Posts Tagged ‘personalized medicine’

Reflections from the Turning the Tide Against Cancer Conference

October 16, 2014

Following the Turning the Tide Against Cancer national conference last Thursday, I’ve had time to reflect on what we must do next to improve how patients experience cancer care.

To make that change we must acknowledge key truths.

Science is driving us towards more targeted solutions and novel approaches to treatment. The clinical trials and regulations necessary to get new products to market must change with the science. They must be nimble to keep pace with the extraordinary advances we are seeing.

Coverage and payment decisions must also allow patients access to advanced treatments and not disincentivize innovations in health care. While new products sometimes seem expensive, we must remember that costs are saved by getting the right treatment to the right patient at the outset of care.

We must educate. Each of us has a responsibility and a role to play in educating all participants in health care about the changes we are seeing in cancer. We must educate patients and their families about the complexity of decision-making and share information with them so that each patient can make informed choices. We must provide resources so health care providers are able to stay current with the latest developments in care. We also must educate policy makers to ensure that negative unintended consequences of policies are avoided.

Everyone working in health care wants to provide patients with the absolute best treatments imaginable – and those we have not yet even begun to imagine. Sometimes that will require addressing very tough questions in order for personalized medicine to be fully integrated into the health system.

Finally, we should celebrate the improvements made in cancer care from innovations in clinical trial design, to imaginative diagnostic testing of solid tumors that allow for better targeting of treatments, and tests that help physicians and patients make the best treatment choices. Advances are being made in science that are extending and improving lives. This is no small feat, and while our goals are great, we must celebrate the achievements that have taken us this far, and acknowledge the policies and pathways that made those discoveries possible.

We have an opportunity and responsibility to change the future of cancer care through continued education and advocacy. I invite you to help be a part of this change by sharing your thoughts and joining us on this journey to turn the tide against cancer.

Turning the Tide Against Cancer: Collaboration to Improve Outcomes

October 6, 2014

Over the past few weeks, the Age of Personalized Medicine editorial team has been talking to leaders from the co-convening organizations of the second Turning the Tide Against Cancer Through Sustain Medical Innovation national conference, which will be held on October 9, 2014, in Washington, D.C.

Our conversation with Margaret Foti, Ph.D., M.D. (h.c.), chief executive officer of the American Association for Cancer Research, on the need for continued collaboration across the diverse spectrum of stakeholders in the cancer community, can be viewed below.

We also heard from Edward Abrahams Ph.D., president of the Personalized Medicine Coalition and Marcia A. Kean, M.B.A., chairman of Feinstein Kean Healthcare, on the need for policies that encourage and keep pace with innovation in cancer research and personalized medicine.

Visit the Turning the Tide Against Cancer website to register for the conference and learn more about ways you or your organization can support the ongoing initiative. If you are unable to attend the conference in person, please join us via webcast on October 9 by visiting the Turning the Tide Against Cancer homepage. The Age of Personalized Medicine will also be tweeting live from the conference using #T3cancer.

Turning the Tide Against Cancer: Policies to Sustain Innovation

October 1, 2014

Leading up to the second Turning the Tide Against Cancer Through Sustain Medical Innovation national conference on October 9, 2014, in Washington, D.C., the Age of Personalized Medicine editorial team had the chance to sit down with leaders from the initiative’s co-convening organizations to talk about the current cancer research and care landscape, what progress has been made since the start of the initiative, and the upcoming conference.

Last week, we heard from Edward Abrahams Ph.D., president of the Personalized Medicine Coalition on the importance of patient-centered care and value, and the need for policies that encourage innovation in cancer research and personalized medicine in order to improve patient outcomes.

Our conversation with Marcia A. Kean, M.B.A., chairman of Feinstein Kean Healthcare, on the importance of enacting policies that keep pace with the rapid development of innovative cancer treatments, can be viewed below. Stay tuned for our video interview with Margaret Foti, Ph.D., M.D. (h.c.), chief executive officer of the American Association for Cancer Research.

Visit the Turning the Tide Against Cancer website to register for the conference and learn more about ways you or your organization can support the ongoing initiative. The Age of Personalized Medicine will also be tweeting live from the conference on October 9 using #T3cancer.

