Real World Health Care Blog

Tag Archives: science

Research America Wants YOU to Support Public and Private Sector Investment in Research!

We’ll be live blogging today from the National Health Research Forum: Straight Talk about the Future of Medical and Health Research at Newseum.  Sponsored by Celgene, Johnson & Johnson, Onyx Pharmaceuticals, TEVA, Genentech, University of Maryland School of Medicine, and HudsonAlpha Institute for Biotechnology, we’re pleased to bring you updates from inside the room!

{Note from Editor: our updates below paraphrase speaker comments so that we can share the points being made in real time without the benefit of a recording to verify or use a transcript}

The first panel is titled, “Where will medical research be in 2023?”

We’re underway with our first panel.  Dr Anthony Fauci, Director, National Institute of Allergy and Infectious Diseases, is told that he does a good job of advocating for his division. Has the pendulum shifted away from investment in research?

Dr. Fauci: When you have even a modest increase in a budget, you can preserve the fundamental research activities. But the problem is the budget has been flat for 10 years and inflation takes a bigger toll. As a nation we have to make a commitment that fundamental research should not be part of a discretionary pool.  So any cutting of a budget hits the discretionary pool.  Some people get it and some don’t when we tell this story that basic research is what it is we’ve done for you.  Five years from now we’re not going to be able to tell anyone if we continue to stay flat and not even account for inflation.

John Seffrin, PhD, CEO American Cancer Society: Research America has doubled the NIH budget in a period of five years.  But we haven’t made the case to the public to explain the need for long term investment.  The tsunami of non-communicable diseases coming down the road – we need to convince the public of the need to invest.

France Cordova, PhD, Director, National Science Foundation: Industry gets the need to invest in basic research and advocacy for it, but it seems to be number 10 on the priority list.

Dr. Fauci: I can assure you that no one will stop me and say, with regard to the ebola outbreak, why didn’t we invest more in basic research years ago?  They’ll say: why don’t you have a vaccine now?

Dr. Cordova: We need to make science more accessible, to what I like to call “K to gray.”  With this science is accessible to all ages.

Panel 2: Code Red Again: Can We End the Assault on Public Health Research and Practice

Tom Frieden, MD, MPH, Director, Centers for Disease Control and Prevention: We save $3 for every $1 invested in vaccines, in the health sector alone, and $10 overall…..

Only for this event would I take an hour off from ebola.  The ebola epidemic is worse than is recognized. Reported cases are a small fraction of the total.  Despite maximum efforts – 100 field officers – the largest response in CDC history.  If we had  invested in systems that would find, respond, prevent we could have prevented this disaster…..

Public health is a “best buy.” It keeps us safe and prevents disaster. Every dollar investment in public health pays off.  Ebola, drug resistance – ongoing threats – it will take a funding commitment and partnerships. We at CDC have a partnership with CMS that never existed before.  Public health is the governments responsibility but the government can’t do it alone…..

Handwashing in hospital is not up to par, neither is blood pressure maintenance. We have room to grow in health quality.  Hospital acquired infections are too common…..

The average person can have a big impact on the government. One person is able to change policy. One person made government improve and keep open TB clinics in NY….

With regard to health disparities, high blood pressure and heart attacks is the single largest cause of differences among the races.  The second big issue is teen pregnancy.  These are two areas where intervening makes a huge difference.

Georges Benjamin, MD, Exec Director, American Public Health Association.  The public thinks we have a better protection system than we do….

Seeing your doctor 2-4 times a year is a system where patients think they are staying healthy, but many times this is not effective.  We need to create a better system to keep patients healthy.  Prevention Research should be funded more…..

Saying that you support research, and actually voting to increase or keep funding for research is different.  We need to hold policy makers accountable. Meet with local or state elected officials, with out an agenda, so when you do have an agenda they are more willing to help….

Lynn Goldman, MD, MS, MPH, Dean, Milken Institute School of Public Health, George Washington University:  In the US we feel comfortable that we have systems in place, however it would be possible for a slightly different pathogen to arise that we could not protect ourselves from. We’re doing very little about antibiotic resistant pathogens. We’re not going to be able to control something like that…..

The pay off for the American tax payer is tremendous for increasing vaccinations…..

Richard Kronick, PhD, Director, Agency for Healthcare Research and Quality, HHS: Is there a perception problem? Yes. The public can see that price and accessibility are not perfect, but most people see safety and quality as a non-issue, when it is, especially in less developed countries…..

