News and Articles

8/15/2012 Prospective Observational Studies to Assess Comparative Effectiveness: The ISPOR Good Research Practices Task Force Report
In both the United States and Europe there has been an increased interest in using comparative effectiveness research of interventions to inform health policy decisions. Prospective observational studies will undoubtedly be conducted with increased frequency to assess the comparative effectiveness of different treatments, including as a tool for “coverage with evidence development,” “risk-sharing contracting,” or key element in a “learning health-care system.”
8/1/2012 Real-world and late-phase studies in "pharmerging" markets: challenges and best practices in implementation
Published in the August 2012 issue of SCRIP Regulatory Affairs,
Real-world and late-phase research can help bio-pharma companies maximise product value and minimise risk. Michelle Builliard and Ken Lee report that the "pharmerging" markets are building a healthy environment for these types of studies and explain how companies can clear the hurdles to implementing such research.
6/4/2012 US and European Perspectives on Interventional and Observational Research Designs in Post-Marketing Safety
Applied Clinical Trials,
Pharmacovigilance and safety of medicines are key priorities for regulatory agencies, with a strong focus on ensuring that both risks and benefits are monitored throughout a medicinal product's lifecycle. For marketed products, sources of adverse reaction reports include those received spontaneously from health professionals as well as those derived from post-authorization studies and trials.
5/1/2012 Are Phase Labels Still Relevant?
Applied Clinical Trials,
In the biopharmaceutical industry, research terminology largely reflects that phased regulatory drug development process. Around the time of product approval, there are many descriptors - late phase or late-phase research, registration/pre-registration research, peri/post approval research, postmarket research, Phase IIIb/IV research - but no formal definition exists, and the ones available are used inconsistently.
5/1/2012 Real-World Research: Improving Clinical and Financial Outcomes
Group Practice Journal,
Practices are facing increasing financial and clinical pressures to expand electronic data collection and reporting and collect more clinical outcomes data demonstrating the quality of patient care being delivered. As practices implement new IT systems and projects, they should also evaluate how to leverage these new tools for improving financial and clinical outcomes.
4/1/2012 Generating Real-World Evidence for Rare Diseases
ISPOR Connections,
Defined by regulatory standards in the United States as any disease that affects fewer than 200,000 lives, and in Europe as one that affects 5 in 10,000 lives and is also life-threatening or chronically debilitating, more than 50 million people worldwide suffer from a rare disease [1,2]. To date, more than 7000 rare diseases have been identified, such as cystic fibrosis, sickle cell anemia, hemophilia, Wilson’s Disease, and Gaucher Disease. While the high costs of emerging therapies demands more evidence of benefit, evidence development represents a significant challenge for these diseases.
2/1/2012 Making Informed Decisions: Assessing the Strengths and Weaknesses of Study Designs and Analytic
Comparative effectiveness research (CER) is defined as “the conduct and synthesis of research comparing the benefits and harms of different interventions and strategies to prevent, diagnose, treat and monitor health conditions in “’real-world’ settings.”1 The goal of CER is to improvepatient outcomes by providing decision-makers, such as patients, providers, policy-makers, and payers, with information as to which interventions are most effective for specific types of patients.
9/12/2011 Insider Insights: Outcome Sciences
CWWeekly,
What can CROs learn from disease patient registries that sponsors, disease associations, patient advocate groups and medical professional organizations don’t already know about the major researched diseases?
9/1/2011 The Power of Observation
Pharmaceutical Executive,
As the name implies, in observational studies one 'observes.' The choice of therapy is determined by the patient and her physician rather than protocol, and, because participation does not impact which treatment a patient receives, these studies can enroll a greater diversity of patients. As such, these studies are generally viewed as more 'real-world' than classic clinical trials. As the importance of these studies have grown, so have efforts to develop best practice guidelines in the design, implementation, and evaluation of these studies, so that high-quality evidence can be used for decision-making among regulators, payers, physicians, and patients.
