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Recommendations for Medicare Clinical Trial Reimbursement

Clinical trials have become an essential component of modern medical care because they are the best means of finding out which health care interventions work and which do not. Trials produce information of value to future patients, and frequently benefit the people enrolled in them. Medicare beneficiaries and taxpayers have a shared interest in the development of reliable information about health care interventions, because such information provides the basis for rational health care and resource allocation decisions by providers and patients.

Although neither regulations nor specific policy directives have been issued about the reimbursement of routine patient care costs in clinical trials, the impression is widespread that the Medicare statute excludes from reimbursement all or some such costs (see chapter 2). This impression is apparent from the nature of legislation introduced in Congress to assure reimbursement for routine patient care costs, from statements of HCFA officials, and from remarks of providers. In chapter 2, the committee also established that, in practice, Medicare reimburses many claims for routine patient care. Thus, a wide gap separates many people's impressions of HCFA's rules and the demonstrable facts of actual reimbursement.

The committee is recommending explicit policy to legitimize reimbursement for much—though not all—of the care rendered to participants in clinical trials. Reimbursement should not be denied solely because the care is delivered as part of a clinical trial. In addition, the recommendations apply to all clinical trials involving any type of intervention in any aspect of health care and for any illness, for care that would be eligible for Medicare reimbursement outside of trials. They apply whether government, industry, or other private sources support or sponsor the trial.



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Extending Medicare Reimbursement in Clinical Trials 3 Recommendations for Medicare Clinical Trial Reimbursement Clinical trials have become an essential component of modern medical care because they are the best means of finding out which health care interventions work and which do not. Trials produce information of value to future patients, and frequently benefit the people enrolled in them. Medicare beneficiaries and taxpayers have a shared interest in the development of reliable information about health care interventions, because such information provides the basis for rational health care and resource allocation decisions by providers and patients. Although neither regulations nor specific policy directives have been issued about the reimbursement of routine patient care costs in clinical trials, the impression is widespread that the Medicare statute excludes from reimbursement all or some such costs (see chapter 2). This impression is apparent from the nature of legislation introduced in Congress to assure reimbursement for routine patient care costs, from statements of HCFA officials, and from remarks of providers. In chapter 2, the committee also established that, in practice, Medicare reimburses many claims for routine patient care. Thus, a wide gap separates many people's impressions of HCFA's rules and the demonstrable facts of actual reimbursement. The committee is recommending explicit policy to legitimize reimbursement for much—though not all—of the care rendered to participants in clinical trials. Reimbursement should not be denied solely because the care is delivered as part of a clinical trial. In addition, the recommendations apply to all clinical trials involving any type of intervention in any aspect of health care and for any illness, for care that would be eligible for Medicare reimbursement outside of trials. They apply whether government, industry, or other private sources support or sponsor the trial.

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Extending Medicare Reimbursement in Clinical Trials Medicare reimbursement should not hinge on a judgment by HCFA about the quality of the trial. However, HCFA does have a legitimate interest in assuring that trials meet currently accepted standards for scientific merit and protection of research participants. The committee recommends limiting reimbursement to trials that have been reviewed and accepted by all relevant institutional review boards (IRBs). The committee does not believe that HCFA should evaluate every trial for which a reimbursement claim is submitted, but it should have access to documentation of a trial's scientific merit and the fact of IRB approval, upon request. The committee believes the need for such review would be quite rare. A new reimbursement policy following these recommendations would satisfy two conditions the committee believes to be key: (1) beneficiaries would not be denied reimbursement merely because they have volunteered to participate in a clinical trial; (2) the new rules would not impose excessive administrative burdens on HCFA, its fiscal intermediaries and carriers, or investigators, providers, or participants in clinical trials. Explicit rules would have the added benefit of increasing the uniformity of reimbursement decisions made by Medicare fiscal intermediaries and carriers in different parts of the country. Greater uniformity would, in turn, decrease the uncertainty about reimbursement when providers and patients embark on a clinical trial. The fundamental principle is that reimbursement decisions should be made independent of whether a beneficiary is receiving care in or out of a clinical trial. Reimbursement should be provided for all services to which the beneficiary would be generally entitled under Medicare. This rule would provide reimbursement for a large share of services to participants in clinical trials. The fundamental rule would apply to routine care in all trials comparing standard approved interventions (drugs, devices, procedures, or other). It also would apply to routine care in trials of new versus standard drugs. No new reimbursement policy is needed for routine care in trials of most investigational medical devices. Under the 1995 agreement between FDA and HCFA, reimbursement for participants in trials of devices is allowed when the device is "investigational," but not when it is "experimental" (i.e., when the device itself is not a new type but is a modified version of an existing approved device or is being used for a new indication). For such devices the underlying questions of safety and effectiveness have already been answered. Such devices are classified in "category B.'' Devices for which initial questions of safety and effectiveness have not been answered are designated "category A." The committee believes that reimbursement should be provided for procedures under rules analogous to those applied to medical devices; procedures that are modifications of, or new uses for existing procedures should be reimbursed at the same level as the existing, accepted procedure. Conversely, as with category A devices, Medicare should not pay routinely for procedures that are considered "experimental," although HCFA may (and should) choose to do so in specific trials. The committee recognizes that classifying procedures may be challenging. A "gray zone" in which a reimbursement decision is unclear will

