Archive for the ‘targeted therapies’ Category

Required Reading: June 2014

June 24, 2014

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

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

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.

Finding My Future, or, How to Coexist with Cancer

March 24, 2014

In October 2007, my identity was stolen.

In September 2007, I was a 39 year-old wife and mother of two young girls. Then, suddenly, I was a 39 year-old wife, mother of two young girls, and Stage IV lung cancer patient.

At the time of my diagnosis, I learned a majority of late-stage lung cancer patients die within one year. Just one year. One birthday. One summer. Would I make it to another Christmas? I didn’t know. Already a “glass half empty” kind of person, I wondered if this was my death sentence as the sense of a future ahead of me drifted away.

For the first six months, I tried traditional treatments, and I felt terrible. I was finding out what it felt like to experience the decline of death.

But here I am. More than six years later, alive and kicking. And personalized medicine is the reason.

My experience with molecular testing and targeted therapies
My first line of treatment was the classic carboplatin-taxol combo, combined with bevacizumab—the first of the newer, targeted therapies. Even though I was epidermal growth factor receptor (EGFR) negative and might not respond, I fit the common demographic for success closely enough that my doctor and I decided to try erlotinib with bevacizumab. That run lasted over two and a half years—precious time. I spent that time with versions of the most common side effects but otherwise felt pretty normal and lived life actively, something I never expected to do again.

During that time, I learned about the clinical trial for crizotinib on the news, and after three different people contacted me to pass along the story, I considered it an omen of sorts and asked to have my tumor specimen tested to see if I was a fit for this new personalized treatment.

When I found out I was ALK positive—and therefore likely to respond to the treatment—I felt relief. I knew how promising crizotinib already was and now I had my next plan in place. I have been on this targeted therapy for more than two years and I have been living a virtually symptom-free, normal existence with my daughters, with the hope for more.

Targeted therapies have been invaluable to my treatment. I know that I am very fortunate and remain in the minority to have my disease controlled so well. Frankly, that fuels my feeling of responsibility to do something productive to change things. So many—too many—die so quickly.

From my perspective, there are two important considerations for targeted therapies.

  1. We should use molecular testing as a way to inform treatment—not exclude access to drugs: I am enthusiastic about the value of molecular testing, and I also believe that its value lies not in its exclusionary potential, but in informing the priorities of a treatment plan.

    Having benefited over a period of almost three years from erlotinib when I tested negative for the genetic marker, I’d hate to see that same possibility be denied outright to others. I know that for some cancers and other illnesses, there is a concern about “overtreatment,” but in the lung cancer world, we’ll take whatever we get because our odds are so poor.

  2. We should better keep healthcare providers educated and up-to-date on the latest in molecular testing: I have heard horror stories about oncologists who don’t know much about the testing, never mention it to their patients, and trudge down the traditional paths without pause.

    We need better approaches to ensure doctors have access to the latest research and tools for diagnosis and treatment. I worry about the patient that doesn’t have the tools to advocate for their own healthcare.

Millions of people are depending on scientists, business people, and policymakers to keep working hard and collaborating to bring access to promising therapies to those in need and accelerate medical breakthroughs.

Now, I live with Stage IV lung cancer. It is a part of my body and part of my identity. My outlook on life has changed dramatically since my diagnosis, but my ability to take care of my daughters and live my life hasn’t at all. And that is incredible.

Molecular testing and personalized medicine gave me my life back, and my sense of a future back. While I’m realistic enough to know that my daughters are fairly certain to lose their mother before they are grown, I also know I have tools to fight with, and a responsibility to share my story.

We need earlier detection.
We need the tools to cure.
We need to increase survival for those, like me, who live with cancer.

Ten Years into Personalized Medicine: What We’ve Learned and What’s Next

January 15, 2014

Ten years ago, the sequencing of the entire human genome, along with the development of aggregate “omics” technologies began giving rise to a fundamentally new capability for the practice of medicine – the ability to predict and track disease risks on a personalized basis, to understand diseases mechanistically, and to target therapy to treat an individual’s specific disease.

Based on the potential impact of these technologies, I predicted in my 2002 Chairman’s address to the Association of American Medical Colleges, that medicine would be transformed from being disease-focused and reactive to being proactive and personalized. I believed that the “one-size-fits-all” approach to disease care was outmoded and would soon be replaced by one that would prevent and treat disease on a personalized basis.

