Author Archive

Targeted Therapies: From Promise to Reality

August 15, 2013

A recent Wall Street Journal article featured the impact that the genomics revolution is having on cancer patient care with a focus on lung cancer. The article shares a strong message of hope, detailing the scientific progress made thus far by noting that we have experienced the “most extraordinary decade of progress ever in the long scientific struggle against lung cancer,” while also pointing to the promise further personalized approaches to cancer care holds.

Lung cancer patient Kellie Carey poignantly shares her story of how she had to demand testing for her tumor to identify its mutation, opening the door for her to molecularly targeted drugs that extended her life.

This message echoes what we heard during the briefing Personalized Medicine: How Medical Progress Happens held by the Personalized Medicine Coalition on Capitol Hill just last month — in particular, the experiences of one of the speakers, lung cancer survivor Stephanie Haney (click here for a video interview with Stephanie). Stephanie and Kellie share a disease and a journey as they have both been “lucky” enough to have been diagnosed with lung cancer in the midst of a revolution in care.

For thirty years the available treatments extended life a few weeks, possibly months. Lung cancer diagnosis was an immediate and imminent death sentence. Now, Stephanie, Kellie, and others with genetically identified tumor mutations have access to targeted treatments that are extending their lives years longer than those lung cancer patients on chemotherapy only and without identified mutations.

These are just two examples of the value of innovative therapies, particularly molecularly targeted drugs. 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.

And now it is our promise to Kellie, Stephanie, and others to continue to push forward, toward further discovery, and to drive innovation.  There are nearly 1,000 new medicines in the pipeline for cancer alone.  We can all do our part by supporting policies that enable and encourage innovation and allow the science to guide us.

Examining Policy That Will Support Innovation, Personalized Medicine

July 22, 2013

Policymakers are facing pressure to control federal spending and lower the national debt, with healthcare costs as a focal point of debate to address this pressure. The path that policymakers take will have a significant impact on biomedical progress, the role of U.S. companies as global leaders in life science innovation, and the quality of patient care.

One example of how policy can impact healthcare innovation is playing out right now. The Centers for Medicare and Medicaid Services (CMS) 2013 Gapfill Payment Amounts and Clinical Laboratory Fees Schedule (CLFS) charged regional Medicare Administrative Contractors (MACs) to set new prices for molecular diagnostic test reimbursement through a process called “gapfilling.” This process has resulted in much lower payment amounts for molecular diagnostics which has had unintended consequences.  Some innovators have reported that drafting cooperation agreements to bring new targeted therapies to market has been stifled. Some clinical labs have reported that they might not be able to offer some tests since new payment rates do not cover the costs of running them, which may negatively impact patient access to diagnostic tests and appropriate treatments.

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.

With dramatic advances in science, it has become more important than ever to ensure that policymakers understand the value of biomedical innovation and support pathways that encourage continued advancement and yet we see deficit reduction pressures regularly resurfacing in new healthcare policies.

To that end, PMC is hosting a Congressional Briefing on Monday, July 22. We will examine the role personalized medicine plays in healthcare and how our public policy can drive progress and innovation. At the briefing, PMC will unveil policy principles to ensure that proposed cost-containment efforts do not undermine personalized medicine, but instead, protect innovation, the physician-patient relationship, and patient values and choice. We also will hear the diverse perspectives on innovation from lung cancer survivor and patient advocate, Stephanie Haney; drug developer, Dr. Stephen Eck; diagnostic developer, Patrick Balthrop; and physician, Dr. Amy Abernethy.

At PMC, we look forward to our continued collaboration with the stakeholder community to identify opportunities for public policy to drive progress and innovation.

Angelina Jolie Lends Her Voice and Experience to Personalized Medicine

May 16, 2013

It was great to see Angelina Jolie’s eloquent op-ed in the May 14 edition of The New York Times. She carefully wove her personal experience with the importance of genetic testing and using the resulting information to make an informed health decision. I applaud her decision to go public with her story as a great example of patient empowerment but also as a powerful example of personalized medicine in action.

