Survey Reveals Insights About Awareness, Understanding of Personalized Medicine, Part 1

August 22, 2014 by

Earlier this year, the Personalized Medicine Coalition (PMC) commissioned U.S. Public Opinion About Personalized Medicine, a nationally representative survey of 1,024 adults gauging consumer awareness, knowledge and attitudes about personalized medicine.

The key findings of the survey were positive. Although less than 4 in 10 Americans had heard of it prior to being surveyed, respondents are interested in learning more about personalized medicine and are supportive of the concept. For those who had heard of personalized medicine, their knowledge was shallow and did not associate the term with diagnostic testing or targeted treatments. This highlights the need for education to a willing public.

When the surveyed individuals were given a definition of personalized medicine, approximately two-thirds were receptive and expressed positive opinions about its prospects.

Respondents easily pointed out the major benefits of personalized medicine, noting that it could give them more knowledge to prevent or treat their illness, help them choose the most
effective treatments with their doctors, and lead to a decline in unnecessary treatments, side effects, invasive procedures and trial and error medicine.

Many of the individuals who were surveyed even raised questions, specifically regarding the efficacy, potential risks, cost, access and affordability. A majority agreed that insurance should cover personalized medicine if it is recommended by a doctor.

We’re at the beginning of the golden age of personalized medicine. Armed now with a clear picture of the public’s opinion, we have an opportunity to raise awareness and increase understanding of what personalized medicine is, and how it can transform approaches to healthcare delivery.

Stay tuned for part two of this blog post series as we hear from leading experts, and the researcher who conducted the survey, on what key benefits of personalized medicine they think the public needs to know about in order to embrace this approach to healthcare.

In the meantime, learn more about U.S. Public Opinion About Personalized Medicine and review the survey findings by viewing the slideshow below or visiting the PMC website.

FDA Outlines Personalized Medicine Policy with Publication of LDT Draft Guidance Document, Final Guidance on Companion Diagnostics

August 15, 2014 by

On July 31, FDA announced drastic changes to regulation for personalized medicine products and services when it coupled the release of the long-awaited final guidance document on the regulation of companion diagnostic devices with a proposed framework for regulating laboratory developed tests (LDTs), which was also long-awaited or long-feared, depending on your perspective.

The final guidance on In Vitro Companion Diagnostic Devices was welcomed by the personalized medicine community because in the document, FDA clarified the path for co-developed drug-diagnostic products, and finalized their assertion that new targeted therapeutics will not be kept from the market if the diagnostic kit is not ready at the same time. This enables promising new drugs to come to market while also allowing the laboratory community to fill testing needs in cases where an FDA-approved kit is not available for therapeutic selection, dosing and avoidance decisions.

However, many issues remain to be addressed.

To address concerns that FDA regulation will pose obstacles to an already challenged laboratory industry, there is a rather long transition phase — nine years — and an initial focus on high-risk. FDA defines high-risk LDTs as those with the same intended use as cleared or approved companion diagnostics, LDTs with the same intended use as an FDA-approved Class III medical device, and certain LDTs for determining the safety and efficacy of blood or blood products. This focus and transition period will allow clinical laboratories and FDA time to adjust. By focusing initial regulation on high-risk LDTs, FDA makes a strong argument for the framework, and slices off a rather small segment of the LDT market.

Many have argued that FDA does not have the bandwidth to regulate LDTs. FDA responded to this claim by reiterating enforcement discretion for the vast majority of LDTs and outlining a process for LDT regulation, which might be less onerous than traditional regulatory pathways for medical devices.

Although FDA made great efforts to address concerns about the Agency’s new regulatory enforcement, it did not address perceived conflicts between laboratory regulation under Clinical Laboratory Improvement Amendments (CLIA) and this new framework. Furthermore, FDA intends to use an expert advisory panel to provide recommendations to the Agency on LDTs risks and classification on certain categories of LDTs, as appropriate. I suspect that defining those categories will be contentious and, at times, difficult.

Investors have long argued that clarity is necessary in both regulation and reimbursement for continued advancement of personalized medicine. We now have clarity on FDA’s current thinking although many issues remain unresolved. The community has time to consider this framework and may soon have a chance to provide public comments. And finally, the pharmaceutical industry has the FDA’s assurance that targeted treatments will not be held up by co-development challenges.

Required Reading: July 2014

August 1, 2014 by

Great stories are published daily about the impact personalized medicine is having on individual patients, and the medical community as a whole, but it can be a challenge to stay on top of the news. With that in mind, we bring to you a monthly roundup of the three to five most thought-provoking articles we are reading, sharing and discussing with our colleagues.