Where are where failing? What can your organization do better? and in what time frame? This should have been done yesterday.  We need to not only produce evidence, but make sure that this evidence is used.  We have funded work to show how low-income children are overly prescribed anti-psychotics, but we are not able to implement any rules or policies to change this. ….

Jack Watters, MD, VP, External Medical Affairs, Pfizer: The happiest news is that we are all living longer – a cause for celebration. One of the best thing that’s happening is that healthy younger people are living to be healthier older people…..

I see far more appetite for public/private partnerships, in research, in delivering public health. We should recognize that we are all in this together and we must partner more – I welcome the increased appetite……

Seeing a doctor a couple of times per year is better than not at all, but we need to increase contact between all health professionals and patients (nurses and pharmacists)….

We are seeing an improvement in the private sector.  There is a shift in the appreciation to public health by the pharmaceutical industry. Some money is being used for research, but it is harder to convince policy makers for more.  Public health problems are not as “sexy” as proactive research in medicine for uncured/treated conditions…..

There are simple things we can do to increase public health, for example with depression, just asking a person could make a significant difference.

Panel 3: What’s Right – and Wrong – with the Research Ecosystem?

Moderator Margot Sanger-Katz, Health Care Correspondent, The New York Times:

Pablo Cagnoni, MD, President, Onyx Pharmaceuticals: What do you think of the US Research system? We are not moving as quickly as necessary, we could be moving more quickly. From lab to market, the timing is too long.  That being said, we still have the best ecosystem in the world.

The competitive system keeps priorities in the wrong place.

We have to extend Dr Woodcock’s good work to CBER and get companion diagnostic testing approved rapidly.

There are 2 areas where “big data” is playing out. One is in research, more importantly another is in utilization.  Are we utilizing the right drugs with the right patients?

Kathy Giusti, MBA, Founder and CEO, Multiple Myeloma Research Foundation (was unable to make it to the event)

Robert Hugin, Chairman, CEO, Celgene: What do you think of the US Research system? I think that it is a fact that we have the best research system in the world.  Yes we still need to improve, but we always need to. We have a very competitive system, that makes for a great spirit in the science community, and we are always improving. Transational medicine (bringing research to the patients) does not get the visibility, but it is important and appreciated.

We need to provide more economic incentives to collaborate, to avoid redundancies between different research centers. More transparency would help this problem.

Ways to improve- Prevention research by asking for congressional help, more investments.

At $1.5 billion per drug who can sustain this? Something has to change. It’s not sustainable. We must review the system.

With regard to the increasing costs of drugs: I think we look at this in an inappropriate way, the only way we can capture costs is through price. If someone could create a drug that can cure a cancer (and avoid downstream costs), we have to remember that.  The overall impact is very positive. We do a better job than Europe with access.  We should never be embarrassed to talk about it.  Taking cancer for example. In 1970, the cost was 1 percent of spending for oncology drug spending, now it’s at .5 percent.  Remember, the generics didn’t discover those drugs – they wouldn’t have them without our research. Exchange programs discriminate against the working poor – at Fred Hutch, Memorial Sloan Kettering, MD Anderson, people are kept out of those excellent care facilities….CBO says when Rx costs rise, other healthcare costs go down.  We want to talk about Lindsay Lohan and rehab instead of better access to care.  We have this discussion backwards. (Applause)

We are finally able to bring technology to the research because of the high costs. Ten years from now, our lives will be fundamentally different, because of (advances)…

E. Albert Reece, MD, PhD, MBA, Dean, University of Maryland, School of Medicine: What do you think of the US Research system? Our system is very rigorous, and it works very well, but it is not perfect.  Looking at other countries, our research infrastructure is more rigorous, and that is our strength. Our weakness is that we are not sustainable.

Boom or Busts in research, training young people takes a long time, so there is a constant roller coaster in the amount of researchers that we have. The amount of researchers that we needs is constantly changing also.

I agree with Bob, there have to be improvements and legislation to increase efficiencies.

Janet Woodcock, MD, Director, Center for Drug Evaluation and Research, FDA: What do you think of the US Research system? We don’t have the infrastructure necessary to carry the information, that we find through our research, to the American public, and to implement new findings.

