Mostrando las entradas con la etiqueta Institute of Medicine. Mostrar todas las entradas
Mostrando las entradas con la etiqueta Institute of Medicine. Mostrar todas las entradas

29 mayo, 2013

Tobacco Company Efforts to Influence the Food and Drug Administration-Commissioned Institute of Medicine

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  • Crystal E. Tan,
  • Thomas Kyriss,
  • Stanton A. Glantz mail

Background

Spurred by the creation of potential modified risk tobacco products, the US Food and Drug Administration (FDA) commissioned the Institute of Medicine (IOM) to assess the science base for tobacco “harm reduction,” leading to the 2001 IOM report Clearing the Smoke. The objective of this study was to determine how the tobacco industry organized to try to influence the IOM committee that prepared the report.

Methods and Findings

We analyzed previously secret tobacco industry documents in the University of California, San Francisco Legacy Tobacco Documents Library, and IOM public access files. (A limitation of this method includes the fact that the tobacco companies have withheld some possibly relevant documents.) Tobacco companies considered the IOM report to have high-stakes regulatory implications. They developed and implemented strategies with consulting and legal firms to access the IOM proceedings. When the IOM study staff invited the companies to provide information on exposure and disease markers, clinical trial design for safety and efficacy, and implications for initiation and cessation, tobacco company lawyers, consultants, and in-house regulatory staff shaped presentations from company scientists. Although the available evidence does not permit drawing cause-and-effect conclusions, and the IOM may have come to the same conclusions without the influence of the tobacco industry, the companies were pleased with the final report, particularly the recommendations for a tiered claims system (with separate tiers for exposure and risk, which they believed would ease the process of qualifying for a claim) and license to sell products comparable to existing conventional cigarettes (“substantial equivalence”) without prior regulatory approval. Some principles from the IOM report, including elements of the substantial equivalence recommendation, appear in the 2009 Family Smoking Prevention and Tobacco Control Act.

Conclusions

Tobacco companies strategically interacted with the IOM to win several favored scientific and regulatory recommendations.

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19 octubre, 2011

Medical Device Innovation — Is “Better” Good Enough?