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Extending Medicare Reimbursement in Clinical Trials require some special determinations by HCFA. Nonetheless, this provision is an essential component of the overall recommendation. In addition to providing reimbursement through the proposed policies, the committee urges HCFA to use its existing authority to support selected trials and in two ways to assist in the development of new trials. First, HCFA should reimburse for care in selected trials that would not otherwise qualify, or reimburse at a higher rate than would otherwise be allowed for investigational interventions that are more expensive than the standard treatment. Second, HCFA should assist in the development of new trials of particular importance to the Medicare population. In all cases, HCFA should provide clear guidance on how the recommended reimbursement policies should be implemented by providers and the entities that process Medicare claims. Such guidance would promote nationally uniform administration and minimize uncertainty for providers and patients about what will and will not be reimbursed. Finally, although not a requirement of implementing a new policy, the committee recommends prompt completion of a national clinical trials registry. Such a registry will increase HCFA's ability to monitor reimbursement for services in clinical trials, in addition to serving its other functions. RECOMMENDATIONS RECOMMENDATION 1. Medicare should reimburse routine care for patients in clinical trials in the same way it reimburses routine care for patients not in clinical trials. This principle applies to payments for physicians and other providers, routine laboratory and other diagnostic tests, and any other services that comprise routine care for a given patient. All coverage and medical necessity rules and all other restrictions that apply to patients not in clinical trials would apply to care in clinical trials. The committee recommends a broad definition of clinical trials—including all phases and legitimate designs and all sources of sponsorship (government, industry, or other)—all of which should be equally eligible for reimbursement. This definition does not mean, however, that any treatment simply called a "clinical trial" would qualify for reimbursement. To qualify, a clinical trial must have a written protocol that describes a scientifically sound study and have been approved by all relevant IRBs before participants are enrolled. HCFA should articulate criteria for an acceptable trial and IRB review, which investigators would apply to determine whether their studies are eligible for reimbursement. (HCFA could state the criteria in terms of "current NIH standards," e.g., rather than stating specific study characteristics.) The committee recognizes that controversies surround both the quality of current clinical trials and IRBs, but holds that these issues are being addressed in various ways by DHHS and other sectors

