Leadership Commitments to Improve Value in Health Care Finding Common Ground: Workshop Summary (2009) / Chapter Skim
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10 Regulators
Pages 237-268
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From page 237... ...
Roundtable on Evidence-Based Medicine is focused on furthering the use of the best available evidence in clinical decision making, that is, evidence that will help provide an understanding of which diagnostic or treatment intervention, among an array of possible options, is best for a given patient. For medical products, this can happen only if the system for identifying, quantifying, and qualifying the evidence that is gathered on the product throughout its life cycle is formalized.
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From page 238... ...
To achieve the IOM Roundtable's goal of achieving medical care that is more solidly based on the evidence, the careful quantification and qualification of evidence related to medical products needs to be extended throughout the life cycles of products. Food and Drug Administration The FDA has regulatory responsibility for drug and biological products and medical devices, beginning in the premarketing phase and lasting for the duration of a product's life cycle.
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From page 239... ...
The first category, primary clinical effectiveness research, which comprises direct comparisons between interventions or between an intervention and no therapy, includes a range of randomized clinical trial designs as well as observational and cohort studies. Primary clinical effectiveness research delivers the principal data that product developers rely on to demonstrate the safety and effectiveness of an intervention.
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From page 240... ...
As explained in more detail below, FDA's statutory authority may not always permit the agency to require significant primary clinical effectiveness research that both is comparative and provides a sound basis for understanding how a new intervention is best used among the existing treatment options. Typically, the primary focus of the major efficacy studies intended to support the approval of new products is the development of data that establish that an intervention has a beneficial clinical effect (i.e., that it is effective and safe for a given use in a defined population)
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From page 241... ...
In addition to the efforts noted above, CMS's Coverage with Evidence Development protocol, the clinical trials policy, and the use of registries support this work. CMS recognizes that collaborative partnerships with a variety of organizations, individuals, and institutions are necessary to support quality measurement efforts.
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From page 242... ...
, these "dual eligibles" depended on Medicaid to help them purchase prescription drugs, and because Medicare has a limited long-term care benefit, many adults over age 65 years depend on Medicaid to provide community and institutionally based long-term care. Across this spectrum of healthcare services, the states are moving steadily toward the greater use of clinical evidence to guide their policies.
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From page 243... ...
This authority encompasses everything from requirements relating to financial solvency to access to health insurance coverage policies and the oversight of certain clinical issues. Because healthcare organizations and providers depend on insurers for much of their revenue, the regulatory actions of the states play a major role in not only how medical services are financed for millions of Americans but also how health care is delivered in any given location.
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From page 244... ...
Clinical evidence influences a subset of these activities, primarily the definition of mandatory services for inclusion in policies and the determination of when covered services must be provided. ACTIVITY CATEGORIES Premarketing Review of Drug and Device Products Large quantities of primary clinical data are generated pursuant to FDA requirements to demonstrate the effectiveness and safety of drug and biological products and medical devices before marketing.
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From page 245... ...
Various organizations have suggested that studies conducted to better understand the optimal use of multiple related interventions might best be coordinated by an independent entity capable of identifying evidentiary needs, prioritizing those needs, and funding research to address the priorities on behalf of a broad coalition of affected parties. Postmarketing Monitoring of Drug and Medical Device Products During the postmarketing phase of a product's life cycle, FDA oversees a mostly passive safety surveillance system -- that is, it largely depends on spontaneous voluntary reports from healthcare professionals and patients of suspected adverse events noted during standard patient care -- but it may also require the development of additional clinical data after a drug is marketed (so-called Phase IV studies)
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From page 246... ...
FDA Communication Activities FDA oversees both the generation of evidence about the uses of medical products and the communication of that information to healthcare professionals and, to some extent, patients. Prescription drug labeling or prescribing information is the primary information tool for communicating drug information to healthcare professionals.
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From page 247... ...
The safety of the medical products that FDA regulates has always been a key focus of FDA's commitment to its mission to protect and promote the public health, and monitoring of safety postmarketing is an important piece of any effort to strengthen the evidence base. A number of efforts are under way at FDA that, once they are implemented, will strengthen the nation's ability to identify the risks related to medical products and to collect, quantify, and qualify the risk information to help inform the growing evidence base.
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From page 248... ...
To build a solid evidence base for medical products, FDA needs to develop not only a way to collect, quantify, and qualify relevant information but also a way to communicate key findings to point of care in the clinic. FDA is working to modernize the processes by which it collects, analyzes, and communicates to the public important risk information, including the creation of user-friendly information sheets for healthcare professionals on emerging safety issues and a periodic, World Wide Web–based newsletter for healthcare professionals on important drug safety information.
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From page 249... ...
For example, today, medical product safety information is largely nonstandardized, making even the most basic analyses and data-mining efforts difficult and time-consuming. The lack of data standards also impedes the development of newer analytic tools.
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From page 250... ...
When an application for an NCD is approved, experts review the available scientific and clinical evidence to determine the effectiveness of the item or service in question. A judgment about the adequacy of the evidence for making coverage decisions depends on the methodological quality of the available research and the magnitude of the effect of an item or service on specific clinical outcomes.