6/1/2011 Patient Registries for Understanding Natural History of Disease
iHealth Connections,
Understanding the natural history of a disease- its characteristics, management, and outcomes with or without treatment - is critical for developing new therapies, identifying best practice guidelines for treatment, and improving patient outcomes. The natural history of a disease may vary across different groups or geographic regions, and it often changes over time as new therapies are introduced. In many cases, the natural histories of diseases are not well described. The need to develop and conduct studies to better understand the natural history of diseases has been recognized by a wide range of stakeholders, including funding agencies, regulatory bodies, researchers, clinicians, and patient advocacy groups.
3/2/2011 A Registry of Registries
ClinPage,
There is a new federal effort to organize disease registries. It's a meta-repository of all disease registries—a big list. The FDA will be advising on its design but play no formal role in creating or running it, according to Richard Gliklich, president and CEO of Outcome. His company is gathering expert brainpower on the project and will build the registry. The details of the design are still to be finalized. The Cambridge, Mass., firm specializes in running patient registries and other databases. It typically supports hundreds of them at any given time.The grant is from the Agency for Healthcare Research and Quality (AHRQ), the primary federal clearinghouse for figuring out if treatments work and are cost-effective. To the extent that any part of the U.S. government is doing research on how to compare the costs and clinical profiles of two drugs, AHRQ is it. Gliklich says the registry of registries will serve several roles.
3/1/2011 The State of REMS in the US
Regulatory Affairs Pharma
Although it has been three years since the passage of the US Food and Drug Administration Amendments Act of 2007 and the risk evaluation and mitigation strategies (REMS) programme it introduced, there still exists a debate over what the clear reasons are for REMS, when the FDA should require one and how effective they have actually been in mitigating risks. The agency began approving REMS in 2008 and, as of January this year, 175 REMS had been approved.
3/1/2011 Patient Registries and Rare Diseases
Applied Clinical Trials,
With more than 25 million people affected in the United States - and 50 million worldwide - rare disease research and treatment is an important medical necessity. Yet, little research has been done on many rare diseases, leaving both physicians and patients with a lack of treatment options, and, in some cases, with only a basic understanding of the disease itself. New studies focusing on rare diseases often face challenges related to small patient populations, the lifelong nature of the diseases, and variations among disease sub types. Increasingly, researchers are turning to patient registries to fill some knowledge gaps, as registries offer an opportunity to learn much about the way patients are diagnosed and treated and about how they respond to various management approaches.
2/28/2011 HIT Exchange Media Interview - Dr. Francis X. Campion
HIT Exchange Media spoke with Outcome Sciences, Inc. Director of Provider Programs, Francis X. Campion, MD, on the movement of data from an electronic health record to a registry.
1/26/2011 CMS Meaningful Use - An Imperative Opportunity for Hospitals
Western Pennsylvania Hospital News and More,
The Centers for Medicare and Medicaid Services (CMS) Meaningful Use (MU) program is in full gear for hospitals and physicians. Under the American Recovery and Reinvestment Act (ARRA) of 2009, the Health Information Technology component, entitled the HITECH Act, incentivizes hospitals and doctors to adopt electronic health records (EHRs) and to use them in a “meaningful” way to improve the quality and efficiency of patient care. The Act has specific provisions to establish a population- focus of care, including the use of health information exchange (HIE) networks at a regional level and public health initiatives.
1/1/2011 A Registry of Registries? The US Backs the Idea for Patients
Nature Medicine,
Building on the success of ClinicalTrials.gov, a registry of nearly 100,000 federally and privately funded clinical trials around the world, the US government is now planning to build a registry of patient registries. The ultimate goal of the effort is to create a one-stop shop where physicians, patients and researchers can find these lists of individuals who have made themselves available for observational medical studies.
10/15/2010 Effectiveness of Antiviral Treatment in Human Influenza A(H5N1) Infections: Analysis of a Global Patient Registry
Journal of Infectious Diseases,
Although the world was challenged in 2009 by a pandemic caused by influenza A(H1N1), human infections with H5N1 virus continue to be reported and remain a threat. The existence of an extensive avian reservoir makes it likely that the H5N1 virus will continue to pose a threat to human health. The high mortality rate associated with H5N1 (reported by the World Health Organization [WHO] as 59% [1, 2]) and the novelty of the virus to humans give cause for concern that, should the virus acquire increased capacity to transmit from human to human, it might trigger a pandemic that exacts a very high human and economic cost.