Since its inception ten years ago, many of those predictions have been realized; personalized medicine has begun to have major impacts on components of medical practice and has engendered health care industries estimated to grow to $450 billion by 2015 with $42 billion related to drugs, devices, and diagnostics. Molecular diagnostics have gained traction in cardiology, rheumatology, transplantation, endocrinology, and, in particular, oncology. Targeted therapies have revolutionized cancer therapy and whole genome sequencing is providing insights into baseline health risks and understanding of some diseases. However, while the use of personalized medicine tools to treat disease is gaining traction, the transformation of medical practice to being proactive, strategic, and personalized; i.e., personalized health care, has been slow to develop.

There is, however, evidence that this movement is gaining momentum and with increases in health care consumerism, a more predictable regulatory environment, and changes in medical reimbursement to reward better outcomes, the adoption of personalized health care is inevitable. Personalized medicine will go beyond the use of technologies to individualize disease care to finally transforming the approach to care itself by enabling individuals and care providers to foster proactive, personalized care. In turn, the clinical adoption of proactive, personalized care will broaden the need for personalized medicine technologies thus spurring the growth of this industry.

Appreciating the value of a ten-year review of personalized medicine, the Personalized Medicine World Conference will host, and I will moderate, a panel discussion titled, “Ten Years into Personalized Medicine: What We’ve Learned & What’s Next” on January 27, 2014 in Mountain View, Calif., with luminaries including Kim Popovits of Genomic Health, Randy Scott from InVitae, Brook Byers of KPCB, and Jay Flatley from Illumina, opining on what they initially anticipated, what they’ve learned, and what’s coming next.

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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. For more information and the full agenda, visit: 2014sv.pmwcintl.com.

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.

Angelina Jolie Lends Her Voice and Experience to Personalized Medicine

May 16, 2013

It was great to see Angelina Jolie’s eloquent op-ed in the May 14 edition of The New York Times. She carefully wove her personal experience with the importance of genetic testing and using the resulting information to make an informed health decision. I applaud her decision to go public with her story as a great example of patient empowerment but also as a powerful example of personalized medicine in action.

Despite all the advances we have made in recent years, we all know that the concept of personalized medicine is still foreign to most Americans. PMC recently conducted focus groups which strongly reminded us of the uphill battle that remains to gain public understanding of and support for personalized medicine. In our groups only one or two out of 52 participants were able to correctly describe the concept.

We found that people are very enthusiastic about personalized medicine once they understand it but it is a complex concept which is often misunderstood. It turns out that giving clear, specific examples of personalized diagnostics and treatments is the best way to explain what personalized medicine is and the benefits it can bring.

Ms. Jolie’s contribution demonstrates that without patients who are educated, empowered to get tested and to act, the progress and innovation could be for naught. We need to make sure that patients are knowledgeable enough to pursue personalized options for their own health but also so that they are motivated to support policies that foster continued progress in this area.

Angelina Jolie’s op-ed is an important and high-profile contribution to the discourse about patient experience and personalized medicine.  Her voice echoes the words of Adriana Jenkins, who courageously shared her battle against breast cancer and joins other advocates like Donna Cryer, who works tirelessly to remind all stakeholders about how personalize medicine is a patient-centric approach to healthcare. Courageous individuals, like Ms. Jolie, who publicly share their personal stories have the power to bolster both public and media discourse around patient empowerment, for example with CNN “Early Start” co-anchor Zoraida Sambolin’s disclosure of her own breast cancer diagnosis and treatment decision live on air.

I hope that Ms. Jolie’s op-ed raises awareness of personalized medicine and the options patients have to take control of their healthcare.  No single organization alone can raise public awareness on the scale that we need but through a steady stream of individual op-eds, blog posts, conversations, etc., we in the personalized medicine community can spotlight more stories like this and begin to educate the public.  I look forward to hearing more voices from the community and hope that their contributions will support personalized medicine research, patient and provider engagement, and thoughtful policy.

Shaping the Future of Personalized Medicine with TEDMED

March 22, 2013

I had the opportunity recently to participate in a TEDMED Great Challenges panel discussion entitled, “Shaping the Future of Personalized Medicine.” My fellow panelists and Challenge Teammates included representatives from 23andMe, Foundation Medicine, Illumina, and InformedDNA.