Despite all the advances we have made in recent years, we all know that the concept of personalized medicine is still foreign to most Americans. PMC recently conducted focus groups which strongly reminded us of the uphill battle that remains to gain public understanding of and support for personalized medicine. In our groups only one or two out of 52 participants were able to correctly describe the concept.

We found that people are very enthusiastic about personalized medicine once they understand it but it is a complex concept which is often misunderstood. It turns out that giving clear, specific examples of personalized diagnostics and treatments is the best way to explain what personalized medicine is and the benefits it can bring.

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.

Angelina Jolie’s op-ed is an important and high-profile contribution to the discourse about patient experience and personalized medicine.  Her voice echoes the words of Adriana Jenkins, who courageously shared her battle against breast cancer and joins other advocates like Donna Cryer, who works tirelessly to remind all stakeholders about how personalize medicine is a patient-centric approach to healthcare. Courageous individuals, like Ms. Jolie, who publicly share their personal stories have the power to bolster both public and media discourse around patient empowerment, for example with CNN “Early Start” co-anchor Zoraida Sambolin’s disclosure of her own breast cancer diagnosis and treatment decision live on air.

I hope that Ms. Jolie’s op-ed raises awareness of personalized medicine and the options patients have to take control of their healthcare.  No single organization alone can raise public awareness on the scale that we need but through a steady stream of individual op-eds, blog posts, conversations, etc., we in the personalized medicine community can spotlight more stories like this and begin to educate the public.  I look forward to hearing more voices from the community and hope that their contributions will support personalized medicine research, patient and provider engagement, and thoughtful policy.

Evidence, Coverage, and Incentives: A PMC/BIO Solutions Summit Conference

April 10, 2013

Personalized medicine is changing the way medicine is practiced, and how drugs and biologics are developed.  Systemic changes are needed to reflect this and support product development, but current economic, regulatory, and practice disincentives are impacting and slowing development. For example, medical advances often take years to be recognized in clinical care guidelines and cost containment policies often prevent new diagnostics from being priced at a level that supports innovation.

The Personalized Medicine Coalition (PMC) and the Biotechnology Industry Organization (BIO) have joined forces to address these challenges. A series of PMC/BIO Solutions Summits will convene stakeholders with the charge to identify and discuss potential solutions to these and other barriers.

The first of this series, Evidence, Coverage & Incentives, will be held Wednesday, April 17, in Washington, D.C. I encourage you to join this full-day conversation uniting stakeholders from across industry as we work to ensure that our healthcare system benefits from all personalized medicine promises to offer.

Specific discussions will focus on:

  • Challenges of reimbursement;
  • Evidentiary standards and data requirement for payer coverage;
  • Clinical guidelines;
  • Investing in molecular diagnostics;
  • Issues impacting market access; and
  • Incentivizing personalized medicine development.

The challenges are daunting, and it will take cooperation among all stakeholders to bring the benefits of our scientific advances to fruition. This summit will work toward solutions to foster innovation in our collective thinking and to identify pathways for improving the overall quality and efficiency of care. I encourage you to lend your voice to these efforts.

This blog also appears on the BIOtechNOW website.

Putting the Pieces Together for Personalized Medicine

April 8, 2013

At the Personalized Medicine Coalition, our goal is to open the path to innovation in the science and practice of personalized medicine. “Personalized medicine” is the tailoring of medical treatment to the individual characteristics of each patient. For personalized medicine to work, many pieces must align, including detailed knowledge of the genomics underlying a given disease, effective targeted treatments, and accompanying diagnostic tests to identify the patients who are most likely to benefit.

Those pieces do not line up overnight but, instead, each aspect advances as knowledge builds cumulatively over time. For example, the technology to sequence genes has come a long way in the last 15 years. According to our latest edition of The Case for Personalized Medicine, the cost of sequencing the human genome dropped from $300 million in 2001 to $5,000 in late 2011. This dramatic price drop reflects the evolving science that built over time. Without the initial $1 billion investment to sequence the human genome by public and private partners, this progress would not have been possible.

