It is a part of the heart that most people have never pondered, let alone heard of, in their lives. But the left atrial appendage – a physical trait that all humans share – is worth knowing about because it is involved in the vast majority of strokes. Now, a prestigious new grant obtained by a team from the USF Health Morsani College of Medicine and Tampa General Hospital could significantly enhance preventive treatments.

Thanks to the innovative work of Dr. Hiram Bezerra, professor at the USF Health Morsani College of Medicine and director of the TGH Interventional Cardiology Center of Excellence, the $460,000 grant could lead to key improvements of an existing procedure to block the opening to the left atrial appendage (LAA) in certain patients who are at high risk for stroke. And it ultimately could deliver safer, faster, and more effective results for patients.

This marks the first time a National Institutes of Health R01 grant – designed to support advanced, hypothesis-driven research projects with strong preliminary data – has been awarded to USF Health’s Division of Cardiology Sciences in the Morsani College of Medicine.

“I think this is reflective of the journey we are on to become a national presence in the forefront of cardiology,” said Dr. Guilherme Oliveira, chief of the division and Ed C. Wright Professor and Chair of Cardiovascular Research, as well as co-director of the USF Heart Institute for Research. Dr. Oliveira also is vice president and chief of the Tampa General Hospital Heart & Vascular Institute.

“This has never been done here before – the ability to get an R01 grant for our division that basically is developing a new technology – with potential clinical applications going all the way from basic pre-clinical engineering of an innovation and taking it all the way to the bedside,” said Dr. Oliveira. “And I think it’s very telling of where we are with the type of talent we’ve been able to attract to USF and Tampa General.”

In this case, the grant, three years in the works, will allow Dr. Bezerra and his team to produce a better, more streamlined approach for dealing with the left atrial appendage – an area in the heart’s left atrium akin to a little pocket. While the structure may help lower pressure in the atrium, it also is possible for blood to pool there in patients with atrial fibrillation, a type of irregular heartbeat, and raises the risk of a clot that could travel to the brain.

“The actual magical aspect of it, and what we are trying to achieve, is a therapy that is offered for stroke prevention,” said Dr. Bezerra. “More than 90 percent of strokes originate from the left atrial appendage chamber. And by occluding the left atrial appendage, you will prevent a stroke in a population prone to have one – the atrial fibrillation, or AFib, population.”

Those suffering from AFib experience an array of symptoms that include an irregular heartbeat, a racing heart, shortness of breath, fatigue and chest pain. People with the condition are some five times more likely to suffer a stroke than those without it – with some 12 million in the U.S. estimated to have AFib by 2030.

“The patients we are targeting have AFib and for some reasons are not a good candidate for the standard preventive therapy of blood thinners,” Dr. Bezerra added. “The next treatment in line is occluding the left atrial appendage. And we are talking about hundreds of thousands of patients in the United States.”

The primary device in the U.S. used to block the left atrial appendage is called the Watchman, manufactured by Boston Scientific, with some doctors employing the Amplatzer Amulet heart device from Abbott. In the current protocol, a patient typically receives a transesophageal echocardiogram two weeks ahead of the procedure to examine the structure and functioning of the heart and evaluate the size of the appendage.

This allows doctors to plan the procedure and select the device. Patients commonly receive general anesthesia for the procedure, which again involves a transesophageal echocardiogram as a real-time guide. In most cases, patients return home the next day.

“But we are pushing to implement a workflow that is less resource intense, and that we believe is actually safer to do,” Bezerra said.

The grant proposed that patients will not have to undergo general anesthesia for the procedure, and a single cardiac MRI would be employed, allowing a patient to just come in once and not have to undergo a separate pre-imaging appointment. The scanner on the day of the procedure would perform the sizing to determine the best device to use. And it would also allow for improved visibility in real-time guidance during the procedure – providing live, higher-resolution images than the current method affords.

“It will all be done with a single modality,” Dr. Bezerra explained. “In addition, the patient is awake. There is no additional cost of the intracardiac echo, or the inconvenience of general anesthesia. And it increases the chances of a patient to go home the same day.”

Dr. Bezerra wrote the grant to be tied specifically to the Watchman because it is more frequently used. But ultimately, replicating the procedure on a different device would not be difficult to achieve. The grant includes a pre-clinical stage at Cleveland’s Case Western University followed by a clinical phase at USF Health and Tampa General. He estimates that it could be available for use on USF Health patients at TGH in three years.

