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New Approaches for Practical Challenges in Personalized Medicine

January 27, 2014

When I received the invitation to speak at the Personalized Medicine World Conference (PWMC), I was excited. When I saw the program and faculty, I was honored. When I received my “assignment,” I was intimidated. To share the podium with many of the luminaries in the field of genomics — even to hear them speak — is tremendously exciting. In contrast, how can they really be interested in hearing me speak on the real-world challenges providers and payers face in bringing genomics advances from bench to bedside more quickly?  I cannot answer this question. Although based solely on the number of conferences to which I am invited on this topic, someone must care. They should. Making the science “work” is only the first step in moving from bench to bedside.

As I was preparing my talk, I reflected on the story Sid Mukherjee, M.D., Ph.D., tells in his fabulous book, The Emperor of All Maladies: A Biography of Cancer. I’m not usually a big non-fiction fan, but if you are involved in cancer care in any way, this is a must read. Perhaps the book resonated because my professional career spanned many of the seminal events described in the book. That, and I share Dr. Mukherjee’s general perspective on the challenges of making meaningful advances in cancer treatment. The bottom line: cancer is tough. Just when we think we’ve made some great progress, it teaches us humility. Still, it is impossible not to be excited and enthusiastic about how far we have come in our understanding of the genetics of cancer. I really think we are experiencing a sea change. We must begin to think about navigating these changing currents in the world of health care reform in order to take best advantage of that progress.

So what are the practical challenges? There are many. And many share the underlying theme that the old paradigms do not work so well. Traditional methods of quality control in test performance, i.e. analytical validity, are a big problem. Proficiency testing and standards do not currently exist. To be honest, most doctors do not think about them when ordering a test. The billing and coding of molecular tests have been undergoing dramatic evolution but is still not suited to multi-analyte assays. Perhaps the biggest challenges lie in the area of clinical utility, which impacts providers, payers and regulatory agencies. Patients are impacted in a huge way. Most people have an idea of where we need to go, but we have a shortage of ideas about how to get there. Finally, all of this occurs in the setting of unsustainable growth in health care spending, and the near uniform agreement that we need to spend our money in a more intelligent, impactful way.

We have a lot of work to do together. It is important to remember we are all on the same side. We have a chance to make a huge difference, but we need to do it right.

What do you think our greatest challenges and opportunities are today? I look forward to the discussion in California.

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

The Future of Sequencing in Silicon Valley

January 9, 2014

Those working in (or reporting on) the fascinating fields of genomics and personalized medicine will have no shortage of conferences to attend in early 2014, including the increasingly important JP Morgan Healthcare Conference in San Francisco; the annual Advances in Genome Biology and Technology (AGBT) meeting on Florida’s Marco Island in February; and Eric Topol’s superb gathering next March, The Future of Genomic Medicine, held on the Scripps campus overlooking the Pacific Ocean.

The meeting I’m most looking forward to, however, is the 6th Annual Personalized Medicine World Conference (PMWC), to be held on January 27th and 28th in Silicon Valley. This event has flourished in recent years, offering a magnificent opportunity to meet and hear from world-renowned experts at the forefront of genomics, molecular diagnostics and personalized medicine.

The overarching theme of PMWC 2014 is: The Arrival of Actionable Personalized Medicine: The Age of Guided Disease Management. The opening session includes discussions featuring personalized medicine luminaries including Brook Byers, Randy Scott, Lee Hood, and NHGRI director Eric Green. I expect vigorous debate of pressing issues including the impact of the Supreme Court’s gene patent decision, the development of targeted therapies in cancer and neurological disorders, as well as regulatory and reimbursement trends.

PMWC 2014 will also showcase the remarkable pace of implementation of next-generation sequencing (NGS) in a clinical context, as the cost of a full genome sequence has plunged to just a few thousand dollars. Diagnostic companies and medical centers are now routinely offering comprehensive genome analysis, as evidenced by the recent report from Christine Eng and colleagues at Baylor College of Medicine in the New England Journal of Medicine on the first 250 patients studied using whole-exome sequencing.

On the eve of PMWC 2014, the conference organizers are hosting a special event to honor Jay Flatley, CEO of Illumina. Since the acquisition of the British biotech company Solexa in 2007, Illumina has been a dominant leader in NGS technology.