Likewise, we see the same accumulation of knowledge related to personalized treatments over time. Even within one medicine, our understanding of its potential benefits and indications evolves.

Today, we can sub-classify certain diseases based on genetics: Melanoma can be BRAF positive or non-small cell lung cancer can be EGFR positive or ALK positive. Making these distinctions has led to targeting these gene mutations with specific treatments, leading to improvements in patient outcomes and quality of life. Tomorrow, our scientific knowledge base will continue to grow and is coupled with the technological advances that are enabling the analysis and interpretation of findings like no other time in history.

Increasingly, our expanding knowledge of the role of genetic variation in patient response to treatments will drive continuous learning about the optimal role and value of treatment regimens and diagnostic/treatment combinations. This emerging capability holds great potential to improve patient care and healthcare value, but it is at odds with our conventional approaches to assessing value at a point in time based on broad average study results.

To foster continued progress, the private and public sectors must work together to develop policies that align with scientific advances. We need adequate reimbursement for advanced diagnostics and targeted therapies, and flexible methods to access the value of personalized medicines, rather than one-size-fits-all determinations that don’t take the individual into account. We need policies that recognize the many factors that come into play to make personalized medicine possible, including the research, development, and performance of molecular testing.

At the Personalized Medicine Coalition, we firmly believe that we can and must bring personalized medicine to all patients. Collaboration among the many stakeholders involved will move us closer to this goal.

– A version of this blog originally appeared in The Catalyst series highlighting incremental innovation posted at the PhRMA website.

Shaping the Future of Personalized Medicine with TEDMED

March 22, 2013

I had the opportunity recently to participate in a TEDMED Great Challenges panel discussion entitled, “Shaping the Future of Personalized Medicine.” My fellow panelists and Challenge Teammates included representatives from 23andMe, Foundation Medicine, Illumina, and InformedDNA.

As we discussed the challenges, opportunities and benefits of personalized medicine, three themes emerged.

First, personalized medicine, at its root, is about empowering patients to participate in their own healthcare. Trends in popular culture are meshing with advances in technology to allow consumers access to their health information and the ability to make informed decisions, and our culture is changing in that many now demand to be an involved party in their healthcare.

Amber Trivedi of InformedDNA noted that the power of personalized medicine lies not only in treatment, but prevention. As a genetic counselor, the most common questions asked by her patients are:  “What does my genetic information mean to me, and what will it mean to my children?” 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.

Second, as Michael Pellini, the CEO of Foundation Medicine said, data has to be “accurate and actionable” to have value. However, we cannot discount the potential future benefits of the data discoveries that are underway today. We must find a balance between supporting ongoing data discovery and analysis, while also pushing for data that are actionable now.

Third, traditional healthcare models need to continue to shift to aid in the advancement of personalized medicine. Research cannot continue to solely focus on large population studies; payers need to develop innovative approaches to improve reimbursement policies; and healthcare professionals need training and resources to enable the adoption of personalized medicine in the clinical world.

Finally, when asked what we found to be the most invigorating about personalized medicine, my fellow panelists were quick to share the advances they see on the horizon, including an explosion in targeted therapies aided by new technologies; the shift of cancer to a chronic disease; revolutionizing the treatment of infectious disease through the application of lessons learned from personalized medicine advances in cancer; and deeper data mining enabled by technology currently used in other fields.

Regardless of our individual areas of focus within the world of personalized medicine, this Challenge Team is energized and optimistic about the future of medicine.

Follow the Great Challenges conversation, and submit your questions or comments on Twitter using #GreatChallenges, or at TEDMED.

The Myth of Average: Why Individual Patient Differences Matter

November 26, 2012

Everyone understands the need to bring greater cost-efficiency to American healthcare. That desire to contain spending drives changes in both the public and private sectors. Yet, what we’ve learned – and are learning more each day through advances in personalized medicine – is that there is no single solution for the healthcare needs of an entire population.

