Balancing the Need for Guidance, Communications, and Education to Support Innovation in Personalized Medicine Diagnostics

Recently, I had the opportunity to moderate a thought-provoking panel at the PMC/BIO Solutions Summit. The summit brought together key stakeholders to discuss solutions to barriers in the development of innovative personalized medicine diagnostics. A big question for those developing potentially game-changing technologies in an increasingly cost conscious environment is the need for “Evidentiary Standards and Data Requirements for Payer Coverage.”

Determining the data requirements for coverage is becoming an increasingly frustrating issue for diagnostics manufacturers, which face rising demands for evidence but continued lack of clarity about payer standards for evidence-based decision-making, leading many to ask the question, “Why can’t payers just tell us what their standards are?” Complicating the picture is that diagnostics can come to market via different pathways with different levels of supporting evidence (e.g., companion diagnostics reviewed by FDA for clinical validity with the companion therapeutic and tests developed, validated, and introduced to the market by laboratories).

The panelists – who represented leaders from industry, non-profit advocacy, and government working to create solutions for some of these market access barriers – noted a couple of issues at play. One is that having a payer “pick list” or hard criteria for coverage removes the flexibility that is so often needed in these gray-area coverage decisions. The second is that given the volume of products they are evaluating, most payers don’t have the bandwidth to be experts in the nuances of the trial design for every technology. Third is that across all stakeholders, there is a wide range of knowledge on innovative products and personalized medicine and that basic education for the majority of stakeholders to better understand these products is lacking.

Several lessons and next steps came out of this discussion. 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.

At the end of the day, it may be incumbent on the molecular diagnostic community to shape the paradigm for evidence requirements so that payers can act as enablers, rather than watchdogs.

Putting the Pieces Together for Personalized Medicine

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.

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?

Randy Scott: Bringing Metcalfe’s Law to Genomic Medicine

Each year, the Personalized Medicine Coalition recognizes an individual whose contributions in science, business, and/or policy have helped advance the frontiers of personalized medicine. This year, the Leadership in Personalized Medicine Award was presented to Randy Scott, Ph.D., during the Harvard Personalized Medicine Conference on November 28, 2012 in Boston, Massachusetts. 

Receiving the 2012 Leadership in Personalized Medicine Award from the Personalized Medicine Coalition (PMC), Randy Scott reflected on his success as the founder and former CEO of Genomic Health, but also looked ahead to new opportunities with his latest venture, InVitae Corporation.

Scott received the award this week at the 8th Annual Personalized Medicine Conference at Harvard Medical School. “Randy has transformed our understanding of how medicine can be practiced by creating one of the most successful personalized medicine companies to date,” stated Brook Byers, a partner with Kleiner Perkins Caufield & Byers and a previous honoree. Past winners of the PMC Award include Janet Woodcock (FDA), Elizabeth Nabel (NIH), Ralph Synderman (Chancellor Emeritus, Duke University), and Leroy Hood (Institute for Systems Biology).

After a successful stint at Incyte, Scott founded Genomic Health in 2000 and led the firm for nine years, overseeing the development of the Oncotype Dx gene expression test for breast cancer. He modestly shared the credit with numerous colleagues. “My contribution was I probably did a good job of hiring a lot of people at Genomic Health who are way smarter than I was,” he said, naming in particular co-founder Joffre Baker, CMO Steven Shak, and CEO Kim Popovits.

As a graduate student in the early 1980s, Scott said he had been excited about biotech but worried he was too late. “All the exciting genes had been cloned! TPA, Factor VIII, human growth hormone, insulin,” he recalled thinking. Today, Scott said, “we’re on the precipice of incredible accelerating change in this field… Everything we’ve experienced to date pales in comparison to what we’re going to experience in the next 5-10 years.”

But he also shed some personal insight into the launch of his latest venture, InVitae Corporation. He said he is “unabashedly excited” about the future of personalized medicine. “Personalized medicine is really when disease happens to you—your friends or your family. Suddenly it’s no longer just an industry we’re working in but something so personal, so intense, and so emotional. We should never forget that.”

The Network Effect

Scott said reading Intel founder Andy Grove’s book Only the Paranoid Survive in the mid-90s, during his tenure at Incyte racing to identify human genes, was highly influential. In the book, Grove discussed the impact of Moore’s Law on the revolution in computing; Scott saw parallels with the biotech industry. “The way we were sequencing DNA [at the time] was so embarrassingly simple,” he said. Just as computing costs were plummeting, Scott reasoned it was inevitable that sequencing costs would also fall.