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Extending Medicare Reimbursement in Clinical Trials of government, and should not be addressed routinely in HCFA's reimbursement decisions. Medicare should reimburse routine patient care costs, but not all costs in clinical trials. Medicare should not reimburse the costs of experimental interventions (except category B devices for which reimbursement is allowed under agreement with FDA, and certain procedures as described in recommendation 2), of data collection and record keeping that would not be required but for the trial, or of other services to clinical trial participants necessary solely to satisfy data collection needs of the clinical trial ("protocol-induced costs"). These costs should remain the responsibility of research sponsors, private and public. Medicare should continue its current practice of reimbursing costs of treating conditions that result as unintended consequences (complications) of clinical trials. RECOMMENDATION 2. HCFA should reimburse surgeons (or other practitioners) for treating patients in randomized clinical trials involving procedures that are variations or modifications of accepted procedures, or new uses for accepted procedures. Under the current interpretation of Medicare reimbursement rules, the committee believes that surgeons and others performing surgical or other procedures in trials might not be eligible to be reimbursed for those services. Therefore, the committee recommends that procedures that have become widely accepted as a part of standard medical practice, but which, as part of a clinical trial, are being rigorously evaluated, or are being modified or applied for new indications to determine the incremental risks and benefits, should be eligible for reimbursement at the rate for the standard procedure. Conversely, types of procedures for which initial questions of safety and efficacy have not been resolved would not be eligible for reimbursement. Unlike the basic recommendation regarding routine patient care costs, which applies to all clinical trials, this recommendation would limit reimbursement to randomized trials (the equivalent of "phase 3" trials for drugs and devices). The committee believes this limitation is appropriate in order to avoid providing reimbursement for uncontrolled experimentation by practitioners. The introduction of new drugs and devices is governed by FDA under a formal system that involves phased trials (see chapter 1). In contrast, the introduction of new procedures is not governed by any regulatory authority. In their early phases, procedures are modified or tried for different indications in clinical practice, but rarely in formal trials. However, once a new or modified procedure has been defined and developed to the point that it is distinct enough from the predicate procedure, it may be tested against the standard treatment (the predicate procedure or other accepted treatment) in a formal randomized trial. Medicare should provide reimbursement to the surgeon or other practitioner for treating patients in such trials.

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Extending Medicare Reimbursement in Clinical Trials Further clarification may be needed to make clear the committee's intent with regard to reimbursement for procedures on patients in clinical trials. The committee is expressing no judgments about when trials of procedures should or should not be carried out, or who should be involved in them if they are. This recommendation is not intended to influence the criteria or processes HCFA uses to decide on coverage of new procedures under usual medical care. It applies only when a trial of a procedure is being done—for the all the reasons that trials are done—and claims for reimbursement for the procedure are submitted by practitioners. HCFA's initial task in implementing this recommendation will be to develop definitions for classifying procedures analogous to "category A" and "category B" devices. These definitions describing what is and is not allowed will be applied in the field when claims are submitted. HCFA should not be required to rule routinely on the eligibility of procedures before bills may be submitted. In the same way that providers are responsible for following reimbursement rules for all services under Medicare, they will be responsible for applying the rules appropriately in the case of procedures in clinical trials. Fiscal intermediaries and carriers audit these interpretations by providers in clinical trials, as they now audit bills from providers for care outside of clinical trials. Advice or an interpretation could, of course, be requested of HCFA at any time. In addition, HCFA would retain the right to initiate its own review, without being asked, if it believes there is an issue to be explored, to carry out a random check, or for another reason. The committee recognizes that creating definitions that neatly separate "category A" and "category B" procedures will not be simple, and disagreements are inescapable about where the line between "A" and ''B" should be drawn in specific cases. Table 3-1 gives a few examples of how the definitions might work. Wherever the separation lies, some procedures will fall into a "gray zone." HCFA can narrow the gray zone by applying the definitions to a wide range of real and hypothetical procedures, and stating whether the procedures would or would not be eligible for reimbursement. To deal with cases in which uncertainty remains, HCFA should set up a process to rule quickly on reimbursement eligibility. With accumulated experience, the number of gray zone cases should decline, as has been the case with FDA classification of devices into categories A and B.