A Preview of the Turning the Tide Against Cancer National Conference – Part 1

September 29, 2014

Leading up to the Turning the Tide Against Cancer 2014 national conference, the Age of Personalized Medicine editorial team posed two questions to leaders in the cancer community who will be speaking at the event on October 9 in Washington, D.C. In part one, we asked speakers to share their insights on the role of innovation in advancing cancer research and care. Check back for part two where we ask leaders to define patient-centered and explain how patient-centric approaches can improve healthcare.

QUESTION: What role does medical innovation play in paving the pathway to progress towards a sustainable and high-value, patient-centric cancer care system?

Here’s what some of the conference speakers had to say:

………………

abernethy

Amy P. Abernethy, M.D., Ph.D., Professor of Medicine, Duke University School of Medicine; Chief Medical Officer and Senior Vice President of Oncology, Flatiron Health, Inc.

“One area of important medical innovation is the generation and use of data. This is not just hype around ‘big data’ but the recognition that we need amalgamation of data streams that tell the whole person’s story. This includes information about the cancer itself, and also the individual’s personal life: their values and needs, their personal experiences, as well as biometric monitoring. The longitudinal story expressed through data can then be used to support many innovations in cancer care. It can act as a clinical annotation stream to inform biological research such as annotation of biospecimens, biomarkers and basic discovery. It can be used to optimize cancer care delivery, or used for quality monitoring and to highlight gaps in care to be resolved. It can be used to compare differing interventions to figure out what works for whom and when. Advancing high quality and sustainable medical care is dependent on innovations around the generation and use of data.”

………………

Newton F. Crenshaw, Vice President, North American Oncology Commercial Operations, Global Business Development  and Advocacy, Eli Lilly and Company

Newton F. Crenshaw, Vice President, North American Oncology Commercial Operations, Global Business Development and Advocacy, Eli Lilly and Company

“Medical innovation is absolutely central to advancing the fight against the over 200 types of cancer. Each new advance, no matter how small, contributes to our scientific understanding of this collection of diseases, and provides new hope and cures to people with cancer. This cycle of continuous innovation has had impressive results and generated savings throughout the health care system: since 1975, the 5-year survival rate for cancer patients has increased by about 40%. To sustain–and accelerate–this progress, our society must continue to foster and reward innovation.”

………………

Coehlo Photo2Tony Coelho, Former Member, U.S. House of Representatives; Chairman, Partnership to Improve Patient Care

“The rapid pace of medical innovation in oncology is increasing our ability to provide more personalized, patient-centered care (based on their biomarkers, quality of life considerations, etc.). Achieving more efficient delivery of high quality care will require continued medical innovation, including development of new treatments, improvements to existing treatments, and increasing efficiencies in the delivery system that support higher quality care and an overall a reduction in the economic and health burden of disease. But innovation is worthless without access to it.”


Visit the Turning the Tide Against Cancer website to register for the conference and learn more about ways you or your organization can support the ongoing initiative. The Age of Personalized Medicine will also be tweeting live from the conference on October 9. Join the conversation with #T3cancer.

Required Reading: September 2014

September 26, 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 September 2014 installment of Required Reading.


Medical Calculators Use Big Data to Help Patients Make Choices by Laura Landro, The Wall Street Journal

Michael Kattan, chairman of the department of quantitative health sciences at Cleveland Clinic’s Lerner Research Institute, discusses sophisticated risk calculators, or “nomograms,” that can combine a patient’s unique characteristics, such as age, gender, race, extent and type of disease and other health factors; compare them with the vast databases of similar cases and studies; and use them to predict probable outcomes depending on the treatment a patient chooses.

FDA’s Shuren Defends Plan to Issue Guidance for LDTs at House Hearing by Michael D. Williamson, Bloomberg BNA

On September 9, the House Energy and Commerce Committee’s Subcommittee on Health held a hearing to examine the regulation of laboratory developed tests (LDTs) as a continuation of the committee’s 21st Century Cures initiative. Members heard testimonies from various witnesses on recently released guidance from the U.S. Food and Drug Administration (FDA) and its impact on innovation and the practice of precision medicine. Read more about the FDA’s proposed framework for regulating LDTs.

