Real World Health Care Blog

Tag Archives: therapies

Alzheimer’s Association Outlines Research Priorities & Treatment Horizons

This week, Real World Health Care brings you a conversation with Maria Carrillo, PhD, Alzheimer’s Association Chief Scientific Officer. Formed in 1980, the Alzheimer’s Association advances research to end Alzheimer’s and dementia while enhancing care for those living with the disease.

Real World Health Care: According to your literature, more than 5 million Americans suffer from Alzheimer’s disease. What do the majority of these patients most need in terms of treatments: treatments to address the symptoms of the disease, or treatments to stop or slow the progression of the disease? Why?

Maria Carrillo, Alzheimer's Association

Maria Carrillo, Alzheimer’s Association

Maria Carillo: What people need most is hope – hope that comes from significant progress in Alzheimer’s research that they can understand and that benefits their day to day life. Thanks to the amazing work of thousands of Alzheimer’s Association advocates, and many dedicated legislators, we have in place a U.S. National Plan to Address Alzheimer’s Disease. The Plan calls for real progress in treating and preventing Alzheimer’s disease by 2025. An expert workgroup convened by the Alzheimer’s Association recommended a federal investment of $2 billion per year for 10 years to make this achievement a real possibility. We are making strides toward that level of commitment – we’re over half way there – but we still have a way to go.

To more directly answer your question, both symptomatic and preventive therapies have a lot of value. People with Alzheimer’s, and those at high risk of getting it in the near-term, need something to help them now. Greatly reducing or eliminating dementia symptoms and/or pushing back the onset of dementia by ten, or even five, years, will act as something like prevention for the great majority of people – they will live most if not all of their lives free from debilitating symptoms.

Finally, we all look forward to the development of proven ways to reduce risk and prevent Alzheimer’s disease and other dementias – for the millions of lives saved, the billions of dollars saved, and the enormous suffering eliminated.

RWHC: What are some of the most promising areas of treatment research that the Alzheimer’s Association is funding?

MC: The Alzheimer’s Association is the largest private, nonprofit funder of Alzheimer’s research, awarding more than $350 million to more than 2,300 projects. Our goals are to (1) advance our understanding of Alzheimer’s, (2) help identify new treatments, (3) improve care, and (4) further our knowledge of brain health and Alzheimer’s prevention.

The Alzheimer’s Association has provided essential startup funding to, and/or funding to expand the scope of, three major Alzheimer’s disease prevention trials currently underway – the A4 Trial, DIAN TU, and API APOE4.

The Alzheimer’s Association grants program has funded – and continues to fund – some of the most important research threads in Alzheimer’s science. These topics and ideas move the field forward by contributing to knowledge about Alzheimer’s, refining research questions, and yielding clues to causes and treatments. Examples include:

  • Sex, gender and vulnerability to Alzheimer’s and other dementias
  • Commonalities between cancer and Alzheimer’s
  • Effects of oxidative stress and inflammation in Alzheimer’s
  • Vascular contributions to Alzheimer’s
  • Protein misfolding and Alzheimer’s
  • Tau toxicity in Alzheimer’s
  • Biomarkers for Alzheimer’s
  • Development of new scales for Alzheimer’s, including pain and clinical meaningfulness
  • Vaccines/Immunotherapies for Alzheimer’s
  • Insulin and insulin-degrading enzyme in Alzheimer’s
  • Down syndrome and Alzheimer’s
  • Patient ability to consent in Alzheimer’s
  • Differences in Alzheimer’s in minority communities
  • Blood pressure control and Alzheimer’s

RWHC: What are some of the biggest challenges or obstacles that Alzheimer’s researchers are facing in terms of developing new treatments? What can or should be done to overcome those challenges or obstacles, and how is the Alzheimer’s Association helping to overcome them?

MC: The two biggest issues/obstacles are insufficient funding for Alzheimer’s research and the need for more participants for clinical studies.