10/1/2010 Why Observational Studies Should Be Among The Tools Used In Comparative Effectiveness Research
Health Affairs,
Comparative effectiveness has been defined as “the conduct and synthesis of research comparing the benefits and harms of different interventions and strategies to prevent, diagnose, treat and monitor health conditions in ‘real world’ settings.”1 The Patient Protection and Affordable Care Act of 2010 established a new center, the Patient-Centered Outcomes Research Institute, for comparative effectiveness research. It also called for the creation of a methods committee to “develop and improve the science and methods of comparative clinical effectiveness research.”
8/3/2010 Leveraging Electronic Health Records
RAJ Pharma,
Regulators are increasingly using EHRs in their post-market surveillance activities. The emphasis on improving the safety of drugs and devices has increased in recent years, leading to efforts by regulators around the world to develop new methods and initiatives. While pre-market safety data remain vital, much of the attention has shifted towards improving the collection and reporting of post-market data. Post-market safety data may allow decision-makers to more effectively identify and characterise rare events not seen in clinical trials or events that occur in patients who are different (eg older or with more co-morbidities) than those included in clinical trials.
6/10/2010 GRACE Principles: Recognizing High-Quality Observational Studies of Comparative Effectiveness
The American Journal of Managed Care,
Comparative effectiveness (CE) has been defined as “the conduct and synthesis of research comparing the benefits and harms of different interventions and strategies to prevent, diagnose, treat, and monitor health conditions in ‘real world’ settings.”
4/1/2010 Envisioning the Quality-Focused Practice of the Future
Group Practice Journal,
With the recent flurry of political initiatives and legislation surrounding evidence-based medicine and outcomes-based reimbursement, a reform movement has begun to take shape. Although it is being expressed through different models, including accountable care organizations, performance-based care coordination, payment bundling, patient-centered medical homes, and gainsharing, they all touch on the same underlying theme behind all the proposed legislation - improving quality and reducing costs. Elements could be taken from each model to create what politicians are envisioning as the practice of the future.
3/12/2010 Power of observation propels comparative effectiveness research into the mainstream
SCRIP,
US agencies are making inroads in the use of comparative effectiveness research to support more robust decision-making. Observational studies are an emerging force in this area and promise to overcome some of the limitations of randomised clinical trials. Such studies have enjoyed some success in fields including cancer, cardiovascular disease and diabetes. Yet some stakeholders fear the advent of the rationing of care, explain Dr Richard Gliklich and Michelle Leavy
2/25/2010 State of the Industry (HIMSS Convention News)
HealthNewsDigest.com,
In February 2009, the U.S. government enacted the American Recovery and Reinvestment Act (ARRA) to serve as a stimulus to the American economy. With the intention of improving the quality of patient care and ultimately reducing overall healthcare costs, the act included an investment of $147.7 billion dollars in Medicaid, health insurance subsidies, health information technology, health research institutes, prevention and wellness, effectiveness research on healthcare treatments, and more.
1/1/2010 Patient Registries: They May Ease the Challenges of PQRI Reporting
MGMA Connexion,
Is the Centers for Medicare & Medicaid Services’ (CMS’) Physician Quality Reporting Initiative (PQRI) the future of reimbursement for quality improvement? If so, is it worth the effort? Can it help your medical practice improve the quality of care? The registry reporting method may help.
8/24/2009 Registries Rising: FDA Looking At TNF Inhibitors; AHRQ Updates Standards
The Pink Sheet,
The FDA Amendments Act of 2007 gave the agency many new safety powers and the potential for promotional restrictions and post-marketing fines have understandably received much industry attention. But FDA also appears to be drawing upon its beefed-up authority to increase use of a much older, low-tech safety effort: the patient registry.