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From page 251... ...
Two current applications of the CMS CED protocol include the implantable cardioveter defibrillator and positron emission tomography registries, which collect information about patients receiving these treatments. On the basis of the recommendations from the Medicare Evidence Development and Coverage Advisory Committee, in July 2007, the clinical trial policy was expanded to include paying for the investigational clinical service if covered by Medicare outside a trial or required through an NCD (i.e., CED)
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From page 252... ...
Department of Health and Human Services (HHS) as standards pursuant to the Health Insurance Portability and Accountability Act are the International Classification of Diseases, 9th edition (ICD-9)
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From page 253... ...
A number of states are involved in pay-for-performance and disease management initiatives, both of which draw on clinical research and system reorganization to improve the quality of care and, it is hoped, health outcomes. State Insurance Regulation Determining which services must be included in insurance coverage can be a controversial and highly political process in which clinical evidence plays an important but not conclusive role.
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From page 254... ...
Evidence is often helpful in determining the questions of the scope of practice and competency that inevitably arise in these decisions. The rationale for insisting on good evidence of effectiveness before enactment of a mandate continues to grow because mandates almost always increase the cost of premiums, and cost is the primary reason that millions of Americans are unable to obtain health insurance coverage.
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From page 255... ...
To the extent that these examinations require would-be physicians to understand how to assess and appropriately apply clinical evidence, they are a critical instrument for helping ensure that the many efforts to improve and produce clinical evidence actually succeed in affecting medical practice. Continuing medical education Most states require physicians to complete a minimum amount of continuing education to maintain an active medical license.
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From page 256... ...
The goal is to work together to develop new standards and identify new methods and technologies that will improve safety, boost the quality of information derived from clinical trials, and make the research process more efficient.3 Facilitating Development of More Personalized Interventions Personalized medical interventions present significant challenges to a regulatory process that has historically been geared to evaluating interventions with fairly general populations. Increasingly, selecting the correct 3 For more detail, see http://www.fda.gov/oc/initiatives/criticalpath/partnership.html.
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From page 257... ...
Several agencies, including FDA and CMS, are involved in the Biomarker Consortium, a broad-based group of interested parties devoted to identifying and qualifying the next generation of biomarkers. Advancing Innovative Clinical Trial Designs FDA is committed to the implementation of innovative trial designs.
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From page 258... ...
They will also improve the efficiencies of clinical trials and product development efforts for subsequent products. Safety Rapid advances in science and technology have increased the complexity of medical products, resulting in increased attention to safety-related issues by the broad healthcare community.
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From page 259... ...
It is very important that FDA work with the healthcare community's public and private sectors -- payers, healthcare practitioners, provider organizations, medical product manufacturers, academia, patients, the states, and other agencies in the federal government -- to identify potentially serious problems resulting from the use of medical products (e.g., adverse reactions and effects, product use errors, and product quality problems) and get that information to the public as quickly as possible.
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From page 260... ...
This should enhance the analytic ability to detect and interpret medical product-related adverse events, regardless of their source. Similar efforts should be pursued in other bioinformation settings.
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From page 261... ...
Sources include healthcare claims, information submitted by clinical trial investigators, manufacturers, professional societies, hospitals, physicians, federal agencies, technology assessments, registries, and other monitored data systems. This data gathering needs to be better coordinated so that current knowledge is available in a standard and open manner.
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From page 262... ...
State-Level Initiatives Medicaid Local Coverage Decisions States are working with each other, not-for-profit organizations, federal partners, and others to expand the use and availability of evidence in clinical and administrative decision making. Many states are in the process of designing and purchasing new Medicaid information systems.
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From page 263... ...
Increasingly, their deliberations are shaped by the inclusion of research evidence. A growing understanding of the nature of good-quality clinical evidence and its strengths and limitations seems to be evident.
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From page 264... ...
The document calls for physicians to have the ability to critically appraise research and to appropriately incorporate research findings into self-assessment and practice improvement in each of these domains. This clear articulation of the objective of medical education and training will help prepare physicians to sort through and use the vast amounts of clinical evidence that will emerge from the efforts of other healthcare institutions.
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From page 265... ...
There needs to be, in addition to existing structures like the VA or cancer cooperative groups, additional consortia of investigators who conduct clinical trials who can rapidly be signed up, identify local site managers, and initiate studies. Models for this also exist (e.g., the International Studies of Infarct Survival in Europe and perhaps the Department of Clinical Research Informatics and the Thrombolysis in Myocardial Ischemia study in the United States)
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From page 266... ...
An initial first step might be the creation of a coordinating entity to help identify priorities and design strategies for the establishment of an infrastructure to better support controlled studies and gather data on an ongoing basis. Support the Sentinel Safety System Initiative Cooperative work between FDA and the healthcare community -- payers, healthcare practitioners, provider organizations, medical product manufacturers, academia, patients, the states, and the federal government -- is important to enhance the ability to identify problems with medical products and provide information to the healthcare community as quickly as possible.
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From page 267... ...
2005. Drug safety oversight board meetings public summaries.
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