This is the July 2014 installment of Required Reading.


Nobody is Average but What to Do About It? The Challenge of Individualized Disease Prevention Based on Genomics by Muin J. Khoury, CDC Genomics and Health Impact Blog

When it comes to individual health risks, there is no such thing as average, yet most health guidelines and recommendations are tailored to “average” individuals in the population. This blog post by Muin J. Khoury, director of the Office of Public Health Genomics at the Centers for Disease Control and Prevention highlights some of the challenges to actualizing the concept of individualized disease prevention and best utilizing each individual’s biological uniqueness.

The Emotional Side of Personalized Medicine by Ide Mills, Genome

For more than 30 years, Ide Mills worked as an oncology social worker, health educator, and communication strategist. Now as a woman living with advanced, ALK-positive non-small cell lung cancer, Mills tells Genome magazine about her experience transitioning from intravenous chemotherapy to a twice-daily pill regimen to help treat her disease. Her story details the challenges – and improvements – she experienced adjusting to the concept of oral cancer therapy and taking an active role in her healthcare.

23andMe Co-founder Anne Wojcicki’s Washington Charm Offensive by Ariana Eunjung Cha, The Washington Post

When she founded genetic company 23andMe more than six years ago, Anne Wojcicki’s ultimate goal was for people to be in control of their own healthcare. Wojcicki is still determined to change the way traditional healthcare works in the United States by shifting the focus to individuals instead of institutions. 23andMe is currently working with the U.S. Food and Drug Administration to get approval for its direct-to-consumer personal health reports that analyze an individual’s DNA.

As Sequencing Moves into Clinical Use, Insurers Balk by Julie Steenhuysen, Reuters 

A number of major insurers are beginning to address the increasing availability and usage of gene-sequencing tests by seeking proof that the results will lead to meaningful treatments among the estimated 2 million Americans with a serious, undiagnosed disease. Genetic experts have responded saying that gene-sequencing tests, such as exome sequencing, are bringing hope to families by more than doubling the chances they will get a diagnosis and saving them money by not spending it on multiple tests of a single gene. 

Developing New Tools to Support Regulatory Use of “Next Gen Sequencing” Data by Carolyn A. Wilson, Ph.D., FDAVoice blog

In last month’s Required Reading, we shared an article from The New York Times about next-generation sequencing (NGS). Learn more about the private cloud-based environment called the High-Performance Integrated Virtual Environment (HIVE) that the Center for Biologics Evaluation and Research supported the development of to help prepare the U.S. Food and Drug Administration to review and understand the interpretation and significance of data in regulatory submissions that include NGS. 

BREAKING NEWS: FDA Notifies Congress of its Intent to Publish Framework for Regulatory Oversight of Laboratory Developed Tests (LDTs)

July 31, 2014 by

Summers in D.C. are notoriously slow. FDA, however, has added excitement to this summer by informing Congress of its intent to publish a long-awaited framework for LDT regulation.

In its notice to Congress, FDA included what appears to be a draft of the document. After the mandatory 60-day Congressional review, the draft guidance document will be formally issued for public comment.

Within the draft framework, FDA proposes a risk-based, phased system of oversight. They recognize community concerns around access and do not intend to interrupt the marketing and sale of currently available tests. Furthermore, FDA expresses the intent to continue using enforcement discretion for forensic and organ transplantation uses, traditional LDTs, and LDTs for unmet needs.

The document outlines the history of LDT regulation, FDA’s policy of enforcement discretion, and how personalized medicine has caused FDA to reconsider that policy.

We will continue to provide updates on the development of framework for regulatory oversight of LDTs, with additional in-depth commentary next week on this issue and the related news of FDA’s final guidance on companion diagnostics.

For additional information on the current regulation of LDTs, please read PMC’s report “Pathways for Oversight of Diagnostics.”

Ensuring a Personalized Future through Diagnostics Policies

July 30, 2014 by

In a time of rapidly rising healthcare costs, personalized medicine offers a more efficient approach to prevention, diagnosis and treatment.

However, special considerations and thoughtful policies that acknowledge the emerging and varied technology (and challenging science) at play are needed in order to ensure access to personalized diagnostics and treatments, while also encouraging further investment and development of technologies. Just as personalized medicine is moving us away from a one-size-fits-all approach to healthcare, we must too move away from one-size-fits-all approaches to reimbursement.