But Illumina and the rest of the NGS field are bracing for another seismic event as exciting new technologies, led by nanopore sequencing, are poised to emerge. Just a few weeks ago, I was privileged to attend a live demo of Oxford Nanopore’s new MinION sequencer, as portable as a smartphone, along with a couple of dozen sequencing experts. Judging from the enthused reaction of the assembled guests, I would anticipate a commercial launch sometime in 2014.

So there will be plenty to talk about when, on Day 2 of PMWC 2014, I’ll be moderating a special panel discussion – Killer apps, genome interpretation and the future of NGS – featuring five outstanding authorities in DNA sequencing. The panelists include Stanford University’s Steve Quake, the co-founder of Fluidigm and Helicos; Michael Hunkapiller, chief executive of Pacific Biosciences (and formerly of Applied Biosystems); Cliff Reid, the founding CEO of Complete Genomics, now part of BGI; Stefan Roever, CEO of nanopore sequencing start-up Genia Technologies; and Maneesh Jain, who handles business development at Ion Torrent.

One of the panelists suggested – not entirely tongue-in-cheek — that we subtitle the session: Will anyone succeed in knocking Illumina off its perch? I suggest you book a place at PMWC 2014 and find out!

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 Look for additional posts from speakers and participants prior to the 6th Annual Personalized Medicine World Conference on January 27-28, 2014. 

The Personalized Medicine Revolution Will Require Revolutionary Changes In How We Care For Cancer Patients

December 18, 2013

In the following post, Dr. Leonard Lichtenfeld, Deputy Chief Medical Officer for the American Cancer Society, shares his thoughts on how to improve the cancer patient care delivery process and reflects on conversations from a roundtable held by the Turning the Tide Against Cancer initiative.

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I recently attended a meeting in Washington that got me to thinking about the fact that as we revolutionize cancer research and treatment, we are also going to have to revolutionize cancer care. And that may prove to be an even more daunting task than finding new treatments for the disease itself.

The meeting was sponsored by a collaboration called “Turning The Tide Against Cancer“. The organizers brought together experts from a variety of disciplines ranging from insurance companies and economists to advocacy groups and highly regarded cancer specialists to discuss policy solutions to support innovation in cancer research and care. Walking in, I anticipated this was going to be another one of those sessions where we talked about funding for research, bringing research into clinical trials, and having patients get access to new drugs. But I was wrong. The discussions quickly steered into a different direction: what do we need to do to make the cancer care system work for patients?

Of course there were the continuing themes of “big data” and the impact of genomics on drug development and patient care, but a surprising amount of the discussion centered around new payment models, quality of care, and fundamental redesign of medical care to become more patient centric. And although we talked a lot about data gathering and analysis, what stuck with me was the redesign piece. I thought the discussion around redesign would focus on personalized medicine, but we spent a lot of time on changing the fundamental structure of cancer care and payment.

How are those two linked? Did we miss our focus?

The answer? If we don’t change the way the system is working, we won’t realize the promise of personalized medicine. Seems pretty simple and straight forward until you start thinking about the implications.

Our science is moving forward at a rapid pace, in significant ways. We are learning about the human/cancer genome, we have many new drugs in the pipeline that are targeted to the abnormal genetic signatures of cancer cells, we have research reports on a regular basis about a new finding that may predict who is at higher risk of developing certain cancers, or new tests to predict whether someone has an aggressive or indolent cancer. But if we don’t rethink the nuts and bolts of delivering the care, we won’t be able to get these remarkable tools and discoveries into the hands of patients and doctors. That in turn means we won’t be able to offer patients the benefits of the phenomenal advances we have made in cancer research and drug development. Heck, we won’t even have the tools to help develop the drugs or learn who the patients are who may benefit from the drugs or how patients respond to the drugs.

All of this means we have to figure out how transform the system. We need to be able to capture real data from real people. We need to have computer systems that talk to one another. We need to have a payment system that rewards quality-based and innovative patient care.

The list goes on, but you get the point. Being “patient centered”–which is a bit of a buzzword these days–is a call to action. The problem is that the barriers to change are substantial, and many more people and institutions pay lip service to the concept of patient centered cancer care than work to make their care truly patient centric.