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Source: NEJM
Last year, the United States spent $95 billion on medical devices, nearly half of the $200 billion spent on devices worldwide.1 Our investment in devices has yielded impressive gains in length and quality of life from products such as implantable cardioverter–defibrillators, pacemakers, and artificial joints (cardiovascular and orthopedic devices account for more than 35% of the market1). Roughly 10 million Americans have symptomatic knee osteoarthritis,2 a leading cause of disability and the most common indication for total knee arthroplasty. More than 600,000 total knee arthroplasty procedures are performed annually in the United States; 85% of recipients report functional improvement, and the annual failure rate is 0.5 to 1.6%.3 Inspired by these successes, medical device innovation continues. Each year for the past decade, the Food and Drug Administration (FDA) has approved more than 35 new systems or components for total knee arthroplasty. Most are designed to improve durability, and their manufacturers cite laboratory studies showing reductions in wear. Advertising campaigns promote innovative implants for younger, more physically active patients, expanding the market for knee arthroplasty.
But oversight of device innovation is currently under scrutiny. Safety concerns have been raised over total joint components and other devices approved through the FDA's 510(k) clearance process, whereby devices perceived as posing a low risk of complications are approved for marketing without clinical trials. These concerns led the Institute of Medicine to recommend eliminating the 510(k) process, calling it ineffective and unsalvageable.4 The current oversight system has been simultaneously faulted for inadequate assurance of safety and efficacy and for suppressing innovation. Since regulatory approval hinges on claims of similarity to previously approved devices, the process may encourage the development of devices that provide only small improvements at higher cost than their predecessors. The trade-offs between incremental improvement and the additional costs and technical complexity of the required procedure are poorly understood and seldom investigated rigorously.
When adequately powered randomized trials are not feasible, a model-based approach can offer insight into the interplay among device efficacy and durability, patient characteristics, costs, and long-term outcomes. We used a validated “state-transition” computer-simulation model of the natural history and management of knee osteoarthritis5 to forecast clinical outcomes associated with hypothetical “innovative” total knee implants as compared with existing implants. We considered cohorts of persons with end-stage, symptomatic knee osteoarthritis, stratified by age and presence of coexisting conditions at the time of arthroplasty. We used a range of values for the potential reduction in the likelihood of long-term implant failure with hypothetical innovative implants and estimated the proportion of each patient cohort that would remain alive with their original (standard or innovative) implant intact 20 years after surgery. We examined the effects of increasing the risk of short-term failure while simultaneously decreasing the rate of long-term failure, as might be expected from a device offering improved survival at the expense of greater technical complexity. (Details are presented in the Supplementary Appendix, available with the full text of this article at NEJM.org.)
According to our model, by 20 years after a standard total knee arthroplasty, 19% of people who were healthy and 50 to 59 years of age at the time of the surgery and 86% of those who were 70 to 79 years of age and had coexisting conditions would have died; 65% and 11% of these groups, respectively, would be alive with their original implant intact. In part because of the much higher risk of death among older patients, the cumulative risk of requiring revision surgery within 20 years after a primary total knee arthroplasty would be twice as high among younger, healthier patients than among older patients with coexisting conditions (18% vs. 9%; see graph
Cumulative Risk of Revision Surgery 20 Years after Total Knee Arthroplasty with a Standard Implant and with an Innovative Implant, According to Computer-Simulation Modeling.
). Innovative implants with long-term failure rates 70% lower than those of current implants (an improvement similar to those that some manufacturers have demonstrated in the laboratory) would reduce the cumulative risk of revision by 11% among healthy 50-to-59-year-olds and 6% among 70-to-79-year-olds with coexisting conditions. If short-term failure rates quintupled (as recent data on innovative orthopedic devices suggest they could), the reductions in cumulative risk of revision would be lessened by 35% among healthy 50-to-59-year-olds and 59% among 70-to-79-year-olds with coexisting conditions, potentially offsetting the benefits of decreases in long-term failure.
Our findings suggest that there can be no one-size-fits-all approach to the use of innovative devices. In the case of total knee arthroplasty, a patient's life expectancy has a marked effect on his or her anticipated benefit from improvements in durability over existing implants, whose survival rates are already excellent. Given the low annual failure rate of existing implants, even significant reductions in long-term failure rates would have little effect on overall implant survival in older, sicker patients. This finding is even more significant when innovative implants have greater short-term failure rates (possibly attributable to the learning curve associated with new technology). There are also additional trade-offs that should be considered in evaluating and pricing innovative devices. For example, innovations are typically accompanied by cost increases, and devices providing small, incremental clinical benefits may be less likely to offer good value for any additional investment.
We believe that our approach and the insights it can offer extend well beyond knee implants. Total knee implants are similar to many medical devices — such as hip and spinal implants, other orthopedic hardware, and ophthalmologic implants — in that they improve quality of life rather than survival. Thus, our work has implications for the development and adoption of any medical device offering improved long-term clinical benefit at increased initial cost. These analyses demonstrate that even small decreases in long-term device failure can provide clinical value, but these innovations are unlikely to provide equal benefit to all patients. Innovative technologies may also increase the risk of short-term complications, owing to increased complexity of the procedure or the greater technical skill required to optimally implement such advances — a phenomenon that is rarely captured in laboratory-based testing. Furthermore, these technologies typically cost more than their predecessors. These considerations may further restrict the populations in which an innovative device offers good value.
Our goal is not to set limits on who receives which implants, but to illustrate a model-based approach to improving new-device evaluation. Decisions about the marketing, use, and pricing of medical devices are often made in the absence of robust outcomes data. As the current controversy over the 510(k) process attests, traditional approaches to clinical investigation and evaluation are poorly suited to exploring and balancing the competing considerations at play — for instance, estimating likely improvements in long-term efficacy and device durability, factoring in the competing risks when devices are used in older or higher-risk patients, and determining our willingness to pay for incremental improvements. A model-based assessment can help to define the circumstances under which the diffusion of medical device innovations to ever-expanding patient populations is clinically and economically justified.
Model-based evaluations could help define the thresholds for complication and efficacy rates and costs that would be required to improve on existing device performance while maintaining acceptable economic value. This information could then inform postmarketing surveillance efforts, triggering reviews at prespecified efficacy or complication thresholds and facilitating rapid application of new data as they become available. Manufacturers could use such data to improve device development; researchers could identify target populations for evaluating novel technologies; insurers could identify opportunities for value-based reimbursement; and consumers could be educated about what clinical benefits they are getting for their money. The complex trade-offs between short- and long-term health and economic consequences of technological innovation may not be captured by even the most sophisticated randomized trials. Model-based approaches may provide invaluable insights for evaluating medical device innovation and merit consideration as a standard component of the evaluation process.
Disclosure forms provided by the authors are available with the full text of this article at NEJM.org.
SOURCE INFORMATION
From Yale School of Medicine (L.G.S.) and Yale School of Public Health (A.D.P.) — both in New Haven, CT; the Veterans Affairs Connecticut Healthcare System, West Haven, CT (L.G.S.); and Brigham and Women's Hospital (B.N.R., D.H.S., I.G., H.G., J.N.K., E.L.), Harvard Medical School (D.H.S., E.L.), Harvard School of Public Health (J.N.K.), and Boston University School of Public Health (E.L.) — all in Boston.