The Alzheimer’s Association and its nationwide constituency is the leading advocate for increased research funding at the federal level, and also the leading nonprofit funder of Alzheimer’s research. That said, many more voices need to be heard – every day and through every vehicle – to ensure that the federal government understands exactly how high a priority this issue is, and allocates the needed funds.

Recruiting and retaining clinical trial participants is now the greatest obstacle, other than funding, to developing new and better treatments for Alzheimer’s disease. Before any drug or therapy can be used in medical practice, it must be rigorously tested to certify that it is safe and effective.

To address the growing need for clinical trial participants, the Alzheimer’s Association launched Alzheimer’s Association TrialMatch – a free clinical studies matching service. More than 100,000 individuals have registered to search for Alzheimer’s clinical trials using TrialMatch.

Alzheimer’s Association TrialMatch provides users with comprehensive clinical trial information and a customized list of studies they may seek to enroll in. TrialMatch is open to everyone, including people with Alzheimer’s or other dementias, their caregivers, family members, and anyone who wants to get involved in the fight against Alzheimer’s.

There are several other registries also working to identify and marshal individuals for Alzheimer’s disease research. The primary need is for increased awareness that these services exist, and that participating in Alzheimer’s research is one of the greatest gifts that one can give to current and future generations.

RWHC: Is a preventative or curative treatment on the horizon? Do you think this is a disease that can ultimately be prevented before it starts or cured after it starts?

MC: Similar to the way we address heart disease, the preferred strategy for Alzheimer’s would include:

  • Early detection methods that identify those most at risk. Preferably these would be simple, inexpensive and non-invasive, such as a blood test.
  • Effective methods to track the biological course of the disease, and the impact of drugs and other interventions.
  • Preventive and risk reduction strategies – lifestyle and pharmacological – that can be engaged in by everyone throughout life.
  • Multiple effective treatments – that address the disease in multiple ways – for those who, despite the points above, suffer from dementia symptoms.

The Alzheimer’s Association believes that all these things are possible in the relative near-term with the appropriate investment in research.

We have made great strides in treating and preventing many diseases – even major killers such as cancer, heart disease, and HIV/AIDS – when we have made the issue a high priority and made the resources available for research. Now is the time to do the same for Alzheimer’s disease.

RWHC: What sort of initiatives or activities can we expect to see from the Alzheimer’s Association over the next few years?

MC: We still do not completely understand the causes of Alzheimer’s disease, and many questions remain about exactly how it progresses. The Alzheimer’s Association plans to redouble its efforts to support and spotlight in the basic science of the disease, for example at the Alzheimer’s Association International Conference. Increasing our knowledge in these areas should uncover multiple opportunities for therapeutic targets.

At the same time, the Association is deeply involved in clinical trials aiming to prevent Alzheimer’s disease. In addition to the funding initiatives mentioned earlier, we collaborate with Fidelity Biosciences Research Initiative (FBRI) and the principal investigators of all the major prevention studies in the Collaboration for Alzheimer’s Prevention (CAP) seeking to facilitate the sharing of information, resources and expertise that may speed the discovery of new preventive treatments. CAP was first convened in 2011 to help researchers learn from and support each other’s work; share data; harmonize data gathering and trial outcomes to allow for comparability across studies; and hold open, informal dialogue with regulators.

Inflammation is a factor that contributes to the cause and progression of many diseases and conditions. This is emerging as a significant area now in Alzheimer’s research. The Alzheimer’s Association’s Part the Cloud initiative, for example, is making the development of new therapies in this area a target of multi-million dollars in research funding.

Newly launched is Alzheimer’s Combination Therapy Opportunities (ACTO), which aims to provide pilot funding to explore combination therapy opportunities in Alzheimer’s disease. This program will support a biomarker-based clinical trial testing of repurposed drug combinations through Phase II proof of concept.

The Alzheimer’s Association also continues to prioritize issues such as the impact of sex/gender, race/ethnicity, education, and socioeconomic status on the development and progression of Alzheimer’s and other dementias.