This challenge is going to receive a much-needed focus at the forum, “The Myth of Average: Why Individual Patient Differences Matter,” taking place on November 30 in Washington, D.C. As a co-sponsor for this full-day discussion, the Personalized Medicine Coalition applauds our colleagues at the National Pharmaceutical Council, National Health Council, and WellPoint for constructing an event at which the dialogue can truly make a difference. Leaders from the Centers for Medicare and Medicaid Services (CMS) and the Patient-Centered Outcomes Research Institute (PCORI) will be on hand, alongside patient representatives and experts in biopharmaceutical innovation and medical research.

Speakers will share their perspectives on how health policy decisions can impact patient care and specifically inform a broader understanding that comparative effectiveness research has an important role to play in improving clinical care. You can view the full agenda at http://www.regonline.com/builder/site/tab2.aspx?EventID=1129899. I hope to see you there.

Do Not Stymie Innovation by Denying Reimbursement

October 4, 2012

Last week the public comment period closed for the Centers for Medicare and Medicaid Services (CMS) proposed payment determination for Multianalyte Assays with Algorithmic Analyses (MAAAs) in the CY 2013 New and Reconsidered Clinical Laboratory Fee Schedule (CLFS) Test Codes and Preliminary Payment Determinations (“the Preliminary Determination”).

MAAAs are procedures that utilize multiple results derived from assays of various types. The American Medical Association gives the following definition: “Algorithmic analysis, using the results of these assays as well as other patient information (if used), is then performed and reported typically as a numeric score(s) or as a probability. MAAAs are typically unique to a single clinical laboratory or manufacturer. The results of individual component procedure(s) that are inputs to the MAAAs may be provided on the associated laboratory report; however these assays are not reported separately using additional codes.”

MAAAs are not new.  CMS and private payers routinely pay for them and many of them, having been added to clinical care guidelines, are now considered standard of care.

The proposal, as it stands, will likely prevent providers from receiving reimbursement for MAAAs. Without a clear reimbursement pathway, we risk stifling innovation by sending a powerful message to developers and providers that they cannot recoup investments made in the development, clinical validation, and commercialization of innovative diagnostic products.

Innovators focused on developing new therapies and accompanying diagnostics have difficult decisions to make regarding what products to invest in and bring to market. They must have confidence that future innovative diagnostics will be recognized and valued or improvements in patient care could be at risk.

At the urging of our members, the Personalized Medicine Coalition (PMC) sent a letter to CMS on this issue, noting that the Preliminary Determination:

  • Reverses current practice as many of the MAAAs are well-established tests that have been covered and reimbursed by Medicare for several years and are medically necessary given their status as the standard of care in treatment guidelines;
  • Jeopardizes personalized medicine and medical innovation by not separately recognizing and valuing the MAAA CPT codes; and
  • Represents a lack of transparency in the CMS decision-making process.

PMC recommended that the Preliminary Determination be altered to take into consideration these perspectives, shared by all PMC member organizations, to ensure that the decision does not inadvertently impact current patient care and the future of biomedical innovation.

Healthcare delivery and our research enterprise continue to change as innovators apply new scientific discoveries to the development of new therapies to treat and manage illnesses.  Our reimbursement policies must also adjust in step in order to support our evolving healthcare system.

Today, physicians across the country use sophisticated diagnostics to guide treatment decisions, a trend that we expect will continue. The rise in personalized medicine will lead to efficiencies in the healthcare system that increase the quality of patient care while saving patients exposure to unnecessary treatments that may not help them. Let us not halt innovation in healthcare by hasty policy decisions, such as the Preliminary Determination, that are not fully vetted for unintended consequences.

