Making the Case for Personalized Medicine

As lawmakers, industry, consumer groups and the general public debate the future of health care, personalized medicine continues to focus attention-with good reason. In a time of unprecedented scientific breakthroughs and technological advancements, personalized health care has the capacity to detect the onset of disease at its earliest stages, preempt the progression of disease, and, at the same time, increase the efficiency of the health care system by improving quality, accessibility, and affordability.

With that in mind, I’m excited to announce the release of the fourth edition of Personalized Medicine Coalition’s signature document, The Case for Personalized Medicine, which notes the steady development of the field. Most notably, in 2006, there were 13 prominent examples of personalized drugs, treatments and diagnostics on the market. In 2011, there were 72, and today there are 113-a 57 percent increase in the last three years.

Along with this steady growth, we also examine opportunities for the continued development and adoption of personalized medicine as several factors come into play: the cost of genetic sequencing declines, the pharmaceutical industry increases its commitment to personalized treatment, and the public policy landscape evolves. While momentum is building, much remains to be done to keep up with the evolving developments in science and technology, according to the report. With all this progress comes a greater responsibility to actually help patients by advancing personalized medicine to make it standard practice in the near future.

According to The Case, personalized medicine can accomplish the following:

• Shift the emphasis in medicine from reaction to prevention
• Direct the selection of optimal therapy and reduce trial-and-error prescribing
• Help avoid adverse drug reactions
• Increase patient adherence to treatment
• Improve quality of life
• Reveal additional or alternative uses for medicines and drug candidates
• Help control the overall cost of health care

But to advance personalized medicine, we need intelligent coverage and payment policies, clearer regulatory guidelines, and modernized professional education to prepare the next generation of doctors and other health care professionals for personalized medicine.

PMC is pleased to partner with BIO and release this report at the Personalized Medicine & Diagnostics Forum at this year’s meeting. The Forum takes place next Wednesday, June 25 at 9 a.m. PT in room 7AB and will explore many of the salient issues facing personalized medicine, as discussed in The Case. If you’re onsite at BIO this year, come check out the forum. We’d love to hear your thoughts on the current personalized medicine landscape and what’s to come.

The new edition of The Case for Personalized Medicine will be available online June 25 at 9 a.m. ET, but in the meantime, check out PMC’s other publications here.

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This blog post originally appeared in BIOtechNow, the blog of the Biotechnology Industry Organization (BIO).

 

A Look at the Regulation of Diagnostics

Participants at the upcoming Personalized Medicine World Conference 2014 (PMWC) will be engaging in thoughtful debate and discussion on some of the biggest topics in the field of personalized medicine. I anticipate a lively discussion with Andrew Fish, Executive Director of AdvaMedDx on the topic of regulatory issues in molecular diagnostics.

The regulation of diagnostic products is one of the most contentious issues within the personalized medicine community today. Regulatory issues have led to confusion and uncertainty in the industry due to the involvement of multiple agencies with varying standards. Consensus on solutions among kit manufactures and laboratory developed test companies has been hard to come by.

At the Personalized Medicine Coalition (PMC), we have heard from some who would prefer that the status quo is maintained; however, PMC contends that the status quo is not an option. While our members may not agree upon the exact course of action, it is time to acknowledge that action is needed to build a consensus around the development of an efficient, cost-effective process for bringing safe, high quality diagnostic tests to market in which patients, physicians, and payers can have confidence.

To initiate this process, PMC published Personalized Medicine Regulation: Pathways for Oversight of Diagnostics. The paper established baseline knowledge of the status of diagnostic regulation, and set the groundwork for future collaboration among industry, government, and other organizations.

To learn more about this important issue, with a look at the complex and diverse perspectives from key stakeholders, as well as to explore potential solutions, join me on January 28 at the PMWC 2014. Our discussion will look at areas of agreement and disagreement regarding the regulation of molecular diagnostics and likely scenarios for the future.