“The plan is for me now to make a few trips to Cleveland, when it’s time for the animal experiments and to help facilitate that,” Dr. Bezerra said. “The next step will be testing for MRI compatibility and starting basic engineering work. A lot of bench and pre-clinical work still needs to take place before we can offer it to patients.”

Dr. Oliveira put it in perspective: “This is the holy grail of grants – where you go, as I said, from a bench concept and have a grant that will support the development of that product all the way to the bedside. It is not easy to do outside of the industry.”

The research also will be a natural fit for work that other physicians, such as Dr. Bibhu Mohanty, already are doing at USF and TGH to advance stroke care, Dr. Bezerra said. Dr. Mohanty, an associate professor in Internal Medicine at the College of Medicine, is an interventional cardiology specialist.

“This grant will complement our very active multidisciplinary neurocardiac program led by Dr. Mohanty in close collaboration with Neurology and Electrophysiology,” Dr. Bezerra said. “With the addition of this translation grant, USF/TGH will continue to be on the very cutting edge of stroke prevention.”

Story by Dave Scheiber for USF Health News.

USF Health researchers have launched several clinical trials as part of the world-wide effort to reduce the severity and even prevent COVID-19.

In connection with Tampa General Hospital, the USF Health researchers are leading the examination of a range of medications and treatment protocols that could impact the disease at a symptoms-level for patients and at the cellular level of the virus.

“The USF Health Office of Clinical Research and supporting parties are making strides that we’ve never made before,” said Rachel Karlnoski PhD, CHRC, director of Clinical Research Operations for USF Health.

“The typical start-up timeframe for new clinical trials in an academic medical center is 90 days or more. Our goal for COVID-19 trials was five days or less and we have succeeded. Communication, collaboration and prioritization from our budget and contract analysts, onsite legal counsel, USF IRB, central IRBs, investigators, study teams and regulatory manager have enabled our success.”

The united, expedited efforts paid off and now USF Health and TGH are conducting multiple front-line studies that link to larger efforts across the country, all in sync for finding treatments and cures for COVID-19.

“I am humbled by the response from our MCOM research staff, and their willingness to volunteer and put themselves at risk,” Karlnoski said. “We have over 30 volunteers from all departments comprised of nurses, coordinators, regulatory specialists, and data analysts who are ready to be deployed when a COVID trial becomes available. The silver lining behind the pandemic is the visibility it has brought to the importance of clinical research in the advancement of medicine. I am optimistic that we will find an effective treatment.”

One of the main overarching research efforts is the inclusion of data related to COVID-19 as part of a national registry tracking many aspects of the virus and those infected with it, such as pregnant women and their babies. USF Health and TGH providers are actively submitting information to this registry.

Among the clinical trials being set up or currently underway are:

• Testing sarilumab to determine effectiveness in blocking inflammation in the lungs in hospitalized severely ill patients.
• Two studies, one in adults and the other in children, are looking at the emergency use of remdesivir, an antiviral drug that may help kill the virus. These studies are for critically ill COVID-19 patients.
• Several studies looking at hydroxychloroquine, on three main fronts:
  • For severely ill patients: Given to patients coming into the emergency department who are severely ill and are going to be hospitalized.
  • For health care workers: A multi-site clinical trial involving 15,000 health care workers across the country to determine if hydroxychloroquine can prevent COVID-19.
  • For outpatient use: Given to those who test positive for COVID-19 but are not severely ill and don’t need to be hospitalized.
• Using convalescent sera/plasma from people who have recovered from COVID-19 in those who currently have the disease to test the sera’s ability treat these COVID-19.
• Testing 3D printed nasal swabs to confirm their ability to prevent so many false negatives compared with standard swabs when used for testing for COVID-19.
• Testing in outpatient settings the use of inhaled nitric oxide to help treat patients with COVID-19 who require supplemental oxygen.
• Studying Ruxolitinib in critically ill patients with COVID-19 and the impact of the medication in easing or stopping the associated cytokine storm, when the patient’s immune system becomes overwhelmed and attacks healthy cells.
• Other studies are looking into ways for treating pneumonia associated with so many cases of patients with COVID-19; assessing the safety and anti-coronavirus response of combined suppression of host nucleotide synthesis in hospitalized adults with COVID-19; testing Brequinar, an antiviral drug for treating COVID-19.