Is money being spent in the right way on the right research?  A problem that we have is that a big amount of research is not able to produce the same findings more than once. We need to be more intentional with research. We are trying to change the way that clinical evaluation is done. It is too expensive and not sustainable. We should not have a brand new clinical trial for every single experimental drug, we need to come up with a system that can be reused, and is therefore more sustainable.

How does the regulatory system change the research ecosystem? It creates a lot of challenges, we’re seeing a shift in pharmaceutical research to drugs that depend on genome, more precise medicine. Because this is so new, there are a lot of uncertainties.

Unless we change the cost and drug development process, then we will not keep improving.  We don’t have the right science to actually make the right drugs, only 2/10 drugs even make it to trials. Efficiency needs to change, and this can change through changing the drug manufacturing process.

Our healthcare system is poorly designed when it comes to non-drug interventions. The final translation into practice – we’re trying to look at more patient centered measures to look for ways to benefit through proven, unconventional benefits.

 

Categories: General

Why We Give to HealthWell Foundation – and Why You Should Too

As the head of a communications strategy shop that helps clients in science, technology, and health care, I encounter a seemingly endless number of organizations that want to do good for society and the planet.  Why then have the WHITECOAT Strategies employees – who serve as editors of Real World Health Care (RWHC) Blog – decided that the HealthWell Foundation should be one of our two charter charities, as our firm becomes a social enterprise in 2014?

David Sheon

David Sheon

Before I answer that, just what is a social enterprise?

A social enterprise is an organization that applies business strategies to maximize improvements in human and environmental well-being, rather than maximizing profits for shareholders.

Social enterprises can be structured as for-profit or non-profit organizations, but their focus is using their proceeds to do good.

We decided that organizations seeking communications firms would like to know that revenue from their work is going to help society.  And our employees like to know that too.

When we made the decision to become a social enterprise, we thought about the impact of our work globally and locally.  And that’s how we arrived at helping CA Bikes, as well as the HealthWell Foundation.

CA Bikes is a nonprofit organization founded by Chris Ategeka, a native of Uganda. The oldest of five children, Chris became an orphan and head of his household at an early age after losing both his parents to HIV/AIDS. After years of poverty and laboring in the fields, a miracle happened, as Chris says, when a woman from the United States started an organization called Y.E.S. Uganda near his village, took him in, and supported him through school. Now, Chris holds a BS and an MS in Mechanical Engineering from the University of California, Berkeley.

Many people living in rural Africa have no access to emergency medical services, and given that the nearest health clinic or hospital is often miles away, this results in needless suffering and deaths. CA Bikes builds and distributes bicycle and motorcycle ambulances to rural African villages and trains partners in their maintenance and use to provide access to life-saving care during medical emergencies. For more information about CA Bikes and to help support their work, click here.

The WHITECOAT team is honored to help Chris fulfill the mission of CA Bikes.

WHITECOAT’s history with the HealthWell Foundation dates to a discussion one of my staff members and I had over three years ago.  She told me that her best friend from college had been diagnosed with a brain tumor. He had insurance through his job, which stuck with him through the medical emergency.  His wife had been laid off of her job a month before the diagnosis.  The emotional toll of the diagnosis was awful.  I knew the couple and their children would find their own way to deal with that and there was nothing we could do. But I felt that perhaps we could do something more to find them financial support.

One call to the HealthWell Foundation was all that was needed.  After reviewing financial records and evaluating the situation, the Foundation tapped a fund reserved for medical emergencies that reimbursed not only for the co-pays associated with medication, but also for the cost of the monthly health insurance premium and related medical expenses.  This program has now transformed into the Emergency Cancer Relief Fund, which WHITECOAT is proud to help launch for HealthWell.

HealthWell has awarded more than 265,000 grants to patients in over 40 disease categories, making a profound difference to over 165,000 people faced with difficult medical circumstances in the U.S.

I hope that at this time of giving, you’ll join me and the WHITECOAT staff by donating to the HealthWell Foundation.

Categories: Cost-Savings

World Hepatitis Day Spotlights Importance of Early Detection to Improve Prevention and Treatment Strategies

This Sunday, July 28, is World Hepatitis Day, an observance that reminds us that hepatitis (inflammation of the liver) remains largely unknown as a major health threat. Approximately half a billion people worldwide and 4.4 million people in the U.S. live with chronic viral hepatitis, with one million deaths resulting from the disease each year.