11 octubre, 2011

Defining Essential Health Benefits — The View from the IOM Committee


Source: NEJM
When Congress enacted the Affordable Care Act (ACA), it mandated that a broad package of “essential health benefits” (EHBs) equivalent to that of a “typical employer plan” be offered by qualified health plans participating in newly created state-based insurance exchanges, as well as by new plans offered to individuals and small employers outside these exchanges. Congress directed the Department of Health and Human Services (DHHS) to flesh out the details. The DHHS, in turn, asked the Institute of Medicine (IOM) to recommend a process for defining and updating the EHB package — but notably, not to develop a specific list of benefits.
In a report released on October 7, the IOM recommended that the initial EHB package be equivalent in scope to what could be purchased by the average premium that a small business would pay on behalf of an employee (see Key Recommendations of the IOM Committee on Defining and Revising an Essential Health Benefits Package for Qualified Health Plans).1 If the DHHS endorsed a more expansive package, the IOM's Committee on Defining and Revising an Essential Health Benefits Package for Qualified Health Plans cautioned in its report, many currently uninsured individuals and small businesses would find it unaffordable, which would undermine the overriding goal of the reform law — to make coverage both meaningful and nearly universal. To underscore its strong emphasis on affordability without elimination of appropriate coverage, the report said EHBs “should become more fully evidence-based, specific, and value-promoting over time,” and if additional services are added, “the package should be offset by savings” from the elimination of outmoded or unnecessary services and the making of prioritized choices among services supported by public dollars.
Over time, under the ACA, an estimated 30 million uninsured individuals and employees of small businesses (fewer than 100 workers) with low-to-moderate incomes will become eligible for federally subsidized coverage through insurance exchanges or expansions of Medicaid programs. Most employees will remain insured through grandfathered or self-funded employer plans offered by large companies and some small ones, and these plans are exempt from the EHB mandate.
The ACA stipulates that health plans must offer, to individuals and small businesses who seek coverage through an insurance exchange or outside of one, packages that “at least” include 10 broad benefit categories: ambulatory patient services; emergency services; hospitalization; maternity and newborn care; mental health and substance abuse disorder services, including behavioral health treatment; prescription drugs; rehabilitative and habilitative services and devices; laboratory services; preventive and wellness services and chronic disease management; and pediatric services, including oral and vision care. The ACA allows states to require health plans to offer benefits beyond the defined set of EHBs, but states must assume the incremental costs for subsidies of any additional mandated benefits.
To standardize benefits and establish a basis for calculating premium subsidies, the law creates four plan levels that are differentiated by their actuarial value — the percentage of covered expenses that a plan is expected to pay out, on average, as distinguished from what is paid out-of-pocket by the consumer. These levels are bronze (paying on average 60% of covered expenses), silver (70%), gold (80%), and platinum (90%). Individuals with incomes up to 400% of the federal poverty level (currently $88,200 for a family of four) will be eligible for tax credits scaled by income to help defray the cost of coverage. Individuals with incomes at or below 133% of the federal poverty level are eligible to enroll in Medicaid, which has little or no patient cost sharing.
The IOM report identifies key challenges that the DHHS will face as it strives to equate the ACA-defined EHBs with the typical small-business package. For one, the conductors of an ACA-mandated Labor Department survey of documents from 3200 employer-sponsored insurance plans found it difficult to describe with much precision the benefits of a “typical” employer package.2 In addition, some benefits mandated by the ACA — such as habilitative services, wellness programs, and pediatric oral and vision care — are generally not included in standard small-employer or even large-group insurance contracts. The report notes that for employers, establishing a budget creates one way to explicitly consider benefit-package tradeoffs when resources are limited.
The committee decided to peg its budgeting target to the estimated average premium for a “silver” package — the second-lowest-priced plan available through an exchange and the level to which the ACA's premium subsidies are linked. The report suggests that the DHHS's selection of benefits be guided by an estimate such as that prepared by the Congressional Budget Office (CBO)3 and converted to 2014 dollars by the IOM committee — $6,933 for an individual policy (see tableEstimated Health Insurance Premiums for Individual and Family Policies without the Affordable Care Act (ACA) and after Its Implementation.) — or a RAND estimate of $5,474 for a silver plan when individual and small-group risk pools are combined.4 To build on those estimates, the IOM recommended that the DHHS seek actuarial advice on the costs of all elements of the EHB plan, including benefit-design features such as the degree of medical management, provider payment rates, and patient cost sharing. Actual premiums for plans would vary, as they do now, with the geographic area, population enrolled, and other factors.
As part of its assigned task, the IOM committee was asked to evaluate definitions of “medical necessity review” that are found in private insurance contracts. The committee concluded that such processes and other medical management techniques (e.g., prior authorization) “with appropriate checks and balances, are necessary to ensure that the package of EHB benefits can be delivered at the most affordable cost.” The ACA requires the establishment of an independent external review of appealed medical necessity cases. The committee decided not to recommend a single national definition of medical necessity but added: “The criteria used for medically necessary services or services that conform to medical necessity are medical services that are (1) clinically appropriate for the individual patient, (2) based on the best scientific evidence, taking into account the available hierarchy of medical evidence, and (3) likely to produce incremental health benefits relative to the next best alternative that justify any added cost.” These criteria are consistent with best practices and supported by legal precedent.
The IOM report acknowledges that “the determination of the EHB is a politically and socially charged endeavor.” Recognizing the controversy the EHBs could provoke, the committee recommends that the DHHS create a “structured interactive process” to advise the department on reconciling “the tensions between comprehensiveness and affordability.” From its beginning, the panel agreed unanimously that if the long-standing problem of rising health care costs is not addressed more aggressively, the ACA's goals of reducing the uninsured population (49.9 million people, or 16.3% of the population, in 2010, according to the U.S. Census Bureau) and offering meaningful benefits could be undermined.
The report said the cost trend will not be moderated only by the definition of EHBs and added: “The committee considered whether complementary Medicare-only or federal-only approaches to reducing rising health costs would be sufficient and concluded they would not be. An all stakeholder strategy is required across the public and private sectors,” and the ACA-created independent payment advisory board, even if it survives strong opposition to its creation, will not include formal engagement of the private sector. By expressing its concern over health care costs whose annual increases outstrip the growth of the economy, the committee has issued a wake-up call for policymakers, clarifying how this spending pattern squeezes out other important competing needs, particularly in a no-growth economy. However, such previous warnings, as often as they have been issued, have largely fallen on deaf ears.

