Leroy Hood, M.D., Ph.D., who will be presented with PMC’s Award for Leadership in Personalized Medicine at this year’s Harvard conference. Dr. Hood envisioned the integration of scientific understanding into clinical practice by developing the first DNA and protein sequencer and synthesizer–laying the foundation for the development of personalized medicine.

I hope you will join me at this year’s Harvard conference, November 9-10, 2011, in Boston and at PMC’s cocktail reception to kick off the conference on November 8, 2011.  We will both celebrate the progress personalized medicine has made, thanks to the leadership of visionaries like Raju Kucherlapati and Leroy Hood; and discuss how we continue to move the paradigm of personalized medicine forward through scientific innovation, collaborative business partnerships, and supportive public policies.

New Personalized Medicine Products Signal Progress for Patients

The approvals of new treatments for two different types of cancer indicate that we are making significant progress in personalized medicine. Although the U.S. Food and Drug Administration (FDA) could further define a more transparent and predictable regulatory environment for personalized medicine products, the agency is clearly serious about personalized medicine. In August it cleared Xalkori® (crizotinib), which targets a rare form of lung cancer, ahead of its own priority six-month review goal and Zelboraf® (vemurafenib), which treats patients with melanoma also well ahead of schedule.

These combination products represent a harbinger of the future of medicine. By applying new genetic understanding of tumors to target treatments, drug and diagnostic companies working together are able to develop drugs that are safer and more effective, offering hope to cancer patients where none existed.

In the case of Xalkori®, Pfizer and Abbott Molecular co-developed the drug and a companion diagnostic test (Vysis ALK Break Apart FISH Probe Kit) to identify the subset of patients who are most likely to respond to the drug.  Around five percent of patients diagnosed with non-small cell lung cancer, the most common form of the disease, have tumors with an anaplastic lymphoma kinase (ALK) gene defect that responds to the drug – about 6,000 patients per year in the United States.  Xalkori® works by blocking proteins produced by the abnormal ALK gene.  It is the first lung cancer treatment developed and approved with a diagnostic test.

Stafford O’Kelly, Abbott Molecular Vice President and President Molecular Diagnostics, noted, “[This collaboration] marks a breakthrough in the advancement of personalized medicine…that will help a subset of lung cancer patients get treatment tailored to their unique genetic profile.”

Also recently approved is a targeted therapy to treat melanoma – the deadliest form of skin cancer.  Zelboraf® is indicated for treatment of patients with melanoma whose tumors express a gene mutation called BRAF V600E, which the drug blocks.  Approved with a diagnostic test (cobas® 4800 BRAF V600 Mutation Test) that determines which patients carry the mutation, the drug was clinically effective in 50 percent of these patients.

These new models for collaborative research and development of treatments are yielding positive results. They should keep us focused on the emerging field of personalized medicine and its promise to deliver the right treatment to the right patient with improved diagnosis, more efficient drugs, and better medical outcomes.

Looking Forward, Looking Back

At the Food and Drug Administration (FDA), I had a wonderful opportunity to spend nearly 20 years working in various capacities: in drug development science, regulatory oversight, and clinical pharmacology. Earlier this summer, I had the privilege to meet with the Personalized Medicine Coalition’s public policy committee to discuss the state of personalized medicine just after my departure from the FDA and before I began my new position leading the pharmacometrics and systems pharmacology initiative in the interdisciplinary Institute of Therapeutic Innovation at the University of Florida’s Research and Academic Center in Lake Nona (Orlando).  Personalized medicine will continue to be a passion for me in my new position.

The timing of my talk was serendipitous:  Dr. Steven Spielberg was just named as the new Deputy Commissioner for Medical Products and Tobacco at FDA. His perspective is strongly rooted in understanding disease at the molecular level, having served as director of the Center for Personalized Medicine and Therapeutic Innovation at Children’s Mercy Hospital in Kansas City, Missouri. And the FDA had just announced the draft guidance document for personalized medicine and companion diagnostics. This guidance should be seen as part of a mosaic of guidance documents from the FDA that will together guide the regulation of personalized medicine products. The Personalized Medicine Coalition has begun putting the mosaic together by listing the relevant guidance documents as a reference tool on its website.

I had a chance to reflect on the new direction FDA has taken over the past several years to embrace what we’re seeing in research and scientific discovery. Like the industrial revolution, the genomic revolution could take 60 years to fully unfold, but in the last ten years, the field and the FDA have made great progress. Many of these changes are leading researchers to re-evaluate how we focus research efforts, assess new therapeutics, and apply our shared learning through the R&D continuum.  As we’ve heard from the industry, researchers are stratifying medicines earlier in the R&D process and about 50% of new drugs have a biomarker that can potentially lead to a personalized approach. While many challenges remain, I am encouraged by the four-fold increase in Investigational New Drugs (INDs) and New Drug Applications (NDAs) that include genomic data since 2008.

To address this growth in genomic submissions, the FDA has new employees with expertise in genomics dedicated to review of these products and efforts to coordinate personalized medicine reviews will continue. I see a new era of personalized medicine not just in oncology but in cardiovascular disease, infectious disease, and neurodegenerative diseases, among others and the FDA is working to be ready.

I called on all members of the Personalized Medicine Coalition to find new ways to enable collaboration and to export the knowledge they are fostering at their companies and organizations to benefits for public health. By doing so, our combined efforts can lead to dramatic advances in improving therapeutics and healthcare. We are only just beginning to understand how new networks and powerful tools for sharing best practices can help us solve some of our most complex disease and drug response questions.