Author Archive

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.

PCORI Announces Comment Period; Feedback Critical to Ensure Quality Research

July 25, 2012

The Patient-Centered Outcomes Research Institute (PCORI) announced this week the public comment period for its landmark draft Methodology Report, which proposes standards for the conduct of patient-centered outcomes research.  While the draft report came out last month, the comment period closes on Friday, September 14, 2012.

The Personalized Medicine Coalition (PMC) has a long history with PCORI starting from when the legislation creating it was being drafted.  We advocated for recognition of personalized medicine in the conduct of comparative effectiveness research, which was incorporated into the legislation.  PMC’s methodology committee is charged with ensuring that the research funded by PCORI examines, among other things, research question responses based on patient molecular subgroup.

As part of the comment period announcement, PCORI stated that feedback received during the comment period would be reviewed for potential incorporation into a revised version of the report. The revised version will be considered for adoption by the PCORI Board of Governors at its November 2012 public meeting in Boston, Mass.  We have previously urged PCORI to be explicit in how the public comments are considered and incorporated. That, and other institutional development, is essential for the proper functioning of the organization.

The current report does not adequately address personalized medicine as an important factor in improving health outcomes through patient-centered outcomes research methodology. PMC will comment on the methodology report, and we urge our member organizations and others to do the same.  Active input from the community is required so that the research will live up to the high expectations that we have for it.

Congress Recognizes Value of Personalized Medicine Approaches in FDA User Fee Authorization Legislation

June 27, 2012

Regulatory uncertainty has long been a concern for personalized medicine innovators and the Turning the Tide Against Cancer conference earlier this month reinforced the need for regulatory changes to facilitate personalized medicine.  While that uncertainty is not gone, the Food and Drug Administration (FDA) user fee reauthorization legislation, which passed its last major hurdle through Congress yesterday, contains personalized medicine-relevant policy changes that will enhance the regulatory process for new products.

We applaud Congress for passing this legislation and commend the measures it takes to smooth the way for more personalized medicine products.  Provisions directly related to personalized medicine include:

  • The development of FDA’s capacity to review submissions involving pharmacogenomics and biomarkers by integrating and training staff with pharmacogenomics and biomarker expertise into the product review process.
  • The creation of an expedited approval process for “breakthrough therapies” — drugs that are both developed using biomarkers and used to treat serious or life-threatening ailments.
  • A requirement that the FDA notify Congress two months in advance of the Agency’s planned release of any guidance or draft guidance document related to the regulation of laboratory-developed tests.

We look forward to continuing to work with FDA to improve the complex regulatory process for personalized medicine and accelerate adoption new personalized treatments.

Progress in Cancer Highlighted by NEJM Retrospective; Turning the Tide Conference to Catalyze Comprehensive Dialogue on How to Sustain Cancer Innovation

June 8, 2012

In honor of the New England Journal of Medicine’s (NEJM’s) 200th Anniversary, the journal examined how medicine has evolved over the last two centuries, looking in particular at oncology diagnosis, prevention, and treatment. But while there has been tremendous progress in cancer, questions remain:  Where do we go from here?  And how do we get there in an era of immense fiscal discipline?  These are questions that we plan to address on Tuesday at our conference, Turning the Tide Against Cancer Through Sustained Medical Innovation.

In a similar vein to what Siddhartha Mukherjee, M.D., a special guest speaker at the conference, lays out in his book The Emperor of All Maladies: A Biography of Cancer, the authors of the NEJM article “Two Hundred Years of Cancer Research” provide a timeline of major discoveries and advances in cancer research and care.

They show how each milestone is built on the ones that came before it and trace the evolution of cancer progress from the early efforts to control the disease through surgery, advances in radiation, chemotherapy, and the targeted therapies that are redefining cancer treatment today.

The underlying science that made these treatment advances possible takes years to translate into clinical benefits for patients, but the original investments pay off. Our understanding of the genetic basis of cancer became possible only after decades of work on the basic biology of DNA beginning in the 1940s and 50s, but it was not until after the sequencing of the human genome that researchers were able to begin to translate genetics knowledge into new medicines.

Genetic understandings of cancer have led to breakthrough new medicines such as Xalkori® (for non-small cell lung cancer) and Zelboraf® (for melanoma) and more targeted therapies are on the way.  A new report issued by the Pharmaceutical Research and Manufacturers of America (PhRMA) found that there are 981 new medicines and vaccines for cancer in development today, many of which are likely to be personalized medicines.

Tomorrow’s progress in cancer therapies and treatment approaches depend on today’s policy makers recognizing the need for policies that holistically support cancer research and innovation.  In advance of next week’s conference, a Discussion Paper “Sustaining Progress Against Cancer in an Era of Cost Containment” coalesces the views of the conference advisory committee and other leading cancer experts about new models for cancer innovation, how to define value in cancer care, and how policy can support continued progress against cancer.


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