8/19/2009 Registries for Robust Evidence
JAMA,
Although randomized clinical trials (RCTS) are the bedrock for establishing which interventions are efficacious, there is increasing recognition that they cannot address all needs, especially the need to determine, in a timely manner, the safety and effectiveness of different interventions used in the diverse array of patients and settings that make up a health care system.
8/13/2009 E-Prescribing and the Physician Quality Reporting Initiative: Get in While the Getting is Good!
The American Journal of Orthopedics,
Whether you like it or not, e-prescribing (e-Rx) and the Physician Quality Reporting Initiative (PQRI) are here to stay. And in today's tough economy, why not reap the cash benefits?
5/29/2009 FDA Urged to Add Implant Registries, Premarket Data in Sentinel Plan
FDA Week,
FDA is receiving a bevy of recommendations from stakeholders on ways to structure its Sentinel Network initiative, through which FDA will have access to reams of post-market safety data. Orthopedists want to see FDA integrate implant registries into Sentinel and are suggesting methods for FDA to incentivize industry participation.
5/27/2009 Product safety takes centre stage
SCRIP,
Numerous initiatives in the US and Europe show that the focus on medical product safety is at its prime. All healthcare stakeholders are looking towards regulators and thought leaders to determine what the immediate and residual effects will be.
4/15/2009 American Heart Associations Get With the GuidelinesSM (GWTG) Program, developed jointly with Outcome in 2000, recognized in Wall Street Journal
A push to get hospitals to follow prescribed treatment guidelines is showing promising results in improving care for stroke and heart-attack patients and preventing repeat hospitalizations for those with heart failure. Guidelines for treating such patients have been around for years. But surveys have shown that they often aren't followed, especially for women and minorities, and that adherence to the guidelines varies widely around the country. A program called Get With the Guidelines, launched in 2000 by the American Heart Association, aims to improve outcomes and eliminate disparities in care of heart and stroke patients. About 1,600 U.S. hospitals, or about a third of the total, have so far signed up with the program. Following the release of recent studies showing improved outcomes at hospitals enrolled in the program, institutions are joining at an accelerated pace, program officials say.
4/1/2009 Clinical Trials Vs Registries
Applied Clinical Trials,
As health care stakeholders, from regulators to providers to payers, demand more evidence on the safety and effectiveness of drugs and medical devices in real-world use, they are also increasingly asking life science companies to develop patient registries to fulfill this need.
3/24/2009 Comparative Effectiveness Research Coming Soon?
Pharmaceutical Executive,
As the political debate on healthcare reform intensifies, comparative effectiveness research (CER) has increasingly been cited as an important tool for reducing costs and improving quality. The American Recovery and Reinvestment Act put CER on the national radar by setting aside $1.1 billion for this research, and President Obama has identified a role for more CER. However, opponents argue that CER will be used as a means to control costs by limiting patient access to new therapies and interfering with the medical judgment of physicians.
3/1/2009 Medicare's P4P Program Aims to Improve Care
Managed Healthcare Executive,
For many physicians, their first real experience with pay-for-performance will be through the Center for Medicare and Medicaid Services’ (CMS) Physician Quality Reporting Initiative (PQRI). In 2008, approximately 100,000 providers participated in the program. In 2009, along with the incentive payment (increased to 2%) the participation numbers are expected to grow. Facilitating that growth is the fact that in 2009, physicians may submit data via claims or via the new registry reporting method, greatly increasing the ease of participation.
2/10/2009 Quality of Care in Women With Ischemic Stroke in the GWTG Program
Stroke,
Stroke in women is increasingly recognized as an important clinical and public health problem. Because women have a longer life expectancy and have higher stroke mortality in the oldest age groups, more stroke events occur in women than in men. Moreover, this sex difference in the number of stroke events is expected to increase dramatically over the next few decades.
10/17/2008 Changes in FDA’s Approach to Risk
Applied Clinical Trials,
Drug safety efforts in the United States underwent a fundamental change in September 2007 with the passage of the Food and Drug Administration Amendments Act (FDAAA).The Act directs the FDA to develop a systematic, scientifically sound approach to managing the risk–benefit ratio of a drug throughout its lifecycle, with an explicit focus on postapproval safety.