For more information about these and other clinical research taking place at USF Health, please email the Office of Clinical Research at ocr@usf.edu

USF researchers and clinicians heard valuable ideas for connecting with community groups and recruiting more minorities for clinical trials when they attended a community advocacy workshop earlier this month.

The program was an exercise offered by USF Health WE-CARE, an initiative that aims to improve health outcomes among minority populations by increasing minority enrollment and participation in research.

“We are going to cast a broad net to all groups to make sure we are making every attempt we can to capture people and introduce them to your research,” said Kevin Sneed, PharmD, dean of the USF Health College of Pharmacy, senior vice president of USF Health, and director of WE-CARE (Workgroup Enhancing Community Advocacy and Research Engagement).

“We have to build trust and co-create solutions. And we need all of you, the researchers and the community, to shorten the bridge that will get us together and make sure we’re communicating.”

Dr. Kevin Sneed welcomes attendees at the WE-CARE, CAM workshop.

WE-CARE is helping build part of that bridge by connecting clinical researchers with community groups in hopes that more minorities will enroll in research. Clinical trials have historically lacked participation by people from minority populations, skewing research results to reflect presumed outcomes for predominately white and male populations. While African-Americans, Latinos, Asians and mixed sub-groups make up almost 40 percent of the U.S. population, current clinical trial demographics do not reflect that same diversity – non-whites account for less than 5 percent of clinical trial participants.

This narrow pool of DNA variants equates to limited samples for researchers to study as they develop new treatments, and thus limit information on the true effectiveness and on the risks of these treatments in minorities.

WE-CARE, CAM workshop panelists.

Part of the WE-CARE mission is to act as a resource for reviewing research studies and guiding researchers in potential ways of adjusting their effort to incorporate more minorities in their studies. Researchers can submit an abstract to WE-CARE, which goes to the group’s review board and, if approved, WE-CARE will help connect with Tampa Bay area community groups to help with recruitment.

“We can help you find people from underserved, underrepresented groups here in the Tampa Bay area,” Dr. Sneed said to the audience that included many researchers from USF Health and Moffitt Cancer Center.

Both Dr. Sneed and program panelists – made up of leaders from community advocacy groups and non-profit organizations from across the Tampa Bay area – urged researchers in the audience to remain cognizant of the patient’s perspective and to always consider them as individuals.

“The number one thing you can do is to buy into their humanity first,” Dr. Sneed said.

The Nov. 2 workshop was the second event hosted locally aiming to advance clinical research by increasing minority enrollment. About three years ago, a similar workshop was hosted, inspired by and in partnership with 50 Hoops, a national effort for outreach focused mostly on increasing minority male enrollment in clinical trials for prostate cancer. The recent workshop hosted by WE-CARE is based on the CAM model (Community Advocacy Matchmaking) designed by 50 Hoops.

Another concern voiced at the workshop was lack of follow through, and that if researchers want more minority enrollment they will have to engage far more frequently.

“Report back,” Dr. Sneed urged everyone researcher in the room.

“The number one complaint (I hear) is that researchers came into a community, collected, and never came back into the community to give an update. They never heard the result of anything.”

Photos by Eric Younghans, video by Torie Doll, story by Sarah Worth.

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The USF Health Byrd Alzheimer’s Institute unveiled its mobile Memory Research Suite on June 28 – a groundbreaking traveling “clinic” designed to bring the latest clinical drug trials to senior communities across Florida.

That makes the institute the first academic research facility in the state with a mobile clinical trials unit dedicated to finding new treatments for Alzheimer’s disease, a memory-robbing illness that affects more than 5 million Americans and this week claimed the life of legendary women’s college basketball coach Pat Summit.

USF Health Byrd Alzheimer’s Institute leaders cut the ribbon inaugurating the institute’s new mobile Memory Research Suite.

Although current drugs help mask the symptoms of Alzheimer’s disease, there are not yet effective medications to treat the underlying disease or to significantly delay its progression.

David Morgan, PhD, CEO of the Byrd Alzheimer’s Institute, says that 15 investigational drugs for Alzheimer’s disease and other forms of dementia are in the last stage (Phase 3) of clinical study with more in the pipeline, but getting promising drugs to the Food and Drug Administration for review can take four to five years, including more than two years for full enrollment of patients in a trial.

“We need to get drugs to patients sooner,” Dr. Morgan said. “We’re trying to accelerate the entire process, and we think that ideas like this will help do that and increase opportunities for drug study participation for a much broader range of Floridians than exists today.”

