Author Archive

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.

National Bioeconomy Blueprint Showcases Personalized Medicine as Model for Strengthening U.S. Bioeconomy

May 7, 2012

Last week, the White House released its National Bioeconomy Blueprint.  It lays out some strategic objectives designed to help realize the full potential of the U.S. biotechnology sector to generate economic growth by creating jobs and addressing societal needs.

As an example of how the government’s efforts can facilitate the development of a more robust bioeconomy, the report discusses the impact of the Human Genome Project and the development of personalized medicine. The Blueprint cites the Case for Personalized Medicine, 3rd Edition, noting “advances in recent technologies have increased the momentum of personalized medicine – customized healthcare based on specific genetic or other information of an individual patient.”

While we agree that policies are needed to help support research and development (R&D), improve translational and regulatory science, improve regulation in other areas, enhance workforce training, and develop new public-private partnerships and precompetitive collaborations, we are concerned they are not sufficient to allow us to realize Adriana Jenkins’ dying wish, that all patients have access to personalized treatments.

The White House is correct to shine a light on FDA and direct the agency to focus attention on application review times, coordinated parallel reviews of products, and continued improvement of regulatory science. In reality, we are already seeing the benefits of this increased coordination – with FDA’s accelerated review and approval of Kalydeco™ for cystic fibrosis and Xalkori® for non-small cell lung cancer, each with a companion diagnostic approved together and in advance of FDA time lines.

Still, engaging on regulatory science and streamlining FDA processes will only go so far to improve the bioeconomy and bring personalized medicine to patients. For instance, current comparative effectiveness research (CER) and health technology assessment (HTA) models, which are not addressed in the Blueprint, are not well aligned with the science of personalized medicine and such misalignment causes additional barriers to market entry and patient access after FDA approval. The Blueprint highlights coverage with evidence development (CED) as a potential HTA model, but it should be noted that although CED can be a good tool, if done wrong, it can also chill innovation.

Likewise, unclear and unrealistic expectations for obtaining Medicare coverage and adequate payment for personalized medicine products and services are a substantial hurdle, preventing companies from seizing the full scientific potential and translating that into new treatments and the resulting high-paying jobs and economic contributions that follow.

We look forward to working with the Administration and with Congress to shape policies that will help support the ability of companies to continue to develop personalized medicines and bring them to patients, including research and development tax credits, delivery system reforms, and regulatory and reimbursement policies. Given the tremendous potential of personalized medicines, it’s key that we get the policies right to foster companies working on personalized medicine and thereby improve patients’ lives and our economy.

PCORI’s Selby Defines Research Agenda; Tells PMC that Institute Will Solicit Continued Feedback to Ensure CER Supports Personalized Medicine

April 30, 2012

The Patient-Centered Outcomes Research Institute’s (PCORI) Board of Governors held a conference call meeting Wednesday afternoon.  During the call, they announced the finalization of their research agenda and discussed changes made from the initial draft in response to stakeholder comments.

Given the many criticisms PCORI received for being too broad and vague in its draft, I was surprised by how quickly the research agenda was finalized.  I was gratified that the Institute publicly detailed how stakeholder comments shaped the final agenda, as urged by the Personalized Medicine Coalition (PMC) in our letter to the Institute last month.

Last week, PCORI Executive Director Joe Selby also addressed PMC’s public policy committee, discussed the Institute’s rationale for its agenda – which was short on specifics – and answered questions from PMC members.

During his talk, Selby shared that PCORI’s goal for its research agenda was to be a foundation for future work.  Through funding announcements, the Institute hopes to solicit a broad range of research proposals that will offer many options on approaches to implement and conditions to study.

Selby suggested that PCORI aims to keep the door open to provide funding for comparative effectiveness research (CER) studies that may benefit small subpopulations or rare disease research, and not to fund only research addressing common conditions such as heart disease, depression or diabetes.  However, several PMC members pointed out that ultimately PCORI’s funding decisions will pick winners and losers.

Selby encouraged the submission of proposals to the Institute for studies designed to demonstrate what types of treatments will work for different subgroups of patients (as PMC believes all CER studies should aim to do).  PMC was disappointed that he did not commit the Institute to such an approach as we believe the statute requires, although he noted that research announcements will solicit thematic proposals in addition to disease-specific ones that may address some personalized medicine topics.

PMC Members asked Dr. Selby about the development of PCORI’s infrastructure and questioned whether the Institute has what it needs to align CER with personalized medicine.  As I noted previously, PMC does not believe that the Institute has developed the internal structure necessary to carry out its mission at this time.  Dr. Selby informed the PMC policy committee that PCORI welcomes its feedback about how to assure that funded CER-studies align with personalized medicine through its open meetings, public comments, and advisory committees.

To facilitate personalized medicine, CER must explain not only what works best – but also for whom.  The future of medicine depends on a careful and critical answer to this critical question.  We are not yet sure that PCORI has embraced this idea.  For example, when the Board met to discuss altering the research agenda based on public comment, it tried to more clearly define the need to study personalized medicine. It failed to cite the most common definition of personalized medicine, and instead, discussed mostly demographic characteristics. The PMC will respond to the altered language with the expectation that the definition of personalized medicine will be accepted into the research agenda language. This is the most basic, first step to aligning CER with personalized medicine.

We valued Dr. Selby’s willingness to dialog with the PMC community and look forward to continued engagement with PCORI to ensure that CER can be supportive of quality health information, tailored to patients’ values and individual biology.