8/1/2008 F-D-triple-A steps out to a mixed reception
SCRIP,
The passage of the Food and Drug Administration Amendments Act (FDAAA) in September 2007 was a major milestone for drug safety efforts in the US. While the extensive new legislation gives the US FDA more resources, authority and regulatory tools to ensure drug safety, the most important point of the Act may be its mandate that the FDA shift its safety paradigm.
7/16/2008 Heart Failure Care in the Outpatient Cardiology Practice Setting: Findings From IMPROVE HF
Circulation: Heart Failure,
Few data exist regarding contemporary care patterns for heart failure (HF) in the outpatient setting. IMPROVE HF is a prospective cohort study designed to characterize current management of patients with chronic HF and ejection fraction ≤35% in a national registry of 167 US outpatient cardiology practices.
6/23/2008 McClellan Envisions Lifecycle Approach to Drug Surveillance
Bio-IT World.com,
Rising drug development costs and public frustration about major drug recalls highlight the need for an active, post-market drug surveillance system, Mark McClellan told attendees at the recent Post-Approval Summit.
6/16/2008 CDER Stresses 'Active' Post-Approval Surveillance
Bio-IT World.com,
Speakers at the recent Post-Approval Summit left no doubt that we are entering a new era of active surveillance of post-approval drugs. Richard Gliklich, chairman of the conference, said a number of forces have recently changed the role of post-approval research from “what had been an afterthought to something far more intrinsic” to the delivery and evaluation of health products
6/2/2008 Implications of the FDA’s increased focus on post-approval safety
SCRIP,
The passage of the Food and Drug Administration Amendments Act (FDAAA) in September 2007 represents a sweeping overhaul of the US system for ensuring the safety of approved drugs. The legislation includes over 200 provisions dealing with nearly all aspects of the FDA and requires substantial changes in the agency’s approach to drug safety. Given the wide impact of the legislation, the FDA will need to move forward carefully to maintain support among multiple stakeholder groups.
5/27/2008 CDER’s Woodcock Details Expanded Authority, Sentinel Concept at Post-Approval Summit
CenterWatch Weekly,
Opening the Post-Approval Summit at Harvard, keynote speaker Janet Woodcock,M.D., director of the Center for Drug Evaluation and Research (CDER), Food and Drug Administration (FDA), outlined her agency’s expanded authorities in the area of post-approval research oversight brought about by the passage of the FDA Amendments Act (FDAAA) into law at the end of March.
5/27/2008 FDA’s Woodcock On FDAAA
Clinpage,
Last week, we quoted Wyeth’s Joseph Camardo on the renewed user fee legislation known as FDAAA. He’s not sure the new post-marketing environment is starkly different from the old one. In thinking of the still-murky FDAAA regulatory landscape, we were reminded of a lyric from The Who: “Meet the new boss, same as the old boss.” Janet Woodcock, head of the FDA Center for Drug Evaluation and Research (CDER), is in the unusual position of being both the new boss and the old boss. Simultaneously.
5/20/2008 Wyeth on FDAAA
Clinpage,
Because of the random sighting of Ira Spector at one conference or another, we’ve always had a soft spot for Wyeth. We don’t know Spector—no need to fret he has been consorting with the news media. But from remarks by Spector and his lieutenants over the years, we’ve gathered Wyeth seems to have anticipated most of the challenges of the eclinical era, and to have aimed for aggressive competitive and operational goals. It’s hard not to admire their efforts.
5/19/2008 Observations from the Post-Approval Summit
Applied Clinical Trials,
“We are moving to an age of active surveillance and this will create new issues, but also solutions,” said Richard Gliklich, MD, Harvard Medical School and president and chief executive officer of Outcome Sciences. This past week high-level speakers from industry, government and academia gathered at the Post-Approval Summit held at Harvard Medical School, to discuss various post-approval topics.
3/3/2008 When Clinical Trials Aren't Enough
Applied Clinical Trials,
Effectiveness and real-world data underlie the global need for more patient registries and standards.
2/22/2008 Clinical trial firms make passage to India
Mass High Tech,
A rapidly growing health-care software company based in Cambridge is expanding its global presence in South Asia with the launch of a joint venture in Mumbai, India.