Perhaps more importantly was the concept of “the network effect.” Just as Metcalfe’s Law—the community value of a network is proportional to the square of the number of its users—drove change in the computing world, so too will it drive the future of biotechnology.

“Having a really cheap genome sequenced is really not very useful. We still see articles in The New York Times, ten years after the genome project, [saying] ‘so what?’ At some level, they’re horribly wrong, and at some level, they’re horribly right. We’ve not yet seen the network effect or the full implication of Moore’s Law.”

Scott said the community is still “1-2 years away from the inflection point” where the cost of sequencing reaches the point that will trigger “massive consumer demand.” The value of genome sequencing will be most strongly felt in the network effect. “How we connect that genomic information across millions and millions of individuals… Somebody can be sitting at a computer, link into the network, and find how a mutation and how it correlates with their patient and a patient somewhere else in the world.”

Scott said he was also a believer in what he called the “Law of Finite Genomes.” The human genome is like a complex finite puzzle with about 150,000 pieces (20,000 genes and 100,000 non-coding RNAs). “All common diseases are really rare diseases,” Scott said, with cancer a prime example. “Medicine goes from an infinite game to a finite game,” he said. By comparing lots of genomic information, we can begin to rule things out.

Patients, Patients, Patients 

Scott was inspired to launch Genomic Health when a close friend was diagnosed with colon cancer in 1999. For the first time, Scott was personally struck by the chasm between science/technology and medicine. “We’ve got to bridge the gap—bring the science into clinical practice,” he said.

The focus at Genomic Health, Scott said, was “patients, patients, patients.”

“I’m not sure we had a model other than this maniacal focus on patients that wouldn’t be denied,” he said. If we could really do the science right, the science would sell.” Genomic Health spent an enormous effort on clinical studies.

“Clinical data wins over physicians, and it is physicians that win over the payors,” Scott said. “The onus is on us as an industry to build the value proposition [for payors]… so physicians have to adopt those products. If physicians adopt, they will drive payers to cover.”

Scott left Genomic Health this year to launch InVitae, spurred by the impact of rare genetic diseases affecting members of his family.

In 2000, Scott’s nephew had a daughter with galactosemia. Fortunately, the disorder was diagnosed within 48 hours of birth, and her diet could be changed, otherwise there could have been “a dramatically different outcome.” In 2005, an adopted nephew collapsed on a tennis court and died from hypertrophic cardiomyopathy. Advanced screening could have saved his life, but nobody knew any family history of cardiac disease, he said.

Finally, one of his wife’s relatives had a young son who developed serious seizures at age 2 years. The infant is developmentally impaired and severely autistic. Earlier this year, Scott revealed that exome sequencing of the child and his parents revealed a single de novo point mutation as the putative cause of the disorder. This is unlikely to provide any tangible medical benefit, but “it gives a clue into potential causes of these disorders,” he said.

Ridiculous Goal 

Scott said his goal in launching InVitae was to bring the power of genetics into the real world of clinical practice. “We have a ridiculous goal,” he said. “We want to aggregate all of the world’s genetic tests into a single assay—for less than the cost of a single assay today!”

In other words, InVitae plans to collapse all Mendelian inherited traits into a single assay that can be performed “reproducibly, at high quality and at reasonable cost for the medical system. So instead of going into these diagnostic odysseys… every parent thinking about conceiving a child can know exactly what their carrier status is and what disease risks lie in their family.”

The initial assay will essentially be an elaborate gene panel, but Scott’s plan is eventually that this will lead into whole-genome sequencing (WGS). Scott believes that “within 10-20 years, everyone in any developed health care system will be able to be provided with a low-cost [WGS] analysis at birth… We’ll be talking about managing your genome over the course of your lifetime.”

As for the question of how to deal with the plethora of data, “that’s Metcalfe’s Law, the network effect,” said Scott. “Much of the data won’t be of value to the patient or physician ordering the test. But collectively, they will be massively valuable to the research community.”