As we discussed the challenges, opportunities and benefits of personalized medicine, three themes emerged.

First, personalized medicine, at its root, is about empowering patients to participate in their own healthcare. Trends in popular culture are meshing with advances in technology to allow consumers access to their health information and the ability to make informed decisions, and our culture is changing in that many now demand to be an involved party in their healthcare.

Amber Trivedi of InformedDNA noted that the power of personalized medicine lies not only in treatment, but prevention. As a genetic counselor, the most common questions asked by her patients are:  “What does my genetic information mean to me, and what will it mean to my children?” The best scenario for personalized medicine in action will come when patients are able to see not only the implications of their genetic dispositions, but also are motivated and empowered to use that information in preventative care.

Second, as Michael Pellini, the CEO of Foundation Medicine said, data has to be “accurate and actionable” to have value. However, we cannot discount the potential future benefits of the data discoveries that are underway today. We must find a balance between supporting ongoing data discovery and analysis, while also pushing for data that are actionable now.

Third, traditional healthcare models need to continue to shift to aid in the advancement of personalized medicine. Research cannot continue to solely focus on large population studies; payers need to develop innovative approaches to improve reimbursement policies; and healthcare professionals need training and resources to enable the adoption of personalized medicine in the clinical world.

Finally, when asked what we found to be the most invigorating about personalized medicine, my fellow panelists were quick to share the advances they see on the horizon, including an explosion in targeted therapies aided by new technologies; the shift of cancer to a chronic disease; revolutionizing the treatment of infectious disease through the application of lessons learned from personalized medicine advances in cancer; and deeper data mining enabled by technology currently used in other fields.

Regardless of our individual areas of focus within the world of personalized medicine, this Challenge Team is energized and optimistic about the future of medicine.

Follow the Great Challenges conversation, and submit your questions or comments on Twitter using #GreatChallenges, or at TEDMED.

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

June 8, 2012

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

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

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

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

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

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

Levin, O’Kelly Provide Perspective on Decades of Personalized Medicine Progress, Urge Action and Support

May 22, 2012

On Tuesday, May 8, 2012, the Personalized Medicine Coalition (PMC) welcomed our chairman, Stafford O’Kelly, President of Abbott Molecular and keynote speaker, Mark Levin, Partner and Co-Founder of Third Rock Ventures to its Eighth Annual State of Personalized Medicine Luncheon. The event brought together PMC’s members, partners and other stakeholders involved in realizing the future of personalized medicine for researchers, industry leaders, patients, caregivers, advocates and policymakers.

Stafford O’Kelly reminded us, in his introductory remarks, of the progress and success we’ve made toward achieving personalized medicine in practice within the past year alone. Just last week we saw that Xalkori is showing progress in fighting certain childhood cancers. O’Kelly pointed out the assurances by the FDA that co-approval of drugs and diagnostics will occur more frequently. He also challenged attendees to think about what they can do “to accelerate the shift toward personalized medicine in a way that will improve treatment outcomes for the patient while, at the same time, lower overall costs for our health care system.”

Mr. Levin sought to use his significant experience and background in venture capital, product development and marketing to answer O’Kelly’s provoking question during his keynote address. He urged attendees to support personalized medicine. He explained that “personalized medicine is one of the most important things in medicine today” and lauded its potential to reduce safety challenges, increase efficacy, and improve productivity in the pharmaceutical industry.

I agree that we are at a point where the scientific and clinical progress made in personalized medicine is undeniable. But to echo Stafford O’Kelly’s call to action:  “We need to get the right stakeholders involved to help develop pathways for accelerating growth in the field….We need commitment from payers, providers and patients.”  In order to continue to make progress against disease and improve outcomes for patients, it is necessary as O’Kelly and Levin said, for the entire ecosystem of players to focus efforts in the direction that scientific discovery points us – toward targeted, patient-centric approaches to research and care.

We hope to continue the discussion around these themes and the future of innovation, specifically in cancer research and care at “Turning the Tide Against Cancer Through Medical Innovation,” a national conference that the Personalized Medicine Coalition, American Association for Cancer Research and Feinstein Kean Healthcare are co-hosting on June 12, 2012.  I encourage you to join us in Washington, D.C. as we look to identify and build support for an environment that sustains innovation and drives our evolution toward personalized cancer care.


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