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 Look for additional posts from speakers and participants prior to the 6th Annual Personalized Medicine World Conference on January 27-28, 2014. For more information and the full agenda, visit: 2014sv.pmwcintl.com.

2013: A Year of Innovation, Inspiration

As we look forward to 2014, we engage in the time-honored tradition of reflecting on the year of activities and progress as we bid 2013 farewell.

For personalized medicine, it was another year with big advances, and big questions. The following captures highlights from The Age of Personalized Medicine Blog for 2013.

We started the year with a call to action from Amy Miller, PMC vice president, public policy, about the power of personalized medicine, the need for “accurate and actionable” data, and for a shift in traditional healthcare models. As Amy noted:

…The power of personalized medicine lies not only in treatment, but prevention. The best scenario for personalized medicine in action will come when patients are able to see not only the implications of their genetic dispositions, but also are motivated and empowered to use that information in preventative care.

In May, personalized medicine took the international spotlight as Angelina Jolie shared with the world her genetic prognosis and personal decision to pursue a course of preventative care. The high-profile case inspired much discussion of the value of personalized medicine, as well as the need for access to diagnostic testing for all:

Ms. Jolie’s contribution demonstrates that without patients who are educated, empowered to get tested and to act, the progress and innovation could be for naught. We need to make sure that patients are knowledgeable enough to pursue personalized options for their own health but also so that they are motivated to support policies that foster continued progress in this area.

Kristin Ciriello Pothier, Diagnostics and Life Science Practice lead at Health Advances, shared key take-aways from the PMC/BIO Solutions Summit panel she moderated on “Evidentiary Standards and Data Requirements for Payer Coverage.”

Ms. Ciriello Pothier examined the challenges facing the personalized medicine diagnostics industry and summarized her panel’s conclusions:

First, panelists agreed that there must be more education for all stakeholders so that each stakeholder can actually evaluate novel products appropriately, a key finding echoed throughout the day. Second, the emphasis on outcomes must shift from only clinical outcomes to clinical outcomes and quality of life for patients. Finally, all panelists agreed the ideal situation is open, trusting lines of communication and split of the responsibility according to expertise.

William Chin, M.D., identified research, collaboration, and innovation as critical components to advancing personalized medicine, while Stephen Eck, M.D., Ph.D., cited the need for funding for basic research, diagnostics testing research, increased healthcare provider education, and reimbursement for diagnostic testing as key.

At the mid-point of 2013, pressures to reduce federal spending elevated the need for policy that will support biomedical research and innovation. PMC responded to these concerns through comments submitted to the Centers for Medicare and Medicaid Services (CMS) regarding the charge on regional Medicare Administrative Contractors (MACs) to set new prices for molecular diagnostic test reimbursement through a process called “gapfilling”:

The Personalized Medicine Coalition (PMC) submitted comments to CMS on the Gapfill Payment Amounts and CLFS, iterating the concerns of PMC members that insufficient payment amounts threaten the sustainability of the laboratory industry and continued investment in the developing field of personalized medicine. As a consequence, this policy has the potential to stifle innovation and progress in healthcare and possibly eliminate the potential for lowering overall costs through the elimination of unnecessary and or ineffective treatments.

In 2013, PMC also released a set of policy principles key to advancing personalized medicine in a deficit-reduction environment. Debuted at a Capitol Hill Briefing, these principles lay the groundwork for consensus-building around key issues to ensure that efforts to contain rising healthcare costs do not undermine continued progress in personalized medicine and protect innovation, the physician-patient relationship, patient values, and choice.

The reality of personalized continues to be realized with patient triumph stories bringing new advances in science, technology, and research to life:

The promise of personalized medicine is very real. Personalized medicine is not an abstract concept for the future of medicine. It is here, it is now, and the true promise has been realized in the lives of Kellie and Stephanie, and the precious days, weeks, months, and years they have taken back from their disease.