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Extending Medicare Reimbursement in Clinical Trials TABLE 3-1. Reimbursement Eligibility for Procedures Under Recommendation 2 Standard Procedure and Indication Innovation and/or Indication Circumstances of Trial, Nature of Innovation Reimbursement Eligibility: Yes/No/HCFA Review Open cholecystectomy Laparoscopic cholecystectomy If neither this nor other "keyhole surgeries" were being reimbursed by HCFA, this would be considered a major departure from standard practice No Open cholecystectomy Laparoscopic cholecystectomy If other "keyhole surgeries" were already being reimbursed by HCFA, this would fall in the "gray zone" HCFA review? Mastectomy for early breast cancer Lumpectomy for early breast cancer Lumpectomy is a variation on mastectomy Yes Standard surgery for parathyroid disease: removal of some parathyroid glands with confirmation by pathology during surgery Surgery for parathyroid disease with physiologic calcium measurement to determine surgical endpoint The procedure is the same; only monitoring is different Yes Optic nerve decompression surgery (ONDS) for pseudotumor cerebrii ONDS for nonarteritic ischemic optic neuropathy New indication for standard procedure, but had been used and reimbursed for new indication outside of trial Yes (this would qualify under recommendation I, because Medicare had been paying for patients outside of trials)

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Extending Medicare Reimbursement in Clinical Trials Open CABG Midline CABG (minimally invasive procedure) Could be considered next-generation open CABG Yes or gray zone Human liver transplant Pig liver transplant Used only in experimental situations; significantly different from standard intervention No Heart transplant or aggressive medical treatment for congestive heart failure Battista procedure: removal of damaged cardiac tissue Completely new procedure. No Pharmacologic or no treatment Fetal cell transplant for Parkinson's disease Completely new procedure No NOTE: Procedures in the table are for illustration only and are treated as though they were new in 1999 and relevant to the Medicare population, even though that is not the case for all examples. In each instance, the judgment about reimbursement eligibility is made assuming that the trial was taking place before HCFA had begun "legitimate reimbursement" for the procedure for the specified indication. By "legitimate reimbursement," we mean that reimbursement was made for claims that disclosed the nature of the procedure and the indication for which it was performed. In this table, "HCFA review" means that the procedure falls in the "gray zone" (or for some other reason, the principal investigator is uncertain about reimbursement eligibility), and a determination of eligibility could be made by HCFA. If a trial is initiated after reimbursement has become standard practice, the "fundamental'' rule—that procedures that are covered in the course of usual medical practice would also be covered in clinical trials—would apply, and reimbursement would be allowed. For any procedure not eligible for reimbursement under the stated rule, investigators could apply to HCFA for reimbursement as an exception.

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Extending Medicare Reimbursement in Clinical Trials The committee has not attempted to specify an institutional mechanism under which HCFA might carry out the tasks required by this recommendation. However, the committee notes that the new Medicare Coverage Advisory Committee* might provide the needed expertise for the task of defining categories A and B and ruling quickly on "gray zone" cases that arise. RECOMMENDATION 3. For claims submitted in accordance with both the fundamental recommendation (No. 1) and the special recommendation for procedures (No. 2), no special precertification by HCFA, or any other administrative process, should be required of clinical trial researchers or providers participating in trials before they submit claims for reimburseable services. Claims should be submitted in the same way they are for treatment outside of trials. Practitioners and institutions would be expected to submit reimbursement claims for services to patients in clinical trials under rules outlined in recommendations 1 and 2. With a clear statement of reimbursement policy, such claims should pose difficulties no different from those arising in the administration of coverage and reimbursement rules for claims for care outside of trials. Investigators and providers would not be routinely required to submit documentation about the trial to HCFA, but HCFA could, at any time, request such documentation to confirm that the clinical trial meets current standards for scientific merit and has the relevant IRB approval. RECOMMENDATION 4. If Medicare or trial sponsors fail to cover clinical care costs, patients should not be billed for those costs above what they would pay if they were not in a trial. This recommendation is not one that can be enforced as part of a reimbursement policy by HCFA; however, the committee believes it is an important principle that could be adopted by clinical trial sponsors and investigators. It also could be incorporated in any legislation passed to implement the committee's recommendations. RECOMMENDATION 5. Medicare members of managed care plans should have the same reimbursement eligibility for care in clinical trials as those enrolled in fee-for-service Medicare, but not beyond the limits of the managed care contract. *    The Medicare Coverage Advisory Committee (MCAC) was established by HCFA to provide guidance on coverage issues. The 120-member committee will function through specialty panels of not more than 15 members each. The MCAC had its first meeting in September 1999.