Bill and Sheila Nagley were among the guests to tour the mobile clinical trials suite. They currently drive monthly from south St. Petersburg to the Byrd Institute in Tampa, where Bill participates in an Alzheimer’s drug clinical trial.

Scheduled to begin operation in August, the Memory Research Suite features a reception area, patient exam room, two testing rooms, an Americans with Disability Act compliant restroom, a phlebotomy area for blood draws as well as central air conditioning and wireless Internet access. The Byrd Institute’s clinical experts staffing the mobile unit will evaluate study participants and administer investigational medications at pre-selected study sites, expected to include memory disorder clinics, outpatient medical clinics, hospitals, churches, retirement communities.

“Up to this point, we were limited to only being able to enroll people willing to travel to our site,” said Amanda Smith, MD, medical director of the Byrd Alzheimer’s Institute. “By bringing a fully-functioning mobile unit to them, we hope to increase enrollment in trials, increase diversity in research, decrease the time it takes to complete the studies and, ultimately shorten the time it takes to get new treatments to the market.”

Ed Funai, MD, chief operating officer of USF Health and vice president of strategic development for USF said: “Today is a defining moment that positions USF Health as a national leader in addressing Alzheimer’s and other memory-related disorders.”

Ed Funai, MD, chief operating officer for USF Health and vice president of strategic development for USF, thanked the Byrd Institute faculty and staff for all they do to help position USF Health as a national leader in the fight against Alzheimer’s disease.

“We all know someone who has been affected by this horrible disease,” Dr. Funai said. “Clinical trials are essential for the development of more effective and efficient prevention, diagnoses and treatments for Alzheimer’s and memory-related disorders… What will take place inside our new mobile Memory Research Suite will make life better for generations to come.”

USF mascot “Rocky” the Bull with David Morgan, PhD, CEO of the Byrd Alzheimer’s Institute.

Following a ribbon-cutting, guests and media toured the 400-square-foot clinical research space on wheels, which was designed in collaboration with industry leaders from research organizations.

Among the guests was Sheila Nagley and her husband Bill, 74, who was diagnosed with Alzheimer’s disease in 2009. They make the more than one hour drive from their home in south St. Petersburg to the Byrd Institute monthly so that Bill can participate in an Eli Lilly-sponsored Phase 3 clinical trial testing whether solanezumab, a monoclonal antibody designed to help reduce build-up of amyloid plaques in the brains of people with early stages of Alzheimer’s, can slow the disease’s progression.

Amanda Smith, MD, medical director of the USF Health Byrd Alzheimer’s Institute

Bill does not know if the IV infusion he receives is the investigational drug or not; half of the participants in the double-blind randomized controlled study receive solanezumab and the other half get a placebo. But, at the end of the 18-month trial all participants are eligible for the medication.

“We don’t mind the drive (to Tampa), but if this portable facility was available closer to us in our community, we’d choose to use it,” Sheila said. “We started coming to the Byrd Institute in 2012, and they’ve really improved our quality of life with all their resources, including a support group and clinical trials. Having access to people actually doing the research is great.”

The mobile unit was specifically designed make cutting-edge clinical research studies in Alzheimer’s disease more accessible to elderly Floridians and their families across the state.

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Video by Sandra C. Roa, and photos by Eric Younghans, USF Health Communications and Marketing

The Top 20 clinical trial researchers at the USF Health Morsani College of Medicine (MCOM) in fiscal year 2015 represent a broad range of disciplines, ranging from allergy and immunology to pediatrics and rheumatology.  More than a quarter (six) of the top 20 are physicians from the Department of Neurology, in particular clinical researchers specializing in multiple sclerosis (MS), Parkinson’s disease, Huntington’s disease, ataxias, amyotrophic lateral sclerosis (ALS) and other movement disorders.

The list below, from the MCOM Office of Clinical Research (OCR), brought in the most funding from new and ongoing clinical trials in fiscal year 2015 (July 1, 2014- June 30, 2015).  These faculty members lead patient-oriented studies — sponsored by industry, biopharma or foundations — testing the safety and effectiveness of new drugs, devices and other therapies, including some observational studies to manage symptoms and assess disease progression.