I encourage you to read more about Kellie and Stephanie, and to draw inspiration from their strength.

At the 9th Annual Personalized Medicine Conference in November, it was an honor to present Kathy Giusti, founder and CEO of the Multiple Myeloma Research Foundation (MMRF) with the 2013 Leadership Award. Ms. Giusti’s story of ingenuity in the face of adversity has led to a redefinition of the role of patient advocacy organizations in research:

Clinically, MMRF has funded research and paved the way for FDA approval for six multiple myeloma treatments in 10 years, and doubled the lifespan for many patients. Today in her remarks, Kathy shared that her daughter, who was just one year old when Kathy received her myeloma diagnosis, is now a 19-year-old college student with a younger brother. Incredible results.

2013 was a year of progress and inspiration. We look forward to the new year ahead and personalized medicine advances on the horizon, bringing new treatments and improved outcomes to patients.

Personalized Medicine Coalition Honors MMRF’s Kathy Giusti with 2013 Leadership Award

Edward Abrahams, Ph.D., President, PMC, (left) and William S Dalton, Ph.D., M.D., Director, Personalized Medicine Institute, Moffitt Cancer Center and CEO, M2Gen, recognize 2013 Leadership in Personalized Medicine Award recipient Kathy Giusti, Founder & CEO of the MMRF (Photo: Justin Knight)

The personalized medicine community has convened in Boston for the Ninth Annual Personalized Medicine Conference. Last night, the Personalized Medicine Coalition welcomed more than 250 attendees to the Boston Museum of Science and the new Hall of Human Life exhibit to kick off the two-day event. As colleagues gathered, the prevailing conversation focused on how far personalized medicine has come. It has been nine years since we first gathered researchers, academics, policymakers, clinicians, patient advocates and other stakeholders to identify the key questions facing – at the time – an emerging approach to healthcare.

Then, and over the years since we first gathered, we have often spoken of the “promise of personalized medicine.” Today, as the Personalized Medicine Conference opened, we focused on how that promise has come to fruition. The personal stories of panelists who carry a dual title of “healthcare expert and patient”, or “genomics expert and caregiver”, riveted the audience and provided a poignant reminder of the need to continue to encourage and enable innovation and access to personalized approaches to care.

One leader in the field, who embodies the promise of personalized medicine is Kathy Giusti, Multiple Myeloma Research Foundation (MMRF) Founder and CEO. This year, the Personalized Medicine Coalition honored Kathy with the 2013 Leadership in Personalized Medicine Award. The award recognizes an individual whose work and contributions are advancing personalized medicine research, product development, reimbursement and policies. Kathy Giusti has done all that and more through her passionate and driven work to empower patients and advance research in multiple myeloma.

The unique and creative business model Kathy employs at MMRF has yielded both clinical and personal results. Clinically, MMRF has funded research and paved the way for FDA approval for six multiple myeloma treatments in 10 years, and doubled the lifespan for many patients. Today in her remarks, Kathy shared that her daughter, who was just one year old when Kathy received her myeloma diagnosis, is now a 19-year-old college student with a younger brother. Incredible results.

The Personalized Medicine Conference continues to be an important gathering for information sharing, consensus development, and, just as critically, these inspirational moments that remind us all that the Age of Personalized Medicine is here as we continue to gain more evidence each day that the science is moving us toward more personalized approaches to research and care.

As Kathy shared at the podium, “I accept this award on behalf of all of you, because you are here as innovators in a field where patients desperately need you. You are here because you are innovators in the field of personalized medicine. You are going to change the lives of thousands and thousands.”

A Response to Ezekiel J. Emanuel’s NYT Op-Ed about the Cost of Cancer Care

Ezekiel J. Emanuel’s “A Plan to Fix Cancer Care” (The New York Times, Op-Ed, March 23, 2013) highlights the need for collaboration and commitment across the oncology community to find solutions for the financial burden cancer poses to our healthcare system.