 

Four benefits of electronic health records

Leaders from industry, academia, and health care discuss the rollout of this technology at The Atlantic’s sixth annual Health Care Forum

Today The Atlantic Health Care Forum brought together leading policymakers and industry experts in medicine, public health, and nutrition to have conversations about the state of the nation’s health care system. The event was sponsored by Siemens, Surescripts, WellPoint, GSK and PhRMA. Real World Health Care attended to share insights from the panel “Health Care Tomorrow: Examining the Tools and Technologies that Will Revolutionize the Future Health Care System.”

Jamie Elizabeth Rosen

Jamie Elizabeth Rosen

Much of the discussion centered around electronic health records, which are increasingly being rolled out in huge hospital systems after the federal government incentivized their adoption to the tune of billions of dollars five years ago. Four themes emerged from the panel, which included top executives from Johns Hopkins Medicine, athenahealth, PhRMA, and Carolinas HealthCare System.

 

1. Enhancing collaboration.

Electronic health records facilitate a team-based approach to hospital care, as well as allowing for better coordination between hospital systems. “What we’re going to see is it’s going to drive team-based clinical care because everyone in the system will have access to the same medical records,” said Dr. Paul Rothman, Dean of the Medical Faculty and Vice President for Medicine at The Johns Hopkins University and Chief Executive Officer at Johns Hopkins Medicine. “You’re going to see an [increased] level of collaboration not only between delivery systems, but also between the patient and the health care provider.”

However, Ed Park, Executive Vice President and Chief Operating Officer, athenahealth, warned that the decades-old technologies that many hospital systems are using are limited in their capabilities. “The current crop of [electronic health records] are documentation tools instead of care management tools,” he said, adding that they are primarily for use by insurers and lawyers. “What I fear is health systems beginning to buy their way into their own prisons that are built of their own IT…as opposed to dealing in an open environment,” he said.

 

2. Enabling patient-centered care.

Electronic health records enable patients to reap greater benefits from telehealth. “Having your information on your iPhone: that’s not far away,” Dr. Rothman said. “[Patients are] going to do EKG’s at home. They’re going to be measuring their blood sugar at home. The patient will have control of the data.”

Electronic records also hold the promise of helping to solve age-old problems in the U.S. health care system, including keeping contact with patients to encourage them to take prescribed treatment regimens. “There is almost $350 billion a year in inefficiency because of lack of compliance and adherence with medications,” said John Castellani, President and Chief Executive Officer, PhRMA. “If you could just get an improvement in whether patients take the medicines that are prescribed, you could capture this great savings.”

“You have kids who have kidney transplants, and you can give them reminders on Facebook that they have to take their medications,” Dr. Rothman added.

 

3. Targeting therapies for increased success.

Electronic medical records can help health care providers ensure that they prescribe the treatments most likely to work for their patients.

“What I think is the promise of electronic medical records is our ability to find subsets of diseases through the broad diseases we treat,” Dr. Rothman said. “Asthma isn’t one disease. Obesity isn’t one disease. Diabetes isn’t one disease. We are going to be able to find subsets of diseases and target therapies [that work]. That’s when you’re going to see efficiency and return on investment.”

 

4. Harnessing the power of big data.

Our health care system has already begun to see the benefits of ‘big data’ with examples such as the discovery of drug side effects and interactions through mining consumer web search data. “We have to use the technologies to bring down the cost of the drug discovery process,” Castellani said.

“Just taking care of the patient, we capture data,” said Dr. Roger Ray, Executive Vice President and Chief Medical Officer, Carolinas HealthCare System. “That allows us to know when a patient…may be at risk for hospital readmission. Having the ability to mine [data]…makes a difference for patients.

“We all, each of us, remember with longing a simpler time when we could scribble and walk off and our job was done,” he added. “What we know now is that’s not very good for the patient. We had no standardization allowing us to help patients avoid lots of different bad outcomes they could have.”