USF Health neurologist Derrick Robertson, MD, director of the USF Multiple Sclerosis Program, speaks with patient Kimberly Creech of New Port Richey, who is enrolled in an open label extension trial for ocrelizumab, a humanized monoclonal antibody that has shown promise in reducing MS relapses and also cutting clinical disability.

Total revenue from the trials was $7.2 million in FY 2015 — up 40 percent from $5.14 million in FY 2013.

“As basic and translational science uncover fundamental causes of disease and new pathways for treatment, the ultimate test of their usefulness to improve human health is the clinical trial,” said Stephen Liggett, MD, vice dean for research at the Morsani College of Medicine. “The success of our clinical research enterprise is due to the untiring efforts of our faculty and staff, and has occurred despite increased regulatory burdens, increased costs, decreased margins, and growth of for-profit clinical research organizations.”

From left: USF Multiple Sclerosis Program staff include Angela Aungst, MPH, CPH, clinical research administrator; Dr. Robertson, director; and Lise’ Casady, MSCN, ARNP-C, nurse practitioner. The MS clinic has 120 patients participating in 20 trials testing drugs for various stages of MS.

Photos by Eric Younghans, USF Health Communications and Marketing

Move strengthens cardiovascular services at region’s only academic health center

Tampa, FL  (Feb. 13, 2014) — Florida Cardiovascular Institute, a leading private cardiology practice, has joined forces with USF Health, creating the largest clinical cardiology practice in Tampa at the region’s only academic health center.

“We’re thrilled Florida Cardiovascular Institute is with us. When we looked at all the pieces, it was a natural fit,” said Jeffrey Lowenkron, MD, CEO of the USF Physicians Group.  “The attractiveness of the scope of services and expertise we offer is higher with the addition of FCI.”

Sitting, l to r, Dr. Arthur Labovitz, chair of the Department of Cardiovascular Sciences at the USF Health Morsani College of Medicine, with Dr. Fadi Matar, acting director of the department’s new FCI Division. Standing, l to r, Dr. Bengt Herweg, director of electrophysiology and arrhythmia services at USF Health with the FCI cardiologists who, along with Dr. Matar, have joined USF Health: Drs. J. Thompson Sullebarger, Joel Fernandez, Dany Sayad, John Ramirez and Olga Kuteyeva.

Adding six FCI cardiologists increases to 18 the number of full-time clinical cardiology faculty members in the USF Health Morsani College of Medicine’s Department of Cardiovascular Sciences.  FCI also brings 7,500 patients and 33 full and part-time nurses, medical assistants, ultrasound technicians and administrative staff to the USF Physicians Group, the largest multispecialty practice on Florida’s west coast.

The group will continue to provide care at its current South Tampa location, 509 S. Armenia Ave. USF Health has leased the 10,000-square-foot, second-floor space occupied by FCI, and in March a new co-branded sign will appear on the building.

“These physicians are all high-quality clinicians who add depth and will enrich our overall practice,” said Arthur Labovitz, MD, professor and chair of the USF Health Department of Cardiovascular Sciences and an acting director of the USF Health Heart Institute.  “They will be a catalyst for advancing our department’s growing presence in the community. We will be better positioned to meaningfully affect how cardiovascular medicine is practiced in the Tampa Bay region.”

FCI was established in 1999 by founding physicians Fadi Matar, MD, and J. Thompson Sullebarger, MD, after they left USF’s medical school to go into private practice.  Dr. Matar, medical director of the cardiac catheterization laboratory at Tampa General Hospital, and Dr. Sullebarger, chief of cardiology at TGH, now return to academic medicine as associate professors in USF’s newly created Division of Florida Cardiovascular Institute. Dr. Matar serves as the division’s acting director.

FCI considered several larger groups in the Tampa Bay area as potential partners. But, Dr. Matar said, the private practice’s longstanding interest in research and education and its work at Tampa General, the university’s major teaching hospital, made USF Health “the best fit.”

Dr. Labovitz and Dr. Matar

“We feel good about the move. We’re growing our family from six cardiologists to 18, and they are all colleagues we’ve worked well with in the past,” Dr. Matar said. “Being part of an academic group on the cutting-edge of studying and offering new therapies makes us better doctors who can provide the highest-level care to our patients.”

The transaction highlights a growing national trend of independent physician groups merging with larger partners as doctors seek to avoid being squeezed by shrinking insurance reimbursement rates, control expenses, and adjust to changes in the health care delivery system driven, in part, by the Affordable Care Act.  What cardiologists and other specialists are getting paid for certain procedures or visits is in many cases declining, while the expense and complexity of running a practice are increasing.