In order to sustain progress while containing cost, though, we must recognize the heterogeneity of the dreaded disease by eschewing a one-size-fits-all approach. We must, in short, devise strategies that make medical sense. These strategies necessarily will involve adaptive clinical trials, an intelligent regulatory policy that encourages linking therapy and diagnosis, and a willingness to pay for products that improve patient care.

Progress is driven by a dynamic process in which the full value of intervention evolves over time. We must be careful not to stifle innovation by overlooking the critical steps that can provide an answer to the correct question, which is: how can we maximize value from our growing, but not always wise, investments in health care?

From The Catalyst (blog) – A Conversation with Edward Abrahams Ph.D., President, Personalized Medicine Coalition

This entry is reposted with permission from PhRMA’s blog, The Catalyst.

ASCO’s Annual Meeting this week highlighted some of the exciting advances that are emerging in the fight against cancer. PhRMA has joined with many other organizations in supporting a conference next week, “Turning the Tide Against Cancer Through Sustained Medical Innovation,” which is focused on the critical issue of how we sustain this progress in an era of increasing cost-cutting pressure. Conference participants will examine how we measure the value of new treatments and how we can promote high quality, patient-centered care.

The conference is being convened by the American Association of Cancer Research, the Personalized Medicine Coalition, and Feinstein Kean Healthcare. We sat down with each of the conference co-hosts about the event and progress in cancer care.

A Conversation with Edward Abrahams Ph.D., President, Personalized Medicine Coalition

Q:  What are the implications of personalized medicine’s growing role in research and patient care in oncology?

Ed Abrahams:  Technological improvements in genetic sequencing and molecular profiling of tumors have given researchers and clinicians a new ability to understand the molecular and genetic characteristics of cancer patients and their tumors.  By more accurately classifying disease, patients can benefit from targeted treatments that are more likely to work.

Simply put, personalized medicine means better patient care. Personalized treatment regimens allow clinicians to select treatments that are more likely to attack specific tumor types.  But personalized medicine also delivers better value to patients and the health system because it enables clinicians to spare patients the expense and side-effects of treatments that are not likely to provide tangible benefits and thereby to help address the challenge of rising health care costs.

We believe that the future of cancer therapeutics lies in molecular biology and genomics, supported by electronic health data systems and advanced health information technologies that support a rapid learning health care system.  Increased collaboration between academia, the biopharmaceutical industry, philanthropic organizations, and patient advocacy groups will maximize the development of new personalized medicine technologies and treatments.

Q:  What are some of the barriers to personalized medicine that need to be addressed to facilitate the adoption of personalized medicine?

Ed Abrahams:  Clearer regulatory and reimbursement pathways are needed to ensure that developers of new personalized medicine products – therapeutics, diagnostics, and others laboratory services – have a reasonable pathway to get their products approved by regulatory agencies and to ensure that products are reimbursed at rates reflective of the innovative value of the product and the investment necessary to develop it.

We must also aligning comparative effectiveness research (CER) with the science of personalized medicine and the fact that we are not always aware of all of the uses for a new therapy at the time of its approval. If we rush to judgment about value too early, we will cut short the process of continuous learning through which value emerges. If we take a traditional “one size fits all” approach to CER that judges “average” value at a point in time, we will stifle innovation.

Finally, our education system must evolve to ensure that our health care workforce is trained in genetics, genomics, and the technologies needed to integrate personalized medicine into cancer treatment decision-making.

Q:  What is your goal for the conference?

Ed Abrahams: This conference will provide stakeholders a forum to discuss major developments in oncology research and development and link the science of personalized medicine with public policy.

We intend the high-level discussion at the conference to illuminate policy solutions that will spur the development of new personalized medicine approaches to cancer.  By brining patients, payers, industry, and academe, we hope to identify ways to continue to deliver high value care in a cost constrained environment and to sustain innovation to improve cancer patient outcomes.