8/28/2007 Podcast Interview: Dr. Nancy Dreyer Speaks to PharmaVoice on the Importance of Registries and the AHRQ Handbook
PharmaVoice
8/1/2007 Designing and Assessing Registries to Evaluate the Safety of Medical Products
ACRP Monitor,
Traditionally, the safety of medical products has been assessed through spontaneous reports of serious adverse events, secondary analyses of administrative data collected for other purposes, and clinical trials and meta-analyses of clinical trials. Recent product recalls and warnings have raised questions about whether these strategies provide enough information about rare clinical events that may occur during conditions of real-world use and whether they are reliable enough to guide decisions on removing products from the market.
5/2/2007 Post-Approval Summit: Politician, Serono Speak at Meeting
ClinPage,
"The Post-Approval Summit at Harvard, now in its third year, is the most cerebral conference on our calendar. It is also the meeting most larded with VIP speakers, including a preponderance of physicians who run a) large swaths of international companies or b) big slices of a regulatory bureaucracy in the U.S. or Europe."
5/1/2007 Post-Approval Needs Reassessed
Applied Clinical Trials,
Physicians, payers, and patients all want evidence of the real-world results of medical products, therapies, and services. And they want those results for larger and more diverse populations than are typically studied before approval to enable them to judge whether such products and services are safe and effective and how quality of care might be improved.
2/1/2007 Post-Approval Research: Safety Net or Power Tool?
BioExecutive,
The method of releasing a new product into the marketplace is a delicate balancing act. A developer naturally wants to release its product ASAP. However, the developer also knows the product has to undergo serious testing before it can safely be made publicly available.
12/18/2006 Will CDISC Demo Drive Demand for EDC/EHR Integration?
Bio-IT World, eCliniqua,
Four major pharmaceutical companies and a California systems integration services firm are providing the "brain and brawn" -- as well as the funding – to demonstrate the feasibility of integrating data capture needs of clinical research and disease prevention with electronic health records (EHRs), says Tanyss Mason, director of communications for the Clinical Data Interchange Standards Consortium (CDISC). At present, "it's not unusual for [investigative] sites to have an EHR as well as multiple EDC [electronic data capture] tools, and it's unwieldy."
12/4/2006 Sponsors Getting 'Proactive' about Phase IV Studies
Bio-IT World, eCliniqua,
For a multitude of reasons, it’s a good idea for sponsor companies to spend more energy on so-called “Phase IV” post-marketing studies. A maturing drug pipeline is at risk of generic erosion unless new indications are found. Medicare and private health plans are demanding more real-world data as a condition of coverage for specific subpopulations. High-profile recalls have drawn unflattering public attention to blockbuster products.
12/1/2006 The Emerging Role of the Patient Registry
Good Clinical Practice Journal,
Patient registries are a valuable post-approval tool, providing evidence of the real-world results of medical products, therapies, and services. While clinical trials give important data about treatment effects in controlled conditions, trial data are often not applicable to the diverse population that may receive the interventions in the post-approval setting. This results in evidence gaps that impede the ability of patients and providers to make informed treatment decisions and of payers to determine what kinds of coverage will be appropriate. Patient registries can close these evidence gaps by offering additional data, to enable decision-makers to judge whether products or procedures are safe and effective.
8/1/2006 Registering Results in the Real World
Good Clinical Practice Journal,
A key area of growth in the e-clinical world is the increasing use of patient registries as a tool for monitoring effectiveness and safety, among other measures. We interviewed Dr Richard Gliklich, president and CEO of Outcome Sciences (d/b/a Outcome), to discuss their increasing importance.
6/2/2006 Personalized Medicine Meets the Real World?
Pharmaceutical Executive,
The era of personalized medicine was heralded long before its arrival. But not until Herceptin (trastizumab) came onto the market in 1998 to treat breast cancer in HER-2 positive patients did companies understand just how much genomics could alter the health-care landscape.

Why Observational Studies Should Be Among The Tools Used In Comparative Effectiveness Research