“There’s been downward pressure on payment,” Dr. Lowenkron said, “so if you’re in a bigger place it’s probably true you’re a little bit safer.”

USF and FCI say that the move made sense for more reasons than the financial advantages of building a bigger practice. There was alignment between the two groups in several key areas, including:

–          Like-minded patient-centered care philosophies.

–          A shared perspective on the importance of USF Health’s academic missions of education and research in making health care better.

–          The desire to train future cardiologists for the challenges and opportunities of a changing healthcare system

Dr.Matar speaks with a patient. He and his FCI colleagues have a keen interest in teaching future cardiologists and engaging in clinical research.

Together the two physician groups can offer patients a fuller spectrum of cardiovascular subspecialty services than either could alone.  For example, Dr. Lowenkron said, FCI will help cover USF’s gaps in interventional cardiology, while USF’s strength in electrophysiology appealed to FCI.

The university’s research infrastructure, including support for grant writing, biostatistics and outcomes research, and the opportunity to participate in investigator-driven studies also appealed to FCI, Dr. Labovitz said.

FCI adds about a dozen industry-sponsored clinical trials to USF’s mix of 24 federally-funded, private and pharmaceutical-sponsored trials testing new cardiovascular drugs or devices and evaluating standards of care.  That means USF Health cardiology patients have access to an impressive array of clinical studies involving heart failure, hypertension, arrhythmias and other cardiovascular diseases, as well as the first genomics trial, in partnership with the American College of Cardiology, examining the link between genetic information and the risk of developing these diseases.

In addition to Dr. Matar and Dr. Sullebarger, the other FCI cardiologists to join USF Health are Joel Fernandez, MD; Olga Kuteyeva, MD, John Ramirez, MD; and Dany Sayad, MD, all assistant professors.

-USF Health-

USF Health’s mission is to envision and implement the future of health. It is the partnership of the USF Health Morsani College of Medicine, the College of Nursing, the College of Public Health, the College of Pharmacy, the School of Biomedical Sciences and the School of Physical Therapy and Rehabilitation Sciences; and the USF Physician’s Group. The University of South Florida is a Top 50 research university in total research expenditures among both public and private institutions nationwide, according to the National Science Foundation. For more information, visit www.health.usf.edu

Photos by Eric Younghans, USF Health Communications

Media contact:
Anne DeLotto Baier, USF Health Communications
(813) 974-3303,  or abaier@health.usf.edu

A series of new papers in the journal Lancet discussing how to increase value and reduce waste in research includes a contribution by USF Distinguished Professor Benjamin Djulbegovic, MD, PhD.

Dr. Djulbegovic, professor of medicine and oncology at the USF Health Morsani College of Medicine, was a co-author of one of five papers published Jan. 8 in the Lancet series: “How to increase value and reduce waste when research priorities are set.”

The article explains why decisions about which research to fund should be based on issues relevant to the users of research — including patients, clinicians and policy makers – and more systematically account for what researchers already know or are investigating.

“The Lancet series effectively points out that at least 50 percent of research investment is wasted, amounting to billions of dollars a year,” Dr. Djulbegovic said.

Benjamin Djulbegovic, MD, PhD

About $240 billion a year is spent globally on biomedical research.  While the enterprise has yielded substantial health improvements, only about half of the study results are made public for use by other researchers and doctors treating patients.  And, 40 to 89 percent of published trial studies could not be replicated – critical to validating study findings – because the interventions tested were poorly or incompletely described.

In their report, lead author Dr. Iain Chalmers, Dr. Djulbegovic and colleagues point out that good research ideas often produce unanticipated results and these disappointments should not be deemed wasteful as long as “the way in which these ideas are prioritized for research is transparent and warranted.”

Unexpected results are different from avoidable or unjustified waste, which encompasses inefficiencies in the way research studies are chosen, designed, conducted, analyzed, disseminated and reported. It includes the tendency not to report or publish negative study findings, which can be valuable in saving time and money by indicating when a drug or medical device does not work, or may even harm patients.

In their Lancet article Dr. Djulbegovic and co-authors make the following recommendations for reducing research waste:

–          Investigate ways to improve the yield from basic science research.

–          Be more transparent about how research funders decide which research to support, making clear how they take into account the needs of potential users of research.