Improved PCORI Infrastructure Needed to Deliver on Personalized CER Mandate

March 19, 2012

As I mentioned previously, the Patient-Centered Outcomes Research Institute (PCORI) issued its draft research priorities earlier this year.  The Personalized Medicine Coalition (PMC) provided comments on the draft that outline how PCORI must address fundamental structural issues to meet Congressional intent.  Specifically, the intent is that the research overseen and conducted by PCORI should support personalized medicine and align comparative effectiveness research with personalized medicine.

In the public comments on the draft research priorities, PMC offered five recommendations, outlining how PCORI can build the infrastructure needed to execute its mission as Congress intended:

 1.  Define a public engagement process: PCORI should outline a transparent process for obtaining input from all stakeholders, including patients, clinical experts and scientists, and detail how stakeholder input will be used. One possibility is to assemble all comments in a document and demonstrate how they informed the final draft.

 2. Establish a personalized medicine expert advisory panel: PCORI has the statutory authority to create expert advisory panels, on any topic, to carry out its mission. To assist PCORI with assuring that its work supports personalized medicine, PMC strongly requests that PCORI develop an expert advisory panel devoted to personalized medicine. As an educational organization dedicated to advancing the field and populated by stakeholders from all sectors of the health care universe, the PMC Clinical Science Committee offers its assistance in identifying potential members for this proposed expert advisory panel.

 3. Improve the science behind comparative effectiveness research (CER): One of PCORI’s Congressionally mandated tasks is to improve the quality of CER by incorporating new information and technological innovations into its studies, reviewing and updating the evidence as necessary and identifying future research that is needed to address information gaps. PMC suggests creating the infrastructure (and the processes) to achieve this goal now as the foundations of the organization are being established.

4. Make the research priorities more specific: The PCORI draft research priorities envisioned by statute were broad, encompassing all aspects of the health care system that relate to high-quality, effective patient care–and specific, calling for a transparent process to identify and prioritize research topics based on explicit criteria and public input. PMC strongly suggests that PCORI’s materials address the second point, assuming that the statute envisions a broad scope of research.

 5. Develop in-house capacity to engage broad scientific and clinical expertise: The mission of PCORI is unique and to carry it out PCORI must call on the capacity of a unique set of individuals to develop calls for research proposals, evaluate them, make awards, follow the progress of the research, and engage the public at all steps in the process. Having this infrastructure “in-house” is a necessary step in the Institute’s development.

We look forward to working with PCORI on these initiatives to strengthen its research agenda and infrastructure.  Personalized CER has great potential to elevate the quality of care in the United States and we are ready to do our part.

PCORI Research Priorities, Will They Support Personalized Medicine?

January 27, 2012

The Board of Directors of the Patient-Centered Outcomes Research Institute (PCORI) met last week, outlined research priorities, and asked for public feedback. What I found most interesting, and a little disheartening, is that the priorities are drafted in vague language, making it difficult to determine how they may (or may not) support personalized medicine.

I hope that as PCORI moves forward, it will give assurance to the Personalized Medicine Coalition (PMC) and other stakeholders that its research supports personalized medicine, as was the intent of Congress at the time of the Institute’s formation.

Specifically, research conducted in the category, “Comparative Assessment of Options for Prevention, Diagnosis, and Treatment,” will compare treatments, but at what level?  Researchers now know more about individual response to some drugs based on biomarker information. For example, when comparing a red pill to a blue pill, it is imperative that biomarker information be included in those examinations, especially when biomarker information is in the label for the drug. A list of drugs with biomarker information in the label can be found on pages 20-25 of The Case for Personalized Medicine, 3rd edition. But, without clearly defining the details of PCORI’s research, we really can’t know whether or how the Institute will consider biomarker information in its priorities and research agenda.

The research priority “Improving Healthcare System can support patient-centered care through innovations, but how? PMC suggests that the research be predictive, preventative, personalized and participatory. By emphasizing the “4Ps,” health system evaluation can support personalized medicine. However, without getting more specific about research priorities, we don’t know whether PCORI will include research that addresses these aspects of care delivery.

To support personalized medicine, the “Communication and Dissemination Research” priority should require researchers to answer one question: “Why does this treatment work and for whom?” (or, more frequently, “How likely is this treatment to work for me, and what are the potential trade-offs?”).  It is not enough, in the PMC’s opinion, to say that one therapy works for most people. PMC suggests that the research should explain why a therapy works (or is more likely to work) and for whom. For example, in research comparing red and blue pills, the communication and dissemination of the research results should stress that although the red pill works for most, the blue pill is particularly effective for people with a specific biomarker.

We are at a pivotal stage in the design of an entity that is tasked with assisting patients, clinicians, purchasers and policymakers in making informed, evidenced-based health decisions. PCORI could foster research that answers the important questions about what interventions work, and for whom. Or, PCORI could be another investigator-driven research institute that allows funded researchers to pursue questions of great scientific import, regardless of their practicality. Such a mission would be redundant with many others, most notably the National Institutes of Health, would be less likely to result in a cohesive national research program, and less likely to ensure personalized medicine is appropriately integrated.

It is my hope that PCORI supports the science that will drive personalized medicine forward, and will engage stakeholders in driving personalized medicine forward, by proposing specific research priorities and research questions to get answers to the questions that science, medicine and, ultimately, patients demand.


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