 

Have electronic medical records impacted your health or that of your patients? Share your thoughts in the comments section.

Companion Diagnostics Target Therapy to the Patients Most Likely to Respond

What cancer patient would want to use trial and error with various chemotherapies when he or she could know first from a diagnostic that one agent in particular would result in the most successful outcome?

David Sheon

David Sheon

A new field has emerged, companion diagnostics, to help predict a patient’s compatibility with chemotherapy or other cancer treatments.  This is a game changer to help ensure drugs are given only to the patients most likely to respond to them. 

According to the Food and Drug Administration (FDA), companion diagnostics already play an important role in determining which therapies are the safest and most effective for a particular patient.  By identifying treatments that work best for specific patients, less money is spent on those that do not work because patients no longer have to endure multiple treatments to find the one that is right for their case. Companion diagnostics, usually created in combination with targeted therapy, not only reduce cost and waste, but also reduce side effects for the patient.

Over the past several years, the FDA has approved the following five companion diagnostics: 

  • Zelboraf and the Cobas Test: The drug Zelboraf specifically treats the melanoma of patients whose tumors express a gene mutation called BRAF V600E. Alberto Gutierrez, Ph.D., Director of the Office of In Vitro Diagnostic Device Evaluation and Safety in the FDA’s Center for Devices and Radiological Health, said that approval “is a great example of how companion diagnostics can be developed and used to ensure patients are exposed to highly effective, more personalized therapies in a safe manner.”
  • Vysis ALK Break Apart FISH Probe Kit: Developed along with the targeted therapy drug Xalkori for patients with late stage, non-small lung cancer who express the abnormal anaplatic lymphoma kinase gene. The test determines if a patient possesses that gene to ensure that the correct treatment is applied.
  • therascreen KRAS RGQ PCR Kit: For those with colorectal cancer who are determining whether the drug Erbitux is right for them. This provides information about the KRAS gene mutation in patients whose colorectal cancer has spread to other parts of the body. If the test shows that the patient does not have the gene mutation, this demonstrates that Erbitux is the correct choice, but not the right one for those with the gene mutation.
  • EGFR Mutation Test: Administered in conjunction with the targeted therapy drug Tarceva, which detects the epidermal growth factor receptor gene mutation in patients with lung cancer. If the patient has the genetic mutation that Tarveca targets, then they are “candidates for receiving Tarceva as first line therapy,” Dr. Gutierrez says. Because this gene is present in approximately 10 percent of patients who have non-small lung cancer, it would go far to help improve the treatment and success rate among these patients.
  • THxID BRAF: Approved alongside two drugs (Tafinlar and Mekinist) – which treat the most dangerous type of skin cancer – this detects the BRAF V600E or V600K gene mutations associated with the disease. If the tumor of the patient contains either of these genes, both drugs are effective. 

Have you – or someone you know – ever been treated with one or more companion diagnostics? What was the experience like and would you recommend it for someone else?

Categories: Cost-Savings

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?

National Patient Safety Program Cuts Bloodstream Infections to Save Lives and Money

Central-line catheters are lifesavers. They’re used in hospitals to deliver therapy where needed and when needed for patients with a wide range of conditions.  Unfortunately, central line-associated bloodstream infections (CLABSIs) result in thousands of deaths each year and billions of dollars in added costs to the U.S. health care system, according to the CDC.

But there’s one collaborative program that has cut CLABSIs in intensive care units by 40 percent, preventing more than 2,000 infections, saving more than 500 lives and avoiding more than $34 million in health care costs. The program, funded by the Agency for Healthcare Research and Quality (AHRQ), used the Comprehensive Unit-based Safety Program (CUSP) to achieve these landmark results.

CLABSIs occur when germs enter the bloodstream through the central line (also known as a central venous catheter), which is placed in a large vein in a patient’s neck, chest or groin to give medication or fluids or to collect blood for medical tests. Such lines are commonly used in intensive care units and can remain in place for weeks or months.