The Personalized Medicine Coalition recently released “The Future of Coverage and Payment for Personalized Medicine Diagnostics.” The white paper takes a critical look at the policies of the Centers for Medicare and Medicaid Services, which in turn inform the decision making process for payers. Three specific challenges to the further implementation of personalized medicine diagnostics are identified:

  • Reimbursement levels that ensure access while also encouraging development
  • Inconsistent standards for evaluating genomic tests
  • A lack of incentives for genomic medicine

As we continue our journey in the post-ACA healthcare landscape, the need for objective, reliable and clearly defined standards for evaluation and reimbursement continue to grow. Uncertainty – especially in the form of payment – does not inspire investment or fuel growth through R&D.

Required Reading: June 2014

June 24, 2014 by

Great stories are published daily about the impact personalized medicine is having on individual patients, and the medical community as a whole, but it can be a challenge to stay on top of the news. With that in mind, we bring to you a monthly roundup of the three to five most thought-provoking articles we are reading, sharing and discussing with our colleagues.

This is the first installment of Required Reading.


Drugmakers Find Breakthroughs in Medicine Tailored to Individuals’ Genetic Makeups by Brady Dennis, The Washington Post

One-size-fits-all blockbuster drugs are giving way to treatments tailored to individuals’ genetic makeups. This article highlights the advantages of these targeted treatments, posing important questions that regulators, drug makers, insurers and patients alike need to consider as more specialized drugs come to market, including who will benefit and how to ensure patients have access to life-saving treatments.

In a First, Test of DNA Finds Root of Illness by Carl Zimmer, The New York Times

Diagnosis is a crucial step in medicine, but it can also be the most difficult. A new DNA sequencing technology, called unbiased next-generation sequencing, can identify a pathogen by testing DNA samples from a patient and then comparing fragments with genetic sequences stored in online databases. This technology has the potential to not only provide speedy diagnoses to critically ill patients, but could lead to more effective treatments for diseases that can be hard to identify.

The Perfect Drug by Erin Biba, Newsweek

This article highlights how continued innovation in our understanding of cancer and the emergence of companies providing personalized treatments helped change one patient’s life by turning his cancer diagnosis from a death sentence into a managed chronic illness.

Keeping the Patient at the Center of Cancer Innovation by Amy Abernethy, M.D., Ph.D. and Arif Kamal, M.D., The Hill’s Congress Blog

Hot on the heels of the 2014 American Society of Clinical Oncology annual meeting and Institute of Medicine’s National Cancer Policy Forum earlier this month, this op-ed focuses on the role of medical innovation and patient-centered approaches in the growing discussion on the cost and value of cancer research and care.

Making the Case for Personalized Medicine

June 18, 2014 by

As lawmakers, industry, consumer groups and the general public debate the future of health care, personalized medicine continues to focus attention-with good reason. In a time of unprecedented scientific breakthroughs and technological advancements, personalized health care has the capacity to detect the onset of disease at its earliest stages, preempt the progression of disease, and, at the same time, increase the efficiency of the health care system by improving quality, accessibility, and affordability.

With that in mind, I’m excited to announce the release of the fourth edition of Personalized Medicine Coalition’s signature document, The Case for Personalized Medicine, which notes the steady development of the field. Most notably, in 2006, there were 13 prominent examples of personalized drugs, treatments and diagnostics on the market. In 2011, there were 72, and today there are 113-a 57 percent increase in the last three years.

Along with this steady growth, we also examine opportunities for the continued development and adoption of personalized medicine as several factors come into play: the cost of genetic sequencing declines, the pharmaceutical industry increases its commitment to personalized treatment, and the public policy landscape evolves. While momentum is building, much remains to be done to keep up with the evolving developments in science and technology, according to the report. With all this progress comes a greater responsibility to actually help patients by advancing personalized medicine to make it standard practice in the near future.

According to The Case, personalized medicine can accomplish the following:

  • Shift the emphasis in medicine from reaction to prevention
  • Direct the selection of optimal therapy and reduce trial-and-error prescribing
  • Help avoid adverse drug reactions
  • Increase patient adherence to treatment
  • Improve quality of life
  • Reveal additional or alternative uses for medicines and drug candidates
  • Help control the overall cost of health care

But to advance personalized medicine, we need intelligent coverage and payment policies, clearer regulatory guidelines, and modernized professional education to prepare the next generation of doctors and other health care professionals for personalized medicine.

PMC is pleased to partner with BIO and release this report at the Personalized Medicine & Diagnostics Forum at this year’s meeting. The Forum takes place next Wednesday, June 25 at 9 a.m. PT in room 7AB and will explore many of the salient issues facing personalized medicine, as discussed in The Case. If you’re onsite at BIO this year, come check out the forum. We’d love to hear your thoughts on the current personalized medicine landscape and what’s to come.