Linda Barlow

Linda Barlow

The goal of World Hepatitis Day is to move from awareness to action to address the “silent epidemic” of viral hepatitis – so named because most people don’t experience symptoms when they first become infected, often not until they develop chronic liver disease many years later.

Stakeholders in government and private industry are stepping up to answer the call, supporting early detection and medical intervention as key starting points to effectively address the epidemic.

Earlier this month, Quest Diagnostics announced a partnership with the CDC to improve public health analysis of hepatitis C screening, diagnosis and treatment for the 3.2 million Americans living with it. Under the collaboration, anonymous patient data will be evaluated to identify and track epidemiological trends in hepatitis C virus infection, testing and treatment and determine how those trends differ based on gender, age, geography and clinical management.

“Our collaboration with the CDC underscores the importance of using diagnostic information to derive useful insights enabling effective prevention, detection and management programs for diseases with significant impact on public health,” Jay Wohlgemuth, M.D., senior vice president, science and innovation, Quest Diagnostics, said in a statement.

Early detection was also the focus of a 2012 National Institutes of Health (NIH) study published in Proceedings of the National Academy of Sciences. In the study, researchers concluded that elevated blood levels of a specific enzyme and a specific protein early on in the course of hepatitis C infection were much more likely to develop into advanced fibrosis or cirrhosis. The study found:

  • The long-term course of chronic hepatitis C is determined early in infection.
  • Rapidly progressive disease correlated with persistent and significant elevations of alanine aminotransferase (ALT), an enzyme released when the liver is damaged or diseased.
  • Rapidly progressive disease correlated with persistent and significant elevations of the protein MCP-1 (CCL-2), a chemokine that is critical to the induction of progressive fibrogenesis and ultimately cirrhosis.

Armed with this information, clinicians are expected to make a fairly accurate assessment of which patients are likely to develop advanced disease rapidly. Instead of waiting for a new class of drugs to be approved, these patients are likely to be pressed to start treatment right away – with the goal of treating the virus before it causes cirrhosis of the liver.

Because hepatitis does not result in symptoms until serious liver damage occurs, getting tested is also crucial. In fact, the CDC recommends that everyone born from 1945-65 get a one-time test for hepatitis C because they are five times more likely than American adults in other age categories to be infected and face an increased risk of dying from hepatitis C-related illnesses.

The first FDA-approved hepatitis C genotype test is now available in the U.S. From Abbott, the fully automated Realtime HCV Genotype II test determines the specific type or strain of the HVC virus present in the blood of an HCV-infected individual.

To locate organizations where you can access services including Hepatitis testing, vaccines and treatment, click here. You can also take this 5-minute Hepatitis Risk Assessment to obtain a personalized report from the CDC.

Early awareness and prevention-based practices are crucial to avoiding hepatitis. But what else can be done to ensure access to and availability of reliable and cost-effective screening and diagnostics, in addition to safe and simple treatment regimens for people with the disease?

We hope this post serves as a resource for journalists covering or interested in writing stories about World Hepatitis Day and related issues. Also stay tuned for our follow-up post next week that will address the cost-savings implications of vaccination and early treatment of hepatitis.

Patient Venture Philanthropies: Catalyzing the Development and Delivery of Therapeutic Breakthroughs

Linda Barlow

Linda Barlow

How are patient-focused organizations making tangible advances in creating life-saving and life-enhancing therapeutic innovations? The experiences of at least two foundations show that collaboration with stakeholders across industries – private, government, academia, insurers and clinicians – is a good place to start.

One example of success is JDRF (formerly the Juvenile Diabetes Research Foundation), the only global organization with a strategic plan to systematically eradicate the effects of type 1 diabetes (T1D) from people’s lives. As a foundation with cure, treatment and prevention strategies that drive the core of its mission, JDRF funds $530 million in scientific research across 17 countries. JDRF’s highest priority is funding research to deliver a cure for T1D and its complications. It is also committed to:

  • Developing better treatments that will transform the way people of all ages with T1D treat the disease at any stage, in order to help them live healthier lives; and
  • Preventing T1D, to keep future generations from developing the disease.

JDRF focuses its funding on therapies and devices that are truly impactful, either in the sense of bringing something to market more quickly or by reaching the largest possible number of patients.