Make no mistake: this is a gargantuan task. There are formidable obstacles to overcome which will favor keeping the status quo in place. Progress will be slow and difficult, but we have to keep remembering that we really haven’t tackled the issue of assuring every cancer patient and their loved ones that they got the right treatment for their disease at the appropriate cost and at the appropriate time. Fragmentation, duplication, and lack of information and access are no longer acceptable.

So it’s no longer just about the science and the research, as important as they may be. It’s not just surgery, chemotherapy, and radiation therapy. It’s about the care delivery process and the quality of that care. Reconstructing from the ground up. Thinking about new ways to help patients. It’s about nutrition, psychological and spiritual support, and financial guidance. It’s about making certain that people who live in rural areas can get care that is up to date and accessible through care collaborations designed to serve their needs and their expectations.

Is it too much to ask that patients, their families, and caregivers have genuine assurance that they are getting the best care available? Is it too much to ask that systems be in place to assure quality care? Is it too much to ask that we have computers that actually talk to each other? Is it too much to ask that in the most difficult moment of one’s life, they have an assurance that what was done for them was appropriate?

Don’t expect easy or quick answers to this situation. It took us decades of fundamental research to get us to the place we are today with the human genome. It is probably going to take a similar amount of time to change cancer practice. And I suspect changing practice is going to be even tougher to figure out than the gene thing.

But if we don’t do it, the power of what we will have to offer patients simply won’t be there for everyone. And that is something none of us should accept.

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A version of this blog originally appeared in Dr. Len’s Cancer Blog at the American Cancer Society.

Advancing Personalized Medicine through Research, Collaboration, and Innovation

November 21, 2013

The healthcare community is at an important juncture; we must work together to ensure that policies and regulations align to help us meet the unique challenges and full potential of personalized medicine. Science and technology drive what we can achieve, but they also evolve over time. We, too, must evolve and adapt to this rapidly changing environment.

Indeed, as I told participants at the 9th Annual Harvard Personalized Medicine Conference , the continuing development of personalized medicines holds promise to define our age and change everything we do. These treatments will affect how we approach once seemingly intractable medical challenges, how we research and develop new medicines, and how patients interact with physicians and our healthcare delivery system.

The biopharmaceutical industry is strongly committed to researching and developing personalized medicines.  A Tuft’s University survey found that biopharmaceutical companies increased their investment in personalized medicines by 75 percent between 2005 and 2010. In fact, between 12 and 50 percent of all compounds currently being researched are potential personalized medicines, which are targeting a growing range of diseases that may be identifiable through a genetic test or biomarker – from depression to obesity to many forms of cancer. Through these advances, we are shifting from reactive to proactive medicine, possibly enabling us to identify a patient’s risk before symptoms appear, and to transform our approaches to diagnosis and treatment.

But with these hopes, there are hurdles.  We all know that we need forward-looking policies that foster innovation and a regulatory system that is in line with advances in science. But we also need strong collaboration among biopharmaceutical companies, academics, patients, hospitals, government labs and others to pave the way for the science and its application.

Achieving the full power and potential of personalized medicine is an urgent priority. But it is an attainable goal only if we work together. On behalf of our patients, I urge everyone in our healthcare community to continue working across sectors and disciplines to advance personalized medicine.

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A version of this blog originally appeared in The Catalyst posted at the PhRMA website.

Strategies for Accelerating Progress in Personalized Medicine

November 5, 2013

Last year around this time, I wrote a blog aimed toward skeptics of personalized medicine, citing the progress we have made in lung cancer as just one example of how well this approach works for treating diseases. As I have reflected upon the advances we have made in areas like cancer and diabetes and how personalized medicine now seems to be the cornerstone for drug discovery and development for many pharmaceutical companies, it has become clear to me that there are still a number of areas for continued investment and focus.

What can we do to continue to grow the field of personalized medicine, find new and exciting therapies, and maintain the momentum we have seen over the past few years?

I believe that developing a personalized medicine strategy is the answer, but this is far from simple.

It begins by first appreciating that one needs to study the diversity inherent in diseases. When a new medicine is introduced in human clinical trials, for example, it is important to describe and understand the diversity of human responses to the medicine and to determine ways to identify those who will benefit the most from the medicine.

While we would ideally like to have such a clear scientific understanding of a disease that we can predict in advance who the likely benefiting patients are (which can be done in some instances), in most cases the personalization of a medicine comes from the observations made in clinical studies of diverse populations.