Thanks in part to CUSP, progress is being made to protect people from these infections. In fact, nearly 60 percent fewer bloodstream infections occurred in hospital ICU patients with central lines in 2009 than in 2001. This decrease in infections saved up to 27,000 lives and $1.1 billion in excess medical costs. More recently, CLABSIs dropped 41 percent from 2008 to 2011, up from a 32 percent reduction in 2010.

CUSP Programs, like the one used in the AHRQ project, are being used by a number of state health departments to help prevent CLABSIs. CUSP combines clinical best practices with an understanding of the science of safety, improved safety culture and an increased focus on teamwork. It helps clinicians understand how to identify safety problems and gives them the tools to tackle those problems.

“In the CLABSI project, we learned that the principles of CUSP worked to make care safer, and that clinical teams could sustain those improvements over time,” said Jeff Brady, MD, MPH, Associate Director, Center for Quality Improvement and Patient Safety. “The CUSP toolkit, which is a free resource on AHRQ’s web site, is designed to help clinical teams improve any safety problem, not just CLABSIs or infections.”

Indeed, Dr. Brady notes that new projects are already underway to apply CUSP principles to other safety problems like perinatal care and other settings of care, like ambulatory surgery. In addition, AHRQ is developing a CUSP toolkit module to address patient and family engagement – a resource slated for introduction in the late spring.

The bottom line: CLABSIs are preventable and we have the replicable tools we need to protect more patients.

How are health care providers in your area preventing CLABSIs? Are there steps patients can take? If so, what are they?

Filling the Financial Gap When Health Insurance Isn’t Enough

You can’t escape the headlines: rising expenses and high unemployment. And even for the employed, a sharp reduction in health benefits – coupled with a steep increase in out-of-pocket costs, including deductibles, copayments and coinsurance – is making access to life-saving and life-sustaining therapies out of reach for many Americans.

For some individuals and families, these out-of-pocket expenses can total thousands of dollars each month – much more than many folks earn.

When people in these circumstances need help, many turn to Patient Assistance Programs (PAPs), while others apply for financial assistance through independent non-profits such as The HealthWell Foundation. PAPs – which are offered by state governments or drug makers – are designed for those who cannot afford the cost of medication. Groups like Partnership for Prescription Assistance, NeedyMeds, RxAssistRxOutreach, and the National Center for Benefits (provided by the National Council on Aging), empower individuals to sort out their options and get connected to the PAP that’s right for them, sometimes even helping applicants fill out their paperwork.

What do all these organizations have in common? They focus on addressing the financial strains confronting individuals with health insurance who need important medical treatments but cannot cover their associated out-of-pocket costs and premiums.

Individuals like Marianne of Tarpon Springs, Florida, for example. For Marianne, living frugally her whole life didn’t help. Even though she had health insurance, paid all her bills on time, and once earned a good living as a librarian, the 70-year-old breast cancer survivor could not afford the medicine she needed to keep the cancer from coming back.

With no other alternatives to the $500-a-month life-saving medicine, the fixed-income senior citizen didn’t know where to turn. Until her doctors pointed her to the HealthWell Foundation.

Two years later, and thanks to the financial assistance she received from HealthWell, the still-healthy Marianne travels, cycles, and enjoys the life that continues to “delight and amaze” her.

“I am so fortunate,” she says. “I’ve always been glad to give back to others, and now that I’ve needed the help, I know just how precious it is to receive the kindness and compassion of others.”

Marianne is one of many Americans who benefit from organizations like HealthWell, which has provided copayment assistance to more than 164,000 patients since 2004. Without these critical funds, many of those living with chronic and life-altering illnesses would not have the treatments they need in order to live healthier lives.

No child or adult in the U.S. should go without health care because he or she cannot afford it.

How can charitable copayment assistance organizations partner with businesses, government and other stakeholders to achieve lower costs for health care treatment?

The HealthWell Foundation sponsors this blog.

Get the conversation going in the comments section.

Categories: Cost-Savings