KEY RECOMMENDATIONS OF THE IOM COMMITTEE ON DEFINING AND REVISING AN ESSENTIAL HEALTH BENEFITS PACKAGE FOR QUALIFIED HEALTH PLANS.*

1. The secretary of health and human services should establish an essential health benefits (EHB) package including the 10 categories contained in the Affordable Care Act and as guided by a national average premium target. Once developed, the package should be adjusted so that the expected national average premium for a “silver” (second-lowest-price) plan is actuarially equivalent to the average premium small employers would have paid in 2014 for a typical plan. A public deliberative process should be used to make adjustments to the initial EHB package.
2. By January 2013, the secretary should establish a framework for monitoring EHB implementation and updating that accounts for changes in provider payment rates, financial incentives, practice organizations, and other relevant matters. The secretary should implement this framework and coordinate federal efforts to produce and make the data accessible for public use.
3. Beginning in 2015, the secretary should update the EHB package to make it more fully evidence-based, specific, and value-promoting — explicitly incorporating costs. A public deliberative process should be used to inform choices about what to include in or exclude from the updated package.
4. The secretary should permit states administering their own exchanges to adopt variants of the federal EHB package, provided that modifications are consistent with the federal package, not significantly more or less generous, and are subject to public input.
5. The secretary should establish a National Benefits Advisory Council, with members appointed through a nonpartisan process, which should make recommendations annually stemming from its oversight of the EHB package.
6. To ensure that the EHB-defined packages remain affordable and sustainable, the secretary should develop a strategy, in collaboration with others, for aligning the growth rate of health care spending in all sectors with that of the economy.
* Summarized from the Institute of Medicine Committee report.1
Disclosure forms provided by the author are available with the full text of this article at NEJM.org.
This article (10.1056/NEJMp1109982) was published on October 7, 2011, at NEJM.org.

SOURCE INFORMATION

Mr. Iglehart is a national correspondent for the Journal.