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Extending Medicare Reimbursement in Clinical Trials Nearly one Medicare beneficiary in five belongs to a capitated plan—an HMO or some other form of managed care. That number is likely to increase over time. It is vital, therefore, that the committee's recommendations carry over to patients served outside traditional Medicare. Managed care plans must provide all benefits offered under traditional Medicare. (Most offer additional benefits, including coverage of outpatient drugs.) Accordingly, the committee recommends that managed care plans be required to offer Medicare beneficiaries access to clinical trials involving services available within their networks. If, for example, a plan routinely covers a particular drug, it should cover it in a trial, as well. If the plan limits the choice of drugs to those listed on a formulary, the plan should not be required to cover a nonformulary drug in a trial. Under point-of-service plans, patients should have the right to go outside the managed care network to participate in a trial, under the terms stipulated in the plan for point-of-service care, but no such right should be inferred under plans that limit enrollees to the plan's providers. This recommendation is not comprehensive, but is suggestive of a policy for managed care. Full implementation will require additional thought when HCFA adopts a clinical trial reimbursement policy, but the committee urges that the new policy not create obstacles to clinical trial enrollment for beneficiaries in managed care. RECOMMENDATION 6. In addition to providing routine coverage through the proposed policy, the committee urges HCFA to use its existing authority to support selected trials and to assist in the development of new trials. In selected clinical trials, the committee believes that HCFA should do more than pay for routine patient care according to the recommendations already stated. Medicare should (1) provide additional reimbursement in a limited number of trials and (2) identify emerging or current methods of care of particular importance to the Medicare population and work with other organizations to initiate trials. Researchers should be able to apply to HCFA for reimbursement above routine rates in cases meriting special treatment. Such trials could include some interventions that do not qualify under the basic recommendations, such as "category A" procedures, primary and secondary screening, diagnostics, and interventions not usually covered by Medicare (e.g., behavioral interventions). The rationale for extending coverage is straightforward. HCFA has a large stake in determining whether more effective or less costly alternatives to current interventions may exist, preventing ineffective procedures from becoming common practice, and facilitating the identification of innovations that would benefit the Medicare population. For example, a behavioral intervention, which normally would not be covered, might replace a more expensive drug or surgical intervention to the benefit of both patients' health and Medicare finances. HCFA should have sole authority to decide whether to extend coverage and should make such determinations

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Extending Medicare Reimbursement in Clinical Trials expeditiously. The committee assumes that only a few trials would be appropriate for such exceptional treatment each year. In the case of interventions of particular importance to the Medicare population, HCFA should collaborate with the National Institutes of Health (NIH) or others to see that appropriate trials are fielded. HCFA should cover routine patient care costs for these trials along the lines of the committee's basic recommendation. It could also fund other costs as well, under the exceptions procedure described in the preceding paragraph. But the objective is to encourage trials, not necessarily to pay for them. Such an active role would not be new for HCFA. This recommendation follows the model of the ongoing study of lung volume reduction surgery, which grew out of collaboration among HCFA, NIH, and the Agency for Health Care Policy and Research (AHCPR), at HCFA's initiative. RECOMMENDATION 7. Every trial for which some Medicare reimbursement is sought should be entered into a national registry of clinical trials. Reimbursement claims should bear an identification number assigned by the registry. A registry will facilitate ex post audits of reimbursement claims, HCFA's main tool for monitoring clinical trial coverage and detecting potential abuse. But identification of a claim as part of a clinical trial should not be relevant to the reimbursement decision. The committee recognizes that implementation of this recommendation will necessarily take some time. Therefore, the committee's recommendations regarding reimbursement of routine patient care costs do not hinge on the existence of a clinical trials registry. Until a registry is in operation, reimbursement claims for interventions associated with a clinical trial should be denoted on the form, in a manner HCFA specifies. However, a registry would contribute to uniform administration and permit HCFA and others to carry out analyses of clinical trials and the costs of implementing the recommendations put forward here. It should, therefore, be put in place as quickly as possible. Ideally, such a registry should include all publicly and privately sponsored trials before they begin accruing patients, thereby providing a link to all claims for Medicare patients in clinical trials. If the goal of creating such a registry is accepted, the practical question of how best to achieve it must be addressed. It would be possible to build upon the registry currently under development at NIH by broadening the definition of trials to be included and consulting widely on how to present data. Or a separate registry could be created. Whether the registry should operate within NIH or elsewhere merits consideration. The design of the NIH registry has been underway for some time. Its designers claim that it will be functioning, at least in part, by early 2000. Redirecting any ongoing effort will be difficult for reasons that are well understood. If it were concluded that converting this limited registry into an inclusive national registry would be needlessly cumbersome, the creation of a separate comprehensive registry, serving objectives beyond those of NIH, or even of HCFA,

