Six years' experience with interdisciplinary review teams in health technology assessment in Norway.

OBJECTIVES: The aim of this study was to evaluate the use of interdisciplinary review teams that have been the main and central work form in making health technology assessments at the Norwegian Centre for Health Technology Assessment. METHODS: Evaluation questionnaires were sent to all 112 participants in the 17 review teams for the period of January 1998 to June 2003 after completion of the literature assessment. Questions were on the theme/mandate of the assessments, composition of the review team, organization of the work, the working method, and update of the report. RESULTS: The teams ranged from 4 to 14 persons regarded as opinion leaders in their field. The project periods lasted from 4 to 33 months. In all, fifty-five participants gave fifty-eight responses (51.8 percent) to the questionnaires. A total of 83 percent thought the theme was well argued, and 62 percent thought the mandate for the assessments was sufficiently clear. Approximately 80 percent were positive to the composition of the review team. In all, 22 percent expressed that the work method was too extensive and 43 percent wanted more tuition. General comments were that the working method gave competence in assessing medical literature, relevant professional training, and tuition in a working method that ensured the legitimacy of their work. CONCLUSIONS: The review team participants were satisfied with most aspects of the work. The Norwegian Centre for Health Technology Assessment will continue using interdisciplinary review teams in making health technology assessments.

[Clinical use of haematopoietic stem cells from cord blood]. / Klinisk bruk av hematopoetiske stamceller fra navlestrengsblod.

A group of experts appointed by the Norwegian Centre for Health Technology Assessment (SMM) has undertaken a systematic review of available literature on the clinical effectiveness of transplanting haematopoietic stem cells from cord blood. A total of 17 studies form the documentary basis of the review. Autologous transplants of stem cells from cord blood have not been published. Retrospective studies suggest that the clinical effect of allogeneic cord blood transplants, at least in children, is comparable to transplants with allogeneic stem cells from bone marrow or peripheral blood cells. This review demonstrates the need for prospective studies comparing transplantations of cord blood with bone marrow or peripheral blood stem cells.

Status and potential of gene therapy in clinical medicine. Assessment of an emerging health technology through systematic survey of clinical gene therapy protocols and published results.

OBJECTIVE: Somatic gene therapy is a new method in the rapidly expanding field of molecular medicine. Due to recent encouraging results and the promising prospect for some disease groups, Norwegian health authorities wanted to assess somatic therapy with evidence-based standards for strategic use. This article presents the results of this assessment, discussed in the context of the policy-making process in Norway, including ethical and legislational considerations. METHODS: Clinical gene therapy protocols, ongoing or completed with published results, where available, were identified through a systematic survey of descriptive protocols and publications. Preclinical literature was also reviewed. RESULTS: Gene therapy is dominated by preclinical and clinical research. Most of the gene therapy protocols identified are in early phases (phases I and II) with only a few patients in each study. Of the protocols included in the assessment, only three phase III studies are represented. Except for the use of soluble antisense oligonucleotides against cytomegalovirus eye infection, gene therapy is presently not an established treatment modality. Promising results have been observed in treatment of cancer and cardiovascular diseases and, most recently, in inherited severe combined immunodeficiency and hemophilia. Several interesting principles addressing a large panel of conditions are currently being developed and tested. CONCLUSIONS: Gene therapy is developing into an important medical concept that needs to be included within the Norwegian healthcare system. It is recommended that the Norwegian Ministry of Health and Social Affairs fund a national program to boost infrastructure in selected scientific groups both in preclinical and clinical research. The national procedures regulating approval of gene therapy trials should be made more efficient while at the same time allowing for proper control and ethical considerations. It is emphasized that gene therapy trials should be carefully monitored for side effects.