“The challenge is bigger than we anticipated,” according to Jeffrey Brewer, President & CEO of JDRF. “We remain committed to a cure, but are also focused on helping patients live safely and well until a cure comes, through preventive and treatment strategies and treatments.”

Brewer says that JDRF has started working more with industry, noting that historically, the organization focused on academic-based research.

“Forty years of advances in the academic labs has given us the opportunity to translate developments in the labs to companies that will develop therapies to deliver to people,” he says. “Academic research is a critical early component in the pipeline, but company support is also a critical part of the pipeline. That’s why we incentivize companies to do things they otherwise wouldn’t have done.”

“JDRF is willing to take on more risk at an early stage than pharmaceutical companies,” adds Dr. Richard A. Insel, M.D., Chief Scientific Officer, JDRF. “We also are not driven by profit margins or market size. We act as a sort of virtual pharma or biotech company in the early stages of research, and our industry partners step in to bring therapies and devices to market.”

Brewer explains that once companies are able to successfully commercialize therapies and see a financial return, JDRF is “paid back” by those companies, with funds JDRF drives back into more therapeutic research.

“We also work closely with our industry partners and the government, particularly the National Institutes for Health, to make sure resources are being used most effectively and without duplication of effort,” says Insel. “And we work with regulatory agencies to help them better understand what it is like to live with type 1 diabetes so they can evaluate the risk and benefits of new therapies with an eye toward approving them as quickly as possible.”

The Alzheimer’s Drug Discovery Foundation (ADDF) also focuses on prevention, treatment and cure. Using a venture philanthropy model to bridge the worldwide funding gap between basic research and later-stage drug development, ADDF leverages any return on investment to support new research.

The result? ADDF has granted more than $60 million to fund over 400 Alzheimer’s drug discovery programs and clinical trials in academic centers and biotechnology companies in 18 countries.

“Our biomedical venture philanthropy model adapts the operating principles of venture capital investing to the ADDF’s philanthropic mission to advance biomedical research in Alzheimer’s disease. We seek a return on investment for our grants based on the achievement of scientific and/or business milestones. When these milestones are met, funds come back to the Foundation to increase our ability to fund more research,” according to the Foundation.

Source: Parkinsons Action Network

Source: Parkinson’s Action Network

Dr. Insel argues that the participation of patients and families in clinical trials is crucial to translating funding research into impactful therapies.

Do you agree? What other ways can patients living with type 1 diabetes, Alzheimer’s disease or other chronic disease act as advocates for themselves and the disease in general? What other patient venture philanthropy models have you seen work?

Express Scripts Provides Roadmap to Improve Health Care, Reduce Costs and Streamline Delivery of the Medicine Patients Need

You might be in a “utilization management program” and not know what that means or why it matters to your health. Offered by a variety of employers across industries, utilization management programs are designed to help patients evaluate their health care options and make decisions about the type of services they receive.

So how do these programs impact the delivery of specialty medications for cancer, HIV, inflammatory conditions, multiple sclerosis, and more?

MedAdNews.com reports that a new study from Express Scripts demonstrates how such programs can increase efficiency by ensuring that more patients who need safe, affordable and effective medications can access them.

As spending on specialty drugs continues to increase (18.4 percent in 2012, up from 17.1 percent in 2011), finding the most effective ways to improve the delivery of patient care, reduce cost and eliminate waste is more important than ever. Combining innovations from CuraScript and Accredo, Express Scripts draws upon Health Decision Science – which integrates behavioral science, clinical science, and actionable data – as a springboard to achieve just that.

Building upon this scientific, results-driven approach, Express Scripts provides care targeted to specific areas of patient need through Accredo’s Therapeutic Research Centers as part of its Specialty Benefit Services. Here, a broad array of health care providers integrate pharmacy and medical data to offer what Express Scripts describes as comprehensive patient care that strengthens coordination of services, boosts transparency, and produces solutions.

“It’s really about appropriateness and the right thing for a patient who really deserves safe and effective and affordable medication and ruling out waste. What our plans are most interested in is continuing to be able to afford to provide a benefit. This again was a great example of by doing the right thing that patients were able to save a significant amount of money and again preserve affordability,” said Glen Stettin, M.D., senior VP, clinical research and new solutions at Express Scripts.

Does your employer use a multiple cost management program for specialty drugs? If so, what type? If not, do you think your employer should? What might be some advantages or disadvantages?

Categories: Access to Care