Levin, O’Kelly Provide Perspective on Decades of Personalized Medicine Progress, Urge Action and Support

On Tuesday, May 8, 2012, the Personalized Medicine Coalition (PMC) welcomed our chairman, Stafford O’Kelly, President of Abbott Molecular and keynote speaker, Mark Levin, Partner and Co-Founder of Third Rock Ventures to its Eighth Annual State of Personalized Medicine Luncheon. The event brought together PMC’s members, partners and other stakeholders involved in realizing the future of personalized medicine for researchers, industry leaders, patients, caregivers, advocates and policymakers.

Stafford O’Kelly reminded us, in his introductory remarks, of the progress and success we’ve made toward achieving personalized medicine in practice within the past year alone. Just last week we saw that Xalkori is showing progress in fighting certain childhood cancers. O’Kelly pointed out the assurances by the FDA that co-approval of drugs and diagnostics will occur more frequently. He also challenged attendees to think about what they can do “to accelerate the shift toward personalized medicine in a way that will improve treatment outcomes for the patient while, at the same time, lower overall costs for our health care system.”

Mr. Levin sought to use his significant experience and background in venture capital, product development and marketing to answer O’Kelly’s provoking question during his keynote address. He urged attendees to support personalized medicine. He explained that “personalized medicine is one of the most important things in medicine today” and lauded its potential to reduce safety challenges, increase efficacy, and improve productivity in the pharmaceutical industry.

I agree that we are at a point where the scientific and clinical progress made in personalized medicine is undeniable. But to echo Stafford O’Kelly’s call to action:  “We need to get the right stakeholders involved to help develop pathways for accelerating growth in the field….We need commitment from payers, providers and patients.”  In order to continue to make progress against disease and improve outcomes for patients, it is necessary as O’Kelly and Levin said, for the entire ecosystem of players to focus efforts in the direction that scientific discovery points us – toward targeted, patient-centric approaches to research and care.

We hope to continue the discussion around these themes and the future of innovation, specifically in cancer research and care at “Turning the Tide Against Cancer Through Medical Innovation,” a national conference that the Personalized Medicine Coalition, American Association for Cancer Research and Feinstein Kean Healthcare are co-hosting on June 12, 2012.  I encourage you to join us in Washington, D.C. as we look to identify and build support for an environment that sustains innovation and drives our evolution toward personalized cancer care.

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.

Personalized Cancer Care Requires Effective Diagnostic Tools

On July 8th, the New York Times published a lead story “How Bright Promise in Cancer Testing Fell Apart” that questions the value of genetic testing to determine cancer treatment strategies. I wrote a letter to the editor, which the New York Times published on July 12^th and read in part:

The larger reality is that patients benefit from personalized medicine every day.

Safe and effective targeted therapeutics have reshaped treatment for breast cancer and leukemia, for example; and just last month, reports from the American Society of Clinical Oncology of successful clinical trials for targeted therapies in melanoma and lung cancer are expected to do the same.

In fact, complex genomic tests have been on the market for years. A survey by the Tufts Center for the Study of Drug Development found that 94 percent of biopharmaceutical companies are investing in personalized medicine and that 100 percent are using biomarkers to learn more about compounds during the discovery phase of their research.

If it were not for the opportunities this field offers to improve outcomes by matching the right treatments to the right patients, we would not see industry or the federal government making these investments in an effort to advance the frontiers of medicine.

Clearly, as the story reports, some trials will fail and unfortunately, even a single case of fraud is too many.  Nevertheless, to leap to the conclusion (and print it on the front page of the New York Times) that cancer testing is in danger of “falling apart” or to suggest that we are locked into a one-size-fits-all paradigm overlooks current and future capacity to improve diagnosis and target treatment.  We should not lose sight of the value personalized medicine provides to patients while maintaining our commitment to scientifically sound research that supports molecular medicine.