The new edition of The Case for Personalized Medicine will be available online June 25 at 9 a.m. ET, but in the meantime, check out PMC’s other publications here.


This blog post originally appeared in BIOtechNow, the blog of the Biotechnology Industry Organization (BIO).

 

Prescribe by Price, But at What Cost to Patients?

June 16, 2014 by

In recent months, there has been increased discussion about the cost and value of cancer care. These conversations are important, but it’s crucial to make sure the patient’s unique perspective on value isn’t lost in the mix. We must also be sure we are considering each patient’s unique diagnosis, as well as the heterogeneity of their disease.

It is clear that targeted therapies play an important role in delivering personalized care to patients, and in particular, those with many forms of cancer. A recent article in Newsweek profiled a patient who, through a new tumor identification method, was able to receive a highly-targeted treatment for his sarcoma. As noted in the article, “the slow and steady march of innovation in our understanding of cancer and the emergence of companies providing personalized treatments have not only saved Rose’s life but allowed him to turn his cancer from a death sentence into a chronic illness that simply needs regular managing.”

These stories are becoming more common, yet as new targeted treatments work through the regulatory pathways, we are faced with ongoing questions: How do we ensure patients have access to these life-saving drugs and the diagnostics used to select them? And how do we ensure the system incentivizes research and development broadly, so more people benefit from personalized medicine?

Recently, the American Society of Clinical Oncology (ASCO) announced that it will be developing a scorecard, slated to come out this fall, to evaluate cancer drugs based on their cost and value, as well as their efficacy and side effects. This signifies a potential shift in the way that physicians make decisions about their patients’ health, encouraging them to consider the financial implications of interventions alongside patients’ needs. As this framework is developed, it will be important to ensure that the full value of innovative interventions is recognized.

Scientific advances have the potential to provide earlier and better diagnoses, more effective treatments and even possible cures for patients. Personalized approaches to care could provide a patient with more or better quality time to spend with their family or friends, and overall greater long-term value.

As Edward Abrahams, president of the Personalized Medicine Coalition told The Washington Post, these targeted treatments also bring a greater long-term value to the healthcare system as a whole by cutting down the frequency of unnecessary treatments and procedures, and avoiding unnecessary side effects.

Still our continued focus must be on providing high-value care and creating a system that improves patient outcomes and does not place the focus on prescribing drugs based on cost or financial incentives. The Personalized Medicine Coalition, along with the American Association of Cancer Research and Feinstein Kean Healthcare, continues to engage the cancer community to address these questions of cost and value through the Turning the Tide Against Cancer initiative.

Through ongoing dialogue and discussion as part of this initiative, two key themes continually resurface as components that should be considered when defining value: advancing approaches that are patient-centered, and developing new approaches to value assessment that align with and incentivize scientific progress and innovation.

On October 9, 2014, the Personalized Medicine Coalition, the American Association of Cancer Research and Feinstein Kean Healthcare will re-convene the cancer community in Washington, D.C., for a second Turning the Tide Against Cancer national conference to advance the conversation on the value and cost, while supporting a shift to patient-centered cancer research and care.

From The ASCO Meeting In Chicago: A Focus On Cost, Value, And Financial Toxicity Of Cancer Care

June 9, 2014 by

In the following post, Dr. Leonard Lichtenfeld, Deputy Chief Medical Officer for the American Cancer Society, shares his thoughts on the discussion at the 2014 American Society of Clinical Oncology (ASCO) annual meeting regarding the cost and value of cancer care and treatment and mentions an article recently published in Clinical Cancer Research that he co-authored with other members of the Turning the Tide Against Cancer initiative.


At the annual meeting of the American Society of Clinical Oncology (ASCO) here in Chicago, something vitally important is happening: there is an increasing recognition of something no one really wanted to talk about in polite company until now. It is the fact that the costs of many of the new treatments being developed are extraordinary.

The headlines about cost and value of cancer care greeted me when I walked into the McCormick Center in Chicago for the opening sessions of the meeting. This is the leading cancer meeting in the world, and what happens here makes news worldwide, significantly impacting the lives of patients with cancer wherever they may be.

Now there is an increasing recognition of the elephant in the room: the costs of these new treatments are extraordinary. No matter how one chooses to slice and dice the arguments, these drugs are expensive with costs per month of $8000 and upwards getting a lot of attention and increasing concerns, especially at this meeting.

For years, it was whispered in hallways, muttered about with reporters, but now it’s out in the open. The headlines say it, the lecturers say it, the attendees are talking about it. There is an increasing awareness surrounding the value, cost and toxicities–both medical and financial-of cancer treatment. Even as I write this I am sitting in a session devoted to the topic of how we make the choices so necessary for the welfare of our patients and how we should guide those choices.