In contrast to basic laboratory research, where experimental designs seek to minimize variation, the “noise” in clinical trials can actually provide us with important clues to help form the basis of a personalized medicine. Well-designed studies, therefore, should study both the variation of the response as well as the scientific basis for that variation. When done well this can uncover biologic marker that become diagnostic agents for patient selection.

In addition to designing more clinical trials with these two aspects included, I would propose that those of us working in the field of personalized medicine should continue to support efforts that:

  • Fund basic research on the biologic basis of disease. Personalized medicine is based on fundamental scientific discoveries. Without these, there will be no improvement in our understanding of disease.
  • Fund research in diagnostic testing. Companion diagnostics can be useful tools to help healthcare providers and their patients make more informed treatment decisions.
  • Educate physicians and other healthcare providers about personalized medicine. Patients in specialized populations that may respond to a good therapy for their subgroup need to have access to the appropriate tests. This means making sure that those who can order the tests and use them as a part of standard care are aware that these tests and potential therapies exist.
  • Reimburse for diagnostic testing. Personalized medicine drugs will not have any benefit if they are not used and a healthcare provider won’t know to use a potentially life-saving drug unless the correct diagnostic tests have been performed.

Together, we can maintain and even accelerate the progress we have already made in delivering the right drug to the right patient at the right dose, every time.

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Join Dr. Stephen Eck on November 6, 2013 at the 9th Annual Personalized Medicine Conference hosted by Partners HealthCare Center for Personalized Genetic Medicine, Harvard Medical School, and Harvard Business School. 

View Dr. Eck’s video interview “Personalized Medicine: How Progress Happens” online at the Personalized Medicine Coalition.

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.

Developing Policies to Advance Personalized Medicine

August 7, 2013

In my previous post, I highlighted a Hill briefing hosted by the Personalized Medicine Coalition (PMC) and the great examples it provided of the impact of personalized medicine on patients. I was reminded why we must advocate for policies that support continued progress against disease and fulfill the promise of personalized medicine.

PMC provided an excellent starting point at the briefing, releasing a set of policy principles that offer a road map for fostering personalized medicine in a deficit-reduction environment.

As the panelists at the briefing emphasized, policy solutions must start with the patient – putting the patient at the center of decision-making, and supporting a shift in care delivery towards patient-centeredness and patient engagement.

Video highlights from the briefing panelists underscore why policy solutions like those advanced by PMC are so important – many patients continue to face significant unmet medical needs that can be effectively, efficiently met through novel targeted therapies; policy must provide adequate incentives for continued progress, recognizing the ways individual advances build on each other and understanding of an intervention’s optimal role and value evolves over time; and research and evaluation must adapt to rapid changes in clinical practice, the science of personalized medicine, and an increasingly robust “learning healthcare system.”

Below are some key, related statements from PMC’s policy principles:

“PMC believes that a deliberate and comprehensive shift away from health care based on population averages and towards patient-centered care is central to improving health care outcomes and addressing a patient’s perception of value.”

“A health system that focuses on care that is predictive, preemptive, and preventive has the potential to revolutionize health care by allowing clinicians to individualize therapy for patients through the early diagnosis of disease and risk assessment in order to optimize clinical outcomes and to better manage patients before disease symptoms appear.”

“Reform assessment tools (in particular, comparative effectiveness research and health technology assessment) to better align with emerging science and patient-centered health care.”

Coverage or reimbursement policy based on “one-size-fits-all” definitions of comparative clinical or cost effectiveness fail to recognize differences in patient needs and preferences and is misaligned with the progress in personalized medicine. This type of policy has resulted in significant barriers to access to the treatment options that are best for the individual and chills medical progress.

New payment models, such as accountable care organizations (ACOs) and bundled payments, which promote coordination and integrated care, hold potential to shift incentives to high-quality, high-value care for patients. However, if improperly designed, such models will set payment based on current standards of care and discourage advances in medical technology and medical practice.

PMC believes that there must be a continual learning system to aggregate, analyze, and apply evidence-based knowledge to patient care. PMC believes that health informatics and electronic health record (EHR) systems must be used to promote continual learning systems that will help improve patient care, reduce costs, and accelerate the process of drug development.

A version of this blog originally appeared in The Catalyst posted at the PhRMA website.

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.

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

May 13, 2013

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.

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.


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