Experts Warn US in Danger of Losing Biotechnology Edge by Chris Casey, Medical Xpress

The United States is in potential danger of losing its biomedical edge to countries that are aggressively funding research into personalized medicine, according to discussion that emerged at the 21st Century Cures Roundtable on September 5. Roundtable panelists noted that biotechnology is at a crossroads in America, and that funding levels for research have flattened in recent years.

Researcher Urges Wider Genetic Screening for Breast Cancer by Rob Stein, NPR Shots Blog

Mary-Claire King, the geneticist who identified the first breast cancer gene, is recommending that all women get tested for genetic mutations that can cause breast cancer, regardless of their personal or family history. According to a paper she recently published in Proceedings of the National Academy of Science, women who carry mutations in BRCA1 or BRCA2, but have no family history of breast or ovarian cancer, have the same high risks of developing either cancer as those who are identified to be at-risk by virtue of their family history.

Survey Reveals Insights About Awareness, Understanding of Personalized Medicine, Part 2

September 16, 2014

Following the launch of the Personalized Medicine Coalition’s U.S. Public Opinion About Personalized Medicine survey results, the Pharmaceutical Research and Manufacturers of America’s (PhRMA) asked the panelists from our launch event at the National Press Club – What key benefits of personalized medicine do you think the public needs to know about in order to embrace this approach to health care?

The survey, conducted by KRC Research, tells us that most Americans do not know what personalized medicine is, but once the concept is explained to them they are very supportive of advancing the field. In these short video interviews, each of these experts weighs in on how we connect the dots from lack of knowledge to wide support for personalized medicine.

The full set of video responses can be viewed on PhRMA’s Conversations blog, with additional commentary from Raju Kucherlapati, M.D., Professor, Department of Genetics at Harvard Medical School and Mark Richards, Senior Vice President, Management Supervisor at KRC Research.

Amy M. Miller, Ph.D., Executive Vice President, Personalized Medicine Coalition, discusses how personalized medicine is changing the way we experience health care today, including the development of the first cystic fibrosis treatment in over 20 years and other medicines that are improving patients’ quality of life.

Donna R. Cryer, J.D., President and CEO, Global Liver Institute, talks about the importance of educating both patients and clinicians about personalized medicine and its potential benefit, as well as her personal experience as a patient who has benefited from targeted treatments.

Randy Burkholder, Vice President, Policy, PhRMA, highlights the important future of personalized medicine and the commitment of America’s biopharmaceutical research companies to advancing the field and the science of personalized medicine.

Learn more about U.S. Public Opinion About Personalized Medicine and review the survey findings by reading Part 1 of our series or visiting the PMC website.

Required Reading: August 2014

August 28, 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 August 2014 installment of Required Reading.


The Price of Personalization by Timothy Gower, Proto Magazine

This article explores the growing debate over the cost and value of personalized medicines and identifies ways that the healthcare system may need to adapt to accommodate the development and use of increasingly more targeted therapies that work for smaller patient populations.

FDA to Regulate Thousands Of Cancer, Genetic, and Other Diagnostics by Matthew Herper, Forbes

Earlier this month, the U.S. Food and Drug Administration (FDA) announced plans to regulate laboratory developed tests, many of which are diagnostics developed as result of the exploding field of genetics. The new regulatory framework proposes that any test used to diagnose a disease or to decide on a course of treatment will need to be cleared by FDA before it can be utilized.

It’s Time for Us to Think About Cancer Differently by Paul Mejia, Newsweek

A recent genomic study published in the journal Cell suggests that 1 in 10 cancer patients could be more accurately diagnosed if cancer were defined by molecular and genetic characteristics, rather than by where it is located. Researchers believe that reclassifying cancer by identifying the type of cell that caused the disease, instead of the tissue type, could ultimately lead to better treatment in the future.

RNA Combination Therapy for Lung Cancer Offers Promise for Personalized Medicine by Kevin Leonardi, MIT News

Early research at the Koch Institute for Integrative Cancer Research at MIT offers promise for personalized cancer treatments using RNA combination therapies to improve therapeutic response. The development of an efficient delivery system of individual or combined small RNAs to solid tumors could help regulate genetic mutations underlying a given patient’s cancer.

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

August 22, 2014

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

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

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. 


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