Some say the costs of this brave new world of cancer care are justified by the advances they bring to the clinic and the lives of patients. The cost of the drug should reflect the value they provide to society says one company. Others say the costs are justified to allow continued research to advance new drugs. But increasingly, another voice is being heard: one that says the costs are unconscionable and not sustainable. Patients are going bankrupt and some of the drugs are quite toxic or don’t bring sufficient value to the patient (such as meaningfully extending life, or reducing side effects).

This argument and the facts behind it are not going to go away. And they shouldn’t. There are too many forces at work here to suggest that this discussion be tabled. Right now, camps are developing on either end of the spectrum, with many if not most cancer professionals are in a large undecided middle. This war of words includes pharmaceutical company press releases promoting drug development, lectures describing how organizations are trying to develop ways to measure the true value of particular treatments-not just the cost, but what the drug really brings to the table, how many side effects does it have, and what is its “financial toxicity” (the lecturer’s words, not mine) of the medication.

And as this debate rages, I can assure you it’s only the beginning. Listen to the experts and you hear that we are moving forward rapidly to develop new equipment to more rapidly analyze the cancer genome. Another expert exhorts us that we only have drugs to address 5% of the genetic abnormalities we have identified that make a cancer cell a cancer cell. We are looking at a future where literally thousands of drugs may potentially be available to treat the various genetic abnormalities we find in any one person’s cancer (each cancer has a number of these changes, and then with treatment the cell is smart enough to develop a “workaround” which would require a different drug).

One expert made the very appropriate comment that this level of individualization means we are moving toward a day when “every cancer is a rare cancer.” Imagine cancer cells being like fingerprints: each one an individual, except that unlike fingerprints, cancer cells can change their stripes. And we need to meet each of those changes wherever those cells reside in the body.

A couple of months ago I was privileged to be a coauthor on an article published in Clinical Cancer Research talking about these issues. The article was written by members of the Turning The Tide Against Cancer initiative, and described what we have to do to change the system. Refocusing on the entire ecosystem of research, translation of drugs from lab to bedside, clinical trials, clinical practice and how we care for patients was the topic, and all of those have to be addressed. It will be an immense effort that will require intense focus and commitment from many participants to reform what we do and how we do it and then make it work for our patients, wherever they may be. Ultimately, patients must be our focus and always be the center of our attention. Whatever we do must be through the lens of the patient and their families.

What bothers me about all of this? While it’s great this issue is finally getting some attention, I don’t yet see the passion I would expect to see given the serious barriers standing between us and our promised future. We have spent decades getting to this point. We need to get the new effective treatments to patients as quickly as possible, wherever that patient may be. We need new trial systems to evaluate drugs, and perhaps rethink how we study drugs to learn as much as we can from each patient.

And then we need to figure out how to pay for all of this, supporting our research enterprise, pay for the care, and afford the new drugs. Many people, organizations, and corporations have to rethink how they do business. Society must make this process work.

Here we sit, but is it on the edge of a precipice or the edge of an opportunity? Who will take the lead? Who will truly define value? Who will afford the costs?

So many questions, and hopefully some answers are coming. We need a new paradigm, we need a better future. Business as usual is not going to get us where we need to be. If we want to make this cancer’s last century we had best take hold of the opportunity and make it happen. We may not get another chance if we don’t act now, and make our future and our vision our reality.


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

Key SGR Fix Brings Relief to Diagnostics Industry

April 8, 2014 by

On April 1, the diagnostics industry let out a collective sigh of relief as President Obama signed into law HR 4302. Officially known as the Protecting Access to Medicare Act of 2014, many refer to the law as “Doc Fix” or “SGR fix”, focusing on the delay, yet again, of the 24 percent physician pay cut as a part of Medicare’s sustainable growth rate (SGR) formula.

However, my attention, along with that of the diagnostics industry, focused on the provisions in the law that protect clinical diagnostics from unpredictable repricing (subscription required) under the Centers for Medicare and Medicaid Services (CMS) Clinical Lab Fee Schedule (CLFS).

The passage of this law showcases the need and value of the industry to unite in highlighting policies that, though unintended, challenge innovators by creating an uncertain reimbursement environment. Predictable reimbursement policies encourage exploration and innovation within the diagnostics community, improving health care through advances in diagnosis and beyond.

This law acknowledges the transformative scientific advancements the industry has achieved in recent years, catching our policies up with our technologies.

Many questions remain as we look at the implementation of this law, and as such we remain committed to convening the personalized medicine community to work in collaboration with CMS.


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