We’re big fans of “Free the Data!” said Scott. The universe of clinical genetic data “won’t be a database held by one company or one academic institution, but you’ll see a massive movement over the course of the next decade to make data broadly available within the research community.” This will create a huge disruption in medicine, Scott predicted, a shift from phenotypically driven medicine to more of a genotype foundation as sequencing costs fall and the network builds.

“Everything will drive off the genotype and it will move very fast,” he said. “This is a given. To me, this is the investment thesis. This will be the place to be, the chance to help people suffering from rare diseases. At the end of the day, every disease is rare.”

InVitae is building a strong management team. The company recently merged with Locus Development, a start-up co-founded by Sean George and Michele Cargill, founding scientists at Navigenics. Steve Lincoln and Jill Hagenkord, both formerly with Complete Genomics, also joined the cause this year, as did Reece Hart, former manager of research computing and informatics at Genentech.

– This article was first published at the Bio·IT World website on November 30, 2012.

From The Catalyst (blog) – A Conversation with Marcia Kean, Chairman, Feinstein Kean Healthcare

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.

Conversation with Marcia Kean, Chairman, Feinstein Kean Healthcare

Q:  In the course of 30+ interviews of national thought leaders that you conducted to develop a Discussion Paper for the Turn the Tide conference, what themes emerged most strongly? 

Marcia Kean:  I was very surprised at how much consensus there was from such a disparate group of stakeholders from academe, patient advocacy, industry, providers and payers.  There was strong agreement, for example, that as daunting as cancer is, innovation is going to be the best way to overcome both the clinical and economic burden of cancer in coming years, and that above all, innovation has to be protected.

Five major themes recurred.  The first was around the need for an ongoing, community-wide commitment to overcome the issues we face as a society in oncology.  I don’t mean that everyone had the same perspective about those issues – remember, this was a very diverse set of individuals – but rather, there was agreement that we have the opportunity at this moment to do things differently to accelerate research and improve care, and that in order to reward innovation, it’s important for stakeholders to identify overlapping interests rather than remaining rigid in their safe and separate silos.  Creating a joint, defined vision of what constitutes value will help to outline a pathway for development.

Second, while everyone recognizes that we’ve made progress toward patient-centered cancer care, much more needs to be done. We need a better system for measuring value that puts patients’ needs and preferences first, and keeps up with the rapidly evolving scientific and clinical environment. As personalized medicine becomes more central, of course, this kind of dynamic measurement will only become more difficult.

Third, there’s huge excitement about the potentially positive impact of digital technologies to accomplish the dramatic productivity gains in cancer research and care that they’ve done for other fields.  Developing effective tools for shared decision-making and clinical decision support is seen as a major opportunity in that regard.

Fourth, there’s a desire to move to “next-generation” tools for assessing value through Health Technology Assessment, as well as a fervent desire to develop next-generation Comparative Effectiveness Research that aligns with personalized medicine and gives a fuller picture of real-world value. We heard that current approaches aren’t centered enough on patient value, and don’t do an adequate of job keeping pace with how the use and value of tests and treatments evolves over time – they are just too static in a highly dynamic world.

Finally, there’s a strong drumbeat about the shift to models of “continuous learning” that give a more dynamic, patient-centered picture of value, so that every clinical encounter informs our overall knowledge base and research advances are moved rapidly into clinical use.  That virtuous circle is seen as the light on the horizon for all of us.

Q:  Your firm has been very active in efforts to implement health information technology and develop a learning health care system. How will these concepts accelerate progress in cancer care?

Marcia Kean:  In some ways, the field of biomedicine has been the last to benefit from the digital revolution.  For many cultural – and technical – reasons, we just haven’t been able to move data rapidly from lab bench to doctor’s office to hospital to patient, and so on.  But there is a growing cadre of researchers and clinicians and patients who are demanding the kind of ‘data liquidity’ in science and health care that they see in every other sector of their lives, and they are gaining traction.  Once we have seamless data exchange, based on the use of standards in Health Information Technology and bioinformatics tools, we’ll have a solid foundation for a learning health care system. In that picture, everyone wins:  innovators can accelerate and improve research; patients and doctors can get the information they need to make wise decisions; and the efficiency of the process can drive down costs.

However, we need to remember that even a digitally-enabled system doesn’t happen in a vacuum — it has to unfold in a community where every sector is interconnected and working towards a common, patient-centered vision, because all these challenges and solutions are inter-related in a very complex and dynamic ecosystem.