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Extending Medicare Reimbursement in Clinical Trials should be explored. The committee urges the Secretary of HHS to examine this issue promptly, set a timetable for completion of a registry, and seek adequate funding for it. ADMINISTRATIVE AND COST IMPLICATIONS Implementation of the committee's recommendations would likely cause some increase in administrative costs to HCFA. In making its recommendations, the committee strove to minimize potential administrative burdens. It is the committee's judgment that any added administrative costs required by institution of this reimbursement policy will be small. Effects of the committee's recommendations on benefit costs are more important and far more uncertain. For several reasons, the cost impact of these recommendations is likely to be quite small. First, the recommended reimbursement policy is designed to limit payments for an individual to roughly the cost of "standard care" for which he or she would be eligible if not enrolled in the trial. This limitation does not imply that each individual would have chosen standard care that cost the same as the care in the clinical trial, so in individual cases, the cost of actual care in the trial might be higher or lower than forgone care outside the trial. Although the incremental cost of routine care in clinical trials is not known with certainty, it is almost surely small in comparison to the costs otherwise incurred by Medicare. Some clinical trial groups claim that the costs of treating patients in some trials may be less than treating them outside of trials. Second, clinical trials hold the long-term prospect of identifying ineffective interventions, which would fall out of favor in the clinical community, or could be excluded from coverage, in some cases saving Medicare dollars. Finally, only a tiny fraction of Medicare patients participate in clinical trials. No accurate count of clinical trial participants at any point in time exists, but the Lewin Group has estimated that about 265,000 people in the United States participate in clinical trials each year, including about 161,000 Medicare beneficiaries—less than 0.5 percent of the 38 million Medicare enrollees in 1997 (Dobson and Sturm, 1999). Clearly, the proportion of Medicare beneficiaries in clinical trials is quite small. The available evidence suggests that Medicare already pays for a large proportion of routine patient care in such trials, including both costs for which no reimbursement is sought, and claims that are submitted and rejected. The largest effect on Medicare costs could come from the speedier determination of the efficacy of innovative or experimental procedures, drugs, and devices. Some will be cost increasing. Others will be cost reducing. Whether the net effect is to raise or lower total Medicare spending, the speedy determination of what works and what does not work will benefit the Medicare population and the nation as a whole.

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Extending Medicare Reimbursement in Clinical Trials CONCLUSION Clinical trials are integral to modern medical care and to the progress of medical science. Although HCFA has issued little explicit policy about payment for routine patient care for patients in clinical trials, the Medicare statute has been widely interpreted to exclude reimbursement for such care. However, evidence is ample to suggest that providers submit claims for routine care for Medicare beneficiaries in trials without noting the existence of the clinical trial, and HCFA's financial contractors usually pay them. The thrust of the committee's recommendations is that nothing should be done that would materially curtail Medicare's reimbursement for routine patient care costs for patients in clinical trials. On the contrary, HCFA should encourage such trials and even extend reimbursement in a limited number of specifically approved exceptional cases. To achieve these goals, the committee believes that HCFA should assure patients in clinical trials the same reimbursement of routine patient care that is available to patients who are not in trials. Extending reimbursement to certain procedures that represent modifications of current practice and distinguishing those from procedures for which risks and benefits are largely unknown will require some additional effort by HCFA, but it is an essential component of the committee's recommendations. The fundamental recommendation—reimbursement independent of trial participation—should be implemented relatively easily.