–          Systematically assess existing evidence before investing in additional research.

–          Strengthen, develop and use sources of information about research already in progress, insist on publication of protocols at the beginning of studies, and encourage collaboration.

Researchers show that uncertainty about new treatments meets ethical requirements

On average, new treatments perform better in clinical trials only slightly more often than existing treatments, according to a new systematic review led Benjamin Djulbegovic, MD, PhD,  distinguished professor and associate dean of clinical research at the USF Health Morsani College of Medicine.

The fact that experimental treatments are not more effective may seem disappointing, but the authors of the review, published in The Cochrane Library, say their findings satisfy an important ethical requirement for clinical trials.

USF Health’s Dr. Benjamin Djulbegovic led the randomized clinical trials review published in The Cochrane Library.

Randomised trials compare the effects of one treatment to another. In a randomised trial patients are randomly allocated to different treatment groups to ensure that like will be compared with like. When a new treatment is being tested, it is hoped or even expected that it will be better than the established treatment with which it is being compared. These expectations lead to an ethical dilemma. If the researchers already know that one treatment is better, they would be knowingly allocating some people to an inferior treatment. If randomised trials are to be ethical, therefore, only half of new treatments should turn out to be better than existing ones.

Cochrane researchers looked at evidence from 743 publicly funded randomised trials involving 297,744 patients in total. The trials included new, experimental treatments for cancer and neurological disorders, as well as a range of other diseases. On average, only very slightly more than half of new treatments proved to be better than established treatments.

“When we compared the effects of new treatments to established ones, the pattern was almost symmetrical. This is good news, because it means researchers genuinely don’t know whether new treatments are going to be any better,” said Dr. Djulbegovic, who works at USF Health Clinical Research, and the H. Lee Moffitt Cancer Center & Research Institute, at the University of South Florida in Tampa, Florida, US. “So, overall, what we show is that we can expect the new treatments to perform better a little bit more often than established treatments, at least in publicly-funded trials like the ones we considered.”

The researchers found the same pattern in trials going back five decades. The results provide an answer to the question posed 15 years ago in the British Medical Journal by Iain Chalmers, a founder of the Cochrane Collaboration and one of the authors of the review.  “In 1997, in a letter published in the BMJ, I asked ‘What is the prior probability of a proposed new treatment being superior to established treatments?’ I think this review currently provides the best answer to that question,” said Chalmers.

Full citation: Djulbegovic B, Kumar A, Glasziou PP, Perera R, Reljic T, Dent L, Raftery J, Johansen M, Di Tanna GL, Miladinovic B, Soares HP, Vist GE, Chalmers I. New treatments compared to established treatments in randomized trials. Cochrane Database of Systematic Reviews 2012, Issue 10. Art. No.:MR000024. DOI: 10.1002/14651858.MR000024.pub3.

About The Cochrane Library
The Cochrane Library contains high quality health care information, including the Cochrane Database of Systematic Reviews, from the Cochrane Collaboration. Cochrane Systematic Reviews bring together research on the effects of health care and are considered the gold standard for determining the relative effectiveness of different interventions. The Cochrane Collaboration (http://www.cochrane.org) is a UK registered international charity and the world’s leading producer of systematic reviews. It has been demonstrated that Cochrane Systematic Reviews are of comparable or better quality and are updated more often than the reviews published in print journals (Wen J et al; The reporting quality of meta-analyses improves: a random sampling study. Journal of Clinical Epidemiology 2008; 61: 770-775).

In June 2012, the Cochrane Database of Systematic Reviews received an impact factor of 5.715, from Thomson ISI, placing it in the top ten general and internal medicine journals.

 The Cochrane Library is published by Wiley on behalf of The Cochrane Collaboration.

 The Cochrane Library Podcasts: a collection of podcasts on a selection of Cochrane Reviews by authors of reviews in this issue will be available from www.cochrane.org/podcasts.

 Accessing The Cochrane Library
The Cochrane Library can be accessed at www.thecochranelibrary.com. Guest users may access abstracts and plain language summaries for all reviews in the database, and members of the media may request full access to the contents of the Library. For further information, see contact details below. A number of countries, including countries in the World Bank’s list of low- and low-middle income economies (countries with a gross national income (GNI) per capita of less than $4700), have national provisions by which some or all of their residents are able to access The Cochrane Library for free. To find out more, please visit www.thecochranelibrary.com/FreeAccess.