PMC’s Suggestions to PCORI’s Methodology Committee

September 14, 2012

The September 14, 2012 (11:59 PM ET) deadline for submitting public comment on the Draft Methodology Report recently published by the Patient-Centered Outcomes Research Institute’s (PCORI) Methodology Committee quickly approaches.

We encourage you all to submit comment as the revised version will be considered for adoption by the PCORI Board of Governors at its November 2012 public meeting in Boston, Mass.

PCORI was designed to do something new and very different – to define how practical, feasible, and useful patient-centered research is to be conducted and the legislation authorizing it directed the methodology committee to address the role of molecular and genetic variation in comparative effectiveness research. With this bold goal outlined, the Personalized Medicine Coalition (PMC) issued its comments to Dr. Joe Selby and Dr. Sherine Gabriel, the Executive Director and Chair of PCORI on September 13, 2012. You can find a copy of that letter here.

Citing our prior work with the Lewin Group, PMC stressed the importance of PCORI moving beyond conventional CER analysis, noting that “comparative effectiveness research (CER) studies to date have rarely accommodated the collection and reporting of genomic, behavioral, environmental, and other individual patient differences.”

To ensure that the tenets of personalized medicine outlined in the authorizing legislation are upheld, PMC reiterated our call for creation of a PCORI Advisory Panel on Personalized Medicine and Innovation and for establishment of standards for updating studies and analyses based on changes in the science or evidence base. We also are making recommendations to address:

  1. Heterogeneity of Treatment Effectiveness Standards: Recommend that either a separate section on standards for biomarkers and genetic variables be added to the report or an additional section on heterogeneity of treatment effectiveness be developed to guide the use of biomarker data in patient-centered outcomes research.
  2. Diagnostic Test Study Standards: Develop a new paradigm for collecting and analyzing evidence by recommending variations in new clinical trial design, using patient registries to identify respondents, analyzing archived biospecimens, and implementing retrospective analysis of laboratory data.
  3. Stakeholder Engagement: Release a table outlining the categories of specific comments received in all future comment submission periods with an explanation of how PCORI addressed the comments as a way to re-emphasize the importance of stakeholder engagement.

Approaching One-Year Anniversary at FDA, Stephen Spielberg Highlights Agency’s Progress in Personalized Medicine

August 20, 2012

In his address to the Personalized Medicine Coalition (PMC) Policy Committee at our most recent meeting, Stephen Spielberg, M.D., Ph.D., Deputy Commissioner for Medical Products and Tobacco at the U.S. Food and Drug Administration (FDA), announced that the Agency will develop a catalog of personalized medicine-related activities. The catalog, as Dr. Spielberg described, will provide a full accounting of the activities at the agency, including all regulatory divisions and regulatory science.

While reflecting on his 11-month tenure at the agency, Dr. Spielberg also noted that the largest area for advancing personalized medicine was through communication among stakeholders and FDA Centers.  He noted that “PMC is so important because we need dialogue; no one has a lock on complete information.”

The attendees of the policy meeting were pleased to hear that already, less than a year into Dr. Spielberg’s appointment at the agency, he was working to encourage collaboration and communication across divisions.  Dr. Spielberg was previously Director of the Center for Personalized Medicine and Therapeutic Innovation at Children’s Mercy Hospital, one of the founding members of the PMC and so has experience in bringing groups together to advance personalized medicine.

Dr. Spielberg outlined his optimism for personalized medicine at the FDA, noting that one third of the new drug approvals currently in review are for targeted or orphan indications.  He said that by year’s end, we could expect guidance documents on companion diagnostics and co-development, as well as the previously mentioned catalog.

These guidance documents and a FDA catalog may prove to be additional stepping stones to a broader understanding of the impact of personalized medicine on scientific research and clinical medicine.  At PMC, we will continue to work with FDA to promote broader engagement across the ecosystem of stakeholders and greater transparency around research and drug development.  We look forward to the issuance of these materials later this year and will engage a public discourse about them.


Follow

Get every new post delivered to your Inbox.

Join 192 other followers

%d bloggers like this: