U.S. Food and Drug Administration approval of AmBisome (liposomal amphotericin B) for treatment of visceral leishmaniasis.
In August 1997, AmBisome (liposomal amphotericin B, Nexstar, San Dimas, CA) was the first drug approved for the treatment of visceral leishmaniasis by the U.S. Food and Drug Administration. The growing recognition of emerging and reemerging infections warrants that safe and effective agents to treat such infections be readily available in the United States. The following discussion of the data submitted in support of the New Drug Application for AmBisome for the treatment of visceral leishmaniasis shows the breadth of data from clinical trials that can be appropriate to support approval for drugs to treat tropical diseases. (+info)
Advances in the biological therapy and gene therapy of malignant disease.
Biological and gene therapy of cancer have become important components of clinical cancer research. Advances in this area are based on evidence for the presence of tumor antigens, antitumor immune responses, evasion of host control by tumors, and the recognition of host defense failure in cancer patients. These mechanisms are being corrected or exploited in the development of biological and gene therapy. Over the last decade, 9 biological therapies have received Food and Drug Administration approval, and another 12 appear promising and will likely be approved in the next few years. Our approach to gene therapy has been to allogenize tumors by the direct intratumoral injection of HLA-B7/beta2-microglobulin genes as plasmid DNA in a cationic lipid into patients with malignant melanoma. In four Phase I studies, we found a 36% response by the local injected tumor and a 19% systemic antitumor response. In other cancers, gene transfer, expression, and an intratumoral T-cell response were seen, but no clinical response was seen. A variety of follow-up studies with HLA-B7 and other genes are planned. Evasion of host control is now a major target of gene therapy. Strategies to overcome this include up-regulation of MHC and introduction of cell adhesion molecules into tumor cells, suppression of transforming growth factor and interleukin 10 production by tumor cells, and blockade of the fas ligand-fas interaction between tumor cells and attacking lymphocytes. With these approaches, it seems likely that gene therapy may become the fifth major modality of cancer treatment in the next decade. (+info)
The US Food and Drug Administration investigational device exemptions (IDE) and clinical investigation of cardiovascular devices: information for the investigator.
The conduct of a clinical investigation of a medical device to determine the safety and effectiveness of the device is covered by the investigational device exemptions (IDE) regulation. The purpose of IDE regulation is "to encourage, to the extent consistent with the protection of public health and safety and with ethical standards, the discovery and development of useful devices intended for human use, and to that end to maintain optimum freedom for scientific investigators in their pursuit of this purpose" (Federal Food, Drug, and Cosmetic Act). Conducting a clinical investigation may require an approved IDE application. The US Food and Drug Administration encourages early interaction with the agency through the pre-IDE process during the development of a device or technology and during the preparation of an IDE application. This facilitates approval of the IDE application and progression into the clinical investigation. This paper reviews the terminology and applicability of the IDE regulation and the type of study that requires an IDE application to the Food and Drug Administration. The pre-IDE process and the development of an IDE application for a significant risk study of a cardiovascular device are discussed. (+info)
Availability of immune globulin intravenous for treatment of immune deficient patients--United States, 1997-1998.
Immune globulin intravenous (IGIV) is a lifesaving treatment for patients with primary immunodeficiency. Since November 1997, a shortage of IGIV has existed in the United States. In 1998, the Food and Drug Administration (FDA) required pharmaceutical companies to increase the frequency of reporting on IGIV distribution from biannually to monthly; in addition, FDA facilitated IGIV distribution and informed clinicians about the ongoing shortage. To assess the impact of the IGIV shortage on patient care, in 1998 the Immune Deficiency Foundation (IDF) surveyed physicians caring for immunodeficient patients about whether they have had difficulty obtaining IGIV, measures they have taken because of the shortage, and the effect of the shortage on their patients. This report summarizes data reported to FDA and data obtained from the IDF survey and provides recommendations for IGIV use during the shortage. (+info)
Procedure for expediting determinations of antibiotic susceptibility of gram-negative, urinary tract pathogens.
Standardized direct disk diffusion antibiotic susceptibility testing on monomicrobial urine specimens is compared with the Food and Drug Administration method. The direct procedure yields acceptable data and may conserve 24 h in reporting results. (+info)
Herbal remedies: adverse effects and drug interactions.
A growing number of Americans are using herbal products for preventive and therapeutic purposes. The manufacturers of these products are not required to submit proof of safety and efficacy to the U.S. Food and Drug Administration before marketing. For this reason, the adverse effects and drug interactions associated with herbal remedies are largely unknown. Ginkgo biloba extract, advertised as improving cognitive functioning, has been reported to cause spontaneous bleeding, and it may interact with anticoagulants and antiplatelet agents. St. John's wort, promoted as a treatment for depression, may have monoamine oxidase-inhibiting effects or may cause increased levels of serotonin, dopamine and norepinephrine. Although St. John's wort probably does not interact with foods that contain tyramine, it should not be used with prescription antidepressants. Ephedrine-containing herbal products have been associated with adverse cardiovascular events, seizures and even death. Ginseng, widely used for its purported physical and mental effects, is generally well tolerated, but it has been implicated as a cause of decreased response to warfarin. Physicians must be alert for adverse effects and drug interactions associated with herbal remedies, and they should ask all patients about the use of these products. (+info)
Modernizing the FDA: an incremental revolution.
The U.S. Food and Drug Administration (FDA) is responsible for protecting consumers from unsafe or ineffective drugs and medical devices. The agency's role is defined by a growing and increasingly complex set of statutes, which reflect Congress's desires, on the one hand, to prevent product hazards and, on the other, to expedite FDA review and approval of promising new medical technologies. Congress's latest attempt to calibrate regulation to achieve these goals, the 1997 Food and Drug Administration Modernization Act, endorses certain of the FDA's own innovations and changes in the agency's ways of doing business. (+info)
When is a cost-effectiveness claim valid? How much should the FDA care?
Federal law requires the Food and Drug Administration (FDA) to regulate the promotional claims of prescription drugs and certain devices. Standards of evidence for claims of safety and therapeutic efficacy are rigorous because inappropriate product use may place human life at risk. However, equally demanding criteria for claims of cost-effectiveness of marketed technologies seem to be unnecessary because the consequence of error is principally a bad buy rather than patient harm. Concern exists about the validity of cost-effectiveness studies, the potential for bias, standards for the conduct of cost-effectiveness research, and the needs of managed care. The FDA should moderate its role in regulating cost-effectiveness claims of drugs and devices. This would foster information flow to healthcare providers and insurers and protect the FDA concern regarding false or misleading claims of effectiveness. Although the issues are applicable to both devices and drugs, we draw mainly from the field of pharmacoeconomics because this is where most of the policy has developed. (+info)