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.

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

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

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: Together Moving Comparative Effectiveness Research and Personalized Medicine Forward

Today, I had the privilege of addressing the Patient-Centered Outcomes Research Institute (PCORI) Board of Governors on behalf of the Personalized Medicine Coalition (PMC).  As noted in my previous blog on its member selection, the PCORI Board of Governors was established by the Patient Protection and Affordable Care Act and is tasked with assisting patients, clinicians, purchasers, and policymakers in making informed, evidence-based, health decisions. 

As a science-driven organization, PMC supports health decisions made on sound science.  When working with legislative drafters and the community to develop the legislation creating PCORI, we recognized the need to ensure that the research supported by this group must be personalized and continually updated.  The resulting law creating PCORI aligned the principles of personalized medicine with those of comparative effectiveness research.  It underscores the importance of eliminating an “average effects” approach to CER, which obscures subgroup differences and skews treatment decision-making away from individualized care.

To make sure this opportunity is realized, we requested that the Board designate an ad hoc committee on personalized medicine to work with both the Board and the Methodology Committee.  We believe that such an ad hoc committee will help the Board and methodology committee comply with the Congressional intent that the research overseen and conducted by PCORI be personalized when appropriate.  High levels of communication and collaboration have been essential to the advancement of personalized medicine, and the same will hold true for advancing CER that is aligned with personalized medicine.

Achieving alignment between CER and personalized medicine holds substantial opportunities, but we realize it will be challenging. These challenges cut across a range of issues, from study designs, to operating procedures, to research priorities.  An ad hoc committee will provide a venue for bringing a diversity of expertise and perspectives together to focus on this common objective.  Such a group will help in coordinating research across a range of public and private sector activities and ensure that the most relevant evidence gaps and research questions are identified related to personalized medicine, and will help with matching study designs to questions.

The science of personalized medicine is fast moving and sometimes highly specialized. Legislation and regulation takes time to catch up. The personalized medicine community is committed to seeing that PCORI’s research keeps pace with scientific advancement and welcomes the opportunity to achieve this end.

We are at a crossroads of research, regulation and legislation. It is an exciting time!

CER and Personalized Medicine: More Work to Be Done

I just got back from a meeting of the Board of Governors of the Patient-Centered Outcomes Research Institute in New York City. PCORI, as you probably know, is the independent Institute established by the health reform law to conduct comparative clinical effectiveness research. Regarding CER and personalized medicine, I’ll say now what I said late in late 2009: in the midst of the debate over health care reform — it remains to be seen whether CER will align with personalized medicine. CER that aligns with personalized medicine should be  patient-centered, because it will be structured to recognize and respect patient differences. 

We’ve made important progress toward that goal, with CER statute for the first time referencing genetics and individual patient difference.  I am hopeful we’ll  eventually get there, but we’re not there yet. Personalized medicine was, as far as I saw, mentioned only once during the PCORI Board meeting. NIH Director Francis Collins raised it during a discussion of the report from the PCORI Methodology Committee. Commenting on the Committee’s charter and the “perceived tension between CER and personalized medicine,” he said he “would hope that the Methodology Committee would take that on,” and suggested emphasizing alignment with personalized medicine and patient preferences in the document. I applaud Dr. Collins for raising this point, but we will need more voices joining him to make sure the Board and Methodology Committee to integrate CER and PM. It seems to reinforce some concerns expressed earlier by PMC about lack of an expert in genomics or personalized medicine on the Methodology Committee. The idea proposed by PMC of creating an advisory panel on personalized medicine and innovation could be one step to help ensure alignment between CER and personalized medicine.
   
One of the big news items from the PCORI Board meeting was the appointment of Dr. Joe Selby as the PCORI Executive Director. I was heartened by Dr. Selby’s comments at the meeting, in which he stressed the importance of hearing from, and focusing on, patients. It will be equally important for Dr. Selby and the additional staff he hires to understand that part of the Institute’s charge from Congress is to account for genomics and subpopulation differences. Failure to do so will prevent us from realizing the potential for personalized medicine to improve health care quality and value. 

The next meeting of the PCORI Board of Governors is in Washington, D.C. on July 18 and 19th. I hope you’ll join me there in support of comparative clinical effectiveness research that is centered on patient needs and aligned with personalized medicine.