Inborn errors of metabolism: medical and administrative "orphans". (9/1514)

CONTEXT: Inborn errors of metabolism are genetic conditions that affect the normal biochemical functions of the body in any organ and at any age. More than 500 metabolic diseases are known; almost all are classified as orphan diseases under the US Food and Drug Administration guidelines (incidence < 200,000 persons) and each has its own requirements for diagnosis and treatment. Management of these complex, lifelong, multisystem disorders often requires a coordinated, multidisciplinary approach involving several subspecialists and which may include complex laboratory evaluations, genetic counseling, nutritional therapy, and unusual therapeutic approaches that have been used in only a small number of cases. RESULTS: Not infrequently, inborn errors of metabolism fall outside current standard diagnostic and treatment guidelines of managed care plans. This results in delays in diagnosis and appropriate management, with increased costs to patients and to society. CONCLUSIONS: Patients with inborn errors of metabolism should not be discriminated against and all health plans should specify that access to specialists and metabolic centers are a covered benefit of the plan. The acceptance of treatment guidelines, the development of international disease classification codes for the disorders, and the performance of cost-benefit analyses would all greatly facilitate this process. However, without recognition that these disorders require such services, and steps to provide them by the insurance industry, the care of children with metabolic disorders and other chronic diseases will continue to be a source of frustration and anger among the caregivers and the families they serve.  (+info)

Generic drug product equivalence: current status. (10/1514)

This activity is designed for healthcare professionals involved in the selection of multisource drug products. GOAL: To understand the basis for approval of generic drug products by the Food and Drug Administration. OBJECTIVES: 1. Identify the criteria employed by the Food and Drug Administration to approve generic drug products. 2. Discuss controversial issues that have been raised relative to generic drug products. 3. Identify narrow therapeutic index drugs. 4. Describe the different types of bioequivalence studies that are required by the Food and Drug Administration. 5. Discuss the responsibilities underlying the selection of multisource drug products by healthcare professionals.  (+info)

Technology assessment, coverage decisions, and conflict: the role of guidelines. (11/1514)

As pressure grows for health plans to be accountable for increasing quality of care within a cost-control environment, coverage of new technologies becomes a particularly challenging issue. For a number of reasons, health plans have adopted evidence-based methods for guiding technology decisions. The implementation of these methods has not been free of controversy, and conflicts have arisen between plans and proponents of technologies who often use the political and legal arena in an attempt to secure coverage. Unless these conflicts are resolved, the healthcare system may have difficulty meeting cost and quality objectives. Technology assessment and coverage process guidelines and flexible coverage approaches may be possible ways of resolving these conflicts.  (+info)

Technology assessment of medical devices at the Center for Devices and Radiological Health. (12/1514)

We reviewed the Food and Drug Administration's regulatory process for medical devices and described the issues that arise in assessing device safety and effectiveness during the postmarket period. The Center for Devices and Radiological Health (CDRH), an organization within the Food and Drug Administration, has the legal authority and responsibility for ensuring that medical devices marketed in the United States are both reasonably safe and effective for their intended use. This is an enormous challenge given the diversity of medical devices and the large number of different types of devices on the market. Many scientific and regulatory activities are necessary to ensure device safety and effectiveness, including technology assessment, albeit in a manner quite different from that of conventional technology assessment. The basic approach taken at the CDRH to ensure device safety and effectiveness is to develop an understanding of the way in which a medical device works and how it will perform in clinical situations.  (+info)

Role of technology assessment in health benefits coverage for medical devices. (13/1514)

With the profusion of new medical technology, managed care organizations are faced with the challenge of determining which medical devices and services warrant health benefits coverage. To aid in this decision-making process, managed care companies turn to technology assessment, a process that differs from the Food and Drug Administration's review of medical devices. Health plans typically use a structured approach to implementing coverage requirements in employer group benefits contracts and use technology assessment to evaluate the scientific evidence of effectiveness to support coverage decisions. Also important is the societal context for decisions regarding coverage for new technologies and the options being considered by policy makers for accountability in technology assessment by private insurers and health plans.  (+info)

Transmyocardial laser revascularization: a qualitative systematic review. (14/1514)

OBJECTIVE: To evaluate the status of transmyocardial laser revascularization (TMLR) from an evidence-based perspective to help hospitals make resource management decisions. STUDY DESIGN: Qualitative systematic review of the clinical literature. METHODS: We searched the reference databases MEDLINE, BIOSIS, EMBASE, SciSearch, and Current Contents to identify all articles related to TMLR published between January 1985 and March 1997. We collected, analyzed, and summarized clinical studies in evidence tables. RESULTS: The cumulative evidence available in the medical literature regarding the safety and effectiveness of TMLR encompasses approximately 2000 patients treated worldwide, primarily those with medically refractory angina. Preliminary data suggest that TMLR has an acceptable survival rate and effectively relieves angina in approximately 75% of patients. Data showing improved myocardial perfusion, cardiac function, or prognosis are inconclusive. The mechanism by which TMLR relieves angina is not yet known. CONCLUSIONS: Early evidence regarding TMLR suggests it will be useful for treating patients with end-stage coronary artery disease. Definitive recommendations await critical analysis of the results of ongoing randomized clinical trials, post-market surveillance studies, and third-party payer acceptance.  (+info)

The limited state of technology assessment for medical devices: facing the issues. (15/1514)

Medical devices are an integral part of clinical practice and account for a substantial proportion of the national health budget. Clinical testing and regulation of medical devices, however, is vastly different from and inferior to the testing and regulation of drugs. As managed care organizations begin to exert controls on device use, providers are being caught between the policies of their organizations and the demands of device manufacturers and patients, who want wider access to devices. We outline several reasons for the poor state of medical device evaluations and the dangers of using devices without adequate information, and include the recently developed device assessment and reporting guidelines created by the Task Force on Technology Assessment of Medical Devices.  (+info)

Medical records and privacy: empirical effects of legislation. (16/1514)

OBJECTIVE: To determine the effects of state legislation requiring patient informed consent prior to medical record abstraction by external researchers for a specific study. DATA SOURCES/STUDY SETTING: Informed consent responses obtained from November 1997 through April 1998 from members of a Minnesota-based IPA model health plan. STUDY DESIGN: Descriptive case study of consent to gain access to medical records for a pharmaco-epidemiologic study of seizures associated with use of a pain medication that was conducted as part of the FDA's post-marketing safety surveillance program to evaluate adverse events associated with approved drugs. DATA COLLECTION: The informed consent process approved by an institutional review board consisted of three phases: (1) a letter from the health plan's medical director requesting participation, (2) a second mailing to nonrespondents, and (3) a follow-up telephone call to nonrespondents. PRINCIPAL FINDINGS: Of 140 Minnesota health plan members asked to participate in the medical records study, 52 percent (73) responded and 19 percent (26) returned a signed consent form authorizing access to their records for the study. For 132 study subjects enrolled in five other health plans in states where study-specific consent was not required, health care providers granted access to patient medical records for 93 percent (123) of the members. CONCLUSION: Legislation requiring patient informed consent to gain access to medical records for a specific research study was associated with low participation and increased time to complete that observational study. Efforts to protect patient privacy may come into conflict with the ability to produce timely and valid research to safeguard and improve public health.  (+info)